PMID- 25898053 OWN - NLM STAT- MEDLINE DA - 20150422 DCOM- 20150504 LR - 20150507 IS - 1538-3598 (Electronic) IS - 0098-7484 (Linking) VI - 313 IP - 15 DP - 2015 Apr 21 TI - Outcomes following gene therapy in patients with severe Wiskott-Aldrich syndrome. PG - 1550-63 LID - 10.1001/jama.2015.3253 [doi] AB - IMPORTANCE: Wiskott-Aldrich syndrome is a rare primary immunodeficiency associated with severe microthrombocytopenia. Partially HLA antigen-matched allogeneic hematopoietic stem cell (HSC) transplantation is often curative but is associated with significant comorbidity. OBJECTIVE: To assess the outcomes and safety of autologous HSC gene therapy in Wiskott-Aldrich syndrome. DESIGN, SETTING, AND PARTICIPANTS: Gene-corrected autologous HSCs were infused in 7 consecutive patients with severe Wiskott-Aldrich syndrome lacking HLA antigen-matched related or unrelated HSC donors (age range, 0.8-15.5 years; mean, 7 years) following myeloablative conditioning. Patients were enrolled in France and England and treated between December 2010 and January 2014. Follow-up of patients in this intermediate analysis ranged from 9 to 42 months. INTERVENTION: A single infusion of gene-modified CD34+ cells with an advanced lentiviral vector. MAIN OUTCOMES AND MEASURES: Primary outcomes were improvement at 24 months in eczema, frequency and severity of infections, bleeding tendency, and autoimmunity and reduction in disease-related days of hospitalization. Secondary outcomes were improvement in immunological and hematological characteristics and evidence of safety through vector integration analysis. RESULTS: Six of the 7 patients were alive at the time of last follow-up (mean and median follow-up, 28 months and 27 months, respectively) and showed sustained clinical benefit. One patient died 7 months after treatment of preexisting drug-resistant herpes virus infection. Eczema and susceptibility to infections resolved in all 6 patients. Autoimmunity improved in 5 of 5 patients. No severe bleeding episodes were recorded after treatment, and at last follow-up, all 6 surviving patients were free of blood product support and thrombopoietic agonists. Hospitalization days were reduced from a median of 25 days during the 2 years before treatment to a median of 0 days during the 2 years after treatment. All 6 surviving patients exhibited high-level, stable engraftment of functionally corrected lymphoid cells. The degree of myeloid cell engraftment and of platelet reconstitution correlated with the dose of gene-corrected cells administered. No evidence of vector-related toxicity was observed clinically or by molecular analysis. CONCLUSIONS AND RELEVANCE: This study demonstrated the feasibility of the use of gene therapy in patients with Wiskott-Aldrich syndrome. Controlled trials with larger numbers of patients are necessary to assess long-term outcomes and safety. FAU - Hacein-Bey Abina, Salima AU - Hacein-Bey Abina S AD - Biotherapy Department, Necker Children's Hospital, Assistance Publique-Hopitaux de Paris, Paris, France2Biotherapy Clinical Investigation Center, Groupe Hospitalier Universitaire Ouest, Assistance Publique-Hopitaux de Paris, INSERM, Paris, France3Unite de. FAU - Gaspar, H Bobby AU - Gaspar HB AD - Section of Molecular and Cellular Immunology, University College London Institute of Child Health, London, England6Department of Clinical Immunology, Hospital National Health Service Foundation Trust, London, England. FAU - Blondeau, Johanna AU - Blondeau J AD - Biotherapy Department, Necker Children's Hospital, Assistance Publique-Hopitaux de Paris, Paris, France2Biotherapy Clinical Investigation Center, Groupe Hospitalier Universitaire Ouest, Assistance Publique-Hopitaux de Paris, INSERM, Paris, France. FAU - Caccavelli, Laure AU - Caccavelli L AD - Biotherapy Department, Necker Children's Hospital, Assistance Publique-Hopitaux de Paris, Paris, France2Biotherapy Clinical Investigation Center, Groupe Hospitalier Universitaire Ouest, Assistance Publique-Hopitaux de Paris, INSERM, Paris, France. FAU - Charrier, Sabine AU - Charrier S AD - INSERM U951, Unite Mixte de Recherche S951, Molecular Immunology and Innovative Biotherapies, University of Evry, Evry, France8Genethon, Evry, France. FAU - Buckland, Karen AU - Buckland K AD - Section of Molecular and Cellular Immunology, University College London Institute of Child Health, London, England6Department of Clinical Immunology, Hospital National Health Service Foundation Trust, London, England. FAU - Picard, Capucine AU - Picard C AD - Centre d'Etude des Deficits Immunitaires, Hopital Necker-Enfants Malades, Assistance Publique-Hopitaux de Paris, Paris, France10Paris Descartes-Sorbonne Paris Cite University, Imagine Institute, Paris, France11Immunology and Pediatric Hematology Departmen. FAU - Six, Emmanuelle AU - Six E AD - Paris Descartes-Sorbonne Paris Cite University, Imagine Institute, Paris, France12INSERM Unite Mixte de Recherche 1163, Laboratory of Human Lymphohematopoiesis, Paris, France. FAU - Himoudi, Nourredine AU - Himoudi N AD - Section of Molecular and Cellular Immunology, University College London Institute of Child Health, London, England6Department of Clinical Immunology, Hospital National Health Service Foundation Trust, London, England. FAU - Gilmour, Kimberly AU - Gilmour K AD - Section of Molecular and Cellular Immunology, University College London Institute of Child Health, London, England6Department of Clinical Immunology, Hospital National Health Service Foundation Trust, London, England. FAU - McNicol, Anne-Marie AU - McNicol AM AD - Section of Molecular and Cellular Immunology, University College London Institute of Child Health, London, England6Department of Clinical Immunology, Hospital National Health Service Foundation Trust, London, England. FAU - Hara, Havinder AU - Hara H AD - Section of Molecular and Cellular Immunology, University College London Institute of Child Health, London, England6Department of Clinical Immunology, Hospital National Health Service Foundation Trust, London, England. FAU - Xu-Bayford, Jinhua AU - Xu-Bayford J AD - Department of Clinical Immunology, Hospital National Health Service Foundation Trust, London, England. FAU - Rivat, Christine AU - Rivat C AD - Section of Molecular and Cellular Immunology, University College London Institute of Child Health, London, England6Department of Clinical Immunology, Hospital National Health Service Foundation Trust, London, England. FAU - Touzot, Fabien AU - Touzot F AD - Biotherapy Department, Necker Children's Hospital, Assistance Publique-Hopitaux de Paris, Paris, France2Biotherapy Clinical Investigation Center, Groupe Hospitalier Universitaire Ouest, Assistance Publique-Hopitaux de Paris, INSERM, Paris, France10Paris D. FAU - Mavilio, Fulvio AU - Mavilio F AD - Genethon, Evry, France. FAU - Lim, Annick AU - Lim A AD - Groupe Immunoscope, Immunology Department, Institut Pasteur, Paris, France. FAU - Treluyer, Jean-Marc AU - Treluyer JM AD - Clinical Research Center Necker-Enfants Malades and Cochin Hospital Assistance Publique-Hopitaux de Paris, Paris Descartes University. FAU - Heritier, Sebastien AU - Heritier S AD - Paris Descartes-Sorbonne Paris Cite University, Imagine Institute, Paris, France11Immunology and Pediatric Hematology Department, Assistance Publique-Hopitaux de Paris, Paris, France. FAU - Lefrere, Francois AU - Lefrere F AD - Biotherapy Department, Necker Children's Hospital, Assistance Publique-Hopitaux de Paris, Paris, France. FAU - Magalon, Jeremy AU - Magalon J AD - Biotherapy Department, Necker Children's Hospital, Assistance Publique-Hopitaux de Paris, Paris, France2Biotherapy Clinical Investigation Center, Groupe Hospitalier Universitaire Ouest, Assistance Publique-Hopitaux de Paris, INSERM, Paris, France. FAU - Pengue-Koyi, Isabelle AU - Pengue-Koyi I AD - Biotherapy Department, Necker Children's Hospital, Assistance Publique-Hopitaux de Paris, Paris, France2Biotherapy Clinical Investigation Center, Groupe Hospitalier Universitaire Ouest, Assistance Publique-Hopitaux de Paris, INSERM, Paris, France10Paris D. FAU - Honnet, Geraldine AU - Honnet G AD - Genethon, Evry, France. FAU - Blanche, Stephane AU - Blanche S AD - Paris Descartes-Sorbonne Paris Cite University, Imagine Institute, Paris, France11Immunology and Pediatric Hematology Department, Assistance Publique-Hopitaux de Paris, Paris, France. FAU - Sherman, Eric A AU - Sherman EA AD - Department of Microbiology, University of Pennsylvania School of Medicine, Philadelphia. FAU - Male, Frances AU - Male F AD - Department of Microbiology, University of Pennsylvania School of Medicine, Philadelphia. FAU - Berry, Charles AU - Berry C AD - Department of Microbiology, University of Pennsylvania School of Medicine, Philadelphia. FAU - Malani, Nirav AU - Malani N AD - Department of Microbiology, University of Pennsylvania School of Medicine, Philadelphia. FAU - Bushman, Frederic D AU - Bushman FD AD - Department of Microbiology, University of Pennsylvania School of Medicine, Philadelphia. FAU - Fischer, Alain AU - Fischer A AD - Paris Descartes-Sorbonne Paris Cite University, Imagine Institute, Paris, France11Immunology and Pediatric Hematology Department, Assistance Publique-Hopitaux de Paris, Paris, France12INSERM Unite Mixte de Recherche 1163, Laboratory of Human Lymphohematop. FAU - Thrasher, Adrian J AU - Thrasher AJ AD - Section of Molecular and Cellular Immunology, University College London Institute of Child Health, London, England6Department of Clinical Immunology, Hospital National Health Service Foundation Trust, London, England. FAU - Galy, Anne AU - Galy A AD - INSERM U951, Unite Mixte de Recherche S951, Molecular Immunology and Innovative Biotherapies, University of Evry, Evry, France8Genethon, Evry, France. FAU - Cavazzana, Marina AU - Cavazzana M AD - Biotherapy Department, Necker Children's Hospital, Assistance Publique-Hopitaux de Paris, Paris, France2Biotherapy Clinical Investigation Center, Groupe Hospitalier Universitaire Ouest, Assistance Publique-Hopitaux de Paris, INSERM, Paris, France10Paris D. LA - eng GR - R01 AI082020/AI/NIAID NIH HHS/United States PT - Clinical Trial PT - Journal Article PT - Multicenter Study PT - Research Support, Non-U.S. Gov't PL - United States TA - JAMA JT - JAMA JID - 7501160 RN - 0 (Wiskott-Aldrich Syndrome Protein Family) SB - AIM SB - IM CIN - JAMA. 2015 Apr 21;313(15):1522-3. PMID: 25898049 MH - Adolescent MH - Child MH - Child, Preschool MH - Feasibility Studies MH - Gene Expression MH - *Genetic Therapy/adverse effects MH - *Genetic Vectors MH - *Hematopoietic Stem Cell Transplantation/adverse effects MH - Humans MH - Infant MH - Infant, Newborn MH - *Lentivirus MH - Male MH - Severity of Illness Index MH - Wiskott-Aldrich Syndrome/genetics/immunology/*therapy MH - Wiskott-Aldrich Syndrome Protein Family/*genetics EDAT- 2015/04/22 06:00 MHDA- 2015/05/06 06:00 CRDT- 2015/04/22 06:00 AID - 2275447 [pii] AID - 10.1001/jama.2015.3253 [doi] PST - ppublish SO - JAMA. 2015 Apr 21;313(15):1550-63. doi: 10.1001/jama.2015.3253. PMID- 25826002 OWN - NLM STAT- MEDLINE DA - 20150401 DCOM- 20150430 IS - 1077-5552 (Print) IS - 1077-5552 (Linking) VI - 21 DP - 2015 TI - Exercise-induced increases in cell free DNA in human plasma originate predominantly from cells of the haematopoietic lineage. PG - 164-73 AB - The role of cell free DNA (cfDNA) has been intensively discussed under various pathological conditions and after acute bouts of exercise. To date, there is still no conclusive evidence concerning the cellular origin of cfDNA and the entire mechanism leading to elevated cfDNA concentrations in human plasma and serum. Here, we investigated the cellular origin of cfDNA in sex-mismatched haematopoietic stem cell transplantation (HSCT) and liver transplantation (LT) patients by determining the relative proportion of Y-chromosomal to total nuclear cfDNA. Total nuclear cfDNA and Y-chromosomal cfDNA concentrations were determined in blood plasma before and after an incremental exercise test via quantitative real-time PCR (qPCR). Female HSCT patients showed high proportions of Y-chromosomal cfDNA. Both total nuclear and Y-chromosomal cfDNA increased significantly and in a highly correlated fashion due to exercise. In male HSCT patients with female donors less than 10% of the cfDNA was of Y-chromosomal origin at any point in time and even though the total amount of cfDNA increased during exercise, no increases in Y-chromosomal DNA could be detected. The percentage of Y-chromosomal cfDNA in female LT patients with male donors was very low and levels remained unchanged during exercise. This indicates that cells not derived from the bone marrow, in this case transplanted liver cells, represented only a minor fraction of cfDNA in blood plasma and were not released during acute physical exercise. Even though many physiological conditions may be altered in transplant patients versus healthy people, our results strongly suggest that cells from the haematopoietic lineage are the main source of cfDNA released during acute bouts of exercise. CI - Copyright (c) 2015 International Society of Exercise and Immunology. All rights reserved. FAU - Tug, Suzan AU - Tug S AD - Department of Sports Medicine, Disease Prevention and Rehabilitation, Johannes Gutenberg-University Mainz, Mainz, Germany. FAU - Helmig, Susanne AU - Helmig S AD - Department of Sports Medicine, Disease Prevention and Rehabilitation, Johannes Gutenberg-University Mainz, Mainz, Germany. FAU - Deichmann, Eva Ricarda AU - Deichmann ER AD - Department of Sports Medicine, Disease Prevention and Rehabilitation, Johannes Gutenberg-University Mainz, Mainz, Germany. FAU - Schmeier-Jurchott, Anna AU - Schmeier-Jurchott A AD - Department of Internal Medicine III, Hematology, Oncology and Pneumology, University Medical Center, Johannes Gutenberg-University, Mainz, Germany. FAU - Wagner, Eva AU - Wagner E AD - Department of Internal Medicine III, Hematology, Oncology and Pneumology, University Medical Center, Johannes Gutenberg-University, Mainz, Germany. FAU - Zimmermann, Tim AU - Zimmermann T AD - Department of Internal Medicine I, Transplant Hepatology, University Medical Center, Johannes Gutenberg-University, Mainz, Germany. FAU - Radsak, Markus AU - Radsak M AD - Department of Internal Medicine III, Hematology, Oncology and Pneumology, University Medical Center, Johannes Gutenberg-University, Mainz, Germany. FAU - Giacca, Mauro AU - Giacca M AD - Molecular Medicine Laboratory, International Centre for Genetic Engineering and Biotechnology, Trieste, Italy and Department of Medical, Surgical, and Health Sciences, University of Trieste, Trieste, Italy. FAU - Simon, Perikles AU - Simon P AD - Department of Sports Medicine, Disease Prevention and Rehabilitation, Johannes Gutenberg-University Mainz, Mainz, Germany. LA - eng PT - Comparative Study PT - Journal Article PT - Research Support, Non-U.S. Gov't PL - Germany TA - Exerc Immunol Rev JT - Exercise immunology review JID - 9505535 RN - 9007-49-2 (DNA) SB - IM MH - Adult MH - Cell Lineage MH - Cell Nucleus/chemistry MH - Chromosomes, Human, Y/chemistry MH - DNA/*blood MH - *Exercise MH - Exercise Test MH - Female MH - *Hematopoietic Stem Cell Transplantation MH - Hematopoietic Stem Cells/*chemistry MH - Hepatocytes/chemistry MH - Humans MH - *Liver Transplantation MH - Male MH - Middle Aged MH - Organ Specificity MH - Pilot Projects MH - Plasma MH - Running/*physiology MH - Tissue Donors MH - Young Adult OTO - NOTNLM OT - Y-chromosomal PCR OT - cell free DNA OT - exercise OT - graft rejection OT - sex-mismatched transplantation EDAT- 2015/04/01 06:00 MHDA- 2015/05/01 06:00 CRDT- 2015/04/01 06:00 PST - ppublish SO - Exerc Immunol Rev. 2015;21:164-73. PMID- 25802913 OWN - NLM STAT- MEDLINE DA - 20150324 DCOM- 20150430 IS - 1167-7422 (Print) IS - 1167-7422 (Linking) VI - 24 IP - 157 DP - 2015 Feb TI - First-line treatment of acute lymphoblastic leukaemia in children. PG - 39 LA - eng PT - Journal Article PL - France TA - Prescrire Int JT - Prescrire international JID - 9439295 SB - T MH - Age Factors MH - Antineoplastic Combined Chemotherapy Protocols/adverse effects/*therapeutic use MH - Chemotherapy, Adjuvant MH - Child MH - *Hematopoietic Stem Cell Transplantation/adverse effects/mortality MH - Humans MH - Neoadjuvant Therapy MH - Philadelphia Chromosome MH - Precursor Cell Lymphoblastic Leukemia-Lymphoma/diagnosis/genetics/mortality/*therapy MH - Time Factors MH - Treatment Outcome EDAT- 2015/03/25 06:00 MHDA- 2015/05/01 06:00 CRDT- 2015/03/25 06:00 PST - ppublish SO - Prescrire Int. 2015 Feb;24(157):39. PMID- 25617154 OWN - NLM STAT- MEDLINE DA - 20150226 DCOM- 20150513 LR - 20150226 IS - 1472-6882 (Electronic) IS - 1472-6882 (Linking) VI - 15 DP - 2015 TI - A pilot study to evaluate the feasibility of individualized yoga for inpatient children receiving intensive chemotherapy. PG - 2 LID - 10.1186/s12906-015-0529-3 [doi] AB - BACKGROUND: Fatigue is an important problem in paediatric cancer patients and yoga may be an effective intervention. The primary objective was to determine the feasibility of individualized yoga for hospitalized children receiving intensive chemotherapy. METHODS: We included English-speaking children and adolescents aged 7-18 years receiving intensive chemotherapy or haematopoietic stem cell transplantation (HSCT). Yoga was conducted three times weekly for three weeks. The primary outcome was feasibility, defined as ability to deliver at least 60% of planned sessions. Secondary outcomes were parent-reported Pediatric Quality of Life Inventory (PedsQL) Multidimensional Fatigue Scale, Fatigue Scale-Parent, PedsQL Generic Core Scales and PedsQL Acute Cancer Module. RESULTS: Between January and October 2013, 11 patients were enrolled. Median age was 14.0 (range 7.7-16.4) years and 6 (55%) were boys. Yoga was feasible with 10/11 participants meeting the threshold for feasibility. The median number of yoga sessions was 9 (range 3-13). No adverse events were attributed to yoga. Mean+/-standard deviation for the day 21 proxy-reported PedsQL general fatigue scores was 55.6+/-15.5. Qualitative comments suggested design changes for future yoga studies. CONCLUSIONS: Individualized yoga is feasible for inpatient children receiving intensive chemotherapy. Future work will include development and conduct of a randomized trial for fatigue amelioration. TRIAL REGISTRATION: ClinicalTrials.gov NCT02105389. FAU - Diorio, Caroline AU - Diorio C FAU - Schechter, Tal AU - Schechter T FAU - Lee, Michelle AU - Lee M FAU - O'Sullivan, Cathy AU - O'Sullivan C FAU - Hesser, Tanya AU - Hesser T FAU - Tomlinson, Deborah AU - Tomlinson D FAU - Piscione, Janine AU - Piscione J FAU - Armstrong, Christine AU - Armstrong C FAU - Tomlinson, George AU - Tomlinson G FAU - Sung, Lillian AU - Sung L LA - eng SI - ClinicalTrials.gov/NCT02105389 PT - Clinical Trial PT - Journal Article DEP - 20150124 PL - England TA - BMC Complement Altern Med JT - BMC complementary and alternative medicine JID - 101088661 RN - 0 (Antineoplastic Agents) SB - IM MH - Adolescent MH - Antineoplastic Agents/*therapeutic use MH - Child MH - Fatigue/complications/etiology MH - Feasibility Studies MH - Female MH - Hematopoietic Stem Cell Transplantation MH - Humans MH - Individualized Medicine/adverse effects/*methods MH - Inpatients MH - Male MH - Neoplasms/complications/*drug therapy/*therapy MH - Parents MH - Pilot Projects MH - Quality of Life MH - *Yoga PMC - PMC4308944 OID - NLM: PMC4308944 EDAT- 2015/01/27 06:00 MHDA- 2015/05/15 06:00 CRDT- 2015/01/25 06:00 PHST- 2014/02/05 [received] PHST- 2015/01/16 [accepted] PHST- 2015/01/24 [aheadofprint] AID - s12906-015-0529-3 [pii] AID - 10.1186/s12906-015-0529-3 [doi] PST - epublish SO - BMC Complement Altern Med. 2015 Jan 24;15:2. doi: 10.1186/s12906-015-0529-3. PMID- 25596117 OWN - NLM STAT- MEDLINE DA - 20150227 DCOM- 20150512 IS - 1473-5644 (Electronic) IS - 0022-2615 (Linking) VI - 64 IP - Pt 3 DP - 2015 Mar TI - Risk factors, outcomes and epidemiology associated with Clostridium difficile infection in patients with haematological malignancies in a tertiary care hospital in China. PG - 209-16 LID - 10.1099/jmm.0.000028 [doi] AB - The purpose of this study was to evaluate the risk factors, outcomes and epidemiology associated with Clostridium difficile infection (CDI) in patients with haematological malignancies in a tertiary care hospital in China. C. difficile screening was performed on patients admitted for chemotherapy or haematopoietic stem cell transplantation between 2009 and 2013. C. difficile isolates were analysed by multilocus sequence typing, and a retrospective chart review was performed on all patients with a positive toxin assay. CDI was diagnosed in 21 haematology-oncology ward patients and 14 marrow transplantation service patients for a cumulative incidence of 1.89/1000 and 3.69/1000 patient-days, respectively. Univariate analyses showed that patients who received etoposide had an increased risk of CDI (odds ratio 4.25, 95 % confidence interval 1.32-13.64). There was only one patient death, for which CDI was not the primary cause. Ten sequence types (STs) were identified, of which ST-3 and ST-54 were the most common; the hypervirulent ST-1 (ribotype 027) and ST-11 (ribotype 078) C. difficile strains were not detected in the patients in this study. The incidence of CDI did not differ between patients receiving chemotherapy and those receiving haematopoietic stem cell transplantation. The only risk factor for chemotherapy patients was treatment with etoposide. CI - (c) 2015 The Authors. FAU - Gu, Si-Lan AU - Gu SL AD - Collaborative Innovation Center for Diagnosis and Treatment of Infectious Diseases, State Key Laboratory for Diagnosis and Treatment of Infectious Diseases, First Affiliated Hospital, School of Medicine, Zhejiang University, Hangzhou, Zhejiang, PR China. FAU - Chen, Yun-Bo AU - Chen YB AD - Collaborative Innovation Center for Diagnosis and Treatment of Infectious Diseases, State Key Laboratory for Diagnosis and Treatment of Infectious Diseases, First Affiliated Hospital, School of Medicine, Zhejiang University, Hangzhou, Zhejiang, PR China. FAU - Lv, Tao AU - Lv T AD - Collaborative Innovation Center for Diagnosis and Treatment of Infectious Diseases, State Key Laboratory for Diagnosis and Treatment of Infectious Diseases, First Affiliated Hospital, School of Medicine, Zhejiang University, Hangzhou, Zhejiang, PR China. FAU - Zhang, Xue-wu AU - Zhang XW AD - Hematology Department, First Affiliated Hospital, School of Medicine, Zhejiang University, Hangzhou, PR China. FAU - Wei, Ze-Qing AU - Wei ZQ AD - Collaborative Innovation Center for Diagnosis and Treatment of Infectious Diseases, State Key Laboratory for Diagnosis and Treatment of Infectious Diseases, First Affiliated Hospital, School of Medicine, Zhejiang University, Hangzhou, Zhejiang, PR China. FAU - Shen, Ping AU - Shen P AD - Collaborative Innovation Center for Diagnosis and Treatment of Infectious Diseases, State Key Laboratory for Diagnosis and Treatment of Infectious Diseases, First Affiliated Hospital, School of Medicine, Zhejiang University, Hangzhou, Zhejiang, PR China. FAU - Li, Lan-Juan AU - Li LJ AD - Collaborative Innovation Center for Diagnosis and Treatment of Infectious Diseases, State Key Laboratory for Diagnosis and Treatment of Infectious Diseases, First Affiliated Hospital, School of Medicine, Zhejiang University, Hangzhou, Zhejiang, PR China ljli@zju.edu.cn. LA - eng PT - Journal Article PT - Research Support, Non-U.S. Gov't DEP - 20150116 PL - England TA - J Med Microbiol JT - Journal of medical microbiology JID - 0224131 RN - 0 (Anti-Infective Agents) RN - 0 (Antineoplastic Agents, Phytogenic) RN - 6PLQ3CP4P3 (Etoposide) SB - IM MH - Adolescent MH - Adult MH - Aged MH - Anti-Infective Agents/therapeutic use MH - Antineoplastic Agents, Phytogenic/*therapeutic use MH - China/epidemiology MH - Clostridium Infections/blood/drug therapy/*epidemiology/microbiology MH - Clostridium difficile/classification/genetics/*isolation & purification MH - Cross Infection MH - Enterocolitis, Pseudomembranous/blood/drug therapy/epidemiology/microbiology MH - Etoposide/*therapeutic use MH - Feces/microbiology MH - Female MH - Hematologic Neoplasms/*complications/drug therapy/therapy MH - Hematopoietic Stem Cell Transplantation/*adverse effects MH - Humans MH - Incidence MH - Male MH - Middle Aged MH - Multilocus Sequence Typing MH - Retrospective Studies MH - Ribotyping MH - Risk Factors MH - Tertiary Care Centers MH - Young Adult EDAT- 2015/01/18 06:00 MHDA- 2015/05/13 06:00 CRDT- 2015/01/18 06:00 PHST- 2015/01/16 [aheadofprint] AID - jmm.0.000028 [pii] AID - 10.1099/jmm.0.000028 [doi] PST - ppublish SO - J Med Microbiol. 2015 Mar;64(Pt 3):209-16. doi: 10.1099/jmm.0.000028. Epub 2015 Jan 16. PMID- 25521756 OWN - NLM STAT- MEDLINE DA - 20150317 DCOM- 20150511 IS - 1365-2141 (Electronic) IS - 0007-1048 (Linking) VI - 169 IP - 1 DP - 2015 Apr TI - Combined cord blood and bone marrow transplantation from the same human leucocyte antigen-identical sibling donor for children with malignant and non-malignant diseases. PG - 103-10 LID - 10.1111/bjh.13267 [doi] AB - Umbilical cord blood (UCB) from an human leucocyte antigen (HLA)-identical sibling can be used for transplantation of patients with malignant and non-malignant diseases. However, the low cellular content of most UCB units represents a limitation to this approach. An option to increase cell dose is to harvest bone marrow (BM) cells from the same donor and infuse them along with the UCB. We studied 156 children who received such a combined graft between 1992 and 2011. Median age was 7 years and 78% of patients (n = 122) were transplanted for non-malignant diseases, mainly haemoglobinopathies. Acute leukaemia (n = 26) was the most frequent malignant diagnosis. Most patients (91%) received myeloablative conditioning. Median donor age was 1.7 years, median infused nucleated cell dose was 24.4 x 10(7) /kg and median follow-up was 41 months. Sixty-days neutrophil recovery occurred in 96% of patients at a median of 17 d. The probabilities of grade-II-IV acute and chronic graft-versus-host disease (GVHD) were 19% and 10%, respectively. Four-year overall survival was 90% (68% malignant; 97% non-malignant diseases) with 3% probability of death. In conclusion, combined UCB and BM transplantation from an HLA-identical sibling donor is an effective treatment for children with malignant and non-malignant disorders with high overall survival and low incidence of GVHD. CI - (c) 2014 John Wiley & Sons Ltd. FAU - Tucunduva, Luciana AU - Tucunduva L AD - Hospital Saint Louis, AP-HP, and IUH University Paris VII, Eurocord, Paris, France; Centro de Oncologia, Hospital Sirio-Libanes, Sao Paulo, Brazil. FAU - Volt, Fernanda AU - Volt F FAU - Cunha, Renato AU - Cunha R FAU - Locatelli, Franco AU - Locatelli F FAU - Zecca, Marco AU - Zecca M FAU - Yesilipek, Akif AU - Yesilipek A FAU - Caniglia, Maurizio AU - Caniglia M FAU - Gungor, Tayfun AU - Gungor T FAU - Aksoylar, Serap AU - Aksoylar S FAU - Fagioli, Franca AU - Fagioli F FAU - Bertrand, Yves AU - Bertrand Y FAU - Addari, Maria Carmen AU - Addari MC FAU - de la Fuente, Josu AU - de la Fuente J FAU - Winiarski, Jacek AU - Winiarski J FAU - Biondi, Andrea AU - Biondi A FAU - Sengeloev, Henrik AU - Sengeloev H FAU - Badell, Isabel AU - Badell I FAU - Mellgren, Karin AU - Mellgren K FAU - de Heredia, Cristina Diaz AU - de Heredia CD FAU - Sedlacek, Petr AU - Sedlacek P FAU - Vora, Ajay AU - Vora A FAU - Rocha, Vanderson AU - Rocha V FAU - Ruggeri, Annalisa AU - Ruggeri A FAU - Gluckman, Eliane AU - Gluckman E CN - Eurocord and the EBMT Paediatric working party LA - eng PT - Clinical Trial PT - Journal Article PT - Multicenter Study DEP - 20141218 PL - England TA - Br J Haematol JT - British journal of haematology JID - 0372544 SB - IM MH - Acute Disease MH - Adolescent MH - Adult MH - Allografts MH - *Bone Marrow Transplantation MH - Child MH - Child, Preschool MH - Chronic Disease MH - *Cord Blood Stem Cell Transplantation MH - Disease-Free Survival MH - Female MH - Follow-Up Studies MH - Graft vs Host Disease/etiology/*mortality/*therapy MH - Humans MH - Infant MH - Leukemia/*mortality/*therapy MH - Living Donors MH - Male MH - Survival Rate MH - *Transplantation Conditioning OTO - NOTNLM OT - bone marrow OT - cord blood OT - haematopoietic stem cell transplantation EDAT- 2014/12/19 06:00 MHDA- 2015/05/12 06:00 CRDT- 2014/12/19 06:00 PHST- 2014/09/02 [received] PHST- 2014/11/17 [accepted] PHST- 2014/12/18 [aheadofprint] AID - 10.1111/bjh.13267 [doi] PST - ppublish SO - Br J Haematol. 2015 Apr;169(1):103-10. doi: 10.1111/bjh.13267. Epub 2014 Dec 18. PMID- 25495919 OWN - NLM STAT- MEDLINE DA - 20150317 DCOM- 20150511 IS - 1365-2141 (Electronic) IS - 0007-1048 (Linking) VI - 169 IP - 1 DP - 2015 Apr TI - Immunotherapy with the trifunctional anti-CD20 x anti-CD3 antibody FBTA05 (Lymphomun) in paediatric high-risk patients with recurrent CD20-positive B cell malignancies. PG - 90-102 LID - 10.1111/bjh.13242 [doi] AB - Children with B cell malignancies refractory to standard therapy are known to have a poor prognosis and very limited treatment options. Here, we report on the treatment and follow-up of ten patients diagnosed with relapsed or refractory mature B-cell Non Hodgkin Lymphoma (B-NHL), Burkitt leukaemia (B-AL) or pre B-acute lymphoblastic leukaemia (pre B-ALL). All children were treated with FBTA05 (now designated Lymphomun), an anti-CD3 x anti-CD20 trifunctional bispecific antibody (trAb) in compassionate use. Within individual treatment schedules, Lymphomun was applied (a) after allogeneic stem cell transplantation (allo-SCT, n = 6) to induce sustained long-term remission, or (b) stand alone prior to subsequent chemotherapy to eradicate residual disease before allo-SCT (n = 4). Nine of ten children displayed a clinical response: three stable diseases (SD), one partial remission (PR) and five induced or sustained complete remissions (CR). Five of these nine responders died during follow-up. The other patients still maintain CR with a current overall survival of 874-1424 days (median: 1150 days). In conclusion, despite the dismal clinical prognosis of children refractory to standard therapy, immunotherapy with Lymphomun resulted in a favourable clinical outcome in this cohort of refractory paediatric patients. CI - (c) 2014 John Wiley & Sons Ltd. FAU - Schuster, Friedhelm R AU - Schuster FR AD - Department of Paediatric Oncology, Haematology and Immunology, University of Duesseldorf, Duesseldorf, Germany. FAU - Stanglmaier, Michael AU - Stanglmaier M FAU - Woessmann, Wilhelm AU - Woessmann W FAU - Winkler, Beate AU - Winkler B FAU - Siepermann, Meinolf AU - Siepermann M FAU - Meisel, Roland AU - Meisel R FAU - Schlegel, Paul G AU - Schlegel PG FAU - Hess, Jurgen AU - Hess J FAU - Lindhofer, Horst AU - Lindhofer H FAU - Borkhardt, Arndt AU - Borkhardt A FAU - Buhmann, Raymund AU - Buhmann R LA - eng PT - Clinical Trial PT - Journal Article PT - Multicenter Study PT - Research Support, Non-U.S. Gov't DEP - 20141211 PL - England TA - Br J Haematol JT - British journal of haematology JID - 0372544 RN - 0 (Antibodies, Bispecific) RN - 0 (Bi20 antibody) SB - IM MH - Adolescent MH - Allografts MH - Antibodies, Bispecific/*administration & dosage/adverse effects MH - Burkitt Lymphoma/mortality/pathology/*therapy MH - Child MH - Child, Preschool MH - Disease-Free Survival MH - Female MH - Follow-Up Studies MH - Humans MH - *Immunotherapy MH - Male MH - Neoplasm, Residual MH - Precursor B-Cell Lymphoblastic Leukemia-Lymphoma/mortality/pathology/*therapy MH - *Stem Cell Transplantation MH - Survival Rate OTO - NOTNLM OT - B-cell malignancies OT - anti-CD20 x anti-CD3 OT - bispecific antibody OT - children OT - immunotherapy EDAT- 2014/12/17 06:00 MHDA- 2015/05/12 06:00 CRDT- 2014/12/16 06:00 PHST- 2014/07/14 [received] PHST- 2014/11/04 [accepted] PHST- 2014/12/11 [aheadofprint] AID - 10.1111/bjh.13242 [doi] PST - ppublish SO - Br J Haematol. 2015 Apr;169(1):90-102. doi: 10.1111/bjh.13242. Epub 2014 Dec 11. PMID- 25474147 OWN - NLM STAT- MEDLINE DA - 20150318 DCOM- 20150518 IS - 1532-3145 (Electronic) IS - 0363-8715 (Linking) VI - 39 IP - 2 DP - 2015 Mar-Apr TI - The role of screening sinus computed tomography in pediatric hematopoietic stem cell transplant patients. PG - 228-31 LID - 10.1097/RCT.0000000000000185 [doi] AB - OBJECTIVE: The objective of this study was to evaluate pretransplant sinus computed tomography (CT) as predictor of post-hematopoietic stem cell transplant sinusitis. METHODS: We evaluated pretransplant and posttransplant CT findings in 100 children using the Lund-Mackay system and "common-practice" radiology reporting and correlated these with the presence of acute sinusitis. RESULTS: Fourteen percent of patients with normal screening CT developed posttransplant sinusitis, compared with 23% with radiographic abnormalities and 22% with clinical sinusitis alone, not statistically significant. Sensitivity of CT findings for clinical sinusitis ranged between 19% and 56%. Except for mucosal thickening (71% specificity), other findings had high specificity between 92% and 97%, particularly when combined. Lund-Mackay score change of 10 or greater from baseline was associated with a 2.8-fold increased likelihood of having sinusitis (P < 0.001). CONCLUSIONS: Screening CT can serve as a baseline, with a Lund-Mackay score change of 10 or greater constituting a significant threshold. The strongest correlation with the presence of acute sinusitis was seen with combined CT findings. FAU - Zamora, Carlos A AU - Zamora CA AD - From the *Division of Neuroradiology, The Russell H. Morgan Department of Radiology and Radiological Science, Johns Hopkins University School of Medicine, Baltimore, MD; daggerDivision of Blood and Marrow Transplantation, Children's National Medical Center, Washington, DC; and double daggerSidney Kimmel Cancer Center, Department of Pediatrics and Oncology, and section signDivision of Pediatric Radiology, The Russell H. Morgan Department of Radiology and Radiological Science, Johns Hopkins University School of Medicine, Baltimore, MD. FAU - Oppenheimer, Avi G AU - Oppenheimer AG FAU - Dave, Hema AU - Dave H FAU - Symons, Heather AU - Symons H FAU - Huisman, Thierry A G M AU - Huisman TA FAU - Izbudak, Izlem AU - Izbudak I LA - eng PT - Evaluation Studies PT - Journal Article PL - United States TA - J Comput Assist Tomogr JT - Journal of computer assisted tomography JID - 7703942 SB - IM MH - Adolescent MH - Child MH - Child, Preschool MH - Female MH - *Hematopoietic Stem Cell Transplantation MH - Humans MH - Infant MH - Male MH - Paranasal Sinuses/*radiography MH - Postoperative Complications/*radiography MH - Predictive Value of Tests MH - *Preoperative Care MH - Retrospective Studies MH - Sinusitis/*radiography MH - *Tomography, X-Ray Computed MH - Young Adult EDAT- 2014/12/05 06:00 MHDA- 2015/05/20 06:00 CRDT- 2014/12/05 06:00 AID - 10.1097/RCT.0000000000000185 [doi] PST - ppublish SO - J Comput Assist Tomogr. 2015 Mar-Apr;39(2):228-31. doi: 10.1097/RCT.0000000000000185. PMID- 25465233 OWN - NLM STAT- MEDLINE DA - 20150305 DCOM- 20150501 IS - 1432-0584 (Electronic) IS - 0939-5555 (Linking) VI - 94 IP - 4 DP - 2015 Apr TI - Multicenter analysis of treatment outcomes in adult patients with lymphoblastic lymphoma who received hyper-CVAD induction followed by hematopoietic stem cell transplantation. PG - 617-25 LID - 10.1007/s00277-014-2258-y [doi] AB - The hyperfractionated cyclophosphamide, vincristine, doxorubicin, and dexamethasone (hyper-CVAD) regimen has been widely used for lymphoblastic lymphoma (LBL) as a primary treatment. However, there is few data about its treatment outcome in Asian patients. Thus, we conducted this study to evaluate the efficacy of hyper-CVAD induction and stem cell transplantation (SCT) consolidation in LBL patients. The treatment responses of 49 patients treated with the hyper-CVAD regimen were retrospectively analyzed in 13 institutions. Given 24 patients who responded to hyper-CVAD underwent consolidation treatment with SCT, overall survival (OS) and progression-free survival (PFS) of patients who received SCT were compared with patients who did not. The overall response rate was 79 %: 73 % (36/49) complete responses, 6 % (3/49) partial responses, and 4 % (2/49) induction deaths. The major limitation for the delivery of the planned hyper-CVAD cycles was hematological toxicity. Among 39 responders, 24 patients underwent autologous (n = 16) and allogeneic SCT (n = 8) consolidation. Their 3-year OS and PFS rates were 76 and 78 %, respectively, and there was no difference in survival outcomes between autologous and allogeneic SCT. However, 15 patients without SCT consolidation showed poorer PFS even though they all achieved complete response. Thus, only seven patients maintained their response at the time of analysis. In conclusion, the hyper-CVAD regimen is effective for remission induction in LBL, and SCT consolidation after hyper-CVAD induction produced better clinical outcomes than did continuation of hyper-CVAD. FAU - Jeong, Seong Hyun AU - Jeong SH AD - Department of Hematology-Oncology, Ajou University School of Medicine, Suwon, South Korea. FAU - Moon, Joon Ho AU - Moon JH FAU - Kim, Jin Seok AU - Kim JS FAU - Yang, Deok-Hwan AU - Yang DH FAU - Park, Yong AU - Park Y FAU - Cho, Seok Goo AU - Cho SG FAU - Kwak, Jae-Yong AU - Kwak JY FAU - Eom, Hyeon Seok AU - Eom HS FAU - Won, Jong Ho AU - Won JH FAU - Hong, Jun Shik AU - Hong JS FAU - Oh, Sung Yong AU - Oh SY FAU - Lee, Ho Sup AU - Lee HS FAU - Kim, Seok Jin AU - Kim SJ LA - eng PT - Journal Article PT - Multicenter Study DEP - 20141203 PL - Germany TA - Ann Hematol JT - Annals of hematology JID - 9107334 RN - 57-22-7 (Vincristine) RN - 7S5I7G3JQL (Dexamethasone) RN - 80168379AG (Doxorubicin) RN - 8N3DW7272P (Cyclophosphamide) RN - CVAD protocol SB - IM MH - Adolescent MH - Adult MH - Antineoplastic Combined Chemotherapy Protocols/*therapeutic use MH - Combined Modality Therapy MH - Cyclophosphamide/therapeutic use MH - Dexamethasone/therapeutic use MH - Doxorubicin/therapeutic use MH - Female MH - *Hematopoietic Stem Cell Transplantation MH - Humans MH - Induction Chemotherapy/*methods MH - Male MH - Middle Aged MH - Precursor Cell Lymphoblastic Leukemia-Lymphoma/diagnosis/mortality/*therapy MH - Retrospective Studies MH - Survival Analysis MH - Treatment Outcome MH - Vincristine/therapeutic use MH - Young Adult EDAT- 2014/12/04 06:00 MHDA- 2015/05/02 06:00 CRDT- 2014/12/04 06:00 PHST- 2014/04/26 [received] PHST- 2014/11/10 [accepted] PHST- 2014/12/03 [aheadofprint] AID - 10.1007/s00277-014-2258-y [doi] PST - ppublish SO - Ann Hematol. 2015 Apr;94(4):617-25. doi: 10.1007/s00277-014-2258-y. Epub 2014 Dec 3. PMID- 25387866 OWN - NLM STAT- MEDLINE DA - 20150305 DCOM- 20150508 IS - 1097-0142 (Electronic) IS - 0008-543X (Linking) VI - 121 IP - 6 DP - 2015 Mar 15 TI - Phase 1/2 study of nilotinib prophylaxis after allogeneic stem cell transplantation in patients with advanced chronic myeloid leukemia or Philadelphia chromosome-positive acute lymphoblastic leukemia. PG - 863-71 LID - 10.1002/cncr.29141 [doi] AB - BACKGROUND: Allogeneic stem cell transplantation (SCT) remains the standard treatment for advanced chronic myeloid leukemia (CML) and Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph(+) ALL). Relapsed disease is the major cause of treatment failure, especially when SCT is given in the setting of advanced disease. Tyrosine kinase inhibitors can be given after transplantation prophylactically or after the detection of minimal residual disease (MRD) to reduce the relapse risk. METHODS: Posttransplant nilotinib was started after the achievement of sustained engraftment and the resolution of transplant-related toxicities. Nilotinib was continued until progression or unacceptable toxicity. RESULTS: Twenty-two patients with advanced CML (n = 15) or Ph(+) ALL (n = 7) underwent SCT with human leukocyte antigen-matched siblings (n = 11), unrelated donors (n = 7), or alternative donors (n = 4). Sixteen patients were given prophylactic nilotinib maintenance, which was started at a median of 38 days after transplantation. Six patients stopped the treatment because of toxicities (mostly gastrointestinal and hepatic). After nilotinib maintenance, 11 patients achieved (n = 9) or maintained (n = 2) a complete molecular response (CMR), and only 1 of them later relapsed. Four of the 5 patients not achieving CMR relapsed. At a median follow-up of 46 months, 9 patients were alive, and 13 had died. The 2-year overall and progression-free survival rates were 55% (95% confidence interval [CI], 34%-75%) and 45% (95% CI, 25%-66%), respectively. Among the 16 nilotinib recipients, the rates were 69% (95% CI, 46%-92%) and 56% (95% CI, 32%-81%), respectively. The 2-year nonrelapse mortality and relapse rates for all patients were 32% (95% CI, 17%-58%) and 23% (95% CI, 11%-49%), respectively. CONCLUSIONS: Nilotinib is relatively safe and effective prophylactic therapy for the prevention of relapse after SCT. It may control MRD and convert patients to CMR, which is associated with prolonged survival. These observations merit further study in larger scale studies. CI - (c) 2014 American Cancer Society. FAU - Shimoni, Avichai AU - Shimoni A AD - Division of Hematology and Bone Marrow Transplantation, Chaim Sheba Medical Center, Tel-Hashomer, Israel. FAU - Volchek, Yulia AU - Volchek Y FAU - Koren-Michowitz, Maya AU - Koren-Michowitz M FAU - Varda-Bloom, Nira AU - Varda-Bloom N FAU - Somech, Raz AU - Somech R FAU - Shem-Tov, Noga AU - Shem-Tov N FAU - Yerushalmi, Ronit AU - Yerushalmi R FAU - Nagler, Arnon AU - Nagler A LA - eng SI - ClinicalTrials.gov/NCT00750659 PT - Clinical Trial, Phase I PT - Clinical Trial, Phase II PT - Journal Article PT - Research Support, Non-U.S. Gov't DEP - 20141111 PL - United States TA - Cancer JT - Cancer JID - 0374236 RN - 0 (4-methyl-N-(3-(4-methylimidazol-1-yl)-5-(trifluoromethyl)phenyl)-3-((4-pyridin-3 -ylpyrimidin-2-yl)amino)benzamide) RN - 0 (Protein Kinase Inhibitors) RN - 0 (Pyrimidines) SB - AIM SB - IM MH - Adolescent MH - Adult MH - Aged MH - Disease-Free Survival MH - Female MH - Hematopoietic Stem Cell Transplantation/adverse effects/*methods MH - Humans MH - Leukemia, Myelogenous, Chronic, BCR-ABL Positive/*therapy MH - Male MH - Middle Aged MH - Precursor Cell Lymphoblastic Leukemia-Lymphoma/*therapy MH - Protein Kinase Inhibitors/*therapeutic use MH - Pyrimidines/*therapeutic use MH - Transplantation Conditioning/adverse effects/*methods MH - Young Adult OTO - NOTNLM OT - Philadelphia-positive OT - acute lymphoblastic leukemia OT - chronic myeloid leukemia OT - nilotinib OT - stem cell transplantation EDAT- 2014/11/13 06:00 MHDA- 2015/05/09 06:00 CRDT- 2014/11/13 06:00 PHST- 2014/04/27 [received] PHST- 2014/09/27 [revised] PHST- 2014/10/01 [accepted] PHST- 2014/11/11 [aheadofprint] AID - 10.1002/cncr.29141 [doi] PST - ppublish SO - Cancer. 2015 Mar 15;121(6):863-71. doi: 10.1002/cncr.29141. Epub 2014 Nov 11. PMID- 25332061 OWN - NLM STAT- MEDLINE DA - 20150311 DCOM- 20150519 IS - 1537-2995 (Electronic) IS - 0041-1132 (Linking) VI - 55 IP - 3 DP - 2015 Mar TI - Effect of umbilical cord blood prefreeze variables on postthaw viability. PG - 629-35 LID - 10.1111/trf.12873 [doi] AB - BACKGROUND: Assessment of the overall postthaw (PT) viability of an umbilical cord blood (UCB) unit is an important criterion for determining the quality of the unit for transplantation. Overall PT viability is a measure of cellular damage that can occur to the UCB during collection, storage, processing, freezing, and thawing. STUDY DESIGN AND METHODS: This study investigated factors measured before freezing of the UCB that could affect overall PT viability of the stem cell unit from 257 collected cord blood samples. The analysis included hematologic variables, cord blood collection characteristics, and stem cell separation and preservation factors. RESULTS: Each of the variables, postprocess (PP) neutrophils (%), PP hematocrit, overall PP viability (%), freeze rate ( degrees C/min), and time from collection to freezing (hr) were shown to contribute to overall PT viability. Each UCB sample was given a calculated "viability prediction" (VP) score based on the influence or impact of each of these variables. This score was compared to the measured PT viability. Variables with a low VP score had correspondingly low PT viability, indicating more overall damage to the cells. The results showed that the higher the VP score, the higher the PT viability. CONCLUSION: These findings provide a framework for identifying those units that are most likely to have a high overall PT viability and hence an increased likelihood of successful engraftment of the CB-sourced stem cells. The VP score could aid in the selection of a donor cord blood unit for transplantation. CI - (c) 2014 AABB. FAU - Pope, Belinda AU - Pope B AD - Pathology Department, Sydney Adventist Hospital, Wahroonga, NSW, Australia; Australasian Research Institute, Sydney Adventist Hospital, Wahroonga, NSW, Australia; Faculty of Medicine, University of New South Wales, Sydney, NSW, Australia. FAU - Hokin, Bevan AU - Hokin B FAU - Grant, Ross AU - Grant R LA - eng PT - Comparative Study PT - Journal Article DEP - 20141021 PL - United States TA - Transfusion JT - Transfusion JID - 0417360 RN - 0 (Anticoagulants) RN - 0 (Antigens, CD34) RN - 0 (Citrates) RN - 0 (Pharmaceutical Solutions) RN - 51404-37-6 (citrate phosphate dextrose) RN - IY9XDZ35W2 (Glucose) SB - IM MH - Anticoagulants MH - Antigens, CD34/analysis MH - Blood Cell Count MH - *Blood Preservation/methods MH - Blood Specimen Collection MH - Cell Survival MH - Citrates MH - *Cord Blood Stem Cell Transplantation MH - *Cryopreservation/methods MH - Fetal Blood/*cytology MH - Glucose MH - Hematocrit MH - Hematopoietic Stem Cells/cytology MH - Humans MH - Infant, Newborn MH - Neutrophils/cytology MH - Pharmaceutical Solutions EDAT- 2014/10/22 06:00 MHDA- 2015/05/20 06:00 CRDT- 2014/10/22 06:00 PHST- 2014/05/11 [received] PHST- 2014/08/05 [revised] PHST- 2014/08/07 [accepted] PHST- 2014/10/21 [aheadofprint] AID - 10.1111/trf.12873 [doi] PST - ppublish SO - Transfusion. 2015 Mar;55(3):629-35. doi: 10.1111/trf.12873. Epub 2014 Oct 21. PMID- 25234166 OWN - NLM STAT- MEDLINE DA - 20150305 DCOM- 20150508 IS - 1476-5551 (Electronic) IS - 0887-6924 (Linking) VI - 29 IP - 3 DP - 2015 Mar TI - Allogeneic and autologous stem cell transplantation for hepatosplenic T-cell lymphoma: a retrospective study of the EBMT Lymphoma Working Party. PG - 686-8 LID - 10.1038/leu.2014.280 [doi] AB - The objective of this registry study was to analyse the outcome of patients who underwent allogeneic or autologous haematopoietic stem cell transplantation (HSCT) for hepatosplenic T-cell lymphoma (HSTL), a rare and extremely aggressive peripheral T-cell lymphoma subtype. Patients were eligible if they had histologically verified HSTL and underwent HSCT between 2003 and 2011. Seventy-six patients were identified in the European Society for Bone and Marrow Transplantation database. Additional baseline and follow-up information could be obtained from the referring centres for 36 patients. Eleven of these were excluded following histopathology review, leaving 25 patients in the final study cohort (alloHSCT 18, autoHSCT 7). With a median follow-up of 36 months, 2 patients relapsed after alloHSCT, resulting in a 3-year progression-free survival of 48%. After autoHSCT, 5 patients relapsed and subsequently died. This study indicates that graft-versus-lymphoma activity conferred by alloHSCT can result in long-term survival for a substantial proportion of patients with HSTL. FAU - Tanase, A AU - Tanase A AD - 1] EBMT Lymphoma Working Party, Paris, France [2] BMT Department, Fundeni Clinical Institute, Fundeni Hospital, Bucharest, Romania. FAU - Schmitz, N AU - Schmitz N AD - 1] EBMT Lymphoma Working Party, Paris, France [2] Department of Hematology and Stem Cell Transplantation, Asklepios Klinik St Georg, Hamburg, Germany. FAU - Stein, H AU - Stein H AD - Pathodiagnostik Berlin, Berlin, Germany. FAU - Boumendil, A AU - Boumendil A AD - EBMT Lymphoma Working Party, Paris, France. FAU - Finel, H AU - Finel H AD - EBMT Lymphoma Working Party, Paris, France. FAU - Castagna, L AU - Castagna L AD - Humanitas Cancer Center, Rozzano, Italy. FAU - Blaise, D AU - Blaise D AD - Institut Paoli Calmettes, Marseille, France. FAU - Milpied, N AU - Milpied N AD - CHU Bordeaux, Bordeaux, France. FAU - Sucak, G AU - Sucak G AD - Gazi Universitesi Tip Fakultesi, Ankara, Turkey. FAU - Sureda, A AU - Sureda A AD - 1] EBMT Lymphoma Working Party, Paris, France [2] Servei d' Hematologia, Institut Catala d'Oncologia- Hospital Duran i Reynals, Barcelona, Spain. FAU - Thomson, K AU - Thomson K AD - University College London Hospital, London, UK. FAU - Vandenberghe, E AU - Vandenberghe E AD - St James's Hospital, Dublin, Ireland. FAU - Vitek, A AU - Vitek A AD - Institute of Hematology and Blood Transfusion, Prague, Czech Republic. FAU - Dreger, P AU - Dreger P AD - 1] EBMT Lymphoma Working Party, Paris, France [2] Department of Medicine, University of Heidelberg, Heidelberg, Germany. CN - Lymphoma Working Party of the EBMT LA - eng PT - Journal Article DEP - 20140919 PL - England TA - Leukemia JT - Leukemia JID - 8704895 SB - IM MH - Adolescent MH - Adult MH - Aged MH - Cooperative Behavior MH - Databases, Factual MH - Europe MH - Female MH - Follow-Up Studies MH - *Hematopoietic Stem Cell Transplantation MH - Humans MH - Liver Neoplasms/mortality/pathology/*therapy MH - Lymphoma, T-Cell, Peripheral/mortality/pathology/*therapy MH - Male MH - Middle Aged MH - Recurrence MH - *Registries MH - Retrospective Studies MH - Splenic Neoplasms/mortality/pathology/*therapy MH - Survival Analysis MH - Transplantation, Autologous MH - Transplantation, Homologous MH - Treatment Outcome IR - Avivi I FIR - Avivi, Irit IR - Bazarbachi A FIR - Bazarbachi, Ali IR - Bethge W FIR - Bethge, Wolfgang IR - Espigado I FIR - Espigado, Ildefonso IR - Faber E FIR - Faber, Edgar IR - Fegueux N FIR - Fegueux, Nathalie IR - Glass B FIR - Glass, Bertram IR - Hunter H FIR - Hunter, Hannah IR - Ifrah N FIR - Ifrah, Norbert IR - Ljungman P FIR - Ljungman, Per IR - Michallet M FIR - Michallet, Mauricette IR - Moraleda JM FIR - Moraleda, Jose Maria IR - Or R FIR - Or, Reuven IR - Poire X FIR - Poire, Xavier IR - Pretnar J FIR - Pretnar, Jose IR - Rambaldi A FIR - Rambaldi, Alessandro IR - Sargyn D FIR - Sargyn, Deniz IR - Sica S FIR - Sica, Simona IR - Snowden J FIR - Snowden, John IR - Spyridonidis A FIR - Spyridonidis, Alexandros IR - Vernant JP FIR - Vernant, Jean Paul IR - Wahlin A FIR - Wahlin, Anders IR - Yeshurun M FIR - Yeshurun, Moshe EDAT- 2014/09/23 06:00 MHDA- 2015/05/09 06:00 CRDT- 2014/09/20 06:00 PHST- 2014/07/07 [received] PHST- 2014/09/05 [revised] PHST- 2014/09/11 [accepted] PHST- 2014/09/19 [aheadofprint] AID - leu2014280 [pii] AID - 10.1038/leu.2014.280 [doi] PST - ppublish SO - Leukemia. 2015 Mar;29(3):686-8. doi: 10.1038/leu.2014.280. Epub 2014 Sep 19. PMID- 25215306 OWN - NLM STAT- MEDLINE DA - 20140912 DCOM- 20150512 IS - 2314-7156 (Electronic) IS - 2314-7156 (Linking) VI - 2014 DP - 2014 TI - Clinical features and genetic analysis of 20 Chinese patients with X-linked hyper-IgM syndrome. PG - 683160 LID - 10.1155/2014/683160 [doi] AB - X-linked hyper-IgM syndrome (XHIGM) is one type of primary immunodeficiency diseases, resulting from defects in the CD40 ligand/CD40 signaling pathways. We retrospectively analyzed the clinical and molecular features of 20 Chinese patients diagnosed and followed up in hospitals affiliated to Shanghai Jiao Tong University School of Medicine from 1999 to 2013. The median onset age of these patients was 8.5 months (range: 20 days-21 months). Half of them had positive family histories, with a shorter diagnosis lag. The most common symptoms were recurrent sinopulmonary infections (18 patients, 90%), neutropenia (14 patients, 70%), oral ulcer (13 patients, 65%), and protracted diarrhea (13 patients, 65%). Six patients had BCGitis. Six patients received hematopoietic stem cell transplantations and four of them had immune reconstructions and clinical remissions. Eighteen unique mutations in CD40L gene were identified in these 20 patients from 19 unrelated families, with 12 novel mutations. We compared with reported mutation results and used bioinformatics software to predict the effects of mutations on the target protein. These mutations reflected the heterogeneity of CD40L gene and expanded our understanding of XHIGM. FAU - Wang, Lin-Lin AU - Wang LL AD - Department of Allergy and Immunology, Shanghai Children's Medical Center, Shanghai Jiao Tong University School of Medicine, Shanghai 200127, China. FAU - Zhou, Wei AU - Zhou W AD - Department of Nephrology and Rheumatology, Shanghai Children's Medical Center, Shanghai Jiao Tong University School of Medicine, Shanghai 200127, China. FAU - Zhao, Wei AU - Zhao W AD - Department of Allergy and Immunology, Shanghai Children's Medical Center, Shanghai Jiao Tong University School of Medicine, Shanghai 200127, China ; Division of Allergy and Immunology, Department of Pediatrics, Virginia Commonwealth University, Richmond, VA 23298, USA. FAU - Tian, Zhi-Qing AU - Tian ZQ AD - Department of Allergy and Immunology, Shanghai Children's Medical Center, Shanghai Jiao Tong University School of Medicine, Shanghai 200127, China. FAU - Wang, Wei-Fan AU - Wang WF AD - Department of Allergy and Immunology, Shanghai Children's Medical Center, Shanghai Jiao Tong University School of Medicine, Shanghai 200127, China. FAU - Wang, Xiao-Fang AU - Wang XF AD - Department of Allergy and Immunology, Shanghai Children's Medical Center, Shanghai Jiao Tong University School of Medicine, Shanghai 200127, China. FAU - Chen, Tong-Xin AU - Chen TX AD - Department of Allergy and Immunology, Shanghai Children's Medical Center, Shanghai Jiao Tong University School of Medicine, Shanghai 200127, China ; Department of Nephrology and Rheumatology, Shanghai Children's Medical Center, Shanghai Jiao Tong University School of Medicine, Shanghai 200127, China ; Division of Immunology, Institute of Pediatric Translational Medicine, Shanghai Jiao Tong University School of Medicine, 1678 Dongfang Road, Shanghai 200127, China. LA - eng PT - Journal Article DEP - 20140820 PL - United States TA - J Immunol Res JT - Journal of immunology research JID - 101627166 RN - 0 (BCG Vaccine) RN - 147205-72-9 (CD40 Ligand) SB - IM MH - Adolescent MH - Adult MH - Age of Onset MH - Aged MH - Aged, 80 and over MH - Anemia/etiology MH - Asian Continental Ancestry Group/*genetics MH - BCG Vaccine/adverse effects MH - CD40 Ligand/genetics MH - Child MH - Child, Preschool MH - China MH - Genetic Testing MH - Hematopoietic Stem Cell Transplantation MH - Humans MH - Hyper-IgM Immunodeficiency Syndrome, Type 1/complications/*diagnosis/*genetics/therapy MH - Infant MH - Middle Aged MH - Mutation MH - Mycobacterium Infections/etiology MH - Prognosis MH - Transplantation, Homologous MH - Young Adult PMC - PMC4158165 OID - NLM: PMC4158165 EDAT- 2014/09/13 06:00 MHDA- 2015/05/13 06:00 CRDT- 2014/09/13 06:00 PHST- 2014/05/27 [received] PHST- 2014/07/31 [accepted] PHST- 2014/08/20 [epublish] AID - 10.1155/2014/683160 [doi] PST - ppublish SO - J Immunol Res. 2014;2014:683160. doi: 10.1155/2014/683160. Epub 2014 Aug 20. PMID- 25213459 OWN - NLM STAT- MEDLINE DA - 20140912 DCOM- 20150519 IS - 1747-4094 (Electronic) IS - 1747-4094 (Linking) VI - 7 IP - 5 DP - 2014 Oct TI - Haploidentical stem cell transplantation for the treatment of leukemia: current status. PG - 635-47 LID - 10.1586/17474086.2014.954543 [doi] AB - Haploidentical stem cell transplantation (haplo-SCT), either with T-cell depletion or T-cell replete, has been a reliable source of stem cells for patients with high-risk leukemia who do not have matched donors because it provides comparable outcomes to human leukocyte antigen-matched sibling donor transplantation, unrelated donor transplantation and umbilical cord blood transplantation. Factors, such as the Hematopoietic Cell Transplantation-Specific Comorbidity Index, associated with transplant outcomes may help us design risk-stratification-directed intervention to improve the prognosis of leukemia patients. Preliminary results of novel protocols, including co-transplant of haploidentical allografts and cord blood, as well as human leukocyte antigen-mismatched stem cell microtransplantation, for leukemia have shown that these approaches are feasible. Several strategies for enhancing the graft-versus-leukemia effects significantly decreased the relapse rate after haplo-SCT. Future direction of research will focus on perfecting available haplo-SCT protocols and determining the optimal time of haplo-SCT for leukemia and 'fit' haploidentical transplant candidates. FAU - Chang, Ying-Jun AU - Chang YJ AD - Peking University People's Hospital and Peking University Institute of Hematology, Beijing Key Laboratory of Hematopoietic Stem Cell Transplantation, No. 11 South Street of Xizhimen, Xicheng District, Beijing 100044, PR China. FAU - Wang, Yu AU - Wang Y FAU - Huang, Xiao-Jun AU - Huang XJ LA - eng PT - Journal Article PT - Research Support, Non-U.S. Gov't PT - Review DEP - 20140902 PL - England TA - Expert Rev Hematol JT - Expert review of hematology JID - 101485942 RN - 0 (HLA Antigens) SB - IM MH - Clinical Trials as Topic MH - Cord Blood Stem Cell Transplantation MH - Graft vs Host Disease/etiology MH - HLA Antigens/immunology MH - Hematopoietic Stem Cell Transplantation MH - Humans MH - Leukemia/*therapy MH - *Stem Cell Transplantation MH - T-Lymphocytes/cytology/immunology MH - Transplantation, Homologous MH - Unrelated Donors OTO - NOTNLM OT - graft-versus-leukemia effects OT - haploidentical stem cell transplantation OT - leukemia OT - relapse EDAT- 2014/09/13 06:00 MHDA- 2015/05/20 06:00 CRDT- 2014/09/13 06:00 PHST- 2014/09/02 [aheadofprint] AID - 10.1586/17474086.2014.954543 [doi] PST - ppublish SO - Expert Rev Hematol. 2014 Oct;7(5):635-47. doi: 10.1586/17474086.2014.954543. Epub 2014 Sep 2. PMID- 25209879 OWN - NLM STAT- MEDLINE DA - 20150311 DCOM- 20150519 LR - 20150318 IS - 1537-2995 (Electronic) IS - 0041-1132 (Linking) VI - 55 IP - 3 DP - 2015 Mar TI - Initial serum ferritin predicts number of therapeutic phlebotomies to iron depletion in secondary iron overload. PG - 611-22 LID - 10.1111/trf.12854 [doi] AB - BACKGROUND: Therapeutic phlebotomy is increasingly used in patients with transfusional siderosis to mitigate organ injury associated with iron overload (IO). Laboratory response variables and therapy duration are not well characterized in such patients. STUDY DESIGN AND METHODS: We retrospectively evaluated 99 consecutive patients undergoing therapeutic phlebotomy for either transfusional IO (TIO, n = 88; 76% had undergone hematopoietic transplantation) or nontransfusional indications (hyperferritinemia or erythrocytosis; n = 11). Complete blood cell count, serum ferritin (SF), transferrin saturation, and transaminases were measured serially. Phlebotomy goal was an SF level of less than 300 mug/L. RESULTS: Mean SF levels before phlebotomy among TIO and nontransfusional subjects were 3093 and 396 mug/L, respectively. Transfusion burden in the TIO group was 94 +/- 108 (mean +/- SD) RBC units; approximately half completed therapy with 24 +/- 23 phlebotomies (range, 1-103). One-third were lost to follow-up. Overall, 15% had mild adverse effects, including headache, nausea, and dizziness, mainly during first phlebotomy. Prior transfusion burden correlated poorly with initial ferritin and total number of phlebotomies to target in the TIO group. However, number of phlebotomies to target was strongly correlated with initial SF (R(2) = 0.8; p < 0.0001) in both TIO and nontransfusional groups. ALT decreased significantly with serial phlebotomy in all groups (mean initial and final values, 61 and 39 U/L; p = 0.03). CONCLUSIONS: Initial SF but not transfusion burden predicted number of phlebotomies to target in patients with TIO. Despite good treatment tolerance, significant losses to follow-up were noted. Providing patients with an estimated phlebotomy number and follow-up duration, and thus a finite endpoint, may improve compliance. Hepatic function improved with iron offloading. CI - Published 2014. This article is a U.S. Government work and is in the public domain in the USA. FAU - Panch, Sandhya R AU - Panch SR AD - Hematology/Transfusion Medicine, National Heart, Lung and Blood Institute, National Institutes of Health, Bethesda, Maryland. FAU - Yau, Yu Ying AU - Yau YY FAU - West, Kamille AU - West K FAU - Diggs, Karen AU - Diggs K FAU - Sweigart, Tamsen AU - Sweigart T FAU - Leitman, Susan F AU - Leitman SF LA - eng GR - ZIA CL002107-14/Intramural NIH HHS/United States PT - Comparative Study PT - Journal Article DEP - 20140911 PL - United States TA - Transfusion JT - Transfusion JID - 0417360 RN - 0 (Hemoglobins) RN - 0 (Transferrin) RN - 9007-73-2 (Ferritins) RN - EC 2.6.1.1 (Aspartate Aminotransferases) RN - EC 2.6.1.2 (Alanine Transaminase) SB - IM MH - Adolescent MH - Adult MH - Alanine Transaminase/blood MH - Aspartate Aminotransferases/blood MH - Blood Transfusion/adverse effects MH - Dizziness/etiology MH - Endpoint Determination MH - Erythrocyte Indices MH - Ferritins/*blood MH - Hematologic Diseases/therapy MH - Hematopoietic Stem Cell Transplantation MH - Hemoglobins/analysis MH - Humans MH - Iron Overload/blood/etiology/*therapy MH - Middle Aged MH - Nausea/etiology MH - Neoplasms/therapy MH - *Phlebotomy/adverse effects MH - Retrospective Studies MH - Transferrin/analysis MH - Young Adult PMC - PMC4362798 MID - NIHMS620751 OID - NLM: NIHMS620751 [Available on 03/01/16] OID - NLM: PMC4362798 [Available on 03/01/16] EDAT- 2014/09/12 06:00 MHDA- 2015/05/20 06:00 CRDT- 2014/09/12 06:00 PMCR- 2016/03/01 00:00 PHST- 2014/04/21 [received] PHST- 2014/07/26 [revised] PHST- 2014/07/28 [accepted] PHST- 2014/09/11 [aheadofprint] AID - 10.1111/trf.12854 [doi] PST - ppublish SO - Transfusion. 2015 Mar;55(3):611-22. doi: 10.1111/trf.12854. Epub 2014 Sep 11. PMID- 25199184 OWN - NLM STAT- MEDLINE DA - 20140909 DCOM- 20150515 IS - 1757-790X (Electronic) VI - 2014 DP - 2014 TI - Combined therapy of insulin-producing cells and haematopoietic stem cells offers better diabetic control than only haematopoietic stem cells' infusion for patients with insulin-dependent diabetes. LID - 10.1136/bcr-2013-201238 [doi] LID - bcr2013201238 [pii] AB - Insulin-dependent diabetes mellitus (IDDM) is a chronic condition characterised by impaired blood sugar metabolism and autoimmunity. We report two children: a 5-year-old girl on exogenous insulin therapy of 30 IU/day and a 9-year-old boy on short-acting insulin 30 IU/day, long-acting insulin 70 IU/day, with IDDM since 4 and 7 years, respectively. We infused in vitro-generated donor bone marrow (BM)-derived haematopoietic stem cells (HSC) in patient 1 and insulin-secreting cells trans-differentiated from autologous adipose tissue-derived mesenchymal stem cells along with BM-HSC in patient 2 under non-myeloablative conditioning. Patient 1 improved during the initial 6 months, but then again lost metabolic control with increased blood sugar levels and insulin requirement of 32 IU/day; we lost her to follow-up after 18 months. Patient 2, over follow-up of 24.87 months, has stable blood sugar levels with glycosylated haemoglobin of 6.4% and present insulin requirement of 15 IU/day. CI - 2014 BMJ Publishing Group Ltd. FAU - Dave, Shruti D AU - Dave SD AD - Department of Pathology, Laboratory Medicine, Transfusion Services and Immunohematology, G. R. Doshi and K. M. Mehta Institute of Kidney Diseases & Research Centre (IKDRC), Dr. H. L. Trivedi Institute of Transplantation Sciences (ITS), Ahmedabad, Gujarat, India. FAU - Trivedi, Hargovind L AU - Trivedi HL AD - Department of Nephrology and Transplantation Medicine, G. R. Doshi and K. M. Mehta Institute of Kidney Diseases & Research Centre (IKDRC), Dr. H. L. Trivedi Institute of Transplantation Sciences (ITS), Ahmedabad, Gujarat, India. FAU - Gopal, Saroj C AU - Gopal SC AD - Department of Paediatric Surgery, Banaras Hindu University, Varanasi, Uttar Pradesh, India. FAU - Chandra, Tulika AU - Chandra T AD - Department of Transfusion Medicine, King George's Medical University, Lucknow, Uttar Pradesh, India. LA - eng PT - Case Reports PT - Journal Article DEP - 20140908 PL - England TA - BMJ Case Rep JT - BMJ case reports JID - 101526291 RN - 0 (Blood Glucose) RN - 0 (Hemoglobin A, Glycosylated) RN - 0 (Insulin) SB - IM MH - Adipose Tissue MH - Blood Glucose/metabolism MH - Cell Differentiation MH - Child MH - Child, Preschool MH - Diabetes Mellitus, Type 1/blood/*surgery MH - Female MH - *Hematopoietic Stem Cell Transplantation MH - *Hematopoietic Stem Cells MH - Hemoglobin A, Glycosylated/metabolism MH - Humans MH - *Insulin/therapeutic use MH - Insulin-Secreting Cells/*transplantation MH - Male MH - *Mesenchymal Stem Cell Transplantation MH - *Mesenchymal Stromal Cells EDAT- 2014/09/10 06:00 MHDA- 2015/05/16 06:00 CRDT- 2014/09/09 06:00 AID - bcr-2013-201238 [pii] AID - 10.1136/bcr-2013-201238 [doi] PST - epublish SO - BMJ Case Rep. 2014 Sep 8;2014. pii: bcr2013201238. doi: 10.1136/bcr-2013-201238. PMID- 25182680 OWN - NLM STAT- MEDLINE DA - 20140903 DCOM- 20150522 IS - 1534-6307 (Electronic) IS - 1523-3774 (Linking) VI - 16 IP - 10 DP - 2014 Oct TI - Tissue-engineering strategies to repair chondral and osteochondral tissue in osteoarthritis: use of mesenchymal stem cells. PG - 452 LID - 10.1007/s11926-014-0452-5 [doi] AB - Focal chondral or osteochondral lesions can be painful and disabling because they have insufficient intrinsic repair potential, and constitute one of the major extrinsic risk factors for osteoarthritis (OA). Attention has, therefore, been paid to regenerative therapeutic procedures for the early treatment of cartilaginous defects. Current treatments for OA are not regenerative and have little effect on the progressive degeneration of joint tissue. One major reason for this underrepresentation of regenerative therapy is that approaches to treating OA with cell-based strategies have to take into consideration the larger sizes of the defects, as compared with isolated focal articular-cartilage defects, and the underlying disease process. Here, we review current treatment strategies using mesenchymal stem cells (MSCs) for chondral and osteochondral tissue repair in trauma and OA-affected joints. We discuss tissue-engineering approaches, in preclinical large-animal models and clinical studies in humans, which use crude bone-marrow aspirates and MSCs from different tissue sources in combination with bioactive agents and materials. FAU - Grassel, Susanne AU - Grassel S AD - Experimental Orthopedics, Centre for Medical Biotechnology, BioPark 1, Department of Orthopedic Surgery, University Hospital Regensburg, Regensburg, Germany, susanne.graessel@klinik.uni-regensburg.de. FAU - Lorenz, Julia AU - Lorenz J LA - eng PT - Journal Article PT - Research Support, Non-U.S. Gov't PT - Review PL - United States TA - Curr Rheumatol Rep JT - Current rheumatology reports JID - 100888970 SB - IM MH - Cartilage, Articular/*pathology MH - Humans MH - *Mesenchymal Stem Cell Transplantation MH - Osteoarthritis/pathology/*therapy MH - Wound Healing PMC - PMC4182613 OID - NLM: PMC4182613 EDAT- 2014/09/04 06:00 MHDA- 2015/05/23 06:00 CRDT- 2014/09/04 06:00 AID - 10.1007/s11926-014-0452-5 [doi] PST - ppublish SO - Curr Rheumatol Rep. 2014 Oct;16(10):452. doi: 10.1007/s11926-014-0452-5. PMID- 25180821 OWN - NLM STAT- MEDLINE DA - 20140903 DCOM- 20150518 IS - 1932-6203 (Electronic) IS - 1932-6203 (Linking) VI - 9 IP - 9 DP - 2014 TI - Complete suppression of the gut microbiome prevents acute graft-versus-host disease following allogeneic bone marrow transplantation. PG - e105706 LID - 10.1371/journal.pone.0105706 [doi] AB - The hypothesis that elimination of facultative and strict anaerobic microorganisms from the gastro-intestinal tract by antimicrobial drugs in the period of time around allogeneic bone marrow transplantation (BMT) prevents acute graft-versus-host disease (GVHD), was examined in a cohort of 112 children grafted between 1989 and 2002 for hematological malignancies. All patients received T-cell replete marrow from human leukocyte antigens (HLA) matched sibling donors under identical transplantation conditions. To eliminate microorganisms from the gastro-intestinal tract, total gastro-intestinal decontamination (GID) was applied by high doses of non-absorbable antimicrobial drugs while the graft recipient was maintained in strict protective isolation. About half of the children (51%) proved to be successfully decontaminated, and about half (49%) unsuccessfully. One recipient got acute GVHD in the first group and 8 in the second group (p = 0.013). The degree of success of total GID was decisive for the occurrence of acute GVHD, irrespective of the presence of other risk factors such as higher age of recipient and/or donor, female donor for male recipient and carriership or reactivation of herpesviruses. Our results demonstrate that successful total GID of the graft recipient prevents moderate to severe acute GVHD. We suppose that substantial translocation of gastro-intestinal microorganisms or parts of these, functioning as microbial-associated molecular patterns (MAMP's), triggering macrophages/dendritic cells via pattern recognizing receptors (PRR's) is prohibited. As a consequence the initiation and progression of an inflammatory process leading to acute GVHD is inhibited. FAU - Vossen, Jaak M AU - Vossen JM AD - Department of Pediatrics, Leiden University Medical Center, Leiden, The Netherlands. FAU - Guiot, Harry F L AU - Guiot HF AD - Department of Medical Microbiology, Leiden University Medical Center, Leiden, The Netherlands. FAU - Lankester, Arjan C AU - Lankester AC AD - Department of Pediatrics, Leiden University Medical Center, Leiden, The Netherlands. FAU - Vossen, Ann C T M AU - Vossen AC AD - Department of Medical Microbiology, Leiden University Medical Center, Leiden, The Netherlands. FAU - Bredius, Robbert G M AU - Bredius RG AD - Department of Pediatrics, Leiden University Medical Center, Leiden, The Netherlands. FAU - Wolterbeek, Ron AU - Wolterbeek R AD - Department of Medical Statistics and Bioinformatics, Leiden University Medical Center, Leiden, The Netherlands. FAU - Bakker, Hanny D J AU - Bakker HD AD - Department of Pediatrics, Leiden University Medical Center, Leiden, The Netherlands. FAU - Heidt, Peter J AU - Heidt PJ AD - Department of Pediatrics, Leiden University Medical Center, Leiden, The Netherlands; Biomedical Primate Research Centre, Rijswijk, The Netherlands. LA - eng PT - Journal Article DEP - 20140902 PL - United States TA - PLoS One JT - PloS one JID - 101285081 SB - IM MH - Bone Marrow Transplantation/*adverse effects MH - Child MH - Communicable Diseases/complications MH - Confounding Factors (Epidemiology) MH - Disease-Free Survival MH - Female MH - Gastrointestinal Tract/*microbiology MH - Graft vs Host Disease/blood/microbiology/mortality/*prevention & control MH - Humans MH - Male MH - *Microbiota MH - Siblings MH - Tissue Donors MH - Transplantation, Homologous/adverse effects MH - Treatment Outcome PMC - PMC4152127 OID - NLM: PMC4152127 EDAT- 2014/09/03 06:00 MHDA- 2015/05/20 06:00 CRDT- 2014/09/03 06:00 PHST- 2014 [ecollection] PHST- 2014/05/06 [received] PHST- 2014/07/22 [accepted] PHST- 2014/09/02 [epublish] AID - 10.1371/journal.pone.0105706 [doi] AID - PONE-D-14-17390 [pii] PST - epublish SO - PLoS One. 2014 Sep 2;9(9):e105706. doi: 10.1371/journal.pone.0105706. eCollection 2014. PMID- 25151594 OWN - NLM STAT- MEDLINE DA - 20140825 DCOM- 20150512 IS - 2212-4411 (Electronic) VI - 118 IP - 3 DP - 2014 Sep TI - Oral squamous cell carcinoma positive for p16/human papilloma virus in post allogeneic stem cell transplantation: 2 cases and review of the literature. PG - e74-8 LID - 10.1016/j.oooo.2014.05.025 [doi] LID - S2212-4403(14)00521-5 [pii] AB - Complications of allogeneic hematopoietic stem cell transplantation (allo-HSCT) includes the risk of secondary malignancies. This may be related to mechanisms including radiation and chemotherapy regimens, chronic graft-versus-host disease, inflammation, and prolonged imunosuppression. Oral squamous cell carcinoma (OSCC) is a complication associated with chronic graft-versus-host disease after allo-HSCT. Although human papillomavirus (HPV) is known to be associated with OSCC, the role of HPV in development of OSCC in post-HSCT patients has not been studied. We identified 2 cases of OSCC in allo-HSCT recipients. Both biopsy specimens tested positive for p16(INK4A), a surrogate marker for HPV. We propose that the association of OSCC and HPV in patients after allo-HSCT may not be incidental. Clinical implications of these cases may imply the need for a HPV screening, early intervention, and consideration of anti-HPV vaccination in this population. The effectiveness of such interventions could be validated in a prospective clinical study. CI - Copyright (c) 2014 Elsevier Inc. All rights reserved. FAU - Katz, Joseph AU - Katz J AD - Department of Oral Diagnostic Sciences, College of Dentistry, University of Florida. Electronic address: Jkatz@dental.ufl.edu. FAU - Islam, Mohammed Nadim AU - Islam MN AD - Department of Oral Diagnostic Sciences, College of Dentistry, University of Florida. FAU - Bhattacharyya, Indraneel AU - Bhattacharyya I AD - Department of Oral Diagnostic Sciences, College of Dentistry, University of Florida. FAU - Sandow, Pamela AU - Sandow P AD - Department of Oral Diagnostic Sciences, College of Dentistry, University of Florida. FAU - Moreb, Jan S AU - Moreb JS AD - Department of Medicine, College of Medicine, University of Florida. LA - eng PT - Case Reports PT - Journal Article PT - Review DEP - 20140613 PL - United States TA - Oral Surg Oral Med Oral Pathol Oral Radiol JT - Oral surgery, oral medicine, oral pathology and oral radiology JID - 101576782 RN - 0 (Cyclin-Dependent Kinase Inhibitor p16) SB - D SB - IM MH - Adolescent MH - Aged MH - Biopsy MH - Carcinoma, Squamous Cell/radiotherapy/surgery/*virology MH - Cyclin-Dependent Kinase Inhibitor p16/*analysis MH - Hematopoietic Stem Cell Transplantation/*adverse effects MH - Humans MH - Male MH - Mouth Neoplasms/radiotherapy/surgery/*virology MH - Papillomaviridae/*isolation & purification/pathogenicity EDAT- 2014/08/26 06:00 MHDA- 2015/05/13 06:00 CRDT- 2014/08/25 06:00 PHST- 2014/04/23 [received] PHST- 2014/05/20 [revised] PHST- 2014/05/31 [accepted] PHST- 2014/06/13 [aheadofprint] AID - S2212-4403(14)00521-5 [pii] AID - 10.1016/j.oooo.2014.05.025 [doi] PST - ppublish SO - Oral Surg Oral Med Oral Pathol Oral Radiol. 2014 Sep;118(3):e74-8. doi: 10.1016/j.oooo.2014.05.025. Epub 2014 Jun 13. PMID- 25139202 OWN - NLM STAT- MEDLINE DA - 20140904 DCOM- 20150513 IS - 1756-8722 (Electronic) IS - 1756-8722 (Linking) VI - 7 DP - 2014 TI - Multicenter phase II study of a combination of cyclosporine a, methotrexate and mycophenolate mofetil for GVHD prophylaxis: results of the Chinese Bone Marrow Transplant Cooperative Group (CBMTCG). PG - 59 LID - 10.1186/s13045-014-0059-3 [doi] AB - BACKGROUND: Improvement of current GVHD prophylactic therapies remains an important goal in the allo-HSCT. We have described a novel prophylaxis regimen in a single institution trial. The Chinese Bone Marrow Transplant Cooperative Group (CBMTCG) initiated a phase II multicenter study. METHODS: The study was designed as a prospective, single arm phase II open-label, multicenter clinical trial. The primary endpoint was improvement of aGVHD by 25% over historical control (40%) in Chinese patients. 508 patients were enrolled. All of the patients received cyclosporine A (CsA), methotrexate (MTX) and mycophenolate mofetil (MMF) (0.5-1.0 g daily for 30 days) as GVHD prophylaxis regimen. RESULTS: The primary endpoint was met with cumulative incidences of grades 2 to 4 and grades 3 to 4 aGVHD of 23.2% and 10.3%, respectively. Incidence for cGVHD was 67.4%. The non-relapse mortality (NRM) rate was 18.4% at 2 years. The probabilities of leukemia free survival (LFS) for non-advanced stage and advanced stage patients at 2 years were 69.7% and 44.8% respectively (p = 0.000). Recipient age >/= 40 years, advanced stage and Busulfan-Fludarabine(BuFlu) conditioning regimen were identified as major risk factors for aGVHD. Recipient age >/= 40 years, BuFlu conditioning regimens, female donor/male recipient and prior aGVHD were associated with cGVHD. Despite lower RM (relapse mortality), patients with grade 2-4 aGVHD had higher NRM and worse OS and LFS compared to patients with grade 0-1 aGVHD. In contrast, patients with cGVHD had better OS and LFS and lower RM compared to patients without cGVHD. CONCLUSION: The novel GVHD regimen decreased the risk for aGVHD by 42% without improving the risk for cGVHD compared to historical controls. Development of aGVHD was associated with worse OS and LFS as well as higher NRM. In contrast, cGVHD was associated with improved OS and LFS likely attributed to a GVL effect. FAU - Lai, Yong-Rong AU - Lai YR FAU - Chen, Yu-Hong AU - Chen YH FAU - Hu, Deng-Ming AU - Hu DM FAU - Jiang, Ming AU - Jiang M FAU - Liu, Qi-Fa AU - Liu QF FAU - Liu, Lin AU - Liu L FAU - Hou, Jian AU - Hou J FAU - Schwarzenberger, Paul AU - Schwarzenberger P FAU - Li, Qiao-Chuan AU - Li QC FAU - Zhang, Zhong-Ming AU - Zhang ZM FAU - Liu, Kai-Yan AU - Liu KY FAU - Huang, Xiao-Jun AU - Huang XJ AD - Beijing Key Laboratory of Hematopoietic Stem Cell Transplantation, Peking University People's Hospital, Peking University Institute of Hematology, Beijing, China. xjhrm@medmail.com.cn. LA - eng PT - Clinical Trial, Phase II PT - Journal Article PT - Multicenter Study PT - Research Support, Non-U.S. Gov't DEP - 20140821 PL - England TA - J Hematol Oncol JT - Journal of hematology & oncology JID - 101468937 RN - 0 (Drug Combinations) RN - 0 (Immunosuppressive Agents) RN - 83HN0GTJ6D (Cyclosporine) RN - 9242ECW6R0 (mycophenolate mofetil) RN - HU9DX48N0T (Mycophenolic Acid) RN - YL5FZ2Y5U1 (Methotrexate) SB - IM MH - Adolescent MH - Adult MH - Bone Marrow Transplantation/*adverse effects MH - Cyclosporine/*administration & dosage MH - Drug Combinations MH - Female MH - Graft vs Host Disease/epidemiology/*prevention & control MH - Hematopoietic Stem Cell Transplantation/adverse effects MH - Humans MH - Immunosuppressive Agents/*administration & dosage MH - Incidence MH - Kaplan-Meier Estimate MH - Leukemia/mortality/therapy MH - Male MH - Methotrexate/*administration & dosage MH - Middle Aged MH - Mycophenolic Acid/administration & dosage/*analogs & derivatives MH - Risk Factors MH - Transplantation Conditioning/methods MH - Young Adult PMC - PMC4237802 OID - NLM: PMC4237802 EDAT- 2014/08/21 06:00 MHDA- 2015/05/15 06:00 CRDT- 2014/08/21 06:00 PHST- 2014/01/21 [received] PHST- 2014/07/31 [accepted] PHST- 2014/08/21 [aheadofprint] AID - s13045-014-0059-3 [pii] AID - 10.1186/s13045-014-0059-3 [doi] PST - epublish SO - J Hematol Oncol. 2014 Aug 21;7:59. doi: 10.1186/s13045-014-0059-3. PMID- 25128615 OWN - NLM STAT- MEDLINE DA - 20140912 DCOM- 20150519 IS - 1523-6536 (Electronic) IS - 1083-8791 (Linking) VI - 20 IP - 10 DP - 2014 Oct TI - Impaired interferon-alpha production by plasmacytoid dendritic cells after cord blood transplantation in children: implication for post-transplantation toll-like receptor ligand-based immunotherapy. PG - 1501-7 LID - 10.1016/j.bbmt.2014.06.007 [doi] LID - S1083-8791(14)00355-3 [pii] AB - Plasmacytoid dendritic cells (pDCs) initiate both innate and adaptive immune responses, making them attractive targets for post-transplantation immunotherapy, particularly after cord blood transplantation (CBT). Toll-like receptor (TLR) agonists are currently studied for pDC stimulation in various clinical settings. Their efficacy depends on pDC number and functionality, which are unknown after CBT. We performed a longitudinal study of pDC reconstitution in children who underwent bone marrow transplantation (BMT) and single-unit CBT. Both CBT and unrelated BMT patients received antithymocyte globulin as part of their graft-versus-host disease prophylaxis regimen. pDC blood counts were higher in CBT patients than in healthy volunteers from 2 to 9 months after transplantation, whereas they remained lower in BMT patients. We showed that cord blood progenitors gave rise in vitro to a 500-fold increase in functional pDCs over bone marrow counterparts. Upon stimulation with a TLR agonist, pDCs from both CBT and BMT recipients upregulated T cell costimulatory molecules, whereas interferon-alpha (IFN-alpha) production was impaired for 9 months after CBT. TLR agonist treatment is thus not expected to induce IFN-alpha production by pDCs after CBT, limiting its immunotherapeutic potential. Fortunately, in vitro production of large amounts of functional pDCs from cord blood progenitors paves the way for the post-transplantation adoptive transfer of pDCs. CI - Copyright (c) 2014 American Society for Blood and Marrow Transplantation. Published by Elsevier Inc. All rights reserved. FAU - Charrier, Emily AU - Charrier E AD - Groupe de Recherche en Transplantation et Immunologie du Sang de Cordon (GRETISC), Centre de Cancerologie Charles Bruneau, Centre de Recherche du CHU Sainte-Justine, Montreal, Quebec, Canada; Departement de Sciences Biomedicales, Universite de Montreal, Quebec, Canada. FAU - Cordeiro, Paulo AU - Cordeiro P AD - Groupe de Recherche en Transplantation et Immunologie du Sang de Cordon (GRETISC), Centre de Cancerologie Charles Bruneau, Centre de Recherche du CHU Sainte-Justine, Montreal, Quebec, Canada. FAU - Brito, Rose-Marie AU - Brito RM AD - Groupe de Recherche en Transplantation et Immunologie du Sang de Cordon (GRETISC), Centre de Cancerologie Charles Bruneau, Centre de Recherche du CHU Sainte-Justine, Montreal, Quebec, Canada. FAU - Harnois, Michael AU - Harnois M AD - Groupe de Recherche en Transplantation et Immunologie du Sang de Cordon (GRETISC), Centre de Cancerologie Charles Bruneau, Centre de Recherche du CHU Sainte-Justine, Montreal, Quebec, Canada; Departement de Microbiologie et d'Immunologie, Universite de Montreal, Quebec, Canada. FAU - Mezziani, Samira AU - Mezziani S AD - Groupe de Recherche en Transplantation et Immunologie du Sang de Cordon (GRETISC), Centre de Cancerologie Charles Bruneau, Centre de Recherche du CHU Sainte-Justine, Montreal, Quebec, Canada. FAU - Herblot, Sabine AU - Herblot S AD - Groupe de Recherche en Transplantation et Immunologie du Sang de Cordon (GRETISC), Centre de Cancerologie Charles Bruneau, Centre de Recherche du CHU Sainte-Justine, Montreal, Quebec, Canada. FAU - Le Deist, Francoise AU - Le Deist F AD - Groupe de Recherche en Transplantation et Immunologie du Sang de Cordon (GRETISC), Centre de Cancerologie Charles Bruneau, Centre de Recherche du CHU Sainte-Justine, Montreal, Quebec, Canada; Departement de Microbiologie et d'Immunologie, Universite de Montreal, Quebec, Canada; Departement de Pediatrie, Universite de Montreal, Quebec, Canada. FAU - Duval, Michel AU - Duval M AD - Groupe de Recherche en Transplantation et Immunologie du Sang de Cordon (GRETISC), Centre de Cancerologie Charles Bruneau, Centre de Recherche du CHU Sainte-Justine, Montreal, Quebec, Canada; Departement de Sciences Biomedicales, Universite de Montreal, Quebec, Canada; Departement de Microbiologie et d'Immunologie, Universite de Montreal, Quebec, Canada; Departement de Pediatrie, Universite de Montreal, Quebec, Canada. Electronic address: michel.duval@umontreal.ca. LA - eng PT - Journal Article PT - Research Support, Non-U.S. Gov't DEP - 20140814 PL - United States TA - Biol Blood Marrow Transplant JT - Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation JID - 9600628 RN - 0 (Antigens, CD) RN - 0 (Antilymphocyte Serum) RN - 0 (CpG ODN 2216) RN - 0 (Immunosuppressive Agents) RN - 0 (Interferon-alpha) RN - 0 (Oligodeoxyribonucleotides) RN - 0 (TLR9 protein, human) RN - 0 (Toll-Like Receptor 9) SB - IM MH - Adolescent MH - Antigens, CD/genetics/immunology MH - Antilymphocyte Serum/therapeutic use MH - *Bone Marrow Transplantation MH - Cell Count MH - Cell Proliferation MH - Child MH - *Cord Blood Stem Cell Transplantation MH - Dendritic Cells/drug effects/*immunology/pathology MH - Female MH - Gene Expression MH - Graft vs Host Disease/prevention & control MH - Humans MH - Immunosuppressive Agents/therapeutic use MH - *Immunotherapy MH - Interferon-alpha/antagonists & inhibitors/biosynthesis MH - Leukemia/genetics/immunology/pathology/*therapy MH - Longitudinal Studies MH - Male MH - Oligodeoxyribonucleotides/*therapeutic use MH - Toll-Like Receptor 9/*agonists/genetics/immunology MH - Transplantation Conditioning MH - Transplantation, Homologous OTO - NOTNLM OT - Cord blood transplantation OT - Interferon-alpha OT - Plasmacytoid dendritic cells OT - Toll-like receptor (TLR)-9 agonist EDAT- 2014/08/17 06:00 MHDA- 2015/05/20 06:00 CRDT- 2014/08/17 06:00 PHST- 2014/02/13 [received] PHST- 2014/06/05 [accepted] PHST- 2014/08/14 [aheadofprint] AID - S1083-8791(14)00355-3 [pii] AID - 10.1016/j.bbmt.2014.06.007 [doi] PST - ppublish SO - Biol Blood Marrow Transplant. 2014 Oct;20(10):1501-7. doi: 10.1016/j.bbmt.2014.06.007. Epub 2014 Aug 14. PMID- 25115172 OWN - NLM STAT- MEDLINE DA - 20140819 DCOM- 20150515 IS - 1747-5341 (Electronic) IS - 1747-5341 (Linking) VI - 9 DP - 2014 TI - Reassessing the approach to informed consent: the case of unrelated hematopoietic stem cell transplantation in adult thalassemia patients. PG - 13 LID - 10.1186/1747-5341-9-13 [doi] AB - INTRODUCTION: The informed consent process is the legal embodiment of the fundamental right of the individual to make decisions affecting his or her health., and the patient's permission is a crucial form of respect of freedom and dignity, it becomes extremely important to enhance the patient's understanding and recall of the information given by the physician. This statement acquires additional weight when the medical treatment proposed can potentially be detrimental or even fatal. This is the case of thalassemia patients pertaining to class 3 of the Pesaro classification where Allogenic hematopoietic stem cell transplantation (HSCT) remains the only potentially curative treatment. Unfortunately, this kind of intervention is burdened by an elevated transplantation-related mortality risk (TRM: all deaths considered related to transplantation), equal to 30% according to published reports. In thalassemia, the role of the patient in the informed consent process leading up to HSCT has not been fully investigated. This study investigated the hypothesis that information provided by physicians in the medical scenario of HSCT is not fully understood by patients and that misunderstanding and communication biases may affect the clinical decision-making process. METHODS: A questionnaire was either mailed or given personally to 25 patients. A second questionnaire was administered to the 12 physicians attending the patients enrolled in this study. Descriptive statistics were used to evaluate the communication factors. RESULTS: The results pointed out the difference between the risks communicated by physicians and the risks perceived by patients. Besides the study highlighted the mortality risk considered to be acceptable by patients and that considered to be acceptable by physicians. CONCLUSIONS: Several solutions have been suggested to reduce the gap between communicated and perceived data. A multi-disciplinary approach may possibly help to attenuate some aspects of communication bias. Several tools have also been proposed to fill or to attenuate the gap between communicated and perceived data. But the most important tool is the ability of the physician to comprehend the right place of conscious consent in the relationship with the patient. FAU - Pisu, Salvatore AU - Pisu S AD - Department of Public Health, Clinical and Molecular Medicine, University of Cagliari, Cagliari, Italy. salvatore.pisu@libero.it. FAU - Caocci, Giovanni AU - Caocci G FAU - d'Aloja, Ernesto AU - d'Aloja E FAU - Efficace, Fabio AU - Efficace F FAU - Vacca, Adriana AU - Vacca A FAU - Piras, Eugenia AU - Piras E FAU - Orofino, Maria Grazia AU - Orofino MG FAU - Addari, Carmen AU - Addari C FAU - Pintor, Michela AU - Pintor M FAU - Demontis, Roberto AU - Demontis R FAU - Demuru, Federica AU - Demuru F FAU - Pittau, Maria Rita AU - Pittau MR FAU - Collins, Gary S AU - Collins GS FAU - La Nasa, Giorgio AU - La Nasa G LA - eng PT - Journal Article DEP - 20140812 PL - England TA - Philos Ethics Humanit Med JT - Philosophy, ethics, and humanities in medicine : PEHM JID - 101258058 SB - IM MH - Adolescent MH - Adult MH - Communication MH - Female MH - Graft vs Host Disease MH - Hematopoietic Stem Cell Transplantation/*adverse effects MH - Humans MH - *Informed Consent/psychology MH - Kaplan-Meier Estimate MH - Male MH - Questionnaires MH - Risk Assessment MH - Thalassemia/*therapy MH - Transplantation, Autologous MH - *Unrelated Donors MH - Young Adult PMC - PMC4136633 OID - NLM: PMC4136633 EDAT- 2014/08/15 06:00 MHDA- 2015/05/16 06:00 CRDT- 2014/08/14 06:00 PHST- 2013/07/28 [received] PHST- 2014/06/29 [accepted] PHST- 2014/08/12 [aheadofprint] AID - 1747-5341-9-13 [pii] AID - 10.1186/1747-5341-9-13 [doi] PST - epublish SO - Philos Ethics Humanit Med. 2014 Aug 12;9:13. doi: 10.1186/1747-5341-9-13. PMID- 25102358 OWN - NLM STAT- MEDLINE DA - 20140808 DCOM- 20150511 IS - 1424-3997 (Electronic) IS - 0036-7672 (Linking) VI - 144 DP - 2014 TI - Cell therapies for tendons: old cell choice for modern innovation. PG - w13989 LID - 10.4414/smw.2014.13989 [doi] LID - Swiss Med Wkly. 2014;144:w13989 [pii] AB - Although tissue engineering and cell therapies are becoming realistic approaches for medical therapeutics, it is likely that musculoskeletal applications will be among the first to benefit on a large scale. Cell sources for tissue engineering and cell therapies for tendon pathologies are reviewed with an emphasis on small defect tendon injuries as seen in the hand which could adapt well to injectable cell administration. Specifically, cell sources including tenocytes, tendon sheath fibroblasts, bone marrow or adipose-derived stem cells, amniotic cells, placenta cells and platelet-derivatives have been proposed to enhance tendon regeneration. The associated advantages and disadvantages for these different strategies will be discussed and evolving regulatory requirements for cellular therapies will also be addressed. Human progenitor tenocytes, along with their clinical cell banking potential, will be presented as an alternative cell source solution. Similar cell banking techniques have already been described with other progenitor cell types in the 1950's for vaccine production, and these "old" cell types incite potentially interesting therapeutic options that could be improved with modern innovation for tendon regeneration and repair. FAU - Petrou, Ilias G AU - Petrou IG AD - Plastic, Reconstructive and Hand Surgery, Unit of Regenerative Medicine, University Hospital of Lausanne, Switzerland. FAU - Grognuz, Anthony AU - Grognuz A AD - Plastic, Reconstructive and Hand Surgery, Unit of Regenerative Medicine, University Hospital of Lausanne, Switzerland. FAU - Hirt-Burri, Nathalie AU - Hirt-Burri N AD - Plastic, Reconstructive and Hand Surgery, Unit of Regenerative Medicine, University Hospital of Lausanne, Switzerland. FAU - Raffoul, Wassim AU - Raffoul W AD - Plastic, Reconstructive and Hand Surgery, Unit of Regenerative Medicine, University Hospital of Lausanne, Switzerland. FAU - Applegate, Lee Ann AU - Applegate LA AD - Plastic, Reconstructive and Hand Surgery, Unit of Regenerative Medicine, University Hospital of Lausanne, Switzerland. LA - eng PT - Journal Article PT - Research Support, Non-U.S. Gov't PT - Review DEP - 20140807 PL - Switzerland TA - Swiss Med Wkly JT - Swiss medical weekly JID - 100970884 SB - IM MH - Adipose Tissue MH - Bone Marrow MH - Embryonic Stem Cells/transplantation MH - European Union MH - Female MH - Humans MH - *Mesenchymal Stem Cell Transplantation/legislation & jurisprudence MH - Placenta MH - Pregnancy MH - *Regeneration MH - Tendon Injuries/*therapy MH - Tendons/cytology/*physiology MH - Tissue Engineering/*legislation & jurisprudence MH - Umbilical Cord EDAT- 2014/08/08 06:00 MHDA- 2015/05/12 06:00 CRDT- 2014/08/08 06:00 PHST- 2014/08/07 [epublish] PHST- 2014 [ecollection] AID - 10.4414/smw.2014.13989 [doi] AID - smw-13989 [pii] PST - epublish SO - Swiss Med Wkly. 2014 Aug 7;144:w13989. doi: 10.4414/smw.2014.13989. eCollection 2014. PMID- 25081956 OWN - NLM STAT- MEDLINE DA - 20140814 DCOM- 20150514 IS - 1573-3572 (Electronic) IS - 1068-9583 (Linking) VI - 21 IP - 3 DP - 2014 Sep TI - The role of social and cognitive processes in the relationship between fear network and psychological distress among parents of children undergoing hematopoietic stem cell transplantation. PG - 223-33 LID - 10.1007/s10880-014-9403-6 [doi] AB - The current study examined whether cognitive and social processing variables mediated the relationship between fear network and depression among parents of children undergoing hematopoietic stem cell transplant (HSCT). Parents whose children were initiating HSCT (N = 179) completed survey measures including fear network, Beck Depression Inventory, cognitive processing variables (positive reappraisal and self-blame) and social processing variables (emotional support and holding back from sharing concerns). Fear network was positively correlated with depression (p < .001). Self-blame and holding back emerged as individual partial mediators in the relationship between fear network and depression. Together they accounted for 34.3% of the variance in the relationship between fear network and depression. Positive reappraisal and emotional support did not have significant mediating effects. Social and cognitive processes, specifically self-blame and holding back from sharing concerns, play a negative role in parents' psychological adaptation to fears surrounding a child's HSCT. FAU - Virtue, Shannon Myers AU - Virtue SM AD - Department of Population Science, Rutgers Cancer Institute of New Jersey, 195 Little Albany, New Brunswick, NJ, 08903, USA, myerssb@cinj.rutgers.edu. FAU - Manne, Sharon AU - Manne S FAU - Mee, Laura AU - Mee L FAU - Bartell, Abraham AU - Bartell A FAU - Sands, Stephen AU - Sands S FAU - Ohman-Strickland, Pamela AU - Ohman-Strickland P FAU - Gajda, Tina Marie AU - Gajda TM LA - eng GR - R01 CA127488/CA/NCI NIH HHS/United States GR - R01 CA127488/CA/NCI NIH HHS/United States PT - Journal Article PT - Multicenter Study PT - Research Support, N.I.H., Extramural PL - United States TA - J Clin Psychol Med Settings JT - Journal of clinical psychology in medical settings JID - 9435680 SB - IM MH - Adaptation, Psychological/*physiology MH - Adult MH - Attitude to Health MH - Child MH - Cognition/*physiology MH - Cross-Sectional Studies MH - Fear/*psychology MH - Female MH - Hematopoietic Stem Cell Transplantation/*psychology MH - Humans MH - Male MH - Parents/*psychology MH - Social Behavior MH - Stress, Psychological/*psychology PMC - PMC4153406 MID - NIHMS618088 OID - NLM: NIHMS618088 [Available on 09/01/15] OID - NLM: PMC4153406 [Available on 09/01/15] EDAT- 2014/08/02 06:00 MHDA- 2015/05/15 06:00 CRDT- 2014/08/02 06:00 PMCR- 2015/09/01 00:00 AID - 10.1007/s10880-014-9403-6 [doi] PST - ppublish SO - J Clin Psychol Med Settings. 2014 Sep;21(3):223-33. doi: 10.1007/s10880-014-9403-6. PMID- 25066883 OWN - NLM STAT- MEDLINE DA - 20140915 DCOM- 20150520 LR - 20150319 IS - 1873-5967 (Electronic) IS - 1386-6532 (Linking) VI - 61 IP - 2 DP - 2014 Oct TI - Human herpesvirus 6 can be detected in cerebrospinal fluid without associated symptoms after allogeneic hematopoietic cell transplantation. PG - 289-92 LID - 10.1016/j.jcv.2014.07.001 [doi] LID - S1386-6532(14)00264-9 [pii] AB - BACKGROUND: Human herpesvirus 6 (HHV-6) is an opportunistic pathogen after hematopoietic cell transplantation (HCT) that is associated with central nervous system (CNS) dysfunction. OBJECTIVES: The aim of this study was to determine the frequency and significance of HHV-6 DNA detection in cerebrospinal fluid (CSF) after HCT. STUDY DESIGN: We identified patients with HHV-6 DNA in CSF using quantitative PCR. Patients with neurologic symptoms and HHV-6 DNA in CSF without identification of an alternative etiology were categorized as having HHV-6 CNS dysfunction. RESULTS: Among 3902 allogeneic HCT recipients from 1998 to 2012, 51 of 124 tested patients had HHV-6 DNA in CSF; 37 met criteria for HHV-6 CNS dysfunction and 14 (27%) did not. Patients with an alternative diagnosis had longer time to HHV-6 detection and lower viral load in CSF. Six patients without HHV-6 CNS dysfunction were not treated and had no morbidity attributable to HHV-6. Kaplan-Meier analysis demonstrated poor overall survival among all patients. Variables associated with higher all-cause mortality in a multivariable Cox model included alternative diagnosis (adjusted hazard ratio [aHR], 8.4; 95% CI, 1.7-40.9; P = 0.009) and higher peak plasma viral load (log(10) scale) (aHR, 1.4; 95% CI, 1.1-1.9; P = 0.01). CONCLUSION: We identified a number of allogeneic HCT recipients with HHV-6 DNA in CSF who did not meet criteria for HHV-6 CNS dysfunction. All patients had poor survival. Whether CSF HHV-6 DNA detection in patients without associated CNS dysfunction independently contributes to mortality and warrants treatment is unclear; management of these patients warrants further investigation. CI - Copyright (c) 2014 Elsevier B.V. All rights reserved. FAU - Hill, Joshua A AU - Hill JA AD - Division of Allergy and Infectious Diseases, University of Washington, Seattle, WA, United States; Vaccine and Infectious Disease Division, Fred Hutchinson Cancer Research Center, Seattle, WA, United States. Electronic address: joshuaah@uw.edu. FAU - Boeckh, Michael J AU - Boeckh MJ AD - Division of Allergy and Infectious Diseases, University of Washington, Seattle, WA, United States; Vaccine and Infectious Disease Division, Fred Hutchinson Cancer Research Center, Seattle, WA, United States. FAU - Sedlak, Ruth Hall AU - Sedlak RH AD - Department of Laboratory Medicine, University of Washington, Seattle, WA, United States. FAU - Jerome, Keith R AU - Jerome KR AD - Vaccine and Infectious Disease Division, Fred Hutchinson Cancer Research Center, Seattle, WA, United States; Department of Laboratory Medicine, University of Washington, Seattle, WA, United States. FAU - Zerr, Danielle M AU - Zerr DM AD - Vaccine and Infectious Disease Division, Fred Hutchinson Cancer Research Center, Seattle, WA, United States; Seattle Children's Research Institute, Seattle, WA, United States. LA - eng GR - CA18029/CA/NCI NIH HHS/United States GR - HL093294/HL/NHLBI NIH HHS/United States GR - K24 HL093294/HL/NHLBI NIH HHS/United States GR - P01 CA018029/CA/NCI NIH HHS/United States PT - Journal Article PT - Research Support, N.I.H., Extramural PT - Research Support, Non-U.S. Gov't DEP - 20140712 PL - Netherlands TA - J Clin Virol JT - Journal of clinical virology : the official publication of the Pan American Society for Clinical Virology JID - 9815671 RN - 0 (DNA, Viral) SB - IM MH - Adolescent MH - Adult MH - Cerebrospinal Fluid/*virology MH - Child MH - Cohort Studies MH - DNA, Viral/*cerebrospinal fluid MH - Encephalitis, Viral/*virology MH - Female MH - Hematopoietic Stem Cell Transplantation/*adverse effects MH - Herpesvirus 6, Human/*isolation & purification MH - Humans MH - Male MH - Middle Aged MH - Real-Time Polymerase Chain Reaction MH - Roseolovirus Infections/*virology MH - Survival Analysis MH - Transplantation, Homologous/*adverse effects MH - Young Adult PMC - PMC4165730 MID - NIHMS613425 OID - NLM: NIHMS613425 [Available on 10/01/15] OID - NLM: PMC4165730 [Available on 10/01/15] OTO - NOTNLM OT - CNS OT - Encephalitis OT - Herpesvirus OT - Neurologic OT - Transplant OT - hhv-6 EDAT- 2014/07/30 06:00 MHDA- 2015/05/21 06:00 CRDT- 2014/07/29 06:00 PMCR- 2015/10/01 00:00 PHST- 2014/05/02 [received] PHST- 2014/06/29 [revised] PHST- 2014/07/04 [accepted] PHST- 2014/07/12 [aheadofprint] AID - S1386-6532(14)00264-9 [pii] AID - 10.1016/j.jcv.2014.07.001 [doi] PST - ppublish SO - J Clin Virol. 2014 Oct;61(2):289-92. doi: 10.1016/j.jcv.2014.07.001. Epub 2014 Jul 12. PMID- 25064228 OWN - NLM STAT- MEDLINE DA - 20140820 DCOM- 20150514 IS - 1437-9813 (Electronic) IS - 0179-0358 (Linking) VI - 30 IP - 9 DP - 2014 Sep TI - Histological features of primary tumors after induction or high-dose chemotherapy in high-risk neuroblastoma. PG - 919-26 LID - 10.1007/s00383-014-3564-0 [doi] AB - PURPOSE: In the recent years in Japan, an increasing number of patients with neuroblastoma (NB) are being treated by the "delayed local treatment (DL)" policy, undergoing surgery after the completion of high-dose chemotherapy with hematopoietic stem cell rescue (HDC). We reviewed the histopathological findings of second-look operations, including those of patients treated with DL. PATIENTS: From 1998 to 2013, 26 patients with high-risk NB underwent radical operation following chemotherapy. Surgery was performed after induction chemotherapy in 17 cases (standard; STD), whereas 9 cases completed induction chemotherapy and HDC before undergoing tumor resection (DL). The amount of necrosis and the degree of differentiation within the post-treatment tumor were assessed. RESULTS: Eighty-eight percent of the tumors showed necrosis in more than 1/3 of the specimen. Two DL cases showed complete disappearance of viable tumor cells. Amount of necrosis did not affect the prognosis of the patient. Tumors with immature, poorly differentiated phenotypes showed an extremely aggressive thereafter. Though not statistically proven, (123)I-MIBG (metaiodobenzylguanidine) uptake may be correlated with the amount of viable cells remaining within the tumor, but not with the degree of differentiation. CONCLUSIONS: Our results support the previous reports advocating that tumors that sustain unfavorable histology after chemotherapy behave aggressively thereafter. FAU - Hishiki, Tomoro AU - Hishiki T AD - Department of Pediatric Surgery, Chiba Children's Hospital, 579-1 Heta-cho, Midori-ku, Chiba, 266-0007, Japan, hishiki-tmr@umin.ac.jp. FAU - Horie, Hiroshi AU - Horie H FAU - Higashimoto, Yasuyuki AU - Higashimoto Y FAU - Yotsumoto, Katsumi AU - Yotsumoto K FAU - Komatsu, Shugo AU - Komatsu S FAU - Okimoto, Yuri AU - Okimoto Y FAU - Kakuda, Harumi AU - Kakuda H FAU - Taneyama, Yuichi AU - Taneyama Y FAU - Saito, Takeshi AU - Saito T FAU - Terui, Keita AU - Terui K FAU - Mitsunaga, Tetsuya AU - Mitsunaga T FAU - Nakata, Mitsuyuki AU - Nakata M FAU - Ochiai, Hidemasa AU - Ochiai H FAU - Hino, Moeko AU - Hino M FAU - Ando, Kumiko AU - Ando K FAU - Yoshida, Hideo AU - Yoshida H FAU - Iwai, Jun AU - Iwai J LA - eng PT - Journal Article DEP - 20140727 PL - Germany TA - Pediatr Surg Int JT - Pediatric surgery international JID - 8609169 RN - 0 (Radiopharmaceuticals) RN - 35MRW7B4AD (3-Iodobenzylguanidine) SB - IM MH - 3-Iodobenzylguanidine/diagnostic use MH - Antineoplastic Combined Chemotherapy Protocols/*therapeutic use MH - Child MH - Child, Preschool MH - Female MH - Follow-Up Studies MH - Humans MH - Induction Chemotherapy/*methods MH - Infant MH - Japan MH - Male MH - Neuroblastoma/*drug therapy/*radionuclide imaging/surgery MH - Radiopharmaceuticals/diagnostic use MH - Retrospective Studies MH - Second-Look Surgery/methods MH - Survival Analysis MH - Treatment Outcome EDAT- 2014/07/30 06:00 MHDA- 2015/05/15 06:00 CRDT- 2014/07/28 06:00 PHST- 2014/07/15 [accepted] PHST- 2014/07/27 [aheadofprint] AID - 10.1007/s00383-014-3564-0 [doi] PST - ppublish SO - Pediatr Surg Int. 2014 Sep;30(9):919-26. doi: 10.1007/s00383-014-3564-0. Epub 2014 Jul 27. PMID- 25041793 OWN - NLM STAT- MEDLINE DA - 20140815 DCOM- 20150511 IS - 1399-3046 (Electronic) IS - 1397-3142 (Linking) VI - 18 IP - 6 DP - 2014 Sep TI - Safety and feasibility of granulocyte colony-stimulating factor (G-CSF) primed bone marrow (BM) using three days of G-CSF priming as stem cell source for pediatric allogeneic BM transplantation. PG - 625-30 LID - 10.1111/petr.12314 [doi] AB - There are limited data on the optimal dosing and schedule of G-CSF priming prior to BM harvest. We evaluated the safety and efficacy of three days of G-CSF of primed BM from related pediatric donors. Forty-five children were treated. All donors received 5 mug/kg per day of G-CSF as a single subcutaneous injection for three consecutive days prior to the BM harvest. The median age of the donors was seven yr (range, 0.8-18) and no donor experienced major adverse events related to G-CSF administration. The median age for the recipients was five yr (0.3-16 yr). Thirty-five patients had non-malignant disorders. The median dose of nucleated (TNC) and CD34+, CD3 cells infused per recipient weight was 5.4 x 10(8) /kg (range, 0.61-17), 4.7 x 10(6) /kg (range, 1.6-19), and 43.8 x 10(6) /kg (range, 1.8-95), respectively. All patients achieved neutrophil and platelets engraftment, at a median of 15 (range, 10-22) and 23 days (range, 13-111), respectively. At a median follow up of 60 months (range 12-100), the estimated five yr overall and EFS was 91% and 80%, respectively. Collection of BM following three days of G-CSF priming from pediatric donors is safe and results in high TNC and CD34+ cell yield. CI - (c) 2014 John Wiley & Sons A/S. Published by John Wiley & Sons Ltd. FAU - Hussein, Ayad Ahmed AU - Hussein AA AD - Bone Marrow and Stem Cell Transplantation Program, King Hussein Cancer Center (KHCC), Amman, Jordan. FAU - Sharma, Shanta AU - Sharma S FAU - Al-Zaben, Abdulhadi AU - Al-Zaben A FAU - Frangoul, Haydar AU - Frangoul H LA - eng PT - Journal Article DEP - 20140707 PL - Denmark TA - Pediatr Transplant JT - Pediatric transplantation JID - 9802574 RN - 143011-72-7 (Granulocyte Colony-Stimulating Factor) SB - IM MH - Adolescent MH - *Bone Marrow Transplantation MH - Child MH - Child, Preschool MH - Feasibility Studies MH - Female MH - Graft Survival MH - Granulocyte Colony-Stimulating Factor/administration & dosage/*pharmacology MH - Hematopoietic Stem Cell Mobilization/*methods MH - Humans MH - Infant MH - Jordan MH - Male MH - Transplantation Conditioning/methods MH - Transplantation, Homologous MH - Treatment Outcome OTO - NOTNLM OT - bone marrow transplantation OT - granulocyte colony-stimulating factor primed bone marrow OT - pediatric EDAT- 2014/07/22 06:00 MHDA- 2015/05/12 06:00 CRDT- 2014/07/22 06:00 PHST- 2014/06/02 [accepted] PHST- 2014/07/07 [aheadofprint] AID - 10.1111/petr.12314 [doi] PST - ppublish SO - Pediatr Transplant. 2014 Sep;18(6):625-30. doi: 10.1111/petr.12314. Epub 2014 Jul 7. PMID- 25041660 OWN - NLM STAT- MEDLINE DA - 20140815 DCOM- 20150511 IS - 1399-3046 (Electronic) IS - 1397-3142 (Linking) VI - 18 IP - 6 DP - 2014 Sep TI - Pulmonary function following allogeneic stem cell transplantation in childhood: a retrospective cohort study of 51 patients. PG - 617-24 LID - 10.1111/petr.12313 [doi] AB - HSCT is associated with a high risk of late morbidity. The aim of this study was to evaluate the frequency, time frame, risk factors, and possible etiology of pulmonary dysfunction following allogeneic HSCT in childhood. We evaluated the pulmonary function of 51 HSCT patients (>6 yr), by including FVC and FEV1 values prior to (baseline) and annually up to five yr after HSCT. A Cox proportional hazards model was used to analyze the risk factors for a pulmonary event. Over half (59%) of the patients developed pulmonary dysfunction, mainly consisting of restrictive abnormalities. Acute GvHD (HR 4.31, 95% CI 1.47-12.63), chronic GvHD (HR 10.20, 95% CI 2.42-43.03), and an abnormal baseline pulmonary function (HR 4.82, 95% CI 1.02-22.84) were associated with post-transplant dysfunction. FEV1 (p < 0.001) and FVC (p < 0.001) declined significantly by 12 months after HSCT and both remained below the pre-HSCT level at up to four yr post-transplantation. HSCT in childhood is associated with early and persistent restrictive impairment of pulmonary function. Patients with extensive chronic GvHD are particularly vulnerable to severe pulmonary dysfunction. Scheduled pulmonary function testing is warranted as part of the follow-up of survivors of HSCT in childhood. CI - (c) 2014 John Wiley & Sons A/S. Published by John Wiley & Sons Ltd. FAU - Madanat-Harjuoja, L M AU - Madanat-Harjuoja LM AD - Finnish Cancer Registry, Helsinki, Finland. FAU - Valjento, S AU - Valjento S FAU - Vettenranta, K AU - Vettenranta K FAU - Kajosaari, M AU - Kajosaari M FAU - Dyba, T AU - Dyba T FAU - Taskinen, M AU - Taskinen M LA - eng PT - Journal Article PT - Research Support, Non-U.S. Gov't DEP - 20140707 PL - Denmark TA - Pediatr Transplant JT - Pediatric transplantation JID - 9802574 SB - IM MH - Adolescent MH - Biopsy MH - Child MH - Female MH - Hematopoietic Stem Cell Transplantation/*adverse effects MH - Humans MH - Immunosuppression/methods MH - Lung Diseases/*etiology/*physiopathology MH - Male MH - Respiratory Function Tests MH - Retrospective Studies MH - Risk Factors MH - Transplantation Conditioning MH - Transplantation, Homologous MH - Treatment Outcome MH - Young Adult OTO - NOTNLM OT - childhood cancer OT - graft vs. host disease OT - late effects OT - pulmonary function OT - stem cell transplantation EDAT- 2014/07/22 06:00 MHDA- 2015/05/12 06:00 CRDT- 2014/07/22 06:00 PHST- 2014/05/30 [accepted] PHST- 2014/07/07 [aheadofprint] AID - 10.1111/petr.12313 [doi] PST - ppublish SO - Pediatr Transplant. 2014 Sep;18(6):617-24. doi: 10.1111/petr.12313. Epub 2014 Jul 7. PMID- 25040134 OWN - NLM STAT- MEDLINE DA - 20140827 DCOM- 20150515 IS - 1399-0039 (Electronic) IS - 0001-2815 (Linking) VI - 84 IP - 3 DP - 2014 Sep TI - Validation of statistical imputation of allele-level multilocus phased genotypes from ambiguous HLA assignments. PG - 285-92 LID - 10.1111/tan.12390 [doi] AB - Genetic matching for loci in the human leukocyte antigen (HLA) region between a donor and a patient in hematopoietic stem cell transplantation (HSCT) is critical to outcome; however, methods for HLA genotyping of donors in unrelated stem cell registries often yield results with allelic and phase ambiguity and/or do not query all clinically relevant loci. We present and evaluate a statistical method for in silico imputation of HLA alleles and haplotypes in large ambiguous population data from the Be The Match((R)) Registry. Our method builds on haplotype frequencies estimated from registry populations and exploits patterns of linkage disequilibrium (LD) across HLA haplotypes to infer high resolution HLA assignments. We performed validation on simulated and real population data from the Registry with non-trivial ambiguity content. While real population datasets caused some predictions to deviate from expectation, validations still showed high percent recall for imputed results with average recall >76% when imputing HLA alleles from registry data. We simulated ambiguity generated by several HLA genotyping methods to evaluate the imputation performance on several levels of typing resolution. On average, imputation percent recall of allele-level HLA haplotypes was >95% for allele-level typing, >92% for intermediate resolution typing and >58% for serology (low-resolution) typing. Thus, allele-level HLA assignments can be imputed through the application of a set of statistical and population genetics inferences and with knowledge of haplotype frequencies and self-identified race and ethnicities. CI - (c) 2014 John Wiley & Sons A/S. Published by John Wiley & Sons Ltd. FAU - Madbouly, A AU - Madbouly A AD - Bioinformatics Research, National Marrow Donor Program, Minneapolis, MN, USA. FAU - Gragert, L AU - Gragert L FAU - Freeman, J AU - Freeman J FAU - Leahy, N AU - Leahy N FAU - Gourraud, P-A AU - Gourraud PA FAU - Hollenbach, J A AU - Hollenbach JA FAU - Kamoun, M AU - Kamoun M FAU - Fernandez-Vina, M AU - Fernandez-Vina M FAU - Maiers, M AU - Maiers M LA - eng PT - Journal Article PT - Research Support, U.S. Gov't, Non-P.H.S. PT - Validation Studies DEP - 20140711 PL - England TA - Tissue Antigens JT - Tissue antigens JID - 0331072 RN - 0 (HLA Antigens) SB - IM MH - Alleles MH - Computer Simulation/statistics & numerical data MH - *Ethnic Groups MH - Gene Frequency MH - Genetic Loci/genetics MH - Genotype MH - HLA Antigens/*genetics MH - Haplotypes MH - *Hematopoietic Stem Cell Transplantation MH - Histocompatibility Testing/*methods/statistics & numerical data MH - Humans MH - Linkage Disequilibrium MH - Models, Genetic MH - Registries MH - Tissue Donors MH - United States OTO - NOTNLM OT - expectation maximization OT - human leukocyte antigen OT - imputation OT - maximum likelihood OT - typing ambiguity OT - typing resolution EDAT- 2014/07/22 06:00 MHDA- 2015/05/16 06:00 CRDT- 2014/07/22 06:00 PHST- 2013/08/15 [received] PHST- 2014/03/28 [revised] PHST- 2014/05/12 [accepted] PHST- 2014/07/11 [aheadofprint] AID - 10.1111/tan.12390 [doi] PST - ppublish SO - Tissue Antigens. 2014 Sep;84(3):285-92. doi: 10.1111/tan.12390. Epub 2014 Jul 11. PMID- 25039687 OWN - NLM STAT- MEDLINE DA - 20140815 DCOM- 20150511 IS - 1399-3046 (Electronic) IS - 1397-3142 (Linking) VI - 18 IP - 6 DP - 2014 Sep TI - High-dose chemotherapy with autologous stem cell rescue for treatment of retinoblastoma: report of five cases. PG - 631-6 LID - 10.1111/petr.12321 [doi] AB - RB is a primarily pediatric cancer arising from the retina, initiated by biallelic loss of the RB1 gene. We report five children with bilateral RB (n = 3), extra-ocular disseminated RB, or disseminated relapsed RB, who were treated with tandem high-dose chemotherapy and autologous stem cell rescue. All patients received at least 2.2 x 10(6) /kg CD34(+) (median, 3.9 x 10(6) /kg) cells. The preparative regimen for course 1 was carboplatin, thiotepa, etoposide, and for course 2, CM and melphalan. ANC of at least 0.5 x 10(9) /L occurred at a median of 11 days (range, 10-12) and 15 days (range, 12-16) after the first and second procedure, respectively. Platelet engraftment occurred at a median of 13 days (range, 12-17) and 15 days (range, 14-22) after the first and second procedure, respectively. All of the five patients treated remain alive and disease free at the last follow-up time, ranging between 21 and 44 months after completion of autologous transplant. Additional therapy was required in one patient, in whom enucleation had to be performed because of early disease relapse, refractory to local therapy. Intensification of chemotherapy with repeated high-dose chemotherapy and autologous rescue appears an acceptable choice in selected cases with bilateral or extra-ocular disease, either recurrent or refractory. CI - (c) 2014 John Wiley & Sons A/S. Published by John Wiley & Sons Ltd. FAU - Caselli, Desiree AU - Caselli D AD - Medical Direction, Azienda Ospedaliero Universitaria Meyer Children Hospital, Firenze, Italy. FAU - Tamburini, Angela AU - Tamburini A FAU - La Torre, Agostino AU - La Torre A FAU - Pollazzi, Liliana AU - Pollazzi L FAU - Tintori, Veronica AU - Tintori V FAU - Bambi, Franco AU - Bambi F FAU - Caputo, Roberto AU - Caputo R FAU - Arico, Maurizio AU - Arico M LA - eng PT - Case Reports PT - Journal Article DEP - 20140714 PL - Denmark TA - Pediatr Transplant JT - Pediatric transplantation JID - 9802574 SB - IM MH - Antineoplastic Combined Chemotherapy Protocols/*therapeutic use MH - Child, Preschool MH - Combined Modality Therapy MH - Female MH - *Hematopoietic Stem Cell Transplantation MH - Humans MH - Male MH - Retinoblastoma/*therapy MH - Transplantation, Autologous OTO - NOTNLM OT - autologous stem cell transplant OT - enucleation OT - retinoblastoma EDAT- 2014/07/22 06:00 MHDA- 2015/05/12 06:00 CRDT- 2014/07/22 06:00 PHST- 2014/06/17 [accepted] PHST- 2014/07/14 [aheadofprint] AID - 10.1111/petr.12321 [doi] PST - ppublish SO - Pediatr Transplant. 2014 Sep;18(6):631-6. doi: 10.1111/petr.12321. Epub 2014 Jul 14. PMID- 25034961 OWN - NLM STAT- MEDLINE DA - 20140912 DCOM- 20150519 IS - 1523-6536 (Electronic) IS - 1083-8791 (Linking) VI - 20 IP - 10 DP - 2014 Oct TI - Does the hematopoietic cell transplantation specific comorbidity index (HCT-CI) predict transplantation outcomes? A prospective multicenter validation study of the Kanto Study Group for Cell Therapy. PG - 1553-9 LID - 10.1016/j.bbmt.2014.06.005 [doi] LID - S1083-8791(14)00352-8 [pii] AB - Recent advances in allogeneic hematopoietic stem cell transplantation have led to increasing use of this modality in older patients who tend to have been more heavily pretreated and have more comorbidities. Thus, the evaluation of comorbidity is of increasing importance to more precisely assess the benefits and risks of the transplantation procedure. Researchers from Seattle developed the hematopoietic cell transplantation-specific comorbidity index (HCT-CI), which was associated with the risk of mortality in several retrospective studies. However, its clinical utility has not been extensively documented in prospective studies. The aim of the present study was to evaluate the utility of the HCT-CI prospectively in a multicenter setting. Overall survival (OS) and nonrelapse mortality (NRM) at 2 years were 59% and 20%, respectively (n = 243). We found that the HCT-CI in its original scale failed to predict OS and NRM in this set of patients. Thus, we applied a flexible HCT-CI risk scoring system (restratifying scores from 0 to 3 to indicate low risk, and scores of 4 or higher as high-risk). The flexible HCT-CI was found to predict 2-year NRM and OS better than the original HCT-CI (NRM: P = .01, OS: P = .003). In subgroup analysis, we evaluated the usefulness of the original HCT-CI for patients excluding those who received cord blood transplantation (n = 186). Both 2-year OS and 2-year NRM were not significantly different according to the original HCT-CI (P = .304, P = .996), but with the flexible HCT-CI, there were significant differences in 2-year OS and 2-year NRM (P = .005 and P = .005, respectively). Multivariate analysis identified age >50, performance status (PS) <90, donor type (HLA-mismatched/unrelated donor), and the flexible HCT-CI >/=4 as significant predictors for worse OS at 2 years. However, the flexible HCT-CI did not remain a significant predictor for NRM at 2 years in multivariate analysis, whereas age, PS, and donor type did. The HCT-CI did not consistently predict both NRM and OS, but it still can be a useful tool in combination with other factors, such as PS and age. Furthermore, the HCT-CI, although potentially useful for capturing pretransplantation comorbidity and risk assessment, may need further validation before its adoption for routine clinical use. CI - Copyright (c) 2014 American Society for Blood and Marrow Transplantation. Published by Elsevier Inc. All rights reserved. FAU - Nakaya, Aya AU - Nakaya A AD - Division of Hematology, Department of Medicine, Keio University School of Medicine, Tokyo, Japan. Electronic address: nakaya1016@yahoo.co.jp. FAU - Mori, Takehiko AU - Mori T AD - Division of Hematology, Department of Medicine, Keio University School of Medicine, Tokyo, Japan. FAU - Tanaka, Masatsugu AU - Tanaka M AD - Department of Hematology, Kanagawa Cancer Center, Kanagawa, Japan. FAU - Tomita, Naoto AU - Tomita N AD - Department of Rheumatology/Hematology/Infection Disease, Yokohama City University Hospital, Kanagawa, Japan. FAU - Nakaseko, Chiaki AU - Nakaseko C AD - Department of Hematology, Chiba University Hospital, Chiba, Japan. FAU - Yano, Shingo AU - Yano S AD - Division of Clinical Oncology and Hematology, Department of Internal Medicine, Jikei University School of Medicine, Tokyo, Japan. FAU - Fujisawa, Shin AU - Fujisawa S AD - Department of Hematology, Yokohama City University Medical Center, Kanagawa, Japan. FAU - Sakamaki, Hisashi AU - Sakamaki H AD - Division of Hematology, Tokyo Metropolitan Komagome Hospital, Tokyo, Japan. FAU - Aotsuka, Nobuyuki AU - Aotsuka N AD - Department of Hematology, Narita Red Cross Hospital, Chiba, Japan. FAU - Yokota, Akira AU - Yokota A AD - Department of Internal Medicine, Chiba Aoba Municipal Hospital, Chiba, Japan. FAU - Kanda, Yoshinobu AU - Kanda Y AD - Department of Hematology, Saitama Medical Center, Jichi Medical University, Saitama, Japan. FAU - Sakura, Toru AU - Sakura T AD - Department of Hematology, Saiseikai Maebashi Hospital, Gunma, Japan. FAU - Nanya, Yasuhito AU - Nanya Y AD - Department of Hematology and Oncology, The Institute of Medical Science, The University of Tokyo, Tokyo, Japan. FAU - Saitoh, Takayuki AU - Saitoh T AD - Department of Medicine and Clinical Science, Gunma University Graduate School of Medicine, Gunma, Japan. FAU - Kanamori, Heiwa AU - Kanamori H AD - Department of Hematology, Kanagawa Cancer Center, Kanagawa, Japan. FAU - Takahashi, Satoshi AU - Takahashi S AD - Division of Molecular Therapy, The Advanced Clinical Research Center, The Institute of Medical Science, The University of Tokyo, Tokyo, Japan. FAU - Okamoto, Shinichiro AU - Okamoto S AD - Division of Hematology, Department of Medicine, Keio University School of Medicine, Tokyo, Japan. LA - eng PT - Journal Article PT - Multicenter Study PT - Validation Studies DEP - 20140715 PL - United States TA - Biol Blood Marrow Transplant JT - Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation JID - 9600628 RN - 0 (Myeloablative Agonists) SB - IM MH - Adolescent MH - Adult MH - Age Factors MH - Aged MH - Female MH - Hematologic Neoplasms/immunology/mortality/pathology/*therapy MH - *Hematopoietic Stem Cell Transplantation MH - Histocompatibility Testing MH - Humans MH - Japan MH - Male MH - Middle Aged MH - Myeloablative Agonists/therapeutic use MH - Prognosis MH - Prospective Studies MH - Recurrence MH - *Research Design MH - Risk Factors MH - Survival Analysis MH - *Transplantation Conditioning MH - Transplantation, Homologous MH - Unrelated Donors OTO - NOTNLM OT - Allogeneic hematopoietic stem cell transplantation OT - Flexible hematopoietic cell transplantation-specific comorbidity index OT - Prospective study EDAT- 2014/07/19 06:00 MHDA- 2015/05/20 06:00 CRDT- 2014/07/19 06:00 PHST- 2014/03/01 [received] PHST- 2014/06/02 [accepted] PHST- 2014/07/15 [aheadofprint] AID - S1083-8791(14)00352-8 [pii] AID - 10.1016/j.bbmt.2014.06.005 [doi] PST - ppublish SO - Biol Blood Marrow Transplant. 2014 Oct;20(10):1553-9. doi: 10.1016/j.bbmt.2014.06.005. Epub 2014 Jul 15. PMID- 25034146 OWN - NLM STAT- MEDLINE DA - 20150305 DCOM- 20150508 IS - 1476-5551 (Electronic) IS - 0887-6924 (Linking) VI - 29 IP - 3 DP - 2015 Mar TI - Mesenchymal stromal cells infusions improve refractory chronic graft versus host disease through an increase of CD5+ regulatory B cells producing interleukin 10. PG - 636-46 LID - 10.1038/leu.2014.225 [doi] AB - Refractory chronic graft-versus-host disease (cGVHD) is a significant complication resulting from allogeneic hematopoietic stem cell transplantation (HSCT). Mesenchymal stromal cells (MSCs) have shown promise for treating refractory cGVHD, but the favorable effects of MSCs therapy in cGVHD are complex and not fully understood. In this prospective clinical study, 20 of 23 cGVHD patients had a complete response or partial response in a 12-month follow-up study. The most marked improvements in cGVHD symptoms were observed in the skin, oral mucosa and liver. Clinical improvement was accompanied by a significantly increased number of interleukin (IL)-10-producing CD5+ B cells. Importantly, CD5+ B cells from cGVHD patients showed increased IL-10 expression after MSCs treatment, which was associated with reduced inflammatory cytokine production by T cells. Mechanistically, MSCs could promote the survival and proliferation of CD5+ regulatory B cells (Bregs), and indoleamine 2, 3-dioxygenase partially participates in the MSC-mediated effects on Breg cells. Thus, CD5+ Breg cells may have an important role in the process of MSC-induced amelioration of refractory cGVHD and may provide new clues to reveal novel mechanisms of action for MSCs. FAU - Peng, Y AU - Peng Y AD - Center for Stem Cell Biology and Tissue Engineering, The Key Laboratory for Stem Cells and Tissue Engineering, Ministry of Education, Sun Yat-Sen University, Guangzhou, China. FAU - Chen, X AU - Chen X AD - Center for Stem Cell Biology and Tissue Engineering, The Key Laboratory for Stem Cells and Tissue Engineering, Ministry of Education, Sun Yat-Sen University, Guangzhou, China. FAU - Liu, Q AU - Liu Q AD - Department of Hematology, Nanfang Hospital, Southern Medical University, Guangzhou, China. FAU - Zhang, X AU - Zhang X AD - Center for Stem Cell Biology and Tissue Engineering, The Key Laboratory for Stem Cells and Tissue Engineering, Ministry of Education, Sun Yat-Sen University, Guangzhou, China. FAU - Huang, K AU - Huang K AD - Department of Pediatrics, Second Affiliated Hospital, Sun Yat-Sen University, Guangzhou, China. FAU - Liu, L AU - Liu L AD - Organ Transplant Center, First Affiliated Hospital, Sun Yat-Sen University, Guangzhou, China. FAU - Li, H AU - Li H AD - Guangdong Institute for Food and Drug Control, Guangdong Food and Drug Administration, Guangzhou, China. FAU - Zhou, M AU - Zhou M AD - Department of Laboratory Medicine, Guangdong General Hospital, Guangzhou, China. FAU - Huang, F AU - Huang F AD - Department of Hematology, Nanfang Hospital, Southern Medical University, Guangzhou, China. FAU - Fan, Z AU - Fan Z AD - Department of Hematology, Nanfang Hospital, Southern Medical University, Guangzhou, China. FAU - Sun, J AU - Sun J AD - Department of Hematology, Nanfang Hospital, Southern Medical University, Guangzhou, China. FAU - Liu, Q AU - Liu Q AD - Center for Stem Cell Biology and Tissue Engineering, The Key Laboratory for Stem Cells and Tissue Engineering, Ministry of Education, Sun Yat-Sen University, Guangzhou, China. FAU - Ke, M AU - Ke M AD - Center for Stem Cell Biology and Tissue Engineering, The Key Laboratory for Stem Cells and Tissue Engineering, Ministry of Education, Sun Yat-Sen University, Guangzhou, China. FAU - Li, X AU - Li X AD - Center for Stem Cell Biology and Tissue Engineering, The Key Laboratory for Stem Cells and Tissue Engineering, Ministry of Education, Sun Yat-Sen University, Guangzhou, China. FAU - Zhang, Q AU - Zhang Q AD - Department of Oral and Maxillofacial Surgery and Pharmacology, School of the Dental Medicine, University of Pennsylvania, Pennsylvania, PA, USA. FAU - Xiang, A P AU - Xiang AP AD - 1] Center for Stem Cell Biology and Tissue Engineering, The Key Laboratory for Stem Cells and Tissue Engineering, Ministry of Education, Sun Yat-Sen University, Guangzhou, China [2] Department of Biochemistry, Zhongshan Medical School, Sun Yat-Sen University, Guangzhou, China [3] Cell-Gene Therapy Translational Medicine Research Center, The Third Affiliated Hospital, Sun Yat-Sen University, Guangzhou, China. LA - eng PT - Journal Article PT - Research Support, Non-U.S. Gov't DEP - 20140718 PL - England TA - Leukemia JT - Leukemia JID - 8704895 RN - 0 (IL10 protein, human) RN - 0 (Indoleamine-Pyrrole 2,3,-Dioxygenase) RN - 130068-27-8 (Interleukin-10) SB - IM MH - Adolescent MH - Adult MH - B-Lymphocytes, Regulatory/*metabolism/pathology MH - Cell Proliferation MH - Female MH - Follow-Up Studies MH - Gene Expression MH - Graft vs Host Disease/*prevention & control MH - Hematologic Neoplasms/pathology/therapy MH - *Hematopoietic Stem Cell Transplantation MH - Humans MH - Indoleamine-Pyrrole 2,3,-Dioxygenase/genetics/metabolism MH - Interleukin-10/biosynthesis/*genetics MH - Liver/metabolism/pathology MH - Male MH - *Mesenchymal Stem Cell Transplantation MH - Mesenchymal Stromal Cells/*cytology/metabolism MH - Middle Aged MH - Mouth Mucosa/metabolism/pathology MH - Prospective Studies MH - Skin/metabolism/pathology MH - T-Lymphocytes/metabolism/pathology MH - Transplantation, Homologous EDAT- 2014/07/19 06:00 MHDA- 2015/05/09 06:00 CRDT- 2014/07/19 06:00 PHST- 2014/01/16 [received] PHST- 2014/05/30 [revised] PHST- 2014/06/30 [accepted] PHST- 2014/07/18 [aheadofprint] AID - leu2014225 [pii] AID - 10.1038/leu.2014.225 [doi] PST - ppublish SO - Leukemia. 2015 Mar;29(3):636-46. doi: 10.1038/leu.2014.225. Epub 2014 Jul 18. PMID- 25006868 OWN - NLM STAT- MEDLINE DA - 20140826 DCOM- 20150515 IS - 1557-8518 (Electronic) IS - 1540-4196 (Linking) VI - 12 IP - 7 DP - 2014 Sep TI - Metabolic syndrome appears early after hematopoietic cell transplantation. PG - 367-71 LID - 10.1089/met.2014.0051 [doi] AB - BACKGROUND: Improved survival after allogeneic hematopoietic cell transplantation (allo-HCT) enables us to learn more about potential late complications after HCT, one of which is metabolic syndrome. There are no studies investigating the prevalence or development of metabolic syndrome within the first year post-HCT in adult myeloablative transplant recipients. METHODS: In this retrospective study, we evaluated the prevalence of and risk factors associated with metabolic syndrome early post-HCT in human subjects. Due to lack of complete information regarding all the factors that define metabolic syndrome, we evaluated metabolic characteristics using available objective data referred to as modified metabolic syndrome (MMS). The cohort included 785 patients. RESULTS: We demonstrated that the incidence of MMS was 34% pre-HCT, 48% at day 80 post-HCT, and 40% at 1 year post-HCT. MMS at day 80 post-HCT was predictive of having MMS at 1 year post-HCT. CONCLUSION: These results support the need for nutrition and lifestyle intervention to prevent and treat metabolic abnormalities among patients who survive the acute transplant period. FAU - McMillen, Kerry K AU - McMillen KK AD - 1 Seattle Cancer Care Alliance , Seattle, Washington. FAU - Schmidt, Erin M AU - Schmidt EM FAU - Storer, Barry E AU - Storer BE FAU - Bar, Merav AU - Bar M LA - eng PT - Journal Article DEP - 20140709 PL - United States TA - Metab Syndr Relat Disord JT - Metabolic syndrome and related disorders JID - 101150318 SB - IM MH - Adolescent MH - Adult MH - Aged MH - Female MH - Hematopoietic Stem Cell Transplantation/*adverse effects/statistics & numerical data MH - Humans MH - Lymphoproliferative Disorders/*epidemiology/*therapy MH - Male MH - Metabolic Syndrome X/*epidemiology/*etiology MH - Middle Aged MH - Prevalence MH - Retrospective Studies MH - Risk Factors MH - Transplantation, Homologous MH - Young Adult EDAT- 2014/07/10 06:00 MHDA- 2015/05/16 06:00 CRDT- 2014/07/10 06:00 PHST- 2014/07/09 [aheadofprint] AID - 10.1089/met.2014.0051 [doi] PST - ppublish SO - Metab Syndr Relat Disord. 2014 Sep;12(7):367-71. doi: 10.1089/met.2014.0051. Epub 2014 Jul 9. PMID- 25003459 OWN - NLM STAT- MEDLINE DA - 20140813 DCOM- 20150512 IS - 1536-3708 (Electronic) IS - 0148-7043 (Linking) VI - 73 Suppl 1 DP - 2014 Sep TI - Autologous fat graft and bone marrow-derived mesenchymal stem cells assisted fat graft for treatment of Parry-Romberg syndrome. PG - S99-103 LID - 10.1097/SAP.0000000000000238 [doi] AB - BACKGROUND: Progressive facial hemiatrophy, also called Parry-Romberg syndrome (PRS), is characterized by slowly progressive atrophy of one side of the face and primarily involves the subcutaneous tissue and fat. The restoration of facial contour and symmetry in patients affected by PRS still remains a challenge clinically. Fat graft is a promising treatment but has some shortcomings, such as unpredictability and low rate of graft survival due to partial necrosis. To obviate these disadvantages, fat graft assisted by bone marrow-derived mesenchymal stem cells (BMSCs) was used to treat PRS patients and the outcome was evaluated in comparison with the conventional treatment by autologous fat graft. METHODS: Autologous fat graft was harvested by tumescent liposuction. Bone marrow-derived mesenchymal stem cells were then isolated by human Lymphocytes Separation Medium through density gradient centrifugation. Twenty-six patients were treated with autologous fat graft only (group A), whereas 10 other patients were treated with BMSC-assisted fat graft (group B). The Coleman technique was applied in all fat graft injections. RESULTS: The follow-up period was 6 to 12 months in this study, In group A, satisfactory outcome judged by symmetrical appearances was obtained with 1 injection in 12 patients, 2 injections in 8 patients, and 3 injections in 4 patients. However, the result of 1 patient was not satisfactory and 1 patient was overcorrected. In group B, 10 patients obtained satisfactory outcomes and almost reached symmetry by 1 injection. No complications (infection, hematoma, or subcutaneous mass) were observed. CONCLUSIONS: The results suggest that BMSC-assisted fat graft is effective and safe for soft tissue augmentation and may be superior to conventional lipoinjection. Additional study is necessary to further evaluate the efficacy of this technique. FAU - Jianhui, Zhao AU - Jianhui Z AD - *Department of Plastic Surgery, Xijing Hospital, Fourth Military Medical University, Xi'an, Shaanxi; and daggerDepartment of Plastic Surgery, Chinese PLA General Hospital, Beijing, China. FAU - Chenggang, Yi AU - Chenggang Y FAU - Binglun, Lu AU - Binglun L FAU - Yan, Han AU - Yan H FAU - Li, Yang AU - Li Y FAU - Xianjie, Ma AU - Xianjie M FAU - Yingjun, Su AU - Yingjun S FAU - Shuzhong, Guo AU - Shuzhong G LA - eng PT - Evaluation Studies PT - Journal Article PT - Research Support, Non-U.S. Gov't PL - United States TA - Ann Plast Surg JT - Annals of plastic surgery JID - 7805336 SB - IM MH - Adipose Tissue/*transplantation MH - Adolescent MH - Adult MH - Bone Marrow Cells MH - Facial Hemiatrophy/*surgery MH - Female MH - Humans MH - Male MH - *Mesenchymal Stem Cell Transplantation MH - Reconstructive Surgical Procedures/methods MH - Transplantation, Autologous MH - Young Adult EDAT- 2014/07/09 06:00 MHDA- 2015/05/13 06:00 CRDT- 2014/07/09 06:00 AID - 10.1097/SAP.0000000000000238 [doi] PST - ppublish SO - Ann Plast Surg. 2014 Sep;73 Suppl 1:S99-103. doi: 10.1097/SAP.0000000000000238. PMID- 25001087 OWN - NLM STAT- MEDLINE DA - 20140708 DCOM- 20150514 IS - 1559-131X (Electronic) IS - 1357-0560 (Linking) VI - 31 IP - 8 DP - 2014 Aug TI - Haploidentical hematopoietic stem cell transplantation following myeloablative conditioning regimens in hematologic diseases with G-CSF-mobilized peripheral blood stem cells grafts without T cell depletion: a single center report of 38 cases. PG - 81 LID - 10.1007/s12032-014-0081-x [doi] AB - Many Chinese patients with hematologic diseases, who need allogeneic hematopoietic stem cell transplantation (HSCT), lack a human leukocyte antigen-matched donor. To save these patients and to avoid collecting donor bone marrow graft, we adopted haploidentical peripheral blood HSCT with granulocyte colony stimulating factor (G-CSF) mobilized peripheral blood stem cells as the grafts without ex vivo T cell depletion. Thirty-eight patients were enrolled, and they received myeloablative preconditioning. Thirty-five patients attained a successful neutrophil and platelet recovery. The median time for the neutrophil recovery was 16 days (range of 10-23 days), and the median time for the platelet recovery was 19 days (range of 10-66 days). During the follow-up at a median time of 33.1 weeks (range of 1.1-412.6 weeks), eleven (28.9 %) patients developed aGVHD grade I-II and seven (18.4 %) patients developed aGVHD grade III-IV. The incidence of cGVHD was 27.6 %, and nine (23.7 %) patients died within the first 100 days after transplantation. The cumulative survival proportions at 1 and 2 years were 52.51 +/- 8.57 % and 43.76 +/- 9.11 %, respectively. These results suggested that the G-CSF-primed peripheral blood stem cell grafts, without in vitro T cell depletion, could be an appropriate stem cell source for Haplo-HSCT. FAU - Lu, Rui-Nan AU - Lu RN AD - Department of Hematology, Jiangsu Province Hospital, The First Affiliated Hospital of Nanjing Medical University, Nanjing, 210029, China. FAU - Miao, Kou-Rong AU - Miao KR FAU - Zhang, Run AU - Zhang R FAU - Hong, Ming AU - Hong M FAU - Xu, Ji AU - Xu J FAU - Zhu, Yu AU - Zhu Y FAU - Zhu, Hua-Yuan AU - Zhu HY FAU - Qu, Xiao-Yan AU - Qu XY FAU - Wang, Shuai AU - Wang S FAU - Wang, Li AU - Wang L FAU - Fan, Lei AU - Fan L FAU - Shen, Wen-Yi AU - Shen WY FAU - Lu, Hua AU - Lu H FAU - Qiu, Hong-Xia AU - Qiu HX FAU - Zhang, Xiao-Yan AU - Zhang XY FAU - Chen, Li-Juan AU - Chen LJ FAU - Xu, Wei AU - Xu W FAU - Li, Jian-Yong AU - Li JY FAU - Wu, Han-Xin AU - Wu HX FAU - Qian, Si-Xuan AU - Qian SX LA - eng PT - Clinical Trial PT - Journal Article PT - Research Support, Non-U.S. Gov't DEP - 20140708 PL - United States TA - Med Oncol JT - Medical oncology (Northwood, London, England) JID - 9435512 RN - 0 (Antibodies, Monoclonal) RN - 0 (Recombinant Fusion Proteins) RN - 0 (basiliximab) RN - 143011-72-7 (Granulocyte Colony-Stimulating Factor) SB - IM MH - Adolescent MH - Adult MH - Antibodies, Monoclonal/therapeutic use MH - Female MH - Graft vs Host Disease/epidemiology/prevention & control MH - Granulocyte Colony-Stimulating Factor/administration & dosage/*therapeutic use MH - Haplotypes MH - Hematopoietic Stem Cell Mobilization/adverse effects/*methods MH - Hematopoietic Stem Cell Transplantation/adverse effects/*methods MH - Humans MH - Leukemia/mortality/*therapy MH - Male MH - Middle Aged MH - Peripheral Blood Stem Cell Transplantation/*methods MH - Recombinant Fusion Proteins/therapeutic use MH - Survival Analysis MH - *T-Lymphocytes MH - Tissue Donors MH - Transplantation Conditioning/*methods MH - Treatment Outcome MH - Young Adult EDAT- 2014/07/09 06:00 MHDA- 2015/05/15 06:00 CRDT- 2014/07/09 06:00 PHST- 2014/05/20 [received] PHST- 2014/06/13 [accepted] PHST- 2014/07/08 [aheadofprint] AID - 10.1007/s12032-014-0081-x [doi] PST - ppublish SO - Med Oncol. 2014 Aug;31(8):81. doi: 10.1007/s12032-014-0081-x. Epub 2014 Jul 8. PMID- 24986711 OWN - NLM STAT- MEDLINE DA - 20140813 DCOM- 20150512 IS - 1544-2241 (Electronic) IS - 1544-1873 (Linking) VI - 12 IP - 3 DP - 2014 Sep TI - Bone morbidity in childhood leukemia: epidemiology, mechanisms, diagnosis, and treatment. PG - 300-12 LID - 10.1007/s11914-014-0222-3 [doi] AB - Skeletal abnormalities are commonly seen in children and adolescents with leukemia. The spectrum ranges from mild pain to debilitating osteonecrosis (ON) and fractures. In this review, we summarize the skeletal manifestations, provide an update on therapeutic strategies for prevention and treatment, and discuss the most recent advances in musculoskeletal research. Early recognition of skeletal abnormalities and strategies to optimize bone health are essential to prevent long-term skeletal sequelae and diminished quality of life observed in children and adolescents with leukemia. FAU - Mostoufi-Moab, Sogol AU - Mostoufi-Moab S AD - Department of Pediatrics, The Children's Hospital of Philadelphia, The University of Pennsylvania Perelman School of Medicine, 34th Street and Civic Center Boulevard, Philadelphia, PA, 19104, USA, moab@email.chop.edu. FAU - Halton, Jacqueline AU - Halton J LA - eng PT - Journal Article PT - Review PL - United States TA - Curr Osteoporos Rep JT - Current osteoporosis reports JID - 101176492 SB - IM MH - Bone Diseases/*complications/diagnosis/therapy MH - Child MH - Hematopoietic Stem Cell Transplantation MH - Humans MH - Leukemia/complications/therapy MH - Osteonecrosis/complications/diagnosis/therapy MH - Precursor Cell Lymphoblastic Leukemia-Lymphoma/*complications/therapy MH - Spinal Fractures/complications/diagnosis/therapy PMC - PMC4131149 OID - NLM: PMC4131149 EDAT- 2014/07/06 06:00 MHDA- 2015/05/13 06:00 CRDT- 2014/07/03 06:00 AID - 10.1007/s11914-014-0222-3 [doi] PST - ppublish SO - Curr Osteoporos Rep. 2014 Sep;12(3):300-12. doi: 10.1007/s11914-014-0222-3. PMID- 24979733 OWN - NLM STAT- MEDLINE DA - 20140912 DCOM- 20150519 IS - 1523-6536 (Electronic) IS - 1083-8791 (Linking) VI - 20 IP - 10 DP - 2014 Oct TI - Immunologic resolution of human chronic graft-versus-host disease. PG - 1508-15 LID - 10.1016/j.bbmt.2014.06.030 [doi] LID - S1083-8791(14)00397-8 [pii] AB - To determine the role of regulatory T lymphocytes (Tregs) in the pathogenesis of human chronic graft-versus-host disease (GVHD) and its clinical resolution, we evaluated long-term recipients of pediatric allogeneic hematopoietic stem cell transplantation (HSCT). Seventy-one recipients were evaluated, 30 of whom had a history of chronic GVHD, including 16 with active chronic GVHD and 14 with resolved chronic GVHD. There were no significant clinical differences and no differences in the frequency of Tregs (CD4(+), CD127(-), CD25(+)) between the recipients with active chronic GVHD and those with resolved chronic GVHD. Using the Miyara/Sakaguchi classification scheme to identify functional Tregs, a decreased frequency of functional resting Tregs (rTregs) was identified in recipients with active chronic GVHD (P = .009 compared with normal donors; P = .001 compared with HSCT recipients without history of chronic GVHD; P = .005 compared with recipients with resolved chronic GVHD). The frequency and number of recent thymic emigrants in rTregs were normal in recipients with resolved chronic GVHD, but persistently decreased in recipients with active chronic GVHD. These results support the hypothesis that the reestablishment of normal numbers of functional rTregs is required for the clinical resolution of chronic GVHD. CI - Copyright (c) 2014 American Society for Blood and Marrow Transplantation. Published by Elsevier Inc. All rights reserved. FAU - Mahadeo, Kris M AU - Mahadeo KM AD - Division of Blood and Marrow Transplantation, Children's Center for Cancer and Blood Diseases, Children's Hospital Los Angeles, and Department of Pediatrics, Keck School of Medicine, University of Southern California, Los Angeles, California. FAU - Masinsin, Bernadette AU - Masinsin B AD - Division of Blood and Marrow Transplantation, Children's Center for Cancer and Blood Diseases, Children's Hospital Los Angeles, and Department of Pediatrics, Keck School of Medicine, University of Southern California, Los Angeles, California. FAU - Kapoor, Neena AU - Kapoor N AD - Division of Blood and Marrow Transplantation, Children's Center for Cancer and Blood Diseases, Children's Hospital Los Angeles, and Department of Pediatrics, Keck School of Medicine, University of Southern California, Los Angeles, California. FAU - Shah, Ami J AU - Shah AJ AD - Division of Blood and Marrow Transplantation, Children's Center for Cancer and Blood Diseases, Children's Hospital Los Angeles, and Department of Pediatrics, Keck School of Medicine, University of Southern California, Los Angeles, California. FAU - Abdel-Azim, Hisham AU - Abdel-Azim H AD - Division of Blood and Marrow Transplantation, Children's Center for Cancer and Blood Diseases, Children's Hospital Los Angeles, and Department of Pediatrics, Keck School of Medicine, University of Southern California, Los Angeles, California. FAU - Parkman, Robertson AU - Parkman R AD - Division of Blood and Marrow Transplantation, Children's Center for Cancer and Blood Diseases, Children's Hospital Los Angeles, and Department of Pediatrics, Keck School of Medicine, University of Southern California, Los Angeles, California. Electronic address: rparkman@usc.edu. LA - eng PT - Journal Article DEP - 20140627 PL - United States TA - Biol Blood Marrow Transplant JT - Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation JID - 9600628 RN - 0 (Antigens, CD) RN - 0 (Myeloablative Agonists) SB - IM MH - Adolescent MH - Antigens, CD/genetics/immunology MH - CD4 Lymphocyte Count MH - Cell Proliferation MH - Child MH - Child, Preschool MH - Chronic Disease MH - Cross-Sectional Studies MH - Female MH - Gene Expression MH - Graft vs Host Disease/genetics/immunology/pathology/*therapy MH - Hematologic Diseases/genetics/immunology/pathology/*therapy MH - *Hematopoietic Stem Cell Transplantation MH - Humans MH - Infant MH - Male MH - Myeloablative Agonists/therapeutic use MH - T-Lymphocytes, Regulatory/drug effects/*immunology/pathology MH - *Transplantation Conditioning MH - Transplantation, Homologous OTO - NOTNLM OT - Chronic graft-versus-host disease OT - Regulatory T lymphocytes OT - Thymic function EDAT- 2014/07/01 06:00 MHDA- 2015/05/20 06:00 CRDT- 2014/07/01 06:00 PHST- 2014/04/08 [received] PHST- 2014/06/23 [accepted] PHST- 2014/06/27 [aheadofprint] AID - S1083-8791(14)00397-8 [pii] AID - 10.1016/j.bbmt.2014.06.030 [doi] PST - ppublish SO - Biol Blood Marrow Transplant. 2014 Oct;20(10):1508-15. doi: 10.1016/j.bbmt.2014.06.030. Epub 2014 Jun 27. PMID- 24978300 OWN - NLM STAT- MEDLINE DA - 20140815 DCOM- 20150511 IS - 1399-3046 (Electronic) IS - 1397-3142 (Linking) VI - 18 IP - 6 DP - 2014 Sep TI - Successful second unrelated cord blood transplantation in a child with juvenile myelomonocytic leukemia. PG - 651-2 LID - 10.1111/petr.12312 [doi] FAU - Alsultan, Abdulrahman AU - Alsultan A AD - Department of Oncology, King Abdulaziz Medical City, National Guard Health Affairs, Riyadh, Saudi Arabia; Department of Pediatrics, College of Medicine, King Saud University, Riyadh, Saudi Arabia. aalsultan@gmail.com. FAU - Jarrar, Mohammed AU - Jarrar M FAU - Mushaqbah, Walid AU - Mushaqbah W FAU - Al-Sudairy, Reem AU - Al-Sudairy R FAU - Jawdat, Dunia AU - Jawdat D LA - eng PT - Case Reports PT - Letter DEP - 20140630 PL - Denmark TA - Pediatr Transplant JT - Pediatric transplantation JID - 9802574 RN - 0 (Immunosuppressive Agents) SB - IM MH - *Cord Blood Stem Cell Transplantation MH - Humans MH - Immunosuppressive Agents/*administration & dosage MH - Infant MH - Leukemia, Myelomonocytic, Juvenile/*therapy MH - Male MH - Splenectomy MH - Transplantation Conditioning/methods MH - Transplantation, Homologous EDAT- 2014/07/01 06:00 MHDA- 2015/05/12 06:00 CRDT- 2014/07/01 06:00 PHST- 2014/06/30 [aheadofprint] AID - 10.1111/petr.12312 [doi] PST - ppublish SO - Pediatr Transplant. 2014 Sep;18(6):651-2. doi: 10.1111/petr.12312. Epub 2014 Jun 30. PMID- 24977928 OWN - NLM STAT- MEDLINE DA - 20140815 DCOM- 20150511 IS - 1399-3046 (Electronic) IS - 1397-3142 (Linking) VI - 18 IP - 6 DP - 2014 Sep TI - A trial of alemtuzumab adjunctive therapy in allogeneic hematopoietic cell transplantation with minimal conditioning for severe combined immunodeficiency. PG - 609-16 LID - 10.1111/petr.12310 [doi] AB - For infants with SCID the ideal conditioning regimen before allogeneic HCT would omit cytotoxic chemotherapy to minimize short- and long-term complications. We performed a prospective pilot trial with alemtuzumab monotherapy to overcome NK-cell mediated immunologic barriers to engraftment. We enrolled four patients who received CD34-selected haploidentical cells, two of whom failed to engraft donor T cells. The two patients who engrafted had delayed T-cell reconstitution, despite rapid clearance of circulating alemtuzumab. Although well-tolerated, alemtuzumab failed to overcome immunologic barriers to donor engraftment. Furthermore, alemtuzumab may slow T-cell development in patients with SCID in the setting of a T-cell depleted graft. CI - (c) 2014 John Wiley & Sons A/S. Published by John Wiley & Sons Ltd. FAU - Dvorak, Christopher C AU - Dvorak CC AD - Division of Pediatric Allergy, Immunology, and Blood and Marrow Transplant, University of California San Francisco, Benioff Children's Hospital, San Francisco, CA, USA. FAU - Horn, Biljana N AU - Horn BN FAU - Puck, Jennifer M AU - Puck JM FAU - Adams, Stuart AU - Adams S FAU - Veys, Paul AU - Veys P FAU - Czechowicz, Agnieszka AU - Czechowicz A FAU - Cowan, Morton J AU - Cowan MJ LA - eng GR - UL1 RR024131/RR/NCRR NIH HHS/United States PT - Journal Article PT - Research Support, N.I.H., Extramural PT - Research Support, Non-U.S. Gov't DEP - 20140630 PL - Denmark TA - Pediatr Transplant JT - Pediatric transplantation JID - 9802574 RN - 0 (Antibodies, Monoclonal, Humanized) RN - 3A189DH42V (alemtuzumab) SB - IM MH - Antibodies, Monoclonal, Humanized/adverse effects/*therapeutic use MH - California MH - Chemotherapy, Adjuvant MH - Child MH - *Hematopoietic Stem Cell Transplantation MH - Histocompatibility Testing MH - Humans MH - Infant MH - Male MH - Pilot Projects MH - Prospective Studies MH - Severe Combined Immunodeficiency/drug therapy/immunology/*therapy MH - Transplantation Conditioning MH - Transplantation, Homologous MH - Treatment Outcome OTO - NOTNLM OT - alemtuzumab OT - engraftment OT - haploidentical OT - severe combined immunodeficiency EDAT- 2014/07/01 06:00 MHDA- 2015/05/12 06:00 CRDT- 2014/07/01 06:00 PHST- 2014/05/28 [accepted] PHST- 2014/06/30 [aheadofprint] AID - 10.1111/petr.12310 [doi] PST - ppublish SO - Pediatr Transplant. 2014 Sep;18(6):609-16. doi: 10.1111/petr.12310. Epub 2014 Jun 30. PMID- 24977650 OWN - NLM STAT- MEDLINE DA - 20140815 DCOM- 20150511 LR - 20150408 IS - 1399-3046 (Electronic) IS - 1397-3142 (Linking) VI - 18 IP - 6 DP - 2014 Sep TI - A trial of plerixafor adjunctive therapy in allogeneic hematopoietic cell transplantation with minimal conditioning for severe combined immunodeficiency. PG - 602-8 LID - 10.1111/petr.12309 [doi] AB - For infants with SCID, the ideal conditioning regimen before allogeneic HCT would omit cytotoxic chemotherapy to minimize short- and long-term complications. We performed a prospective pilot trial with G-CSF plus plerixafor given to the host to mobilize HSC from their niches. We enrolled six patients who received CD34-selected haploidentical cells and one who received T-replete matched unrelated BM. All patients receiving G-CSF and plerixafor had generally poor CD34(+) cell and Lin(-) CD34(+) CD38(-) CD90(+) CD45RA(-) HSC mobilization, and developed donor T cells, but no donor myeloid or B-cell engraftment. Although well tolerated, G-CSF plus plerixafor alone failed to overcome physical barriers to donor engraftment. CI - (c) 2014 John Wiley & Sons A/S. Published by John Wiley & Sons Ltd. FAU - Dvorak, Christopher C AU - Dvorak CC AD - Division of Pediatric Allergy, Immunology and Blood and Marrow Transplant, University of California San Francisco Benioff Children's Hospital, San Francisco, CA, USA. FAU - Horn, Biljana N AU - Horn BN FAU - Puck, Jennifer M AU - Puck JM FAU - Czechowicz, Agnieszka AU - Czechowicz A FAU - Shizuru, Judy A AU - Shizuru JA FAU - Ko, Rose M AU - Ko RM FAU - Cowan, Morton J AU - Cowan MJ LA - eng GR - R01 AI078248/AI/NIAID NIH HHS/United States GR - UL1 RR024131/RR/NCRR NIH HHS/United States GR - UL1 TR000004/TR/NCATS NIH HHS/United States PT - Journal Article PT - Research Support, N.I.H., Extramural DEP - 20140630 PL - Denmark TA - Pediatr Transplant JT - Pediatric transplantation JID - 9802574 RN - 0 (Heterocyclic Compounds) RN - 143011-72-7 (Granulocyte Colony-Stimulating Factor) RN - 155148-31-5 (JM 3100) SB - IM MH - Adolescent MH - California MH - Chemotherapy, Adjuvant MH - Child MH - Combined Modality Therapy MH - Drug Therapy, Combination MH - Female MH - Granulocyte Colony-Stimulating Factor/therapeutic use MH - Hematopoietic Stem Cell Mobilization MH - *Hematopoietic Stem Cell Transplantation MH - Heterocyclic Compounds/*therapeutic use MH - Histocompatibility Testing MH - Humans MH - Infant MH - Male MH - Pilot Projects MH - Prospective Studies MH - Severe Combined Immunodeficiency/drug therapy/immunology/*therapy MH - Transplantation Conditioning MH - Treatment Outcome PMC - PMC4134761 MID - NIHMS601007 OID - NLM: NIHMS601007 [Available on 09/01/15] OID - NLM: PMC4134761 [Available on 09/01/15] OTO - NOTNLM OT - engraftment OT - haploidentical OT - plerixafor OT - severe combined immunodeficiency EDAT- 2014/07/01 06:00 MHDA- 2015/05/12 06:00 CRDT- 2014/07/01 06:00 PMCR- 2015/09/01 00:00 PHST- 2014/05/28 [accepted] PHST- 2014/06/30 [aheadofprint] AID - 10.1111/petr.12309 [doi] PST - ppublish SO - Pediatr Transplant. 2014 Sep;18(6):602-8. doi: 10.1111/petr.12309. Epub 2014 Jun 30. PMID- 24973628 OWN - NLM STAT- MEDLINE DA - 20140912 DCOM- 20150519 IS - 1523-6536 (Electronic) IS - 1083-8791 (Linking) VI - 20 IP - 10 DP - 2014 Oct TI - Clostridium difficile infection after allogeneic hematopoietic stem cell transplant: strain diversity and outcomes associated with NAP1/027. PG - 1626-33 LID - 10.1016/j.bbmt.2014.06.025 [doi] LID - S1083-8791(14)00375-9 [pii] AB - Allogeneic hematopoietic stem cell transplantation (HSCT) recipients are at high risk for developing Clostridium difficile infection (CDI). We studied the incidence, risk factors, NAP1/027 prevalence, and clinical outcomes, including acute lower gastrointestinal graft-versus-host disease (GI GVHD), associated with early CDI in this population. A retrospective review was conducted of patients who underwent allogeneic HSCT at Memorial Sloan Kettering Cancer Center from January 1, 2005 to September 30, 2010. Early CDI was defined as infection occurring from day -10 to day +40 from stem cell infusion. Among 793 patients who received allogeneic HSCTs, early CDI occurred in 11.9%; 56% cases were between day -5 and day +5. Overall incidence was 25.2 cases/10,000 at-risk days. There was a high prevalence of NAP1/027 strains during peak incidence (61% in 2008). NAP1/027 was the most common strain in both adult and pediatric cases (24% and 23%, respectively). CDI was clinically mild, including those due to NAP1/027. Metronidazole was the primary treatment for 91 of 94 patients, 7 of 8 cases refractory to metronidazole had no response to vancomycin, and none was due to NAP1/027. Relapse of CDI was common (31%). The cumulative incidence of GI GVHD in patients with and without early CDI was 6.8% and 8%, respectively (P = .5). Most cases of CDI occurred during conditioning or immediately after transplant. Despite high prevalence of NAP1/027, we found only mild disease. Most patients were treated successfully with metronidazole, irrespective of NAP1/027 status. There was no significant association between early CDI and subsequent development of GI GVHD. This study demonstrates the high incidence of CDI early after allogeneic HSCT with wide diversity among infecting strains. Despite the high prevalence of NAP1/027, the disease is mild but relapses are common. No association was found between CDI and subsequent development of GI GVHD. CI - Copyright (c) 2014 American Society for Blood and Marrow Transplantation. Published by Elsevier Inc. All rights reserved. FAU - Kamboj, Mini AU - Kamboj M AD - Services of Infectious Diseases, Department of Medicine, Memorial Sloan Kettering Cancer Center, New York, New York; Department of Medicine, Weill Medical College of Cornell University, New York, New York. Electronic address: kambojm@mskcc.org. FAU - Xiao, Kun AU - Xiao K AD - Services of Infectious Diseases, Department of Medicine, Memorial Sloan Kettering Cancer Center, New York, New York. FAU - Kaltsas, Anna AU - Kaltsas A AD - Services of Infectious Diseases, Department of Medicine, Memorial Sloan Kettering Cancer Center, New York, New York; Department of Medicine, Weill Medical College of Cornell University, New York, New York. FAU - Huang, Yao-Ting AU - Huang YT AD - Services of Infectious Diseases, Department of Medicine, Memorial Sloan Kettering Cancer Center, New York, New York. FAU - Sun, Janet AU - Sun J AD - Services of Infectious Diseases, Department of Medicine, Memorial Sloan Kettering Cancer Center, New York, New York. FAU - Chung, Dick AU - Chung D AD - Services of Infectious Diseases, Department of Medicine, Memorial Sloan Kettering Cancer Center, New York, New York. FAU - Wu, Saliangi AU - Wu S AD - Department of Hematology, Queen Elizabeth Hospital, Hong Kong. FAU - Sheahan, Anna AU - Sheahan A AD - Services of Infectious Diseases, Department of Medicine, Memorial Sloan Kettering Cancer Center, New York, New York. FAU - Sepkowitz, Kent AU - Sepkowitz K AD - Services of Infectious Diseases, Department of Medicine, Memorial Sloan Kettering Cancer Center, New York, New York; Department of Medicine, Weill Medical College of Cornell University, New York, New York. FAU - Jakubowski, Ann A AU - Jakubowski AA AD - Department of Medicine, Weill Medical College of Cornell University, New York, New York; Adult Bone Marrow Transplant, Department of Medicine, Memorial Sloan Kettering Cancer Center, New York, New York. FAU - Papanicolaou, Genovefa AU - Papanicolaou G AD - Services of Infectious Diseases, Department of Medicine, Memorial Sloan Kettering Cancer Center, New York, New York; Department of Medicine, Weill Medical College of Cornell University, New York, New York. LA - eng GR - K23 AI083880/AI/NIAID NIH HHS/United States PT - Journal Article PT - Research Support, N.I.H., Extramural DEP - 20140625 PL - United States TA - Biol Blood Marrow Transplant JT - Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation JID - 9600628 RN - 0 (Anti-Infective Agents) RN - 0 (Myeloablative Agonists) RN - 140QMO216E (Metronidazole) SB - IM MH - Adolescent MH - Adult MH - Aged MH - Anti-Infective Agents/therapeutic use MH - Child MH - Child, Preschool MH - Clostridium Infections/*drug therapy/etiology/immunology/microbiology MH - Clostridium difficile/drug effects/*genetics/isolation & purification MH - Female MH - Gastrointestinal Tract/immunology/microbiology MH - Graft vs Host Disease/immunology/microbiology/*prevention & control MH - Hematologic Neoplasms/pathology/*therapy MH - *Hematopoietic Stem Cell Transplantation MH - Humans MH - Infant MH - Infant, Newborn MH - Male MH - Metronidazole/therapeutic use MH - Middle Aged MH - Myeloablative Agonists/adverse effects MH - Recurrence MH - Retrospective Studies MH - *Transplantation Conditioning MH - Transplantation, Homologous MH - Treatment Outcome OTO - NOTNLM OT - Allogeneic hematopoietic stem cell transplantation OT - Clostridium difficile infection OT - Immunocompromised host OT - NAP1/027 strain EDAT- 2014/06/29 06:00 MHDA- 2015/05/20 06:00 CRDT- 2014/06/29 06:00 PHST- 2014/03/25 [received] PHST- 2014/06/16 [accepted] PHST- 2014/06/25 [aheadofprint] AID - S1083-8791(14)00375-9 [pii] AID - 10.1016/j.bbmt.2014.06.025 [doi] PST - ppublish SO - Biol Blood Marrow Transplant. 2014 Oct;20(10):1626-33. doi: 10.1016/j.bbmt.2014.06.025. Epub 2014 Jun 25. PMID- 24972252 OWN - NLM STAT- MEDLINE DA - 20140912 DCOM- 20150519 IS - 1523-6536 (Electronic) IS - 1083-8791 (Linking) VI - 20 IP - 10 DP - 2014 Oct TI - Participation in clinical research: perspectives of adult patients and parents of pediatric patients undergoing hematopoietic stem cell transplantation. PG - 1604-11 LID - 10.1016/j.bbmt.2014.06.020 [doi] LID - S1083-8791(14)00370-X [pii] AB - Despite major improvements over the past several decades, many patients undergoing hematopoietic stem cell transplantations (HSCT) continue to suffer from significant treatment-related morbidity and mortality. Clinical research studies (trials) have been integral to advancing the standard of care in HSCT. However, 1 of the biggest challenges with clinical trials is the low participation rate. Although barriers to participation in cancer clinical trials have been previously explored, studies specific to HSCT are lacking. The current study was undertaken to examine the knowledge, attitudes, and perceptions of HSCT patients regarding clinical trials. As members of focus groups, participants responded to open-ended questions that assessed factors influencing decision-making about HSCT clinical trials. Suggestions for improvements in the recruitment process were also solicited among participants. Seventeen adult HSCT patients and 6 parents of pediatric HSCT patients participated in the study. The median age was 56 years (range, 18 to 70) and 44 years (range, 28 to 54) for adult patients and parents, respectively. Participants universally indicated that too much information was provided within the informed consents and they were intimidated by the medical and legal language. Despite the large amount of information provided to them at the time of study enrollment, the participants had limited knowledge retention and recall of study details. Nevertheless, participants reported overall positive experiences with clinical trial participation and many would readily choose to participate again. A common concern among participants was the uncertainty of study outcome and general lack of feedback about results at the end of the study. Participants suggested that investigators provide more condensed and easier to understand informed consents and follow-up of study findings. These findings could be used to help guide the development of improved consent documents and enhanced participation in research studies, thereby affecting the future design of HSCT research protocols. CI - Copyright (c) 2014 American Society for Blood and Marrow Transplantation. Published by Elsevier Inc. All rights reserved. FAU - Keusch, Florian AU - Keusch F AD - Institute for Social Research, University of Michigan, Ann Arbor, Michigan. FAU - Rao, Rohini AU - Rao R AD - Blood and Marrow Transplantation Program, University of Michigan, Ann Arbor, Michigan; Department of Pediatrics, University of Michigan, Ann Arbor, Michigan. FAU - Chang, Lawrence AU - Chang L AD - Blood and Marrow Transplantation Program, University of Michigan, Ann Arbor, Michigan; Department of Pediatrics, University of Michigan, Ann Arbor, Michigan. FAU - Lepkowski, James AU - Lepkowski J AD - Institute for Social Research, University of Michigan, Ann Arbor, Michigan. FAU - Reddy, Pavan AU - Reddy P AD - Blood and Marrow Transplantation Program, University of Michigan, Ann Arbor, Michigan; Department of Internal Medicine, University of Michigan, Ann Arbor, Michigan. FAU - Choi, Sung Won AU - Choi SW AD - Blood and Marrow Transplantation Program, University of Michigan, Ann Arbor, Michigan; Department of Pediatrics, University of Michigan, Ann Arbor, Michigan. Electronic address: sungchoi@med.umich.edu. LA - eng GR - 1K23AI091623/AI/NIAID NIH HHS/United States GR - K23 AI091623/AI/NIAID NIH HHS/United States PT - Journal Article PT - Research Support, N.I.H., Extramural PT - Research Support, Non-U.S. Gov't DEP - 20140624 PL - United States TA - Biol Blood Marrow Transplant JT - Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation JID - 9600628 SB - IM MH - Adolescent MH - Adult MH - Aged MH - Clinical Trials as Topic/*psychology MH - Female MH - Focus Groups MH - *Health Knowledge, Attitudes, Practice MH - Hematologic Diseases/psychology/therapy MH - *Hematopoietic Stem Cell Transplantation MH - Humans MH - Informed Consent/psychology MH - Male MH - Middle Aged MH - Patient Participation/*psychology MH - Transplantation, Homologous PMC - PMC4163094 MID - NIHMS617383 OID - NLM: NIHMS617383 OID - NLM: PMC4163094 OTO - NOTNLM OT - Bone marrow transplantation OT - Clinical trials OT - Focus groups OT - Hematopoietic stem cell transplantation OT - Patient research participation OT - Qualitative research study EDAT- 2014/06/28 06:00 MHDA- 2015/05/20 06:00 CRDT- 2014/06/28 06:00 PHST- 2014/05/06 [received] PHST- 2014/06/12 [accepted] PHST- 2014/06/24 [aheadofprint] AID - S1083-8791(14)00370-X [pii] AID - 10.1016/j.bbmt.2014.06.020 [doi] PST - ppublish SO - Biol Blood Marrow Transplant. 2014 Oct;20(10):1604-11. doi: 10.1016/j.bbmt.2014.06.020. Epub 2014 Jun 24. PMID- 24972251 OWN - NLM STAT- MEDLINE DA - 20140912 DCOM- 20150519 IS - 1523-6536 (Electronic) IS - 1083-8791 (Linking) VI - 20 IP - 10 DP - 2014 Oct TI - Anti-HLA antibodies other than against HLA-A, -B, -DRB1 adversely affect engraftment and nonrelapse mortality in HLA-mismatched single cord blood transplantation: possible implications of unrecognized donor-specific antibodies. PG - 1634-40 LID - 10.1016/j.bbmt.2014.06.024 [doi] LID - S1083-8791(14)00374-7 [pii] AB - The impact of anti-HLA antibodies, except for donor-specific anti-HLA-A, -B, -DRB1 antibodies, on engraftment was retrospectively evaluated in 175 single cord blood transplantations (CBT). Patients and donors had been typed at HLA-A, -B, and -DRB1 antigens, and anti-HLA antibodies had been screened before transplantation to avoid the use of cord blood (CB) units with corresponding antigens. The median age was 59 (range, 17 to 74) years. Overall, 61% were male, 89% had high-risk disease status, 77% received myeloablative conditioning regimens, and over 80% were heavily transfused patients. Sixty-nine of the 175 (39.4%) were positive for anti-HLA antibodies. Thirty-nine patients had antibodies only against HLA-A, -B, or -DRB1, 13 had antibodies only against HLA-C, -DP, -DQ, or -DRB3/4/5, and 17 had antibodies both against HLA-C, -DP, -DQ, or -DRB3/4/5 and against HLA-A, -B, or -DRB1. Because CB units had not been typed at HLA-C, -DP, -DQ, or -DRB3/4/5, it was possible that antibodies against them were unrecognized donor-specific antibodies. Patients with antibodies only against HLA-A, -B, or -DRB1 showed comparable neutrophil engraftment rates to those without antibodies (89.7% versus 83%, P = .65), whereas patients having antibodies against C, DP, DQ, or -DRB3/4/5 showed lower engraftment rate (66.7%, P = .12), which became statistically significant in a subgroup of HLA-mismatched donor-recipient pairs (50%, P = .01). Our results demonstrated that the presence of donor nonspecific anti-HLA-A, -B, -DRB1 antibodies had no significant influence on engraftment, whereas anti-HLA-C, -DP, -DQ, or -DRB3/4/5 antibodies adversely affect engraftment, possibly because of unrecognized donor-specific anti-HLA antibodies against them, especially in HLA-mismatched CBT. CI - Copyright (c) 2014 American Society for Blood and Marrow Transplantation. Published by Elsevier Inc. All rights reserved. FAU - Yamamoto, Hisashi AU - Yamamoto H AD - Department of Hematology, Toranomon Hospital, Tokyo, Japan. FAU - Uchida, Naoyuki AU - Uchida N AD - Department of Hematology, Toranomon Hospital, Tokyo, Japan; Okinaka Memorial Institute for Medical Research, Tokyo, Japan. Electronic address: nuchida@toranomon.gr.jp. FAU - Matsuno, Naofumi AU - Matsuno N AD - Department of Hematology, Toranomon Hospital, Tokyo, Japan. FAU - Ota, Hikari AU - Ota H AD - Department of Hematology, Toranomon Hospital, Tokyo, Japan; Department of Transfusion Medicine, Toranomon Hospital, Tokyo, Japan. FAU - Kageyama, Kosei AU - Kageyama K AD - Department of Hematology, Toranomon Hospital, Tokyo, Japan. FAU - Wada, Sachie AU - Wada S AD - Department of Hematology, Toranomon Hospital, Tokyo, Japan. FAU - Kaji, Daisuke AU - Kaji D AD - Department of Hematology, Toranomon Hospital, Tokyo, Japan. FAU - Nishida, Aya AU - Nishida A AD - Department of Hematology, Toranomon Hospital, Tokyo, Japan. FAU - Ishiwata, Kazuya AU - Ishiwata K AD - Department of Hematology, Toranomon Hospital, Tokyo, Japan. FAU - Takagi, Shinsuke AU - Takagi S AD - Department of Hematology, Toranomon Hospital, Tokyo, Japan. FAU - Tsuji, Masanori AU - Tsuji M AD - Department of Hematology, Toranomon Hospital, Tokyo, Japan. FAU - Asano-Mori, Yuki AU - Asano-Mori Y AD - Department of Hematology, Toranomon Hospital, Tokyo, Japan. FAU - Yamamoto, Go AU - Yamamoto G AD - Department of Hematology, Toranomon Hospital, Tokyo, Japan. FAU - Izutsu, Koji AU - Izutsu K AD - Department of Hematology, Toranomon Hospital, Tokyo, Japan; Okinaka Memorial Institute for Medical Research, Tokyo, Japan. FAU - Masuoka, Kazuhiro AU - Masuoka K AD - Department of Hematology, Toranomon Hospital, Tokyo, Japan. FAU - Wake, Atsushi AU - Wake A AD - Department of Hematology, Toranomon Hospital, Tokyo, Japan. FAU - Yoneyama, Akiko AU - Yoneyama A AD - Department of Infectious Diseases, Toranomon Hospital, Tokyo, Japan. FAU - Makino, Shigeyoshi AU - Makino S AD - Department of Transfusion Medicine, Toranomon Hospital, Tokyo, Japan. FAU - Taniguchi, Shuichi AU - Taniguchi S AD - Department of Hematology, Toranomon Hospital, Tokyo, Japan; Okinaka Memorial Institute for Medical Research, Tokyo, Japan. LA - eng PT - Journal Article DEP - 20140624 PL - United States TA - Biol Blood Marrow Transplant JT - Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation JID - 9600628 RN - 0 (HLA Antigens) RN - 0 (Isoantibodies) RN - 0 (Myeloablative Agonists) SB - IM MH - Adolescent MH - Adult MH - Aged MH - Antibody Specificity MH - *Cord Blood Stem Cell Transplantation MH - Female MH - *Graft Survival MH - HLA Antigens/classification/*immunology MH - Hematologic Neoplasms/immunology/mortality/*therapy MH - Histocompatibility Testing MH - Humans MH - Isoantibodies/*biosynthesis/immunology MH - Male MH - Middle Aged MH - Myeloablative Agonists/therapeutic use MH - Prognosis MH - Recurrence MH - Retrospective Studies MH - Survival Analysis MH - Tissue Donors MH - *Transplantation Conditioning MH - Transplantation, Homologous OTO - NOTNLM OT - Anti-HLA antibodies OT - Cord blood transplantation OT - Nondonor specific EDAT- 2014/06/28 06:00 MHDA- 2015/05/20 06:00 CRDT- 2014/06/28 06:00 PHST- 2014/04/17 [received] PHST- 2014/06/17 [accepted] PHST- 2014/06/24 [aheadofprint] AID - S1083-8791(14)00374-7 [pii] AID - 10.1016/j.bbmt.2014.06.024 [doi] PST - ppublish SO - Biol Blood Marrow Transplant. 2014 Oct;20(10):1634-40. doi: 10.1016/j.bbmt.2014.06.024. Epub 2014 Jun 24. PMID- 24963044 OWN - NLM STAT- MEDLINE DA - 20140808 DCOM- 20150429 IS - 1528-0020 (Electronic) IS - 0006-4971 (Linking) VI - 124 IP - 6 DP - 2014 Aug 7 TI - The ethics of a proposed study of hematopoietic stem cell transplant for children with "less severe" sickle cell disease. PG - 861-6 LID - 10.1182/blood-2014-05-575209 [doi] AB - Hematopoietic stem cell transplant (HSCT) is the only cure for sickle cell disease (SCD). HSCT using an HLA-identical sibling donor is currently an acceptable treatment option for children with severe SCD, with expected HSCT survival >95% and event-free survival >85%. HSCT for children with less severe SCD (children who have not yet suffered overt disease complications or only had mild problems) is controversial. It is important to consider the ethical issues of a proposed study comparing HLA-identical sibling HSCT to best supportive care for children with less severe SCD. In evaluating the principles of nonmaleficence, respect for individual autonomy, and justice, we conclude that a study of HLA-identical sibling HSCT for all children with SCD, particularly hemoglobin SS and Sbeta(0)-thalassemia disease, is ethically sound. Future work should explore the implementation of a large trial to help determine whether HSCT is a beneficial treatment of children with less severe SCD. CI - (c) 2014 by The American Society of Hematology. FAU - Nickel, Robert S AU - Nickel RS AUID- ORCID: http://orcid.org/0000-0003-4277-7523 AD - Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta, Emory University, Atlanta, GA; and. FAU - Hendrickson, Jeanne E AU - Hendrickson JE AD - Laboratory Medicine, Yale University, New Haven, CT. FAU - Haight, Ann E AU - Haight AE AD - Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta, Emory University, Atlanta, GA; and. LA - eng PT - Journal Article DEP - 20140624 PL - United States TA - Blood JT - Blood JID - 7603509 RN - 0 (HLA Antigens) SB - AIM SB - IM MH - Anemia, Sickle Cell/complications/mortality/*therapy MH - Child MH - Clinical Trials as Topic/ethics MH - Graft vs Host Disease/etiology MH - HLA Antigens MH - Hematopoietic Stem Cell Transplantation/adverse effects/*ethics MH - Humans MH - Living Donors/ethics MH - Risk Factors MH - Siblings EDAT- 2014/06/26 06:00 MHDA- 2015/04/30 06:00 CRDT- 2014/06/26 06:00 PHST- 2014/06/24 [aheadofprint] AID - blood-2014-05-575209 [pii] AID - 10.1182/blood-2014-05-575209 [doi] PST - ppublish SO - Blood. 2014 Aug 7;124(6):861-6. doi: 10.1182/blood-2014-05-575209. Epub 2014 Jun 24. PMID- 24963042 OWN - NLM STAT- MEDLINE DA - 20140815 DCOM- 20150427 LR - 20150319 IS - 1528-0020 (Electronic) IS - 0006-4971 (Linking) VI - 124 IP - 7 DP - 2014 Aug 14 TI - MPD-RC 101 prospective study of reduced-intensity allogeneic hematopoietic stem cell transplantation in patients with myelofibrosis. PG - 1183-91 LID - 10.1182/blood-2014-04-572545 [doi] AB - From 2007 to 2011, 66 patients with primary myelofibrosis or myelofibrosis (MF) preceded by essential thrombocythemia or polycythemia vera were enrolled into a prospective phase 2 clinical trial of reduced-intensity allogeneic hematopoietic stem cell transplantation (AHSCT), Myeloproliferative Disorder Research Consortium 101 trial. The study included patients with sibling donors (n = 32) receiving fludarabine/melphalan (FluMel) as a preparative regimen and patients with unrelated donors (n = 34) receiving conditioning with FluMel plus anti-thymocyte globulin (ATG). Patient characteristics in the 2 cohorts were similar. Engraftment occurred in 97% of siblings and 76% of unrelated transplants, whereas secondary graft failure occurred in 3% and 12%, respectively. With a median follow-up of 25 months for patients alive, the overall survival (OS) was 75% in the sibling group (median not reached) and 32% in the unrelated group (median OS: 6 months, 95% confidence interval [CI]: 3, 25) (hazard ratio 3.9, 95% CI: 1.8,8.9) (P < .001). Nonrelapse mortality was 22% in sibling and 59% in unrelated AHSCT. Survival correlated with type of donor, but not with the degree of histocompatibility match, age, or JAK2(V617F) status. In patients with MF with sibling donors, AHSCT is an effective therapy, whereas AHSCT from unrelated donors with FluMel/ATG conditioning led to a high rate of graft failure and limited survival. This trial was registered at www.clinicaltrials.gov as #NCT00572897. CI - (c) 2014 by The American Society of Hematology. FAU - Rondelli, Damiano AU - Rondelli D AUID- ORCID: http://orcid.org/0000-0001-5243-401X AD - University of Illinois Hospital & Health Sciences System and University of Illinois Cancer Center, Chicago, IL; Myeloproliferative Disorders Research Consortium, Mount Sinai School of Medicine, New York, NY; FAU - Goldberg, Judith D AU - Goldberg JD AUID- ORCID: http://orcid.org/0000-0003-3758-6976 AD - Myeloproliferative Disorders Research Consortium, Mount Sinai School of Medicine, New York, NY; New York University School of Medicine, New York, NY; FAU - Isola, Luis AU - Isola L AD - Myeloproliferative Disorders Research Consortium, Mount Sinai School of Medicine, New York, NY; Mount Sinai Medical Center, New York, NY; FAU - Price, Leah S AU - Price LS AD - Myeloproliferative Disorders Research Consortium, Mount Sinai School of Medicine, New York, NY; New York University School of Medicine, New York, NY; FAU - Shore, Tsiporah B AU - Shore TB AD - Myeloproliferative Disorders Research Consortium, Mount Sinai School of Medicine, New York, NY; Weill Cornell Medical College, New York, NY; FAU - Boyer, Michael AU - Boyer M AUID- ORCID: http://orcid.org/0000-0002-0452-2987 AD - University of Utah School of Medicine, Salt Lake City, UT; FAU - Bacigalupo, Andrea AU - Bacigalupo A AD - Myeloproliferative Disorders Research Consortium, Mount Sinai School of Medicine, New York, NY; Ospedale San Martino, Genoa, Italy; FAU - Rambaldi, Alessandro AU - Rambaldi A AD - Myeloproliferative Disorders Research Consortium, Mount Sinai School of Medicine, New York, NY; Azienda Ospedaliera Papa Giovanni XXIII, Bergamo, Italy; FAU - Scarano, Marco AU - Scarano M AD - Myeloproliferative Disorders Research Consortium, Mount Sinai School of Medicine, New York, NY; Foundation Mario Negri Sud, S. Maria Imbaro, Italy; FAU - Klisovic, Rebecca B AU - Klisovic RB AD - Myeloproliferative Disorders Research Consortium, Mount Sinai School of Medicine, New York, NY; The Ohio State University Comprehensive Cancer Center, Columbus, OH; FAU - Gupta, Vikas AU - Gupta V AD - Myeloproliferative Disorders Research Consortium, Mount Sinai School of Medicine, New York, NY; Princess Margaret Hospital Cancer Center, Toronto, ON, Canada; FAU - Andreasson, Bjorn AU - Andreasson B AD - Myeloproliferative Disorders Research Consortium, Mount Sinai School of Medicine, New York, NY; Nephro-Urology Hospital Organization, Uddevalla, Sweden; FAU - Mascarenhas, John AU - Mascarenhas J AUID- ORCID: http://orcid.org/0000-0002-8400-0483 AD - Myeloproliferative Disorders Research Consortium, Mount Sinai School of Medicine, New York, NY; Mount Sinai Medical Center, New York, NY; FAU - Wetzler, Meir AU - Wetzler M AD - Myeloproliferative Disorders Research Consortium, Mount Sinai School of Medicine, New York, NY; Roswell Park Cancer Institute, Buffalo, NY; FAU - Vannucchi, Alessandro M AU - Vannucchi AM AD - Myeloproliferative Disorders Research Consortium, Mount Sinai School of Medicine, New York, NY; University of Florence, Florence, Italy; FAU - Prchal, Josef T AU - Prchal JT AD - Myeloproliferative Disorders Research Consortium, Mount Sinai School of Medicine, New York, NY; University of Utah School of Medicine, Salt Lake City, UT; FAU - Najfeld, Vesna AU - Najfeld V AD - Myeloproliferative Disorders Research Consortium, Mount Sinai School of Medicine, New York, NY; Mount Sinai Medical Center, New York, NY; FAU - Orazi, Attilio AU - Orazi A AD - Myeloproliferative Disorders Research Consortium, Mount Sinai School of Medicine, New York, NY; Weill Cornell Medical College, New York, NY; FAU - Weinberg, Rona S AU - Weinberg RS AD - Myeloproliferative Disorders Research Consortium, Mount Sinai School of Medicine, New York, NY; New York Blood Center, New York, NY; and. FAU - Miller, Crystal AU - Miller C AD - Myeloproliferative Disorders Research Consortium, Mount Sinai School of Medicine, New York, NY; Mount Sinai Medical Center, New York, NY; FAU - Barosi, Giovanni AU - Barosi G AD - Myeloproliferative Disorders Research Consortium, Mount Sinai School of Medicine, New York, NY; Istituto Di Ricovero e Cura a Carattere Scientifico Policlinico S. Matteo Foundation, Pavia, Italy. FAU - Silverman, Lewis R AU - Silverman LR AD - Myeloproliferative Disorders Research Consortium, Mount Sinai School of Medicine, New York, NY; Mount Sinai Medical Center, New York, NY; FAU - Prosperini, Giuseppe AU - Prosperini G AD - Myeloproliferative Disorders Research Consortium, Mount Sinai School of Medicine, New York, NY; Foundation Mario Negri Sud, S. Maria Imbaro, Italy; FAU - Marchioli, Roberto AU - Marchioli R AD - Myeloproliferative Disorders Research Consortium, Mount Sinai School of Medicine, New York, NY; Foundation Mario Negri Sud, S. Maria Imbaro, Italy; FAU - Hoffman, Ronald AU - Hoffman R AD - Myeloproliferative Disorders Research Consortium, Mount Sinai School of Medicine, New York, NY; Mount Sinai Medical Center, New York, NY; LA - eng SI - ClinicalTrials.gov/NCT00572897 GR - 1 P01 CA108671-01A2/CA/NCI NIH HHS/United States GR - P01 CA108671/CA/NCI NIH HHS/United States PT - Clinical Trial, Phase II PT - Journal Article PT - Multicenter Study PT - Research Support, N.I.H., Extramural DEP - 20140624 PL - United States TA - Blood JT - Blood JID - 7603509 RN - 0 (Antilymphocyte Serum) RN - EC 2.7.10.2 (JAK2 protein, human) RN - EC 2.7.10.2 (Janus Kinase 2) RN - FA2DM6879K (Vidarabine) RN - P2K93U8740 (fludarabine) RN - Q41OR9510P (Melphalan) SB - AIM SB - IM MH - Adult MH - Aged MH - Analysis of Variance MH - Antilymphocyte Serum/therapeutic use MH - Blood Donors MH - Female MH - Follow-Up Studies MH - Graft vs Host Disease/etiology/prevention & control MH - Hematopoietic Stem Cell Transplantation/adverse effects/*methods MH - Histocompatibility MH - Humans MH - Janus Kinase 2/genetics MH - Kaplan-Meier Estimate MH - Male MH - Melphalan/therapeutic use MH - Middle Aged MH - Mutation MH - Primary Myelofibrosis/genetics/*therapy MH - Prospective Studies MH - Siblings MH - Transplantation Conditioning/methods MH - Transplantation, Homologous MH - Treatment Outcome MH - Unrelated Donors MH - Vidarabine/analogs & derivatives/therapeutic use PMC - PMC4133490 OID - NLM: PMC4133490 EDAT- 2014/06/26 06:00 MHDA- 2015/04/29 06:00 CRDT- 2014/06/26 06:00 PHST- 2014/06/24 [aheadofprint] AID - blood-2014-04-572545 [pii] AID - 10.1182/blood-2014-04-572545 [doi] PST - ppublish SO - Blood. 2014 Aug 14;124(7):1183-91. doi: 10.1182/blood-2014-04-572545. Epub 2014 Jun 24. PMID- 24960628 OWN - NLM STAT- MEDLINE DA - 20140912 DCOM- 20150519 IS - 1523-6536 (Electronic) IS - 1083-8791 (Linking) VI - 20 IP - 10 DP - 2014 Oct TI - Factors affecting the outcome of related allogeneic hematopoietic cell transplantation in patients with Fanconi Anemia. PG - 1599-603 LID - 10.1016/j.bbmt.2014.06.016 [doi] LID - S1083-8791(14)00366-8 [pii] AB - Hematopoietic cell transplantation (HCT) can cure bone marrow failure in patients with Fanconi Anemia (FA), and it is generally accepted that these patients should receive low-intensity conditioning because of the underlying DNA repair defect in their cells. Outcomes for recipients of matched related HCT have generally been favorable, but only a few studies have scrutinized the factors that may affect the eventual outcome of these patients. This retrospective analysis of 94 pediatric patients with FA who underwent related HCT at King Faisal Specialist Hospital & Research Center was carried out to attempt to identify factors that may affect outcome. Results showed overall survival (OS) probabilities of 92.5%, 89%, and 86% at 1, 5, and 10 years, respectively. In univariate analysis, use of higher dose cyclophosphamide (CY) (60 mg/kg) conditioning was associated with a better 10-year OS than lower dose CY (20 mg/kg) conditioning (91% versus 82%, respectively; P = .035), and use of radiation-containing regimens was associated with a significantly lower 10-year OS than nonradiation regimens (76% versus 91%, respectively; P = .005). Of the 4 regimens used in this study, the fludarabine-based regimen was associated with the highest survival (95.2%; P = .034). The use of the higher dose CY (60 mg/kg) was associated with a significantly increased incidence of hemorrhagic cystitis (HC) (20% versus 5.6% respectively; P = .049). Three patients (3%) developed squamous cell carcinoma (2 oropharyngeal and 1 genitourinary), at 9.4, 5.4, and 13.3 years after HCT; 2 of them had radiation-containing conditioning. In conclusion, our data suggest that although using a higher dose CY (60 mg/kg) conditioning regimen may be associated with better survival, it is also associated with a significantly increased risk of HC. The addition of fludarabine to the low-dose CY (20 mg/kg) is associated with the best survival. On the other hand, radiation-containing regimens are associated with significantly lower survival. CI - Copyright (c) 2014 American Society for Blood and Marrow Transplantation. Published by Elsevier Inc. All rights reserved. FAU - Ayas, Mouhab AU - Ayas M AD - Department of Pediatric Hematology Oncology, King Faisal Specialist Hospital and Research Center, Riyadh, Saudi Arabia. Electronic address: mouhab@kfshrc.edu.sa. FAU - Siddiqui, Khawar AU - Siddiqui K AD - Department of Pediatric Hematology Oncology, King Faisal Specialist Hospital and Research Center, Riyadh, Saudi Arabia. FAU - Al-Jefri, Abdullah AU - Al-Jefri A AD - Department of Pediatric Hematology Oncology, King Faisal Specialist Hospital and Research Center, Riyadh, Saudi Arabia. FAU - El-Solh, Hassan AU - El-Solh H AD - Department of Pediatric Hematology Oncology, King Faisal Specialist Hospital and Research Center, Riyadh, Saudi Arabia. FAU - Al-Ahmari, Ali AU - Al-Ahmari A AD - Department of Pediatric Hematology Oncology, King Faisal Specialist Hospital and Research Center, Riyadh, Saudi Arabia. FAU - Khairy, Ashraf AU - Khairy A AD - Department of Pediatric Hematology Oncology, King Faisal Specialist Hospital and Research Center, Riyadh, Saudi Arabia. FAU - Markiz, Samer AU - Markiz S AD - Department of Pediatric Hematology Oncology, King Faisal Specialist Hospital and Research Center, Riyadh, Saudi Arabia. FAU - Shahin, Hasan AU - Shahin H AD - Department of Pediatric Hematology Oncology, King Faisal Specialist Hospital and Research Center, Riyadh, Saudi Arabia. FAU - Al-Musa, Abdulrahman AU - Al-Musa A AD - Department of Pediatric Hematology Oncology, King Faisal Specialist Hospital and Research Center, Riyadh, Saudi Arabia. FAU - Al-Seraihy, Amal AU - Al-Seraihy A AD - Department of Pediatric Hematology Oncology, King Faisal Specialist Hospital and Research Center, Riyadh, Saudi Arabia. LA - eng PT - Journal Article DEP - 20140621 PL - United States TA - Biol Blood Marrow Transplant JT - Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation JID - 9600628 RN - 0 (Myeloablative Agonists) RN - 8N3DW7272P (Cyclophosphamide) RN - FA2DM6879K (Vidarabine) RN - P2K93U8740 (fludarabine) SB - IM MH - Adolescent MH - Carcinoma, Squamous Cell/chemically induced/immunology/mortality/*pathology MH - Child MH - Child, Preschool MH - Cyclophosphamide/adverse effects MH - Cystitis/chemically induced/immunology/mortality/*pathology MH - Fanconi Anemia/immunology/mortality/pathology/*therapy MH - Female MH - Gamma Rays/adverse effects MH - *Hematopoietic Stem Cell Transplantation MH - Hemorrhage/chemically induced/immunology/mortality/*pathology MH - Histocompatibility Testing MH - Humans MH - Infant MH - Male MH - Myeloablative Agonists/adverse effects MH - Oropharyngeal Neoplasms/chemically induced/immunology/mortality/*pathology MH - Retrospective Studies MH - Siblings MH - Survival Analysis MH - Transplantation Conditioning/*methods MH - Transplantation, Homologous MH - Unrelated Donors MH - Vidarabine/adverse effects/analogs & derivatives OTO - NOTNLM OT - Cyclophosphamide OT - Fanconi anemia OT - Radiation OT - Stem cell transplantation EDAT- 2014/06/25 06:00 MHDA- 2015/05/20 06:00 CRDT- 2014/06/25 06:00 PHST- 2014/03/11 [received] PHST- 2014/06/11 [accepted] PHST- 2014/06/21 [aheadofprint] AID - S1083-8791(14)00366-8 [pii] AID - 10.1016/j.bbmt.2014.06.016 [doi] PST - ppublish SO - Biol Blood Marrow Transplant. 2014 Oct;20(10):1599-603. doi: 10.1016/j.bbmt.2014.06.016. Epub 2014 Jun 21. PMID- 24957143 OWN - NLM STAT- MEDLINE DA - 20140808 DCOM- 20150429 LR - 20150501 IS - 1528-0020 (Electronic) IS - 0006-4971 (Linking) VI - 124 IP - 6 DP - 2014 Aug 7 TI - Trends in survival of patients with primary plasma cell leukemia: a population-based analysis. PG - 907-12 LID - 10.1182/blood-2014-03-565051 [doi] AB - Primary plasma cell leukemia (pPCL) is a rare malignancy with an aggressive course and poor outcome. There has been significant improvement in the survival of multiple myeloma patients over the past decade as a result of incorporating autologous stem cell transplantation (ASCT) and novel agents into treatment regimens. However, it is unknown whether these therapies have had a similar impact on the survival of patients with pPCL. We conducted an analysis of the Surveillance, Epidemiology, and End Results database to evaluate the trends in survival of 445 patients with pPCL between 1973 and 2009. The widespread availability of ASCT and use of novel agents in the upfront setting of multiple myeloma and pPCL began after 1995 and 2006, respectively. The median overall survival based on periods of diagnosis were 5, 6, 4, and 12 months for those diagnosed during 1973-1995, 1996-2000, 2001-2005, and 2006-2009, respectively (P = .001). Thus, the current study confirms the recent survival improvement in pPCL within a large US population that may be associated with the use of better therapeutic strategies. CI - (c) 2014 by The American Society of Hematology. FAU - Gonsalves, Wilson I AU - Gonsalves WI AD - Division of Hematology. FAU - Rajkumar, S Vincent AU - Rajkumar SV AUID- ORCID: http://orcid.org/0000-0002-5862-1833 AD - Division of Hematology, Department of Laboratory Medicine and Pathology, Mayo Clinic, Rochester, MN. FAU - Go, Ronald S AU - Go RS AD - Division of Hematology. FAU - Dispenzieri, Angela AU - Dispenzieri A AD - Division of Hematology. FAU - Gupta, Vinay AU - Gupta V AD - Division of Hematology. FAU - Singh, Preet P AU - Singh PP AD - Division of Hematology. FAU - Buadi, Francis K AU - Buadi FK AD - Division of Hematology. FAU - Lacy, Martha Q AU - Lacy MQ AD - Division of Hematology. FAU - Kapoor, Prashant AU - Kapoor P AD - Division of Hematology. FAU - Dingli, David AU - Dingli D AD - Division of Hematology. FAU - Lust, John A AU - Lust JA AD - Division of Hematology, Department of Laboratory Medicine and Pathology, Mayo Clinic, Rochester, MN. FAU - Zeldenrust, Steven R AU - Zeldenrust SR AD - Division of Hematology. FAU - Hayman, Suzanne R AU - Hayman SR AD - Division of Hematology. FAU - Kyle, Robert A AU - Kyle RA AD - Division of Hematology. FAU - Gertz, Morie A AU - Gertz MA AD - Division of Hematology. FAU - Kumar, Shaji K AU - Kumar SK AD - Division of Hematology. LA - eng GR - CA100707/CA/NCI NIH HHS/United States GR - CA107476/CA/NCI NIH HHS/United States GR - CA168762/CA/NCI NIH HHS/United States GR - CA62242/CA/NCI NIH HHS/United States GR - UL1 TR000135/TR/NCATS NIH HHS/United States GR - UL1 TR000135/TR/NCATS NIH HHS/United States PT - Journal Article PT - Research Support, N.I.H., Extramural PT - Research Support, Non-U.S. Gov't DEP - 20140623 PL - United States TA - Blood JT - Blood JID - 7603509 SB - AIM SB - IM MH - Adolescent MH - Adult MH - Aged MH - Aged, 80 and over MH - Child MH - Female MH - Humans MH - Kaplan-Meier Estimate MH - Leukemia, Plasma Cell/drug therapy/*mortality/therapy MH - Male MH - Middle Aged MH - Multiple Myeloma/drug therapy/mortality/therapy MH - Prognosis MH - SEER Program/statistics & numerical data MH - Stem Cell Transplantation MH - Survival Analysis MH - Time Factors MH - Transplantation, Autologous MH - United States/epidemiology MH - Young Adult PMC - PMC4126330 OID - NLM: PMC4126330 [Available on 08/07/15] EDAT- 2014/06/25 06:00 MHDA- 2015/04/30 06:00 CRDT- 2014/06/25 06:00 PMCR- 2015/08/07 00:00 PHST- 2014/06/23 [aheadofprint] AID - blood-2014-03-565051 [pii] AID - 10.1182/blood-2014-03-565051 [doi] PST - ppublish SO - Blood. 2014 Aug 7;124(6):907-12. doi: 10.1182/blood-2014-03-565051. Epub 2014 Jun 23. PMID- 24954547 OWN - NLM STAT- MEDLINE DA - 20140912 DCOM- 20150519 LR - 20150416 IS - 1523-6536 (Electronic) IS - 1083-8791 (Linking) VI - 20 IP - 10 DP - 2014 Oct TI - Parametric response mapping as an indicator of bronchiolitis obliterans syndrome after hematopoietic stem cell transplantation. PG - 1592-8 LID - 10.1016/j.bbmt.2014.06.014 [doi] LID - S1083-8791(14)00364-4 [pii] AB - The management of bronchiolitis obliterans syndrome (BOS) after hematopoietic cell transplantation presents many challenges, both diagnostically and therapeutically. We developed a computed tomography (CT) voxel-wise methodology termed parametric response mapping (PRM) that quantifies normal parenchyma, functional small airway disease (PRM(fSAD)), emphysema, and parenchymal disease as relative lung volumes. We now investigate the use of PRM as an imaging biomarker in the diagnosis of BOS. PRM was applied to CT data from 4 patient cohorts: acute infection (n = 11), BOS at onset (n = 34), BOS plus infection (n = 9), and age-matched, nontransplant control subjects (n = 23). Pulmonary function tests and bronchoalveolar lavage were used for group classification. Mean values for PRM(fSAD) were significantly greater in patients with BOS (38% +/- 2%) when compared with those with infection alone (17% +/- 4%, P < .0001) and age-matched control subjects (8.4% +/- 1%, P < .0001). Patients with BOS had similar PRM(fSAD) profiles, whether a concurrent infection was present or not. An optimal cut-point for PRM(fSAD) of 28% of the total lung volume was identified, with values >28% highly indicative of BOS occurrence. PRM may provide a major advance in our ability to identify the small airway obstruction that characterizes BOS, even in the presence of concurrent infection. CI - Copyright (c) 2014 American Society for Blood and Marrow Transplantation. Published by Elsevier Inc. All rights reserved. FAU - Galban, Craig J AU - Galban CJ AD - Department of Radiology, University of Michigan, Ann Arbor, Michigan. Electronic address: cgalban@med.umich.edu. FAU - Boes, Jennifer L AU - Boes JL AD - Department of Radiology, University of Michigan, Ann Arbor, Michigan. FAU - Bule, Maria AU - Bule M AD - Department of Radiology, University of Michigan, Ann Arbor, Michigan. FAU - Kitko, Carrie L AU - Kitko CL AD - Blood and Marrow Transplant Program, University of Michigan, Ann Arbor, Michigan; Department of Pediatrics and Communicable Diseases, University of Michigan, Ann Arbor, Michigan. FAU - Couriel, Daniel R AU - Couriel DR AD - Blood and Marrow Transplant Program, University of Michigan, Ann Arbor, Michigan; Department of Internal Medicine, University of Michigan, Ann Arbor, Michigan. FAU - Johnson, Timothy D AU - Johnson TD AD - Department of Biostatistics, University of Michigan, Ann Arbor, Michigan. FAU - Lama, Vihba AU - Lama V AD - Department of Internal Medicine, University of Michigan, Ann Arbor, Michigan. FAU - Telenga, Eef D AU - Telenga ED AD - Department of Pulmonary Diseases, University of Groningen, University Medical Center Groningen, Groningen, the Netherlands. FAU - van den Berge, Maarten AU - van den Berge M AD - Department of Pulmonary Diseases, University of Groningen, University Medical Center Groningen, Groningen, the Netherlands. FAU - Rehemtulla, Alnawaz AU - Rehemtulla A AD - Department of Radiation Oncology, University of Michigan, Ann Arbor, Michigan. FAU - Kazerooni, Ella A AU - Kazerooni EA AD - Department of Radiology, University of Michigan, Ann Arbor, Michigan. FAU - Ponkowski, Michael J AU - Ponkowski MJ AD - Blood and Marrow Transplant Program, University of Michigan, Ann Arbor, Michigan. FAU - Ross, Brian D AU - Ross BD AD - Department of Radiology, University of Michigan, Ann Arbor, Michigan. FAU - Yanik, Gregory A AU - Yanik GA AD - Blood and Marrow Transplant Program, University of Michigan, Ann Arbor, Michigan; Department of Pediatrics and Communicable Diseases, University of Michigan, Ann Arbor, Michigan; Department of Internal Medicine, University of Michigan, Ann Arbor, Michigan. LA - eng GR - P01 CA085878/CA/NCI NIH HHS/United States GR - P01CA085878/CA/NCI NIH HHS/United States GR - P50 CA093990/CA/NCI NIH HHS/United States GR - P50CA93990/CA/NCI NIH HHS/United States GR - R01 HL122438/HL/NHLBI NIH HHS/United States GR - T32 EB005172/EB/NIBIB NIH HHS/United States GR - T32EB005172/EB/NIBIB NIH HHS/United States PT - Clinical Trial PT - Journal Article PT - Research Support, N.I.H., Extramural DEP - 20140618 PL - United States TA - Biol Blood Marrow Transplant JT - Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation JID - 9600628 RN - 0 (Myeloablative Agonists) SB - IM MH - Adolescent MH - Adult MH - Aged MH - Bronchiolitis Obliterans/etiology/immunology/microbiology/*radiography MH - Bronchoalveolar Lavage Fluid/microbiology MH - Case-Control Studies MH - Child MH - Female MH - Hematologic Neoplasms/complications/immunology/microbiology/*radiography MH - *Hematopoietic Stem Cell Transplantation MH - Humans MH - Lung/immunology/microbiology/*radiography MH - Male MH - Middle Aged MH - Myeloablative Agonists/therapeutic use MH - Prospective Studies MH - Respiratory Function Tests MH - Syndrome MH - Tomography, X-Ray Computed/*methods MH - Transplantation Conditioning/*methods MH - Transplantation, Homologous PMC - PMC4163140 MID - NIHMS606721 OID - NLM: NIHMS606721 [Available on 10/01/15] OID - NLM: PMC4163140 [Available on 10/01/15] OTO - NOTNLM OT - Bronchiolitis obliterans syndrome OT - Pulmonary OT - Transplantation EDAT- 2014/06/24 06:00 MHDA- 2015/05/20 06:00 CRDT- 2014/06/24 06:00 PMCR- 2015/10/01 00:00 PHST- 2014/03/20 [received] PHST- 2014/06/10 [accepted] PHST- 2014/06/18 [aheadofprint] AID - S1083-8791(14)00364-4 [pii] AID - 10.1016/j.bbmt.2014.06.014 [doi] PST - ppublish SO - Biol Blood Marrow Transplant. 2014 Oct;20(10):1592-8. doi: 10.1016/j.bbmt.2014.06.014. Epub 2014 Jun 18. PMID- 24953019 OWN - NLM STAT- MEDLINE DA - 20140912 DCOM- 20150519 IS - 1523-6536 (Electronic) IS - 1083-8791 (Linking) VI - 20 IP - 10 DP - 2014 Oct TI - Fibrin glue therapy for severe hemorrhagic cystitis after allogeneic hematopoietic stem cell transplantation. PG - 1612-7 LID - 10.1016/j.bbmt.2014.06.018 [doi] LID - S1083-8791(14)00368-1 [pii] AB - Hemorrhagic cystitis (HC) occurring after allogeneic transplantation significantly affects quality of life and, in some cases, becomes intractable, increasing the risk of death. To date, its therapy is not established. We used the hemostatic agent fibrin glue (FG) to treat 35 patients with refractory post-transplantation HC. Of 322 adult patients undergoing an allogeneic transplantation for hematological malignancy, 35 developed grade >/= 2 HC refractory to conventional therapy and were treated with FG, diffusely sprayed on bleeding mucosa by an endoscopic applicator. The cumulative incidence of pain discontinuation and complete remission, defined as regression of all symptoms and absence of hematuria, was 100% at 7 days and 83% +/- 7%, respectively, at 50 days from FG application. The 6-month probability of overall survival for all 35 patients and for the 29 in complete remission was 49% +/- 8% and 59% +/- 9%, respectively. In the matched-pair analysis, the 5-year probability of overall survival for the 35 patients with HC and treated with FG was not statistically different from that of the comparative cohort of 35 patients who did not develop HC (32% +/- 9% versus 37% +/- 11%, P = not significant). FG therapy is a feasible, effective, repeatable, and affordable procedure for treating grade >/=2 HC after allogeneic transplantation. CI - Copyright (c) 2014 American Society for Blood and Marrow Transplantation. Published by Elsevier Inc. All rights reserved. FAU - Tirindelli, Maria Cristina AU - Tirindelli MC AD - Hematology Stem Cell Transplant Transfusion Medicine and Cellular Therapy, Campus Bio-Medico University Hospital, Rome, Italy. Electronic address: m.tirindelli@unicampus.it. FAU - Flammia, Gerardo Paolo AU - Flammia GP AD - Division of Urology, Campus Bio-Medico University Hospital, Rome, Italy. FAU - Bove, Pierluigi AU - Bove P AD - Division of Urology, Tor Vergata University Hospital, Rome, Italy. FAU - Cerretti, Raffaella AU - Cerretti R AD - Stem Cell Transplant Unit, Department of Hematology, Tor Vergata University, Rome, Italy. FAU - Cudillo, Laura AU - Cudillo L AD - Stem Cell Transplant Unit, Department of Hematology, Tor Vergata University, Rome, Italy. FAU - De Angelis, Gottardo AU - De Angelis G AD - Stem Cell Transplant Unit, Department of Hematology, Tor Vergata University, Rome, Italy. FAU - Picardi, Alessandra AU - Picardi A AD - Stem Cell Transplant Unit, Department of Hematology, Tor Vergata University, Rome, Italy. FAU - Annibali, Ombretta AU - Annibali O AD - Hematology Stem Cell Transplant Transfusion Medicine and Cellular Therapy, Campus Bio-Medico University Hospital, Rome, Italy. FAU - Nobile, Carolina AU - Nobile C AD - Hematology Stem Cell Transplant Transfusion Medicine and Cellular Therapy, Campus Bio-Medico University Hospital, Rome, Italy. FAU - Cerchiara, Elisabetta AU - Cerchiara E AD - Hematology Stem Cell Transplant Transfusion Medicine and Cellular Therapy, Campus Bio-Medico University Hospital, Rome, Italy. FAU - Dentamaro, Teresa AU - Dentamaro T AD - Division of Hematology and Transplant, Sant'Eugenio Hospital, Rome, Italy. FAU - De Fabritiis, Paolo AU - De Fabritiis P AD - Division of Hematology and Transplant, Sant'Eugenio Hospital, Rome, Italy. FAU - Lanti, Alessandro AU - Lanti A AD - Division of Blood Bank, Department of Immunohematology, Tor Vergata University, Rome, Italy. FAU - Ferraro, Angelo Salvatore AU - Ferraro AS AD - Division of Blood Bank, Department of Immunohematology, Tor Vergata University, Rome, Italy. FAU - Sergi, Federico AU - Sergi F AD - Division of Urology, Campus Bio-Medico University Hospital, Rome, Italy. FAU - Di Piazza, Fabio AU - Di Piazza F AD - Stem Cell Transplant Unit, Department of Hematology, Tor Vergata University, Rome, Italy. FAU - Avvisati, Giuseppe AU - Avvisati G AD - Hematology Stem Cell Transplant Transfusion Medicine and Cellular Therapy, Campus Bio-Medico University Hospital, Rome, Italy. FAU - Arcese, William AU - Arcese W AD - Stem Cell Transplant Unit, Department of Hematology, Tor Vergata University, Rome, Italy. CN - Rome Transplant Network LA - eng PT - Journal Article PT - Research Support, Non-U.S. Gov't DEP - 20140620 PL - United States TA - Biol Blood Marrow Transplant JT - Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation JID - 9600628 RN - 0 (Fibrin Tissue Adhesive) RN - 0 (Hemostatics) RN - 0 (Myeloablative Agonists) SB - IM MH - Adolescent MH - Adult MH - Aged MH - Aged, 80 and over MH - Cystitis/chemically induced/immunology/mortality/*surgery MH - Cystoscopy MH - Female MH - Fibrin Tissue Adhesive/*therapeutic use MH - Hematologic Neoplasms/*drug therapy/immunology/pathology MH - *Hematopoietic Stem Cell Transplantation MH - Hemorrhage/chemically induced/immunology/mortality/*surgery MH - Hemostatics/*therapeutic use MH - Humans MH - Male MH - Middle Aged MH - Myeloablative Agonists/adverse effects MH - Survival Analysis MH - Transplantation Conditioning/*methods MH - Transplantation, Homologous OTO - NOTNLM OT - Allogeneic transplantation OT - Fibrin glue therapy OT - Hemorrhagic cystitis EDAT- 2014/06/24 06:00 MHDA- 2015/05/20 06:00 CRDT- 2014/06/24 06:00 PHST- 2014/04/24 [received] PHST- 2014/06/13 [accepted] PHST- 2014/06/20 [aheadofprint] AID - S1083-8791(14)00368-1 [pii] AID - 10.1016/j.bbmt.2014.06.018 [doi] PST - ppublish SO - Biol Blood Marrow Transplant. 2014 Oct;20(10):1612-7. doi: 10.1016/j.bbmt.2014.06.018. Epub 2014 Jun 20. PMID- 24933658 OWN - NLM STAT- MEDLINE DA - 20140912 DCOM- 20150519 IS - 1523-6536 (Electronic) IS - 1083-8791 (Linking) VI - 20 IP - 10 DP - 2014 Oct TI - Poor outcome with nonmyeloablative conditioning regimen before cord blood transplantation for patients with high-risk acute myeloid leukemia compared with matched related or unrelated donor transplantation. PG - 1560-5 LID - 10.1016/j.bbmt.2014.06.006 [doi] LID - S1083-8791(14)00353-X [pii] AB - Allogeneic hematopoietic stem cell transplantation (Allo-HSCT) is recommended for patients with high-risk acute myeloid leukemia (AML). In many situations, a matched related (MRD) or matched unrelated donor (MUD) is lacking, in which case unrelated cord blood units (UCB) provide an alternative. We analyzed the outcome of consecutive high-risk AML patients prepared with reduced-intensity conditioning (RIC) regimens and allografted with UCB (n = 32) and compared their outcome with high-risk AML patients who underwent transplantation with MRD/MUD (n = 49) in the same period of time. Grade III to IV acute graft-versus-host disease (GVHD) occurred slightly more frequently in the UCB group (25%) than in the MRD/MUD group (8%) (P = .069). Conversely, we found a lower incidence of extensive chronic GVHD in the UCB group (6%) than in the MRD/MUD group (20%, P = .085). Nonrelapse mortality at 4 years was 16% and 22% in the UCB and MRD/MUD groups, respectively (P = .529). The cumulative incidence of relapse at 4 years was significantly higher in the UCB group (60%) than in the MRD/MUD group (27%, P = .006). Leukemia-free survival (LFS) and overall survival (OS) at 4 years were 25% and 34%, respectively, in the UCB group and 50% and 56%, respectively, in the MRD/MUD group (LFS, P = .029; OS, P = .072). Multivariate analyses adjusted by cytogenetics and disease status at the time of Allo-HSCT revealed that use of UCB remained an independent predictive factor of shorter LFS (hazard ratio, 2.0; 95% confidence interval, 1.1 to 3.6; P = .018), and was associated with a trend for shorter OS (hazard ratio, 1.7; 95% confidence interval, .9 to 3.2; P = .093). Whereas UCB provides an alternative for patients with high-risk AML lacking an MRD/MUD, the high incidence of relapse after RIC-based UCB Allo-HSCT is a concern. Attempts to improve leukemic control with UCB Allo-HSCT are warranted, as well as the evaluation of other alternative donors in this context. CI - Copyright (c) 2014 American Society for Blood and Marrow Transplantation. Published by Elsevier Inc. All rights reserved. FAU - Devillier, Raynier AU - Devillier R AD - Hematology Department, Transplantation Program, Institut Paoli Calmettes, Marseille, France; Aix-Marseille Universite, Marseille, France; Inserm UMR 1068/Centre de Recherche en Cancerologie de Marseille, Marseille, France. Electronic address: devillierr@ipc.unicancer.fr. FAU - Harbi, Samia AU - Harbi S AD - Hematology Department, Transplantation Program, Institut Paoli Calmettes, Marseille, France; Aix-Marseille Universite, Marseille, France. FAU - Furst, Sabine AU - Furst S AD - Hematology Department, Transplantation Program, Institut Paoli Calmettes, Marseille, France. FAU - Crocchiolo, Roberto AU - Crocchiolo R AD - Hematology Department, Transplantation Program, Institut Paoli Calmettes, Marseille, France. FAU - El-Cheikh, Jean AU - El-Cheikh J AD - Hematology Department, Transplantation Program, Institut Paoli Calmettes, Marseille, France. FAU - Castagna, Luca AU - Castagna L AD - Hematology Department, Transplantation Program, Institut Paoli Calmettes, Marseille, France; Humanitas Cancer Center, Hematology Unit, Instituto Clinico Humanitas, Rozzano, Milano, Italy. FAU - Etienne, Anne AU - Etienne A AD - Hematology Department, Transplantation Program, Institut Paoli Calmettes, Marseille, France. FAU - Calmels, Boris AU - Calmels B AD - Cell Therapy Facility, Institut Paoli Calmettes, Marseille, France. FAU - Lemarie, Claude AU - Lemarie C AD - Cell Therapy Facility, Institut Paoli Calmettes, Marseille, France. FAU - Prebet, Thomas AU - Prebet T AD - Hematology Department, Transplantation Program, Institut Paoli Calmettes, Marseille, France; Inserm UMR 1068/Centre de Recherche en Cancerologie de Marseille, Marseille, France. FAU - Granata, Angela AU - Granata A AD - Hematology Department, Transplantation Program, Institut Paoli Calmettes, Marseille, France. FAU - Charbonnier, Aude AU - Charbonnier A AD - Hematology Department, Transplantation Program, Institut Paoli Calmettes, Marseille, France. FAU - Rey, Jerome AU - Rey J AD - Hematology Department, Transplantation Program, Institut Paoli Calmettes, Marseille, France. FAU - Chabannon, Christian AU - Chabannon C AD - Aix-Marseille Universite, Marseille, France; Inserm UMR 1068/Centre de Recherche en Cancerologie de Marseille, Marseille, France; Cell Therapy Facility, Institut Paoli Calmettes, Marseille, France. FAU - Faucher, Catherine AU - Faucher C AD - Hematology Department, Transplantation Program, Institut Paoli Calmettes, Marseille, France. FAU - Vey, Norbert AU - Vey N AD - Hematology Department, Transplantation Program, Institut Paoli Calmettes, Marseille, France; Aix-Marseille Universite, Marseille, France; Inserm UMR 1068/Centre de Recherche en Cancerologie de Marseille, Marseille, France. FAU - Blaise, Didier AU - Blaise D AD - Hematology Department, Transplantation Program, Institut Paoli Calmettes, Marseille, France; Aix-Marseille Universite, Marseille, France; Inserm UMR 1068/Centre de Recherche en Cancerologie de Marseille, Marseille, France. LA - eng PT - Journal Article DEP - 20140614 PL - United States TA - Biol Blood Marrow Transplant JT - Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation JID - 9600628 RN - 0 (Antineoplastic Agents) SB - IM MH - Acute Disease MH - Adolescent MH - Adult MH - Aged MH - Antineoplastic Agents/therapeutic use MH - Chronic Disease MH - *Cord Blood Stem Cell Transplantation MH - Cytogenetic Analysis MH - Female MH - Graft vs Host Disease/immunology/mortality/pathology/*therapy MH - *Hematopoietic Stem Cell Transplantation MH - Histocompatibility Testing MH - Humans MH - Leukemia, Myeloid, Acute/immunology/mortality/pathology/*therapy MH - Male MH - Middle Aged MH - Prognosis MH - Recurrence MH - Retrospective Studies MH - Survival Analysis MH - Transplantation Conditioning/*methods MH - Transplantation, Homologous MH - Unrelated Donors OTO - NOTNLM OT - Acute myeloid leukemia OT - Allogeneic hematopoietic stem cell transplantation OT - Cord blood transplantation OT - Reduced-intensity conditioning regimen EDAT- 2014/06/17 06:00 MHDA- 2015/05/20 06:00 CRDT- 2014/06/17 06:00 PHST- 2014/05/05 [received] PHST- 2014/06/02 [accepted] PHST- 2014/06/14 [aheadofprint] AID - S1083-8791(14)00353-X [pii] AID - 10.1016/j.bbmt.2014.06.006 [doi] PST - ppublish SO - Biol Blood Marrow Transplant. 2014 Oct;20(10):1560-5. doi: 10.1016/j.bbmt.2014.06.006. Epub 2014 Jun 14. PMID- 24931578 OWN - NLM STAT- MEDLINE DA - 20140912 DCOM- 20150513 IS - 1532-2742 (Electronic) IS - 0163-4453 (Linking) VI - 69 IP - 4 DP - 2014 Oct TI - Impact of peri-transplant vancomycin and fluoroquinolone administration on rates of bacteremia in allogeneic hematopoietic stem cell transplant (HSCT) recipients: a 12-year single institution study. PG - 341-51 LID - 10.1016/j.jinf.2014.06.004 [doi] LID - S0163-4453(14)00163-7 [pii] AB - BACKGROUND: We analyzed the effect of peri-transplant prophylaxis on the epidemiology of bacteremia in a 12-year contemporary cohort of allogeneic HSCT recipients at our center. METHODS: This was an observational study of 1052 consecutive adult HSCT from 2000 to 2011. Formal prophylaxis with vancomycin only, fluoroquinolone (FQ) only, or vancomycin + FQ was implemented in 2006. The cumulative incidence of day 100 bacteremia was compared between the Early Period (2000-2005) and the Recent Period (2006-2011). Predictors for pre-engraftment bacteremia were analyzed with Cox-proportional hazard models in a subcohort of 821 HSCT who received myeloablative or reduced intensity conditioning (MA/RIC). RESULTS: The incidence of bacteremia decreased in the Recent Period (32% vs 27%; P = 0.002), whereas the rates of resistance in gram-negative rods (GNR) and vancomycin-resistant enterococci (VRE) were similar between the two Periods (P values are not statistically significant.) In multivariate analyses, prophylaxis with vancomycin only or vancomycin + FQ was protective (HR = 0.5; CI = 0.30-0.72) and (HR = 0.3; CI = 0.12-0.52, P < 0.01). Vancomycin or vancomycin + FQ eliminated viridans streptococcal bacteremia (VSB); vancomycin + FQ decreased GNR bacteremia (HR = 0.35; CI = 0.15-0.85). CONCLUSIONS: Vancomycin-based prophylaxis peri-transplant in MA/RIC HSCT was associated with elimination of VSB and may be considered at centers with high incidence of this infection. CI - Copyright (c) 2014 The British Infection Association. Published by Elsevier Ltd. All rights reserved. FAU - Seo, Susan K AU - Seo SK AD - Department of Medicine, Infectious Disease Service, Memorial Sloan-Kettering Cancer Center, New York, NY, USA; Department of Medicine, Weill Cornell Medical College, Cornell University, New York, NY, USA. Electronic address: Seos@mskcc.org. FAU - Xiao, Kun AU - Xiao K AD - Department of Medicine, Infectious Disease Service, Memorial Sloan-Kettering Cancer Center, New York, NY, USA. FAU - Huang, Yao-Ting AU - Huang YT AD - Department of Medicine, Infectious Disease Service, Memorial Sloan-Kettering Cancer Center, New York, NY, USA. FAU - Jongwutiwes, Ubonvan AU - Jongwutiwes U AD - Department of Medicine, Infectious Disease Service, Memorial Sloan-Kettering Cancer Center, New York, NY, USA. FAU - Chung, Dick AU - Chung D AD - Department of Medicine, Infectious Disease Service, Memorial Sloan-Kettering Cancer Center, New York, NY, USA. FAU - Maloy, Molly AU - Maloy M AD - Department of Medicine, Adult Bone Marrow Transplant Service, Memorial Sloan-Kettering Cancer Center, New York, NY, USA. FAU - Giralt, Sergio AU - Giralt S AD - Department of Medicine, Adult Bone Marrow Transplant Service, Memorial Sloan-Kettering Cancer Center, New York, NY, USA; Department of Medicine, Weill Cornell Medical College, Cornell University, New York, NY, USA. FAU - Barker, Juliet N AU - Barker JN AD - Department of Medicine, Adult Bone Marrow Transplant Service, Memorial Sloan-Kettering Cancer Center, New York, NY, USA; Department of Medicine, Weill Cornell Medical College, Cornell University, New York, NY, USA. FAU - Jakubowski, Ann A AU - Jakubowski AA AD - Department of Medicine, Adult Bone Marrow Transplant Service, Memorial Sloan-Kettering Cancer Center, New York, NY, USA; Department of Medicine, Weill Cornell Medical College, Cornell University, New York, NY, USA. FAU - Papanicolaou, Genovefa A AU - Papanicolaou GA AD - Department of Medicine, Infectious Disease Service, Memorial Sloan-Kettering Cancer Center, New York, NY, USA; Department of Medicine, Weill Cornell Medical College, Cornell University, New York, NY, USA. Electronic address: papanicg@mskcc.org. LA - eng GR - P01 CA023766/CA/NCI NIH HHS/United States GR - P01CA023766/CA/NCI NIH HHS/United States PT - Journal Article PT - Observational Study PT - Research Support, N.I.H., Extramural DEP - 20140612 PL - England TA - J Infect JT - The Journal of infection JID - 7908424 RN - 0 (Anti-Bacterial Agents) RN - 0 (Fluoroquinolones) RN - 6Q205EH1VU (Vancomycin) SB - IM MH - Adolescent MH - Adult MH - Aged MH - Anti-Bacterial Agents/*administration & dosage MH - Bacteremia/epidemiology/microbiology/*prevention & control MH - Female MH - Fluoroquinolones/*administration & dosage MH - Hematopoietic Stem Cell Transplantation/adverse effects/*methods MH - Humans MH - Male MH - Middle Aged MH - New York/epidemiology MH - Proportional Hazards Models MH - Risk Factors MH - Vancomycin/*administration & dosage MH - Young Adult PMC - PMC4163089 MID - NIHMS610363 OID - NLM: NIHMS610363 [Available on 10/01/15] OID - NLM: PMC4163089 [Available on 10/01/15] OTO - NOTNLM OT - Allogeneic transplant OT - Bacteremia OT - Fluoroquinolone prophylaxis OT - Vancomycin prophylaxis OT - Viridans streptococci EDAT- 2014/06/17 06:00 MHDA- 2015/05/15 06:00 CRDT- 2014/06/17 06:00 PMCR- 2015/10/01 00:00 PHST- 2014/04/21 [received] PHST- 2014/05/29 [revised] PHST- 2014/06/04 [accepted] PHST- 2014/06/12 [aheadofprint] AID - S0163-4453(14)00163-7 [pii] AID - 10.1016/j.jinf.2014.06.004 [doi] PST - ppublish SO - J Infect. 2014 Oct;69(4):341-51. doi: 10.1016/j.jinf.2014.06.004. Epub 2014 Jun 12. PMID- 24931494 OWN - NLM STAT- MEDLINE DA - 20140807 DCOM- 20150512 IS - 1399-0012 (Electronic) IS - 0902-0063 (Linking) VI - 28 IP - 8 DP - 2014 Aug TI - The cell composition of infused donor lymphocyte has different impact in different types of allogeneic hematopoietic stem cell transplantation. PG - 926-34 LID - 10.1111/ctr.12404 [doi] AB - Donor lymphocyte infusion (DLI) is often used to enhance the graft-versus-leukemia (GVL) effect after allogeneic hematopoietic stem cell transplantation (allo-HSCT). In this study, we first evaluated the impact of the cell composition of a modified DLI (mDLI) on the prognoses of patients. A total of 194 patients undergoing allo-HSCT were enrolled and received mDLI for various clinical reasons. The infused cellular components of the mDLI were examined by flow cytometry. The results showed that infusion with a lower dose of CD14(+) cells (<0.33 x 10(8) /kg) was an independent risk factor for the occurrence of II-IV acute graft-versus-host disease (aGVHD) (HR = 0.104, p = 0.032) in human leukoctye antigen-identical transplant patients. In addition, a dose of CD14(+) cells greater than the 50th percentile was associated with a lower incidence of hematological relapse and longer disease-free survival (DFS) after the mDLI (relapse: HR = 0.193, p = 0.007; DFS: HR = 0.259, p = 0.016). However, we also found that a greater number of CD14(+) cells were an independent risk factor for II-IV aGVHD (HR = 1.758, p = 0.034) in haploidentical allo-HSCT. In conclusion, our data were the first to demonstrate that the cell composition of a 56 mDLI played a distinct role in different types of allo-HSCT. This finding provided a novel approach for the development of cellular therapies by manipulating the components of infused cells. CI - (c) 2014 John Wiley & Sons A/S. Published by John Wiley & Sons Ltd. FAU - Zhao, Xiao-Su AU - Zhao XS AD - Peking University People's Hospital, Peking University Institute of Hematology, Beijing, China; Beijing Key Laboratory of Hematopoietic Stem Cell Transplantation, Beijing, China. FAU - Wang, Yu AU - Wang Y FAU - Yan, Chen-Hua AU - Yan CH FAU - Wang, Jing-Zhi AU - Wang JZ FAU - Zhang, Xiao-Hui AU - Zhang XH FAU - Xu, Lan-Ping AU - Xu LP FAU - Liu, Kai-Yan AU - Liu KY FAU - Huang, Xiao-Jun AU - Huang XJ LA - eng PT - Journal Article DEP - 20140707 PL - Denmark TA - Clin Transplant JT - Clinical transplantation JID - 8710240 RN - 0 (Antigens, CD14) SB - IM MH - Adolescent MH - Adult MH - Antigens, CD14/*metabolism MH - Child MH - Child, Preschool MH - Female MH - Follow-Up Studies MH - Graft vs Host Disease/etiology/mortality/*prevention & control MH - Hematologic Neoplasms/*therapy MH - *Hematopoietic Stem Cell Transplantation MH - Humans MH - *Lymphocyte Transfusion MH - Lymphocytes/*classification/metabolism MH - Male MH - Middle Aged MH - Neoplasm Recurrence, Local/etiology/mortality/*prevention & control MH - Prognosis MH - Retrospective Studies MH - Risk Factors MH - Survival Rate MH - Tissue Donors MH - Transplantation, Homologous MH - Young Adult OTO - NOTNLM OT - allogeneic hematopoietic stem cell transplantation OT - composition OT - donor lymphocyte infusion OT - graft-versus-host disease OT - outcome EDAT- 2014/06/17 06:00 MHDA- 2015/05/13 06:00 CRDT- 2014/06/17 06:00 PHST- 2014/06/11 [accepted] PHST- 2014/07/07 [aheadofprint] AID - 10.1111/ctr.12404 [doi] PST - ppublish SO - Clin Transplant. 2014 Aug;28(8):926-34. doi: 10.1111/ctr.12404. Epub 2014 Jul 7. PMID- 24930759 OWN - NLM STAT- MEDLINE DA - 20140815 DCOM- 20150511 IS - 1399-3046 (Electronic) IS - 1397-3142 (Linking) VI - 18 IP - 6 DP - 2014 Sep TI - Successful treatment of non-Hodgkin's lymphoma using R-CHOP in a patient with Wiskott-Aldrich syndrome followed by a reduced-intensity stem cell transplant. PG - E208-11 LID - 10.1111/petr.12297 [doi] AB - WAS is an X-linked primary immunodeficiency characterized by microthrombocytopenia, eczema, recurrent infections, and increased incidence of autoimmunity and malignancy. HSCT is the only curative treatment for WAS. Herein, we report the case of a 17-yr-old boy with WAS who received an unrelated HSCT while in complete remission of diffuse large B-cell lymphoma after chemotherapy. Pretransplant conditioning consisted of fludarabine, busulfan, and total body irradiation (4 Gy). GvHD prophylaxis consisted of tacrolimus and short-course methotrexate. Following HSCT, rapid and stable engraftment was observed. Platelet count gradually increased, and the generalized eczema improved. The patient developed grade II acute GvHD and limited chronic GvHD on days 30 and 210, respectively, which resolved with immunosuppressive treatment. Symptoms caused by the reactivation of human herpes virus-6, BK virus, and VZV were observed from days 21, 60, and 96, respectively; they were resolved after conservative treatment and acyclovir administration. No other regimen-related toxicity was observed. Complete donor bone marrow chimerism was achieved one month after transplantation. RIST is an effective therapeutic option for older children with WAS accompanied by malignant lymphoma. CI - (c) 2014 John Wiley & Sons A/S. Published by John Wiley & Sons Ltd. FAU - Koga, Yuhki AU - Koga Y AD - Department of Pediatrics, Graduate school of Medical Sciences, Kyushu University, Fukuoka, Japan. FAU - Takada, Hidetoshi AU - Takada H FAU - Suminoe, Aiko AU - Suminoe A FAU - Ohga, Shouichi AU - Ohga S FAU - Hara, Toshiro AU - Hara T LA - eng PT - Case Reports PT - Journal Article DEP - 20140614 PL - Denmark TA - Pediatr Transplant JT - Pediatric transplantation JID - 9802574 RN - 0 (Antibodies, Monoclonal, Murine-Derived) RN - 0 (Immunosuppressive Agents) RN - 0 (R-CHOP protocol) RN - 57-22-7 (Vincristine) RN - 80168379AG (Doxorubicin) RN - 8N3DW7272P (Cyclophosphamide) RN - VB0R961HZT (Prednisone) SB - IM MH - Adolescent MH - Antibodies, Monoclonal, Murine-Derived/therapeutic use MH - Antineoplastic Combined Chemotherapy Protocols/*therapeutic use MH - Combined Modality Therapy MH - Cyclophosphamide/therapeutic use MH - Doxorubicin/therapeutic use MH - Graft vs Host Disease/drug therapy MH - Humans MH - Immunosuppressive Agents/therapeutic use MH - Lymphoma, Non-Hodgkin/*therapy MH - Male MH - Prednisone/therapeutic use MH - *Stem Cell Transplantation MH - Vincristine/therapeutic use MH - Wiskott-Aldrich Syndrome/*complications/*therapy OTO - NOTNLM OT - Wiskott-Aldrich syndrome OT - allogeneic bone marrow transplantation OT - malignant lymphoma OT - reduced-intensity stem cell transplantation EDAT- 2014/06/17 06:00 MHDA- 2015/05/12 06:00 CRDT- 2014/06/17 06:00 PHST- 2014/04/25 [accepted] PHST- 2014/06/14 [aheadofprint] AID - 10.1111/petr.12297 [doi] PST - ppublish SO - Pediatr Transplant. 2014 Sep;18(6):E208-11. doi: 10.1111/petr.12297. Epub 2014 Jun 14. PMID- 24923536 OWN - NLM STAT- MEDLINE DA - 20140912 DCOM- 20150519 IS - 1523-6536 (Electronic) IS - 1083-8791 (Linking) VI - 20 IP - 10 DP - 2014 Oct TI - Reduced-intensity conditioning hematopoietic cell transplantation is an effective treatment for patients with SLAM-associated protein deficiency/X-linked lymphoproliferative disease type 1. PG - 1641-5 LID - 10.1016/j.bbmt.2014.06.003 [doi] LID - S1083-8791(14)00350-4 [pii] AB - X-linked lymphoproliferative disease type 1 (XLP1) is a rare immune deficiency caused by mutations in SH2D1A. Allogeneic hematopoietic cell transplantation (HCT) is often performed because of the morbidity and mortality associated with XLP1. There is limited experience using reduced-intensity conditioning (RIC) regimens for these patients. Here we report our 8-year single-center experience. Sixteen consecutive patients diagnosed with XLP1 underwent allogeneic HCT between 2006 and 2013 after a RIC regimen consisting of alemtuzumab, fludarabine, and melphalan. Patient phenotypes included hemophagocytic lymphohistiocytosis (HLH) after Epstein-Barr virus (n = 5) or human herpesvirus 6 (n = 1), macrophage activation syndrome (n = 1), interstitial pneumonitis and encephalitis (n = 1), B cell lymphoma (n = 8), and hypogammaglobulinemia (n = 2). One patient was asymptomatic. Fourteen of 16 patients received 8/8 HLA-matched unrelated or related bone marrow grafts, whereas 2 patients received mismatched unrelated grafts. Acute graft-versus-host disease (GVHD) prophylaxis consisted of methylprednisolone and cyclosporine in all but 1 patient, who additionally received methotrexate. All patients had hematopoietic recovery. There were no cases of hepatic veno-occlusive disease or pulmonary hemorrhage. One patient (6%) developed acute GVHD and later also developed chronic GVHD (6%). Five patients (31%) developed mixed chimerism. Only 1 patient with mixed chimerism (6%) experienced a decline of donor chimerism to less than 50% but returned to full donor chimerism after infusion of donor lymphocytes and a CD34(+) selected stem cell boost. Infectious complications were frequent, particularly viral reactivation. One-year survival estimated by Kaplan-Meier analysis was 80%, with long-term survival estimated at 71%. Survival was similar for patients with or without a history of HLH (86% versus 75%, respectively, P = .70). There were no occurrences of lymphoma or HLH after HCT. RIC HCT with alemtuzumab, fludarabine, and melphalan is an effective treatment for patients with XLP1, offering good survival rates regardless of prior disease manifestations, including HLH. CI - Copyright (c) 2014 American Society for Blood and Marrow Transplantation. Published by Elsevier Inc. All rights reserved. FAU - Marsh, Rebecca A AU - Marsh RA AD - Division of Bone Marrow Transplantation and Immune Deficiency, Cancer and Blood Diseases Institute, Cincinnati Children's Hospital, Cincinnati, Ohio. Electronic address: rebecca.marsh@cchmc.org. FAU - Bleesing, Jack J AU - Bleesing JJ AD - Division of Bone Marrow Transplantation and Immune Deficiency, Cancer and Blood Diseases Institute, Cincinnati Children's Hospital, Cincinnati, Ohio. FAU - Chandrakasan, Shanmuganathan AU - Chandrakasan S AD - Division of Bone Marrow Transplantation and Immune Deficiency, Cancer and Blood Diseases Institute, Cincinnati Children's Hospital, Cincinnati, Ohio. FAU - Jordan, Michael B AU - Jordan MB AD - Division of Bone Marrow Transplantation and Immune Deficiency, Cancer and Blood Diseases Institute, Cincinnati Children's Hospital, Cincinnati, Ohio. FAU - Davies, Stella M AU - Davies SM AD - Division of Bone Marrow Transplantation and Immune Deficiency, Cancer and Blood Diseases Institute, Cincinnati Children's Hospital, Cincinnati, Ohio. FAU - Filipovich, Alexandra H AU - Filipovich AH AD - Division of Bone Marrow Transplantation and Immune Deficiency, Cancer and Blood Diseases Institute, Cincinnati Children's Hospital, Cincinnati, Ohio. LA - eng PT - Journal Article DEP - 20140609 PL - United States TA - Biol Blood Marrow Transplant JT - Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation JID - 9600628 RN - 0 (Antibodies, Monoclonal, Humanized) RN - 0 (Immunosuppressive Agents) RN - 0 (Intracellular Signaling Peptides and Proteins) RN - 0 (Myeloablative Agonists) RN - 0 (SH2D1A protein, human) RN - 3A189DH42V (alemtuzumab) RN - FA2DM6879K (Vidarabine) RN - P2K93U8740 (fludarabine) RN - Q41OR9510P (Melphalan) SB - IM MH - Antibodies, Monoclonal, Humanized/therapeutic use MH - Child MH - Child, Preschool MH - Female MH - Graft vs Host Disease/immunology/prevention & control MH - *Hematopoietic Stem Cell Transplantation MH - Histocompatibility Testing MH - Humans MH - Immunosuppressive Agents/therapeutic use MH - Infant MH - Intracellular Signaling Peptides and Proteins/genetics/immunology MH - Lymphoproliferative Disorders/*drug therapy/*genetics/mortality/pathology MH - Male MH - Melphalan/therapeutic use MH - Mutation MH - Myeloablative Agonists/*therapeutic use MH - Prognosis MH - Retrospective Studies MH - Survival Analysis MH - Transplantation Chimera/genetics/immunology MH - Transplantation Conditioning/*methods MH - Transplantation, Homologous MH - Unrelated Donors MH - Vidarabine/analogs & derivatives/therapeutic use OTO - NOTNLM OT - Alemtuzumab OT - Allogeneic hematopoietic cell transplant OT - Bone marrow transplant OT - Reduced-intensity conditioning OT - SH2D1A OT - SLAM-associated protein OT - X-linked lymphoproliferative disease OT - XLP EDAT- 2014/06/14 06:00 MHDA- 2015/05/20 06:00 CRDT- 2014/06/14 06:00 PHST- 2014/03/31 [received] PHST- 2014/06/03 [accepted] PHST- 2014/06/09 [aheadofprint] AID - S1083-8791(14)00350-4 [pii] AID - 10.1016/j.bbmt.2014.06.003 [doi] PST - ppublish SO - Biol Blood Marrow Transplant. 2014 Oct;20(10):1641-5. doi: 10.1016/j.bbmt.2014.06.003. Epub 2014 Jun 9. PMID- 24914821 OWN - NLM STAT- MEDLINE DA - 20140912 DCOM- 20150519 IS - 1523-6536 (Electronic) IS - 1083-8791 (Linking) VI - 20 IP - 10 DP - 2014 Oct TI - Varicella-zoster reactivation after allogeneic stem cell transplantation without routine prophylaxis--the incidence remains high. PG - 1646-9 LID - 10.1016/j.bbmt.2014.06.002 [doi] LID - S1083-8791(14)00349-8 [pii] AB - One-year prophylaxis with acyclovir has been shown to effectively prevent varicella-zoster virus (VZV) reactivation after allogeneic hematopoietic stem cell transplantation (HSCT) in a cohort that underwent transplantation in the beginning of the 2000s. Transplantation procedures have since changed considerably and reduced-intensity conditioning (RIC) is nowadays common. We investigated VZV reactivation without routine prophylaxis in a cohort of HSCT patients, 50% of whom had received RIC. The cumulative 2-year incidence of VZV reactivation was 20.7%. Risk factors in a multivariate analysis were treatment with mesenchymal stromal cells (relative hazard [RH], 1.65; confidence interval [CI], 1.07 to 2.54; P = .02), total body irradiation >/=6 Gy (RH, 1.55; CI, 1.14 to 2.13; P = .006), engraftment later than day 16 (RH, 1.46; CI, 1.07 to 2.00; P = .02), and age 0 to 19 years (RH, 1.68; CI, 1.21 to 2.35; P = .002). There was no difference in VZV reactivation between patients receiving myeloablative conditioning or RIC. VZV-related complications occurred in 29% of the patients with reactivation; most common were disseminated disease and postherpetic neuralgia. No single low-risk group for VZV reactivation could be identified. We conclude that VZV reactivation remains common after HSCT and carries a high complication rate, warranting prophylaxis. CI - Copyright (c) 2014 American Society for Blood and Marrow Transplantation. Published by Elsevier Inc. All rights reserved. FAU - Blennow, Ola AU - Blennow O AD - Division of Infectious Diseases, Department of Medicine, Karolinska Institutet, Karolinska University Hospital, Stockholm, Sweden; Centre for Allogeneic Stem Cell Transplantation, Karolinska Institutet, Karolinska University Hospital, Stockholm, Sweden. Electronic address: ola.blennow@karolinska.se. FAU - Fjaertoft, Gustav AU - Fjaertoft G AD - Centre for Allogeneic Stem Cell Transplantation, Karolinska Institutet, Karolinska University Hospital, Stockholm, Sweden. FAU - Winiarski, Jacek AU - Winiarski J AD - Department of Pediatrics, Clintec, Karolinska Institutet, Karolinska University Hospital, Stockholm, Sweden. FAU - Ljungman, Per AU - Ljungman P AD - Division of Hematology, Department of Medicine, Karolinska Institutet, Karolinska University Hospital, Stockholm, Sweden. FAU - Mattsson, Jonas AU - Mattsson J AD - Centre for Allogeneic Stem Cell Transplantation, Karolinska Institutet, Karolinska University Hospital, Stockholm, Sweden. FAU - Remberger, Mats AU - Remberger M AD - Centre for Allogeneic Stem Cell Transplantation, Karolinska Institutet, Karolinska University Hospital, Stockholm, Sweden. LA - eng PT - Journal Article PT - Research Support, Non-U.S. Gov't DEP - 20140607 PL - United States TA - Biol Blood Marrow Transplant JT - Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation JID - 9600628 RN - 0 (Myeloablative Agonists) SB - IM MH - Adolescent MH - Adult MH - Age Factors MH - Aged MH - Child MH - Child, Preschool MH - Female MH - Hematologic Neoplasms/immunology/pathology/*therapy MH - Hematopoietic Stem Cell Transplantation/*adverse effects MH - Herpes Zoster/etiology/*pathology/virology MH - Herpesvirus 3, Human/pathogenicity/*physiology MH - Humans MH - Infant MH - Male MH - Mesenchymal Stem Cell Transplantation/adverse effects MH - Middle Aged MH - Myeloablative Agonists/therapeutic use MH - Neuralgia, Postherpetic/etiology/*pathology/virology MH - Prognosis MH - Retrospective Studies MH - Risk Factors MH - Time Factors MH - Transplantation Conditioning/*methods MH - Transplantation, Homologous MH - Virus Activation OTO - NOTNLM OT - Allogeneic OT - Hematopoietic stem cell transplantation OT - Herpes zoster reactivation OT - Prophylaxis OT - Varicella-zoster virus EDAT- 2014/06/11 06:00 MHDA- 2015/05/20 06:00 CRDT- 2014/06/11 06:00 PHST- 2014/04/10 [received] PHST- 2014/06/03 [accepted] PHST- 2014/06/07 [aheadofprint] AID - S1083-8791(14)00349-8 [pii] AID - 10.1016/j.bbmt.2014.06.002 [doi] PST - ppublish SO - Biol Blood Marrow Transplant. 2014 Oct;20(10):1646-9. doi: 10.1016/j.bbmt.2014.06.002. Epub 2014 Jun 7. PMID- 24910380 OWN - NLM STAT- MEDLINE DA - 20140912 DCOM- 20150519 LR - 20150505 IS - 1523-6536 (Electronic) IS - 1083-8791 (Linking) VI - 20 IP - 10 DP - 2014 Oct TI - Exercise and stress management training prior to hematopoietic cell transplantation: Blood and Marrow Transplant Clinical Trials Network (BMT CTN) 0902. PG - 1530-6 LID - 10.1016/j.bbmt.2014.05.027 [doi] LID - S1083-8791(14)00325-5 [pii] AB - Studies show that engaging patients in exercise and/or stress management techniques during hematopoietic cell transplantation (HCT) improves quality of life. The Blood and Marrow Transplant Clinical Trials Network tested the efficacy of training patients to engage in self-directed exercise and stress management during HCT. The study randomized 711 patients at 21 centers to receive 1 of 4 training interventions before HCT: a self-directed exercise program, a self-administered stress management program, both, or neither. Participants completed self-reported assessments at enrollment and up to 180 days after HCT. Randomization was stratified by center and transplant type. There were no differences in the primary endpoints of the Physical Component Summary and Mental Component Summary scales of the Medical Outcomes Study Short Form 36 at day +100 among the groups, based on an intention-to-treat analysis. There also were no differences in overall survival, days of hospitalization through day +100 post-HCT, or in other patient-reported outcomes, including treatment-related distress, sleep quality, pain, and nausea. Patients randomized to training in stress management reported more use of those techniques, but patients randomized to training in exercise did not report more physical activity. Although other studies have reported efficacy of more intensive interventions, brief training in an easy-to-disseminate format for either self-directed exercise or stress management was not effective in our trial. CI - Copyright (c) 2014 American Society for Blood and Marrow Transplantation. All rights reserved. FAU - Jacobsen, Paul B AU - Jacobsen PB AD - Department of Health Outcomes and Behavior, Moffitt Cancer Center, Tampa, Florida. FAU - Le-Rademacher, Jennifer AU - Le-Rademacher J AD - Division of Biostatistics, Medical College of Wisconsin, Milwaukee, Wisconsin. FAU - Jim, Heather AU - Jim H AD - Department of Health Outcomes and Behavior, Moffitt Cancer Center, Tampa, Florida. FAU - Syrjala, Karen AU - Syrjala K AD - Clinical Research Division, Fred Hutchinson Cancer Research Center, Seattle, Washington. FAU - Wingard, John R AU - Wingard JR AD - Division of Hematology/Oncology, Department of Medicine, University of Florida College of Medicine, Gainesville, Florida. FAU - Logan, Brent AU - Logan B AD - Division of Biostatistics, Medical College of Wisconsin, Milwaukee, Wisconsin. FAU - Wu, Juan AU - Wu J AD - The EMMES Corporation, Washington, DC. FAU - Majhail, Navneet S AU - Majhail NS AD - Blood and Marrow Transplant Program, Cleveland Clinic, Cleveland, Ohio. FAU - Wood, William AU - Wood W AD - Lineberger Comprehensive Cancer Center, University of North Carolina at Chapel Hill, Chapel Hill, North Carolina. FAU - Rizzo, J Douglas AU - Rizzo JD AD - Center for International Blood and Marrow Transplant Research, Department of Medicine, Medical College of Wisconsin, Milwaukee, Wisconsin. FAU - Geller, Nancy L AU - Geller NL AD - Office of Biostatistics Research, National Heart, Lung, and Blood Institute, Bethesda, Maryland. FAU - Kitko, Carrie AU - Kitko C AD - Pediatric Blood and Marrow Transplant Program, University of Michigan, Ann Arbor, Michigan. FAU - Faber, Edward AU - Faber E AD - Division of Hematology/Oncology, University of Nebraska Medical Center, Omaha, Nebraska. FAU - Abidi, Muneer H AU - Abidi MH AD - Department of Medical Oncology, Wayne State University/Karmanos Cancer Institute, Detroit, Michigan. FAU - Slater, Susan AU - Slater S AD - Center for Hematologic Malignancies, Knight Cancer Institute, Oregon Health and Science University, Portland, Oregon. FAU - Horowitz, Mary M AU - Horowitz MM AD - Center for International Blood and Marrow Transplant Research, Department of Medicine, Medical College of Wisconsin, Milwaukee, Wisconsin. FAU - Lee, Stephanie J AU - Lee SJ AD - Clinical Research Division, Fred Hutchinson Cancer Research Center, Seattle, Washington. Electronic address: sjlee@fhcrc.org. LA - eng GR - U10 HL069246/HL/NHLBI NIH HHS/United States GR - U10 HL069294/HL/NHLBI NIH HHS/United States GR - U10 HL069301/HL/NHLBI NIH HHS/United States GR - U10 HL069310/HL/NHLBI NIH HHS/United States GR - U10 HL069330/HL/NHLBI NIH HHS/United States GR - U10 HL108945/HL/NHLBI NIH HHS/United States GR - U10 HL108987/HL/NHLBI NIH HHS/United States GR - U10 HL109137/HL/NHLBI NIH HHS/United States GR - U10 HL109322/HL/NHLBI NIH HHS/United States GR - U10 HL109526/HL/NHLBI NIH HHS/United States GR - U10HL069294/HL/NHLBI NIH HHS/United States GR - U24 CA076518/CA/NCI NIH HHS/United States PT - Journal Article PT - Multicenter Study PT - Randomized Controlled Trial PT - Research Support, N.I.H., Extramural DEP - 20140606 PL - United States TA - Biol Blood Marrow Transplant JT - Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation JID - 9600628 RN - 0 (Myeloablative Agonists) SB - IM MH - Adolescent MH - Adult MH - Aged MH - *Exercise Therapy MH - Female MH - Hematologic Neoplasms/immunology/mortality/*psychology/*therapy MH - *Hematopoietic Stem Cell Transplantation MH - Humans MH - Intention to Treat Analysis MH - Male MH - Mental Status Schedule MH - Middle Aged MH - Myeloablative Agonists/therapeutic use MH - Prognosis MH - Quality of Life MH - Self Report MH - Stress, Psychological/*therapy MH - Survival Analysis MH - *Transplantation Conditioning MH - Transplantation, Homologous PMC - PMC4163109 MID - NIHMS603210 OID - NLM: NIHMS603210 [Available on 10/01/15] OID - NLM: PMC4163109 [Available on 10/01/15] OTO - NOTNLM OT - Allogeneic hematopoietic cell transplantation OT - Autologous hematopoietic cell transplantation OT - Exercise OT - Quality of life OT - Stress management EDAT- 2014/06/10 06:00 MHDA- 2015/05/20 06:00 CRDT- 2014/06/10 06:00 PMCR- 2015/10/01 00:00 PHST- 2014/04/29 [received] PHST- 2014/05/27 [accepted] PHST- 2014/06/06 [aheadofprint] AID - S1083-8791(14)00325-5 [pii] AID - 10.1016/j.bbmt.2014.05.027 [doi] PST - ppublish SO - Biol Blood Marrow Transplant. 2014 Oct;20(10):1530-6. doi: 10.1016/j.bbmt.2014.05.027. Epub 2014 Jun 6. PMID- 24910379 OWN - NLM STAT- MEDLINE DA - 20140912 DCOM- 20150519 IS - 1523-6536 (Electronic) IS - 1083-8791 (Linking) VI - 20 IP - 10 DP - 2014 Oct TI - Unmanipulated haploidentical transplants compared with other alternative donors and matched sibling grafts. PG - 1573-9 LID - 10.1016/j.bbmt.2014.05.029 [doi] LID - S1083-8791(14)00329-2 [pii] AB - We studied 459 consecutive patients with hematologic malignancies, median age 44 years (range, 15 to 71 years), who underwent transplantation with grafts from identical sibling donors (SIB; n = 176), matched unrelated donors (MUD; n = 43), mismatched unrelated donors (mmUD; n = 43), unrelated cord blood (UCB; n = 105) or HLA-haploidentical family donors (HAPLO; n = 92). Graft-versus-host disease (GVHD) prophylaxis consisted of cyclosporine and methotrexate in the SIB recipients; antithymocyte globulin for the MUD, mmUD, and UCB recipients; and post-transplantation cyclophosphamide, cyclosporine, and mycophenolate in the HAPLO recipients. Conditioning regimens were mostly myeloablative (69%). Advanced disease phase was more frequent, but not significantly so, in the HAPLO and mmUD groups (P = .08). Acute GVHD grade II-IV was significantly less frequent in the HAPLO, UCB, and MUD groups (14% to 21%) compared with the SIB (31%) and mmUD (42%) groups (P < .001), and there was a trend toward less moderate-severe chronic GVHD in the HAPLO and UCB groups (P = .053). The proportion of patients off cyclosporine at 1 year ranged from 55% for the SIB group to 81% for the HAPLO group (P < .001). Transplantation-related mortality at 2 years was lower in the HAPLO and SIB groups (18% to 24%) compared with the MUD, mmUD, and UCB groups (33% to 35%; P = .10). Relapse rate was comparable in the 5 groups (P = .80). The 4-year actuarial survival was 45% in the SIB group, 43% in the MUD group, 40% in the mmUD group, 34% in the UCB group, and 52% in the HAPLO group (P = .10). In multivariate analysis, advanced disease was a negative predictor of survival (hazard ratio [HR], 2.4; P < .0001), together with a diagnosis of acute leukemia (HR, 1.8; P = .0001); HAPLO grafts were comparable to SIB (P = .80), whereas UCB had inferior survival (P = .03). In conclusion, unmanipulated haploidentical family donor transplants are an additional option for patients lacking a matched sibling donor. CI - Copyright (c) 2014 American Society for Blood and Marrow Transplantation. Published by Elsevier Inc. All rights reserved. FAU - Raiola, Anna Maria AU - Raiola AM AD - Division of Hematology and Marrow Transplantation, Istituto Ricerca Carattere Scientifico (IRCCS), San Martino Istituto Tumori (IST), Genova, Italy. FAU - Dominietto, Alida AU - Dominietto A AD - Division of Hematology and Marrow Transplantation, Istituto Ricerca Carattere Scientifico (IRCCS), San Martino Istituto Tumori (IST), Genova, Italy. FAU - di Grazia, Carmen AU - di Grazia C AD - Division of Hematology and Marrow Transplantation, Istituto Ricerca Carattere Scientifico (IRCCS), San Martino Istituto Tumori (IST), Genova, Italy. FAU - Lamparelli, Teresa AU - Lamparelli T AD - Division of Hematology and Marrow Transplantation, Istituto Ricerca Carattere Scientifico (IRCCS), San Martino Istituto Tumori (IST), Genova, Italy. FAU - Gualandi, Francesca AU - Gualandi F AD - Division of Hematology and Marrow Transplantation, Istituto Ricerca Carattere Scientifico (IRCCS), San Martino Istituto Tumori (IST), Genova, Italy. FAU - Ibatici, Adalberto AU - Ibatici A AD - Division of Hematology and Marrow Transplantation, Istituto Ricerca Carattere Scientifico (IRCCS), San Martino Istituto Tumori (IST), Genova, Italy. FAU - Bregante, Stefania AU - Bregante S AD - Division of Hematology and Marrow Transplantation, Istituto Ricerca Carattere Scientifico (IRCCS), San Martino Istituto Tumori (IST), Genova, Italy. FAU - Van Lint, Maria Teresa AU - Van Lint MT AD - Division of Hematology and Marrow Transplantation, Istituto Ricerca Carattere Scientifico (IRCCS), San Martino Istituto Tumori (IST), Genova, Italy. FAU - Varaldo, Riccardo AU - Varaldo R AD - Division of Hematology and Marrow Transplantation, Istituto Ricerca Carattere Scientifico (IRCCS), San Martino Istituto Tumori (IST), Genova, Italy. FAU - Ghiso, Anna AU - Ghiso A AD - Division of Hematology and Marrow Transplantation, Istituto Ricerca Carattere Scientifico (IRCCS), San Martino Istituto Tumori (IST), Genova, Italy. FAU - Gobbi, Marco AU - Gobbi M AD - Chair of Hematology, University of Genova, Italy. FAU - Carella, Angelo Michele AU - Carella AM AD - Division of Clinical Hematology, IRCCS San Martino IST, Genova, Italy. FAU - Signori, Alessio AU - Signori A AD - Chair of Medical Statistics, University of Genova, Italy. FAU - Galaverna, Federica AU - Galaverna F AD - Division of Hematology and Marrow Transplantation, Istituto Ricerca Carattere Scientifico (IRCCS), San Martino Istituto Tumori (IST), Genova, Italy. FAU - Bacigalupo, Andrea AU - Bacigalupo A AD - Division of Hematology and Marrow Transplantation, Istituto Ricerca Carattere Scientifico (IRCCS), San Martino Istituto Tumori (IST), Genova, Italy. Electronic address: andrea.bacigalupo@hsanmartino.it. LA - eng PT - Comparative Study PT - Journal Article PT - Research Support, Non-U.S. Gov't DEP - 20140605 PL - United States TA - Biol Blood Marrow Transplant JT - Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation JID - 9600628 RN - 0 (HLA Antigens) RN - 0 (Immunosuppressive Agents) RN - 0 (Myeloablative Agonists) SB - IM MH - Adolescent MH - Adult MH - Aged MH - *Cord Blood Stem Cell Transplantation MH - Female MH - Gene Expression MH - Graft vs Host Disease/*prevention & control MH - HLA Antigens/genetics/immunology MH - Haplotypes MH - Hematologic Neoplasms/genetics/immunology/mortality/*therapy MH - *Hematopoietic Stem Cell Transplantation MH - Histocompatibility Testing MH - Humans MH - Immunosuppressive Agents/therapeutic use MH - Male MH - Middle Aged MH - Myeloablative Agonists/therapeutic use MH - Prospective Studies MH - Recurrence MH - Siblings MH - Survival Analysis MH - Transplantation Conditioning/*methods MH - Transplantation, Homologous MH - Unrelated Donors OTO - NOTNLM OT - Allogeneic transplant OT - Cord blood OT - Haploidentical OT - Unrelated EDAT- 2014/06/10 06:00 MHDA- 2015/05/20 06:00 CRDT- 2014/06/10 06:00 PHST- 2014/04/28 [received] PHST- 2014/05/30 [accepted] PHST- 2014/06/05 [aheadofprint] AID - S1083-8791(14)00329-2 [pii] AID - 10.1016/j.bbmt.2014.05.029 [doi] PST - ppublish SO - Biol Blood Marrow Transplant. 2014 Oct;20(10):1573-9. doi: 10.1016/j.bbmt.2014.05.029. Epub 2014 Jun 5. PMID- 24910378 OWN - NLM STAT- MEDLINE DA - 20140912 DCOM- 20150519 IS - 1523-6536 (Electronic) IS - 1083-8791 (Linking) VI - 20 IP - 10 DP - 2014 Oct TI - Vitamin d levels affect outcome in pediatric hematopoietic stem cell transplantation. PG - 1537-43 LID - 10.1016/j.bbmt.2014.05.030 [doi] LID - S1083-8791(14)00330-9 [pii] AB - The importance of vitamin D in immunologic processes has recently emerged, but whether it has any impact on the course of allogeneic hematopoietic stem cell transplantation (HSCT) has not been determined. Reports indicate that HSCT recipients, particularly children, often suffer from vitamin D deficiency. This study investigated the role of vitamin D in 123 children undergoing HSCT from 2004 to 2011. Vitamin D (ie, serum calcidiol) was analyzed in collected cryostored samples. Patients were grouped according to pre-HSCT calcidiol level: insufficient (<50 nm/L, n = 38) and sufficient (>/=50 nm/L, n = 85). Older children who underwent transplants from January through June and children of Middle Eastern or African origin were more commonly found in the insufficient group. Acute grades II to IV graft-versus-host disease occurred more frequently in the vitamin D sufficient group (47% versus 30%, P = .05), whereas no difference was demonstrated for chronic graft-versus-host disease. The neutrophil granulocytes rose significantly faster in the vitamin D sufficient group. No difference in lymphocyte counts, immunoglobulin levels, or infectious disease burden during the first year post-HSCT were observed. Among children with malignancies, overall survival was significantly better in the sufficient group (87% versus 50%, P = .01). In addition, rejection (0% versus 11%, P = .06) and relapse (4% versus 33%, P = .03) rates were lower in patients with sufficient vitamin D levels. To conclude, vitamin D may have an important impact on the outcome of pediatric HSCT, particularly in patients with malignant disease. Further studies investigating whether vitamin D acts as an immunomodulator or is merely a surrogate marker of patient health or nutritional status are warranted. CI - Copyright (c) 2014 American Society for Blood and Marrow Transplantation. Published by Elsevier Inc. All rights reserved. FAU - Hansson, Magnus E A AU - Hansson ME AD - Division of Pediatrics, Department of Clinical Science, Intervention, and Technology, Karolinska Institutet, Stockholm, Sweden. Electronic address: magnus.e.a.hansson@ki.se. FAU - Norlin, Anna-Carin AU - Norlin AC AD - Division of Therapeutic Immunology, Department of Laboratory Medicine, Karolinska Institutet, Stockholm, Sweden; Department of Clinical Immunology and Transfusion Medicine, Karolinska University Laboratory, Karolinska University Hospital, Stockholm, Sweden. FAU - Omazic, Brigitta AU - Omazic B AD - Division of Therapeutic Immunology, Department of Laboratory Medicine, Karolinska Institutet, Stockholm, Sweden; Department of Clinical Immunology and Transfusion Medicine, Karolinska University Laboratory, Karolinska University Hospital, Stockholm, Sweden. FAU - Wikstrom, Ann-Charlotte AU - Wikstrom AC AD - Department of Clinical Immunology and Transfusion Medicine, Karolinska University Laboratory, Karolinska University Hospital, Stockholm, Sweden; Department of Biosciences and Nutrition, Karolinska Institutet, Stockholm, Sweden. FAU - Bergman, Peter AU - Bergman P AD - Center for Infectious Medicine, Department of Medicine, Karolinska Institutet, Stockholm, Sweden; Department of Clinical Microbiology, Karolinska University Laboratory, Karolinska University Hospital, Stockholm, Sweden. FAU - Winiarski, Jacek AU - Winiarski J AD - Division of Pediatrics, Department of Clinical Science, Intervention, and Technology, Karolinska Institutet, Stockholm, Sweden; Hematology/Immunology/SCT Section, Astrid Lindgren Children's Hospital, Stockholm, Sweden. FAU - Remberger, Mats AU - Remberger M AD - Division of Therapeutic Immunology, Department of Laboratory Medicine, Karolinska Institutet, Stockholm, Sweden; Center for Allogeneic Stem Cell Transplantation, Karolinska University Hospital, Stockholm, Sweden. FAU - Sundin, Mikael AU - Sundin M AD - Division of Pediatrics, Department of Clinical Science, Intervention, and Technology, Karolinska Institutet, Stockholm, Sweden; Hematology/Immunology/SCT Section, Astrid Lindgren Children's Hospital, Stockholm, Sweden. LA - eng PT - Journal Article PT - Research Support, Non-U.S. Gov't DEP - 20140605 PL - United States TA - Biol Blood Marrow Transplant JT - Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation JID - 9600628 RN - 0 (Myeloablative Agonists) RN - 1406-16-2 (Vitamin D) SB - IM MH - Acute Disease MH - Adolescent MH - Child MH - Child, Preschool MH - Chronic Disease MH - Female MH - Follow-Up Studies MH - Graft vs Host Disease/blood/immunology/mortality/*therapy MH - Hematologic Diseases/blood/immunology/mortality/*therapy MH - *Hematopoietic Stem Cell Transplantation MH - Humans MH - Infant MH - Infant, Newborn MH - Male MH - Myeloablative Agonists/therapeutic use MH - Prognosis MH - Recurrence MH - Retrospective Studies MH - Survival Analysis MH - *Transplantation Conditioning MH - Transplantation, Homologous MH - Vitamin D/*blood MH - Vitamin D Deficiency/blood/immunology/mortality/*therapy MH - Young Adult OTO - NOTNLM OT - 25-OH-vitamin D OT - Bone marrow transplantation OT - Micronutrient EDAT- 2014/06/10 06:00 MHDA- 2015/05/20 06:00 CRDT- 2014/06/10 06:00 PHST- 2014/03/31 [received] PHST- 2014/05/28 [accepted] PHST- 2014/06/05 [aheadofprint] AID - S1083-8791(14)00330-9 [pii] AID - 10.1016/j.bbmt.2014.05.030 [doi] PST - ppublish SO - Biol Blood Marrow Transplant. 2014 Oct;20(10):1537-43. doi: 10.1016/j.bbmt.2014.05.030. Epub 2014 Jun 5. PMID- 24907627 OWN - NLM STAT- MEDLINE DA - 20140912 DCOM- 20150519 LR - 20150415 IS - 1523-6536 (Electronic) IS - 1083-8791 (Linking) VI - 20 IP - 10 DP - 2014 Oct TI - Donor chimerism early after reduced-intensity conditioning hematopoietic stem cell transplantation predicts relapse and survival. PG - 1516-21 LID - 10.1016/j.bbmt.2014.05.025 [doi] LID - S1083-8791(14)00323-1 [pii] AB - The impact of early donor cell chimerism on outcomes of T cell-replete reduced-intensity conditioning (RIC) hematopoietic stem cell transplantation (HSCT) is ill defined. We evaluated day 30 (D30) and 100 (D100) total donor cell chimerism after RIC HSCT undertaken between 2002 and 2010 at our institution, excluding patients who died or relapsed before D30. When available, donor T cell chimerism was also assessed. The primary outcome was overall survival (OS). Secondary outcomes included progression-free survival (PFS), relapse, and nonrelapse mortality (NRM). We evaluated 688 patients with hematologic malignancies (48% myeloid and 52% lymphoid) and a median age of 57 years (range, 18 to 74) undergoing RIC HSCT with T cell-replete donor grafts (97% peripheral blood; 92% HLA-matched), with a median follow-up of 58.2 months (range, 12.6 to 120.7). In multivariable analysis, total donor cell and T cell chimerism at D30 and D100 each predicted RIC HSCT outcomes, with D100 total donor cell chimerism most predictive. D100 total donor cell chimerism <90% was associated with increased relapse (hazard ratio [HR], 2.54; 95% confidence interval [CI], 1.83 to 3.51; P < .0001), impaired PFS (HR, 2.01; 95% CI, 1.53 to 2.65; P < .0001), and worse OS (HR, 1.50; 95% CI, 1.11 to 2.04, P = .009), but not with NRM (HR, .76; 95% CI, .44 to 2.27; P = .33). There was no additional utility of incorporating sustained D30 to D100 total donor cell chimerism or T cell chimerism. Low donor chimerism early after RIC HSCT is an independent risk factor for relapse and impaired survival. Donor chimerism assessment early after RIC HSCT can prognosticate for long-term outcomes and help identify high-risk patient cohorts who may benefit from additional therapeutic interventions. CI - Copyright (c) 2014 American Society for Blood and Marrow Transplantation. Published by Elsevier Inc. All rights reserved. FAU - Koreth, John AU - Koreth J AD - Hematologic Malignancies Division, Dana-Farber Cancer Institute, Boston, Massachusetts. Electronic address: john_koreth@dfci.harvard.edu. FAU - Kim, Haesook T AU - Kim HT AD - Division of Biostatistics and Computational Biology, Dana-Farber Cancer Institute, Boston, Massachusetts. FAU - Nikiforow, Sarah AU - Nikiforow S AD - Hematologic Malignancies Division, Dana-Farber Cancer Institute, Boston, Massachusetts. FAU - Milford, Edgar L AU - Milford EL AD - Tissue Typing Laboratory, Brigham and Women's Hospital, Boston, Massachusetts. FAU - Armand, Philippe AU - Armand P AD - Hematologic Malignancies Division, Dana-Farber Cancer Institute, Boston, Massachusetts. FAU - Cutler, Corey AU - Cutler C AD - Hematologic Malignancies Division, Dana-Farber Cancer Institute, Boston, Massachusetts. FAU - Glotzbecker, Brett AU - Glotzbecker B AD - Hematologic Malignancies Division, Dana-Farber Cancer Institute, Boston, Massachusetts. FAU - Ho, Vincent T AU - Ho VT AD - Hematologic Malignancies Division, Dana-Farber Cancer Institute, Boston, Massachusetts. FAU - Antin, Joseph H AU - Antin JH AD - Hematologic Malignancies Division, Dana-Farber Cancer Institute, Boston, Massachusetts. FAU - Soiffer, Robert J AU - Soiffer RJ AD - Hematologic Malignancies Division, Dana-Farber Cancer Institute, Boston, Massachusetts. FAU - Ritz, Jerome AU - Ritz J AD - Hematologic Malignancies Division, Dana-Farber Cancer Institute, Boston, Massachusetts. FAU - Alyea, Edwin P 3rd AU - Alyea EP 3rd AD - Hematologic Malignancies Division, Dana-Farber Cancer Institute, Boston, Massachusetts. LA - eng GR - P01 CA142106/CA/NCI NIH HHS/United States GR - P01 CA142106-06A1/CA/NCI NIH HHS/United States GR - R01 CA183559-01/CA/NCI NIH HHS/United States PT - Journal Article PT - Research Support, N.I.H., Extramural PT - Research Support, Non-U.S. Gov't DEP - 20140604 PL - United States TA - Biol Blood Marrow Transplant JT - Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation JID - 9600628 RN - 0 (Myeloablative Agonists) SB - IM MH - Adolescent MH - Adult MH - Aged MH - Female MH - Graft Survival MH - Hematologic Neoplasms/diagnosis/immunology/mortality/*therapy MH - *Hematopoietic Stem Cell Transplantation MH - Histocompatibility Testing MH - Humans MH - Male MH - Middle Aged MH - Myeloablative Agonists/therapeutic use MH - Prognosis MH - Recurrence MH - Retrospective Studies MH - Survival Analysis MH - T-Lymphocytes/*immunology/pathology MH - Tissue Donors MH - Transplantation Chimera/genetics/*immunology MH - Transplantation Conditioning/*methods MH - Transplantation, Homologous OTO - NOTNLM OT - Allogeneic transplantation OT - Chimerism OT - Reduced-intensity EDAT- 2014/06/08 06:00 MHDA- 2015/05/20 06:00 CRDT- 2014/06/08 06:00 PHST- 2014/04/07 [received] PHST- 2014/05/22 [accepted] PHST- 2014/06/04 [aheadofprint] AID - S1083-8791(14)00323-1 [pii] AID - 10.1016/j.bbmt.2014.05.025 [doi] PST - ppublish SO - Biol Blood Marrow Transplant. 2014 Oct;20(10):1516-21. doi: 10.1016/j.bbmt.2014.05.025. Epub 2014 Jun 4. PMID- 24907626 OWN - NLM STAT- MEDLINE DA - 20140912 DCOM- 20150519 IS - 1523-6536 (Electronic) IS - 1083-8791 (Linking) VI - 20 IP - 10 DP - 2014 Oct TI - Comparison of chimerism and minimal residual disease monitoring for relapse prediction after allogeneic stem cell transplantation for adult acute lymphoblastic leukemia. PG - 1522-9 LID - 10.1016/j.bbmt.2014.05.026 [doi] LID - S1083-8791(14)00324-3 [pii] AB - Little data are available on the relative merits of chimerism and minimal residual disease (MRD) monitoring for relapse prediction after allogeneic hematopoietic stem cell transplantation (HCT). We performed a retrospective analysis of serial chimerism assessments in 101 adult HCT recipients with acute lymphoblastic leukemia (ALL) and of serial MRD assessments in a subgroup of 22 patients. All patients had received myeloablative conditioning. The cumulative incidence of relapse was significantly higher in the patients with increasing mixed chimerism (in-MC) compared with those with complete chimerism, low-level MC, and decreasing MC, but the sensitivity of in-MC detection with regard to relapse prediction was only modest. In contrast, MRD assessment was highly sensitive and specific. Patients with MRD positivity after HCT had the highest incidence of relapse among all prognostic groups analyzed. The median time from MRD positivity to relapse was longer than the median time from detection of in-MC, but in some cases in-MC preceded MRD positivity. We conclude that MRD assessment is a powerful prognostic tool that should be included in the routine post-transplantation monitoring of patients with ALL, but chimerism analysis may provide additional information in some cases. Integration of these tools and clinical judgment should allow optimal decision making with regard to post-transplantation therapeutic interventions. CI - Copyright (c) 2014 American Society for Blood and Marrow Transplantation. Published by Elsevier Inc. All rights reserved. FAU - Terwey, Theis Helge AU - Terwey TH AD - Department of Hematology, Oncology and Tumor Immunology, Charite-University Medicine Berlin, Berlin, Germany. Electronic address: theis.terwey@charite.de. FAU - Hemmati, Philipp Georg AU - Hemmati PG AD - Department of Hematology, Oncology and Tumor Immunology, Charite-University Medicine Berlin, Berlin, Germany. FAU - Nagy, Marion AU - Nagy M AD - Institute of Legal Medicine and Forensic Sciences, Charite-University Medicine Berlin, Berlin, Germany. FAU - Pfeifer, Heike AU - Pfeifer H AD - Department of Medicine II, Goethe University Hospital, Frankfurt, Germany. FAU - Gokbuget, Nicola AU - Gokbuget N AD - Department of Medicine II, Goethe University Hospital, Frankfurt, Germany. FAU - Bruggemann, Monika AU - Bruggemann M AD - Department of Hematology, University Hospital Schleswig-Holstein, Kiel, Germany. FAU - Le Duc, Tanja Melinh AU - Le Duc TM AD - Department of Hematology, Oncology and Tumor Immunology, Charite-University Medicine Berlin, Berlin, Germany. FAU - le Coutre, Philipp AU - le Coutre P AD - Department of Hematology, Oncology and Tumor Immunology, Charite-University Medicine Berlin, Berlin, Germany. FAU - Dorken, Bernd AU - Dorken B AD - Department of Hematology, Oncology and Tumor Immunology, Charite-University Medicine Berlin, Berlin, Germany. FAU - Arnold, Renate AU - Arnold R AD - Department of Hematology, Oncology and Tumor Immunology, Charite-University Medicine Berlin, Berlin, Germany. LA - eng PT - Comparative Study PT - Journal Article DEP - 20140604 PL - United States TA - Biol Blood Marrow Transplant JT - Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation JID - 9600628 RN - 0 (Myeloablative Agonists) SB - IM MH - Adolescent MH - Adult MH - Bone Marrow/metabolism/pathology MH - Female MH - *Hematopoietic Stem Cell Transplantation MH - Humans MH - Male MH - Middle Aged MH - Myeloablative Agonists/therapeutic use MH - Neoplasm, Residual MH - Precursor Cell Lymphoblastic Leukemia-Lymphoma/genetics/immunology/mortality/*therapy MH - Prognosis MH - Recurrence MH - Retrospective Studies MH - Survival Analysis MH - Transplantation Chimera/genetics/*immunology MH - *Transplantation Conditioning MH - Transplantation, Homologous MH - Treatment Outcome OTO - NOTNLM OT - Acute lymphoblastic leukemia OT - Chimerism OT - Minimal residual disease OT - Relapse OT - Stem cell transplantation EDAT- 2014/06/08 06:00 MHDA- 2015/05/20 06:00 CRDT- 2014/06/08 06:00 PHST- 2014/03/26 [received] PHST- 2014/05/27 [accepted] PHST- 2014/06/04 [aheadofprint] AID - S1083-8791(14)00324-3 [pii] AID - 10.1016/j.bbmt.2014.05.026 [doi] PST - ppublish SO - Biol Blood Marrow Transplant. 2014 Oct;20(10):1522-9. doi: 10.1016/j.bbmt.2014.05.026. Epub 2014 Jun 4. PMID- 24906983 OWN - NLM STAT- MEDLINE DA - 20140901 DCOM- 20150514 IS - 1432-5195 (Electronic) IS - 0341-2695 (Linking) VI - 38 IP - 9 DP - 2014 Sep TI - Regenerative therapy with mesenchymal stem cells at the site of malignant primary bone tumour resection: what are the risks of early or late local recurrence? PG - 1825-35 LID - 10.1007/s00264-014-2384-0 [doi] AB - PURPOSE: There is concern that regenerative cell-based therapies at the site of malignant primary bone tumours could result in increased risk of local tumour recurrence. We therefore investigated the long-term risks for site-specific recurrences in patients who had received an autologous bone marrow derived mesenchymal stem cell suspension to improve healing at the host-to-allograft bone junction of the reconstruction after bone tumour resection. METHODS: A total of 92 patients were treated from 1993 to 2003 with bone marrow-derived mesenchymal stem cells after bone tumour resection. Patients were monitored for cancer incidence from the date of first operation (1993) until death, or until 31 December 2013. The mean follow-up time was 15.4 years (range ten to 20 years). The average number of MSCs returned to the patient was 234,000 MSCs +/- 215,000. The primary outcome was to evaluate the risk of tumorigenesis recurrence at the cell therapy treatment sites with radiographs and/or MRIs. The relative risk of cancer recurrence was expressed as the ratio of observed and expected number of cases according to three different control populations. RESULTS: Thirteen recurrences were found at the treatment sites among the 92 patients. The expected number of recurrences based on incidence in the three cohort populations was between 15 and 20 for the same cancer, age and sex distribution. The standardized incidence ratio (equal to observed cancers divided by expected cancers) for the entire follow-up period and for all recurrences was between 0.65 and 0.86 (95 % CI 0.60-1.20). CONCLUSION: This study found no increased cancer local recurrence risk in patients after application of autologous cell-based therapy using bone marrow-derived mesenchymal stem cells at the treatment site after an average follow-up period of 15.4 years, ranging from ten to 20 years. FAU - Hernigou, Philippe AU - Hernigou P AD - Orthopaedic Surgery, Hopital Henri Mondor, Creteil, France, philippe.hernigou@wanadoo.fr. FAU - Flouzat Lachaniette, Charles Henri AU - Flouzat Lachaniette CH FAU - Delambre, Jerome AU - Delambre J FAU - Chevallier, Nathalie AU - Chevallier N FAU - Rouard, Helene AU - Rouard H LA - eng PT - Journal Article DEP - 20140607 PL - Germany TA - Int Orthop JT - International orthopaedics JID - 7705431 SB - IM MH - Adolescent MH - Adult MH - Bone Neoplasms/*surgery/*therapy MH - Bone Regeneration MH - Bone Transplantation/*methods MH - Cell- and Tissue-Based Therapy/*methods MH - Child MH - Female MH - Follow-Up Studies MH - Humans MH - Incidence MH - Magnetic Resonance Imaging MH - Male MH - *Mesenchymal Stem Cell Transplantation MH - Neoplasm Recurrence, Local/*epidemiology MH - Retrospective Studies MH - Risk Factors MH - Time Factors MH - Treatment Outcome MH - Young Adult EDAT- 2014/06/08 06:00 MHDA- 2015/05/15 06:00 CRDT- 2014/06/08 06:00 PHST- 2014/05/08 [received] PHST- 2014/05/12 [accepted] PHST- 2014/06/07 [aheadofprint] AID - 10.1007/s00264-014-2384-0 [doi] PST - ppublish SO - Int Orthop. 2014 Sep;38(9):1825-35. doi: 10.1007/s00264-014-2384-0. Epub 2014 Jun 7. PMID- 24862638 OWN - NLM STAT- MEDLINE DA - 20140912 DCOM- 20150519 LR - 20150430 IS - 1523-6536 (Electronic) IS - 1083-8791 (Linking) VI - 20 IP - 10 DP - 2014 Oct TI - Mismatched related and unrelated donors for allogeneic hematopoietic cell transplantation for adults with hematologic malignancies. PG - 1485-92 LID - 10.1016/j.bbmt.2014.05.015 [doi] LID - S1083-8791(14)00313-9 [pii] AB - Two parallel phase II trials in adults with hematologic malignancies demonstrated comparable survival after reduced-intensity conditioning and transplantation of either 2 HLA-mismatched umbilical cord blood (UCB) units or bone marrow from HLA-haploidentical relatives. Donor choice is often subject to physician practice and institutional preference. Despite clear preliminary evidence of equipoise between HLA-haploidentical related donor and double unrelated donor UCB transplantation, the actual prospect of being randomized between these 2 very different donor sources is daunting to patients and their treating physicians alike. Under these circumstances, it is challenging to conduct a phase III randomized trial in which patients are assigned to the UCB or haploidentical bone marrow arms. Therefore, we aimed to provide an evidence-based review and recommendations for selecting donors for adults without an HLA-matched sibling or an HLA-matched adult unrelated donor. CI - Copyright (c) 2014 American Society for Blood and Marrow Transplantation. Published by Elsevier Inc. All rights reserved. FAU - Eapen, Mary AU - Eapen M AD - Center for International Blood and Marrow Transplant Research, Department of Medicine, Medical College of Wisconsin, Milwaukee, Wisconsin. Electronic address: meapen@mcw.edu. FAU - O'Donnell, Paul AU - O'Donnell P AD - Fred Hutchinson Cancer Research Center, University of Washington, Seattle, Washington. FAU - Brunstein, Claudio G AU - Brunstein CG AD - Blood and Marrow Transplant Program, University of Minnesota, Minneapolis, Minnesota. FAU - Wu, Juan AU - Wu J AD - EMMES Corporation, Rockville, Maryland. FAU - Barowski, Kate AU - Barowski K AD - EMMES Corporation, Rockville, Maryland. FAU - Mendizabal, Adam AU - Mendizabal A AD - EMMES Corporation, Rockville, Maryland. FAU - Fuchs, Ephraim J AU - Fuchs EJ AD - Sidney Kimmel Cancer Center, Johns Hopkins University, Baltimore, Maryland. LA - eng GR - P01 CA015396/CA/NCI NIH HHS/United States GR - U01 HL069294/HL/NHLBI NIH HHS/United States GR - U10 HL069294/HL/NHLBI NIH HHS/United States GR - U10 HL069294/HL/NHLBI NIH HHS/United States GR - U10 HL069301/HL/NHLBI NIH HHS/United States GR - U10 HL069330/HL/NHLBI NIH HHS/United States GR - U10 HL109137/HL/NHLBI NIH HHS/United States GR - U10 HL109526/HL/NHLBI NIH HHS/United States GR - U24 CA076518/CA/NCI NIH HHS/United States GR - U24 CA76518/CA/NCI NIH HHS/United States PT - Journal Article PT - Research Support, N.I.H., Extramural PT - Review DEP - 20140523 PL - United States TA - Biol Blood Marrow Transplant JT - Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation JID - 9600628 RN - 0 (HLA Antigens) RN - 0 (Myeloablative Agonists) SB - IM MH - *Bone Marrow Transplantation MH - *Cord Blood Stem Cell Transplantation MH - Decision Trees MH - Gene Expression MH - HLA Antigens/genetics/immunology MH - Haplotypes MH - Hematologic Neoplasms/genetics/immunology/mortality/*therapy MH - Histocompatibility Testing MH - Humans MH - Myeloablative Agonists/therapeutic use MH - Randomized Controlled Trials as Topic MH - Recurrence MH - Siblings MH - Survival Analysis MH - Transplantation Conditioning/*methods MH - Transplantation, Homologous MH - *Unrelated Donors PMC - PMC4163123 MID - NIHMS601053 OID - NLM: NIHMS601053 [Available on 10/01/15] OID - NLM: PMC4163123 [Available on 10/01/15] OTO - NOTNLM OT - Alternative donor transplantation OT - Donor selection algorithm OT - Hematologic malignancy EDAT- 2014/05/28 06:00 MHDA- 2015/05/20 06:00 CRDT- 2014/05/28 06:00 PMCR- 2015/10/01 00:00 PHST- 2014/05/13 [received] PHST- 2014/05/14 [accepted] PHST- 2014/05/23 [aheadofprint] AID - S1083-8791(14)00313-9 [pii] AID - 10.1016/j.bbmt.2014.05.015 [doi] PST - ppublish SO - Biol Blood Marrow Transplant. 2014 Oct;20(10):1485-92. doi: 10.1016/j.bbmt.2014.05.015. Epub 2014 May 23. PMID- 24840443 OWN - NLM STAT- MEDLINE DA - 20140520 DCOM- 20150512 IS - 1873-3573 (Electronic) IS - 0039-9140 (Linking) VI - 125 DP - 2014 Jul TI - Comprehensive plasma profiling for the characterization of graft-versus-host disease biomarkers. PG - 265-75 LID - 10.1016/j.talanta.2014.03.017 [doi] LID - S0039-9140(14)00198-2 [pii] AB - Acute graft-versus-host disease (aGVHD) remains a life-threatening complication of hematopoietic stem cell transplantation (HSCT) therefore limiting its application. To optimize the management of aGVHD and reduce therapy-related toxicity, early specific markers are needed. The main objective of this study was to uncover diagnostic biomarkers by comparing plasma protein profiles of patients at the time of acute GVHD diagnosis with those of patients undergoing HSCT without aGVHD. Additional analysis of samples taken 15 days before aGVHD diagnosis was also performed to evaluate the potential of our newly discovered biomarkers for early diagnosis. To get complementary information from plasma samples, we used three different proteomic approaches, namely 2D-DIGE, SELDI-TOF-MS and 2D-LC-MS(E). We identified and confirmed by the means of independent techniques, the differential expression of several proteins indicating significantly increased inflammation response and disturbance in the coagulation cascade. The variation of these proteins was already observed 15 days before GVHD diagnosis, suggesting the potential early detection of the disease before symptoms appearance. Finally, logistic regression analysis determined a composite biomarker panel comprising fibrinogen, fragment of fibrinogen beta chain, SAA, prothrombin fragments, apolipoprotein A1 and hepcidin that optimally discriminated patients with and without GVHD. The area under the receiver operating characteristic curve distinguishing these 2 groups was 0.95. CI - Copyright (c) 2014 Elsevier B.V. All rights reserved. FAU - De Bock, Muriel AU - De Bock M AD - GIGA Research (GIGA-I(3), GIGA-cancer, and GIGA-Neuroscience), University of Liege, CHU, B36, B-4000 Liege, Belgium. FAU - Beguin, Yves AU - Beguin Y AD - GIGA Research (GIGA-I(3), GIGA-cancer, and GIGA-Neuroscience), University of Liege, CHU, B36, B-4000 Liege, Belgium; Department of Medicine, Division of Hematology, University and CHU of Liege, Liege, Belgium. FAU - Leprince, Pierre AU - Leprince P AD - GIGA Research (GIGA-I(3), GIGA-cancer, and GIGA-Neuroscience), University of Liege, CHU, B36, B-4000 Liege, Belgium. FAU - Willems, Evelyne AU - Willems E AD - Department of Medicine, Division of Hematology, University and CHU of Liege, Liege, Belgium. FAU - Baron, Frederic AU - Baron F AD - GIGA Research (GIGA-I(3), GIGA-cancer, and GIGA-Neuroscience), University of Liege, CHU, B36, B-4000 Liege, Belgium; Department of Medicine, Division of Hematology, University and CHU of Liege, Liege, Belgium. FAU - Deroyer, Celine AU - Deroyer C AD - GIGA Research (GIGA-I(3), GIGA-cancer, and GIGA-Neuroscience), University of Liege, CHU, B36, B-4000 Liege, Belgium. FAU - Seidel, Laurence AU - Seidel L AD - Department of Statistics, University of Liege, CHU, B-4000 Liege, Belgium. FAU - Cavalier, Etienne AU - Cavalier E AD - Department of Clinical Chemistry, CHU of Liege, CHU, B-4000 Liege, Belgium. FAU - de Seny, Dominique AU - de Seny D AD - GIGA Research (GIGA-I(3), GIGA-cancer, and GIGA-Neuroscience), University of Liege, CHU, B36, B-4000 Liege, Belgium. FAU - Malaise, Michel AU - Malaise M AD - GIGA Research (GIGA-I(3), GIGA-cancer, and GIGA-Neuroscience), University of Liege, CHU, B36, B-4000 Liege, Belgium. FAU - Gothot, Andre AU - Gothot A AD - GIGA Research (GIGA-I(3), GIGA-cancer, and GIGA-Neuroscience), University of Liege, CHU, B36, B-4000 Liege, Belgium. FAU - Merville, Marie-Paule AU - Merville MP AD - GIGA Research (GIGA-I(3), GIGA-cancer, and GIGA-Neuroscience), University of Liege, CHU, B36, B-4000 Liege, Belgium. FAU - Fillet, Marianne AU - Fillet M AD - GIGA Research (GIGA-I(3), GIGA-cancer, and GIGA-Neuroscience), University of Liege, CHU, B36, B-4000 Liege, Belgium; Department of Analytical Pharmaceutical Chemistry, CIRM, Institute of Pharmacy, University of Liege, CHU, B36, B-4000 Liege, Belgium. Electronic address: Marianne.fillet@ulg.ac.be. LA - eng PT - Journal Article PT - Research Support, Non-U.S. Gov't DEP - 20140318 PL - Netherlands TA - Talanta JT - Talanta JID - 2984816R RN - 0 (Biological Markers) RN - 0 (Blood Proteins) RN - 0 (Proteome) SB - IM MH - Adolescent MH - Adult MH - Aged MH - Area Under Curve MH - Biological Markers/*blood MH - Blood Proteins/*analysis MH - Chromatography, Liquid MH - Cohort Studies MH - Electrophoresis, Gel, Two-Dimensional MH - Female MH - Graft vs Host Disease/*blood MH - Hematologic Neoplasms/blood/therapy MH - Hematopoietic Stem Cell Transplantation MH - Humans MH - Inflammation MH - Male MH - Middle Aged MH - Prospective Studies MH - Proteome MH - Proteomics/*methods MH - Regression Analysis MH - Spectrometry, Mass, Matrix-Assisted Laser Desorption-Ionization MH - Transplantation Conditioning MH - Transplantation, Homologous MH - Young Adult OTO - NOTNLM OT - Biomarkers OT - Clinical proteomics OT - Composite panel OT - Graft-versus-host disease EDAT- 2014/05/21 06:00 MHDA- 2015/05/13 06:00 CRDT- 2014/05/21 06:00 PHST- 2013/10/13 [received] PHST- 2014/02/28 [revised] PHST- 2014/03/11 [accepted] PHST- 2014/03/18 [aheadofprint] AID - S0039-9140(14)00198-2 [pii] AID - 10.1016/j.talanta.2014.03.017 [doi] PST - ppublish SO - Talanta. 2014 Jul;125:265-75. doi: 10.1016/j.talanta.2014.03.017. Epub 2014 Mar 18. PMID- 24802329 OWN - NLM STAT- MEDLINE DA - 20140507 DCOM- 20150514 IS - 1559-131X (Electronic) IS - 1357-0560 (Linking) VI - 31 IP - 6 DP - 2014 Jun TI - High-dose idarubicin plus busulfan as conditioning regimen to autologous stem cell transplantation: promising post-remission therapy for acute myeloid leukemia in first complete remission? PG - 980 LID - 10.1007/s12032-014-0980-x [doi] AB - The optimal post-remission therapy (PRT) for acute myeloid leukemia (AML) remains uncertain. We reported 32 AML patients in first complete remission (CR1) undergoing autologous hematopoietic stem cell transplantation (ASCT) with a characteristic conditioning regimen, termed I-Bu, based on high-dose idarubicin plus busulfan, which considerably strengthened antileukemic activity. Most patients were in better or intermediate-risk group except that cytogenetic or molecular risk information was missing for 18.7 % of the patients. Unpurged peripheral blood stem cells were used in all the cases. The adverse effects were mild and reversible. Only one case of transplant-related mortality was observed. All the patients in this study acquired hematopoietic reconstitution after ASCT. After a median follow-up of 30 (6-119) months, 24 patients (75.0 %) were alive in which 20 (62.5 %) patients were in continuous CR. There were 11 (34.4 %) patients who relapsed after HSCT. Cumulative relapse probability was about 40 % after 24 months. Median OS and DFS have not been reached. Patients in the better and intermediate-risk group had different clinical outcomes, but the differences were not statistically significant. ASCT with I-Bu regimen is possibly promising PRT for better and intermediate-risk AML patients in CR1. FAU - Hong, Ming AU - Hong M AD - Department of Hematology, The First Affiliated Hospital of Nanjing Medical University, Jiangsu Province Hospital, Nanjing, 210029, China. FAU - Miao, Kou-Rong AU - Miao KR FAU - Zhang, Run AU - Zhang R FAU - Lu, Hua AU - Lu H FAU - Liu, Peng AU - Liu P FAU - Xu, Wei AU - Xu W FAU - Chen, Li-Juan AU - Chen LJ FAU - Zhang, Su-Jiang AU - Zhang SJ FAU - Wu, Han-Xin AU - Wu HX FAU - Qiu, Hong-Xia AU - Qiu HX FAU - Li, Jian-Yong AU - Li JY FAU - Qian, Si-Xuan AU - Qian SX LA - eng PT - Clinical Trial PT - Journal Article PT - Research Support, Non-U.S. Gov't DEP - 20140507 PL - United States TA - Med Oncol JT - Medical oncology (Northwood, London, England) JID - 9435512 RN - G1LN9045DK (Busulfan) RN - ZRP63D75JW (Idarubicin) SB - IM MH - Adolescent MH - Adult MH - Busulfan/administration & dosage/*therapeutic use MH - Disease-Free Survival MH - Female MH - *Hematopoietic Stem Cell Transplantation/mortality MH - Humans MH - Idarubicin/*administration & dosage/therapeutic use MH - Leukemia, Myeloid, Acute/drug therapy/mortality/*therapy MH - Male MH - Middle Aged MH - Retrospective Studies MH - Transplantation Conditioning/*methods MH - Transplantation, Autologous MH - Treatment Outcome MH - Young Adult EDAT- 2014/05/08 06:00 MHDA- 2015/05/15 06:00 CRDT- 2014/05/08 06:00 PHST- 2013/11/08 [received] PHST- 2014/04/21 [accepted] PHST- 2014/05/07 [aheadofprint] AID - 10.1007/s12032-014-0980-x [doi] PST - ppublish SO - Med Oncol. 2014 Jun;31(6):980. doi: 10.1007/s12032-014-0980-x. Epub 2014 May 7. PMID- 24797542 OWN - NLM STAT- MEDLINE DA - 20140718 DCOM- 20150507 IS - 1179-2019 (Electronic) IS - 1174-5878 (Linking) VI - 16 IP - 4 DP - 2014 Aug TI - Improving outcomes in children with sickle cell disease: treatment considerations and strategies. PG - 255-66 LID - 10.1007/s40272-014-0074-4 [doi] AB - Over the past decades there has been a significant improvement in the care of patients with sickle cell disease (SCD) in high-income countries. However, more needs to be learned about the complex pathophysiology and the factors that contribute to the development of end organ damage from the disease. While antibiotic prophylaxis and appropriate treatment of infections have resulted in a significant reduction of early mortality, management of the painful episodes and prevention of organ damage remain a challenge. Hydroxyurea is the only medication approved as disease-modifying therapy, and bone marrow transplant as curative treatment is not available to most patients. In low-income countries with the highest disease burden, early mortality is high due to limited resources for systematic screening, early diagnosis, and disease management. In order to improve outcomes in patients with SCD in high-income countries, better and widespread implementation of known disease-modifying therapies and the development of newer therapies targeting key pathophysiologic pathways are required. In low-income countries with high disease burden, innovative approaches to develop low-cost diagnostic devices and treatments that can be implemented to scale are needed to combat early mortality from the disease. Sustainable solutions in low-resource settings require evidence-based affordable interventions that can be integrated into primary and secondary healthcare systems. FAU - Amid, Ali AU - Amid A AD - Department of Pediatrics, University of Toronto, Toronto, ON, Canada. FAU - Odame, Isaac AU - Odame I LA - eng PT - Journal Article PT - Review PL - New Zealand TA - Paediatr Drugs JT - Paediatric drugs JID - 100883685 RN - X6Q56QN5QC (Hydroxyurea) SB - IM MH - Acute Chest Syndrome/therapy MH - Anemia, Sickle Cell/complications/*therapy MH - Antibiotic Prophylaxis MH - Child MH - Costs and Cost Analysis MH - Hematopoietic Stem Cell Transplantation MH - Humans MH - Hydroxyurea/adverse effects/therapeutic use MH - Pain Management EDAT- 2014/05/07 06:00 MHDA- 2015/05/08 06:00 CRDT- 2014/05/07 06:00 AID - 10.1007/s40272-014-0074-4 [doi] PST - ppublish SO - Paediatr Drugs. 2014 Aug;16(4):255-66. doi: 10.1007/s40272-014-0074-4. PMID- 24760212 OWN - NLM STAT- MEDLINE DA - 20141002 DCOM- 20150518 IS - 1806-9460 (Electronic) IS - 1516-3180 (Linking) VI - 132 IP - 3 DP - 2014 TI - Genetic diversity among volunteer donors of bone marrow in southeastern Brazil, according to the HLA system. PG - 158-62 LID - S1516-31802014005000005 [pii] AB - CONTEXT AND OBJECTIVE: Checking the histocompatibility of the molecules of the human leukocyte antigen (HLA) system is vital for performing bone marrow transplantation with allogeneic material. The objective of this study was to characterize bone marrow donors according to gender, age, ethnicity and HLA groups at a regional hemotherapy center in Brazil. DESIGN AND SETTING: Descriptive study on registered donors at a regional hemotherapy center in a public university hospital in the southeastern region of Brazil. METHODS: The records of 66,780 donors who were registered between 2005 and June 2011 were consulted, and the variables studied were tabulated. RESULTS: There were equal numbers of male and female donors and 82.8% of them were under 45 years of age. In terms of ethnicity, 77.3% declared themselves to be white, 15.0% mixed race, 5.7% black and 2% others. In terms of immunogenetic characterization, the most frequent HLA-A allelic group was HLA-A*02, with 39.20% of the donors; in the HLA-B allelic group, the most common was HLA-B*35, with 14.18%; while in the HLA-DRB1 allelic group, the most frequent was HLA-DRB1*03, with 17.03%. Comparison between these results and data from the Brazilian Bone Marrow Donor Registry (REDOME) showed that there were demographic and immunogenetic differences due to the history of immigration in the region of Ribeirao Preto, in southeastern Brazil. CONCLUSIONS: The results reinforce the importance of understanding the demographic and immunogenic profile of regions of Brazil, in order to reduce the waiting time for a histocompatible donor. FAU - Roque, Leticia Sarni AU - Roque LS AD - Biomedical Scientist in the Regional Center for Hematology, Faculdade de Medicina de Ribeirao Preto, Universidade de Sao Paulo, Ribeirao Preto, Sao Paulo, Brazil. FAU - Telarolli-Junior, Rodolpho AU - Telarolli-Junior R AD - Department of Biological Sciences, Universidade Estadual Paulista, Araraquara, Sao Paulo, Brazil. FAU - Loffredo, Leonor Castro Monteiro AU - Loffredo LC AD - Department of Social Dentistry, Universidade Estadual Paulista, Araraquara, Sao Paulo, Brazil. LA - eng PT - Journal Article DEP - 20140414 PL - Brazil TA - Sao Paulo Med J JT - Sao Paulo medical journal = Revista paulista de medicina JID - 100897261 RN - 0 (HLA Antigens) RN - 0 (HLA-A Antigens) RN - 0 (HLA-B Antigens) RN - 0 (HLA-DRB1 Chains) SB - IM MH - Adolescent MH - Adult MH - Age Factors MH - *Bone Marrow MH - Bone Marrow Transplantation MH - Brazil MH - Female MH - *Genetic Variation MH - HLA Antigens/*genetics MH - HLA-A Antigens/genetics MH - HLA-B Antigens/genetics MH - HLA-DRB1 Chains/genetics MH - Humans MH - Male MH - Middle Aged MH - *Registries MH - Sex Factors MH - *Tissue Donors MH - Young Adult EDAT- 2014/04/25 06:00 MHDA- 2015/05/20 06:00 CRDT- 2014/04/25 06:00 PHST- 2012/10/27 [received] PHST- 2013/07/16 [accepted] PHST- 2014/04/14 [aheadofprint] AID - S1516-31802014005000005 [pii] PST - ppublish SO - Sao Paulo Med J. 2014;132(3):158-62. Epub 2014 Apr 14. PMID- 24728310 OWN - NLM STAT- MEDLINE DA - 20140901 DCOM- 20150514 IS - 1432-5195 (Electronic) IS - 0341-2695 (Linking) VI - 38 IP - 9 DP - 2014 Sep TI - Percutaneous grafting with bone marrow autologous concentrate for open tibia fractures: analysis of forty three cases and literature review. PG - 1845-53 LID - 10.1007/s00264-014-2342-x [doi] AB - PURPOSE: Tibial fractures are the most common lower limb fractures. Some criteria such as open fractures and increasing open stage are known to be associated with high delayed union and pseudarthrosis rate. In cases of delayed or nonunion, classical treatment is autologous cancelous bone graft which is associated with high morbidity rate. The ideal treatment would be a percutaneous harvesting and grafting technique. As bone marrow autologous concentrate (BMAC) presents both advantages, we evaluated this technique from 2002 to 2007. METHODS: This was a retrospective study of 43 cases of open tibial fractures with initial surgical treatment. The criteria of inclusion were open fracture and nonunion, delayed union or suspicion of delayed union. RESULTS: In 23 cases (53.5 %) BMAC was successful. The success group had received significantly more CFU-F than the failure group (469 vs 153.10(3), p = 0.013). A threshold of 360.10(3) CFU-F grafted could be established over which there was 100 % success. BMAC done before 110 days after fracture had 47 % success and BMAC done since 110 days after fracture had 73 % success. BMAC success rate decreased with increasing initial fracture skin open stage. There was no BMAC success in cases of a fracture with a remaining gap of more than 4 mm. We had no complications with the technique at the iliac harvesting zone and tibia injection point. CONCLUSION: BMAC is a technique that should be considered as one of the different alternatives for management of long-bone delayed and nonunion because of its effectiveness, low complication rate, preservation of bone stock and low cost. FAU - Le Nail, Louis-Romee AU - Le Nail LR AD - Service de Chirurgie Orthopedique et Traumatologique 2, Hopital Trousseau, Universite Francois-Rabelais de Tours, CHU de Tours, 37044, Tours cedex 09, France. FAU - Stanovici, Julien AU - Stanovici J FAU - Fournier, Joseph AU - Fournier J FAU - Splingard, Marie AU - Splingard M FAU - Domenech, Jorge AU - Domenech J FAU - Rosset, Philippe AU - Rosset P LA - eng PT - Case Reports PT - Journal Article PT - Review DEP - 20140413 PL - Germany TA - Int Orthop JT - International orthopaedics JID - 7705431 SB - IM MH - Administration, Cutaneous MH - Adolescent MH - Adult MH - Aged MH - Aged, 80 and over MH - Bone Marrow Transplantation/*methods MH - Bone Transplantation/*methods MH - Female MH - Fractures, Malunited/*surgery MH - Humans MH - Ilium/cytology MH - Male MH - Middle Aged MH - Retrospective Studies MH - Tibial Fractures/*surgery MH - Time Factors MH - Treatment Outcome MH - Young Adult EDAT- 2014/04/15 06:00 MHDA- 2015/05/15 06:00 CRDT- 2014/04/15 06:00 PHST- 2014/03/13 [received] PHST- 2014/03/23 [accepted] PHST- 2014/04/13 [aheadofprint] AID - 10.1007/s00264-014-2342-x [doi] PST - ppublish SO - Int Orthop. 2014 Sep;38(9):1845-53. doi: 10.1007/s00264-014-2342-x. Epub 2014 Apr 13. PMID- 24721685 OWN - NLM STAT- MEDLINE DA - 20140812 DCOM- 20150511 IS - 1543-2920 (Electronic) IS - 0899-8493 (Linking) VI - 26 IP - 3 DP - 2014 Aug TI - Exercise intolerance and the impact of physical activity in children treated with hematopoietic stem cell transplantation. PG - 358-64 LID - 10.1123/pes.2013-0156 [doi] AB - Hematopoietic stem-cell transplant (SCT) is increasingly used to treat children with cancer, and survival following SCT is improving. One predominant consequence of childhood cancer therapy is increased physical morbidity, which is worse in pediatric SCT recipients compared with children treated with chemotherapy or radiation alone. There are many factors that contribute to exercise intolerance and reduced physical function during the pretransplant, peritransplant, and posttransplant phases. These include side effects from chemotherapy or radiation, excessive immobility due to bed rest, infections, the negative effects of immunosuppressants, and graft vs host disease, all of which can impair cardiorespiratory fitness, muscle strength, and muscle function. Few studies have investigated the effects of exercise in childhood SCT recipients. In a small number of published studies, exercise interventions have been demonstrated to improve cardiorespiratory fitness, preserve or increase muscle mass, and improve muscle strength in children following SCT. The use of exercise as medicine may be a noninvasive and nonpharmaceutical treatment to target physical complications post-SCT. Researchers and health-care professionals should work together to develop exercise prescription guidelines for this unique and important population. FAU - West, Sarah L AU - West SL AD - Physiology and Experimental Medicine, The Hospital for Sick Children, Toronto, ON, Canada. FAU - Gassas, Adam AU - Gassas A FAU - Schechter, Tal AU - Schechter T FAU - Egeler, R Maarten AU - Egeler RM FAU - Nathan, Paul C AU - Nathan PC FAU - Wells, Greg D AU - Wells GD LA - eng PT - Journal Article PT - Research Support, Non-U.S. Gov't PT - Review DEP - 20140410 PL - United States TA - Pediatr Exerc Sci JT - Pediatric exercise science JID - 8909729 SB - IM MH - Child MH - Exercise/physiology/psychology MH - *Exercise Therapy/methods MH - *Exercise Tolerance MH - Hematopoietic Stem Cell Transplantation/*adverse effects MH - Humans EDAT- 2014/04/12 06:00 MHDA- 2015/05/12 06:00 CRDT- 2014/04/12 06:00 PHST- 2014/04/10 [aheadofprint] AID - 2013-0156 [pii] AID - 10.1123/pes.2013-0156 [doi] PST - ppublish SO - Pediatr Exerc Sci. 2014 Aug;26(3):358-64. doi: 10.1123/pes.2013-0156. Epub 2014 Apr 10. PMID- 24619150 OWN - NLM STAT- MEDLINE DA - 20140909 DCOM- 20150515 LR - 20150220 IS - 1573-2665 (Electronic) IS - 0141-8955 (Linking) VI - 37 IP - 5 DP - 2014 Sep TI - Effects of hematopoietic stem cell transplantation on acyl-CoA oxidase deficiency: a sibling comparison study. PG - 791-9 LID - 10.1007/s10545-014-9698-3 [doi] AB - OBJECTIVE: Acyl-CoA oxidase (ACOX1) deficiency is a rare disorder of peroxisomal very-long chain fatty acid oxidation. No reports detailing attempted treatment, longitudinal imaging, or neuropathology exist. We describe the natural history of clinical symptoms and brain imaging in two siblings with ACOX1 deficiency, including the younger sibling's response to allogeneic unrelated donor hematopoietic stem cell transplantation (HSCT). METHODS: We conducted retrospective chart review to obtain clinical history, neuro-imaging, and neuropathology data. ACOX1 genotyping were performed to confirm the disease. In vitro fibroblast and neural stem cell fatty acid oxidation assays were also performed. RESULTS: Both patients experienced a fatal neurodegenerative course, with late-stage cerebellar and cerebral gray matter atrophy. Serial brain magnetic resonance imaging in the younger sibling indicated demyelination began in the medulla and progressed rostrally to include the white matter of the cerebellum, pons, midbrain, and eventually subcortical white matter. The successfully engrafted younger sibling had less brain inflammation, cortical atrophy, and neuronal loss on neuro-imaging and neuropathology compared to the untreated older sister. Fibroblasts and stem cells demonstrated deficient very long chain fatty acid oxidation. INTERPRETATION: Although HSCT did not halt the course of ACOX1 deficiency, it reduced the extent of white matter inflammation in the brain. Demyelination continued because of ongoing neuronal loss, which may be due to inability of transplant to prevent progression of gray matter disease, adverse effects of chronic corticosteroid use to control graft-versus-host disease, or intervention occurring beyond a critical point for therapeutic efficacy. FAU - Wang, Raymond Y AU - Wang RY AD - Division of Metabolic Disorders, CHOC Children's, 1201 W. La Veta Blvd., Orange, CA, 92868, USA, rawang@choc.org. FAU - Monuki, Edwin S AU - Monuki ES FAU - Powers, James AU - Powers J FAU - Schwartz, Phillip H AU - Schwartz PH FAU - Watkins, Paul A AU - Watkins PA FAU - Shi, Yang AU - Shi Y FAU - Moser, Ann AU - Moser A FAU - Shrier, David A AU - Shrier DA FAU - Waterham, Hans R AU - Waterham HR FAU - Nugent, Diane J AU - Nugent DJ FAU - Abdenur, Jose E AU - Abdenur JE LA - eng GR - P30 CA006973/CA/NCI NIH HHS/United States PT - Case Reports PT - Comparative Study PT - Journal Article PT - Research Support, Non-U.S. Gov't DEP - 20140312 PL - Netherlands TA - J Inherit Metab Dis JT - Journal of inherited metabolic disease JID - 7910918 RN - EC 1.3.3.6 (Acyl-CoA Oxidase) SB - IM MH - Acyl-CoA Oxidase/*deficiency MH - Brain/pathology MH - Brain Diseases, Metabolic, Inborn/enzymology/pathology/*therapy MH - Child, Preschool MH - Fatal Outcome MH - Female MH - Head Movements/physiology MH - Hematopoietic Stem Cell Transplantation/*methods MH - Humans MH - Infant MH - Infant, Newborn MH - Male MH - Muscle Hypotonia/etiology MH - Neural Stem Cells/transplantation MH - Siblings MH - Treatment Outcome PMC - PMC4332804 MID - NIHMS659777 OID - NLM: NIHMS659777 OID - NLM: PMC4332804 EDAT- 2014/03/13 06:00 MHDA- 2015/05/16 06:00 CRDT- 2014/03/13 06:00 PHST- 2013/09/16 [received] PHST- 2014/02/17 [accepted] PHST- 2014/02/14 [revised] PHST- 2014/03/12 [aheadofprint] AID - 10.1007/s10545-014-9698-3 [doi] PST - ppublish SO - J Inherit Metab Dis. 2014 Sep;37(5):791-9. doi: 10.1007/s10545-014-9698-3. Epub 2014 Mar 12. PMID- 24444355 OWN - NLM STAT- MEDLINE DA - 20140826 DCOM- 20150512 IS - 1757-6512 (Electronic) IS - 1757-6512 (Linking) VI - 5 IP - 1 DP - 2014 TI - Considerations in designing systems for large scale production of human cardiomyocytes from pluripotent stem cells. PG - 12 LID - 10.1186/scrt401 [doi] AB - Human pluripotent stem cell (hPSC)-derived cardiomyocytes have attracted attention as an unlimited source of cells for cardiac therapies. One of the factors to surmount to achieve this is the production of hPSC-derived cardiomyocytes at a commercial or clinical scale with economically and technically feasible platforms. Given the limited proliferation capacity of differentiated cardiomyocytes and the difficulties in isolating and culturing committed cardiac progenitors, the strategy for cardiomyocyte production would be biphasic, involving hPSC expansion to generate adequate cell numbers followed by differentiation to cardiomyocytes for specific applications. This review summarizes and discusses up-to-date two-dimensional cell culture, cell-aggregate and microcarrier-based platforms for hPSC expansion. Microcarrier-based platforms are shown to be the most suitable for up-scaled production of hPSCs. Subsequently, different platforms for directing hPSC differentiation to cardiomyocytes are discussed. Monolayer differentiation can be straightforward and highly efficient and embryoid body-based approaches are also yielding reasonable cardiomyocyte efficiencies, whereas microcarrier-based approaches are in their infancy but can also generate high cardiomyocyte yields. The optimal target is to establish an integrated scalable process that combines hPSC expansion and cardiomyocyte differentiation into a one unit operation. This review discuss key issues such as platform selection, bioprocess parameters, medium development, downstream processing and parameters that meet current good manufacturing practice standards. FAU - Chen, Allen AU - Chen A FAU - Ting, Sherwin AU - Ting S FAU - Seow, Jasmin AU - Seow J FAU - Reuveny, Shaul AU - Reuveny S FAU - Oh, Steve AU - Oh S LA - eng PT - Journal Article PT - Review DEP - 20140121 PL - England TA - Stem Cell Res Ther JT - Stem cell research & therapy JID - 101527581 SB - IM MH - Humans MH - Myocytes, Cardiac/*cytology/metabolism MH - Pluripotent Stem Cells/*cytology/metabolism MH - Stem Cell Research MH - Stem Cell Transplantation/*methods/standards MH - Tissue Engineering/*methods/standards PMC - PMC4055057 OID - NLM: PMC4055057 EDAT- 2014/01/22 06:00 MHDA- 2015/05/13 06:00 CRDT- 2014/01/22 06:00 PHST- 2014/01/21 [aheadofprint] AID - scrt401 [pii] AID - 10.1186/scrt401 [doi] PST - epublish SO - Stem Cell Res Ther. 2014 Jan 21;5(1):12. doi: 10.1186/scrt401. PMID- 24211377 OWN - NLM STAT- MEDLINE DA - 20140127 DCOM- 20150511 IS - 1878-3511 (Electronic) IS - 1201-9712 (Linking) VI - 19 DP - 2014 Feb TI - Prophylactic role of long-term ultra-low-dose acyclovir for varicella zoster virus disease after allogeneic hematopoietic stem cell transplantation. PG - 26-32 LID - 10.1016/j.ijid.2013.09.020 [doi] LID - S1201-9712(13)00312-3 [pii] AB - OBJECTIVES: To evaluate the prophylactic role of long-term ultra-low-dose acyclovir for varicella zoster virus (VZV) disease after allogeneic hematopoietic stem cell transplantation (HSCT). METHODS: We evaluated 141 patients who were planned to receive acyclovir at 200mg/day until the end of immunosuppressive therapy and for at least 1 year after HSCT in our center between June 2007 and June 2012. RESULTS: The cumulative incidence of VZV disease after HSCT was 4.5% at 1 year and 18.3% at 2 years. Protocol violation was the only independent significant factor that increased the incidence of VZV disease (hazard ratio (HR) 7.50, 95% confidence interval (CI) 3.60-15.63). Excluding patients with protocol violation, the discontinuation of acyclovir was the only significant factor for the development of VZV disease (HR 5.90, 95% CI 1.56-22.37). Six patients experienced breakthrough VZV disease, but four of these six had not taken acyclovir for several weeks before breakthrough VZV disease. On the other hand, the cumulative incidence of VZV disease after the cessation of acyclovir was 28.4% at 1 year and 38.0% at 2 years. The proportion of disseminated VZV disease was only 7% and no patient died directly of VZV disease. CONCLUSIONS: This study shows that long-term ultra-low-dose acyclovir appears to be effective for preventing VZV disease, especially disseminated VZV disease, after allogeneic HSCT. We recommend continuing acyclovir until the end of immunosuppressive therapy and for at least 1 year after HSCT, but additional strategies such as the administration of varicella vaccine may be needed to eradicate VZV disease. CI - Copyright (c) 2013 The Authors. Published by Elsevier Ltd.. All rights reserved. FAU - Kawamura, Koji AU - Kawamura K AD - Division of Hematology, Saitama Medical Center, Jichi Medical University, 1-847, Amanuma-cho, Omiya-ku, Saitama-city, Saitama 330-8503, Japan. FAU - Wada, Hidenori AU - Wada H AD - Division of Hematology, Saitama Medical Center, Jichi Medical University, 1-847, Amanuma-cho, Omiya-ku, Saitama-city, Saitama 330-8503, Japan. FAU - Yamasaki, Ryoko AU - Yamasaki R AD - Division of Hematology, Saitama Medical Center, Jichi Medical University, 1-847, Amanuma-cho, Omiya-ku, Saitama-city, Saitama 330-8503, Japan. FAU - Ishihara, Yuko AU - Ishihara Y AD - Division of Hematology, Saitama Medical Center, Jichi Medical University, 1-847, Amanuma-cho, Omiya-ku, Saitama-city, Saitama 330-8503, Japan. FAU - Sakamoto, Kana AU - Sakamoto K AD - Division of Hematology, Saitama Medical Center, Jichi Medical University, 1-847, Amanuma-cho, Omiya-ku, Saitama-city, Saitama 330-8503, Japan. FAU - Ashizawa, Masahiro AU - Ashizawa M AD - Division of Hematology, Saitama Medical Center, Jichi Medical University, 1-847, Amanuma-cho, Omiya-ku, Saitama-city, Saitama 330-8503, Japan. FAU - Sato, Miki AU - Sato M AD - Division of Hematology, Saitama Medical Center, Jichi Medical University, 1-847, Amanuma-cho, Omiya-ku, Saitama-city, Saitama 330-8503, Japan. FAU - Machishima, Tomohito AU - Machishima T AD - Division of Hematology, Saitama Medical Center, Jichi Medical University, 1-847, Amanuma-cho, Omiya-ku, Saitama-city, Saitama 330-8503, Japan. FAU - Terasako, Kiriko AU - Terasako K AD - Division of Hematology, Saitama Medical Center, Jichi Medical University, 1-847, Amanuma-cho, Omiya-ku, Saitama-city, Saitama 330-8503, Japan. FAU - Kimura, Shun-Ichi AU - Kimura S AD - Division of Hematology, Saitama Medical Center, Jichi Medical University, 1-847, Amanuma-cho, Omiya-ku, Saitama-city, Saitama 330-8503, Japan. FAU - Kikuchi, Misato AU - Kikuchi M AD - Division of Hematology, Saitama Medical Center, Jichi Medical University, 1-847, Amanuma-cho, Omiya-ku, Saitama-city, Saitama 330-8503, Japan. FAU - Nakasone, Hideki AU - Nakasone H AD - Division of Hematology, Saitama Medical Center, Jichi Medical University, 1-847, Amanuma-cho, Omiya-ku, Saitama-city, Saitama 330-8503, Japan. FAU - Yamazaki, Rie AU - Yamazaki R AD - Division of Hematology, Saitama Medical Center, Jichi Medical University, 1-847, Amanuma-cho, Omiya-ku, Saitama-city, Saitama 330-8503, Japan. FAU - Kanda, Junya AU - Kanda J AD - Division of Hematology, Saitama Medical Center, Jichi Medical University, 1-847, Amanuma-cho, Omiya-ku, Saitama-city, Saitama 330-8503, Japan. FAU - Kako, Shinichi AU - Kako S AD - Division of Hematology, Saitama Medical Center, Jichi Medical University, 1-847, Amanuma-cho, Omiya-ku, Saitama-city, Saitama 330-8503, Japan. FAU - Tanihara, Aki AU - Tanihara A AD - Division of Hematology, Saitama Medical Center, Jichi Medical University, 1-847, Amanuma-cho, Omiya-ku, Saitama-city, Saitama 330-8503, Japan. FAU - Nishida, Junji AU - Nishida J AD - Division of Hematology, Saitama Medical Center, Jichi Medical University, 1-847, Amanuma-cho, Omiya-ku, Saitama-city, Saitama 330-8503, Japan. FAU - Kanda, Yoshinobu AU - Kanda Y AD - Division of Hematology, Saitama Medical Center, Jichi Medical University, 1-847, Amanuma-cho, Omiya-ku, Saitama-city, Saitama 330-8503, Japan. Electronic address: ycanda-tky@umin.ac.jp. LA - eng PT - Journal Article DEP - 20131106 PL - Canada TA - Int J Infect Dis JT - International journal of infectious diseases : IJID : official publication of the International Society for Infectious Diseases JID - 9610933 RN - 0 (Antiviral Agents) RN - X4HES1O11F (Acyclovir) SB - IM MH - Acyclovir/*administration & dosage MH - Adolescent MH - Adult MH - Aged MH - Antiviral Agents/*administration & dosage/therapeutic use MH - Female MH - Hematopoietic Stem Cell Transplantation/*adverse effects MH - Herpes Zoster/diagnosis/*prevention & control/virology MH - Herpesvirus 3, Human/*immunology MH - Humans MH - Incidence MH - Male MH - Middle Aged MH - Retrospective Studies MH - Risk Factors MH - Time Factors MH - Transplantation, Homologous/adverse effects MH - Virus Activation MH - Young Adult OTO - NOTNLM OT - Allogeneic hematopoietic stem cell transplantation OT - Disseminated VZV disease OT - Long-term acyclovir OT - Varicella zoster virus disease EDAT- 2013/11/12 06:00 MHDA- 2015/05/12 06:00 CRDT- 2013/11/12 06:00 PHST- 2013/04/28 [received] PHST- 2013/09/09 [revised] PHST- 2013/09/27 [accepted] PHST- 2013/11/06 [aheadofprint] AID - S1201-9712(13)00312-3 [pii] AID - 10.1016/j.ijid.2013.09.020 [doi] PST - ppublish SO - Int J Infect Dis. 2014 Feb;19:26-32. doi: 10.1016/j.ijid.2013.09.020. Epub 2013 Nov 6. PMID- 23965908 OWN - NLM STAT- MEDLINE DA - 20140311 DCOM- 20150512 LR - 20150328 IS - 1539-2570 (Electronic) IS - 0271-6798 (Linking) VI - 34 IP - 3 DP - 2014 Apr-May TI - Osteochondromas after radiation for pediatric malignancies: a role for expanded counseling for skeletal side effects. PG - 331-5 LID - 10.1097/BPO.0000000000000081 [doi] AB - BACKGROUND: A relationship has been reported between total body irradiation (TBI) and later development of osteochondromas in children who receive radiation therapy as conditioning before hematopoietic stem cell transplantation (HSCT). The goal of this study was to better characterize osteochondromas occurring in these children. METHODS: We identified all children (0 to 18 y) who received an allogeneic HSCT and TBI from 2000 to 2012 from a blood and marrow transplant (BMT) database. Thereafter, we identified those who developed osteochondromas through a chart review. In addition, we searched for diagnosis and operative codes from 1996 to 2012 in our pediatric orthopaedic clinical records, isolating osteochondroma patients with a history of radiation exposure. RESULTS: Four patients who underwent allogeneic HSCT and were later diagnosed with osteochondromas were identified from the BMT database (N=233 children); all 4 were among a group of 72 patients who received TBI. Three patients were identified from orthopaedic records. The cohort included 5 boys and 2 girls with acute lymphoblastic leukemia (N=5) or neuroblastoma (N=2), diagnosed at a median age of 2.0 years. Therapy for all patients included chemotherapy, radiation therapy (TBI, N=5; abdominal, N=2), and HSCT. A diagnosis of osteochondroma was made at a median age of 11.7 years (range, 5 to 16 y), on average 8.6 years after radiation therapy. Diagnosis was incidental in 2 patients and secondary to symptoms (pain or genu valgum) in 5. Locations of osteochondromas were the proximal tibia (N=3), distal tibia, distal femur, distal ulna, and the distal phalanx (N=1 each). Three patients underwent surgical resection. CONCLUSIONS: Children may be more likely to develop osteochondromas after early exposure to radiation therapy, which may cause pain and require surgical resection. To the best of our knowledge, this is the first reported case of a radiation-induced osteochondroma causing lower extremity malalignment. Patients typically present to the pediatric orthopaedist's attention when symptomatic, but there may be an expanded role for counseling for potential for long-term skeletal effects in this group. LEVEL OF EVIDENCE: Level IV, case series. FAU - King, Elizabeth A AU - King EA AD - Departments of *Orthopaedic Surgery daggerPediatrics & Communicable Diseases double daggerRadiation Oncology, University of Michigan, Mott Children's Hospital, Ann Arbor, MI. FAU - Hanauer, David A AU - Hanauer DA FAU - Choi, Sung Won AU - Choi SW FAU - Jong, Nahbee AU - Jong N FAU - Hamstra, Daniel A AU - Hamstra DA FAU - Li, Ying AU - Li Y FAU - Farley, Frances A AU - Farley FA FAU - Caird, Michelle S AU - Caird MS LA - eng GR - K23 AI091623/AI/NIAID NIH HHS/United States PT - Journal Article PL - United States TA - J Pediatr Orthop JT - Journal of pediatric orthopedics JID - 8109053 SB - IM MH - Adolescent MH - Bone Neoplasms/etiology/*radiography MH - Child MH - Child, Preschool MH - Cohort Studies MH - *Counseling/methods MH - Female MH - Hematopoietic Stem Cell Transplantation/*adverse effects MH - Humans MH - Infant MH - Male MH - Neoplasms, Radiation-Induced/etiology/*radiography MH - Osteochondroma/etiology/*radiography MH - Retrospective Studies MH - Whole-Body Irradiation/*adverse effects PMC - PMC3930617 MID - NIHMS539428 OID - NLM: NIHMS539428 OID - NLM: PMC3930617 EDAT- 2013/08/24 06:00 MHDA- 2015/05/13 06:00 CRDT- 2013/08/23 06:00 AID - 10.1097/BPO.0000000000000081 [doi] PST - ppublish SO - J Pediatr Orthop. 2014 Apr-May;34(3):331-5. doi: 10.1097/BPO.0000000000000081. PMID- 23895431 OWN - NLM STAT- MEDLINE DA - 20131007 DCOM- 20150511 IS - 1399-3062 (Electronic) IS - 1398-2273 (Linking) VI - 15 IP - 5 DP - 2013 Oct TI - Low-dose acyclovir prophylaxis for the prevention of herpes simplex virus disease after allogeneic hematopoietic stem cell transplantation. PG - 457-65 LID - 10.1111/tid.12118 [doi] AB - BACKGROUND: Currently, acyclovir (ACV) at 1000 mg/day is widely used as prophylaxis in the early phase of hematopoietic stem cell transplant (HSCT) in Japan. However, low-dose ACV (200 mg/day) has been shown to prevent varicella zoster virus reactivation in the middle and late phases of HSCT. METHODS: Therefore, in this study, we decreased the dose of ACV to 200 mg/day in the early phase after HSCT. We analyzed 93 consecutive herpes simplex virus (HSV)-seropositive patients who underwent allogeneic HSCT for the first time in our center between June 2007 and December 2011. RESULTS: Before August 2009, 38 patients received oral ACV at 1000 mg/day (ACV1000) until day 35 after HSCT, whereas 55 patients received oral ACV at 200 mg/day (ACV200) after September 2009. We compared the cumulative incidence of HSV infection in the 2 groups. Oral ACV was changed to intravenous administration because of intolerance in 66% and 45% of the patients in the ACV1000 and ACV200 groups, respectively (P = 0.060). The probability of severe stomatitis (Bearman grade II-III) was 76% and 60% in the ACV1000 and ACV200 groups, respectively (P = 0.12). The number of patients who developed HSV disease before day 100 after HSCT was 0 in the ACV1000 group and 2 in the ACV200 group, with a cumulative incidence of 3.6% (P = 0.43). HSV disease in the latter 2 patients was limited to the lips and tongue and was successfully treated with ACV or valacyclovir at a treatment dose. CONCLUSION: ACV at 200 mg/day appeared to be effective for preventing HSV disease in the early phase after HSCT. CI - (c) 2013 John Wiley & Sons A/S. FAU - Kawamura, K AU - Kawamura K AD - Division of Hematology, Saitama Medical Center, Jichi Medical University, Saitama, Japan. FAU - Wada, H AU - Wada H FAU - Yamasaki, R AU - Yamasaki R FAU - Ishihara, Y AU - Ishihara Y FAU - Sakamoto, K AU - Sakamoto K FAU - Ashizawa, M AU - Ashizawa M FAU - Sato, M AU - Sato M FAU - Machishima, T AU - Machishima T FAU - Terasako, K AU - Terasako K FAU - Kimura, S I AU - Kimura SI FAU - Kikuchi, M AU - Kikuchi M FAU - Nakasone, H AU - Nakasone H FAU - Yamazaki, R AU - Yamazaki R FAU - Kanda, J AU - Kanda J FAU - Kako, S AU - Kako S FAU - Tanihara, A AU - Tanihara A FAU - Nishida, J AU - Nishida J FAU - Kanda, Y AU - Kanda Y LA - eng PT - Journal Article DEP - 20130729 PL - Denmark TA - Transpl Infect Dis JT - Transplant infectious disease : an official journal of the Transplantation Society JID - 100883688 RN - 0 (Antiviral Agents) RN - X4HES1O11F (Acyclovir) SB - IM MH - Acyclovir/*administration & dosage MH - Adolescent MH - Adult MH - Aged MH - Antiviral Agents/*administration & dosage MH - Female MH - Hematopoietic Stem Cell Transplantation/*adverse effects MH - Herpes Simplex/drug therapy/*prevention & control MH - Humans MH - Incidence MH - Japan MH - Male MH - Middle Aged MH - Retrospective Studies MH - Simplexvirus/*drug effects MH - Transplantation, Homologous/adverse effects MH - Virus Activation MH - Young Adult OTO - NOTNLM OT - acyclovir OT - allogeneic hematopoietic stem cell transplantation OT - herpes simplex virus disease EDAT- 2013/07/31 06:00 MHDA- 2015/05/12 06:00 CRDT- 2013/07/31 06:00 PHST- 2012/12/02 [received] PHST- 2013/01/22 [revised] PHST- 2013/02/04 [revised] PHST- 2013/02/17 [accepted] PHST- 2013/07/29 [aheadofprint] AID - 10.1111/tid.12118 [doi] PST - ppublish SO - Transpl Infect Dis. 2013 Oct;15(5):457-65. doi: 10.1111/tid.12118. Epub 2013 Jul 29. PMID- 23890253 OWN - NLM STAT- MEDLINE DA - 20131007 DCOM- 20150511 IS - 1399-3062 (Electronic) IS - 1398-2273 (Linking) VI - 15 IP - 5 DP - 2013 Oct TI - Increased susceptibility for aspergillosis and post-transplant immune deficiency in patients with gene variants of TLR4 after stem cell transplantation. PG - 533-9 LID - 10.1111/tid.12115 [doi] AB - INTRODUCTION: Toll-like receptors (TLRs) detect invading pathogens through several pattern-recognition mechanisms and play a central role in the regulation of the immune system. In allogeneic hematopoietic stem cell transplantation (HSCT), the frequent opportunistic fungal infections remain an important cause of mortality and morbidity in these highly immunocompromised patients. METHODS: We analyzed 154 patients after allogeneic HSCT for acute leukemia for TLR4 gene variants 1063A/G (D299G) and 1363C/T (T399I) with their respective donors, and correlated the results with the incidence of invasive aspergillosis (IA) infection after transplant. RESULTS: Probable and proven IA in recipients was significantly increased if either recipients or donors exhibited one of the two TLR4 gene variants. In addition, recipients with TLR gene variants and IA showed a delayed T cell and NKT cell immune reconstitution after transplant. Increased susceptibility for IA was not associated with an increased rate of death-in-remission or decreased estimate for overall survival. CONCLUSION: These findings reinforce the importance of genetic variants in innate immunity and IA among the recipients of allogeneic HSCT. CI - (c) 2013 John Wiley & Sons A/S. FAU - Koldehoff, M AU - Koldehoff M AD - Department of Bone Marrow Transplantation, West German Cancer Center, Medical School of University of Duisburg-Essen, Essen, Germany. FAU - Beelen, D W AU - Beelen DW FAU - Elmaagacli, A H AU - Elmaagacli AH LA - eng PT - Journal Article PT - Research Support, Non-U.S. Gov't DEP - 20130725 PL - Denmark TA - Transpl Infect Dis JT - Transplant infectious disease : an official journal of the Transplantation Society JID - 100883688 RN - 0 (TLR4 protein, human) RN - 0 (Toll-Like Receptor 4) RN - Immune Deficiency Disease SB - IM MH - Adolescent MH - Adult MH - Aged MH - Aspergillosis/epidemiology/etiology/*genetics/immunology MH - Aspergillus/*isolation & purification MH - Female MH - Genetic Variation MH - Genotype MH - Hematopoietic Stem Cell Transplantation/*adverse effects MH - Humans MH - Immunocompromised Host MH - Immunologic Deficiency Syndromes MH - Incidence MH - Leukemia/complications MH - Male MH - Middle Aged MH - Polymorphism, Single Nucleotide/*genetics MH - Signal Transduction MH - Toll-Like Receptor 4/*genetics MH - Young Adult OTO - NOTNLM OT - HSCT OT - SNP OT - TLR4 OT - allogeneic transplantation OT - invasive aspergillosis EDAT- 2013/07/31 06:00 MHDA- 2015/05/12 06:00 CRDT- 2013/07/30 06:00 PHST- 2012/10/02 [received] PHST- 2013/01/09 [revised] PHST- 2013/02/03 [accepted] PHST- 2013/07/25 [aheadofprint] AID - 10.1111/tid.12115 [doi] PST - ppublish SO - Transpl Infect Dis. 2013 Oct;15(5):533-9. doi: 10.1111/tid.12115. Epub 2013 Jul 25. PMID- 23294601 OWN - NLM STAT- MEDLINE DA - 20141022 DCOM- 20150514 IS - 1555-3892 (Electronic) IS - 0963-6897 (Linking) VI - 23 IP - 9 DP - 2014 TI - Improvement in poor graft function after allogeneic hematopoietic stem cell transplantation upon administration of mesenchymal stem cells from third-party donors: a pilot prospective study. PG - 1087-98 LID - 10.3727/096368912X661319 [doi] AB - Poor graft function (PGF) is a refractory complication that occurs after allogeneic hematopoietic stem cell transplantation (allo-HSCT). In the present study, we prospectively evaluated the efficacy and safety of mesenchymal stem cells (MSCs) expanded from the bone marrow of a third-party donor to patients with PGF after allo-HSCT. Twenty patients with PGF (7 with primary and 13 with secondary PGF) received MSCs (1 x 10(6)/kg) one to three times at 28-day intervals. Seventeen patients were responsive to MSCs, whereas three were not. Within the first 100 days after MSC treatment, 13 patients developed 20 episodes of infection. Moreover, five patients experienced cytomegalovirus-DNA viremia, and seven experienced Epstein-Barr virus (EBV)-DNA viremia within the first 100 days after MSC treatment; three of the latter developed EBV-associated posttransplant lymphoproliferative disorders (PTLD) within the follow-up period. Grade II acute graft-versus-host disease (GVHD) occurred in one patient, and local chronic GVHD occurred in two patients after receiving MSC treatment, including one acute GVHD and one chronic GVHD, respectively, after accepting donor lymphocyte infusions due to PTLD. After a follow-up period of an average of 508 days (range 166-904 days) posttransplantation, 11 patients died. No short-term toxic side effects were observed after MSC treatment. Two patients experienced leukemic relapse. With the exception of three patients with PTLD, no secondary tumors occurred. These results indicate that MSCs derived from the bone marrow of a third-party donor are beneficial in the treatment of both primary and secondary PGF that develops after allo-HSCT. However, additional studies will be needed to determine whether such treatment might increase the risk of EBV infection and reactivation or the development of EBV-associated PTLD. FAU - Liu, Xiaodan AU - Liu X AD - Department of Hematology, Nanfang Hospital, Southern Medical University, Guangzhou, China. FAU - Wu, Meiqing AU - Wu M FAU - Peng, Yanwen AU - Peng Y FAU - Chen, Xiaoyong AU - Chen X FAU - Sun, Jing AU - Sun J FAU - Huang, Fen AU - Huang F FAU - Fan, Zhiping AU - Fan Z FAU - Zhou, Hongsheng AU - Zhou H FAU - Wu, Xiuli AU - Wu X FAU - Yu, Guopan AU - Yu G FAU - Zhang, Xian AU - Zhang X FAU - Li, Yonghua AU - Li Y FAU - Xiao, Yang AU - Xiao Y FAU - Song, Chaoyang AU - Song C FAU - Xiang, Andy Peng AU - Xiang AP FAU - Liu, Qifa AU - Liu Q LA - eng PT - Clinical Trial PT - Journal Article PT - Multicenter Study PT - Research Support, Non-U.S. Gov't PL - United States TA - Cell Transplant JT - Cell transplantation JID - 9208854 RN - 0 (DNA, Viral) SB - IM MH - Adolescent MH - Adult MH - Anemia, Aplastic/mortality/therapy MH - Cytomegalovirus/genetics MH - Cytomegalovirus Infections/etiology/virology MH - DNA, Viral/analysis MH - Epstein-Barr Virus Infections/etiology/virology MH - Female MH - Follow-Up Studies MH - Graft vs Host Disease/etiology MH - *Hematopoietic Stem Cell Transplantation/adverse effects MH - Herpesvirus 4, Human/genetics MH - Humans MH - Leukemia/mortality/therapy MH - Male MH - *Mesenchymal Stem Cell Transplantation/adverse effects MH - Mesenchymal Stromal Cells/cytology MH - Middle Aged MH - Pilot Projects MH - Prospective Studies MH - Survival Rate MH - Transplantation, Homologous MH - Young Adult EDAT- 2013/01/09 06:00 MHDA- 2015/05/15 06:00 CRDT- 2013/01/09 06:00 AID - 10.3727/096368912X661319 [doi] AID - content-cog_09636897_ct0832liu [pii] PST - ppublish SO - Cell Transplant. 2014;23(9):1087-98. doi: 10.3727/096368912X661319.