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Stem Cell Transplantation for Diamond-Blackfan Anemia. A Retrospective Study on Behalf of the Severe Aplastic Anemia Working Party of the European Blood and Marrow Transplantation Group (EBMT)
M. Miano, DJ. Eikema, J. de la Fuente, P. Bosman, A. Ghavamzadeh, F. Smiers, H. Sengeløv, A. Yesilipek, R. Formankova, P. Bader, MÁ. Díaz Pérez, Y. Bertrand, C. Niemeyer, S. Diallo, M. Ansari, TA. Bykova, M. Faraci, S. Bonanomi, J. Gozdzik, TM....
Jazyk angličtina Země Spojené státy americké
Typ dokumentu časopisecké články
- MeSH
- aplastická anemie * terapie MeSH
- Diamondova-Blackfanova anemie * terapie MeSH
- dítě MeSH
- kostní dřeň MeSH
- lidé MeSH
- retrospektivní studie MeSH
- transplantace hematopoetických kmenových buněk * MeSH
- Check Tag
- dítě MeSH
- lidé MeSH
- Publikační typ
- časopisecké články MeSH
Data on stem cell transplantation (SCT) for Diamond-Blackfan Anemia (DBA) is limited. We studied patients transplanted for DBA and registered in the EBMT database. Between 1985 and 2016, 106 DBA patients (median age, 6.8 years) underwent hematopoietic stem cell transplantation from matched-sibling donors (57%), unrelated donors (36%), or other related donors (7%), using marrow (68%), peripheral blood stem cells (20%), both marrow and peripheral blood stem cells (1%), or cord blood (11%). The cumulative incidence of engraftment was 86% (80% to 93%), and neutrophil recovery and platelet recovery were achieved on day +18 (range, 16 to 20) and +36 (range, 32 to 43), respectively. Three-year overall survival and event-free survival were 84% (77% to 91%) and 81% (74% to 89%), respectively. Older patients were significantly more likely to die (hazard ratio, 1.4; 95% confidence interval, 1.06 to 1.23; P < .001). Outcomes were similar between sibling compared to unrelated-donor transplants. The incidence of acute grades II to IV of graft-versus-host disease (GVHD) was 30% (21% to 39%), and the incidence of extensive chronic GVHD was 15% (7% to 22%). This study shows that SCT may represent an alternative therapeutic option for transfusion-dependent younger patients.
Armed Forces Bone Marrow Transplant Centre Rawalpindi Pakistan
BMT Unit Istituto Giannina Gaslini Genova Italy
Bone Marrow Transplantation Unit Saint Louis Hospital APHP Paris France
Center for Pediatric Oncology and Hematology Vilnius University Vilnius Lithuania
Centre for Haematology Imperial College London London United Kingdom
CHU de Liège University of Liège Liège Belgium
CHU Lille Hématogie pédiatrique Lille France
Churchill Hospital Oxford United Kingdom
Department of Clinical Medicine and Surgery University of Naples Naples Italy
Department of Hematology Jules Bordet Institute Brussels Belgium
Department of Pediatrics Leiden University Medical Center Leiden Netherlands
EBMT Statistics EBMT Data Office Leiden Netherlands
Haematology Unit IRCCS Istituto Giannina Gaslini Genova Italy
Haematology Unit Ospedale Civile Pescara Italy
Hematology and Immunology Department Hopital Robert Debré Université de Paris Paris France
Hematology Oncology and Stem Cell Transplantation Research Center Shariati Hospital Tehran Iran
Hospital Universitario La Paz Madrid Spain
Institute of Pediatric Hematology and Oncology Civil Hospital of Lyon Lyon France
Jagiellonian University Medical Collage Kraków Poland
MBBM Foundation University of Milano Bicocca San Gerardo Hospital Monza Italy
Medical Park Antalya Hospital Antalya Turkey
National Institute of Children's Diseases Bratislava Slovakia
Onco Hématologie Pédiatrique CHU de Montpellier Montpellier France
Rigshospitalet Copenhagen Copenhagen Denmark
Sahlgrenska University Hospital Gothenburg Sweden
Citace poskytuje Crossref.org
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