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 Paediatric Haematology and Stem Cell Transplantation Central Hospital of Southern Pest National Institute of Hematology and Infectious Diseases Budapest Hungary

Department of Pediatrics Hematology Oncology and Hematopoietic Stem Cell Transplant Unit Hospital Infantil Universitario Niño Jesús Madrid Spain

Department of Pediatrics Leiden University Medical Center Leiden Netherlands

Division for Stem Cell Transplantation and Immunology Department for Children and Adolescents University Hospital Frankfurt Frankfurt Germany

Division of Pediatric Hematology and Oncology Department of Pediatrics and Adolescent Medicine Medical Center Faculty of Medicine University of Freiburg Freiburg Germany

Division of Pediatric Stem Cell Therapy Department of Pediatric Oncology Hematology and Clinical Immunology Heinrich Heine University Düsseldorf Germany

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

Lower Silesian Center for Cellular Transplantation Ludwik Hirszfeld Institute of Immunology and Experimental Therapy Polish Academy of Sciences Wrocław 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

Pediatric Blood and Marrow Transplantation Program Children's Hospital University Hospital of Würzburg Würzburg Germany

Pediatric Oncology and Hematology Department of Paediatrics Gynaecology and Obstetrics Geneva University Hospital Geneva Switzerland

Raisa Gorbacheva Memorial Scientific Institute of Children Oncology Hematology and Transplantation 1st Pavlov State Medical University of St Petersburg St Petersburg Russia

Rigshospitalet Copenhagen Copenhagen Denmark

Sahlgrenska University Hospital Gothenburg Sweden

St Anna Kinderspital Vienna Austria

University Hospital Motol Prague Czech Republic

References provided by Crossref.org