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)

Maurizio Miano, Dirk-Jan Eikema, Josu de la Fuente, Paul Bosman, Ardeshir Ghavamzadeh, Frans Smiers, Henrik Sengeløv, Akif Yesilipek, Renata Formankova, Peter Bader, Miguel Ángel Díaz Pérez, Yves Bertrand, Charlotte Niemeyer, Safiatou Diallo, Marc Ansari, Tatiana A Bykova, Maura Faraci, Sonia Bonanomi, Jolanta Gozdzik, Tariq Mahmood SattiIvana Bodova, Matthias Wölfl, Vanderson G Rocha, Karin Mellgren, Jelena Rascon, Wolfgang Holter, Andrzej Lange, Roland Meisel, Yves Beguin, Yasmina Mozo, Gergely Kriván, Anne Sirvent, Benedicte Bruno, Jean Hugues Dalle, Daniela Onofrillo, Stefano Giardino, Antonio M Risitano, Régis Peffault de Latour, Carlo Dufour

Abstract

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.

Original languageEnglish
JournalTransplantation and cellular therapy
Volume27
Issue number3
Pages (from-to)274.e1-274.e5
ISSN2666-6367
DOIs
Publication statusPublished - Mar 2021

Keywords

  • Anemia, Aplastic/therapy
  • Anemia, Diamond-Blackfan/therapy
  • Bone Marrow
  • Child
  • Hematopoietic Stem Cell Transplantation
  • Humans
  • Retrospective Studies

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