en
Scientific article
Open access
English

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)

Published inTransplantation and cellular therapy, vol. 27, no. 3, p. 274.e1-274.e5
First online date2020-12-25
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.

eng
Keywords
  • Bone Marrow Failure
  • Congenital disorder
  • Diamond-Blackfan Anemia
  • Stem Cell Transplantation
  • Anemia, Aplastic / therapy
  • Anemia, Diamond-Blackfan / therapy
  • Bone Marrow
  • Child
  • Hematopoietic Stem Cell Transplantation
  • Humans
  • Retrospective Studies
Citation (ISO format)
MIANO, Maurizio et al. 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). In: Transplantation and cellular therapy, 2020, vol. 27, n° 3, p. 274.e1–274.e5. doi: 10.1016/j.jtct.2020.12.024
Main files (1)
Article (Published version)
Identifiers
ISSN of the journal2666-6367
32views
4downloads

Technical informations

Creation10/03/2022 12:21:17 PM
First validation08/10/2023 7:35:12 AM
Update time08/10/2023 7:35:12 AM
Status update08/10/2023 7:35:12 AM
Last indexation02/01/2024 10:30:48 AM
All rights reserved by Archive ouverte UNIGE and the University of GenevaunigeBlack