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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) Publisher Pubmed



Miano M1 ; Eikema DJ2 ; De La Fuente J3 ; Bosman P2 ; Ghavamzadeh A4 ; Smiers F5 ; Sengelov H6 ; Yesilipek A7 ; Formankova R8 ; Bader P9 ; Diaz Perez MA10 ; Bertrand Y11 ; Niemeyer C12 ; Diallo S13 Show All Authors
Authors
  1. Miano M1
  2. Eikema DJ2
  3. De La Fuente J3
  4. Bosman P2
  5. Ghavamzadeh A4
  6. Smiers F5
  7. Sengelov H6
  8. Yesilipek A7
  9. Formankova R8
  10. Bader P9
  11. Diaz Perez MA10
  12. Bertrand Y11
  13. Niemeyer C12
  14. Diallo S13
  15. Ansari M14
  16. Bykova TA15
  17. Faraci M16
  18. Bonanomi S17
  19. Gozdzik J18
  20. Satti TM19
  21. Bodova I20
  22. Wolfl M21
  23. Rocha VG22
  24. Mellgren K23
  25. Rascon J24
  26. Holter W25
  27. Lange A26
  28. Meisel R27
  29. Beguin Y28
  30. Mozo Y29
  31. Krivan G30
  32. Sirvent A31
  33. Bruno B32
  34. Dalle JH33
  35. Onofrillo D34
  36. Giardino S16
  37. Risitano AM35
  38. De Latour RP36
  39. Dufour C1

Source: Transplantation and Cellular Therapy Published:2021


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. © 2021
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