Related and unrelated donor transplantation for β-thalassemia major: Results of an international survey Journal Article


Authors: Li, C.; Mathews, V.; Kim, S.; George, B.; Hebert, K.; Jiang, H.; Li, C.; Zhu, Y.; Keesler, D. A.; Boelens, J. J.; Dvorak, C. C.; Agarwal, R.; Auletta, J. J.; Goyal, R. K.; Hanna, R.; Kasow, K.; Shenoy, S.; Smith, A. R.; Walters, M. C.; Eapen, M.
Article Title: Related and unrelated donor transplantation for β-thalassemia major: Results of an international survey
Abstract: Westudied 1110 patients with b-thalassemia major aged ≤25 years who received transplants with grafts from HLA-matched related (n 5 677; 61%), HLA-mismatched related (n 5 78; 7%), HLA-matched unrelated (n 5 252; 23%), and HLA-mismatched unrelated (n 5 103; 9%) donors between 2000 and 2016. Ninety percent of transplants were performed in the last decade. Eight-five percent of patients received ≥20 transfusions and 88% were inadequately chelated. All patients received myeloablative-conditioning regimen. Overall and event-free survival were highest for patients aged ≤6 years and after HLA-matched related and HLA-matched unrelated donor transplantation. The 5-year probabilities of overall survival for patients aged ≤6 years, 7 to 15 years, and 16 to 25 years, adjusted for donor type and conditioning regimen were 90%, 84%, and 63%, respectively (P <001). The corresponding probabilities for event-free survival were 86%, 80%, and 63% (P <.001). Overall and eventfree survival did not differ between HLA-matched related and HLA-matched unrelated donor transplantation (89% vs 87% and 86% vs 82%, respectively). Corresponding probabilities after mismatched related and mismatched unrelated donor transplantation were 73% vs 83% and 70% vs 78%. In conclusion, if transplantation is considered as a treatment option it should be offered early (age ≤6 years). An HLA-matched unrelated donor is a suitable alternative if an HLA-matched relative is not available. © 2019 by The American Society of Hematology.
Keywords: adolescent; adult; child; controlled study; event free survival; major clinical study; overall survival; busulfan; fludarabine; review; cyclophosphamide; melphalan; thiotepa; hla matching; myeloablative conditioning; beta thalassemia; blood transfusion; chelation therapy; bone marrow transplantation; hla antigen; thalassemia major; unrelated donor; treosulfan; human; male; female; priority journal; related donor
Journal Title: Blood Advances
Volume: 3
Issue: 17
ISSN: 2473-9529
Publisher: American Society of Hematology  
Date Published: 2019-09-10
Start Page: 2562
End Page: 2570
Language: English
DOI: 10.1182/bloodadvances.2019000291
PUBMED: 31471325
PROVIDER: scopus
PMCID: PMC6737407
DOI/URL:
Notes: Review -- Export Date: 1 October 2019 -- Source: Scopus
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MSK Authors
  1. Jaap Jan Boelens
    18 Boelens