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Open Access Publications from the University of California

Related and unrelated donor transplantation for β-thalassemia major: results of an international survey.

  • Author(s): Li, Chunfu
  • Mathews, Vikram
  • Kim, Soyoung
  • George, Biju
  • Hebert, Kyle
  • Jiang, Hua
  • Li, Changgang
  • Zhu, Yiping
  • Keesler, Daniel A
  • Boelens, Jaap Jan
  • Dvorak, Christopher C
  • Agarwal, Rajni
  • Auletta, Jeffery J
  • Goyal, Rakesh K
  • Hanna, Rabi
  • Kasow, Kimberly
  • Shenoy, Shalini
  • Smith, Angela R
  • Walters, Mark C
  • Eapen, Mary
  • et al.

We studied 1110 patients with β-thalassemia major aged ≤25 years who received transplants with grafts from HLA-matched related (n = 677; 61%), HLA-mismatched related (n = 78; 7%), HLA-matched unrelated (n = 252; 23%), and HLA-mismatched unrelated (n = 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 event-free 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.

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