عنوان

Related and unrelated donor transplantation for β-thalassemia major:

Number

LA37s8c658

Title Proper

Related and unrelated donor transplantation for β-thalassemia major:

General Material Designation

[Article]

First Statement of Responsibility

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

Title Proper by Another Author

results of an international survey.

Text of Note

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.

Date of Publication

2019

Title

UCSF

Electronic name

[Article]

275578

a

Y