Document Type

Article

Publication Date

9-10-2019

Identifier

DOI: 10.1182/bloodadvances.2019000291; PMCID: PMC6737407

Abstract

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.

Journal Title

Blood Adv

Volume

3

Issue

17

First Page

2562

Last Page

2570

MeSH Keywords

Adolescent; Adult; Age Factors; Child; Child, Preschool; Hematopoietic Stem Cell Transplantation; Histocompatibility; Humans; Myeloablative Agonists; Surveys and Questionnaires; Survival Analysis; Tissue Donors; Transplantation Conditioning; Transplantation, Homologous; Unrelated Donors; Young Adult; beta-Thalassemia

Keywords

Adolescent; Adult; Age Factors; Child; Child, Preschool; Hematopoietic Stem Cell Transplantation; Histocompatibility; Humans; Myeloablative Agonists; Surveys and Questionnaires; Survival Analysis; Tissue Donors; Transplantation Conditioning; Transplantation, Homologous; Unrelated Donors; Young Adult; beta-Thalassemia

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