Haematopoietic stem cell transplantation for children with transfusion-dependent thalassaemia in Malaysia.

Authors

Xin Yee Chiew, Syaza Ab Rahman, Mohamad Shafiq Azanan, Shekhar Krishnan, Lee Lee Chan, Hai Peng Lin, Hany Ariffin

Published in

Singapore medical journal. Feb 02, 2026. Epub Feb 02, 2026.

Abstract

The study aimed to determine the outcome of children with transfusion-dependent thalassaemia (TDT) who underwent haematopoietic stem cell transplantation (HSCT) at Universiti Malaya Medical Centre, Malaysia.
This was a retrospective analysis of 115 children (aged <18 years) who underwent 128 transplant procedures between 1 June 1987 and 30 December 2023.
The study included 53 boys, and the median age at time of HSCT was 5 years. One-third of patients were in Pesaro class 3. The 15-year overall survival and thalassaemia-free survival (TFS) were 83% and 71%, respectively. Transplant-related mortality (TRM) was 16%. Multivariate analysis revealed that patients who were aged >7 years and did not receive pre-HSCT anti-thymocyte globulin (ATG) had the worst outcomes (TFS 52%). In 62 patients who received busulfan-cyclophosphamide conditioning, 32 had addition of ATG that improved TFS (78% vs. 64%, P = 0.02). Graft failure, seen in 26% of patients, was the main contributing factor to TRM. Omission of ATG in the conditioning regimen was the main cause of graft failure (relative risk [RR] 8.26, 95% confidence interval [CI] 1.68-40.51; P = 0.009). The incidence of Grade II-IV graft-versus-host disease (GVHD) was 26%. Non-inclusion of ATG also significantly increased the risk of GVHD (RR 8.52, 95% CI 1.95-37.25; P = 0.04).
Haematopoietic stem cell transplantation is a feasible curative option for children with TDT in Malaysia, with the best outcomes observed in patients undergoing transplantation before the age of 7 years. Our study also highlights the role of ATG in improving HSCT outcomes in this population.

PMID:
42466977
Bibliographic data and abstract were imported from PubMed on 17 Jul 2026.

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