HLA-matched related donor transplantation in children with transfusiondependent thalassaemia: A single-centre experience in a resource-limited setting – Sri Lanka

Transfusion-dependent thalassaemia (TDT) is a significant health burden in low- and middle-income countries (LMIC) such as Sri Lanka. In LMIC, haematopoietic stem cell transplant (HSCT) remains the only curative option as it's a low-cost treatment modality compared to gene therapy. We present the outcomes of the first government-funded HSCT programme for TDT at the Lady Ridgeway Hospital for Children (LRH), Colombo.
A retrospective analysis includes 14 children with confirmed TDT who underwent matched family donor transplants at LRH between December 2021 and June 2025.
The median age at transplant was 10 years and 2 months. Nine children were in Pesaro class 2 and three were in class 3. Myeloablative conditioning regimens used included Bu/Cy/ATG and Treo/Thio/Flu. All patients achieved neutrophil and platelet engraftment, with median times of 17 and 20 days, respectively. The most common complication was neutropenic fever, followed by sinusoidal obstructive syndrome (n=6), more frequent in the Bu/Cy/ATG group. Two patients developed grade 2-3 acute gut GVHD; no chronic GVHD was observed. At a median follow-up of 387 days, transplant-related mortality was 0%, with 100% overall and thalassaemia-free survival. Twelve patients maintained full donor chimerism; two had mixed chimerism, with one requiring donor lymphocyte infusion.
This single-centre experience demonstrates that HLA-matched sibling HSCT is a safe and effective curative treatment for TDT, even in a resource-limited government setting. With careful patient selection, appropriate conditioning and inter-national mentorship, HSCT can be successfully implemented and scaled in LMICs as part of national thalassaemia strategies.