Outcome of Haematopoietic Stem Cell Transplant in Beta-Thalassaemia Major: Single Centre Experience from a Low- and...

To determine the outcome of haematopoietic stem transplant (HSCT) in beta-thalassaemia major (BTM) patients.

Descriptive Study. Place and Duration of the Study: Department of Clinical Haematology, Armed Forces Bone Marrow Transplant Centre (AFBMTC), Rawalpindi, Pakistan, from April 2018 to December 2023.

All cases of BTM undergoing HLA-matched allogeneic HSCT after myeloablative conditioning were included. Cases undergoing second HSCT and HSCT with treosulfan-based conditioning were excluded. Age, gender, complications, mortality, and associated factors were evaluated.

This study analysed the data of 118 cases of BTM including 71 (60.2%) males undergoing HSCT after myeloablative conditioning during the study period. The mean age at the time of HSCT was 85.7 ± 33.6 months. Eighty-one (68.6%) cases were in Pesaro Class III. Neutropenic fever was the most common complication documented in 117 (99.2%) cases. Mortality at day-100 was 14 (11.9%). Overall treatment-related mortality (TRM) was 23 (19.4%). In univariate analysis, factors having a statistically significant association with TRM were graft failure (p = 0.001), Pesaro class (p = 0.03), severity of acute graft versus host disease (aGVHD) (p = 0.02), and veno-occlusive disease (VOD) (p = 0.02). The median follow-up time was 26.87 ± 16.60 months with overall survival (OS) and disease free survival (DFS) rates of 80.5% and 78.0%, respectively.

The OS of around 80% is promising which can be further improved with better transfusion services, regular iron chelation, and HSCT at a younger age.

Haematopoietic stem cell transplant, Beta-thalassaemia major, Pakistan.