Matched Unrelated Donor (MUD) Hematopoietic Stem Cell Transplant in Thalassemia Patient: An Outcome-Based Analysis of Pediatric Cohort in Tertiary Cancer Care Centre of North India

Background & Aims Allogeneic hematopoietic stem cell transplantation (Allo-HSCT) stands as the sole curative option for the thalassaemia patient, apart from gene therapy. In India, improvement in accessibility of unrelated donors HSCT; has resulted in an increase in the subset of patients undergoing MUD-HSCT for hemoglobinopathies. Our objective is to assess the spectrum of outcomes in patients undergoing MUD-HSCT for transfusion dependent thalassemia (TDT). Material & Methods All pediatric thalassaemia patients who underwent MUD-HSCT from 2014 – Aug 2023 were included in the study. The cohort was followed up and analysed till Sep 2023. Overall-survival (OS), GvHD thalassaemia free survival (GTFS) and Thalassaemia-free-survival (TFS) were assessed. Descriptive statistics detailed central tendency, while Kaplan-Meier estimated OS and TFS. Results Eighteen patients were included with a median age at HSCT was 9.5 years (3 - 15). Male: Female ratio was 2.5: 1. Majority of the patients were in Nanfeng Class III group constituting 66.6% of the cohort. Median CD34 dose was 4.8 × 10*6/kg. Median time to neutrophils and platelets engraftment was 15 days (12 -22) and 15 days (10-26). CMV reactivation was seen in n=5 (26.3%) patients. GVHD was seen in n=14 (73.7%) patients (acute, n=9, chronic n=5) with most of the patients developing grade 1-2 skin GVHD. Rejection was observed in n=2 (10.5%). Treatment-Related-Mortality (TRM) at day +100 was n=2 (10.5%). During the last follow-up, 17 patients were alive and 2 patients died due to TRM within 100 days. At a median follow-up of 17.7 months, OS, GTFS and TFS was 89.5%, 63.2% and 79% respectively. Conclusion We assessed the outcomes of matched unrelated donor hematopoietic stem cell transplantation (MUD-HSCT) in thalassemia patients in India. Despite some complications, MUD-HSCT showed promising results, with high OS and TFS rates at a median follow-up of 17.7 months, highlighting its importance as a curative option when compatible family donors are unavailable.