Paediatric Bone Marrow Transplant Outcomes.
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OBJECTIVE: To evaluate clinical outcomes in paediatric patients after bone marrow transplantation (BMT), including overall survival (OS), post- transplant complications, and quality of life (QoL). STUDY DESIGN: An observational study. Place and Duration of the Study: Department of Bone Marrow Transplant, Gambat Institute of Medical Sciences, Gambat, Pakistan, from January 2020 to June 2025. METHODOLOGY: This study included all paediatric patients (age ≤15 years) who received BMT and had all necessary medical records and follow-up data. OS and complications were noted; disease-free survival (DFS) was estimated using the Kaplan-Meier method. RESULTS: A total of 59 paediatric patients were included, with a mean age of 9 ±4.1 years and male predominance (59.3%). Most patients (67.3%) belonged to low-income families. The most frequent indication for transplantation was beta-thalassaemia major (57.9%), followed by aplastic anaemia (32.2%) and acute myeloid leukaemia (5.1%). All donors were related, with siblings most commonly. Bone marrow was the predominant stem cell source (84.7%). Engraftment was achieved in 88.2% of cases, with median neutrophil and platelet recovery at 16 and 22 days, respectively. OS was 78%, and DFS was 74.6%. Acute graft-versus-host disease (GVHD) occurred in 16.9% and chronic GVHD in 15.8%. Major complications included mucositis (50.8%), neutropenic infections (59.3%), fungal infections (40.7%), and cytomegalovirus reactivation (23.7%). Graft failure occurred in 16.9% (n = 10) of patients, while neutropenic sepsis remained the leading cause of mortality. Thirty- four (57.9%) patients were able to resume normal daily activities, and 50% (n = 30) successfully returned to school. Among survivors, the majority rated their post-transplant QoL positively, with 25.4% (n = 15) rating it as excellent, 38.8% (n = 23) as good and 13.1% (n = 8) as poor. CONCLUSION: Paediatric BMT had 78% OS and 74.6% DFS. Strengthening infection control, post-transplant surveillance, and infrastructure investment remains essential for further improving outcomes in similar settings. KEY WORDS: Paediatric bone marrow transplantation, Resource-limited settings, Graft-versus-host disease, Transplant outcomes, beta-thalassaemia major.