*pediatric transplant data from a center in a remote area in interior sindh, Pakistan*

Abstract

Abstract Background: The Gambat Institute of Medical Sciences (GIMS), located in a remote region of interior Sindh, is only public-sector facility in Pakistan to provide free bone marrow transplantation services. GIMS was established to address the healthcare needs of a population facing widespread poverty, high infectious diseases burden, and low literacy rate. Since its inception, the center has successfully performed hundreds of bone marrow transplants, including a significant number in pediatric patients. This study evaluates clinical outcomes and challenges encountered while delivering pediatric transplants in a resource-limited setting Objective: This study aimed to evaluate clinical outcomes in pediatric patients, including overall survival, post-transplant complications, and overall quality of life. Study Design: This study was designed retrospectively. Duration and Place of the Study: This study was conducted at GIMS, a resource-limited tertiary care facility located in interior Sindh, Pakistan. The analysis focused exclusively on pediatric patients between January 15, 2020 and June 2025 Material and Methods: This study included all 129 BMT patients, including 59 pediatric patients (age ≤ 15 years) who received bone marrow transplants at the center throughout this time period. Patients were included if they had all necessary medical records and follow-up data. OS and DFS were estimated using the Kaplan-Meier method. Related and unrelated donors were selected as per our donor selection criteria. Bone marrow stem cell and Peripheral blood system (PBSC) were the most utilized sources of transplantation. Results: Results: Of the 59 pediatric patients analyzed, the median age was 8 years, with 64.4% being male. Most patients (81.4%) belonged to low socioeconomic backgrounds. The leading indication for transplant was BTM(72.9%), followed by SAA (23.7%), with less common cases including Fanconi anemia, acute leukemia, and inherited platelet disorders. All donors were related donors, with bone marrow being predominant stem cell source (84.7%). Engraftment was achieved in 89.8% of cases. OS and DFS rates were 79.7% and 71.2%, respectively. Post-transplant complications included acute GVHD in 27.1% and chronic GVHD in 13.6% of patients. CMV reactivation occurred in 18.6%, and fungal infections were observed in 10.2% of cases. Despite these complications, 40.7% of children successfully returned to school, reflecting encouraging functional recovery Outcomes: Despite these challenges, the overall survival rate for pediatric patients at GIMS was comparable to those reported in developed countries. This success is attributed to the dedicated efforts of the medical team, comprehensive post-transplant care, and the resilience of the patients and their families. The disease-free survival rate and quality of life post-transplant were encouraging, showcasing the center's ability to provide high-standard care despite limited resources. Challenges: The primary challenges encountered included managing infections, particularly in the context of poverty and limited access to clean water and sanitation. The lack of education among the population also posed difficulties in ensuring adherence to post-transplant care protocols. However, continuous community engagement and education efforts by the GIMS team have helped mitigate some of these issues. Conclusion: Gambat Institute of Medical Sciences is a beacon for pediatric hematological patients requiring bone marrow transplant in Pakistan. In spite of widespread poverty, high infection rates and low education levels in the region, our outcomes at GIMS are similar to those encountered in developed countries. The publication is an atestament to the value of public healthcare programs and high-quality care potentially available even in resource-constrained locations. Keywords: Pediatric bone marrow transplantation, resource-limited settings, graft-versus-host disease, transplant outcomes, developing countries, Gambat Institute of Medical Sciences