Mohana Reddy

Severe thalassemia (ST) is a life-threatening condition common in many low- and middle-income countries. Although bone marrow transplantation (BMT) offers a curative option, its accessibility remains limited because of cost and complexity. This study describes a simplified approach to BMT for hemoglobinopathies that is based on relatively common and inexpensive medicines. This study included 176 pediatric patients with transfusion-dependent thalassemia who received a matched sibling BMT between February 2022 and November 2025 across several centers in Asia and the Middle East. A simplified and low-cost transplant protocol was used, using drugs that are widely available and are listed in the World Health Organization Model List of Essential Medicines. The regimen included fludarabine, dexamethasone, busulfan, and cyclophosphamide. granulocyte colony-stimulating factor–primed bone marrow was used as the graft source, and graft-versus-host disease (GVHD) prophylaxis included cyclosporine and methotrexate. Rejection, mixed-chimerism rates, transplant-related mortality, and GVHD-free, overall survival, and disease-free survival composite outcomes were analyzed. Engraftment was obtained in 97.3% of patients. Thalassemia-free survival was 93.4%, and overall survival was 96%, at a median follow-up of 11.3 months. Rates of moderate to severe acute GVHD were low (3.9%) for a GVHD-free and thalassemia-free composite actuarial outcome of 93.4%. Cytomegalovirus (CMV) reactivation was observed in 19.2% of patients and 1 (0.6%) patient progressed to CMV disease. The approach was equally effective in experienced and newly established transplant centers. A simple, safe, and cost-conscious transplant approach using widely available medicines can achieve excellent outcomes for children with ST in low-resource settings.

Background: Central nervous system (CNS) complications after bone marrow transplant (BMT) for benign hematological diseases mainly Thalassemia major have a major impact on overall survival, disease‐free survival, and transplant‐related mortality. Method: Retrospective data analysis of 595 patients after first matched‐related BMT in four participating centers in the subcontinent from 2010 to 2023 was done to assess the incidence, associated risk factors and outcome of transplant‐related intracranial hemorrhage (ICH). Result: The mean age of the patient population at transplant was 8.32 ± 4.09 years. The incidence of ICH was 4.7%. ICH presented with headache (60%) and hypertension (57%) mostly in the early post‐transplant period (< 28 days post‐BMT). Age at transplant was the positive predictor of developing ICH after BMT. Transplant‐related mortality and rejection was higher in patients with ICH than in the other patients. The overall survival and disease‐free survival of 28 patients with ICH out of 595 patients was 51% and 37%, respectively, as compared to 88% and 76% in the rest of the patients. Transplant‐related mortality was higher in patients with ICH than in the other patients, but there was no statistically significant difference in rejection between the two groups. Conclusion: Transplant‐related ICH affects the overall survival, disease‐free survival, and transplant‐related mortality. ICH presented early post‐BMT with headache and hypertension. Age at BMT was the main predictor that emerged at the time of developing ICH.