Optimizing outcomes and accessibility of matched sibling donor transplant for transfusion-dependent thalassemia in LMICs
10 affiliations
- 1Sankalp India Foundation, Bangalore, India
- 2Bhagwan Mahaweer Jain Hospital-Sankalp Centre for Pediatric Hematology Oncology and BMT, Bangalore, India
- 3Sankalp-Health1 Centre for Paediatric Hematology Oncology and Blood and Marrow Transplant, Ahmedabad, India
- 4Mahatma Gandhi Medical College and Hospital and South East Asian Institute of Technology, Jaipur, India
- 5Al-Mujtaba Hospital, Karbala, Iraq
- 6Dr Akbar Niazi Teaching Hospital, Islamabad, Pakistan
- 7Hue Central Hospital, Hue, Vietnam
- 8National Children’s Medical Center, Tashkent, Uzbekistan
- 9Jagriti InnoHealth Platforms Pvt Ltd, Bangalore, India
- 10Cure2Children Foundation, Florence, Italy
Abstract
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.
Related
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BibTeX
@article{reddy2026optimizing,
title = {{Optimizing outcomes and accessibility of matched sibling donor transplant for transfusion-dependent thalassemia in LMICs}},
author = {Mohana Reddy and Tejashree Sridhar and Vidhi Mayur and Geetha T. Lathe and Deepa Trivedi and Vaibhav Shah and Rakesh Dhanya and Lalith Parmar and Priya Marwah and Hayder Shahad Marzook and Aliya Batool and Kim Hoa Nguyen and Begali Saytov and Amit Sedai and Kumari Ankita and Rajat Kumar Agarwal and Lawrence Faulkner},
journal = {Blood Advances},
year = {2026},
month = jul,
doi = {10.1182/bloodadvances.2025019083},
url = {https://doi.org/10.1182/bloodadvances.2025019083},
note = {CC BY-NC-ND 4.0},
}
RIS
TY - JOUR
TI - Optimizing outcomes and accessibility of matched sibling donor transplant for transfusion-dependent thalassemia in LMICs
AU - Mohana Reddy
AU - Tejashree Sridhar
AU - Vidhi Mayur
AU - Geetha T. Lathe
AU - Deepa Trivedi
AU - Vaibhav Shah
AU - Rakesh Dhanya
AU - Lalith Parmar
AU - Priya Marwah
AU - Hayder Shahad Marzook
AU - Aliya Batool
AU - Kim Hoa Nguyen
AU - Begali Saytov
AU - Amit Sedai
AU - Kumari Ankita
AU - Rajat Kumar Agarwal
AU - Lawrence Faulkner
PY - 2026
DA - 2026/07/22
JO - Blood Advances
DO - 10.1182/bloodadvances.2025019083
UR - https://doi.org/10.1182/bloodadvances.2025019083
AB - 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.
SN - 2473-9529
SN - 2473-9537
ER -