A Cost-Conscious, and Highly Successful Start-up Strategy for Hematopoietic Cell Transplantation in Low- and Middle-Income Countries Supported By DKMS

Sankalp India Foundation, in collaboration with DKMS and Cure2Children, developed a cost-conscious pediatric BMT model for low- and middle-income countries (LMICs). The strategy is supported by the DKMS Access to Treatment and Transplant program (ATT) and hinges on low-risk matched sibling donor (MSD) transplants for severe hemoglobinopathies delivered for an average cost of 12, 000 including follow up. Here we summarize the outcomes of 141 MSD BMTs for hemoglobinopathies done directly in LMICs and compare experienced vs. startup centres assisted by the DKMS ATT program. On-site and online expert guidance relying on a comprehensive online IT platform including electronical medical records and continuing quality improvement tools was implemented. Major emphasis was given to nurses’ training, and professional development. The protocol consisted of pre-transplant immunosuppression with fludarabine/dexamethasone followed one month later by fludarabine, busulfan and cyclophosphamide. G-CSF-primed bone marrow was used as stem cell source followed by cyclosporine/methotrexate prophylaxis. Moderate to severe Graft vs. Host Disease - and Event-Free Survival (GEFS) composite outcome was compared between established (BMJH-Bangalore, CIMS-Ahmedabad, SEAIT-Jaipur, India and ANTH-Islamabad, Pakistan) and startup centres (NCMC-Tashkent, Uzbekistan, HCH-Hue, Vietnam and Al Mujtaba Hospital-Kerbala, Iraq). A total of 141 consecutive first bone marrow transplants (BMTs) were included in the analysis, consisting 128 patients (median age: 6. 04 years; IQR: 4. 1–9. 1 years) from established centres and 13 patients (median age: 5. 12 years; IQR: 4. 1–6. 1 years) from startup centres. Of the 128 patients treated at established centres, 122 were diagnosed with thalassemia major and 6 with sickle cell disease (SCD). All 13 patients at the startup centres had thalassemia major with liver size ≤3 cm below the costal margin at transplantation. GEFS was 94% in the established centres and 100% in the startup centres. Overall survival was 97% in established centres and 100% in startup centres, with a median follow-up of 12. 8 months and 9. 2 months in established respective start up centers. No statistically significant difference in GEFS was observed between the centres as determined by log-rank analysis. The DKMS ATT platform focusing on low-risk MSD BMT for severe hemoglobinopathies seems to be a safe and effective BMT start up approach in LMICs maximizing initial success rates while promoting sustainability.