How to setup a successful transplant program for hemoglobinopathies in developing countries: The Cure2Children approach

Hematopoietic stem cell transplantation (HSCT) remains the only established definitive cure for severe hemoglobinopathies, such as sickle cell disease (SCD) and thalassemia-the most prevalent life-threatening non-communicable disease of childhood globally. HSCT can not only cure over 85% of children with a compatible sibling but also restore normal health-related quality of life in most cases who do not have major irreversible organ damage at transplant. In low- and middle-income countries (LMICs), particularly in sub-Saharan Africa, SCD carrier rate can be up to 30% and 1% of live births have SCD. Relatively simple and inexpensive measures such as newborn screening, early diagnosis, caregiver education, and timely institution of anti-pneumococcal prophylaxis and hydroxyurea therapy can substantially reduce SCD-related mortality and morbidity. Improved prevention and early care should proceed in parallel with the development of transplant services and hope for cure. Cure2Children, an Italian NGO, has supported the startup of several bone marrow transplantation programs in LMICs where over 500 transplants have been performed over the last 10 years, with outcomes not substantially different from high-income countries but at a fraction of the cost. This report summarizes this experience and suggests some strategies to set up new HSCT units.