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

Area of Science:

• Hematology
• Pediatric Oncology
• Public Health

Background:

• Severe hemoglobinopathies, including sickle cell disease (SCD) and thalassemia, are major global childhood health threats.
• Hematopoietic stem cell transplantation (HSCT) is the only definitive cure for these conditions.
• Access to HSCT is limited in low- and middle-income countries (LMICs), despite high SCD prevalence.

Purpose of the Study:

• To summarize the experience of establishing HSCT programs in LMICs.
• To suggest strategies for developing new HSCT units in resource-limited settings.
• To highlight the feasibility and cost-effectiveness of HSCT in LMICs.

Main Methods:

• Review of HSCT program startup and outcomes supported by Cure2Children in LMICs.
• Analysis of transplant success rates and costs compared to high-income countries.
• Identification of key strategies for implementing HSCT services.

Main Results:

• Over 500 HSCT procedures performed in LMICs over 10 years.
• Outcomes in LMICs were comparable to high-income countries.
• HSCT in LMICs was achieved at a significantly lower cost.

Conclusions:

• HSCT is a viable and cost-effective cure for severe hemoglobinopathies in LMICs.
• Developing local HSCT services is crucial for improving patient outcomes.
• Combined efforts in prevention, early care, and transplant development are essential for managing SCD and thalassemia globally.