Hydroxyurea therapy for sickle cell disease in low-income countries

Abstract

Sickle cell disease (SCD) is one of the most common genetic diseases worldwide. It is estimated that annually in Africa, more than 300,000 babies are born with SCD. In sub-Saharan Africa, SCD accounts for about 10 % of all under-5 mortality. Simple interventions including neonatal screening, penicillin prophylaxis, pneumococcal vaccination and malaria prevention significantly reduce early mortality. Disease-modifying therapies help prevent complications and improve quality of life and survival. Hydroxyurea therapy has proven clinical efficacy in patients with SCD in high-income settings, but studies in LICs are limited. Hydroxyurea, orally administered and relatively more affordable, appears to be a practical disease-modifying choice for SCD management in resource-poor settings. Implementation of hydroxyurea therapy is crucial in improving patients survival and quality of life, although considerable barriers exist to studies in LICs where hydroxyurea therapy could have the most impact. Collaborative north-south partnerships are now supporting the initiation of pediatric trials in Africa.