Effectiveness of hydroxyurea (hydroxycarbamide) in a national sickle cell disease programme in Ghana.

Hydroxyurea (hydroxycarbamide; HU) has been shown to be a safe and effective drug for individuals living with sickle cell disease (SCD) in Africa. However, reports of large-scale use of HU outside of controlled trial settings are limited on the continent. Access to HU in Ghana has improved through a major public-private partnership aimed at enhancing holistic care for communities affected by SCD. We evaluated the effectiveness of this programme through the measurement of biomarkers identified from health records, changes in clinical outcomes and quality of life (QoL) reported through interviews with participants and/or caregivers. The haemoglobin (Hb) levels of participants enrolled in the programme between September 2019 and July 2023 (n = 1549) increased on average by 0.55 g/dL (p < 0.001), with 9.2% of participants achieving Hb ≥10 g/dL. Six hundred participants and caregivers interviewed from April to November 2023 reported a significant reduction in the number of pain crises, malarial episodes, incidence of blood transfusions and rate of hospitalisations. Adults and children with SCD reported improved QoL in areas of physical, emotional, social and school-related functioning. Lessons learned are expected to inform future efforts in Ghana and may support HU access programmes in other countries where SCD is highly endemic.