Advancing Sickle Cell Disease Treatment in Sub-Saharan Africa: Challenges and Opportunities for Disease Modifying Therapies.

Sickle cell disease (SCD) is a major individual and public health challenge in sub-Saharan Africa, where over 5 million affected individuals live and where access to disease-modifying treatment is limited. Despite established safety and efficacy of hydroxyurea, its use is limited across the region due to inconsistent healthcare infrastructure, high medication and laboratory costs, inadequate clinician training, and persistent disease stigma. Practical hydroxyurea dosing strategies, integration into national health plans, and a stronger supply chain are necessary to improve access to this life-saving medication. Newly approved medications, such as L-glutamine and crizanlizumab, may provide additional benefits to select patients, but are expensive and unavailable. The increased mortality observed in people on voxelotor in Africa highlights the need to ensure the safety of any new medication in varied settings through high-quality research conducted on the continent. Overall, holistic strategies are needed to improve SCD care in Africa, such as universal screening with connection to comprehensive care that includes disease-modifying treatment, community and healthcare worker education, centers of excellence, and capacity building. SCD management in Africa can be transformed by addressing systemic barriers and leveraging collaborative partnerships, leading to reduced mortality and alleviation of the individual and economic burdens of the disease. It is a moral and economic imperative to prioritize access to SCD treatment in Africa, the region with the greatest disease burden globally.