The evolving treatment landscape for children with sickle cell disease

Sickle cell disease is the most common inherited pathological haemoglobinopathy. Over the past 30 years, disease related morbidity and mortality have improved in high-income countries due to advances in preventive care and treatments. Established disease-modifying therapies, such as hydroxyurea (hydrocarbamide), are continuing to have an important role in the treatment of sickle cell disease, and newer agents also show promise. In the past 5 years, the US Food and Drug Administration approved three additional sickle cell disease-modifying medications, and new gene therapies have been developed as an alternative curative treatment to haematopoietic stem-cell transplantation. In this Review, we discuss the current treatment landscape for paediatric sickle cell disease and emerging innovations in care. We also review the need for close, long-term management for children receiving newer therapies and the importance of ongoing investment in people with sickle cell disease in low-income and middle-income countries.