June 19, marks World Sickle Cell Day, with the 2026 theme focusing on closing the survival gap and a push towards a universal cure. 20 years have passed since the WHO recognised sickle cell disease as a global health problem, and although some progress has been made, there is clearly still substantial work to be done to improve outcomes for those living with the disease around the world.
3 years ago, a Lancet Haematology Commission aimed to define global strategies to improve outcomes for sickle cell disease, focusing on five areas: epidemiology, screening and prevention, established and emerging treatments, cellular therapies, and training and education for health-care providers. With disease-modifying therapies, there have been many hopes and disappointments in recent years. Crizanlizumab and voxelotor have been removed from the American and European markets, and gene therapy remains expensive and inaccessible for those in low-income and middle-income countries. There could be new hope with mitapivat, which is under consideration for US FDA accelerated approval following the results of the phase 3 RISE UP trial. Mitapivat, compared with placebo, significantly improved haemoglobin response, but did not significantly reduce the annualised rate of sickle cell pain crises. Long-term follow-up will be needed to determine whether mitapivat will be a treatment option beyond hydroxurea.