Efficacy and safety of mitapivat in patients with sickle cell disease (RISE UP): a phase 3, double-blind, global, randomised, placebo-controlled trial

Background

Persistent haemolytic anaemia and recurrent vaso-occlusion drive complications and accelerate mortality in sickle cell disease. Mitapivat, an oral allosteric activator of pyruvate kinase, increases ATP and decreases 2,3-DPG with the potential to improve haemolytic anaemia and reduce sickling. We aimed to evaluate the efficacy and safety of mitapivat in patients with sickle cell disease.

Methods