Voxelotor in adolescents and adults with sickle cell disease (HOPE): long-term follow-up results of an international, randomised, double-blind, placebo-controlled, phase 3 trial

RCT (n=274) found treatment with this oral once-daily sickle haemoglobin polymerisation inhibitor resulted in improvements in haemoglobin concentrations vs placebo (mean change baseline to week 72: 1.0, 0.5 and 0.0 g/dL in 1500mg, 900 mg and placebo groups, respectively).

The Lancet Haematology