Results from a multicenter, randomized, clinical trial suggests that voxelotor, which inhibits sickle hemoglobin (HbS) polymerization, may be beneficial for patients with sickle cell disease. The phase 3 Hemoglobin Oxygen Affinity Modulation to Inhibit HbS Polymerization (HOPE) trial randomly assigned 274 participants with sickle cell disease (SCD) in a 1:1:1 ratio to receive either 1500 mg of voxelotor (N=90), 900 mg of voxelotor (N=92) or placebo (N=92) daily for up to 72 weeks. At 24 weeks, 51%, 33%, and 7% of the participants in the 1500 mg voxelotor group, 900 mg voxelotor group, and the placebo group, respectively, had an increase of hemoglobin of more than 1.0 g per deciliter. Hemoglobin levels increased in patients taking voxelotor and markers of hemolysis were reduced, regardless of concomitant hydroxyurea use or baseline anemia. Adverse events were similar in all three groups and were not thought to be associated with voxelotor. Voxelotor could substantially benefit patients with sickle cell disease, but additional studies are also needed to ensure the long-term safety of the drug.
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