Hemoglobinopathies

In the placebo-controlled international HOPE trial, 274 participants, most of whom had sickle cell anemia and two-thirds of whom were receiving hydroxyurea at baseline, were randomly assigned in a 1:1:1 ratio to receive voxelotor orally once daily at 1500 mg or 900 mg, or placebo. Voxelotor significantly increased Hb levels while reducing markers of hemolysis, results that the authors noted “are consistent with inhibition of HbS polymerization and indicate a disease-modifying potential.”