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Deoxygenated sickle cell hemoglobin (HbS) forms polymers that make red blood cells (RBCs) rigid and predisposed to aggregate formation, hemolysis, and vascular occlusion. Voxelotor binds to HbS, increasing its oxygen affinity and inhibiting polymer formation.
To examine the safety and potential benefit of voxelotor, investigators conducted an industry-sponsored, phase I/II, randomized, double-blind, placebo-controlled trial involving 38 adults with HbSS (sickle cell anemia) genotype but mild disease. Patients had a baseline Hb of 8 to 9 g/dL and had experienced ≤1 crisis per year requiring hospitalization. Voxelotor was given orally in doses ranging from 500 to 1000 mg once daily for up to 90 days.
The following were reported:
Markers of hemol…