Inhibition of Sickle Hemoglobin Polymerization in Patients with Sickle Cell Anemia

Investigational agent voxelotor significantly increased hemoglobin levels and reduced hemolysis compared with placebo.

Red blood cell sickling and hemolysis due to polymerization of sickle hemoglobin upon deoxygenation is a hallmark abnormality in those with sickle cell anemia. Accordingly, inhibition of polymerization is a rational therapeutic target, and a prior phase I/II study of voxelotor previously demonstrated safety. Here, investigators conducted an industry-sponsored, randomized, placebo-controlled, double-blind study to evaluate the efficacy and safety of voxelotor (1500-mg and 900-mg doses) in 274 patients with sickle cell anemia.

Eligible patients were aged 12 to 65 years and had confirmed sickle cell disease and between 1 and 10 vaso-occlusive crises (VOCs) in the prior year. Hydroxyurea was permitted if the dose was stable for 3 months, and two…

Reviewing Author

Brady L. Stein, MD, MHS

Disclosures

Leadership Positions in Professional SocietiesAmerican Society of Hematology (Senior Editor, ASH Self-Assessment Program; guideline development)

Brady L. Stein, MD, MHS

Disclosures

Leadership Positions in Professional SocietiesAmerican Society of Hematology (Senior Editor, ASH Self-Assessment Program; guideline development)

Inhibition of Sickle Hemoglobin Polymerization in Patients with Sickle Cell Anemia

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Reviewing Author