Key clinical point: The novel agent voxelotor improves hemoglobin and reduces hemolysis in sickle cell disease.
Major finding: Patients on 1,500 mg of voxelotor had significantly better hemoglobin response compared with patients on placebo (P less than .001).
Study details: A randomized, placebo-controlled trial of 274 adolescents and adults with sickle cell disease.
Disclosures: Global Blood Therapeutics funded the study. Dr. Howard reported consultant/advisory board activity for the company. Dr. Thompson reported grants and/or personal fees from other companies.
Vichinsky E et al. EHA Congress, Abstract S147. N Engl J Med. 2019 Jun 14. doi: 10.1056/NEJMoa1903212.