Background Sickle cell disease (SCD) is a genetic disease that impacts patients' quality of
life, healthcare costs, and life expectancy. Elevated sickle hemoglobin (HbS), which readily
polymerizes, causes red blood cell sickling, leading to chronic hemolytic anemia and
complications often requiring hospitalization and transfusions. In 2019, voxelotor, which
inhibits HbS polymerization, was approved for SCD treatment. Objectives This study uses
real-world evidence to assess voxelotor's effectiveness in SCD patients in typical clinical …

Background: Until late 2019, few treatments had been approved by the FDA for treating
sickle cell disease (SCD). Voxelotor (Oxbryta®) is a sickle hemoglobin-polymerization
inhibitor approved by the FDA in November 2019 for treatment of SCD in adults and
adolescents aged≥ 12 years under an accelerated approval based on results of the pivotal
HOPE study. In HOPE, voxelotor increased average hemoglobin (Hb) by 1.1 g/dL from
baseline in patients with 1-10 vaso-occlusive crises (VOCs) in the previous year and a Hb …