Real-World Experience of Patients With Sickle Cell Disease Treated With Voxelotor: A Multicenter, Retrospective Study

Background: Sickle cell disease (SCD) is an inherited systemic disorder in which sickle hemoglobin (HbS) polymerization triggers red blood cell sickling, chronic hemolytic anemia, and recurrent episodes of vaso-occlusion. SCD-related complications lead to acute and chronic life-threatening events, cumulative organ damage, disability, and early mortality. Voxelotor (Oxbryta ®) tablets are approved in the United States for treatment of SCD in adults and adolescents aged ≥12 years, based on the efficacy and safety data from the randomized, placebo-controlled, multicenter HOPE trial. Voxelotor is an oral, once-daily HbS-polymerization inhibitor that has been shown to increase hemoglobin (Hb) levels and reduce markers of hemolysis. The Retrospective Study to Evaluate Outcomes in Patients with Sickle Cell Disease Treated with Oxbryta (RETRO) is designed to collect, aggregate, and characterize real-world, retrospective laboratory and clinical data on adults and adolescents with SCD treated with voxelotor as part of their usual care at multiple clinical centers in the United States.