Real-World Experience of Voxelotor for the Management of Complications in Sickle Cell Disease

Background: Sickle cell disease (SCD) is an inherited systemic disorder characterized by chronic hemolytic anemia and recurrent vaso-occlusion, which can lead to acute and chronic complications, disability, and early mortality. Patients with SCD are hospitalized frequently and most commonly for vaso-occlusive crises (VOCs). Furthermore, most will require a blood transfusion for prevention or acute management of complications, with >90% of adults receiving ≥1 transfusion in their lifetime. Voxelotor (Oxbryta ®) tablets are indicated for treatment of SCD in adults and adolescents aged ≥12 years. Emerging evidence shows that voxelotor, an oral, once-daily sickle hemoglobin-polymerization inhibitor, may improve the clinical symptoms of SCD and reduce VOC rates as well as the need for transfusions. This study sought to assess the real-world impact of voxelotor treatment on the rates and management of SCD-related complications.