Real-World Effectiveness of Voxelotor for Treating Sickle Cell Disease in The US: A Large Claims Data Analysis

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. Sickle cell disease (SCD) is a systemic, inherited disorder affecting approximately 100,000 people in the United States. It predominantly occurs in ethnic groups of African, Middle Eastern, South Mediterranean, and Indian descent. Mutations in the gene encoding hemoglobin (Hb) subunit β lead to sickle Hb (HbS), which then polymerizes under deoxygenated conditions and thereby disturbs red blood cell (RBC) shape. The consequences include the chronic destruction of RBCs, anemia, and limited oxygen delivery. This, in turn, can affect the function of various organs and increase the risk of morbidity, including disease progression and end-organ damage that can substantially impact the quality of life. Although mortality in children has decreased over time, mortality rates in adults have remained high over the past few decades. Characteristic effects of SCD are chronic anemia, hemolysis, and painful vaso-occlusive crises (VOCs). VOCs cause reoccurring episodes of severe pain and are often the main reason for patient hospitalizations   Abstract source