Pharmacologic Therapies for Sickle Cell Disease

Sickle cell disease (SCD) is a multisystem disease, associated with episodes of acute illness and progressive organ damage. Despite its low prevalence in the United States, SCD is one of the most common severe monogenic disorders worldwide, with an estimated 230,000 affected children born every year in sub-Saharan Africa. As a result of its multiple complications, SCD is associated with an overall decreased life expectancy. In addition, the economic burden of SCD is enormous - a consequence of recurrent and chronic SCD-related complications, frequent absences from school and work, and physical impairment limiting the ability to contribute to society. Although there is an increased understanding of the pathophysiology of SCD, available pharmacologic treatments remain limited. The current approach to the management of SCD includes supportive treatments with folic acid; symptomatic treatment with analgesics, intravenous fluids, oxygen and red blood cell (RBC) transfusion; disease-modifying treatment with hydroxyurea and RBC transfusion; and curative therapy with bone marrow transplantation. Despite the success of bone marrow transplantation as a curative approach in SCD, this modality is limited by its high cost, decreased availability of suitable donors and toxicity. Abstract source