Advancements in Sickle Cell Disease (SCD) Treatment: A Review of Novel Pharmacotherapies and Their Impact on Patient Outcomes

Abstract

Sickle cell disease (SCD) is a genetic hereditary blood disease that disrupts normal beta-globin production. Patients with SCD experience a broad range of symptoms ranging from anemia, pain crises, and jaundice to acute coronary syndrome and stroke. SCD has been treated with hydroxyurea since 1998. Three important pharmacotherapies have been approved by the Food and Drug Administration (FDA) in the past few years. L-Glutamine has shown efficacy in reducing vaso-occlusive pain crises and hospitalization. Crizanlizumab has also shown positive outcomes in patients with SCD. Voxelotor has been studied to be effective in improving hemolytic anemia and the quality of life in SCD patients. These drugs can be used alone or in conjunction with hydroxyurea. Trials have shown that these therapies have significant efficacy. The events of pain, hemolytic anemia, vaso-occlusive crises, and hospitalizations have been reduced by using these agents. In this editorial, we will discuss these advanced treatment options for patients with SCD.