Hope on the Horizon: New and Future Therapies for Sickle Cell Disease

Abstract

This article provides an overview of conventional, new, and future treatment options for sickle cell disease (SCD), a genetic disorder affecting the production of hemoglobin. Current treatments include hydroxyurea, a conventional SCD treatment that increases the levels of fetal hemoglobin, and new treatments such as voxelotor, a recently approved SCD treatment that selectively binds hemoglobin, preventing formation of sickled red blood cells. In addition to discussing the mechanisms of action of current SCD treatments, potential side effects are also discussed, highlighting the need for new treatments that can address the limitations of current treatments and improve the quality of life for people with SCD. Future treatments, such as gene therapy, are also explored as promising treatment options for SCD patients.

A Case Report of Hyperhemolytic Syndrome in Sickle Cell Disease, with a Special Focus on Avoiding the Use of Transfusions

“…The newer disease-modifying treatment approved for the treatment of SCD is voxelotor, which inhibits HbS polymerization and prevents sickle cell formation, while crizanlizumab is an antibody against P-selectin, blocking the interaction between sickled RBCs and P-selectin, thus preventing vaso-occlusive crisis. These may also be effective treatment options [24,25]. Conventional treatment, such as hydroxyurea, increases the formation of HbF in SCD patients and can prevent complications and acute crises [25].…”

“…These may also be effective treatment options [24,25]. Conventional treatment, such as hydroxyurea, increases the formation of HbF in SCD patients and can prevent complications and acute crises [25].…”