Practical Guidance for the Use of Voxelotor in the Management of Sickle Cell Disease

Abstract

Sickle cell disease (SCD) is one of the most common inherited blood disorders. Deoxygenated hemoglobin S (HbS) polymerizes and causes anemia and various end organ effects. Voxelotor acts by increasing HbS oxygen affinity, decreasing anemia and hemolysis. Voxelotor is approved for use in individuals with SCD age 4 years and older. Phase 3 trials demonstrated an increase in hemoglobin levels and a decrease in markers of hemolysis; however, data or benefits related to clinical and quality of life outcomes are relatively limited and varied across different studies. This review summarizes the published clinical trials and research studies focused on the use of voxelotor in SCD to provide an evidence-based practical guide for hematology providers on its utilization in clinical settings, including physicians and independent licensed practitioners.

Keywords:

• sickle cell
• sickle cell anemia
• sickle cell disease
• hemoglobinopathy
• pediatric
• children
• adolescents
• young adults
• adult
• voxelotor
• oxbryta
• disease-modifying therapy

Abbreviations

CTCAE, Common Terminology Criteria for Adverse Events; GBT, Global Blood Therapeutics; SCD, Sickle cell disease; SDM, Shared decision making; FDA, Food and Drug Administration; HbSβ⁰Thal, Hemoglobin S Beta Zero thalassemia; HbSβ+Thal, Hemoglobin S Beta Plus thalassemia.

Acknowledgment

This project was supported by grant (K23HL150232, PI: Badawy) from the National Heart, Lung, and Blood Institute of the National Institutes of Health. The content is solely the responsibility of the authors and does not necessarily represent the National Institutes of Health.

Disclosure

The authors report no relevant conflict or competing interests for this work.