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Sickle cell disease (SCD) describes a group of heritable blood disorders caused by the polymerization of sickle hemoglobin (HbS). HbS polymerization leads to anemia and vaso-occlusion, a process that impedes delivery of oxygen to tissues throughout the body, resulting in end-organ damage (EOD). Given the lifelong complications associated with SCD, identification and treatment of early symptoms in childhood is increasingly important. Voxelotor is an oral therapy that inhibits the polymerization of HbS and offers a unique therapeutic mechanism to reduce the causes of EOD. Voxelotor was approved in December 2021 for the treatment of SCD in patients aged ≥4 years. Clinical data on the use of voxelotor in pediatric patients with SCD is reviewed. Ongoing studies examining the clinical efficacy and safety profile of voxelotor in pediatric patients are compared with similar clinical outcomes in adults with SCD. Planned studies of voxelotor in children are also discussed. Voxelotor provides a unique therapeutic option to target the root causes of EOD and can potentially be used alongside other SCD therapies. Future studies directly observing the impact of voxelotor on EOD will be important for determining treatment strategies.

Voxelotor for the treatment of sickle cell disease in pediatric patients