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Review Article

Promising role of Voxelotor in managing sickle cell disease in children: a narrative review

Amit Agrawal¹  , Gaurav Jadon²  , Japna Singh³, Dalwinder Janjua⁴ 

¹Gandhi Medical College, Bhopal, India
²NMC Speciality Hospital, Dubai, United Arab Emirates
³International Modern Hospital, Dubai, United Arab Emirates
⁴Al Jalila Children's Specialty Hospital, Dubai, United Arab Emirates

Correspondence Amit Agrawal ,Email: agrawaldramit@yahoo.co.in

Received: March 19, 2024; Revised: August 12, 2024   Accepted: August 12, 2024.

Abstract

Sickle cell disease (SCD) is characterized by chronic hemolytic anemia and intermittent vaso-occlusive crises. To date, four disease-modifying drugs have been approved for the treatment of SCD: hydroxyurea (an S-phase inhibitor), L-glutamine (an amino acid), crizanlizumab (a P-selectin inhibitor), and voxelotor (a hemoglobin S polymerization inhibitor). Preclinical studies suggested that voxelotor effectively treats SCD and sickle cell anemia (SCA). In a phase III trial, voxelotor-treated patients showed significantly elevated hemoglobin levels (>1 g/dL from baseline) compared to placebo-treated patients. The group that received voxelotor also showed a greater decrease in hemolytic markers but a comparable incidence of side effects. Six ongoing clinical trials also sought to ascertain the effectiveness and safety of high-dose voxelotor when administered to children younger than 12 years. Studies assessing their long-term efficacy and safety are needed to fully understand the role of voxelotor in treating SCD/SCA. In this review, we discuss the mechanisms, trials to date, and future treatment directions of voxelotor.

Keywords :Sickle cell disease, Sickle cell anemia, Voxelotor, Pharmacokinetics, Pharmacodynamics, Safety