| 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: 19 March 2024 • Revised: 12 August 2024 • Accepted: 12 August 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. |
| Key Words:
Sickle cell disease, Sickle cell anemia, Voxelotor, Pharmacokinetics, Pharmacodynamics, Safety |
|
PDF Links
Download Citation
