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Safety and efficacy of voxelotor in pediatric patients with sickle cell disease aged 4 to 11 years.
- Source :
-
Pediatric blood & cancer [Pediatr Blood Cancer] 2022 Aug; Vol. 69 (8), pp. e29716. Date of Electronic Publication: 2022 Apr 21. - Publication Year :
- 2022
-
Abstract
- Background: Sickle cell disease (SCD) is a devastating, multisystemic disorder that affects millions of people worldwide. The earliest clinical manifestations of SCD can affect infants as young as 6 months of age, and pediatric patients are at risk for acute and life-threatening complications. Early intervention with treatments that target the underlying pathophysiological mechanism of SCD, sickle hemoglobin (HbS) polymerization, are expected to slow disease progression and circumvent disease-associated morbidity and mortality.<br />Procedure: The HOPE-KIDS 1 trial (NCT02850406) is an ongoing four-part, phase 2a, open-label, single- and multiple-dose study to evaluate the pharmacokinetics, efficacy, and safety of voxelotor-a first-in-class HbS polymerization inhibitor-in patients aged 6 months to 17 years with SCD. Initial findings from a cohort of 45 patients aged 4 to 11 years who received voxelotor treatment for up to 48 weeks are reported.<br />Results: Hemoglobin (Hb) response, defined as a >1.0 g/dl increase from baseline, was achieved at week 24 by 47% (n = 16/34) of patients with Hb measurements at baseline and week 24. At week 24, 35% (n = 12/34) and 21% (n = 7/34) of patients had a >1.5 g/dl increase and a >2.0 g/dl increase from baseline in Hb concentration, respectively. Concurrent improvements in hemolytic markers were observed. Voxelotor was well tolerated in this young cohort, with no newly emerging safety signals.<br />Conclusions: Based on its mechanism as an HbS polymerization inhibitor, voxelotor improves Hb levels and markers of hemolysis and has the potential to mitigate SCD-related complications; these results support its use in patients aged ≥4 years.<br /> (© 2022 The Authors. Pediatric Blood & Cancer published by Wiley Periodicals LLC.)
Details
- Language :
- English
- ISSN :
- 1545-5017
- Volume :
- 69
- Issue :
- 8
- Database :
- MEDLINE
- Journal :
- Pediatric blood & cancer
- Publication Type :
- Academic Journal
- Accession number :
- 35451176
- Full Text :
- https://doi.org/10.1002/pbc.29716