Safety and efficacy of voxelotor in pediatric patients with sickle cell disease aged 4 to 11 years.

Jeremie H EsteppRam KalpatthiGerald WoodsSara TrompeterRobert I LiemKacie SimsAdlette InatiBaba Psalm Duniya Inusa Andrew D CampbellConnie PicconeMiguel R AbboudKim Smith-WhitleySandra DixonMargaret TondaCarla WashingtonNoelle M GriffinClark Brown

Published in: Pediatric blood & cancer (2022)

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.

Keyphrases

end stage renal disease
ejection fraction
chronic kidney disease
prognostic factors
risk factors
patient reported outcomes
climate change
risk assessment
human health