Mitapivat versus Placebo for Pyruvate Kinase Deficiency.

David J KuterFrédéric GalactérosAndreas GlenthoejJennifer A RothmanOliver AndresRachael F GraceMarta Morado-AriasD Mark LaytonKoichi OnoderaMadeleine VerhovsekWilma BarcelliniSatheesh ChonatMalia P JudgeErin ZagadailovRengyi XuPeter HawkinsVanessa BeynonSarah GheuensEduard J van Beersnull null

Published in: The New England journal of medicine (2022)

In patients with pyruvate kinase deficiency, mitapivat significantly increased the hemoglobin level, decreased hemolysis, and improved patient-reported outcomes. No new safety signals were identified in the patients who received mitapivat. (Funded by Agios Pharmaceuticals; ACTIVATE ClinicalTrials.gov number, NCT03548220.).

Keyphrases

patient reported outcomes
protein kinase
tyrosine kinase
red blood cell
replacement therapy
randomized controlled trial
double blind
phase iii