Hanny Al-Samkari, Frédéric Galactéros, Andreas Glenthøj, Jennifer A Rothman, Oliver Andres, Rachael F Grace, Marta Morado-Arias, D Mark Layton, Koichi Onodera, Madeleine Verhovsek, Wilma Barcellini, Satheesh Chonat, Malia P Judge, Erin Zagadailov, Rengyi Xu, Peter Hawkins, Vanessa Beynon, Sarah Gheuens, Eduard J van Beers
BACKGROUND: Pyruvate kinase deficiency is a rare, hereditary, chronic condition that is associated with hemolytic anemia. In a phase 2 study, mitapivat, an oral, first-in-class activator of erythrocyte pyruvate kinase, increased the hemoglobin level in patients with pyruvate kinase deficiency. METHODS: In this global, phase 3, randomized, placebo-controlled trial, we evaluated the efficacy and safety of mitapivat in adults with pyruvate kinase deficiency who were not receiving regular red-cell transfusions...
April 14, 2022: New England Journal of Medicine