N Engl J Med 2022 04;386(15):1432-1442
From the Division of Hematology Oncology, Massachusetts General Hospital (H.A.-S.) and the Dana-Farber/Boston Children's Cancer and Blood Disorders Center (R.F.G.), Harvard Medical School, Boston, and Agios Pharmaceuticals, Cambridge (M.P.J., E.Z., R.X., P.H., V.B., S.G.) - all in Massachusetts; Unité des Maladies Génétiques du Globule Rouge, Centre Hospitalier Universitaire Henri Mondor, Créteil, France (F.G.); the Department of Hematology, Copenhagen University Hospital, Rigshospitalet, Copenhagen (A.G.); Duke University Medical Center, Durham, NC (J.A.R.); the Department of Pediatrics, University of Würzburg, Würzburg, Germany (O.A.); the Hematology Department, Hospital Universitario La Paz, Madrid (M.M.-A.); Hammersmith Hospital, Imperial College Healthcare NHS Trust, London (D.M.L.); Tohoku University Hospital, Sendai, Japan (K.O.); McMaster University, Hamilton, ONT, Canada (M.V.); Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan (W.B.); Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta, and the Department of Pediatrics, Emory University, Atlanta (S.C.); and the Benign Hematology Center, Van Creveldkliniek, University Medical Center Utrecht, University Utrecht, Utrecht, the Netherlands (E.J.B.).
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. Read More