Clinically meaningful improvements in patient-reported outcomes in mitapivat-treated patients with pyruvate kinase deficiency

Kevin H M Kuo; Rachael F Grace; Eduard J van Beers; Wilma Barcellini; Andreas Glenthøj; Susanne Holzhauer; Vanessa Beynon; Susan Morris; Junlong Li; Erin Zagadailov; Parija Patel; Hanny Al-Samkari

doi:10.1002/ajh.27312

Clinically meaningful improvements in patient-reported outcomes in mitapivat-treated patients with pyruvate kinase deficiency

Am J Hematol. 2024 Jul;99(7):1415-1419. doi: 10.1002/ajh.27312. Epub 2024 May 7.

Authors

Affiliations

¹ Division of Hematology, University of Toronto, Toronto, Ontario, Canada.
² Dana-Farber/Boston Children's Cancer and Blood Disorder Center, Harvard Medical School, Boston, Massachusetts, USA.
³ Center for Benign Haematology, Thrombosis and Haemostasis, Van Creveldkliniek, University Medical Center Utrecht, Utrecht University, Utrecht, The Netherlands.
⁴ Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan, Italy.
⁵ Department of Haematology, Copenhagen University Hospital, Rigshospitalet, Copenhagen, Denmark.
⁶ Department of Pediatric Hematology and Oncology, Charité University Medicine, Berlin, Germany.
⁷ Agios Pharmaceuticals, Inc., Cambridge, Massachusetts, USA.
⁸ Division of Hematology, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA.

PMID: 38712615
DOI: 10.1002/ajh.27312

Abstract

Clinically meaningful benefits in the signs, symptoms, and impacts of #PKDeficiency as assessed by disease-specific patient-reported outcome measures were observed in mitapivat-treated adult patients in two phase 3 clinical trials.

Publication types

Clinical Trial, Phase III
Letter
Research Support, Non-U.S. Gov't

MeSH terms

Adult
Anemia, Hemolytic, Congenital Nonspherocytic
Female
Humans
Male
Middle Aged
Patient Reported Outcome Measures*
Pyruvate Kinase* / deficiency
Pyruvate Metabolism, Inborn Errors*
Treatment Outcome

Substances

Pyruvate Kinase

Supplementary concepts

Pyruvate Kinase Deficiency of Red Cells

Grants and funding

Agios Pharmaceuticals