Safety and Efficacy of Mitapivat in Pyruvate Kinase Deficiency Academic Article uri icon

Overview

MeSH Major

  • Anemia, Sickle Cell
  • Blood Transfusion
  • Iron Overload

abstract

  • The administration of mitapivat was associated with a rapid increase in the hemoglobin level in 50% of adults with pyruvate kinase deficiency, with a sustained response during a median follow-up of 29 months during the extension phase. Adverse effects were mainly low-grade and transient. (Funded by Agios Pharmaceuticals; ClinicalTrials.gov number, NCT02476916.).

publication date

  • September 5, 2019

Research

keywords

  • Academic Article

Identity

Language

  • eng

Digital Object Identifier (DOI)

  • 10.1056/NEJMoa1902678

PubMed ID

  • 31483964

Additional Document Info

start page

  • 933

end page

  • 944

volume

  • 381

number

  • 10