Pyruvate kinase deficiency

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Tillman Benjamin-2.jpg
Benjamin Tillman, MD
Vanderbilt University
Nashville, TN, USA

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Given the rapid change in evidence in many areas of hematology/oncology, readers are encouraged to consider any guideline published 5+ years ago to be for historical purposes, only.

International expert guidelines

All lines of therapy

Mitapivat monotherapy


Study Dates of enrollment Evidence Comparator Comparative Efficacy
Grace et al. 2019 (AG348-C-003) Not reported Phase 2
Glenthøj et al. 2022 (ACTIVATE-T) 2018-06-26 to 2020-02-04 Nonrandomized phase 3 (RT)
Al-Samkari et al. 2022 (ACTIVATE) 2018-10-01 to 2020-10-09 Phase 3 (E-RT-esc) Placebo Superior hemoglobin response

Supportive therapy

24-week course


  1. AG348-C-003: Grace RF, Rose C, Layton DM, Galactéros F, Barcellini W, Morton DH, van Beers EJ, Yaish H, Ravindranath Y, Kuo KHM, Sheth S, Kwiatkowski JL, Barbier AJ, Bodie S, Silver B, Hua L, Kung C, Hawkins P, Jouvin MH, Bowden C, Glader B. Safety and Efficacy of Mitapivat in Pyruvate Kinase Deficiency. N Engl J Med. 2019 Sep 5;381(10):933-944. link to original article PubMed NCT02476916
  2. ACTIVATE: Al-Samkari H, Galactéros F, Glenthøj A, Rothman JA, Andres O, Grace RF, Morado-Arias M, Layton DM, Onodera K, Verhovsek M, Barcellini W, Chonat S, Judge MP, Zagadailov E, Xu R, Hawkins P, Beynon V, Gheuens S, van Beers EJ; ACTIVATE Investigators. Mitapivat versus Placebo for Pyruvate Kinase Deficiency. N Engl J Med. 2022 Apr 14;386(15):1432-1442. link to original article dosing details in abstract have been reviewed by our editors PubMed NCT03548220
  3. ACTIVATE-T: Glenthøj A, van Beers EJ, Al-Samkari H, Viprakasit V, Kuo KHM, Galactéros F, Chonat S, Porter J, Zagadailov E, Xu R, Oluyadi A, Hawkins P, Gheuens S, Beynon V, Barcellini W; ACTIVATE-T investigators. Mitapivat in adult patients with pyruvate kinase deficiency receiving regular transfusions (ACTIVATE-T): a multicentre, open-label, single-arm, phase 3 trial. Lancet Haematol. 2022 Oct;9(10):e724-e732. Epub 2022 Aug 18. link to original article PubMed NCT03559699