Pyruvate kinase deficiency
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| Section editor | |
|---|---|
 |
Benjamin Tillman, MD Vanderbilt University Nashville, TN, USA |
Guidelines
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
- 2024: Al-Samkari et al. Diagnosis and management of pyruvate kinase deficiency: international expert guidelines PubMed
All lines of therapy
Mitapivat monotherapy
Regimen
| Study | Dates of enrollment | Evidence | Comparator | Comparative Efficacy |
|---|---|---|---|---|
| Grace et al. 2019 (AG348-C-003) | NR | 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 |
References
- 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
- 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 contains dosing details in abstract PubMed NCT03548220
- 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