Clinically meaningful improvements in patient-reported outcomes in mitapivat-treated patients with pyruvate kinase deficiency.
Journal
American journal of hematology
ISSN: 1096-8652
Titre abrégé: Am J Hematol
Pays: United States
ID NLM: 7610369
Informations de publication
Date de publication:
07 May 2024
07 May 2024
Historique:
revised:
19
03
2024
received:
29
09
2023
accepted:
22
03
2024
medline:
7
5
2024
pubmed:
7
5
2024
entrez:
7
5
2024
Statut:
aheadofprint
Résumé
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.
Types de publication
Letter
Langues
eng
Sous-ensembles de citation
IM
Subventions
Organisme : Agios Pharmaceuticals
Informations de copyright
© 2024 The Authors. American Journal of Hematology published by Wiley Periodicals LLC.
Références
Grace RF, Bianchi P, van Beers EJ, et al. Clinical spectrum of pyruvate kinase deficiency: data from the pyruvate kinase deficiency natural history study. Blood. 2018;131:2183‐2192.
Al‐Samkari H, van Beers EJ, Morton DH, et al. Health‐related quality of life and fatigue in children and adults with pyruvate kinase deficiency. Blood Adv. 2022;6:1844‐1853.
Andrae DA, Grace RF, Jewett A, et al. Psychometric validation of the pyruvate kinase deficiency diary and pyruvate kinase deficiency impact assessment in adults in the phase 3 ACTIVATE trial. J Patient Rep Outcomes. 2023;7:112.
Agios Pharmaceuticals Inc. Pyrukynd. Prescribing information. 2022. Accessed June 15, 2023. https://www.accessdata.fda.gov/drugsatfda_docs/label/2022/216196s000lbl.pdf
Al‐Samkari H, Galactéros F, Glenthøj A, et al. Mitapivat versus placebo for pyruvate kinase deficiency. N Engl J Med. 2022;386:1432‐1442.
Glenthøj A, van Beers EJ, Al‐Samkari H, et al. 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;9:e724‐e732.