The New England journal of medicine, 2022-04, Vol.386 (15), p.1432-1442
- Al-Samkari, Hanny
- Galactéros, Frédéric
- Glenthøj, Andreas
- Rothman, Jennifer A
- Andres, Oliver
- Grace, Rachael F
- Morado-Arias, Marta
- Layton, D. Mark
- Onodera, Koichi
- Verhovsek, Madeleine
- Barcellini, Wilma
- Chonat, Satheesh
- Judge, Malia P
- Zagadailov, Erin
- Xu, Rengyi
- Hawkins, Peter
- Beynon, Vanessa
- Gheuens, Sarah
- van Beers, Eduard J
2022
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- Autor(en) / Beteiligte
- Al-Samkari, Hanny
- Galactéros, Frédéric
- Glenthøj, Andreas
- Rothman, Jennifer A
- Andres, Oliver
- Grace, Rachael F
- Morado-Arias, Marta
- Layton, D. Mark
- Onodera, Koichi
- Verhovsek, Madeleine
- Barcellini, Wilma
- Chonat, Satheesh
- Judge, Malia P
- Zagadailov, Erin
- Xu, Rengyi
- Hawkins, Peter
- Beynon, Vanessa
- Gheuens, Sarah
- van Beers, Eduard J
- Titel
- Mitapivat versus Placebo for Pyruvate Kinase Deficiency
- Ist Teil von
- The New England journal of medicine, 2022-04, Vol.386 (15), p.1432-1442
- Ort / Verlag
- United States: Massachusetts Medical Society
- Erscheinungsjahr
- 2022
- Quelle
- MEDLINE
- Beschreibungen/Notizen
- Pyruvate kinase deficiency, the most common genetic lesion in the glycolytic pathway, leads to chronic hemolytic anemia. Mitapivat, an oral agent, can activate some mutant enzymes and restore red-cell ATP generation. In this trial, hemoglobin levels increased from baseline by 1.5 g per deciliter or more at 24 weeks in 40% of the patients with pyruvate kinase deficiency who received mitapivat.
- Sprache
- Englisch
- Identifikatoren
- ISSN: 0028-4793eISSN: 1533-4406DOI: 10.1056/NEJMoa2116634Titel-ID: cdi_proquest_miscellaneous_2650252539
- Format
- –
- Schlagworte
- Adult, Adverse events, Anemia, Anemia, Hemolytic, Congenital Nonspherocytic - drug therapy, Chronic illnesses, Double-Blind Method, Drug dosages, Enzymes, FDA approval, Genetics, Genetics General, Hematology, Hemoglobin, Hemoglobins - analysis, Hemoglobins - drug effects, Hemolysis, Hemolysis - drug effects, Hemolytic anemia, Humans, Medical laboratories, Mutation, Nausea, Oncology, Oncology General, Patients, Piperazines - pharmacology, Piperazines - therapeutic use, Placebos, Pyruvate kinase, Pyruvate Kinase - deficiency, Pyruvate Metabolism, Inborn Errors - drug therapy, Pyruvic acid, Quinolines - pharmacology, Quinolines - therapeutic use