Details

Autor(en) / Beteiligte

• Shah, Ami J.
• Schwartz, Jonathan D.
• Segovia, Jose C.
• Al-Samkari, Hanny
• Beynon, Vanessa
• van Beers, Eduard J.

Titel

Mitapivat versus Placebo for Pyruvate Kinase Deficiency

Ist Teil von

• The New England journal of medicine, 2022-06, Vol.386 (26), p.2538-2539

Ort / Verlag

Boston: Massachusetts Medical Society

Erscheinungsjahr

2022

Quelle

EZB-FREE-00999 freely available EZB journals

Beschreibungen/Notizen

• To the Editor: Al-Samkari et al. (April 14 issue) 1 report that mitapivat was efficacious in the treatment of pyruvate kinase deficiency, and they observed increases from baseline of at least 1.5 g per deciliter in the hemoglobin level in at least 40% of adult patients who had disease emanating from at least one missense PKLR mutation and who were not receiving regular red-cell transfusions. The authors indicated that “all prespecified patient subgroups (including those defined according to a baseline hemoglobin level of <8.5 or ≥8.5 g per deciliter and those defined according to previous splenectomy status) in the mitapivat group . . .