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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 . . .