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The New England journal of medicine, 2016-06, Vol.374 (26), p.2605-2606
Ort / Verlag
United States: Massachusetts Medical Society
Erscheinungsjahr
2016
Quelle
MEDLINE
Beschreibungen/Notizen
To the Editor:
Advanced systemic mastocytosis
1
carries a poor prognosis.
2
Midostaurin, an inhibitor of tyrosine kinases, targets KIT mutants associated with mastocytosis.
3
The French National Reference Center for Mastocytosis (CEREMAST) conducted a prospective survey of patients with mastocytosis who were treated with midostaurin under a transitory-use authorization program. All the patients provided written informed consent.
From August 2012 through April 2015, a total of 28 patients with mastocytosis (including 4 with aggressive disease, 18 with associated hematologic non–mast-cell disease, 3 with mast-cell leukemia, 1 with mast-cell sarcoma, and 2 with progressive smoldering disease) received midostaurin at a dose of 100 . . .