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Efficacy and Safety of Midostaurin in Advanced Systemic Mastocytosis
Author(s) -
Jason Gotlib,
Hanneke C. KluinNelemans,
Tracy I. George,
Cem Akin,
Karl Sotlar,
Olivier Hermine,
Farrukh T. Awan,
Elizabeth O. Hexner,
Michael J. Mauro,
David Sternberg,
Matthieu Villeneuve,
Alice Huntsman Labed,
Eric J. Stanek,
Karin Hartmann,
Hans-Peter Horny,
Peter Valent,
Andreas Reiter
Publication year - 2016
Publication title -
new england journal of medicine
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 19.889
H-Index - 1030
eISSN - 1533-4406
pISSN - 0028-4793
DOI - 10.1056/nejmoa1513098
Subject(s) - systemic mastocytosis , midostaurin , medicine , pathogenesis , disease , immunology , leukemia , oncology
Advanced systemic mastocytosis comprises rare hematologic neoplasms that are associated with a poor prognosis and lack effective treatment options. The multikinase inhibitor midostaurin inhibits KIT D816V, a primary driver of disease pathogenesis.

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