Midostaurin in Advanced Systemic Mastocytosis

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...

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Published in:	The New England journal of medicine Vol. 374; no. 26; pp. 2605 - 2606
Main Authors:	Chandesris, Marie-Olivia, Damaj, Gandhi, Canioni, Danielle, Brouzes, Chantal, Lhermitte, Ludovic, Hanssens, Katia, Frenzel, Laurent, Cherquaoui, Zoubair, Durieu, Isabelle, Durupt, Stéphane, Gyan, Emmanuel, Beyne-Rauzy, Odille, Launay, David, Faure, Cyril, Hamidou, Mohamed, Besnard, Sophie, Diouf, Momar, Schiffmann, Aurélie, Niault, Mathilde, Jeandel, Pierre-Yves, Ranta, Dana, Gressin, Remi, Chantepie, Sylvain, Barete, Stéphane, Dubreuil, Patrice, Bourget, Philippe, Lortholary, Olivier, Hermine, Olivier
Format:	Journal Article
Language:	English
Published:	United States Massachusetts Medical Society 30-06-2016
Subjects:	Age of Onset Antineoplastic Agents - adverse effects Antineoplastic Agents - therapeutic use Follow-Up Studies Humans Leukemia Mastocytosis Mastocytosis, Systemic - drug therapy Medical prognosis Multivariate Analysis Staurosporine - adverse effects Staurosporine - analogs & derivatives Staurosporine - therapeutic use Survival Rate
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Summary:	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 . . .
Bibliography:	SourceType-Other Sources-1 ObjectType-Article-2 content type line 63 ObjectType-Feature-1 ObjectType-Correspondence-3
ISSN:	0028-4793 1533-4406
DOI:	10.1056/NEJMc1515403