Abstract | BACKGROUND: METHODS: RESULTS: The overall response rate was 60% (95% confidence interval [CI], 49 to 70); 45% of the patients had a major response, which was defined as complete resolution of at least one type of mastocytosis-related organ damage. Response rates were similar regardless of the subtype of advanced systemic mastocytosis, KIT mutation status, or exposure to previous therapy. The median best percentage changes in bone marrow mast-cell burden and serum tryptase level were -59% and -58%, respectively. The median overall survival was 28.7 months, and the median progression-free survival was 14.1 months. Among the 16 patients with mast-cell leukemia, the median overall survival was 9.4 months (95% CI, 7.5 to not estimated). Dose reduction owing to toxic effects occurred in 56% of the patients; re-escalation to the starting dose was feasible in 32% of those patients. The most frequent adverse events were low-grade nausea, vomiting, and diarrhea. New or worsening grade 3 or 4 neutropenia, anemia, and thrombocytopenia occurred in 24%, 41%, and 29% of the patients, respectively, mostly in those with preexisting cytopenias. CONCLUSIONS:
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Authors | Jason Gotlib, Hanneke C Kluin-Nelemans, Tracy I George, Cem Akin, Karl Sotlar, Olivier Hermine, Farrukh T Awan, Elizabeth Hexner, Michael J Mauro, David W Sternberg, Matthieu Villeneuve, Alice Huntsman Labed, Eric J Stanek, Karin Hartmann, Hans-Peter Horny, Peter Valent, Andreas Reiter |
Journal | The New England journal of medicine
(N Engl J Med)
Vol. 374
Issue 26
Pg. 2530-41
(Jun 30 2016)
ISSN: 1533-4406 [Electronic] United States |
PMID | 27355533
(Publication Type: Clinical Trial, Phase II, Journal Article, Multicenter Study, Research Support, N.I.H., Extramural, Research Support, Non-U.S. Gov't)
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Chemical References |
- Antineoplastic Agents
- Staurosporine
- midostaurin
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Topics |
- Adult
- Aged
- Aged, 80 and over
- Antineoplastic Agents
(adverse effects, therapeutic use)
- Female
- Humans
- Leukemia, Mast-Cell
(drug therapy, mortality)
- Male
- Mastocytosis, Systemic
(drug therapy, mortality)
- Middle Aged
- Multivariate Analysis
- Staurosporine
(adverse effects, analogs & derivatives, therapeutic use)
- Survival Analysis
- Treatment Outcome
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