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Efficacy and safety of midostaurin in patients with advanced systemic mastocytosis: 10-year median follow-up of a phase II trial.

Author: BerubeC, CorlessC, CoutreS, DeAngeloD J, DutreixC, GeorgeT I, GotlibJ, GraubertT J, LangfordC, LiedtkeM, LinderA, MaJ, MedeirosB, MerkerJ D, PerkinsC, RuffieP-A, SternbergD, WesterveltP

Clinical Trial, Phase IIA Phase II trial is a trial that further evaluates the efficacy and safety of a new drug or treatment, whose safety has been confirmed to some extent in a Phase I trial, in a small number of patients.
Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
Research Support, Non-U.S. Gov'tResearch funding provided by government agencies outside the United States.

Overview

Midostaurin, a multikinase inhibitor, showed promising results in a Phase II trial of patients with advanced systemic mastocytosis. The study found that midostaurin had an overall response rate of 69% and resulted in a significant reduction in bone marrow mast cell burden and serum tryptase levels. Median overall survival for the entire cohort was 40 months.
Paper Details　
Original Abstract of the Article :
Patients with advanced systemic mastocytosis (SM) (e.g. aggressive SM (ASM), SM with an associated hematologic neoplasm (SM-AHN) and mast cell leukemia (MCL)) have limited treatment options and exhibit reduced survival. Midostaurin is an oral multikinase inhibitor that inhibits D816V-mutated KIT, a ...See full text at original site
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引用元：
https://doi.org/10.1038/leu.2017.234

データ提供：米国国立医学図書館（NLM）

Midostaurin: A Beacon of Hope for Advanced Systemic Mastocytosis

The desert of hematological malignancies is a vast and complex terrain, where the delicate balance of blood cells is constantly being challenged. This study investigates the efficacy and safety of midostaurin, a multikinase inhibitor, in treating patients with advanced systemic mastocytosis, a rare and often aggressive condition. Researchers conducted a phase II trial to evaluate the impact of midostaurin on disease progression, survival, and overall health outcomes. The study provides insights into the potential benefits of midostaurin in managing this challenging condition.

Midostaurin: A Promising Therapy for Advanced Systemic Mastocytosis

The study demonstrates the effectiveness of midostaurin in treating advanced systemic mastocytosis. The medication significantly reduced bone marrow mast cell burden, serum tryptase levels, and clinical symptoms. The study also found that midostaurin improved overall survival, particularly in patients with mast cell leukemia. These findings highlight the potential of midostaurin as a valuable therapy for this rare and aggressive condition.

Navigating the Desert of Hematological Malignancies: Midostaurin as a Treatment Option

This study underscores the importance of continued innovation in treating hematological malignancies. The development of midostaurin provides a new and effective treatment option for advanced systemic mastocytosis, offering hope for individuals facing this challenging condition. Like skilled explorers seeking new routes through the desert, researchers continue to search for solutions to manage hematological malignancies and improve the lives of those facing these challenges.

Dr.Camel's Conclusion

The desert of hematological malignancies is a vast and unforgiving terrain, but researchers continue to seek new oases of effective therapies. This study highlights the potential of midostaurin as a beacon of hope for individuals with advanced systemic mastocytosis, offering a promising treatment option and a chance for improved survival and quality of life.

Date :
  1. Date Completed 2019-01-02
  2. Date Revised 2019-01-02
Further Info :

Pubmed ID

28744009

DOI: Digital Object Identifier

10.1038/leu.2017.234

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Article Analysis
・Side Effects of midostaurin
SNS
PICO Info
in preparation
Languages

English

Positive IndicatorAn AI analysis index that serves as a benchmark for how positive the results of the study are. Note that it is a benchmark and requires careful interpretation and consideration of different perspectives.

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