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Efficacy and safety of avapritinib in advanced systemic mastocytosis: interim analysis of the phase 2 PATHFINDER trial.

Author: Alvarez-TwoseIván, BosePrithviraj, DeAngeloDaniel J, DeiningerMichael W, DybedalIngunn, GeorgeTracy I, GotlibJason, HeaneyMark L, HermineOlivier, HexnerElizabeth O, HicksLisa K, LinHui-Min, MarBrenton G, MesaRuben, MitalAndrzej, OhStephen T, PanseJens, PettitKristen M, PlatzbeckerUwe, RadiaDeepti H, ReiterAndreas, SenJayita, SpanLambert, VannucchiAlessandro M

Research Support, N.I.H., ExtramuralResearch funding provided by the NIH to external research institutions and researchers.
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

Avapritinib is a new drug for advanced systemic mastocytosis (AdvSM), a rare cancer. In a study of 62 patients, avapritinib was shown to be effective in 75% of patients, with 19% achieving complete remission. The most common side effects were low white blood cell count, low platelet count, and anemia.
Paper Details　
Original Abstract of the Article :
Advanced systemic mastocytosis (AdvSM) is a rare, KIT D816V-driven hematologic neoplasm characterized by mast cell infiltration and shortened survival. We report the results of a prespecified interim analysis of an ongoing pivotal single-arm phase 2 trial (no. NCT03580655 ) of avapritinib, a p...See full text at original site
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引用元：
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8674139/

データ提供：米国国立医学図書館（NLM）

Avapritinib: A New Hope for Advanced Systemic Mastocytosis

Hematology is a field of medicine that delves into the mysteries of blood and its components, exploring diseases that affect the blood and bone marrow. This study examines the efficacy and safety of avapritinib, a potent inhibitor of KIT D816V, in treating advanced systemic mastocytosis (AdvSM), a rare and often debilitating hematologic neoplasm. The researchers conducted a Phase 2 trial to evaluate the clinical, morphological, and molecular responses to avapritinib in patients with AdvSM.

Avapritinib: A Promising Breakthrough in Mastocytosis Treatment

The study revealed a high rate of clinical, morphological, and molecular responses to avapritinib in patients with AdvSM. The drug showed impressive efficacy in reducing mast cell burden, serum tryptase levels, and KIT D816V variant allele fraction. The researchers also found that avapritinib was generally well tolerated, with manageable adverse events. Imagine avapritinib as a skilled desert explorer, navigating the complex terrain of AdvSM, offering a ray of hope for patients facing this challenging disease.

A New Era in Mastocytosis Treatment

This study represents a significant advance in the treatment of AdvSM, offering a new and potentially more effective option for patients. The researchers emphasize the importance of continued research to further optimize the use of avapritinib and explore its potential benefits in other mast cell disorders. This research marks a promising step forward in the fight against this rare and often debilitating disease.

Dr.Camel's Conclusion

This Phase 2 trial demonstrates the remarkable efficacy and safety of avapritinib in treating advanced systemic mastocytosis. The researchers highlight the drug's potential to improve clinical outcomes and provide a much-needed ray of hope for patients facing this challenging disease. This research marks a significant milestone in the journey to conquer mastocytosis, offering a new oasis in the desert of treatment options.

Date :
  1. Date Completed 2022-02-21
  2. Date Revised 2022-03-09
Further Info :

Pubmed ID

34873345

DOI: Digital Object Identifier

PMC8674139

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Article Analysis
・Effects of avapritinib
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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