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Safety and efficacy of avapritinib in advanced systemic mastocytosis: the phase 1 EXPLORER trial.

Author: BosePrithviraj, DeAngeloDaniel J, DeiningerMichael W, DrummondMark W, EvansErica K, GeorgeTracy I, GotlibJason, HexnerElizabeth O, HornyHans-Peter, LinHui-Min, MarBrenton G, QuieryAlbert T, RadiaDeepti H, RobinsonWilliam A, Schmidt-KittlerOleg, TugnaitMeera, VerstovsekSrdan, WintonElliott F

Research Support, N.I.H., ExtramuralResearch funding provided by the NIH to external research institutions and researchers.
Clinical Trial, Phase IThe first stage of clinical trials in humans. Evaluates the safety, tolerability, and pharmacokinetics of a new drug/treatment.
Research Support, Non-U.S. Gov'tResearch funding provided by government agencies outside the United States.
Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.

Overview

Avapritinib is a new drug for advanced systemic mastocytosis (AdvSM). In a study of 86 patients, avapritinib was effective in 75% of patients, with 36% achieving complete remission. The most common side effects were swelling around the eyes, anemia, diarrhea, low platelet count, and nausea.
Paper Details　
Original Abstract of the Article :
Advanced systemic mastocytosis (AdvSM) is a rare hematologic neoplasm driven by the KIT D816V mutation and associated with poor survival. This phase 1 study ( NCT02561988 ) evaluated avapritinib (BLU-285), a selective KIT D816V inhibitor, in patients with AdvSM. The primary endpoints were the maximu...See full text at original site
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引用元：
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8674134/

データ提供：米国国立医学図書館（NLM）

Avapritinib: A New Hope for Advanced Systemic Mastocytosis

The [Treatment] of [Advanced systemic mastocytosis (AdvSM)] is a challenging undertaking, often met with [Limited treatment options] and [Poor survival rates]. This study explores the [Safety and efficacy] of [Avapritinib], a [Selective KIT D816V inhibitor], in patients with [AdvSM]. It’s like discovering a new well in the desert, offering a potential source of relief for patients struggling with this rare and aggressive condition. The researchers conducted a [Phase 1 clinical trial] to assess the [Maximum tolerated dose, recommended phase 2 dose, and safety] of [Avapritinib]. The results show that [Avapritinib] demonstrated [Significant efficacy] in reducing [Mast cell burden] and inducing [Durable responses], including [Molecular remission] in some patients.

A New Oasis in the Desert: Avapritinib Shows Promise for Advanced Systemic Mastocytosis

The study demonstrates that [Avapritinib] holds promise as a [New and effective treatment] for [AdvSM]. This finding is like discovering a hidden oasis in the desert, offering a sanctuary for patients facing the challenges of this rare and debilitating condition. The study's findings suggest that [Avapritinib] may significantly improve the [Outcomes] for patients with [AdvSM], offering a potential path toward a more hopeful future.

Navigating the Desert of Mastocytosis: A Focus on Precision Medicine

This study underscores the importance of [Precision medicine] in the [Treatment] of [Cancer]. [Avapritinib], with its [Targeted inhibition] of the [KIT D816V mutation], exemplifies the power of [Tailored therapy] in addressing the specific needs of patients with [AdvSM]. It’s like using a map to navigate the desert, selecting the most efficient path for each individual traveler, ensuring a smoother and more successful journey.

Dr.Camel's Conclusion

This study offers promising results for patients with advanced systemic mastocytosis. Avapritinib demonstrates significant efficacy and a favorable safety profile, representing a potential breakthrough in the treatment of this rare and challenging disease.

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

Pubmed ID

34873347

DOI: Digital Object Identifier

PMC8674134

Related Literature

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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