July 24, 2025

#121 - Avapritinib versus Placebo in Indolent Systemic Mastocytosis - PIONEER Trial

39 minutes

What happens when you target the root cause of indolent systemic mastocytosis?

In this episode of The Itch Review, we unpack key findings from the article “Avapritinib versus Placebo in Indolent Systemic Mastocytosis,” published on May 23, 2023, in NEJM Evidence.

Indolent Systemic Mastocytosis (ISM) is a rare chronic disease with life-altering symptoms. Until recently, treatment options were limited. We break down new data from Part 2 of the PIONEER study, which evaluated Avapritinib, a targeted therapy designed to inhibit the KIT D816V mutation that drives ISM.

Dr. Gupta and Dr. Blaiss explain how Avapritinib works, how the study was designed, and what outcomes were measured, including the Total Symptom Score (TSS). Most importantly, we explore what this treatment could mean for patients with moderate to severe ISM.

INFOGRAPHIC

What we cover in our episode about avapritinib versus placebo:

Understanding ISM: How the KIT D816V mutation causes overproduction and overactivation of mast cells.

Why current treatment falls short: The limitations of best supportive care and why many patients remain symptomatic despite multiple medications.

How Avapritinib works: A tyrosine kinase inhibitor that targets the KIT mutation directly, aiming to reduce mast cell activation at its source.

Inside the PIONEER trial: Study design, eligibility criteria, and how outcomes like Total Symptom Score (TSS) and serum tryptase were measured.

What the data showed: Improvements in symptoms, lab markers, and quality of life and what this could mean for long-term ISM care.

The Itch Review, hosted by Dr. Gupta, Kortney, and Dr. Blaiss, explores allergy and immunology studies, breaking down complex research in conversations accessible to clinicians, patients, and caregivers. Each episode provides key insights from journal articles and includes a one-page infographic in the show notes for easy reference.

Made in partnership with The Allergy & Asthma Network.

Thanks to Blueprint Medicines for sponsoring today’s episode.

This podcast is for informational purposes only and does not substitute professional medical advice. Always consult with your healthcare provider for any medical concerns.

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