Avapritinib for Treatment of Indolent Systemic Mastocytosis (Pioneer Trial)
Welcome to the latest episode of the Allergy, Asthma & Immunology Innovations podcast. Our hosts Payel Gupta, MD, and Kortney are joined by allergist Michael Blaiss, MD, for The Itch Review, a series to talk about allergy and immunology research, breaking it down into simple terms.
This episode looks at a medication called avapritinib and how it works to treat the root cause of indolent systemic mastocytosis, or ISM. The hosts review the article “Avapritinib versus Placebo in Indolent Systemic Mastocytosis,” published May 23, 2024 in The New England Journal of Medicine Evidence.
The study asks: What happens when you target the root cause of indolent systemic mastocytosis? You will learn what ISM is, what treatments are available, what the PIONEER study examined, and what it found.
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Thanks to Blueprint Medicines for sponsoring this episode.
What we cover in this episode about avapritinib
- What is ISM? It’s a rare disease in which the body makes too many mast cells. These cells can cause health problems like itching, stomach pain, diarrhea, tiredness, and even serious allergic reactions.
- What is avapritinib? Avapritinib is a pill that blocks the signal that tells mast cells to keep growing. It’s the first treatment to focus on the cause of the disease, not just the symptoms.
- What did the study do? The study looked at adults with ISM who still had bad symptoms even with current treatment.
What did the study find? People who took avapritinib had fewer symptoms and fewer markers for ISM, like serum tryptase. - Why does this matter? For the first time, people with ISM have a treatment that may lower the number of mast cells in their body. It could help them feel better and live a more normal life.
Timestamps for our episode on avapritinib
01:20 – What ISM is and how it affects the body
03:55 – Common symptoms people with ISM may feel
05:06 – How ISM is treated today
07:17 – What avapritinib is and how it helps
12:02 – What the PIONEER Part 2 study is about
13:43 – How doctors measure symptoms using TSS
15:25 – Who was allowed to join the study
18:20 – How the study was set up
21:57 – How patients were split into groups (2 got medicine, 1 got placebo)
25:38 – Why doctors looked at tryptase levels
27:50 – What the main results of the study were
28:57 – What a p-value means in a study
30:46 – Other results the study looked at
33:25 – What side effects doctors checked for
36:17 – What this all means for people with ISM
Resources about mast cell disease
- Mast Cell Disease Toolkit
- What is Mastocytosis? Types, Symptoms, and Treatment
- Mast Cell Disease Glossary of Terms
Other podcast episodes about mast cell disease
