Avapritinib for Treatment of Indolent Systemic Mastocytosis (Pioneer Trial)

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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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This podcast is for informational purposes only and does not substitute professional medical advice. Any mention of brands is also informational and not an endorsement. Always consult with your healthcare provider for any medical questions or concerns.

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.
An infographic reviewing a study comparing avapritinib versus placebo in indolent systemic mastocytosis, summarizing study design, treatment options, key findings, graphs, and implications for patient symptom improvement.
Click image to see full graphic or to download.

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

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