Evidence map›Paper›PMID 40963125›Full record

ArticleAllergy, asthma, and clinical immunology : official journal of the Canadian Society of Allergy and Clinical Immunology2025

Avapritinib reduces symptoms and mast cell burden in systemic mastocytosis.

Paula Nöldeke, Oliver Schmalz, Hans Kvasnicka, Jens Panse, Silke C Hofmann

Abstract read
In one paragraph

Article in Allergy, asthma, and clinical immunology : official journal of the Canadian Society of Allergy and Clinical Immunology, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.

0numbers the graph read from it
0cells of the map it votes in
1citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

1 citing paper in PubMed.

  1. Article
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

5 authors.

Paula NöldekeCenter for Dermatology, Allergology and Dermatosurgery, Helios University Hospital Wuppertal, University Witten/Herdecke, Heusnerstr. 40, 42283, Wuppertal, Germany.
Oliver SchmalzDepartment for Hematology, Oncology and Palliative Medicine, Helios University Hospital Wuppertal, University Witten/Herdecke, Wuppertal, Germany.
Hans KvasnickaInstitute of Pathology, Helios University Hospital Wuppertal, University Witten/Herdecke, Wuppertal, Germany.
Jens PanseCenter for Integrated Oncology Aachen Bonn Cologne Düsseldorf (CIO ABCD), Aachen, Germany.
Silke C HofmannCenter for Dermatology, Allergology and Dermatosurgery, Helios University Hospital Wuppertal, University Witten/Herdecke, Heusnerstr. 40, 42283, Wuppertal, Germany. silke.hofmann@helios-gesundheit.de.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

backgroundMastocytosis is driven by a clonal expansion of mast cells, commonly triggered by the KIT D816V mutation which is present in over 90% of adult patients. Individuals with indolent systemic mastocytosis (ISM) frequently experience recurrent anaphylaxis and mast cell mediator-related symptoms, leading to substantial morbidity. In rare cases, progression to more severe subtypes, such as smoldering systemic mastocytosis (SSM), can occur. CASE PRESENTATION: We describe one patient with ISM and another with ISM transitioning to SSM, both treated with the selective KIT D816V inhibitor avapritinib at a daily dose of 25 mg. Following initiation of avapritinib, both patients exhibited a marked reduction in serum tryptase levels and complete remission of maculopapular cutaneous mastocytosis. Additionally, joint pain, gastrointestinal symptoms, and neurocognitive complaints decreased. Sustained clinical improvement over follow-up periods of 9 and 12 months was consistently reflected in disease-specific patient-reported outcome measures (PROMs).

conclusionsRegular clinical and laboratory monitoring, including serum tryptase and KIT D816V mutation assessment in peripheral blood, is essential in all ISM patients to detect early signs of disease progression. In refractory cases, avapritinib is a promising therapeutic option that can reduce mast cell burden, alleviate symptoms, and enhance overall quality of life.

Indexed as

Mast cellOsteoporosisPROMsTryptase

Identifiers

PMID40963125
PMCPMC12442258

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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.