Evidence map›Paper›PMID 40582709›Full record

ArticleEuropean journal of haematology2025

Momelotinib Is Effective in Treatment for VEXAS Syndrome: Two Cases Within the AGMT Austrian Myeloid Registry.

Dominik Kiem, Michael Leisch, Ildiko Toth, Marie-Christina Mayer, Lisa Pleyer, Richard Greil, Alexander Egle, Thomas Melchardt

Abstract readCase Reports
PubMed Full text at PMC Publisher
In one paragraph

Article in European journal of haematology, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 2 papers.

0numbers the graph read from it
0cells of the map it votes in
2citing 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

2 citing papers in PubMed.

  1. A fatal case of VEXAS syndrome with a brief review.Journal of vascular surgery cases and innovative techniques · 2026
    Article
  2. 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

8 authors.

Dominik KiemDepartment of Internal Medicine III With Haematology, Medical Oncology, Haemostaseology, Infectiology and Rheumatology, Paracelsus Medical University, Salzburg, Austria.
Michael LeischDepartment of Internal Medicine III With Haematology, Medical Oncology, Haemostaseology, Infectiology and Rheumatology, Paracelsus Medical University, Salzburg, Austria.
Ildiko TothDepartment of Internal Medicine III With Haematology, Medical Oncology, Haemostaseology, Infectiology and Rheumatology, Paracelsus Medical University, Salzburg, Austria.
Marie-Christina MayerDepartment of Internal Medicine III With Haematology, Medical Oncology, Haemostaseology, Infectiology and Rheumatology, Paracelsus Medical University, Salzburg, Austria.
Lisa PleyerDepartment of Internal Medicine III With Haematology, Medical Oncology, Haemostaseology, Infectiology and Rheumatology, Paracelsus Medical University, Salzburg, Austria.
Richard GreilDepartment of Internal Medicine III With Haematology, Medical Oncology, Haemostaseology, Infectiology and Rheumatology, Paracelsus Medical University, Salzburg, Austria.
Alexander EgleDepartment of Internal Medicine III With Haematology, Medical Oncology, Haemostaseology, Infectiology and Rheumatology, Paracelsus Medical University, Salzburg, Austria.
Thomas MelchardtDepartment of Internal Medicine III With Haematology, Medical Oncology, Haemostaseology, Infectiology and Rheumatology, Paracelsus Medical University, Salzburg, Austria.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

VEXAS syndrome is caused by somatic mutations in the UBA1 gene and includes features of both autoinflammatory and myeloid diseases. Among several treatment options, JAK inhibitors have proven effective, especially ruxolitinib. However, anemia is often present in VEXAS syndrome. The novel JAK inhibitor momelotinib is approved for myelofibrosis with anemia. Here, we report of two patients within the Austrian Myeloid Registry of the Austrian Group Medical Tumor Therapy (AGMT), with newly diagnosed VEXAS syndrome and anemia, who were treated with momelotinib. Both patients experienced an improvement in anemia, a decrease in inflammation, and the treatment was well tolerated. VEXAS syndrome (acronym for: vacuoles, E1 enzyme, X-linked, autoinflammatory, somatic) is an autoinflammatory disease caused by somatic mutations in the ubiquitin-like modifier activating enzyme 1 (UBA1) gene. Key clinical features comprise hematological as well as rheumatological, features. Associated hematological conditions include cytopenia, bone marrow failure, myelodysplastic syndrome, increased risk for thromboembolic events, and prominent vacuolization of myeloid and erythroid precursor cells in the bone marrow.

Indexed as

Genetic Diseases, X-LinkedJanus Kinase InhibitorsPyrimidinesAgedAnemiaAustriaFemaleHumansMaleMiddle AgedMutationMyelodysplastic SyndromesRegistriesSkin Diseases, GeneticTreatment OutcomeUbiquitin-Activating EnzymesJanus Kinase InhibitorsPyrimidinesUBA1 protein, humanUbiquitin-Activating EnzymesautoinflammationJAK inhibitormyelodysplastic syndromeVEXAS syndrome

Identifiers

PMID40582709
PMCPMC12319881

What OpenQuestion holds

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Registered trials

None linked

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.