Evidence map›Paper›PMID 42154291›Full record

ReviewRheumatology international2026

Exploring glucocorticoid dose-response patterns in VEXAS syndrome: a pilot retrospective study.

Nicolas Giachetti, Jérome Hadjadj, Noémie Gaudré, Christian Lavigne, Valentin Lacombe

Abstract readReview
In one paragraph

Review in Rheumatology international, 2026. 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. Review
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.

Nicolas GiachettiMédecine interne - Immunologie clinique, Centre National de Référence Maladies Auto-immunes Systémiques Rares du Nord, Nord-Ouest, Méditerranée et Guadeloupe (CeRAINOM-Angers), CHU d'Angers, Angers, France.
Jérome HadjadjService de medecine interne, Sorbonne Université, Hôpital Saint-Antoine, CEREMAIAA, AP-HP, Paris, France.ORCID http://orcid.org/0000-0002-2520-3272
Noémie GaudréMédecine interne et polyvalente, CH du Haut-Anjou, 1 quai du Dr Lefèvre, 53200, Château-Gontier, France.
Christian LavigneMédecine interne - Immunologie clinique, Centre National de Référence Maladies Auto-immunes Systémiques Rares du Nord, Nord-Ouest, Méditerranée et Guadeloupe (CeRAINOM-Angers), CHU d'Angers, Angers, France.ORCID http://orcid.org/0000-0002-2538-0076
Valentin LacombeMédecine interne et polyvalente, CH du Haut-Anjou, 1 quai du Dr Lefèvre, 53200, Château-Gontier, France. lacombe.valentin31@gmail.com.ORCID http://orcid.org/0000-0002-7184-752X

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

VEXAS syndrome is characterized by high glucocorticoid (GC) requirements and frequent relapses during tapering, yet dose-response relationships remain poorly defined. We conducted a pilot study to assess the feasibility of granular longitudinal treatment-line reconstruction and to explore GC dose-response patterns associated with disease activity and flare risk. In this retrospective single-center study, we reconstructed detailed treatment trajectories of patients with VEXAS syndrome. Disease courses were segmented into treatment-line periods defined by any therapeutic modification, including GC dose changes. For each period, GC dose, concomitant therapies, and clinical activity were recorded. Associations between GC dose and disease activity were analyzed using multivariable models adjusting for concomitant treatments. Twelve patients were included, representing 315 treatment-line periods over a mean follow-up of 45 months. Mean annual GC exposure was 3.27 ± 2.26 g per patient. Higher GC dose (continuous variable) was independently associated with lower odds of clinical activity (aOR 0.91 per mg increase [95% CI 0.88-0.94], p < 0.001). Exploratory threshold analyses suggested increased flare risk below approximately 15 mg/day (or 0.2 mg/kg/day), with a lower-risk plateau above 20 mg/day. The increased flare risk below 15 mg/day remained significant after adjustment for concomitant therapies (HR 0.24 [95% CI 0.11-0.54], p = 0.001). Organ-specific analyses suggested heterogeneous glucocorticoid sensitivity, with higher doses appearing required for lung involvement and lower doses appearing sufficient for fever, skin, and joint manifestations. This pilot study demonstrates the feasibility of detailed treatment-line reconstruction in VEXAS syndrome and identifies GC dose-response trends. These preliminary findings provide planning parameters for a future adequately powered study aimed at validating dose thresholds and refining tapering strategies in VEXAS.

Indexed as

GlucocorticoidsAdolescentChildDose-Response Relationship, DrugDrug TaperingFemaleHumansMaleMyelodysplastic SyndromesPilot ProjectsRetrospective StudiesSkin Diseases, GeneticTreatment OutcomeGlucocorticoidsDisease flareGlucocorticoid dependenceGlucocorticoid taperingSteroid-sparing therapiesUBA1 mutationVEXAS syndrome

Identifiers

PMID42154291
PMCPMC13186862

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Registered trials

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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.