Autoinflammatory & rare systemic diseases
Disease Trajectories and Glucocorticoid Exposure in VEXAS Syndrome Treated with Cytokine-Directed Therapies
Authors' conclusion
Cytokine-directed therapies alone do not alter the risk of haematologic disease progression or significantly reduce glucocorticoid exposure in VEXAS.
Study context
- Population
- Patients with VEXAS syndrome (81.7% with anti-IL6R or Janus kinase inhibitors (JAKi) exposure)median follow-up 4.8 years
- Participants
- 71 patients
- Exposure
- Event-free survival by UBA1 genotype and CHVAF≥10%; GC exposure and outcomes in relation to anti-IL6R or JAKi initiation
- Outcome
- Event-free survival:
- Transfusion dependence
- Haematopoietic stem cell transplantation
- Death
Methodological detailsprospective follow-up for events, retrospective assessment of laboratory results, GC exposure and clinical measures
- Prospective follow-up for events
- Retrospective assessment of laboratory results, GC exposure and clinical measures
Main finding
As reported in the abstract
Event-free survival:
- Events: transfusion dependence
- HSCT
- Death
Each group vs p.M41LHR for event (transfusion dependence, HSCT or death) (95% CI)
p.M41V
5.7(1.5–20.4)
p.M41L/T with CHVAF≥10%
5.7(1.6–20.8)
Observational data: associations, not effects.
Further findings
Anaemia, elevated C-reactive protein (CRP) and monocytopenia
- p.M41V genotype had the highest risk
Mean GC dose · median follow-up 4.8 (IQR 3.0, 8.1) years
- Mean GC dose >15 mg/day prednisone regardless of variant or disease duration
Clinical remission on ≤10 mg/day prednisone at prospective visits
- Proportion of visits
- 2.7%
Steroid exposure after anti-IL6R or JAKi · 1 year post-treatment
- No clinically meaningful reduction (<5 mg/day difference)
Progression of anaemia after anti-IL6R and JAKi
- No attenuation observed
Original publication
Disease Trajectories and Glucocorticoid Exposure in VEXAS Syndrome Treated with Cytokine-Directed Therapies. Ann Rheum Dis. Published 27 June 2025. doi:10.
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