Autoinflammatory & rare systemic diseases
Treatment outcomes in patients with VEXAS syndrome: a retrospective cohort study
Main finding · Response in those who continued therapyResult — go to the resultRelative risk 2.47 95% CI 1.18–5.20Authors' conclusion
In this UK cohort of patients with VEXAS syndrome, azacitidine and tocilizumab showed superior effectiveness compared with anakinra, baricitinib, and prednisolone only.
Study context
- Population
- Patients with genetically confirmed VEXAS syndrome receiving ≥1 targeted therapy or prednisolone alone, six UK tertiary referral centres, 2014–2024 (98% male; mean age 71 years; 46% myelodysplastic syndrome)71 targeted therapies in 59 patients
- Participants
- 13 / 19 / 13 / 11 / 10
- Exposure
- Targeted therapies vs prednisolone alone
- Azacitidinen = 13
- Tocilizumabn = 19
- Anakinran = 13
- Baricitinibn = 11
- Prednisolone onlyn = 10reference
- Outcome
- Complete response (clinical remission, C-reactive protein (CRP) ≤10 mg/L, prednisolone ≤10 mg/day) and partial response (clinical remission with ≥50% reductions in CRP and GC dose from baseline)
Methodological detailsmulticentre (six tertiary referral centres)
- Multicentre (six tertiary referral centres)
Main finding
Response in those who continued therapy · 6 months
Relative risk
2.47
95% CI 1.18–5.20
P = 0.018
Events by group
Azacitidinevs other therapies
RR 2.47 (95% CI 1.18–5.20); P = 0.018
As reported in the abstract
Observational data: associations, not effects.
Further findings
| Outcome | Values |
|---|---|
| Median CRP, baseline to 6 months | Tocilizumab median 4 (IQR 3–37) mg/L · baseline 30 mg/L [IQR 13-45] · Anakinra median 2 (IQR 1–28) mg/L · baseline 18 mg/L [11-52] |
| Mean haemoglobin, baseline to 6 months (greatest increase) | Azacitidine mean 120 (SD 14.4) g/L · baseline 104 g/L [SD 17.5] |
Predictors of outcome
- Absence of fever or thromboembolism at diagnosis associated with better outcomes
Safety
| Outcome | Values |
|---|---|
| Anakinra discontinuation | Anakinra 62% |
| Infections | Azacitidine 62% · Tocilizumab 47% |
- Anakinra discontinuation: mostly due to severe injection-site reactions (n=5)
Treatment discontinuation (all treatments)
- Discontinued
- 28 (39%) of 71
- Due to serious adverse events
- 12 [17%]
- Due to death
- nine [13%]
Original publication
Treatment outcomes in patients with VEXAS syndrome: a retrospective cohort study. Lancet Rheumatol. Published 21 May 2025. doi:10.
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