Autoinflammatory & rare systemic diseases
Efficacy and safety of targeted therapies in VEXAS syndrome: retrospective study from the FRENVEX
Authors' conclusion
This study shows the benefit of JAKi and IL-6 inhibitors, whereas other therapies have lower efficacy.
Study context
- Population
- Patients with genetically proven VEXAS syndrome who received at least one targeted therapy (median age 71 years)therapies in 110 patients
- Participants
- 194 patients
- Exposure
- Targeted therapies in VEXAS syndrome
- JAKin = 78
- IL-6 inhibitorsn = 51
- IL-1 inhibitorsn = 33
- TNFα blockersn = 20
- Other targeted therapiesn = 12
- Outcome
- Overall response (complete response (CR): clinical remission, C-reactive protein (CRP) ≤10 mg/L and ≤10 mg/day prednisone-equivalent; Partial response (PR): clinical remission and 50% reduction in CRP and GC dose)
- Follow-up
- 3 months
Methodological detailsmulticentre
- Multicentre
Main finding
As reported in the abstract
Overall response (CR and PR) rate
Observational data: associations, not effects.
Further findings
Overall response rate · 6 months
Causes of discontinuation: primary failure / secondary failure (among patients who discontinued)
Primary failure
Secondary failure
Survival without treatment discontinuation
- Significantly longer with JAKi than with the other targeted therapies
Safety
Discontinuation due to serious adverse event or death (among patients who discontinued)
Serious adverse event
Death
Original publication
Efficacy and safety of targeted therapies in VEXAS syndrome: retrospective study from the FRENVEX. Ann Rheum Dis. Published 30 September 2024. doi:10.
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