Autoinflammatory & rare systemic diseases
Low remission rates and high incidence of adverse events in a prospective VEXAS syndrome registry
Authors' conclusion
Patients with VEXAS syndrome required high-dose glucocorticoids to reduce disease activity, and complications-such as malignancy, thrombosis, and infection-occurred frequently within a short observation period.
Study context
- Population
- Patients in Japan suspected of having VEXAS syndrome; all male, median age 73.5 years30 with pathogenic UBA1 variants
- Participants
- 55 patients
- Outcome
- Disease activity, remission and AEs
- Follow-up
- 3 months
Main finding
As reported in the abstract
- Complete remission according to FRENVEX at 3 months
- 2
Observational data: associations, not effects.
Safety
Adverse events (AEs) during 6-month observation period
- AEs
- 28
- Deaths
- 3
- Malignancies (from two cases)
- 4
- Thromboses
- 2
- Infections
- 13
- Mycobacterial infections
- 4
- Inflammation of the lung and cervical region were the most common AEs
Original publication
Low remission rates and high incidence of adverse events in a prospective VEXAS syndrome registry. Rheumatology (Oxford). Published 28 September 2024. doi:10.
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