Navigating through uncertainty-Experience from the UK national VEXAS MDT

Area of Science:

• Rheumatology
• Genetics
• Immunology

Background:

• Vacuoles, E1 enzyme, X-linked, autoinflammatory, somatic (VEXAS) syndrome is a rare, severe autoinflammatory disease.
• Optimal management strategies for VEXAS syndrome are still evolving, lacking formal treatment guidelines.

Purpose of the Study:

• To establish and evaluate the UK national multidisciplinary team (MDT) for VEXAS syndrome.
• To assess the MDT's clinical impact, structure, and perceived value among clinicians.
• To analyze treatment shifts and patient outcomes following MDT review.

Main Methods:

• Retrospective review of 42 patients discussed at the national VEXAS MDT (June 2024-May 2025).
• Extraction of clinical data from referral forms and MDT recommendations.
• Anonymised questionnaire assessing clinician experiences and confidence levels.

Main Results:

• 36 patients had confirmed VEXAS syndrome; most were male (median age 70).
• MDT recommendations shifted from corticosteroid monotherapy to steroid-sparing agents like azacitidine and tocilizumab.
• Increased clinician confidence and value placed on MDT's educational and collaborative aspects.

Conclusions:

• The UK national VEXAS MDT facilitates evidence-informed, multidisciplinary decision-making.
• The MDT promotes targeted VEXAS syndrome therapies and enhances clinical confidence and collaboration.
• The MDT provides crucial support in the absence of established treatment guidelines for VEXAS syndrome.