Navigating through uncertainty-Experience from the UK national VEXAS MDT.

The objective of this study was to describe the establishment, structure and influence of the United Kingdom national multidisciplinary team (MDT) for vacuoles, E1 enzyme, X-linked, autoinflammatory, somatic (VEXAS) syndrome and to assess its clinical outputs and perceived value among participating clinicians. All patients discussed at the national VEXAS MDT between June 2024 and May 2025 were included. Clinical information was extracted from standardised referral forms, and MDT recommendations were reviewed. An anonymised questionnaire evaluated clinicians' experience of the MDT. Forty-two patients from 27 centres were reviewed; 36 (85.7%) had confirmed VEXAS syndrome and 6 had VEXAS-like disease. Almost all were male (41/42), with a median age of 70 years. Before MDT review, 62% received corticosteroid monotherapy. MDT recommendations favoured steroid-sparing strategies, with marked increases in the use of azacitidine (1-22 patients) and tocilizumab (5-15 patients), alongside reduced use of conventional disease-modifying anti-rheumatic drugs (DMARDs). Four patients were referred for haematopoietic stem cell transplantation. Twenty of 43 MDT members completed the survey, reporting greater confidence in managing VEXAS and valuing the MDT's educational and collaborative benefits. The national VEXAS MDT supported evidence-informed, multidisciplinary decision-making, promoted more targeted therapy and strengthened clinical confidence and collaboration in the absence of formal treatment guidelines.