Treatment outcomes in patients with VEXAS syndrome: a retrospective cohort study
Abstract
BACKGROUND
Vacuoles, E1 enzyme, X-linked, autoinflammatory, somatic (VEXAS) syndrome is a recently described autoinflammatory disorder with little therapeutic evidence. We compared treatment outcomes of targeted therapies versus prednisolone alone in the largest UK cohort of patients with VEXAS syndrome to date.
METHODS
In this retrospective cohort study, we analysed the outcomes of targeted therapies in patients with VEXAS syndrome in six tertiary referral centres across the UK between July 22, 2014, and Oct 19, 2024. The inclusion criteria were genetically confirmed VEXAS syndrome and receipt of at least one targeted therapy or prednisolone alone. Patients without clinical information at all timepoints after baseline were excluded. Data collection forms were used to record clinical and biochemical data at the following timepoints: time of diagnosis, initiation of treatment, and follow-up at 3 months, 6 months, and 12 months from the initiation of treatment (±28 days). Laboratory parameters, including C-reactive protein (CRP) and haemoglobin, and glucocorticoid doses were collected at each timepoint and compared between timepoints. Primary outcomes were complete response (ie, clinical remission, CRP ≤10 mg/L, and prednisolone ≤10 mg per day) and partial response (ie, clinical remission with ≥50% reductions in both CRP and glucocorticoid dose from baseline) to treatment. Treatment discontinuation and adverse events were documented for each treatment. Due to the high…
How strong is the evidence?
Evidence Hub reads this as a cohort study, done in people. Its evidence level is 3 of 5 (Moderate · trial / cohort / secondary analysis). Trials without a large randomized design, cohorts and secondary analyses give moderate evidence. Reported size: 59 participants or records.
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