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Here at Last: Treatment Options for VEXAS

Michael Putman, MD, MSCI
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We have known about the VEXAS (vacuoles, E1 enzyme, X-linked, autoinflammatory, somatic) syndrome for nearly 3 years, but prior to ACR Convergence 2023 there has been relatively little to say about how to treat it. To date there have been over twice as many publications about VEXAS (263 publications) as there have been patients described with respect to treatment strategies (116 patients). 

One of the late breaking abstracts may finally have rectified this imbalance, reporting on the “Efficacy and safety of targeted therapies in VEXAS syndrome: retrospective study from the FRENVEX” (Abstract L03). 

This abstract describes a French multicenter retrospective study that included patients with genetically proven VEXAS syndrome. Only those who received a targeted therapy could be included, and response rates (complete, partial, none) were recorded. The authors accumulated an impressive number of patients, who were predictably male (99%) and older (median age 71). Patients frequently required multiple therapies (48%) and rates of death ranged from 5% to 19%, depending on treatment. If nothing else, this would be a large and useful cohort study of patients with VEXAS. 

Thankfully, the authors went the additional step to provide something else – information about treatment strategies. Patients in the study received JAK inhibitors (71%), IL-6 inhibitors (51%), IL-1 inhibitors (30%), and TNF inhibitors (18%) (numbers add up to >100% due to patients receiving multiple agents). Kaplan-Meier curves from the study tell a compelling story; patients who received JAK inhibitors had a significantly lower rate of treatment withdrawal than other agents and a higher rate of complete response at 6 months (26% for JAK, 20% for IL-6).

At face value, these results finally provide clarity about a reasonable approach to patients with VEXAS.

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