Vol. 2 No. 11 (2022)
Health Technology Reviews

Treatment Options for VEXAS Syndrome

Published November 7, 2022

Key Messages

  • VEXAS (vacuoles, E1 enzyme, X-linked, autoinflammatory, somatic) syndrome is a disorder caused by a genetic mutation, and is characterized by hematologic and autoinflammatory symptoms that are severe and progressive. It is typically seen in older men and is considered to be rare.
  • Proposed treatments for VEXAS syndrome include DNA hypomethylating agents such as azacitidine, anti-IL6 monoclonal antibodies such as tocilizumab, and Janus kinase inhibitors such as ruxolitinib.
  • We found 5 non-randomized studies on the effectiveness of the proposed treatments — 4 on azacitidine; 1 on ruxolitinib; and 1 on azacitidine, ruxolitinib, and tocilizumab.
  • There are several limitations to the research studies, most notably that they included a small number of patients who were retrospectively identified as having VEXAS syndrome. Therefore, the effectiveness of tocilizumab, ruxolitinib, and azacitidine for treating VEXAS syndrome is uncertain.
  • We did not find any evidence-based guidelines on the management of VEXAS syndrome.