Canada’s Drug Agency | Drugs, Health Technologies and Systems.
Canada’s Drug Agency | Drugs, Health Technologies and Systems.

Treatment Options for VEXAS Syndrome

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Project Status:
Completed
Project Line:
Health Technology Review
Project Sub Line:
Rapid Review
Project Number:
RC1460-000
Expected finish date:

Question

  1. What is the clinical effectiveness of tocilizumab for patients diagnosed with VEXAS syndrome?
  2. What is the clinical effectiveness of ruxolitinib for patients diagnosed with VEXAS syndrome?
  3. What is the clinical effectiveness of azacitidine for patients diagnosed with VEXAS syndrome?
  4. What are the evidence-based guidelines regarding the treatment options for the management of VEXAS syndrome?

Key Message

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.

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Last Updated : November 11, 2022