Tocilizumab for treatment of cutaneous and systemic manifestations of vacuoles, E1 enzyme, X-linked, autoinflammatory, somatic (VEXAS) syndrome without myelodysplastic syndrome

Vacuoles, E1 enzyme, X-linked, autoinflammatory, somatic (VEXAS) syndrome is a newly described, adult-onset, autoinflammatory disorder caused by somatic mutations in the X-linked UBA1 gene. This syndrome is characterized by fevers, cytopenias, peripherally circulating myeloid and erythroid precursors with cytoplasmic vacuoles and dysplastic changes, pulmonary inflammation, chondritis, vasculitis, and joint pain and swelling.1,2 Nearly all patients with VEXAS present with cutaneous manifestations of the disease, most commonly a robust neutrophilic dermatosis.