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Case report: VEXAS syndrome with excellent response to treatment with azacitidine
Tzvika Porges1, Elli Rosenberg2, Ofir Wolach3
1Hematology Department, Soroka University Medical Center, Yitzhack Rager Blvd 151, Beer Sheva, 84101, Israel. tzvipo@clalit.org.il.
Abstract:
Vacuoles, E1 enzyme, X-linked, auto inflammatory, somatic (VEXAS) syndrome is an inflammatory disorder caused by somatic UBA1 variants and is characterized by late-onset systemic autoimmune inflammation and blood abnormalities. Glucocorticoids ameliorate symptoms effectively. However, other treatment options have limited efficacy and a transient effect. Herein, we describe a case of a 69-year-old male patient with VEXAS syndrome with skin, lung and hematologic involvement. He was treated with glucocorticoids and after the failure with anti IL-1 he began treatment with azacitidine with excellent hematological and clinical response. Azacitidine may be a suitable option for treating VEXAS syndrome, especially due to the relationship between inflammatory symptoms and response to azacitidine.
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