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Clinical reviews
VEXAS SYNDROME: A COMPREHENSIVE REVIEW OF PATHOGENESIS, CLINICAL SPECTRUM, AND THERAPEUTIC STRATEGIES
Emma M Groarke, Benjamin Turturice, Bhavisha A Patel, Kaitlin A Quinn, Alice Fike, Peter C Grayson
PDF12 pages1.9 MB
VEXAS syndrome is a recently identified monogenic disease affecting older men, characterized by severe systemic inflammation and progressive bone marrow failure due to mutations in the UBA1 gene. The review discusses the clinical features, epidemiology, and potential treatment options, including glucocorticoids and emerging therapies like JAK inhibitors and hypomethylating agents. It emphasizes the complexity of managing this condition and the need for further research into effective therapies.
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