Long‐term genetic and clinical remissions after cessation of azacitidine treatment in patients with VEXAS syndrome

Abstract excerpt

VEXAS (vacuoles, E1 enzyme, X-linked, autoinflammatory, somatic) is an inflammatory syndrome caused by acquired mutations in the gene encoding ubiquitin like modifier activating enzyme 1 (UBA1) that is often fatal.1, 2 Allogeneic hematopoietic stem cell transplantation is currently considered the only curative treatment modality.3-6 We were the first to report eradication of virtually all UBA1-mutated cells by...

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