VEXAS syndrome is a rare, adult-onset, life-threatening autoinflammatory disease caused by a genetic mutation. The pathophysiology is still unknown, but new work presented at the 2024 congress of EULAR—The European Alliance of Associations for Rheumatology—aims to provide a molecular and phenotypic characterization of hematopoiesis in VEXAS patients, and to develop cellular and humanized mouse models by gene editing.
Neoadjuvant sacituzumab govitecan demonstrated efficacy in patients with muscle-invasive bladder cancer, but its future role in the space is uncertain.