Article
Successful allogeneic hematopoietic stem cell transplantation in patients with VEXAS syndrome: a 2-center experience.
Blood advances - 8 Feb 2022
Diarra Ava, Duployez Nicolas, Fournier Elise, Preudhomme Claude, Coiteux Valérie, Magro Leonardo, Quesnel Bruno, Heiblig Maël, Sujobert Pierre, Barraco Fiorenza, Balsat Marie, Scanvion Quentin, Hachulla Eric, Launay David, Yakoub-Agha Ibrahim, Terriou Louis
Abstract excerpt
The recently described vacuoles, E1 enzyme, X-linked, autoinflammatory, somatic (VEXAS) syndrome is caused by somatic mutations in UBA1. Patients with VEXAS syndrome display late-onset autoinflammatory symptoms, usually refractory to treatment, and hematologic abnormalities. The identification of an easily-accessible specific marker (UBA1 mutations) is of particular interest as it allows the convergence of...
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