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Efficacy and safety of targeted therapies in VEXAS syndrome: retrospective study from the FRENVEX

2024; BMJ; Volume: 83; Issue: 10 Linguagem: Inglês

10.1136/ard-2024-225640

1468-2060

Jérôme Hadjadj, Yann Nguyen, Dalila Mouloudj, R. Bourguiba, Maël Heiblig, Hassina Aloui, Chloé McAvoy, Valentin Lacombe, Samuel Ardois, Corrado Campochiaro, Alexandre Maria, Cyrille Coustal, Thibault Comont, Estibaliz Lazaro, François Lifermann, G. Le Guenno, Hervé Lobbes, Vincent Grobost, R. Outh, J. Campagne, Anais Dor-Etienne, Alice Garnier, Yvan Jamilloux, Antoine Dossier, Maxime Samson, S. Audia, Barbara Nicolas, Alexis Mathian, B. De Maleprade, B. De Sainte Marie, B. Faucher, Jean‐David Bouaziz, Jonathan Broner, Cyril Dumain, Carole Antoine, Benjamin Carpentier, Brice Castel, Céline Lartigau-Roussin, Étienne Crickx, Geoffroy Volle, Damien Fayard, Paul Decker, T. Moulinet, Anaël Dumont, Alexandre Nguyen, Achille Aouba, Jean-Philippe Martellosio, M. Levavasseur, S. Puigrenier, Pascale Antoine, Jean-Thomas Giraud, Olivier Hermine, Carole Lacout, N. Martis, J.D. Karam, François Chasset, Laurent Arnaud, Paola Marianetti, C. Deligny, Thibaud Chazal, Pascal Woaye-Hune, Murielle Roux-Sauvat, A. Meyer, Pierre Sujobert, Pierre Hirsch, Noémie Abisror, Pierre Fenaux, Olivier Kosmider, Vincent Jachiet, O. Fain, Benjamin Terrier, A. Mékinian, Sophie Georgin‐Lavialle,

Peptidase Inhibition and Analysis

Vacuoles, E1 enzyme, X-linked, autoinflammatory and somatic (VEXAS) syndrome is an adult-onset autoinflammatory disease associated with somatic ubiquitin-like modifier-activating enzyme 1 (UBA1) mutations. We aimed to evaluate the efficacy and safety of targeted therapies.