Article
Efficacy and safety of targeted therapies in VEXAS syndrome: retrospective study from the FRENVEX.
Annals of the rheumatic diseases - 30 Sept 2024
Hadjadj Jerome, Nguyen Yann, Mouloudj Dalila, Bourguiba Rim, Heiblig Mael, Aloui Hassina, McAvoy Chloe, Lacombe Valentin, Ardois Samuel, Campochiaro Corrado, Maria Alexandre, Coustal Cyrille, Comont Thibault, Lazaro Estibaliz, Lifermann Francois, Le Guenno Guillaume, Lobbes Hervé, Grobost Vincent, Outh Roderau, Campagne Julien, Dor-Etienne Anais, Garnier Alice, Jamilloux Yvan, Dossier Antoine, Samson Maxime, Audia Sylvain, Nicolas Barbara, Mathian Alexis, de Maleprade Baptiste, De Sainte-Marie Benjamin, Faucher Benoit, Bouaziz Jean-David, Broner Jonathan, Dumain Cyril, Antoine Carole, Carpentier Benjamin, Castel Brice, Lartigau-Roussin Celine, Crickx Etienne, Volle Geoffroy, Fayard Damien, Decker Paul, Moulinet Thomas, Dumont Anael, Nguyen Alexandre, Aouba Achille, Martellosio Jean-Philippe, Levavasseur Matthieu, Puigrenier Sebastien, Antoine Pascale, Giraud Jean-Thomas, Hermine Olivier, Lacout Carole, Martis Nihal, Karam Jean-Denis, Chasset Francois, Arnaud Laurent, Marianetti Paola, Deligny Christophe, Chazal Thibaud, Woaye-Hune Pascal, Roux-Sauvat Murielle, Meyer Aurore, Sujobert Pierre, Hirsch Pierre, Abisror Noemie, Fenaux Pierre, Kosmider Olivier, Jachiet Vincent, Fain Olivier, Terrier Benjamin, Mekinian Arsène, Georgin-Lavialle Sophie
Abstract excerpt
OBJECTIVES: 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. METHODS: Multicentre retrospective study including patients with genetically proven VEXAS syndrome who had received at least one...
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