AUTHOR=Vitale Antonio , Leone Flavia , Caggiano Valeria , Hinojosa-Azaola Andrea , Martín-Nares Eduardo , Guaracha-Basañez Guillermo Arturo , Torres-Ruiz Jiram , Ayumi Kawakami-Campos Perla , Hissaria Pravin , Callisto Alicia , Beecher Mark , Dagna Lorenzo , Tomelleri Alessandro , Campochiaro Corrado , Frassi Micol , Crisafulli Francesca , Franceschini Franco , Hernández-Rodríguez José , Gómez-Caverzaschi Verónica , Araújo Olga , Sfriso Paolo , Bindoli Sara , Baggio Chiara , Sbalchiero Jessica , Sota Jurgen , Tufan Abdurrahman , Vasi Ibrahim , Piga Matteo , Cauli Alberto , D’Agostino Maria Antonietta , De Paulis Amato , Mormile Ilaria , Mayrink Giardini Henrique A. , Cordeiro Rafael Alves , Gavioli Francesco , Lopalco Giuseppe , Iannone Florenzo , Montecucco Carlomaurizio , Monti Sara , Ruiz-Irastorza Guillermo , Soto-Peleteiro Adriana , Triggianese Paola , Gurnari Carmelo , Viapiana Ombretta , Bixio Riccardo , Vitetta Rosetta , Conticini Edoardo , La Torre Francesco , Ragab Gaafar , Batu Ezgi Deniz , González-García Andrés , Peña-Rodríguez Mercedes , Bocchia Monica , Wiesik-Szewczyk Ewa , Jahnz-Rózyk Karina , de-la-Torre Alejandra , Balistreri Alberto , Frediani Bruno , Fabiani Claudia , Cantarini Luca 

TITLE=Efficacy and safety of conventional disease-modifying antirheumatic drugs in VEXAS syndrome: real-world data from the international AIDA network

JOURNAL=Frontiers in Pharmacology

VOLUME=Volume 16 - 2025

YEAR=2025

URL=https://www.frontiersin.org/journals/pharmacology/articles/10.3389/fphar.2025.1539756

DOI=10.3389/fphar.2025.1539756

ISSN=1663-9812

ABSTRACT=BackgroundVEXAS (vacuoles, E1 enzyme, X-linked, autoinflammatory, somatic) syndrome is an adult-onset autoinflammatory condition resulting in severe, often treatment-refractory inflammation. Currently, there are no established treatment guidelines for VEXAS syndrome.ObjectivesTo assess the efficacy and safety of conventional disease-modifying antirheumatic drugs (cDMARDs) in a cohort of VEXAS patients.MethodsData from VEXAS patients were obtained from the International AIDA Network VEXAS registry.ResultsData from 36 VEXAS patients were evaluated, with 28 (77.8%) treated with cDMARDs as monotherapy - and concomitant glucocorticoids (GC) - and 8 (22.2%) receiving a combination of different cDMARDs plus GC. Complete response (CR), partial response (PR), and failure to cDMARDs monotherapy were reported in 4/22 (18.2%), 11/22 (50%), and 7/22 (31.8%) courses, respectively. All patients were treated with GCs at the start of cDMARD monotherapy, and no GC discontinuation was observed later. No significant differences were observed in the GC dosage from the start of cDMARDs to the 3-month (p = 0.43), 6-month (p = 0.31), and 12-month (p = 0.21) visits. Conversely, the GC sparing resulted to be statistically significant when using methotrexate (p = 0.02). As for cDMARDs combinations, no cases achieved CR, while PR was observed in 5/9 (55.6%). Seventeen adverse events were reported, seven of which led to discontinuation.ConclusionMany VEXAS patients report a partial benefit from cDMARDs, while a smaller yet not negligible number of patients exhibit a CR; cDMARDs remain a viable option for this disorder, especially when the initial GC dosage is low and the need for a steroid-sparing effect is not immediately urgent.