Efficacy and safety of conventional disease-modifying antirheumatic drugs in VEXAS syndrome

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

doi:https://doi.org/10.3389/fphar.2025.1539756

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Efficacy and safety of conventional disease-modifying antirheumatic drugs in VEXAS syndrome

https://repository.urosario.edu.co/handle/10336/46055
https://doi.org/10.3389/fphar.2025.1539756

Autores

Cantarini, Luca

Fabiani, Claudia

Frediani, Bruno

Balistreri, Alberto

De La Torre Cifuentes, Ligia Alejandra

Jahnz-Rózyk, Karina

Wiesik-Szewczyk, Ewa

Bocchia, Monica

Peña-Rodríguez, Mercedes

González-García, Andrés

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Fecha

2025-03-06

Editor

Frontiers

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Abstract

Background: VEXAS (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. Objectives: To assess the efficacy and safety of conventional disease-modifying antirheumatic drugs (cDMARDs) in a cohort of VEXAS patients. Methods: Data from VEXAS patients were obtained from the International AIDA Network VEXAS registry. Results: Data 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. Conclusion: Many 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.