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

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dc.creatorCantarini, Lucaspa
dc.creatorFabiani, Claudiaspa
dc.creatorFrediani, Brunospa
dc.creatorBalistreri, Albertospa
dc.creatorDe La Torre Cifuentes, Ligia Alejandraspa
dc.creatorJahnz-Rózyk, Karinaspa
dc.creatorWiesik-Szewczyk, Ewaspa
dc.creatorBocchia, Monicaspa
dc.creatorPeña-Rodríguez, Mercedesspa
dc.creatorGonzález-García, Andrésspa
dc.creatorDeniz Batu, Ezgispa
dc.creatorRagab, Gaafarspa
dc.creatorLa Torre, Francescospa
dc.creatorConticini, Edoardospa
dc.creatorVitetta, Rosettaspa
dc.creatorBixio, Riccardospa
dc.creatorViapiana, Ombrettaspa
dc.creatorGurnari, Carmelospa
dc.creatorTriggianese, Paolaspa
dc.creatorSoto-Peleteiro, Adrianaspa
dc.creatorRuiz-Irastorza, Guillermospa
dc.creatorMonti, Saraspa
dc.creatorMontecucco, Carlomauriziospa
dc.creatorIannone, Florenzospa
dc.creatorLopalco, Giuseppespa
dc.creatorGavioli, Francescospa
dc.creatorAlves Cordeiro, Rafaelspa
dc.creatorMayrink Giardini, Henrique A.spa
dc.creatorMormile, Ilariaspa
dc.creatorDe Paulis, Amatospa
dc.creatorD’Agostino, Maria Antoniettaspa
dc.creatorCauli, Albertospa
dc.creatorPiga, Matteospa
dc.creatorVasi, Ibrahimspa
dc.creatorTufan, Abdurrahmanspa
dc.creatorSota, Jurgenspa
dc.creatorSbalchiero, Jessicaspa
dc.creatorBaggio, Chiaraspa
dc.creatorBindoli, Saraspa
dc.creatorSfriso, Paolospa
dc.creatorAraújo, Olgaspa
dc.creatorGómez-Caverzaschi, Verónicaspa
dc.creatorHernández-Rodríguez, Joséspa
dc.creatorFranceschini, Francospa
dc.creatorCrisafulli, Francescaspa
dc.creatorFrassi, Micolspa
dc.creatorCampochiaro, Corradospa
dc.creatorTomelleri, Alessandrospa
dc.creatorDagna, Lorenzospa
dc.creatorBeecher, Markspa
dc.creatorCallisto, Aliciaspa
dc.creatorHissaria, Pravinspa
dc.creatorKawakami-Campos, Perla Ayumispa
dc.creatorTorres-Ruiz, Jiramspa
dc.creatorGuaracha-Basañez, Guillermo Arturospa
dc.creatorMartín-Nares, Eduardospa
dc.creatorHinojosa-Azaola, Andreaspa
dc.creatorCaggiano, Valeriaspa
dc.creatorLeone, Flaviaspa
dc.creatorVitale, Antoniospa
dc.date.accessioned2025-07-21T16:38:21Z
dc.date.available2025-07-21T16:38:21Z
dc.date.created2025-03-06spa
dc.date.issued2025-03-06spa
dc.description.abstractBackground: 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.eng
dc.format.mimetypeapplication/pdfspa
dc.identifier.doihttps://doi.org/10.3389/fphar.2025.1539756spa
dc.identifier.issn1663-9812spa
dc.identifier.urihttps://repository.urosario.edu.co/handle/10336/46055
dc.language.isoengspa
dc.publisherFrontiersspa
dc.relation.ispartofFrontiers Pharmacology, 06 March 2025 Sec. Inflammation Pharmacology Volume 16 - 2025spa
dc.relation.urihttps://www.frontiersin.org/journals/pharmacology/articles/10.3389/fphar.2025.1539756/fullspa
dc.rightsAttribution-NonCommercial-NoDerivatives 4.0 Internationalspa
dc.rights.accesRightsinfo:eu-repo/semantics/openAccessspa
dc.rights.accesoAbierto (Texto Completo)spa
dc.rights.urihttp://creativecommons.org/licenses/by-nc-sa/4.0/spa
dc.sourceFrontiers Pharmacologyspa
dc.source.instnameinstname:Universidad del Rosariospa
dc.source.reponamereponame:Repositorio Institucional EdocURspa
dc.subject.keywordFarmacologíaeng
dc.subject.keywordcDMARDseng
dc.subject.keywordClinical outcomeseng
dc.subject.keywordAutoinflammatory diseaseseng
dc.subject.keywordDiagnosiseng
dc.subject.keywordTreatmenteng
dc.titleEfficacy and safety of conventional disease-modifying antirheumatic drugs in VEXAS syndromespa
dc.typearticlespa
dc.type.hasVersioninfo:eu-repo/semantics/publishedVersionspa
dc.type.spaArtículo de Investigaciónspa
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