{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,2]],"date-time":"2026-10-02T14:32:02Z","timestamp":1790951522007,"version":"4.1.0"},"reference-count":37,"publisher":"Frontiers Media SA","license":[{"start":{"date-parts":[[2026,4,29]],"date-time":"2026-04-29T00:00:00Z","timestamp":1777420800000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"content-domain":{"domain":["frontiersin.org"],"crossmark-restriction":true},"short-container-title":["Front. Immunol."],"abstract":"<jats:p>\n                    VEXAS syndrome (\u2018Vacuoles\u2019, \u2018E1 enzyme\u2019, \u2018X-linked\u2019, \u2018Autoinflammatory\u2019 and \u2018Somatic\u2019) is a rare autoinflammatory disorder caused by somatic mutations in the\n                    <jats:italic>UBA1<\/jats:italic>\n                    (ubiquitin-like modifier-activating enzyme 1) gene whose treatment and prognosis remain poorly understood. The GESMD group (Grupo Espa\u00f1ol de S\u00edndromes Mielodispl\u00e1sicos) conducted a retrospective multicenter study of 40 male patients (median age 74 years), to analyze clinical and biological features, treatments, responses, prognosis and outcomes.\n                    <jats:italic>UBA1<\/jats:italic>\n                    mutations were present in all patients, the c.209T&amp;gt;A mutation\u2014previously reported by Garc\u00eda-Escudero et al. as a novel and presumably causative variant in VEXAS syndrome\u2014was associated with older age at diagnosis, a lower frequency of lymph node involvement, and milder anemia. However, these data should be validated in larger cohorts. Overall, 58 treatment lines were administered, including corticosteroids, JAK inhibitors, anti-IL1 and anti-IL6 agents, hypomethylating agents (HMA), and other immunomodulators. Corticosteroid therapy led to rapid early improvement but limited by dependence and tapering difficulties. HMA showed a gradual and sustained increase in global, clinical, and biological responses over time, reaching high proportions of improvement at 12 months despite being used predominantly in more refractory patients (83.3% of patients showed a global response, including 50.0% of complete responses and 33.3% of partial responses). The median time to next treatment or death (TNT-D) was 11 months. Treatment discontinuation occurred in 25 of 58 lines (43.1%), mainly due to lack of efficacy, toxicity, or the need for steroid-sparing. Overall survival data remained immature due to the limited number of events, although gastrointestinal involvement appeared associated with poorer prognosis (\n                    <jats:italic>P<\/jats:italic>\n                    \u00a0=\u00a00.018), while achieving clinical or biological response correlated with better survival (\n                    <jats:italic>P<\/jats:italic>\n                    \u00a0=\u00a00.001 and\n                    <jats:italic>P<\/jats:italic>\n                    \u00a0=\u00a00.003). This study provides a detailed characterization of the Spanish VEXAS cohort, highlighting evolving therapeutic trajectories and the potential role of HMA, and underscores the need for larger prospective studies to define long-term outcomes.\n                  <\/jats:p>","DOI":"10.3389\/fimmu.2026.1743509","type":"journal-article","created":{"date-parts":[[2026,4,29]],"date-time":"2026-04-29T06:07:25Z","timestamp":1777442845000},"update-policy":"https:\/\/doi.org\/10.3389\/crossmark-policy","source":"Crossref","is-referenced-by-count":1,"title":["Clinical, laboratory and genetic characteristics of VEXAS syndrome: a study on behalf of GESMD"],"prefix":"10.3389","volume":"17","author":[{"given":"Marta","family":"Fonseca-Santos","sequence":"first","affiliation":[{"name":"Servicio de Hematolog\u00eda, Hospital Universitario de Salamanca (HUS)","place":["Salamanca, Spain"]},{"name":"Instituto de Investigaci\u00f3n Biom\u00e9dica de Salamanca (IBSAL)","place":["Salamanca, Spain"]},{"name":"Centro de Investigaci\u00f3n del C\u00e1ncer de Salamanca (CIC-IBMCC)","place":["Madrid, Spain"]},{"name":"Universidad de Salamanca (USAL)","place":["Salamanca, Spain"]},{"name":"Grupo Espa\u00f1ol de S\u00edndromes Mielodispl\u00e1sicos (GESMD)","place":["Madrid, Spain"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Borja","family":"Puertas","sequence":"additional","affiliation":[{"name":"Servicio de Hematolog\u00eda, Hospital Universitario de Salamanca (HUS)","place":["Salamanca, Spain"]},{"name":"Instituto de Investigaci\u00f3n Biom\u00e9dica de Salamanca (IBSAL)","place":["Salamanca, Spain"]},{"name":"Centro de Investigaci\u00f3n del C\u00e1ncer de Salamanca (CIC-IBMCC)","place":["Madrid, Spain"]},{"name":"Universidad de Salamanca (USAL)","place":["Salamanca, Spain"]},{"name":"Grupo Espa\u00f1ol de S\u00edndromes Mielodispl\u00e1sicos (GESMD)","place":["Madrid, Spain"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Javier","family":"Arzuaga-M\u00e9ndez","sequence":"additional","affiliation":[{"name":"Grupo Espa\u00f1ol de S\u00edndromes Mielodispl\u00e1sicos (GESMD)","place":["Madrid, Spain"]},{"name":"Hematology Department. Biocruces Bizkaia Health Research Institute, Hospital Universitario Cruces","place":["Barakaldo, Bizkaia, Spain"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Idoya","family":"Ancin-Arteaga","sequence":"additional","affiliation":[{"name":"Grupo Espa\u00f1ol de S\u00edndromes Mielodispl\u00e1sicos (GESMD)","place":["Madrid, Spain"]},{"name":"Hematology Department. Biocruces Bizkaia Health Research Institute, Hospital Universitario Cruces","place":["Barakaldo, Bizkaia, Spain"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sandra","family":"Casta\u00f1o-Diez","sequence":"additional","affiliation":[{"name":"Grupo Espa\u00f1ol de S\u00edndromes Mielodispl\u00e1sicos (GESMD)","place":["Madrid, Spain"]},{"name":"Hematology Department. 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Santa-Catalina","sequence":"additional","affiliation":[{"name":"Oncology Department, Hospital Universitario de Salamanca, IBSAL","place":["Salamanca, Spain"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ra\u00fal","family":"Azibeiro","sequence":"additional","affiliation":[{"name":"Servicio de Hematolog\u00eda, Hospital Universitario de Salamanca (HUS)","place":["Salamanca, Spain"]},{"name":"Instituto de Investigaci\u00f3n Biom\u00e9dica de Salamanca (IBSAL)","place":["Salamanca, Spain"]},{"name":"Centro de Investigaci\u00f3n del C\u00e1ncer de Salamanca (CIC-IBMCC)","place":["Madrid, Spain"]},{"name":"Universidad de Salamanca (USAL)","place":["Salamanca, Spain"]},{"name":"Grupo Espa\u00f1ol de S\u00edndromes Mielodispl\u00e1sicos (GESMD)","place":["Madrid, Spain"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Carlos","family":"Puerta","sequence":"additional","affiliation":[{"name":"Servicio de Hematolog\u00eda, Hospital Universitario de Salamanca (HUS)","place":["Salamanca, Spain"]},{"name":"Instituto de Investigaci\u00f3n Biom\u00e9dica de Salamanca (IBSAL)","place":["Salamanca, Spain"]},{"name":"Centro de Investigaci\u00f3n del C\u00e1ncer de Salamanca (CIC-IBMCC)","place":["Madrid, Spain"]},{"name":"Universidad de Salamanca (USAL)","place":["Salamanca, Spain"]},{"name":"Grupo Espa\u00f1ol de S\u00edndromes Mielodispl\u00e1sicos (GESMD)","place":["Madrid, Spain"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Alba","family":"Mesa-Tudela","sequence":"additional","affiliation":[{"name":"Grupo Espa\u00f1ol de S\u00edndromes Mielodispl\u00e1sicos (GESMD)","place":["Madrid, Spain"]},{"name":"Hematology Department. 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