{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,11,6]],"date-time":"2026-11-06T02:48:25Z","timestamp":1793933305824,"version":"4.8.0"},"reference-count":33,"publisher":"American Society of Hematology","issue":"12","content-domain":{"domain":["ashpublications.org"],"crossmark-restriction":true},"short-container-title":[],"published-print":{"date-parts":[[2025,9,18]]},"abstract":"<jats:sec>\n                    <jats:title>Abstract<\/jats:title>\n                    <jats:p>VEXAS (vacuoles, E1 enzyme, X-linked, autoinflammatory, somatic) syndrome is a severe monogenic disorder caused by somatic mutations in ubiquitin-like modifier activating enzyme 1 (UBA1), characterized by inflammation, cytopenias, and frequent association with myelodysplastic neoplasms (MDS). Steroid dependence is common, and targeted therapies have demonstrated limited efficacy. Azacitidine (AZA), a hypomethylating agent used in MDS, has shown potential in VEXAS syndrome but data remain limited. This multicenter retrospective study assessed AZA efficacy and safety in 88 patients with genetically confirmed VEXAS syndrome from FRENVEX (French VEXAS study group), 80% meeting World Health Organization 2022 MDS criteria. Inflammatory response rates were 41% at 6 months and 54% at 12 months, regardless of MDS status. A total of 50 (61%) patients achieved inflammatory response, with 70% occurring at 6 months, suggesting a delayed median response. Among responders, relapse-free survival on AZA was 90% at 1 year and 85% at 5 years. Of the 12 responders who discontinued AZA, 9 relapsed after a median of 3.1 years (range, 0.4-5.6), with effective reexposure in 4 of 5 patients. Hematological responses included red blood cell transfusion independence in 65% and platelet improvement in 77% of patients. Molecular response, defined as a \u226525% reduction in UBA1 variant allele frequency (VAF), was observed in 65% of patients, all of whom achieved inflammatory and hematological responses; and VAF dropped to &amp;lt;2% in 43% of cases. Infections (34%) and cytopenias (36%) were common, particularly during the first 3 cycles. This study establishes AZA as an effective therapy for VEXAS syndrome, improving inflammation, cytopenias, and UBA1 clonal burden, warranting larger prospective trials.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1182\/blood.2024028133","type":"journal-article","created":{"date-parts":[[2025,5,15]],"date-time":"2025-05-15T16:19:17Z","timestamp":1747325957000},"page":"1450-1461","update-policy":"https:\/\/doi.org\/10.1182\/blood.2019cm0000","source":"Crossref","is-referenced-by-count":44,"title":["Efficacy and safety of azacitidine for VEXAS syndrome: a large-scale retrospective study from FRENVEX"],"prefix":"10.1182","volume":"146","author":[{"given":"Vincent","family":"Jachiet","sequence":"first","affiliation":[{"name":"1Service de M\u00e9decine Interne, H\u00f4pital Saint-Antoine, Centre de R\u00e9f\u00e9rence des Maladies Auto-Inflammatoires et des Amyloses, Assistance Publique-H\u00f4pitaux de Paris, Sorbonne Universit\u00e9, Paris, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-6021-4057","authenticated-orcid":false,"given":"Olivier","family":"Kosmider","sequence":"additional","affiliation":[{"name":"2Service d'H\u00e9matologie Biologique, H\u00f4pital Cochin, Assistance Publique-H\u00f4pitaux de Paris, Paris, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-4924-093X","authenticated-orcid":false,"given":"Maxime","family":"Beydon","sequence":"additional","affiliation":[{"name":"3Institut Pierre Louis d'Epid\u00e9miologie et de Sant\u00e9 Publique, Equipe Pharmaco\u00e9pid\u00e9miologie et \u00c9valuation des Soins, H\u00f4pital Piti\u00e9-Salp\u00e9tri\u00e8re, Assistance Publique-H\u00f4pitaux de Paris, Sorbonne Universit\u00e9, Paris, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"J\u00e9r\u00f4me","family":"Hadjadj","sequence":"additional","affiliation":[{"name":"1Service de M\u00e9decine Interne, H\u00f4pital Saint-Antoine, Centre de R\u00e9f\u00e9rence des Maladies Auto-Inflammatoires et des Amyloses, Assistance Publique-H\u00f4pitaux de Paris, Sorbonne Universit\u00e9, Paris, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-5035-4470","authenticated-orcid":false,"given":"Lin-Pierre","family":"Zhao","sequence":"additional","affiliation":[{"name":"4Service H\u00e9matologie Seniors, H\u00f4pital Saint-Louis, Assistance Publique-H\u00f4pitaux de Paris, Universit\u00e9 Paris Cit\u00e9, Paris, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0009-0003-5352-7949","authenticated-orcid":false,"given":"Vincent","family":"Grobost","sequence":"additional","affiliation":[{"name":"5Service de M\u00e9decine Interne, CHU Estaing, Clermont-Ferrand, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Valentin","family":"Lacombe","sequence":"additional","affiliation":[{"name":"6Service de M\u00e9decine Interne et Polyvalente, Centre Hospitalier du Haut-Anjou, Ch\u00e2teau-Gontier, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-7724-4607","authenticated-orcid":false,"given":"Guillaume","family":"Le Guenno","sequence":"additional","affiliation":[{"name":"5Service de M\u00e9decine Interne, CHU Estaing, Clermont-Ferrand, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-0866-3824","authenticated-orcid":false,"given":"Yann","family":"Nguyen","sequence":"additional","affiliation":[{"name":"7Service de M\u00e9decine Interne, H\u00f4pital Beaujon, Assistance Publique-H\u00f4pitaux de Paris, Universit\u00e9 Paris Cit\u00e9, Clichy, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jean-Beno\u00eet","family":"Arlet","sequence":"additional","affiliation":[{"name":"8Service de M\u00e9decine Interne, H\u00f4pital Europ\u00e9en Georges Pompidou, Assistance Publique-H\u00f4pitaux de Paris, Universit\u00e9 Paris Cit\u00e9, Paris, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-1135-0351","authenticated-orcid":false,"given":"J\u00e9r\u00e9mie","family":"Dion","sequence":"additional","affiliation":[{"name":"9Service de M\u00e9decine Interne et Immunopathologie, Institut Universitaire du Cancer de Toulouse Oncopole, Centre Hospitalier Universitaire de Toulouse, Universit\u00e9 de Toulouse, Toulouse, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-1682-8657","authenticated-orcid":false,"given":"Ma\u00ebl","family":"Heiblig","sequence":"additional","affiliation":[{"name":"10Service d'H\u00e9matologie Clinique, H\u00f4pital Lyon Sud, Hospices Civils de Lyon, Pierre B\u00e9nite, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Alice","family":"Garnier","sequence":"additional","affiliation":[{"name":"11Service d'H\u00e9matologie Clinique, Centre Hospitalier Universitaire de Nantes, Nantes, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Maxime","family":"Samson","sequence":"additional","affiliation":[{"name":"12Service de M\u00e9decine Interne et Immunologie Clinique, Centre de R\u00e9f\u00e9rence Maladies Rares, Maladies Auto-immunes et Auto-inflammatoires syst\u00e9miques Rares de l'Adulte, Centre Hospitalier Universitaire de Dijon Bourgogne, Dijon, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Achille","family":"Aouba","sequence":"additional","affiliation":[{"name":"13Service de M\u00e9decine Interne, Centre Hospitalier Universitaire de Caen, Caen, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-3492-2778","authenticated-orcid":false,"given":"Sylvain","family":"Th\u00e9pot","sequence":"additional","affiliation":[{"name":"14Service des Maladies du Sang, Centre Hospitalier Universitaire de Angers, Angers, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sophie","family":"Dimicoli-Salazar","sequence":"additional","affiliation":[{"name":"15Service d\u2019H\u00e9matologie, H\u00f4pital Haut-L\u00e9v\u00eaque, Centre Hospitalier Universitaire de Bordeaux, Pessac, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Fabien","family":"Dutasta","sequence":"additional","affiliation":[{"name":"16Service de M\u00e9decine Interne, HIA Sainte-Anne, Toulon, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-1533-9264","authenticated-orcid":false,"given":"Beno\u00eet","family":"Faucher","sequence":"additional","affiliation":[{"name":"17Service de M\u00e9decine Interne, H\u00f4pital La Timone, Assistance Publique-H\u00f4pitaux de Marseille, Marseille, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Estibaliz","family":"Lazaro","sequence":"additional","affiliation":[{"name":"18Service de M\u00e9decine Interne, H\u00f4pital Haut-L\u00e9v\u00eaque, Centre Hospitalier Universitaire de Bordeaux, Pessac, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"V\u00e9ronique","family":"Morel","sequence":"additional","affiliation":[{"name":"19Service d'H\u00e9matologie Clinique, H\u00f4pital Piti\u00e9 Salp\u00e9tri\u00e8re, Assistance Publique-H\u00f4pitaux de Paris, Sorbonne Universit\u00e9, Paris, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-7707-5794","authenticated-orcid":false,"given":"Antoine","family":"N\u00e9el","sequence":"additional","affiliation":[{"name":"20Service de M\u00e9decine Interne, Centre Hospitalier Universitaire de Nantes, Nantes, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-6411-884X","authenticated-orcid":false,"given":"Roderau","family":"Outh","sequence":"additional","affiliation":[{"name":"21Service de M\u00e9decine Interne, Centre Hospitalier Perpignan, Perpignan, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-0203-8235","authenticated-orcid":false,"given":"Holy","family":"Bezanahary","sequence":"additional","affiliation":[{"name":"22Service de M\u00e9decine Interne, Centre Hospitalier Universitaire de Limoges, Limoges, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-3052-1424","authenticated-orcid":false,"given":"Julien","family":"Rossignol","sequence":"additional","affiliation":[{"name":"23Service d\u2019H\u00e9matologie, H\u00f4pital Necker, Assistance Publique-H\u00f4pitaux de Paris, Paris, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Anne-Sophie","family":"Alary","sequence":"additional","affiliation":[{"name":"24D\u00e9partement de Biologie du Cancer, Institut Paoli-Calmettes, Marseille, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-0992-8377","authenticated-orcid":false,"given":"Audrey","family":"Bidet","sequence":"additional","affiliation":[{"name":"25Service d\u2019H\u00e9matologie Biologique, H\u00f4pital Haut-L\u00e9v\u00eaque, Centre Hospitalier Universitaire de Bordeaux, Pessac, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Pauline","family":"Blateau","sequence":"additional","affiliation":[{"name":"26Unit\u00e9 de G\u00e9n\u00e9tique Chromosomique, Service de G\u00e9n\u00e9tique Mol\u00e9culaire et Cytog\u00e9nomique, Centre Hospitalier Universitaire de Montpellier, Universit\u00e9 de Montpellier, Montpellier, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Anne","family":"Bouvier","sequence":"additional","affiliation":[{"name":"27Centre Hospitalier Universitaire d'Angers, Laboratoire d'H\u00e9matologie, Angers, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-2903-3135","authenticated-orcid":false,"given":"Guilaine","family":"Boursier","sequence":"additional","affiliation":[{"name":"28Unit\u00e9 des Maladies Autoinflammatoires, Service de G\u00e9n\u00e9tique Mol\u00e9culaire et Cytog\u00e9nomique, Centre de R\u00e9f\u00e9rence des Maladies Auto-Inflammatoires et des Amyloses, Centre Hospitalier Universitaire de Montpellier, Universit\u00e9 de Montpellier, Montpellier, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Matthieu","family":"Decamp","sequence":"additional","affiliation":[{"name":"29Structure F\u00e9d\u00e9rative d'Oncog\u00e9n\u00e9tique Cyto-Mol\u00e9culaire, Centre Hospitalier Universitaire Caen Normandie, Caen, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-4890-336X","authenticated-orcid":false,"given":"Benjamin","family":"Lebecque","sequence":"additional","affiliation":[{"name":"30Service d\u2019H\u00e9matologie Biologique, Centre Hospitalier Universitaire de Estaing, Clermont-Ferrand, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-0095-6999","authenticated-orcid":false,"given":"Yannick","family":"Le Bris","sequence":"additional","affiliation":[{"name":"31Service d\u2019H\u00e9matologie Biologique, Centre Hospitalier Universitaire de Nantes, Nantes, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-1724-4792","authenticated-orcid":false,"given":"Pierre","family":"Sujobert","sequence":"additional","affiliation":[{"name":"32Service d'H\u00e9matologie Biologique, H\u00f4pital Lyon Sud, Hospices Civils de Lyon, Pierre-B\u00e9nite, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Alice","family":"Marceau-Renaut","sequence":"additional","affiliation":[{"name":"33Laboratoire d\u2019H\u00e9matologie, Centre Hospitalier Universitaire de Lille, Lille, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-5675-3154","authenticated-orcid":false,"given":"C\u00e9dric","family":"Pastoret","sequence":"additional","affiliation":[{"name":"34Laboratoire de Biologie, P\u00f4le de Biologie, Centre Hospitalier Universitaire de Rennes, Rennes, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-6894-4329","authenticated-orcid":false,"given":"David","family":"Rizzo","sequence":"additional","affiliation":[{"name":"35Laboratoire d\u2019H\u00e9matologie, Centre Hospitalier Universitaire de Dupuytren, Limoges, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-5263-9987","authenticated-orcid":false,"given":"Nathalie","family":"Boiret-Dupr\u00e9","sequence":"additional","affiliation":[{"name":"30Service d\u2019H\u00e9matologie Biologique, Centre Hospitalier Universitaire de Estaing, Clermont-Ferrand, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Lara","family":"Boucher","sequence":"additional","affiliation":[{"name":"2Service d'H\u00e9matologie Biologique, H\u00f4pital Cochin, Assistance Publique-H\u00f4pitaux de Paris, Paris, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-2728-7243","authenticated-orcid":false,"given":"St\u00e9phanie","family":"Dulucq","sequence":"additional","affiliation":[{"name":"25Service d\u2019H\u00e9matologie Biologique, H\u00f4pital Haut-L\u00e9v\u00eaque, Centre Hospitalier Universitaire de Bordeaux, Pessac, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Franck","family":"Genevieve","sequence":"additional","affiliation":[{"name":"27Centre Hospitalier Universitaire d'Angers, Laboratoire d'H\u00e9matologie, Angers, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0009-0004-2016-7278","authenticated-orcid":false,"given":"Cassandra","family":"Jadeau","sequence":"additional","affiliation":[{"name":"35Laboratoire d\u2019H\u00e9matologie, Centre Hospitalier Universitaire de Dupuytren, Limoges, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Pierre","family":"Lemaire","sequence":"additional","affiliation":[{"name":"36Laboratoire d\u2019H\u00e9matologie, H\u00f4pital Saint-Louis, Assistance Publique-H\u00f4pitaux de Paris, Paris, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Romain","family":"Vazquez","sequence":"additional","affiliation":[{"name":"2Service d'H\u00e9matologie Biologique, H\u00f4pital Cochin, Assistance Publique-H\u00f4pitaux de Paris, Paris, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-0950-979X","authenticated-orcid":false,"given":"Jean-Baptiste","family":"Rieu","sequence":"additional","affiliation":[{"name":"37Laboratoire d'H\u00e9matologie, Institut Universitaire du Cancer de Toulouse Oncopole, Centre Hospitalier Universitaire de Toulouse, Universit\u00e9 de Toulouse, Toulouse, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Olivier","family":"Fain","sequence":"additional","affiliation":[{"name":"1Service de M\u00e9decine Interne, H\u00f4pital Saint-Antoine, Centre de R\u00e9f\u00e9rence des Maladies Auto-Inflammatoires et des Amyloses, Assistance Publique-H\u00f4pitaux de Paris, Sorbonne Universit\u00e9, Paris, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-6668-8854","authenticated-orcid":false,"given":"Sophie","family":"Georgin-Lavialle","sequence":"additional","affiliation":[{"name":"38Service de M\u00e9decine Interne, H\u00f4pital Tenon, Centre de R\u00e9f\u00e9rence des Maladies Auto-Inflammatoires et des Amyloses, Assistance Publique-H\u00f4pitaux de Paris, Sorbonne Universit\u00e9, Paris, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0009-0001-1275-5422","authenticated-orcid":false,"given":"Lucie","family":"Rigolot","sequence":"additional","affiliation":[{"name":"37Laboratoire d'H\u00e9matologie, Institut Universitaire du Cancer de Toulouse Oncopole, Centre Hospitalier Universitaire de Toulouse, Universit\u00e9 de Toulouse, Toulouse, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-9461-0728","authenticated-orcid":false,"given":"Lise","family":"Larcher","sequence":"additional","affiliation":[{"name":"36Laboratoire d\u2019H\u00e9matologie, H\u00f4pital Saint-Louis, Assistance Publique-H\u00f4pitaux de Paris, Paris, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Pierre","family":"Hirsch","sequence":"additional","affiliation":[{"name":"39Service d'H\u00e9matologie Biologique, H\u00f4pital Saint Antoine, Assistance Publique-H\u00f4pitaux de Paris, Sorbonne Universit\u00e9, Paris, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Benjamin","family":"Terrier","sequence":"additional","affiliation":[{"name":"40Service de M\u00e9decine Interne, H\u00f4pital Cochin, Assistance Publique-H\u00f4pitaux de Paris, Paris, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Pierre","family":"Fenaux","sequence":"additional","affiliation":[{"name":"4Service H\u00e9matologie Seniors, H\u00f4pital Saint-Louis, Assistance Publique-H\u00f4pitaux de Paris, Universit\u00e9 Paris Cit\u00e9, Paris, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-2849-3049","authenticated-orcid":false,"given":"Ars\u00e8ne","family":"M\u00e9kinian","sequence":"additional","affiliation":[{"name":"1Service de M\u00e9decine Interne, H\u00f4pital Saint-Antoine, Centre de R\u00e9f\u00e9rence des Maladies Auto-Inflammatoires et des Amyloses, Assistance Publique-H\u00f4pitaux de Paris, Sorbonne Universit\u00e9, Paris, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-6891-9238","authenticated-orcid":false,"given":"Thibault","family":"Comont","sequence":"additional","affiliation":[{"name":"9Service de M\u00e9decine Interne et Immunopathologie, Institut Universitaire du Cancer de Toulouse Oncopole, Centre Hospitalier Universitaire de Toulouse, Universit\u00e9 de Toulouse, Toulouse, France"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"234","reference":[{"issue":"27","key":"2025091811221001700_bib1","doi-asserted-by":"crossref","first-page":"2628","DOI":"10.1056\/NEJMoa2026834","article-title":"Somatic mutations in UBA1 and severe adult-onset autoinflammatory disease","volume":"383","author":"Beck","year":"2020","journal-title":"N Engl J Med"},{"issue":"4","key":"2025091811221001700_bib2","doi-asserted-by":"crossref","first-page":"318","DOI":"10.1001\/jama.2022.24836","article-title":"Estimated prevalence and clinical manifestations of UBA1 variants associated with VEXAS syndrome in a clinical population","volume":"329","author":"Beck","year":"2023","journal-title":"JAMA"},{"issue":"3","key":"2025091811221001700_bib3","doi-asserted-by":"crossref","first-page":"564","DOI":"10.1111\/bjd.20805","article-title":"Further characterization of clinical and laboratory features in VEXAS syndrome: large-scale analysis of a multicentre case series of 116 French patients","volume":"186","author":"Georgin-Lavialle","year":"2022","journal-title":"Br J Dermatol"},{"issue":"3","key":"2025091811221001700_bib4","first-page":"244","article-title":"Spectrum of clonal hematopoiesis in VEXAS syndrome","volume":"142","author":"Gutierrez-Rodrigues","year":"2023","journal-title":"Blood"},{"issue":"10","key":"2025091811221001700_bib5","doi-asserted-by":"crossref","first-page":"1358","DOI":"10.1136\/ard-2024-225640","article-title":"Efficacy and safety of targeted therapies in VEXAS syndrome: retrospective study from the FRENVEX","volume":"83","author":"Hadjadj","year":"2024","journal-title":"Ann Rheum Dis"},{"issue":"9936","key":"2025091811221001700_bib6","doi-asserted-by":"crossref","first-page":"2239","DOI":"10.1016\/S0140-6736(13)61901-7","article-title":"Myelodysplastic syndromes","volume":"383","author":"Ad\u00e8s","year":"2014","journal-title":"The Lancet"},{"issue":"11","key":"2025091811221001700_bib7","doi-asserted-by":"crossref","first-page":"2739","DOI":"10.1038\/s41375-022-01698-8","article-title":"A Phase II prospective trial of azacitidine in steroid-dependent or refractory systemic autoimmune\/inflammatory disorders and VEXAS syndrome associated with MDS and CMML","volume":"36","author":"Mekinian","year":"2022","journal-title":"Leukemia"},{"issue":"4","key":"2025091811221001700_bib8","doi-asserted-by":"crossref","first-page":"969","DOI":"10.1111\/bjh.17893","article-title":"Azacitidine for patients with vacuoles, E1 enzyme, X-linked, autoinflammatory, somatic syndrome (VEXAS) and myelodysplastic syndrome: data from the French VEXAS registry","volume":"196","author":"Comont","year":"2022","journal-title":"Br J Haematol"},{"issue":"8","key":"2025091811221001700_bib9","doi-asserted-by":"crossref","first-page":"e129","DOI":"10.1002\/hem3.129","article-title":"Long-term genetic and clinical remissions after cessation of azacitidine treatment in patients with VEXAS syndrome","volume":"8","author":"Aalbers","year":"2024","journal-title":"Hemasphere"},{"issue":"12","key":"2025091811221001700_bib10","doi-asserted-by":"crossref","first-page":"e661","DOI":"10.1097\/HS9.0000000000000661","article-title":"Azacytidine treatment for VEXAS syndrome","volume":"5","author":"Raaijmakers","year":"2021","journal-title":"Hemasphere"},{"issue":"6","key":"2025091811221001700_bib11","doi-asserted-by":"crossref","first-page":"919","DOI":"10.1007\/s12185-023-03532-y","article-title":"Successful azacitidine therapy for myelodysplastic syndrome associated with VEXAS syndrome","volume":"117","author":"Kataoka","year":"2023","journal-title":"Int J Hematol"},{"issue":"3","key":"2025091811221001700_bib12","doi-asserted-by":"crossref","first-page":"993","DOI":"10.1007\/s00277-023-05611-w","article-title":"VEXAS syndrome: complete molecular remission after hypomethylating therapy","volume":"103","author":"Sockel","year":"2024","journal-title":"Ann Hematol"},{"key":"2025091811221001700_bib13","doi-asserted-by":"crossref","first-page":"1403808","DOI":"10.3389\/fimmu.2024.1403808","article-title":"Case report: VEXAS syndrome: an atypical indolent presentation as sacroiliitis with molecular response to azacitidine","volume":"15","author":"Pereira da Costa","year":"2024","journal-title":"Front Immunol"},{"issue":"12","key":"2025091811221001700_bib14","doi-asserted-by":"crossref","first-page":"5935","DOI":"10.1007\/s00277-024-06072-5","article-title":"Case report: VEXAS syndrome with excellent response to treatment with azacitidine","volume":"103","author":"Porges","year":"2024","journal-title":"Ann Hematol"},{"issue":"5","key":"2025091811221001700_bib15","doi-asserted-by":"crossref","first-page":"e117","DOI":"10.1093\/rheumatology\/keab866","article-title":"Successful treatment with azacitidine in VEXAS syndrome with prominent myofasciitis","volume":"61","author":"Cordts","year":"2022","journal-title":"Rheumatology (Oxford)"},{"issue":"2","key":"2025091811221001700_bib16","doi-asserted-by":"crossref","first-page":"254","DOI":"10.1002\/ajh.27169","article-title":"Diagnostic capabilities, clinical features, and longitudinal UBA1 clonal dynamics of a nationwide VEXAS cohort","volume":"99","author":"Gurnari","year":"2024","journal-title":"Am J Hematol"},{"issue":"26","key":"2025091811221001700_bib17","doi-asserted-by":"crossref","first-page":"3682","DOI":"10.1182\/blood.2020010177","article-title":"Therapeutic options in VEXAS syndrome: insights from a retrospective series","volume":"137","author":"Bourbon","year":"2021","journal-title":"Blood"},{"issue":"7","key":"2025091811221001700_bib18","doi-asserted-by":"crossref","first-page":"1703","DOI":"10.1038\/s41375-022-01613-1","article-title":"The 5th edition of the World Health Organization classification of haematolymphoid tumours: myeloid and histiocytic\/dendritic neoplasms","volume":"36","author":"Khoury","year":"2022","journal-title":"Leukemia"},{"issue":"7","key":"2025091811221001700_bib19","doi-asserted-by":"crossref","first-page":"EVIDoa2200008","DOI":"10.1056\/EVIDoa2200008","article-title":"Molecular international prognostic scoring system for myelodysplastic syndromes","volume":"1","author":"Bernard","year":"2022","journal-title":"NEJM Evid"},{"issue":"12","key":"2025091811221001700_bib20","doi-asserted-by":"crossref","first-page":"2454","DOI":"10.1182\/blood-2012-03-420489","article-title":"Revised international prognostic scoring system for myelodysplastic syndromes","volume":"120","author":"Greenberg","year":"2012","journal-title":"Blood"},{"issue":"10","key":"2025091811221001700_bib21","doi-asserted-by":"crossref","first-page":"1109","DOI":"10.1016\/j.neurol.2022.06.008","article-title":"Azacitidine, a therapeutic option in Lewis and Sumner syndrome associated with VEXAS syndrome","volume":"178","author":"Escoda","year":"2022","journal-title":"Rev Neurol (Paris)"},{"issue":"6","key":"2025091811221001700_bib22","first-page":"1432","article-title":"Very long-term remission with azacitidine in VEXAS syndrome","volume":"110","author":"Zhao","year":"2025","journal-title":"Haematologica"},{"issue":"10","key":"2025091811221001700_bib23","doi-asserted-by":"crossref","first-page":"1020","DOI":"10.1182\/blood-2018-06-857102","article-title":"Proposals for revised IWG 2018 hematological response criteria in patients with MDS included in clinical trials","volume":"133","author":"Platzbecker","year":"2019","journal-title":"Blood"},{"issue":"2","key":"2025091811221001700_bib24","doi-asserted-by":"crossref","first-page":"286","DOI":"10.1111\/bjh.17679","article-title":"Vacuoles in neutrophil precursors in VEXAS syndrome: diagnostic performances and threshold","volume":"195","author":"Lacombe","year":"2021","journal-title":"Br J Haematol"},{"issue":"1","key":"2025091811221001700_bib25","doi-asserted-by":"crossref","first-page":"880","DOI":"10.1186\/s12864-016-3195-z","article-title":"Visualizing tumor evolution with the fishplot package for R","volume":"17","author":"Miller","year":"2016","journal-title":"BMC Genomics"},{"issue":"2","key":"2025091811221001700_bib26","doi-asserted-by":"crossref","first-page":"e828","DOI":"10.1097\/HS9.0000000000000828","article-title":"Quantitative assessment of vacuolization of myeloid precursors in VEXAS syndrome","volume":"7","author":"Rabut","year":"2023","journal-title":"HemaSphere"},{"issue":"2","key":"2025091811221001700_bib27","doi-asserted-by":"crossref","first-page":"826","DOI":"10.1093\/rheumatology\/kead697","article-title":"Treatment experiences with focus on IL-6R inhibition in patients with VEXAS syndrome and a case of remission with azacytidine treatment","volume":"64","author":"Johansen","year":"2025","journal-title":"Rheumatology"},{"issue":"10","key":"2025091811221001700_bib28","doi-asserted-by":"crossref","first-page":"3431","DOI":"10.3324\/haematol.2024.285519","article-title":"De-escalation of corticosteroids and clonal remission in UBA1 mutation-driven VEXAS syndrome with 5-azacytidine","volume":"109","author":"Trikha","year":"2024","journal-title":"Haematologica"},{"issue":"2","key":"2025091811221001700_bib29","doi-asserted-by":"crossref","first-page":"565","DOI":"10.1111\/bjh.19953","article-title":"Hypomethylating agents for patients with VEXAS without myelodysplastic syndrome: Clinical outcome and longitudinal follow-up of vacuolization and UBA1 clonal dynamics","volume":"206","author":"\u00c1lamo","year":"2025","journal-title":"Br J Haematol"},{"issue":"11","key":"2025091811221001700_bib30","doi-asserted-by":"crossref","first-page":"1316","DOI":"10.1182\/blood.2022018602","article-title":"Contribution of mutant HSC clones to immature and mature cells in MDS and CMML, and variations with AZA therapy","volume":"141","author":"Schnegg-Kaufmann","year":"2023","journal-title":"Blood"},{"issue":"3","key":"2025091811221001700_bib31","doi-asserted-by":"crossref","first-page":"372","DOI":"10.1136\/ard-2023-224819","article-title":"Serious infections in patients with VEXAS syndrome: data from the French VEXAS registry","volume":"83","author":"de Valence","year":"2024","journal-title":"Ann Rheum Dis"},{"issue":"12","key":"2025091811221001700_bib32","doi-asserted-by":"crossref","first-page":"4490","DOI":"10.1111\/cas.14653","article-title":"No clear survival benefit of azacitidine for lower-risk myelodysplastic syndromes: a retrospective study of Nagasaki","volume":"111","author":"Toriyama","year":"2020","journal-title":"Cancer Sci"},{"issue":"6","key":"2025091811221001700_bib33","doi-asserted-by":"crossref","first-page":"1485","DOI":"10.1002\/cncr.24894","article-title":"Azacitidine for the treatment of lower risk myelodysplastic syndromes: a retrospective study of 74 patients enrolled in an Italian named patient program","volume":"116","author":"Musto","year":"2010","journal-title":"Cancer"}],"container-title":["Blood"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/ashpublications.org\/blood\/article-pdf\/146\/12\/1450\/2412674\/blood_bld-2024-028133-main.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"syndication"},{"URL":"https:\/\/ashpublications.org\/blood\/article-pdf\/146\/12\/1450\/2412674\/blood_bld-2024-028133-main.pdf","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2025,9,18]],"date-time":"2025-09-18T15:23:46Z","timestamp":1758209026000},"score":1,"resource":{"primary":{"URL":"https:\/\/ashpublications.org\/blood\/article\/146\/12\/1450\/537312\/Efficacy-and-safety-of-azacitidine-for-VEXAS"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2025,9,18]]},"references-count":33,"journal-issue":{"issue":"12","published-print":{"date-parts":[[2025,9,18]]}},"URL":"https:\/\/doi.org\/10.1182\/blood.2024028133","relation":{},"ISSN":["0006-4971","1528-0020"],"issn-type":[{"value":"0006-4971","type":"print"},{"value":"1528-0020","type":"electronic"}],"subject":[],"published-other":{"date-parts":[[2025,9,18]]},"published":{"date-parts":[[2025,9,18]]}}}