Modern Rheumatology最新文献

{"title":"Epidemiological and Clinical Insights into Behçet's Disease in Japan: A 2019 Nationwide Analysis.","authors":"Yuki Mizuki, Nobuyuki Horita, Mitsuhiro Takeno, Akira Meguro, Yohei Kirino, Tatsukata Kawagoe, Takuto Sakono, Mami Ishihara, Shigeaki Ohno, Nobuhisa Mizuki","doi":"10.1093/mr/roaf103","DOIUrl":"https://doi.org/10.1093/mr/roaf103","url":null,"abstract":"<p><strong>Background: </strong>Behçet's disease (BD) is a systemic inflammatory disorder with regional variations in its clinical manifestations. Although its characteristics have evolved over time, comprehensive data on the current epidemiology, treatment trends, and impact on daily life in Japan remain limited.</p><p><strong>Method: </strong>This study analysed the 2019 data from the National Database of Designated Intractable Diseases of Japan, provided by the Japanese Ministry of Health, Labour and Welfare (MHLW). We focused on age distribution, symptom frequency, daily life impact, and treatment trends.</p><p><strong>Results: </strong>In the 2019 database, 10 406 patients with BD were registered. The median age was 55 years, with a higher proportion of women affected (53.2%). Serious manifestations such as ocular, vascular, and CNS involvement were more frequent in males, whereas females showed more mucocutaneous and articular symptoms, representing mild forms of the disease. Pharmacological treatment was common (93.8%), with corticosteroids (44.3%), colchicine (50.9%), infliximab (17.0%), and adalimumab (9.6%) being frequently used. BD significantly affected daily life, with nearly one-third experiencing impaired mobility or depression.</p><p><strong>Conclusion: </strong>This nationwide analysis highlighted sex-based differences in clinical features and found that BD continues to substantially affect quality of life. The widespread use of biologics suggests a shift in therapeutic strategies.</p>","PeriodicalId":18705,"journal":{"name":"Modern Rheumatology","volume":" ","pages":""},"PeriodicalIF":1.9,"publicationDate":"2025-10-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145345939","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Effectiveness and safety of tocilizumab, an anti-interleukin-6 receptor antibody, in 342 Japanese patients with multicentric Castleman disease: a 3-year, prospective, observational study.","authors":"Norihiro Nishimoto, Miho Murakami, Takeshi Johkoh, Seiji Hayashi, Shiro Ohshima, Masao Mizuki, Takahiro Kakehi, Yuichi Kawata, Shinichi Matsuda, Misato Hashizume","doi":"10.1093/mr/roaf100","DOIUrl":"https://doi.org/10.1093/mr/roaf100","url":null,"abstract":"<p><strong>Objectives: </strong>Multicentric Castleman disease (MCD) is a rare lymphoproliferative disorder characterized by multifocal lymphadenopathy and systemic inflammatory symptoms. This single-arm, prospective, observational, postmarketing surveillance (UMIN000023071) assessed the real-world effectiveness and safety of tocilizumab in Japanese patients with MCD.</p><p><strong>Methods: </strong>Patients with MCD received tocilizumab intravenous infusion 8 mg/kg every 2 weeks. Effectiveness outcomes included enlarged lymph node regression; improvement in laboratory findings, associated symptoms, and organ involvement; and change in corticosteroid doses. Safety was assessed through adverse events (AEs).</p><p><strong>Results: </strong>A total of 342 patients were included. Tocilizumab was continued for > 152 weeks in 70.1% of patients. After treatment, 58.9% of patients showed lymph node regression. Laboratory test values were improved, and most evaluable patients reported improvements in associated symptoms, including night sweats, anorexia, fever, and fatigue. Organ involvement, including pulmonary disease, rash, and hepatosplenomegaly, was 'improved' or 'stable' in more than half of evaluable patients. Tocilizumab treatment resulted in corticosteroid dose reduction. The most commonly reported AEs were infections and infestations; respiratory, thoracic, and mediastinal disorders; and gastrointestinal disorders.</p><p><strong>Conclusions: </strong>The effectiveness and safety of tocilizumab in Japanese patients with MCD was confirmed for up to 3 years, without new safety concerns.</p>","PeriodicalId":18705,"journal":{"name":"Modern Rheumatology","volume":" ","pages":""},"PeriodicalIF":1.9,"publicationDate":"2025-10-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145337281","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"The Significance of Participation in Peer Support Groups for People with Rheumatoid Arthritis: A Qualitative Study.","authors":"Izumi Kobayashi, Hideko Nakahara, Mie Fusama","doi":"10.1093/mr/roaf101","DOIUrl":"https://doi.org/10.1093/mr/roaf101","url":null,"abstract":"<p><strong>Objectives: </strong>This study examines how rheumatoid arthritis (RA) peer support groups affect patients' lives and identifies the roles of these groups.</p><p><strong>Methods: </strong>Twelve RA patients from a peer support group participated in a focus group interview discussing reasons for joining, emotional changes, and disease management. Data were analyzed using qualitative methods.</p><p><strong>Results: </strong>The analysis identified 65 codes, organized into 22 subcategories and synthesized into five pre- and five post-membership experience categories. The five pre-membership categories were: prolonged time for diagnosis, persistent anxiety even after starting treatment, expectation of interacting with peers with the same disease, encounter with the peer support group, and decision to join due to worsening condition. The five post-membership categories were meeting peers with whom they could empathize, need to understand their own illnesses, diverse information from other members' experiences, active participation in treatment and a place to belong and share with understanding peers. The group was considered to be a supportive community where patients can obtain necessary information, peer support, and increased treatment motivation.</p><p><strong>Conclusions: </strong>This study indicates that peer support group could be valuable social resources, helping patients manage illness and improve quality of life.</p>","PeriodicalId":18705,"journal":{"name":"Modern Rheumatology","volume":" ","pages":""},"PeriodicalIF":1.9,"publicationDate":"2025-10-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145308533","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Effect of lower limb exercise therapy on the patients with rheumatoid arthritis: an analysis based on severity of joint damage.","authors":"Rie Kurose, Yuji Wakai","doi":"10.1093/mr/roaf102","DOIUrl":"https://doi.org/10.1093/mr/roaf102","url":null,"abstract":"<p><strong>Objective: </strong>Exercise therapy is widely recommended for patients with rheumatoid arthritis (RA), but the ideal parameters and appropriate patients selection remain controversial. This study aimed to assess the effectiveness and safety of lower limb exercise therapy in RA-affected knees, stratified by radiographic severity of joint destruction.</p><p><strong>Methods: </strong>We retrospectively analyzed RA patients who participated in a three-month lower limb exercise therapy. Participants were divided into two groups according to the Larsen grading classification: mild destruction (LG II-III) and advanced destruction (LG IV-V). Functional outcomes and disease activity parameters were compared between two groups of LG II-III and IV-V.</p><p><strong>Results: </strong>Patients in the LG II-III group showed improvements in lower limb functions, including enhanced strength of the gluteus muscles, with no worsening of RA disease activity. Conversely, some patients in the LG IV-V group experienced declines in the 10-meter walk time, timed up and go test (TUG), patient visual analogue scale (VAS), and stair climbing ability. Some patients in the LG IV-V group had intensified anti-rheumatic drug therapy.</p><p><strong>Conclusion: </strong>Lower limb exercise therapy appears safe and effective for RA knees with mild destruction but may risk functional decline in patients with advanced damage. Radiographic evaluation is essential for tailoring therapy to individual joint status.</p>","PeriodicalId":18705,"journal":{"name":"Modern Rheumatology","volume":" ","pages":""},"PeriodicalIF":1.9,"publicationDate":"2025-10-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145308338","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Real-world safety and effectiveness of mepolizumab for patients with eosinophilic granulomatosis with polyangiitis in Japan: a subgroup analysis of the MARS study.","authors":"Tomonori Ishii, Hideaki Kunishige, Mitsuhiro Yoshida, Etsuko Hayashi, Masaki Komatsubara, Rafael Alfonso-Cristancho, Peter Howarth","doi":"10.1093/mr/roaf096","DOIUrl":"https://doi.org/10.1093/mr/roaf096","url":null,"abstract":"<p><p>ObjectivesEvaluate the long-term safety and effectiveness of mepolizumab in subgroups of patients with eosinophilic granulomatosis with polyangiitis, including: disease duration, oral corticosteroid use, relapse, worsening or new-onset symptoms, anti-neutrophil cytoplasm antibodies status, and immunosuppressive therapy use. MethodsData from the 96-week MARS study in Japan were analysed for patients receiving mepolizumab for ≥96 weeks, focusing on adverse events, clinical symptoms, average daily oral corticosteroid dose, and relapse. ResultsOral corticosteroid exposure decreased in most subgroups, particularly in the anti-neutrophil cytoplasm antibodies negative subgroup. Patients on higher average oral corticosteroid doses (>7.5 mg/day versus ≤ 7.5 mg/day, during the observation period) experienced more adverse events, including infection-related adverse events and clinical symptoms, and higher relapse rates. No relapses were observed in patients on an average dose of 0.0 mg/day oral corticosteroids. Reduced oral corticosteroid use was associated with improved clinical outcomes, particularly in patients who remained relapse-free. ConclusionMepolizumab was effective regardless of eosinophilic granulomatosis with polyangiitis duration, anti-neutrophil cytoplasm antibodies status, or immunosuppressant use. Higher oral corticosteroid doses were linked to more adverse events, clinical symptoms, and relapses compared to lower doses. In relapse-free patients, continued mepolizumab use led to fewer adverse events, lower oral corticosteroid doses, and improved symptoms.</p>","PeriodicalId":18705,"journal":{"name":"Modern Rheumatology","volume":" ","pages":""},"PeriodicalIF":1.9,"publicationDate":"2025-10-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145301936","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Comparative Effectiveness and Safety of Peficitinib and Abatacept for Rheumatoid Arthritis: A Multicenter, Inverse Probability Weighting Analysis.","authors":"Kento Ichikawa, Yosuke Kunishita, Satoru Shinoda, Kayo Harita, Chikara Honda, Naoki Suzuki, Kana Higashitani, Yuki Iizuka, Yuji Uzawa, Eriko Ohno, Chiharu Hidekawa, Takaaki Komiya, Natsuki Sakurai, Yuichiro Sato, Masaki Mitsuhashi, Naoki Hamada, Yuji Yoshioka, Naomi Tsuchida, Ayaka Maeda, Lisa Hirahara, Yutaro Soejima, Daiga Kishimoto, Hiroto Nakano, Kaoru Takase-Minegishi, Ryusuke Yoshimi, Yohei Kirino, Tadanobu Okubo, Shigeru Ohno, Shouhei Nagaoka, Hideaki Nakajima","doi":"10.1093/mr/roaf094","DOIUrl":"https://doi.org/10.1093/mr/roaf094","url":null,"abstract":"<p><strong>Objectives: </strong>To examine the real-world effectiveness and safety of peficitinib (PEF) and abatacept (ABT) in patients with rheumatoid arthritis (RA).</p><p><strong>Methods: </strong>In this multicenter, retrospective cohort study, we examined patients who were administered PEF or ABT between July 2019 and July 2022. The primary endpoint was the one-year retention rate of PEF or ABT. The primary analyses were performed using treatment persistence, disease activity measurements, and safety parameters. The inverse probability of treatment weighting (IPTW) was used to adjust for confounding variables.</p><p><strong>Results: </strong>A total of 219 patients were enrolled, with 64 receiving PEF and 155 receiving ABT. The one-year retention rates were 42.8% in the PEF group and 61.0% in the ABT group after IPTW (p = 0.0083). Two years following the initiation of treatment, the mean CDAI and SDAI showed comparable improvement in both groups after IPTW. Treatment discontinuation occurred primarily because of an inadequate therapeutic response (PEF: 41.3%, ABT: 28.1%) and adverse reactions (PEF: 11.1%, ABT: 3.8%).</p><p><strong>Conclusions: </strong>PEF treatment resulted in lower retention rates compared with ABT, whereas both medications demonstrated effectiveness in controlling disease activity for patients who were able to continue treatment. Our findings contribute to informed decision-making in RA treatment in actual clinical practice.</p>","PeriodicalId":18705,"journal":{"name":"Modern Rheumatology","volume":" ","pages":""},"PeriodicalIF":1.9,"publicationDate":"2025-10-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145275318","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Real-world effectiveness of rituximab or intravenous cyclophosphamide versus non-use in patients with severe microscopic polyangiitis and granulomatosis with polyangiitis: a retrospective cohort study of J-CANVAS.","authors":"Satoshi Omura, Takashi Kida, Junya Kitai, Takuya Yanagida, Daiki Nakagomi, Yoshiyuki Abe, Makoto Wada, Naoho Takizawa, Atsushi Nomura, Yuji Kukida, Naoya Kondo, Hirosuke Takagi, Koji Endo, Shintaro Hirata, Naoto Azuma, Tohru Takeuchi, Shoichi Fukui, Kazuro Kamada, Ryo Yanai, Yusuke Matsuo, Yasuhiro Shimojima, Ryo Nishioka, Ryota Okazaki, Tomoaki Takata, Mayuko Moriyama, Ayuko Takatani, Yoshia Miyawaki, Tsuyoshi Shirai, Hiroaki Dobashi, Takafumi Ito, Isao Matsumoto, Toshihiko Takada, Toshiko Ito-Ihara, Nobuyuki Yajima, Takashi Kawaguchi, Takahiro Seno, Yutaka Kawahito","doi":"10.1093/mr/roaf090","DOIUrl":"https://doi.org/10.1093/mr/roaf090","url":null,"abstract":"<p><strong>Objectives: </strong>To evaluate the real-world effectiveness of rituximab (RTX) and intravenous cyclophosphamide (IVCY) compared to non-use for remission induction in microscopic polyangiitis (MPA) and granulomatosis with polyangiitis (GPA).</p><p><strong>Methods: </strong>This observational study emulated a target trial using data from the Japan Collaborative Registry of ANCA-Associated Vasculitis (J-CANVAS). Patients aged ≥20 years with newly diagnosed or relapsing MPA or GPA (2017-2023) were included. RTX or IVCY use within 4 weeks defined the treatment group; others formed the control group. The primary outcome was failure to achieve remission at week 24 (BVAS = 0 and prednisolone ≤ 10 mg/day). Secondary outcomes included a composite of death, kidney failure, and relapse, and serious infection. In inverse probability weighted population, risk ratios were estimated using modified Poisson regression.</p><p><strong>Results: </strong>Among 544 patients (MPA: 413, GPA: 131), 63.6% received RTX or IVCY. The risk ratio for failure to achieve remission was 0.72 (95% CI: 0.61-0.85), and for the composite outcome was 0.57 (95% CI: 0.33-0.97), and for serious infection was 1.03 (95% CI, 0.47-2.25). Results were robust in sensitivity analyses.</p><p><strong>Conclusions: </strong>RTX and IVCY improved short-term outcomes in MPA and GPA without increasing infection risk, supporting their recommendation as standard therapy.</p>","PeriodicalId":18705,"journal":{"name":"Modern Rheumatology","volume":" ","pages":""},"PeriodicalIF":1.9,"publicationDate":"2025-10-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145275390","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Development and validation of case-finding algorithms for identifying patients with systemic lupus erythematosus in an administrative claim database from tertiary care centers in Japan.","authors":"Ken-Ei Sada, Yoshia Miyawaki, Ryo Yanai, Takashi Kida, Akira Ohnishi, Ryusuke Yoshimi, Kunihiro Ichinose, Yasuhiro Shimojima","doi":"10.1093/mr/roaf091","DOIUrl":"https://doi.org/10.1093/mr/roaf091","url":null,"abstract":"<p><strong>Objective: </strong>To develop and validate algorithms for identifying patients with systemic lupus erythematosus (SLE) in Japanese administrative claims databases from tertiary care centers using statistical and machine learning methods.</p><p><strong>Methods: </strong>This retrospective cross-sectional study included 13,538 patients from six hospitals. One-year claims data were linked to chart-confirmed SLE diagnoses. Patients were randomly assigned to training (n = 8,811) and test (n = 3,775) sets; an external validation set (n = 952) was drawn from another hospital. Feature selection used Least Absolute Shrinkage and Selection Operator (LASSO), Boruta, and Recursive Feature Elimination. Logistic regression, random forest, and decision tree models were trained with synthetic oversampling to address class imbalance. Model performance was evaluated using the Area Under the Receiver Operating Characteristic Curve (AUROC), and other standard performance metrics.</p><p><strong>Results: </strong>The random forest model achieved the best performance (AUROC: 0.995; sensitivity: 0.971; specificity: 0.969). A simplified rule based on diagnosis code and anti-double-stranded DNA antibody testing showed high accuracy in both test and validation sets. Adding urine sediment examination modestly improved sensitivity but reduced specificity.</p><p><strong>Conclusion: </strong>A claims-based algorithm incorporating diagnosis codes and standard laboratory tests accurately identified patients with SLE facilitating reliable use of administrative data in real-world research.</p>","PeriodicalId":18705,"journal":{"name":"Modern Rheumatology","volume":" ","pages":""},"PeriodicalIF":1.9,"publicationDate":"2025-10-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145275369","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Presence of Myositis-Specific Autoantibodies May Predict Favorable Outcomes to Rituximab in Patients with Idiopathic Inflammatory Myositis: Retrospective Observational Study.","authors":"Emin Oğuz, Nevzat Koca, Murat Bektas","doi":"10.1093/mr/roaf097","DOIUrl":"https://doi.org/10.1093/mr/roaf097","url":null,"abstract":"<p><strong>Objective: </strong>This study aimed to evaluate the autoantibody profile, including myositis-associated autoantibodies (MAAs) and myositis-specific autoantibodies (MSAs), and their influence on outcomes of rituximab (RTX) treatment in patients with IIM.</p><p><strong>Methods: </strong>This retrospective observational study included data from patients with idiopathic inflammatory myositis (IIM) who met at least one of the following criteria: Bohan and Peter, ACR/EULAR 2017, and ENMC criteria.</p><p><strong>Results: </strong>Fifty-three patients, 72% of whom were female, were included. Among them, 32.1% had a clinical diagnosis of dermatomyositis, 35.8% antisynthetase syndrome (ASS), 15.3% overlap syndrome, 11.3% polymyositis, and 5.7% had immune-mediated necrotizing myopathy. RTX was used in 49% of the patients, and nearly half of them (46%) had ASS. Remission was achieved in 77%, a partial response was observed in 11.5%, and unresponsiveness/active disease was observed in 11.5% of patients receiving RTX. Relapse was lower, and mortality tended to be lower in patients receiving RTX. In multivariate analysis, MSA positivity was associated with remission in those receiving RTX.</p><p><strong>Conclusion: </strong>In our study, RTX was safe and effective in patients with IIM who had an inadequate response or severe disease at admission. Furthermore, the presence of MSAs may predict a favorable response to RTX treatment in patients with IIM.</p>","PeriodicalId":18705,"journal":{"name":"Modern Rheumatology","volume":" ","pages":""},"PeriodicalIF":1.9,"publicationDate":"2025-10-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145251849","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"High Glucocorticoid Dependency and Limited Therapeutic Response in Japanese Patients with VEXAS Syndrome: A Multicentre Retrospective Study.","authors":"Ayaka Maeda, Yohei Kirino, Naomi Tsuchida, Yuri Uchiyama, Daisuke Kobayashi, Tomoyuki Asano, Yuji Ito, Kosuke Katsuo, Ayaka Kato, Ichiro Mori, Hiroyuki Yamaguchi, Toshimasa Shimizu, Satoshi Kobayashi, Mitsumasa Kishimoto, Nobuhiro Oda, Ryo Rokutanda, Ken Nagahata, Hiroshi Fujiwara, Hiroshi Kobayashi, Yoichi Takeuchi, Hiroyuki Hagiyama, Naoki Umeda, Kaori Uchino, Yuji Miyoshi, Yukiko Hidaka, Hiroaki Ida, Keiichi Akasaka, Kaori Kashino, Yusuke Yoshida, Hidenori Ohnishi, Noriyuki Takahashi, Yuichiro Fujieda, Nobuyuki Horita, Kaoru Takase-Minegishi, Naomichi Matsumoto, Hideaki Nakajima","doi":"10.1093/mr/roaf095","DOIUrl":"https://doi.org/10.1093/mr/roaf095","url":null,"abstract":"<p><strong>Objectives: </strong>VEXAS (vacuoles, E1 enzyme, X-linked, autoinflammatory, somatic) syndrome is an autoinflammatory disease caused by somatic variants of the UBA1 gene. Due to uncertainty around treatment strategies and limited therapeutic options, this study aimed to characterize the clinical treatment and outcomes of VEXAS syndrome patients in Japan.</p><p><strong>Methods: </strong>We retrospectively analysed data on clinical manifestations, treatments, and outcomes from 46 male patients diagnosed with VEXAS syndrome. Twelve patients at our institution were evaluated for remission using the French VEXAS group criteria.</p><p><strong>Results: </strong>All patients (median age at onset: 71.4 years) received systemic glucocorticoid therapy (mean maximum dose: 47.4 mg/day; mean minimum dose: 8.5 mg/day). Most patients required continuous glucocorticoid treatment of ≥10 mg/day, and tapering was generally difficult. Among the patients followed at our institution, only 42% (5/12) achieved complete remission at least once during follow-up. Tocilizumab was the most frequently administered immunosuppressant (n = 24, 52.2%); Janus kinase inhibitors and azacitidine, reported to be effective overseas, were prescribed in only three and two cases, respectively.</p><p><strong>Conclusions: </strong>This study highlights the difficulty of treating patients with VEXAS syndrome in Japan. The development of more effective treatments is urgently needed to reduce glucocorticoid dependence and improve patient outcomes.</p>","PeriodicalId":18705,"journal":{"name":"Modern Rheumatology","volume":" ","pages":""},"PeriodicalIF":1.9,"publicationDate":"2025-10-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145251692","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}