Sharfaraz Salam, Jasper M Morrow, Sachit Shah, Michael G Hanna, Mazen M Dimachkie, Pedro M Machado
{"title":"Imaging evaluation of the upper limbs in inclusion body myositis: an unmet need.","authors":"Sharfaraz Salam, Jasper M Morrow, Sachit Shah, Michael G Hanna, Mazen M Dimachkie, Pedro M Machado","doi":"10.55563/clinexprheumatol/q7ozm2","DOIUrl":"10.55563/clinexprheumatol/q7ozm2","url":null,"abstract":"<p><p>Inclusion body myositis (IBM) is an acquired myopathy belonging to the spectrum of idiopathic inflammatory myopathies. It commonly presents in individuals aged above 50 years of age. Characteristic clinical features of IBM include weakness of the quadriceps and finger flexors. There are currently no effective drug treatments for IBM. However as more clinical drug trials are being conducted it is important that more precise outcome measures are developed to track disease progression and assess treatment effects. Imaging techniques such as magnetic resonance imaging (MRI) and ultrasound have been increasingly used to study intramuscular changes within the thigh and calf muscles. In particular quantitative MRI assessments of the lower limb have started to be employed as endpoints for clinical trials in IBM patients. However, in comparison to the lower limb, there is a relative lack in robust imaging biomarkers for the upper limb muscles. It is prudent that this paucity is addressed as the majority of IBM patients have forearm involvement and, in many individuals, upper limb weakness is their main source of disability. Imaging focussed studies thus far indicate preferential flexor digitorium profundus involvement. In this review, we discuss the imaging modalities that have been used to evaluate intramuscular changes and the possible techniques which could be developed further as upper limb biomarkers for IBM.</p>","PeriodicalId":10274,"journal":{"name":"Clinical and experimental rheumatology","volume":" ","pages":"372-378"},"PeriodicalIF":3.4,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143457050","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}
Weijin Zhang, Guohai Huang, Shaoyu Zheng, Jianqun Lin, Shijian Hu, Jinghua Zhuang, Zexuan Zhou, Guangzhou Du, Kedi Zheng, Shaoqi Chen, Qichuan Zhang, Angelina Mikish, Anna-Maria Hoffmann-Vold, Masataka Kuwana, Marco Matucci-Cerinic, Daniel E Furst, Yukai Wang
{"title":"Risk prediction modelling in idiopathic inflammatory myositis-associated interstitial lung disease based on seven factors including serum KL-6 and lung ultrasound B-lines.","authors":"Weijin Zhang, Guohai Huang, Shaoyu Zheng, Jianqun Lin, Shijian Hu, Jinghua Zhuang, Zexuan Zhou, Guangzhou Du, Kedi Zheng, Shaoqi Chen, Qichuan Zhang, Angelina Mikish, Anna-Maria Hoffmann-Vold, Masataka Kuwana, Marco Matucci-Cerinic, Daniel E Furst, Yukai Wang","doi":"10.55563/clinexprheumatol/ylf0oe","DOIUrl":"10.55563/clinexprheumatol/ylf0oe","url":null,"abstract":"<p><strong>Objectives: </strong>To develop a user-friendly nomogram-based predictive model for interstitial lung disease (ILD) in patients with idiopathic inflammatory myositis (IIM).</p><p><strong>Methods: </strong>A retrospective study was conducted at Shantou Central Hospital, encompassing 205 IIM patients diagnosed between January 2013 and December 2022. We used the LASSO regression method in the discovery set to select features for model construction, followed by efficacy verification through AUC of ROC. Afterwards, KL-6 values and LUS B-lines number were added into this model to evaluate whether these 2 factors added to the model efficiency. Finally, a web version was constructed to make it more available.</p><p><strong>Results: </strong>Among the 205 IIM patients, 115 (56.1%) patients were diagnosed with ILD, and 90 (43.9%) did not. The predictive model, derived from the training set, comprised four independent risk factors, including age, presence of respiratory symptoms, anti-melanoma differentiation-associated gene 5 (MDA-5) antibody positivity, and anti-aminoacyl transfer RNA synthetase (anti-ARS) antibodies positivity. Notably, anti-TIF1-γ antibody positivity emerged as a protective factor. The AUC of the ROC based on these 5 factors was 0.876 in the training set and 0.861 in the validation set. The AUC of the ROC based on the 5 factors plus KL-6 was 0.922, 5 factors plus B-line number was 0.949 and 5 factors plus both KL-6 and B-line number was 0.951. Accordingly, a nomogram and a web version were developed.</p><p><strong>Conclusions: </strong>This predictive model demonstrates robust capability to assess ILD risk in IIM patients, particularly when augmented with serum KL-6 level or/and LUS B-line number.</p>","PeriodicalId":10274,"journal":{"name":"Clinical and experimental rheumatology","volume":" ","pages":"260-268"},"PeriodicalIF":3.4,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142364613","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}
Aleksandra H Opinc-Rosiak, Ewelina M Kulesza, Agnieszka J Mikosińska, Olga E Brzezińska, Joanna S Makowska
{"title":"Infections as a predominant cause of death in adult patients with idiopathic inflammatory myopathies.","authors":"Aleksandra H Opinc-Rosiak, Ewelina M Kulesza, Agnieszka J Mikosińska, Olga E Brzezińska, Joanna S Makowska","doi":"10.55563/clinexprheumatol/kcdmct","DOIUrl":"https://doi.org/10.55563/clinexprheumatol/kcdmct","url":null,"abstract":"<p><strong>Objectives: </strong>To evaluate causes of death in the single-centre Polish cohort of patients with idiopathic inflammatory myopathies (IIM) and to identify risk factors associated with fatal outcomes.</p><p><strong>Methods: </strong>Electronic medical database was retrospectively analysed, data on the clinical symptoms, serological profiles, administered treatment and outcomes in IIM patients were collected. Two subgroups were distinguished - the deceased and the survived group. Statistical analysis was performed to identify differences between the subgroups and risk factors contributing to fatal outcomes.</p><p><strong>Results: </strong>79 patients with IIM were identified, most frequently with antisynthetase syndrome, dermatomyositis and polymyositis. Among them 9 patients (11.39%) deceased. The mean age at the time of diagnosis was 57.10 ± 14,59years old. Median diagnostic delay reached 5 months. The majority of the deaths (77.78%) occurred within the first 18 months after IIM diagnosis. As compared to the survivors, patients with fatal outcomes were more frequently affected by cardiac involvement (p=0.026), suffered from concomitant autoimmune disorders (p=0.028) were treated with mycophenolate mofetil (p=0.005) and cyclophosphamide (p=0.006). 89.89% of all deaths in our cohort were caused by various infections, predominantly of the respiratory tract. Although 10.13% of the patients had a history of malignancy, none of the deaths was caused by malignancy.</p><p><strong>Conclusions: </strong>Patients with IIM associated with cardiac involvement and concomitant autoimmune diseases may be at higher risk of fatal outcomes. Infections were the predominant cause of death in our cohort of patients. The majority of fatal outcomes occurred at the relatively early stage of the disease.</p>","PeriodicalId":10274,"journal":{"name":"Clinical and experimental rheumatology","volume":"43 2","pages":"290-299"},"PeriodicalIF":3.4,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143522665","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}
Kristofer M Andreasson, Fabricio Espinosa-Ortega, Heléne Sandlund, Helene Alexanderson
{"title":"Self-reported physical activity and fatigue and its associations to anxiety and depression in adult patients with idiopathic inflammatory myopathies:a MIHRA psychological impact and MIHRA exercise and rehabilitation scientific working groups collaboration.","authors":"Kristofer M Andreasson, Fabricio Espinosa-Ortega, Heléne Sandlund, Helene Alexanderson","doi":"10.55563/clinexprheumatol/5u5ah3","DOIUrl":"10.55563/clinexprheumatol/5u5ah3","url":null,"abstract":"<p><strong>Objectives: </strong>To evaluate self-reported physical activity (PA) levels as well as the relationship between PA, anxiety, depression, quality of life, pain, fatigue, disease activity, and organ damage in patients with idiopathic inflammatory myopathies (IIM).</p><p><strong>Methods: </strong>All adult patients registered at the rheumatology clinic at Karolinska University Hospital in Stockholm, Sweden (2019-2022) were eligible to participate. Questionnaires measuring anxiety, depression (HADS), and PA (IPAQ) were provided during yearly check-up or by mail, due to reduced in-person visits amid the Covid-19 pandemic. Additional data was obtained from the Swedish Rheumatology Quality Registry.</p><p><strong>Results: </strong>Of 488 invited patients, 336 agreed to participate, and 246 completed the questionnaires. Median (range) age was 64 (20-88) years, median disease duration five (0.3-61) years and two-thirds were women. Notably, 82% reported moderate/high level of PA. Probable anxiety and depression were experienced by 25% and 14%, respectively. The findings indicated a potential protective effect of PA against depression (OR 0.23, CI 0.06-0.95). Conversely, patients who were physically inactive had poorer mental health, reduced muscle function, increased organ damage, and fatigue. Fatigue demonstrated a progressive link to heightened risks of anxiety (2%, 2-4%) or depression (3%, 1-6%).</p><p><strong>Conclusions: </strong>Most patients with IIM reported being physically active. The study highlights the potential influence of PA on mental health and its role in mitigating risks associated with depression and fatigue among IIM-patients. It also underscores the importance of patient-reported outcomes, and their role in understanding and improving healthcare interventions. Further research is needed to uncover causes and confirm these associations.</p>","PeriodicalId":10274,"journal":{"name":"Clinical and experimental rheumatology","volume":" ","pages":"230-240"},"PeriodicalIF":3.4,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142281256","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}
Marlise S M S Faria, Akira Yoshida, Naoki Mugii, Pleiades T Inaoka, Takashi Matsushita, Takahisa Gono, Masataka Kuwana, Samuel K Shinjo
{"title":"Different phenotypic manifestations between Brazilian and Japanese anti-MDA5 antibody-positive dermatomyositis: an international tricentric longitudinal study.","authors":"Marlise S M S Faria, Akira Yoshida, Naoki Mugii, Pleiades T Inaoka, Takashi Matsushita, Takahisa Gono, Masataka Kuwana, Samuel K Shinjo","doi":"10.55563/clinexprheumatol/9s7djz","DOIUrl":"10.55563/clinexprheumatol/9s7djz","url":null,"abstract":"<p><strong>Objectives: </strong>Anti-MDA5 autoantibodies are strongly associated with interstitial lung disease (ILD) and rapidly progressive ILD (RP-ILD) in Asian patients with dermatomyositis (DM) or amyopathic DM (ADM). However, this association has not yet been established in Brazilian patients with anti-MDA5(+) DM/ADM. This study aimed to investigate the phenotypic differences between Brazilian and Japanese patients with anti-MDA5(+) DM/ADM, with a particular focus on ILD.</p><p><strong>Methods: </strong>This was an international, tricentric, retrospective cohort study conducted in one Brazilian and two Japanese tertiary centres. Patients diagnosed with anti-MDA5(+) DM/ADM at the three centres were enrolled. Clinical characteristics and outcomes were collected using a pre-standardised protocol and compared between Brazilian and Japanese patients.</p><p><strong>Results: </strong>Thirty-four Brazilian and 65 Japanese patients were analysed. Brazilian patients were younger at the time of diagnosis than Japanese patients. The prevalence of muscle weakness, myalgia, dysphagia, heliotrope rash, V-sign, calcinosis, Raynaud's phenomenon, and digital ulcers was higher in Brazilian patients, whereas mechanic's hands were more prevalent in Japanese patients. The prevalence of ILD was significantly lower in Brazilian patients than in Japanese patients (50.0% vs. 98.5%, p<0.001). RP-ILD was observed in 34 (52.3%) Japanese patients and in only one (3.3%) Brazilian patient (p<0.001). Outcomes including overall survival and the frequency of relapses and complications, such as severe infection and malignancy, were comparable between the two populations.</p><p><strong>Conclusions: </strong>Brazilian patients with anti-MDA5(+) DM/ADM had a higher prevalence of skin and muscle involvement, whereas the prevalence of ILD and RP-ILD was significantly lower than in Japanese patients.</p>","PeriodicalId":10274,"journal":{"name":"Clinical and experimental rheumatology","volume":" ","pages":"193-201"},"PeriodicalIF":3.4,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143188463","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}
Sharfaraz Salam, Jasper M Morrow, Michael P McDermott, Nicholas Zafeiropoulos, John S Thornton, Sachit Shah, Stephen Wastling, Tarek Y Yousry, Richard J Barohn, Michael G Hanna, Mazen M Dimachkie, Pedro M Machado
{"title":"Quantitative muscle magnetic resonance imaging as a biomarker for inclusion body myositis in clinical trials: exploring the in vivo effects of arimoclomol.","authors":"Sharfaraz Salam, Jasper M Morrow, Michael P McDermott, Nicholas Zafeiropoulos, John S Thornton, Sachit Shah, Stephen Wastling, Tarek Y Yousry, Richard J Barohn, Michael G Hanna, Mazen M Dimachkie, Pedro M Machado","doi":"10.55563/clinexprheumatol/5b9lme","DOIUrl":"https://doi.org/10.55563/clinexprheumatol/5b9lme","url":null,"abstract":"<p><strong>Objectives: </strong>To investigate the intramuscular effects of arimoclomol using quantitative magnetic resonance imaging (qMRI) of the thighs in a subset of inclusion body myositis (IBM) participants from a multicentre, randomised, double-blind, placebo-controlled trial, and to further evaluate the utility of qMRI assessments as outcome measures.</p><p><strong>Methods: </strong>Eighteen participants (10 placebo, 8 arimoclomol-treated) were recruited to undergo an MRI at baseline, 12 and 20 months. Spearman correlations between baseline clinical measures and qMRI measurements [fat fraction (FF), remaining muscle area (RMA), magnetisation transfer ratio (MTR), muscle water T2 (T2m) and fat fraction apparent (FFa)] were used to evaluate construct validity. A mixed model repeated measures (MMRM) strategy was employed to estimate mean changes, in order to determine treatment effects on qMRI biomarkers and evaluate responsiveness to disease progression over time. Longitudinal analyses examined Spearman correlations between changes in qMRI and changes in clinical assessments at the last available follow-up.</p><p><strong>Results: </strong>Baseline FF, RMA, MTR and FFa of the thigh and quadriceps demonstrated strong construct validity. No significant treatment effects on the qMRI measures were detected. FF, RMA and FFa demonstrated strong responsiveness to disease progression (standardised response means>0.8, p<0.05) at 20 months. Longitudinal changes in thigh T2m were strongly correlated with changes in myometry and modified timed up and go velocity.</p><p><strong>Conclusions: </strong>Arimoclomol had no significant effects on the qMRI measurements evaluated, consistent with clinical outcomes from the main trial. The qMRI measurements demonstrated both validity and responsiveness, further supporting their potential utility as biomarkers in IBM.</p>","PeriodicalId":10274,"journal":{"name":"Clinical and experimental rheumatology","volume":"43 2","pages":"334-344"},"PeriodicalIF":3.4,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143522731","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":"An update on myositis autoantibodies and insights into pathogenesis.","authors":"Guochun Wang, Neil John McHugh","doi":"10.55563/clinexprheumatol/kyj2cy","DOIUrl":"10.55563/clinexprheumatol/kyj2cy","url":null,"abstract":"<p><p>Myositis-specific autoantibodies (MSAs) are hallmarks of idiopathic inflammatory myopathies (IIMs) and have become increasing valuable in disease diagnosis, phenotyping, and classification. In addition to their clinical utility, emerging data, including findings from several animal studies, suggest that MSAs and autoreactive T cells substantially contribute to the etiopathogenesis of IIMs. This review aims to provide an updated perspective on myositis autoantibodies by focusing on relevant clinical and translational studies.</p>","PeriodicalId":10274,"journal":{"name":"Clinical and experimental rheumatology","volume":" ","pages":"364-371"},"PeriodicalIF":3.4,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142557295","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}
Xiao Liang, Juan Wu, Huaming Ren, Meng Li, Chuping Huang, Jinger Guo Guo, Dongsheng Li, Juan Li, Junqing Zhu
{"title":"Clinical features and prognosis of idiopathic inflammatory myopathies with coexistent multiple myositis-specific antibodies.","authors":"Xiao Liang, Juan Wu, Huaming Ren, Meng Li, Chuping Huang, Jinger Guo Guo, Dongsheng Li, Juan Li, Junqing Zhu","doi":"10.55563/clinexprheumatol/22j41g","DOIUrl":"10.55563/clinexprheumatol/22j41g","url":null,"abstract":"<p><strong>Objectives: </strong>This study aimed to evaluate the clinical significance of the coexistence of 2 or more myositis-specific antibodies (multiple MSAs) in adult patients with idiopathic inflammatory myopathies (IIM).</p><p><strong>Methods: </strong>We assessed a cohort of 202 consecutive patients with IIM. Clinical features and survival rates were compared between patients with and without multiple MSAs.</p><p><strong>Results: </strong>Of those 202 patients, 44 (21.8%) were found to have multiple MSAs. 63.6% of the 44 patients tested positive for anti-aminoacyl-tRNA synthetase antibodies (anti-ARS+) and 52.3% positive for anti-melanoma differentiation-associated protein-5 antibody (anti-MDA5+). The presence of multiple MSAs was associated with less rapidly progressive interstitial lung disease (RP-ILD), fever, rash, periungual erythema, more muscle involvement and dysphagia, higher albumin level, and higher positive rate of ANA antibody in anti-MDA5+ population. In anti-ARS+ population with multiple MSAs, there were more V-neck sign, skin ulcers, dysphagia and peripheral edema. No differences in survival rates were observed between patients with or without multiple MSAs in the overall and anti-ARS+ populations. However, the survival rate in anti-MDA5+ population with multiple MSAs was significantly higher than those without multiple MSAs (p = 0.003). Moreover, multiple MSAs remained an independent protective factor against mortality in multivariable Cox regression analysis of anti-MDA5+ population [HR 0.108 (95% CI 0.013, 0.908), p=0.041].</p><p><strong>Conclusions: </strong>Multiple MSAs coexist in some IIM patients and their existence indicates mixed features from concomitant MSAs in anti-MDA5+ population and anti-ARS+ population. Identifying multiple MSAs could help to discover a more favourable disease phenotype with decreased mortality in anti-MDA5+ population.</p>","PeriodicalId":10274,"journal":{"name":"Clinical and experimental rheumatology","volume":" ","pages":"211-220"},"PeriodicalIF":3.4,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141183629","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}
Julie J Paik, Jiri Vencovský, Christina Charles-Schoeman, Grace C Wright, Ruth Ann Vleugels, Alexandra S Goriounova, Paul N Mudd, Rohit Aggarwal
{"title":"Brepocitinib, a potent and selective TYK2/JAK1 inhibitor: scientific and clinical rationale for dermatomyositis.","authors":"Julie J Paik, Jiri Vencovský, Christina Charles-Schoeman, Grace C Wright, Ruth Ann Vleugels, Alexandra S Goriounova, Paul N Mudd, Rohit Aggarwal","doi":"10.55563/clinexprheumatol/eeglsa","DOIUrl":"10.55563/clinexprheumatol/eeglsa","url":null,"abstract":"<p><p>Dermatomyositis (DM) is a rare and debilitating, systemic, autoimmune disease. While heterogenous in presentation and severity, DM is primarily characterised by a spectrum of skin and muscle disease, which may include proximal muscle weakness and recalcitrant cutaneous eruptions. DM may also be associated with joint pain and stiffness, inflammatory arthritis, dysphagia, fatigue, and calcinosis. The current standard of care for DM includes glucocorticoids, immunosuppressants, and intravenous immunoglobulin (IVIg). Unfortunately, these medications are not uniformly effective and can lead to adverse events, particularly with chronic use, necessitating discontinuation of therapy. Therefore, a substantial unmet need exists for more tailored and efficacious therapies that target DM pathogenesis. Brepocitinib is an oral, once-daily, novel, and specific TYK2/JAK1 inhibitor. Brepocitinib's potent inhibition of TYK2 and JAK1 reduces the signalling of pro-inflammatory cytokines, including IFN-α/β, IL-12, IL-23, and IFNγ, that have been implicated in the pathogenesis of DM. Other JAK inhibitors have been used off-label in both case series and open-label clinical trials in patients with DM; and brepocitinib has demonstrated efficacy in phase 2 clinical trials of several other autoimmune diseases, including plaque psoriasis, psoriatic arthritis, Crohn's disease, hidradenitis suppurativa, and ulcerative colitis. Therefore, there is a strong scientific and clinical rationale for the utility and potential effectiveness of brepocitinib in the treatment of DM patients. Currently, the safety, tolerability, and efficacy of brepocitinib is being evaluated in the largest (n=225) double-blind placebo-controlled phase 3 trial in DM patients to date (VALOR - NCT0543726).</p>","PeriodicalId":10274,"journal":{"name":"Clinical and experimental rheumatology","volume":" ","pages":"354-363"},"PeriodicalIF":3.4,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141615969","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}