ACR open rheumatology最新文献

{"title":"The Design of a Randomized Controlled Active Comparator Strategy Trial for Gout: Treat to Target Serum Urate Versus Treat to Avoid Symptoms.","authors":"Daniel H Solomon, Misti Paudel, Shravani Chitineni, Ana Fernandes, Tammy Pham, Shreya Billa, Chio Yokose, Kiara Tan, Julien J Dedier, Maureen D Dubreuil, John D Fitzgerald, Sally L Hodder, Tuhina Neogi, Michael H Pillinger, Kevin R Riggs, Kenneth G Saag, Paul G Shekelle, Zoe Tseng, Michael Toprover, David S Wei, Michael J Barry, Hyon K Choi","doi":"10.1002/acr2.70012","DOIUrl":"10.1002/acr2.70012","url":null,"abstract":"<p><strong>Objective: </strong>Controversy persists regarding the optimal management of gout in routine primary care. There is a lack of clarity on whether treating to a target serum urate (TTT-SU) versus treating to avoid symptoms (TTASx) is more effective.</p><p><strong>Methods: </strong>We designed a randomized controlled comparative effectiveness trial aimed at patients in primary care who have known gout, have elevated SU levels, and had at least one flare in the previous 12 months. The trial was designed to be pragmatic and incorporated structured input from primary care physicians, rheumatologists, and patients. The TTASx strategy group will receive weeklong courses of typical therapies for gout flares, such as colchicine, naproxen, or an oral glucocorticoid. The TTT-SU strategy group will receive urate-lowering therapy (primarily allopurinol) with dose titration to maintain an SU level <6 mg/dL, colchicine (or naproxen) prophylaxis for the first six months of urate-lowering therapy, and access to the same flare therapies as the TTASx group. Two clinicians (nurses or physicians) per site will be trained in each strategy to manage the patients in each arm without contamination. Gout flares are the primary outcome and are assessed every two weeks by trained study staff masked to treatment assignment using a validated questionnaire. The secondary outcome is quality of life. Blood pressure control, kidney function, glycemic control, and coronary atherosclerosis are exploratory secondary outcomes.</p><p><strong>Results: </strong>Several sites have started prescreening using automated search strategies in their patients' electronic health records. Of the first 1,381 patients found in primary care practices with a history of gout, 691 patients (50%) passed prescreening checks. These potentially eligible participants have a median age of 67 years, 85% are men, median SU levels are 7.2 mg/dL, and 18% are taking low dosages of allopurinol. These patients have been targeted for recruitment efforts that are underway now.</p><p><strong>Conclusion: </strong>This randomized controlled active comparator strategy trial will answer a key question in the treatment of patients with gout in primary care: the comparative effectiveness of TTT-SU versus TTASx in gout. Secondary and exploratory outcomes will add important information regarding the broader extra-articular and quality-of-life effects of lowering SU levels.</p>","PeriodicalId":93845,"journal":{"name":"ACR open rheumatology","volume":"7 3","pages":"e70012"},"PeriodicalIF":2.9,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11932158/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143702333","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"UK Medical Cannabis Registry: An Analysis of Outcomes of Medical Cannabis Therapy for Hypermobility-Associated Chronic Pain.","authors":"Mary Dickinson, Simon Erridge, John Warner-Levy, Evonne Clarke, Katy McLachlan, Ross Coomber, Wendy Holden, James J Rucker, Michael W Platt, Mikael H Sodergren","doi":"10.1002/acr2.70024","DOIUrl":"10.1002/acr2.70024","url":null,"abstract":"<p><strong>Objective: </strong>The study aims to evaluate the clinical outcomes in patients with hypermobility spectrum disorder (HSD) and hypermobile Ehlers-Danlos syndrome (hEDS) with chronic pain following treatment with cannabis-based medicinal products (CBMPs).</p><p><strong>Methods: </strong>This was a case series conducted with the UK Medical Cannabis Registry. The primary outcomes were changes in the following validated patient-reported outcome measures at 1, 3, 6, 12, and 18 months compared with baseline: Short-Form McGill Pain Questionnaire 2 (SF-MPQ-2), pain visual analog scale score (Pain-VAS), Brief Pain Inventory (BPI), five-level EQ-5D (EQ-5D-5L), Single-Item Sleep Quality Scale (SQS), General Anxiety Disorder Seven-Item Scale (GAD-7), and Patient Global Impression of Change. The incidence of adverse events was analyzed as secondary outcomes. Statistical significance was defined as P <0.050.</p><p><strong>Results: </strong>A total of 161 patients met inclusion criteria. Improvements were observed in BPI severity and interference subscales, SF-MPQ-2, and Pain-VAS (P < 0.001). Changes were also seen in the EQ-5D-5L index value, SQS, and GAD-7 (P < 0.001). A total of 50 patients (31.06%) reported one or more adverse event with a total incidence of 601 (373.29%). The most frequent rating for adverse events was moderate (n = 258; 160.25%), with headache being the most common (n = 44; 27.33%).</p><p><strong>Conclusion: </strong>An association was identified between patients with HSD/hEDS with chronic pain and improvements in pain-specific and general health-related quality of life following the commencement of CBMPs. CBMPs were also well tolerated at 18 months. These findings must be interpreted within the context of the limitations of study design but add further weight to calls for randomized controlled trials.</p>","PeriodicalId":93845,"journal":{"name":"ACR open rheumatology","volume":"7 3","pages":"e70024"},"PeriodicalIF":2.9,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11905011/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143627106","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Clinical Images: Back pain, flattened vertebral bodies, and a novel mutation in the TRAPPC2 gene.","authors":"Zhen He, Sheng-Ming Dai, Zhiyong Chen","doi":"10.1002/acr2.70000","DOIUrl":"10.1002/acr2.70000","url":null,"abstract":"","PeriodicalId":93845,"journal":{"name":"ACR open rheumatology","volume":"7 2","pages":"e70000"},"PeriodicalIF":2.9,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11825289/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143416458","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Clinical Images: Joint pain with CHIK sign: a classic clinical diagnosis.","authors":"Sayan Mukherjee, Nishant G Kamble, T G Sundaram, Puneet Kumar","doi":"10.1002/acr2.70008","DOIUrl":"10.1002/acr2.70008","url":null,"abstract":"","PeriodicalId":93845,"journal":{"name":"ACR open rheumatology","volume":"7 2","pages":"e70008"},"PeriodicalIF":2.9,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11834581/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143442811","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Comparative Effectiveness and Safety of the JAK Inhibitors and Biologic Disease-Modifying Antirheumatic Drugs in Treating Children With Nonsystemic Juvenile Idiopathic Arthritis: A Bayesian Meta-Analysis of Randomized Controlled Trials.","authors":"Yuxiang Li, Bin Huang, Sandra Andorf, Xiaomeng Yue, Daniel J Lovell, Hermine I Brunner","doi":"10.1002/acr2.11788","DOIUrl":"10.1002/acr2.11788","url":null,"abstract":"<p><strong>Objective: </strong>We evaluated the efficacy and safety profiles of JAK inhibitors (JAKi) and biologic disease-modifying antirheumatic drugs (bDMARDs) when used with or without methotrexate (MTX) for the treatment of nonsystemic forms of juvenile idiopathic arthritis (nsJIA).</p><p><strong>Methods: </strong>Randomized clinical trials (RCTs) investigating efficacy and safety outcomes of JAKi or bDMARDs for the nsJIA population up to 2023 were searched in ClinicalTrial.gov, PubMed, EMBASE, and Cochrane databases. Bayesian arm-based network meta-analysis compared efficacy as measured by Juvenile Idiopathic Arthritis-American College of Rheumatology 70 (JIA-ACR70) improvement and safety based on rates of serious adverse events (SAEs) among all therapies.</p><p><strong>Results: </strong>Eligible studies included 45 citations from 16 RCTs (7 parallel and 9 withdrawal trials) with a total of 1,821 participants that investigated nine bDMARDs, three with and six without MTX co-treatment, and two JAKis (tofacitinib and baricitnib). The reported SAE incidence rates ranged from 0 to 0.3 per person-year of follow-up; none of the pairwise comparisons were statistically significant. The JIA-ACR70 improvement by 16 weeks of treatment ranged from 11.3% to 89.5%. Compared with controls, significant JIA-ACR70 improvements were observed for etanercept, golimumab, and all three combination therapies (adalimumab+MTX, etanercept+MTX, and infliximab+MTX), with odds ratios ranging from 2.97 to 3.99. No significant pairwise comparisons between bDMARDs and JAKi versus bDMARDs were noted.</p><p><strong>Conclusion: </strong>Overall, no significant evidence was found for efficacy and safety profiles in pairwise comparisons of JAKis and bDMARDs. Future studies will expand the meta-analysis by including non-RCT studies and individual participant data.</p>","PeriodicalId":93845,"journal":{"name":"ACR open rheumatology","volume":"7 2","pages":"e11788"},"PeriodicalIF":2.9,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11834587/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143442936","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"How (Ultra-)Rare Gene Variants Improve Our Understanding of More Common Autoimmune and Inflammatory Diseases.","authors":"Alexandre Belot, Maud Tusseau, Jade Cognard, Sophie Georgin-Lavialle, Guilaine Boursier, Christian M Hedrich","doi":"10.1002/acr2.70003","DOIUrl":"10.1002/acr2.70003","url":null,"abstract":"<p><p>The aim of this study was to explore the impact of rare and ultra-rare genetic variants on the understanding and treatment of autoimmune and autoinflammatory diseases with a focus on systemic lupus erythematosus (SLE) and Behçet syndrome. This review summarizes current research on the monogenic causes of SLE and Behçet syndrome, highlighting the various pathways that can be responsible for these unique phenotypes. In monogenic SLE, the identification of complement and DNASE1L3 deficiencies has elucidated mechanisms of apoptotic body accumulation and extracellular nucleic acid sensing. Type I interferonopathies underline the specific role of DNA/RNA sensing and the interferon overexpression in the development of systemic autoimmunity. Other significant genetic defects include Toll-like receptor hypersignaling and JAK/STATopathies, which contribute to the breakdown of immune tolerance. To date, genetic defects directly affecting B and T cell biology only account for a minority of identified causes of monogenic lupus, highlighting the importance of a tight regulation of mechanistic target of rapamycin and RAS (Rat sarcoma GTPase)/MAPK (mitogen-activated protein kinase) signaling in lupus. In Behçet syndrome, rare variants in TNFAIP3, RELA, and NFKB1 genes have been identified, underscoring the importance of NF-κB overactivation. Additional monogenic diseases such as ELF4, WDR1 mutations and trisomy 8 further illustrate the genetic complexity of this condition. Observations from genetic studies in SLE and Behçet syndrome highlight the complexity of systemic inflammatory diseases in which distinct molecular defects caused by single-gene mutations can promote lupus or Behçet syndromes, often unrecognizable from their genetically complex \"classical\" forms. Insights gained from studying rare genetic variants enhance our understanding of immune function in health and disease, paving the way for targeted therapies and personalized medicine.</p>","PeriodicalId":93845,"journal":{"name":"ACR open rheumatology","volume":"7 2","pages":"e70003"},"PeriodicalIF":2.9,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11834591/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143442884","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Clinical Image: A Novel Capillaroscopic Morphologic Anomaly in a Smoker: The \"∩-Shaped\" Megacapillary.","authors":"Angelo Nigro","doi":"10.1002/acr2.70015","DOIUrl":"10.1002/acr2.70015","url":null,"abstract":"","PeriodicalId":93845,"journal":{"name":"ACR open rheumatology","volume":"7 2","pages":"e70015"},"PeriodicalIF":2.9,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11834582/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143442810","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Social Connections in Older Adults With Systemic Lupus Erythematosus: Patient Perspectives.","authors":"Sarah B Lieber, Sarah R Young, Yvonne Shea, Sarah P Gottesman, Robyn Lipschultz, Dongmei Sun, M Carrington Reid, Lisa A Mandl, Iris Navarro-Millán","doi":"10.1002/acr2.11801","DOIUrl":"10.1002/acr2.11801","url":null,"abstract":"<p><strong>Objective: </strong>Interpersonal relationships are crucial to healthy aging. Social isolation is associated with multiple adverse health outcomes in older adults, including depression. Those with chronic conditions, such as systemic lupus erythematosus (SLE), may be particularly vulnerable to social isolation. In this qualitative study, we elicited lived experiences of older adults with SLE related to social connections and emotional health.</p><p><strong>Methods: </strong>Adults ≥65 years of age with SLE participated in semistructured interviews based on a descriptive phenomenological design to describe the experience of aging with SLE. We collected self-reported data on sociodemographic and disease characteristics, social networks, and health-related quality of life. We probed participants regarding their interpersonal relationships and the effect of SLE on general health in the context of aging. We analyzed qualitative data thematically.</p><p><strong>Results: </strong>Among 30 participants with a mean age of 71.3 years, three themes emerged as essential to characterizing aging with SLE: (1) social isolation, (2) perceived burden to loved ones, and (3) adverse mental health effects of SLE. Participants frequently reported social isolation, often related to SLE disease manifestations rather than unavailability of social networks and situated within the context of burdening loved ones and mental health effects of SLE.</p><p><strong>Conclusion: </strong>Social isolation was commonly reported by older adults with SLE. Larger observational studies are needed to improve characterization of social isolation in this population and understand its association with depression and other adverse health outcomes. Investigational studies integrating strategies to improve social isolation in older adults with SLE may improve their health and well-being.</p>","PeriodicalId":93845,"journal":{"name":"ACR open rheumatology","volume":"7 2","pages":"e11801"},"PeriodicalIF":2.9,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11795054/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143191596","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Use of ¹⁸F-fluorodeoxyglucose Positron Emission Tomography to Monitor Disease Activity in Patients With Giant Cell Arteritis on Tocilizumab.","authors":"Kaitlin A Quinn, Mark A Ahlman, Peter C Grayson","doi":"10.1002/acr2.11797","DOIUrl":"10.1002/acr2.11797","url":null,"abstract":"<p><strong>Objective: </strong>The objective of this study was to assess the value of ¹⁸F-fluorodeoxyglucose (FDG)- positron emission tomography (PET) scans to monitor disease activity in patients with giant cell arteritis (GCA) on tocilizumab.</p><p><strong>Methods: </strong>Patients with GCA were recruited into a prospective cohort in which they underwent clinical, laboratory, and imaging assessments, including FDG-PET. FDG-PET scans were interpreted as active or inactive based on global impression by two independent readers. The PET Vascular Activity Score (PETVAS) was calculated to quantify arterial FDG uptake (scale 0-27). Vascular FDG-PET findings were described in patients with GCA on tocilizumab for at least six months. For patients in established clinical remission, FDG-PET findings were compared between those treated with tocilizumab versus glucocorticoid monotherapy.</p><p><strong>Results: </strong>A total of 36 patients with GCA underwent FDG-PET imaging on tocilizumab. Five patients had active clinical symptoms, and FDG-PET scans were active in all cases (PETVAS range: 20-27). For the 31 patients in clinical remission on tocilizumab, FDG-PET was active in 16 of these patients (52%) across a wide range of PETVAS (range 11-27). There were no associations between FDG-PET activity during remission and historical clinical features or longitudinal outcomes, including relapse risk or angiographic progression of disease. PETVAS was significantly lower during clinical remission in patients treated with tocilizumab compared to an additional 12 patients treated with glucocorticoid monotherapy (PETVAS 18 vs 24; P = 0.02).</p><p><strong>Conclusion: </strong>FDG-PET has limited value to guide management decisions or inform prognosis when obtained during remission in patients with GCA on tocilizumab. Compared to glucocorticoid monotherapy, tocilizumab significantly reduces, but often does not eliminate, vascular inflammation in GCA.</p>","PeriodicalId":93845,"journal":{"name":"ACR open rheumatology","volume":"7 2","pages":"e11797"},"PeriodicalIF":2.9,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11795057/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143191599","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Clinical Images: Vasculitic rash as a presenting symptom of heparin-induced thrombocytopenia in a woman with primary antiphospholipid syndrome.","authors":"Rachel E Elam, Kevin Thompson, Rick Saha, Anam Qureshi, Matthew Powell, Jackson C Elam, Giovanni Carter, Abdullah Kutlar","doi":"10.1002/acr2.11793","DOIUrl":"10.1002/acr2.11793","url":null,"abstract":"","PeriodicalId":93845,"journal":{"name":"ACR open rheumatology","volume":"7 2","pages":"e11793"},"PeriodicalIF":2.9,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11834583/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143442926","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}