Annals of the Rheumatic Diseases最新文献

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Effective use of anti-CD19 chimeric antigen receptor T cells in a case of treatment-resistant granulomatosis with polyangiitis. 抗cd19嵌合抗原受体T细胞在治疗抵抗性肉芽肿病合并多血管炎中的有效应用
IF 20.3 1区 医学
Annals of the Rheumatic Diseases Pub Date : 2025-05-29 DOI: 10.1016/j.ard.2025.04.029
Luisa Uhlmann, Matthias Pierer, Marco Krasselt, Georg Franke, Robert Zeidler, Osama Sabri, Timm Denecke, Ulrich Sack, Ulrike Köhl, Andreas Boldt, Dominic Borie, Ulf Wagner, Uwe Platzbecker, Vladan Vucinic
{"title":"Effective use of anti-CD19 chimeric antigen receptor T cells in a case of treatment-resistant granulomatosis with polyangiitis.","authors":"Luisa Uhlmann, Matthias Pierer, Marco Krasselt, Georg Franke, Robert Zeidler, Osama Sabri, Timm Denecke, Ulrich Sack, Ulrike Köhl, Andreas Boldt, Dominic Borie, Ulf Wagner, Uwe Platzbecker, Vladan Vucinic","doi":"10.1016/j.ard.2025.04.029","DOIUrl":"https://doi.org/10.1016/j.ard.2025.04.029","url":null,"abstract":"","PeriodicalId":8087,"journal":{"name":"Annals of the Rheumatic Diseases","volume":" ","pages":""},"PeriodicalIF":20.3,"publicationDate":"2025-05-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144186345","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Prognosis of older patients with multimorbidity who do not meet the definition of difficult-to-treat rheumatoid arthritis but have uncontrolled disease activity. 不符合难治性类风湿关节炎定义但疾病活动不受控制的老年多病患者的预后
IF 20.3 1区 医学
Annals of the Rheumatic Diseases Pub Date : 2025-05-29 DOI: 10.1016/j.ard.2025.05.005
Satoshi Takanashi, Tsutomu Takeuchi, Yuko Kaneko
{"title":"Prognosis of older patients with multimorbidity who do not meet the definition of difficult-to-treat rheumatoid arthritis but have uncontrolled disease activity.","authors":"Satoshi Takanashi, Tsutomu Takeuchi, Yuko Kaneko","doi":"10.1016/j.ard.2025.05.005","DOIUrl":"https://doi.org/10.1016/j.ard.2025.05.005","url":null,"abstract":"","PeriodicalId":8087,"journal":{"name":"Annals of the Rheumatic Diseases","volume":" ","pages":""},"PeriodicalIF":20.3,"publicationDate":"2025-05-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144186347","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Variants in the DDX6-CXCR5 autoimmune disease risk locus influence the regulatory network in immune cells and salivary gland. DDX6-CXCR5自身免疫性疾病风险位点的变异影响免疫细胞和唾液腺的调节网络。
IF 20.3 1区 医学
Annals of the Rheumatic Diseases Pub Date : 2025-05-29 DOI: 10.1016/j.ard.2025.04.023
Mandi M Wiley, Marcin Radziszewski, Bhuwan Khatri, Michelle L Joachims, Kandice L Tessneer, Anna M Stolarczyk, Songyuan Yao, James Li, Cherilyn Pritchett-Frazee, Audrey A Johnston, Astrid Rasmussen, Juan-Manuel Anaya, Lara A Aqrawi, Sang-Cheol Bae, Eva Baecklund, Albin Björk, Johan G Brun, Sara Magnusson Bucher, Nick Dand, Maija-Leena Eloranta, Fiona Engelke, Helena Forsblad-d'Elia, Cecilia Fugmann, Stuart B Glenn, Chen Gong, Jacques-Eric Gottenberg, Daniel Hammenfors, Juliana Imgenberg-Kreuz, Janicke Liaaen Jensen, Svein Joar Auglænd Johnsen, Malin V Jonsson, Jennifer A Kelly, Sharmily Khanam, Kwangwoo Kim, Marika Kvarnström, Thomas Mandl, Javier Martín, David L Morris, Gaetane Nocturne, Katrine Brække Norheim, Peter Olsson, Øyvind Palm, Jacques-Olivier Pers, Nelson L Rhodus, Christopher Sjöwall, Kathrine Skarstein, Kimberly E Taylor, Phil Tombleson, Gudny Ella Thorlacius, Swamy R Venuturupalli, Edward M Vital, Daniel J Wallace, Lida Radfar, Michael T Brennan, Judith A James, R Hal Scofield, Patrick M Gaffney, Lindsey A Criswell, Roland Jonsson, Silke Appel, Per Eriksson, Simon J Bowman, Roald Omdal, Lars Rönnblom, Blake M Warner, Maureen Rischmueller, Torsten Witte, A Darise Farris, Xavier Mariette, Caroline H Shiboski, Marie Wahren-Herlenius, Marta E Alarcón-Riquelme, Wan-Fai Ng, Kathy L Sivils, Joel M Guthridge, Indra Adrianto, Timothy J Vyse, Betty P Tsao, Gunnel Nordmark, Christopher J Lessard
{"title":"Variants in the DDX6-CXCR5 autoimmune disease risk locus influence the regulatory network in immune cells and salivary gland.","authors":"Mandi M Wiley, Marcin Radziszewski, Bhuwan Khatri, Michelle L Joachims, Kandice L Tessneer, Anna M Stolarczyk, Songyuan Yao, James Li, Cherilyn Pritchett-Frazee, Audrey A Johnston, Astrid Rasmussen, Juan-Manuel Anaya, Lara A Aqrawi, Sang-Cheol Bae, Eva Baecklund, Albin Björk, Johan G Brun, Sara Magnusson Bucher, Nick Dand, Maija-Leena Eloranta, Fiona Engelke, Helena Forsblad-d'Elia, Cecilia Fugmann, Stuart B Glenn, Chen Gong, Jacques-Eric Gottenberg, Daniel Hammenfors, Juliana Imgenberg-Kreuz, Janicke Liaaen Jensen, Svein Joar Auglænd Johnsen, Malin V Jonsson, Jennifer A Kelly, Sharmily Khanam, Kwangwoo Kim, Marika Kvarnström, Thomas Mandl, Javier Martín, David L Morris, Gaetane Nocturne, Katrine Brække Norheim, Peter Olsson, Øyvind Palm, Jacques-Olivier Pers, Nelson L Rhodus, Christopher Sjöwall, Kathrine Skarstein, Kimberly E Taylor, Phil Tombleson, Gudny Ella Thorlacius, Swamy R Venuturupalli, Edward M Vital, Daniel J Wallace, Lida Radfar, Michael T Brennan, Judith A James, R Hal Scofield, Patrick M Gaffney, Lindsey A Criswell, Roland Jonsson, Silke Appel, Per Eriksson, Simon J Bowman, Roald Omdal, Lars Rönnblom, Blake M Warner, Maureen Rischmueller, Torsten Witte, A Darise Farris, Xavier Mariette, Caroline H Shiboski, Marie Wahren-Herlenius, Marta E Alarcón-Riquelme, Wan-Fai Ng, Kathy L Sivils, Joel M Guthridge, Indra Adrianto, Timothy J Vyse, Betty P Tsao, Gunnel Nordmark, Christopher J Lessard","doi":"10.1016/j.ard.2025.04.023","DOIUrl":"https://doi.org/10.1016/j.ard.2025.04.023","url":null,"abstract":"<p><strong>Objectives: </strong>Sjögren's disease (SjD) and systemic lupus erythematosus (SLE) share genetic risk at the DDX6-CXCR5 locus (11q23.3). Identifying and functionally characterising shared SNPs spanning this locus can provide new insights into common genetic mechanisms of autoimmunity.</p><p><strong>Methods: </strong>Transdisease meta-analyses, fine-mapping, and bioinformatic analyses prioritised shared likely functional single nucleotide polymorphisms (SNPs) for allele-specific and cell type-specific functional interrogation using electromobility shift, luciferase reporter, and quantitative chromatin conformation capture assays and clustered regularly interspaced short palindromic repeat (CRISPR) gene regulation.</p><p><strong>Results: </strong>Five shared SNPs were identified as likely functional in primary human immune cells, salivary gland and kidney tissues: rs57494551, rs4936443, rs4938572, rs7117261, and rs4938573. All 5 SNPs exhibited cell type-specific and allele-specific effects on nuclear protein binding affinity and enhancer/promoter regulatory activity in immune, salivary gland epithelial, and kidney epithelial cell models. Mapping of chromatin-chromatin interactions revealed a chromatin regulatory network that expanded beyond DDX6 and CXCR5 to include PHLDB1, lnc-PHLDB1-1, BCL9L, TRAPPC4, among others. Coalescence of functional assays and multiomic data analyses indicated that these SNPs likely modulate the activity of 3 regulatory regions: intronic rs57494551 regulatory region, intergenic SNP haplotype (rs4938572, rs4936443, and rs7117261) regulatory region, and rs4938573 regulatory region upstream of the CXCR5 promoter.</p><p><strong>Conclusions: </strong>Shared genetic susceptibly at the DDX6-CXCR5 locus in SjD and SLE likely alters common mechanisms of autoimmunity, including interferon signalling (DDX6), autophagy (TRAPPC4), and lymphocytic infiltration of disease-target tissues (CXCR5). Further, using multiomic data from patients with SjD, combined with bioinformatic and in vitro functional studies, can provide mechanistic insights into how genetic risk influences the biological pathways that drive complex autoimmunity.</p>","PeriodicalId":8087,"journal":{"name":"Annals of the Rheumatic Diseases","volume":" ","pages":""},"PeriodicalIF":20.3,"publicationDate":"2025-05-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144186348","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Remote monitoring or patient-initiated care in axial spondyloarthritis: a 3-armed randomised controlled noninferiority trial. 轴型脊柱炎的远程监测或患者主动护理:一项三臂随机对照非劣效性试验
IF 20.3 1区 医学
Annals of the Rheumatic Diseases Pub Date : 2025-05-23 DOI: 10.1016/j.ard.2025.04.019
Inger Jorid Berg, Eirik K Kristianslund, Anne Therese Tveter, Joseph Sexton, Gunnstein Bakland, Laure Gossec, Sarah Hakim, Gary J Macfarlane, Ellen Moholt, Sella Aarrestad Provan, Emil E Kvernberg Thomassen, Annette de Thurah, Espen A Haavardsholm, Siri Lillegraven, Nina Osteras
{"title":"Remote monitoring or patient-initiated care in axial spondyloarthritis: a 3-armed randomised controlled noninferiority trial.","authors":"Inger Jorid Berg, Eirik K Kristianslund, Anne Therese Tveter, Joseph Sexton, Gunnstein Bakland, Laure Gossec, Sarah Hakim, Gary J Macfarlane, Ellen Moholt, Sella Aarrestad Provan, Emil E Kvernberg Thomassen, Annette de Thurah, Espen A Haavardsholm, Siri Lillegraven, Nina Osteras","doi":"10.1016/j.ard.2025.04.019","DOIUrl":"https://doi.org/10.1016/j.ard.2025.04.019","url":null,"abstract":"<p><strong>Objectives: </strong>This study aimed to determine whether novel follow-up regimen, remote monitoring, or patient-initiated care is noninferior to usual care in maintaining low disease activity, in patients with axial spondyloarthritis (axSpA).</p><p><strong>Methods: </strong>This is a randomised, controlled, 3-armed, parallel-group, open-label, noninferiority trial. Patients with axSpA in low disease activity on stable treatment with tumour necrosis factor inhibitor were recruited from a Norwegian outpatient clinic. Patients were randomly allocated 1:1:1 to remote monitoring, patient-initiated care, or usual care (control group), with 18 months of follow-up. Primary outcome was mean probability of axSpA Disease Activity Score (ASDAS) of <2.1, compared between groups at 6, 12, and 18 months, with 15% noninferiority margin. Secondary outcomes included other measures of disease activity, physical function, patient satisfaction, change of medication, resource use, and adverse events.</p><p><strong>Results: </strong>Of 243 patients enrolled patients, 235 completed the study (remote monitoring = 75, patient-initiated care = 79, usual care = 81). At the 6-month, 12-month, and 18-month assessments, 90% or more patients in all 3 groups had ASDAS of <2.1. The estimated difference of probability of ASDAS < 2.1 was as follows: usual care vs remote monitoring, -4.1% (97.5% CI, -9.9% to 1.8%); usual care vs patient-initiated care, -1.1% (97.5% CI, -7.2% to 4.9%); and remote monitoring vs patient-initiated care, 2.9% (95% CI, -1.5% to 7.4%). Health providers' resource use was lowest in patient-initiated care; other secondary outcomes were comparable.</p><p><strong>Conclusions: </strong>In patients with axSpA in low disease activity and on stable treatment with tumour necrosis factor inhibitor, follow-up with remote monitoring or patient-initiated care was noninferior to usual care in maintaining low disease activity, supporting the implementation of novel follow-up strategies.</p>","PeriodicalId":8087,"journal":{"name":"Annals of the Rheumatic Diseases","volume":" ","pages":""},"PeriodicalIF":20.3,"publicationDate":"2025-05-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144141203","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
CD4+ tissue-resident memory Th17 cells are a major source of IL-17A in Spondyloarthritis synovial tissue. CD4+组织驻留记忆Th17细胞是脊柱炎滑膜组织中IL-17A的主要来源。
IF 20.3 1区 医学
Annals of the Rheumatic Diseases Pub Date : 2025-05-23 DOI: 10.1016/j.ard.2025.04.018
Feng Liu, Hui Shi, Jason D Turner, Rachel Anscombe, Jiaqi Li, Takuya Sekine, Ariane Hammitzsch, Devika Agarwal, Christopher Mahony, Jiewei Chen, Ben Kendrick, Dajiang Du, Qiang Tong, Lihua Duan, Kyla Dooley, Hai Fang, Ilya Korsunsky, Roopa Madhu, Adam P Cribbs, Matthias Friedrich, Brian D Marsden, Yi-Ling Chen, Graham Ogg, Anna Adams, Warner Chen, Steven Leonardo, Fiona E McCann, Christopher D Buckley, Terence Rooney, Thomas Freeman, Holm H Uhlig, Calliope Dendrou, Adam Croft, Andrew Filer, Paul Bowness, Liye Chen
{"title":"CD4+ tissue-resident memory Th17 cells are a major source of IL-17A in Spondyloarthritis synovial tissue.","authors":"Feng Liu, Hui Shi, Jason D Turner, Rachel Anscombe, Jiaqi Li, Takuya Sekine, Ariane Hammitzsch, Devika Agarwal, Christopher Mahony, Jiewei Chen, Ben Kendrick, Dajiang Du, Qiang Tong, Lihua Duan, Kyla Dooley, Hai Fang, Ilya Korsunsky, Roopa Madhu, Adam P Cribbs, Matthias Friedrich, Brian D Marsden, Yi-Ling Chen, Graham Ogg, Anna Adams, Warner Chen, Steven Leonardo, Fiona E McCann, Christopher D Buckley, Terence Rooney, Thomas Freeman, Holm H Uhlig, Calliope Dendrou, Adam Croft, Andrew Filer, Paul Bowness, Liye Chen","doi":"10.1016/j.ard.2025.04.018","DOIUrl":"https://doi.org/10.1016/j.ard.2025.04.018","url":null,"abstract":"<p><strong>Objectives: </strong>Interleukin (IL)-17A is a key driver of spondyloarthritis (SpA) joint pathology. We aimed to identify its cellular source in synovial tissue from patients with 2 forms of SpA, namely axial SpA (AxSpA) and psoriatic arthritis (PsA).</p><p><strong>Methods: </strong>Synovial tissue from patients with SpA was profiled using single-cell RNA sequencing (scRNA-seq; AxSpA, n = 5; PsA, n = 6) or spatial RNA profiling (PsA, n = 4). CellPhoneDB was used to infer cell-cell communication. Tissue-resident memory Th17 (TRM17)-like cells were generated in vitro using blood memory CD4+ T cells from SpA patients. An epigenetic inhibitor library, siRNA, and clustered regularly interspaced short palindromic repeats (CRISPR) were used to identify epigenetic regulator(s) for TRM17.</p><p><strong>Results: </strong>scRNA-seq showed that CD4+CXCR6+ TRM17 cells are the predominant spontaneous IL17A producers in SpA synovium. Cell-cell communication and single-cell spatial analysis support the interaction between TRM17 and CLEC10A+ dendritic cells, which were activated in SpA. Both sublining and lining fibroblasts in SpA synovium showed evidence of interleukin (IL)-17A activation. In vitro-generated CD4+ TRM17-like cells phenocopied joint tissue TRM17, producing IL-17A/F upon T cell-receptor (TCR) stimulation, which was further enhanced by cytokines. Perturbation of BRD1 inhibited the generation of TRM17-like cells.</p><p><strong>Conclusions: </strong>CD4+ TRM17 cells are the predominant source of IL-17A in SpA synovial tissue. TCR stimulation is essential for the secretion of IL-17A by CD4+TRM17-like cells. The epigenetic regulator bromodomain-containing protein 1 (BRD1) contributes to the generation of CD4+TRM17. Depleting CD4+TRM17 cells in SpA is thus a therapeutic strategy with potential to induce long-term remission.</p>","PeriodicalId":8087,"journal":{"name":"Annals of the Rheumatic Diseases","volume":" ","pages":""},"PeriodicalIF":20.3,"publicationDate":"2025-05-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144141149","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Spatial multiomics decipher fibroblast-macrophage dynamics in systemic sclerosis. 空间多组学解读系统性硬化症成纤维细胞-巨噬细胞动力学。
IF 20.3 1区 医学
Annals of the Rheumatic Diseases Pub Date : 2025-05-22 DOI: 10.1016/j.ard.2025.04.025
Zhijian Li, Aleix Rius Rigau, Wenjie Xie, Linlin Huang, Wenjing Ye, Yi-Nan Li, Alexandru-Emil Matei, Christina Bergmann, Xiaohang Shao, Hejian Zou, Jiucun Wang, Luca Pinello, Jörg H W Distler, Rui He, Minrui Liang
{"title":"Spatial multiomics decipher fibroblast-macrophage dynamics in systemic sclerosis.","authors":"Zhijian Li, Aleix Rius Rigau, Wenjie Xie, Linlin Huang, Wenjing Ye, Yi-Nan Li, Alexandru-Emil Matei, Christina Bergmann, Xiaohang Shao, Hejian Zou, Jiucun Wang, Luca Pinello, Jörg H W Distler, Rui He, Minrui Liang","doi":"10.1016/j.ard.2025.04.025","DOIUrl":"https://doi.org/10.1016/j.ard.2025.04.025","url":null,"abstract":"<p><strong>Objectives: </strong>Stromal-immune crosstalk shapes the pathogenic microenvironment of systemic sclerosis (SSc), but the spatial regulatory networks underlying fibrogenesis remain poorly defined. We aimed to explore tissue organisation and cell coordination in SSc skin, providing spatiotemporal insights into disease mechanisms and bridging the gap between omics discovery and precision medicine.</p><p><strong>Methods: </strong>We performed spatial transcriptomics on skin biopsies from 10 patients with diffuse cutaneous SSc and 4 healthy controls using the 10× Visium platform. These findings were confirmed using higher-resolution Stereo-seq transcriptomics, spatial proteomics, and single-cell RNA sequencing data from patients with SSc, SSc mouse models, and wound-healing reindeer models. In vivo and in vitro studies were conducted to validate the key regulatory pathways.</p><p><strong>Results: </strong>Fourteen skin biopsies were analysed, revealing significant expansion of fibrotic niches enriched with fibroblasts and macrophages in SSc, correlating with clinical severity. We revealed disease-specific cell states of fibroblasts and macrophages and evaluated their spatial dependency and cell-cell communication. Stratification based on signature genes enabled the identification of patients with SSc with progressive disease and treatment-nonresponsive phenotype. ACKR3 (a CXCL12 decoy receptor) was selectively expressed in myofibroblast progenitors, which diminished during differentiation towards mature myofibroblast, potentially serving to regulate CXCL12/CXCR4-mediated proinflammatory macrophage recruitment. Inhibition of CXCR4 attenuated skin and lung fibrosis in experimental fibrosis mouse models.</p><p><strong>Conclusions: </strong>Our spatially resolved atlas uncovered dynamic fibroblast-macrophage interplay as a hallmark of fibrotic niche expansion. These findings offer spatiotemporal insights into disease mechanisms and pave the way for advanced mechanistic and therapeutic studies, bridging the gap between omics discovery and precision medicine.</p>","PeriodicalId":8087,"journal":{"name":"Annals of the Rheumatic Diseases","volume":" ","pages":""},"PeriodicalIF":20.3,"publicationDate":"2025-05-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144131791","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Treating juvenile dermatomyositis to target: Paediatric Rheumatology European Society/Childhood Arthritis and Rheumatology Research Alliance-endorsed recommendations from an international task force. 治疗青少年皮肌炎的目标:儿科风湿病欧洲学会/儿童关节炎和风湿病研究联盟认可的国际工作组的建议。
IF 20.3 1区 医学
Annals of the Rheumatic Diseases Pub Date : 2025-05-22 DOI: 10.1016/j.ard.2025.04.024
Angelo Ravelli, Silvia Rosina, Jayne M MacMahon, Talia Baird, Ana Isabel Rebollo-Giménez, Claas Hinze, Liza J McCann, Ann M Reed, Lisa G Rider, Matilde Arvigo, Brigitte Bader-Meunier, Claudio Bruno, Li Caifeng, Raquel Campanilho-Marques, Sara Cuccato, Chiara Fiorillo, Nikki A Hahn, Adam M Huber, Marc Jansen, Ozgur Kasapcopur, Maria Martha Katsikas, Susan Kim, Polly Livermore, Sue Maillard, Clara Malattia, Angela Nyangore Migowa, Takako Miyamae, Ruth Murphy, Rebecca Nicolai, Charalampia Papadopoulou, Clarissa Pilkington, Helga Sanner, Sujata Sawhney, Elzbieta Smolewska, Stacey E Tarvin, Georgina Tiller, Natasa Toplak, Lucy R Wedderburn, Francesca Bovis, Alessandro Consolaro, Brian M Feldman
{"title":"Treating juvenile dermatomyositis to target: Paediatric Rheumatology European Society/Childhood Arthritis and Rheumatology Research Alliance-endorsed recommendations from an international task force.","authors":"Angelo Ravelli, Silvia Rosina, Jayne M MacMahon, Talia Baird, Ana Isabel Rebollo-Giménez, Claas Hinze, Liza J McCann, Ann M Reed, Lisa G Rider, Matilde Arvigo, Brigitte Bader-Meunier, Claudio Bruno, Li Caifeng, Raquel Campanilho-Marques, Sara Cuccato, Chiara Fiorillo, Nikki A Hahn, Adam M Huber, Marc Jansen, Ozgur Kasapcopur, Maria Martha Katsikas, Susan Kim, Polly Livermore, Sue Maillard, Clara Malattia, Angela Nyangore Migowa, Takako Miyamae, Ruth Murphy, Rebecca Nicolai, Charalampia Papadopoulou, Clarissa Pilkington, Helga Sanner, Sujata Sawhney, Elzbieta Smolewska, Stacey E Tarvin, Georgina Tiller, Natasa Toplak, Lucy R Wedderburn, Francesca Bovis, Alessandro Consolaro, Brian M Feldman","doi":"10.1016/j.ard.2025.04.024","DOIUrl":"https://doi.org/10.1016/j.ard.2025.04.024","url":null,"abstract":"<p><strong>Objectives: </strong>Despite the recent prognostic improvement, a sizeable proportion of patients with juvenile dermatomyositis (JDM) respond suboptimally to contemporary therapies. This study aimed to develop recommendations for treating JDM to target.</p><p><strong>Methods: </strong>A Steering Committee formulated a set of provisional recommendations based on evidence derived from a systematic literature review and a retrospective chart review of patients. These were discussed, amended, and voted on by an international Task Force, including 28 paediatric rheumatologists, 2 specialists in neuromuscular diseases, 1 dermatologist, 1 physical therapist, 1 research nurse, 2 patients with JDM, and 1 parent of a patient with JDM. Items that achieved at least an 80% majority vote were accepted as final recommendations.</p><p><strong>Results: </strong>Although the literature review did not reveal trials that compared a treat-to-target strategy with a nonsteered approach, it provided indirect evidence about specific end points that could serve as targets that facilitated development of recommendations. The group reached consensus on 7 overarching principles and 12 recommendations. It was agreed that both patients/parents and treaters should share decisions in setting treatment targets and therapeutic strategies, with inactive disease as the preferred target and minimal disease activity an alternative one. Inactive disease is targeted to be achieved within 12 months after treatment start. Interim targets include minimal and moderate clinical improvement within 6 weeks and 3 months, respectively, and normalisation of muscle strength within 6 months. High-dose glucocorticoids remain fundamental in the initial management, but progressive tapering and discontinuation within 12 months through optimisation of concomitant immunomodulatory therapy was advised. A research agenda was formulated.</p><p><strong>Conclusions: </strong>The Task Force developed recommendations for treating JDM to target, being aware that the evidence is not strong and needs to be expanded by future research. Implementation of the recommendations in clinical practice will help to reach optimal outcomes for JDM.</p>","PeriodicalId":8087,"journal":{"name":"Annals of the Rheumatic Diseases","volume":" ","pages":""},"PeriodicalIF":20.3,"publicationDate":"2025-05-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144131794","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
In immune-mediated necrotising myopathy, anti-HMGCR antibodies inhibit HMGCR activity, leading to the sarcoplasmic accumulation of lipid droplets and myofibres necrosis. 在免疫介导的坏死性肌病中,抗HMGCR抗体抑制HMGCR活性,导致肌浆内脂滴积聚和肌纤维坏死。
IF 20.3 1区 医学
Annals of the Rheumatic Diseases Pub Date : 2025-05-22 DOI: 10.1016/j.ard.2025.04.027
Margherita Giannini, Giulia Quiring, Mustapha Oulad-Abdelghani, Béatrice Lannes, Yves Allenbach, Olivier Benveniste, Olivier Boyer, Aleksandra Nadaj Pakleza, Bernard Geny, Alain Meyer
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引用次数: 0
Development of salivary gland organoids derived from patient biopsies: a functional model of Sjögren's disease. 从患者活检中获得的唾液腺类器官的发展:Sjögren病的功能模型。
IF 20.3 1区 医学
Annals of the Rheumatic Diseases Pub Date : 2025-05-19 DOI: 10.1016/j.ard.2025.04.020
Loïc Meudec, Negaar Goudarzi, Sacha E Silva-Saffar, Juliette Pascaud, Fanny Jaulin, Quentin Pascal, Thierry Lazure, Rami Bechara, Xavier Mariette, Gaetane Nocturne
{"title":"Development of salivary gland organoids derived from patient biopsies: a functional model of Sjögren's disease.","authors":"Loïc Meudec, Negaar Goudarzi, Sacha E Silva-Saffar, Juliette Pascaud, Fanny Jaulin, Quentin Pascal, Thierry Lazure, Rami Bechara, Xavier Mariette, Gaetane Nocturne","doi":"10.1016/j.ard.2025.04.020","DOIUrl":"https://doi.org/10.1016/j.ard.2025.04.020","url":null,"abstract":"<p><strong>Objectives: </strong>Salivary gland epithelial cells (SGECs) play a key role in Sjögren's disease (SjD) as an active contributor to the pathogenesis. Current models lack clear epithelial readouts. Our aim was to establish a more advanced model by developing salivary gland organoids (SGOs) from labial salivary gland biopsies (LSGBs) of SjD patients and sicca controls.</p><p><strong>Methods: </strong>We included SjD patients fulfilling the American College of Rheumatology/European League Against Rheumatism 2016 criteria and sicca controls. LSGBs were dissociated, encapsulated in extracellular matrix, and submerged in growth expansion medium for long-term culture. SGOs were primarily cultured in differentiation medium and then transferred to a low attachment plate to form differentiated SGOs (DIF-SGOs).</p><p><strong>Results: </strong>We included 13 SjD and 15 controls. In both groups, SGOs were formed, demonstrating long-term culture viability and comparable self-renewal capacity (3.3 ± 1.7 months). DIF-SGOs comparably exhibited mature acinar (aquaporin 5, amylase), ductal (cytokeratins 5 and 7) and myoepithelial (α-smooth muscle actin) markers in both groups. DIF-SGOs were responsive to inflammation by expressing BAFF, CXCL10 and IL7 upon stimulation with poly(I:C) and interferon-α. DIF-SGOs also demonstrated a swelling response to cholinergic stimulation by pilocarpine. We observed significant differences between SjD- and control-derived SGOs. Notably, SjD-derived DIF-SGOs consistently maintained a persistent interferon signature throughout long-term culture. In addition, the swelling capacity was reduced in SjD-derived DIF-SGOs compared to control. However, treatment with tofacitinib enhanced the swelling ability, suggesting a potential effect on saliva production.</p><p><strong>Conclusions: </strong>We successfully developed SGOs from LSGBs of SjD patients, allowing long-term culture and faithfully recapitulating the disease phenotype. This model holds promise as a valuable tool for drug screening.</p>","PeriodicalId":8087,"journal":{"name":"Annals of the Rheumatic Diseases","volume":" ","pages":""},"PeriodicalIF":20.3,"publicationDate":"2025-05-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144109550","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Disease-modifying antirheumatic drug-free remission in psoriatic arthritis: is it attainable and sustainable? A large longitudinal study. 银屑病关节炎的抗风湿病无药物缓解:是否可以实现和可持续?一项大型纵向研究。
IF 20.3 1区 医学
Annals of the Rheumatic Diseases Pub Date : 2025-05-16 DOI: 10.1016/j.ard.2025.04.022
Selinde V J Snoeck Henkemans, Marijn Vis, Gonul Hazal Koc, Jolanda J Luime, Marc R Kok, Ilja Tchetverikov, Karen Visser, Lindy-Anne Korswagen, Jessica Bijsterbosch, Maikel van Oosterhout, Paul Baudoin, Jos H van der Kaap, Annette H M van der Helm-van Mil, Pascal H P de Jong
{"title":"Disease-modifying antirheumatic drug-free remission in psoriatic arthritis: is it attainable and sustainable? A large longitudinal study.","authors":"Selinde V J Snoeck Henkemans, Marijn Vis, Gonul Hazal Koc, Jolanda J Luime, Marc R Kok, Ilja Tchetverikov, Karen Visser, Lindy-Anne Korswagen, Jessica Bijsterbosch, Maikel van Oosterhout, Paul Baudoin, Jos H van der Kaap, Annette H M van der Helm-van Mil, Pascal H P de Jong","doi":"10.1016/j.ard.2025.04.022","DOIUrl":"https://doi.org/10.1016/j.ard.2025.04.022","url":null,"abstract":"<p><strong>Objectives: </strong>According to current management guidelines for psoriatic arthritis (PsA) tapering of disease-modifying antirheumatic drugs (DMARDs) can be considered. However, limited data are available on complete DMARD cessation, also known as disease-modifying antirheumatic drug-free remission (DFR). Therefore, our aim was to investigate whether DFR is achievable and sustainable in PsA and to evaluate possible predictors for sustained disease-modifying antirheumatic drug-free remission (SDFR).</p><p><strong>Methods: </strong>From the Dutch southwest Early Psoriatic Arthritis cohort, all newly diagnosed patients with oligoarticular/polyarticular PsA who were treated with DMARDs were included (n = 451). Prevalence of (S)DFR and flare rates (early and late) were described. DFR was defined as the absence of clinical synovitis for ≥3 months after DMARD cessation, while for SDFR, a period of >1 year was used. Early and late flares were defined as restarting DMARD treatment ≤1 and >1 year after DMARD cessation, respectively. Subsequently, possible predictors for true SDFR, that is, SDFR without flaring were explored.</p><p><strong>Results: </strong>After a median of 5.1 years (IQR, 3.0-7.3 years), 22% of patients with PsA had reached DFR, and after reaching DFR, 4.7% had an early flare. Thus, SDFR was achieved in 14.4% of patients with PsA; 5.3% experienced a late flare, which occurred a median of 1.7 years (IQR, 1.4-2.8 years) after DMARD cessation. Eventually, 9.1% of patients reached true SDFR. Low baseline Disease Activity Index in Psoriatic Arthritis and never using biological or targeted synthetic DMARDs were independent predictors for true SDFR. At the time of true SDFR, the Health Assessment Questionnaire was similar to the general population (median, 0.12; IQR, 0-0.75).</p><p><strong>Conclusions: </strong>DFR is attainable in oligoarticular/polyarticular PsA and is sustainable in 9% of patients. However, the subgroup of patients with PsA with high disease activity at baseline and who require biological or targeted synthetic DMARDs do not achieve true SDFR. To our knowledge, this is the first study to demonstrate that chronicity can potentially be influenced in a proportion of patients with PsA.</p>","PeriodicalId":8087,"journal":{"name":"Annals of the Rheumatic Diseases","volume":" ","pages":""},"PeriodicalIF":20.3,"publicationDate":"2025-05-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144092342","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
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