Pub Date : 2025-07-21DOI: 10.1038/s41584-025-01277-y
Guillermo J. Pons-Estel, María Fernanda Ramírez-Flores, Rosana Quintana, Sang-Cheol Bae, Dzifa Dey, Bernardo A. Pons-Estel, Ingris Peláez-Ballestas
Systemic lupus erythematosus is a complex and increasingly prevalent disease that presents substantial challenges in both diagnosis and management. Diagnostic delays frequently lead to irreversible organ damage, and remain a crucial concern, mainly among vulnerable populations, such as minority ethnic groups and those living in the global south. These delays are exacerbated by the clinical heterogeneity of systemic lupus erythematosus, the lack of specific diagnostic biomarkers, gaps in disease awareness or medical training, and persistent health care disparities, particularly in low-resource settings. This Perspective highlights the urgent need for a standardized definition of diagnostic delay that accounts for both clinical and socio-economic factors. By prioritizing early intervention and expanding access to specialized care, we can improve patient outcomes and reduce the long-term burden of the disease. Delays in the diagnosis of systemic lupus erythematosus negatively affect the treatment, quality of life and outcomes of affected individuals. In this Perspective, the authors provide an overview of the contributing factors and consequences of diagnostic delay in systemic lupus erythematosus and discuss how this urgent issue should be addressed.
{"title":"Addressing the challenge of global delays in diagnosis and treatment of systemic lupus erythematosus","authors":"Guillermo J. Pons-Estel, María Fernanda Ramírez-Flores, Rosana Quintana, Sang-Cheol Bae, Dzifa Dey, Bernardo A. Pons-Estel, Ingris Peláez-Ballestas","doi":"10.1038/s41584-025-01277-y","DOIUrl":"10.1038/s41584-025-01277-y","url":null,"abstract":"Systemic lupus erythematosus is a complex and increasingly prevalent disease that presents substantial challenges in both diagnosis and management. Diagnostic delays frequently lead to irreversible organ damage, and remain a crucial concern, mainly among vulnerable populations, such as minority ethnic groups and those living in the global south. These delays are exacerbated by the clinical heterogeneity of systemic lupus erythematosus, the lack of specific diagnostic biomarkers, gaps in disease awareness or medical training, and persistent health care disparities, particularly in low-resource settings. This Perspective highlights the urgent need for a standardized definition of diagnostic delay that accounts for both clinical and socio-economic factors. By prioritizing early intervention and expanding access to specialized care, we can improve patient outcomes and reduce the long-term burden of the disease. Delays in the diagnosis of systemic lupus erythematosus negatively affect the treatment, quality of life and outcomes of affected individuals. In this Perspective, the authors provide an overview of the contributing factors and consequences of diagnostic delay in systemic lupus erythematosus and discuss how this urgent issue should be addressed.","PeriodicalId":18810,"journal":{"name":"Nature Reviews Rheumatology","volume":"21 9","pages":"566-574"},"PeriodicalIF":32.7,"publicationDate":"2025-07-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144669630","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}
Pub Date : 2025-07-10DOI: 10.1038/s41584-025-01281-2
Hatem El-Shanti
Familial Mediterranean fever (FMF) is a monogenic autoinflammatory disorder with high prevalence in Mediterranean populations. Considerable advances in the management of FMF have been made in the past decade, with respect to the use of biologic drugs and understanding colchicine resistance. The 2024 updated FMF management recommendations are timely and reflect these advances.
{"title":"Guided management of familial Mediterranean fever","authors":"Hatem El-Shanti","doi":"10.1038/s41584-025-01281-2","DOIUrl":"10.1038/s41584-025-01281-2","url":null,"abstract":"Familial Mediterranean fever (FMF) is a monogenic autoinflammatory disorder with high prevalence in Mediterranean populations. Considerable advances in the management of FMF have been made in the past decade, with respect to the use of biologic drugs and understanding colchicine resistance. The 2024 updated FMF management recommendations are timely and reflect these advances.","PeriodicalId":18810,"journal":{"name":"Nature Reviews Rheumatology","volume":"21 10","pages":"578-579"},"PeriodicalIF":32.7,"publicationDate":"2025-07-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144593920","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}
Pub Date : 2025-07-10DOI: 10.1038/s41584-025-01275-0
Csaba Matta, Roland Takács, Mona Dvir-Ginzberg, Stephen M. Richardson, Karoliina Pelttari, Girish Pattappa, Makarand V. Risbud, Ali Mobasheri
Chondrocyte biology is being revolutionized by single-cell multi-omics technologies, revealing cellular heterogeneity within cartilaginous tissues. Although past research has implicated cellular heterogeneity in chondrocyte populations, advances over the past decade in single-cell transcriptomics now enable a more granular, functionally annotated classification of chondrocyte subtypes. These analyses provide crucial insights into the role of these subtypes in cartilage formation, maintenance and disease progression. Chondrocyte populations are implicated in tissue homeostasis, pathogenesis and responses to external stimuli, including pro-inflammatory mediators and novel therapeutic agents. This knowledge opens pathways for developing targeted treatments for diseases such as osteoarthritis and intervertebral disc disease. Insights into the molecular signatures of disease-critical chondrocyte populations provide a foundation for biomarker discovery and therapeutic targeting, and there are exciting opportunities for leveraging these findings to progress regenerative therapies. Spatial and temporal profiling of cellular markers, behaviour and metabolic activity will enhance understanding of disease pathogenesis and chondrosenescence and could possibly enable early intervention for osteoarthritis, thereby preventing irreversible joint damage. Future research must integrate advanced single-cell techniques with computational modelling to unravel the dynamic interplay of chondrocyte populations. These efforts could transform precision medicine in rheumatology, addressing the unmet clinical needs in cartilage-related diseases. This Review provides an update on chondrocyte heterogeneity in cartilaginous tissues in health, disease and senescence focusing on insights gained from single-cell analyses. The authors highlight how single-cell multi-omics techniques could reveal new biomarkers and therapeutic targets for conditions such as osteoarthritis and intervertebral disc degeneration.
{"title":"Insights into chondrocyte populations in cartilaginous tissues at the single-cell level","authors":"Csaba Matta, Roland Takács, Mona Dvir-Ginzberg, Stephen M. Richardson, Karoliina Pelttari, Girish Pattappa, Makarand V. Risbud, Ali Mobasheri","doi":"10.1038/s41584-025-01275-0","DOIUrl":"10.1038/s41584-025-01275-0","url":null,"abstract":"Chondrocyte biology is being revolutionized by single-cell multi-omics technologies, revealing cellular heterogeneity within cartilaginous tissues. Although past research has implicated cellular heterogeneity in chondrocyte populations, advances over the past decade in single-cell transcriptomics now enable a more granular, functionally annotated classification of chondrocyte subtypes. These analyses provide crucial insights into the role of these subtypes in cartilage formation, maintenance and disease progression. Chondrocyte populations are implicated in tissue homeostasis, pathogenesis and responses to external stimuli, including pro-inflammatory mediators and novel therapeutic agents. This knowledge opens pathways for developing targeted treatments for diseases such as osteoarthritis and intervertebral disc disease. Insights into the molecular signatures of disease-critical chondrocyte populations provide a foundation for biomarker discovery and therapeutic targeting, and there are exciting opportunities for leveraging these findings to progress regenerative therapies. Spatial and temporal profiling of cellular markers, behaviour and metabolic activity will enhance understanding of disease pathogenesis and chondrosenescence and could possibly enable early intervention for osteoarthritis, thereby preventing irreversible joint damage. Future research must integrate advanced single-cell techniques with computational modelling to unravel the dynamic interplay of chondrocyte populations. These efforts could transform precision medicine in rheumatology, addressing the unmet clinical needs in cartilage-related diseases. This Review provides an update on chondrocyte heterogeneity in cartilaginous tissues in health, disease and senescence focusing on insights gained from single-cell analyses. The authors highlight how single-cell multi-omics techniques could reveal new biomarkers and therapeutic targets for conditions such as osteoarthritis and intervertebral disc degeneration.","PeriodicalId":18810,"journal":{"name":"Nature Reviews Rheumatology","volume":"21 8","pages":"465-477"},"PeriodicalIF":32.7,"publicationDate":"2025-07-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144594670","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}
Pub Date : 2025-07-08DOI: 10.1038/s41584-025-01284-z
Maria Papatriantafyllou
Skin- and joint-resident CD8+ T cells that share clonality also share phenotypic similarities in psoriatic arthritis.
共享克隆性的皮肤和关节驻留CD8+ T细胞在银屑病关节炎中也具有表型相似性。
{"title":"Tissue-resident memory CD8+ T cells on the skin–joint route","authors":"Maria Papatriantafyllou","doi":"10.1038/s41584-025-01284-z","DOIUrl":"10.1038/s41584-025-01284-z","url":null,"abstract":"Skin- and joint-resident CD8+ T cells that share clonality also share phenotypic similarities in psoriatic arthritis.","PeriodicalId":18810,"journal":{"name":"Nature Reviews Rheumatology","volume":"21 8","pages":"441-441"},"PeriodicalIF":32.7,"publicationDate":"2025-07-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144586417","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}
Pub Date : 2025-07-08DOI: 10.1038/s41584-025-01285-y
Maria Papatriantafyllou
The anti-fibrotic and anti-inflammatory agent nerandomilast has shown promise for the treatment of both idiopathic and progressive pulmonary fibrosis in phase III trials.
抗纤维化和抗炎药nerandomilast在III期试验中显示出治疗特发性和进行性肺纤维化的希望。
{"title":"Nerandomilast slows progression of pulmonary fibrosis","authors":"Maria Papatriantafyllou","doi":"10.1038/s41584-025-01285-y","DOIUrl":"10.1038/s41584-025-01285-y","url":null,"abstract":"The anti-fibrotic and anti-inflammatory agent nerandomilast has shown promise for the treatment of both idiopathic and progressive pulmonary fibrosis in phase III trials.","PeriodicalId":18810,"journal":{"name":"Nature Reviews Rheumatology","volume":"21 8","pages":"441-441"},"PeriodicalIF":32.7,"publicationDate":"2025-07-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144586418","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}
Pub Date : 2025-07-08DOI: 10.1038/s41584-025-01283-0
Holly Webster
A study provides insights into the factors that regulate synovial fibroblast interactions with endothelial cells in RA and subsequent pathogenic neovascularization.
一项研究提供了调节RA中滑膜成纤维细胞与内皮细胞相互作用以及随后的致病性新生血管形成的因素。
{"title":"Synovial fibroblast-mediated neovascularization in RA","authors":"Holly Webster","doi":"10.1038/s41584-025-01283-0","DOIUrl":"10.1038/s41584-025-01283-0","url":null,"abstract":"A study provides insights into the factors that regulate synovial fibroblast interactions with endothelial cells in RA and subsequent pathogenic neovascularization.","PeriodicalId":18810,"journal":{"name":"Nature Reviews Rheumatology","volume":"21 8","pages":"442-442"},"PeriodicalIF":32.7,"publicationDate":"2025-07-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144586416","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}
Pub Date : 2025-07-07DOI: 10.1038/s41584-025-01271-4
Hayley Peters, Jason S. Rockel, Christopher B. Little, Mohit Kapoor
The main homeostatic function of the synovial fluid is joint lubrication. However, during knee osteoarthritis (KOA), synovial fluid becomes modified with drivers of disease that contribute to symptoms (pain) and joint-related pathology. Acting as a sink of factors from both systemic circulation and local tissues, including articular cartilage, subchondral bone, synovium, and the infrapatellar fat pad, the synovial fluid enables bidirectional communication promoting KOA pathogenesis. Synovial fluid constituents might also be detected in circulation, functioning not only as accessible biomarkers but also as potential mediators of KOA-driven systemic effects. Factors deposited in synovial fluid have the ability to affect nervous system activity, acting at the neuronal projections that are integrated into joint tissues from dorsal root ganglia. Non-coding RNAs (microRNAs, long non-coding RNAs, circular RNAs), metabolites, cytokines and other secreted proteins of the synovial fluid in KOA have emerged as biomarkers of disease progression, therapeutic efficacy, and pain. These molecules might also function as molecular mediators of KOA, supporting them as candidates for therapeutic intervention. This review consolidates literature published primarily within the past 4 years, focussing on factors identified within synovial fluid as biomarkers and molecular mediators of KOA symptoms and pathology. Emerging therapeutic modalities to target synovial fluid molecular mediators are also discussed. The synovial fluid lubricates joints while also collecting molecular mediators from surrounding tissues. This Review highlights how molecular analyses of the synovial fluid might provide information on the progression of knee osteoarthritis and treatment efficacy, and identify potential therapeutic strategies targeting synovial fluid mediators in knee osteoarthritis.
{"title":"Synovial fluid as a complex molecular pool contributing to knee osteoarthritis","authors":"Hayley Peters, Jason S. Rockel, Christopher B. Little, Mohit Kapoor","doi":"10.1038/s41584-025-01271-4","DOIUrl":"10.1038/s41584-025-01271-4","url":null,"abstract":"The main homeostatic function of the synovial fluid is joint lubrication. However, during knee osteoarthritis (KOA), synovial fluid becomes modified with drivers of disease that contribute to symptoms (pain) and joint-related pathology. Acting as a sink of factors from both systemic circulation and local tissues, including articular cartilage, subchondral bone, synovium, and the infrapatellar fat pad, the synovial fluid enables bidirectional communication promoting KOA pathogenesis. Synovial fluid constituents might also be detected in circulation, functioning not only as accessible biomarkers but also as potential mediators of KOA-driven systemic effects. Factors deposited in synovial fluid have the ability to affect nervous system activity, acting at the neuronal projections that are integrated into joint tissues from dorsal root ganglia. Non-coding RNAs (microRNAs, long non-coding RNAs, circular RNAs), metabolites, cytokines and other secreted proteins of the synovial fluid in KOA have emerged as biomarkers of disease progression, therapeutic efficacy, and pain. These molecules might also function as molecular mediators of KOA, supporting them as candidates for therapeutic intervention. This review consolidates literature published primarily within the past 4 years, focussing on factors identified within synovial fluid as biomarkers and molecular mediators of KOA symptoms and pathology. Emerging therapeutic modalities to target synovial fluid molecular mediators are also discussed. The synovial fluid lubricates joints while also collecting molecular mediators from surrounding tissues. This Review highlights how molecular analyses of the synovial fluid might provide information on the progression of knee osteoarthritis and treatment efficacy, and identify potential therapeutic strategies targeting synovial fluid mediators in knee osteoarthritis.","PeriodicalId":18810,"journal":{"name":"Nature Reviews Rheumatology","volume":"21 8","pages":"447-464"},"PeriodicalIF":32.7,"publicationDate":"2025-07-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144568815","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}
Pub Date : 2025-07-07DOI: 10.1038/s41584-025-01276-z
Norma Maugeri, Angelo A. Manfredi
Platelets are central players in inflammatory and thrombotic responses that drive the onset and progression of rheumatic diseases. In particular, they regulate immunothrombosis, a defence mechanism in which the immune and blood-clotting systems cooperate to contain infections or vascular damage. Although immunothrombosis can help to preserve blood-vessel integrity and promote healing, it becomes harmful when exaggerated or chronic. In rheumatic diseases, such as systemic lupus erythematosus, systemic sclerosis and antiphospholipid syndrome, immunothrombosis contributes to persistent inflammation, abnormal blood-clot formation and long-term damage to the small blood vessels. It has also been implicated in maintaining autoimmune responses to autoantigens released by neutrophils. Platelets are among the first responders to vascular injury and influence the activity of immune cells, particularly neutrophils, by promoting the formation of neutrophil extracellular traps. Platelets express proteins such as P-selectin and the damage-associated molecule high-mobility group box 1 (HMGB1), which have distinct and non-redundant roles, both via direct interactions locally at sites of vascular damage and systemically via the release of extracellular vesicles. Understanding how platelets contribute to vascular inflammation and clotting in autoimmune settings elucidates disease mechanisms and might lead to the identification of new therapeutic targets. This Review provides an overview of how platelets promote immunothrombosis in rheumatic disease. The authors discuss the different ways in which platelets and immunothrombosis can be targeted for therapeutic intervention.
{"title":"Platelets as drivers of immunothrombosis in rheumatic diseases","authors":"Norma Maugeri, Angelo A. Manfredi","doi":"10.1038/s41584-025-01276-z","DOIUrl":"10.1038/s41584-025-01276-z","url":null,"abstract":"Platelets are central players in inflammatory and thrombotic responses that drive the onset and progression of rheumatic diseases. In particular, they regulate immunothrombosis, a defence mechanism in which the immune and blood-clotting systems cooperate to contain infections or vascular damage. Although immunothrombosis can help to preserve blood-vessel integrity and promote healing, it becomes harmful when exaggerated or chronic. In rheumatic diseases, such as systemic lupus erythematosus, systemic sclerosis and antiphospholipid syndrome, immunothrombosis contributes to persistent inflammation, abnormal blood-clot formation and long-term damage to the small blood vessels. It has also been implicated in maintaining autoimmune responses to autoantigens released by neutrophils. Platelets are among the first responders to vascular injury and influence the activity of immune cells, particularly neutrophils, by promoting the formation of neutrophil extracellular traps. Platelets express proteins such as P-selectin and the damage-associated molecule high-mobility group box 1 (HMGB1), which have distinct and non-redundant roles, both via direct interactions locally at sites of vascular damage and systemically via the release of extracellular vesicles. Understanding how platelets contribute to vascular inflammation and clotting in autoimmune settings elucidates disease mechanisms and might lead to the identification of new therapeutic targets. This Review provides an overview of how platelets promote immunothrombosis in rheumatic disease. The authors discuss the different ways in which platelets and immunothrombosis can be targeted for therapeutic intervention.","PeriodicalId":18810,"journal":{"name":"Nature Reviews Rheumatology","volume":"21 8","pages":"478-493"},"PeriodicalIF":32.7,"publicationDate":"2025-07-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144568816","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}
Pub Date : 2025-07-02DOI: 10.1038/s41584-025-01272-3
Holly Wobma, Stacy P. Ardoin, Challice L. Bonifant, Jennifer C. Cooper, Hanna Kim, Rebecca E. Sadun, Laura Lewandowski, Michael Keller, Robert A. Colbert, Cuoghi Edens, Kimberly DeQuattro, Kyla Driest, Julia Shalen, Ivana Stojkic, Andrea Knight, Colleen Annesley, Kathryn S. Torok, Caitlin W. Elgarten, Toshihiro Onishi, Shaun W. Jackson, Susan Prockop, Nirali N. Shah, Kaveh Ardalan, Margaret Lamb, on behalf of the Integrated Multidisciplinary Paediatric Autoimmunity and Cell Therapy (IMPACT) working group*
Initial success with B cell-targeted chimeric antigen receptor (CAR) T cells for the treatment of systemic lupus erythematosus and other rheumatic diseases has generated enthusiasm for the broad application of this technology outside of the field of oncology. Paediatric patients with severe rheumatic diseases require lifelong therapy with a substantial toxicity burden and a high cost of care. Paradigm-shifting treatments, including CAR T cells, are desperately needed. Although CAR T cell therapy shows promise for paediatric rheumatic diseases, there are unique aspects of care compared with adults, which require careful consideration and expertise. In response, we established the Integrated Multidisciplinary Paediatric Autoimmunity and Cell Therapy (IMPACT) working group, comprising international experts in the fields of paediatric rheumatology, oncology and cellular therapy, immunology and nephrology, to address the challenges of introducing cell therapies to patients with paediatric-onset autoimmune diseases. Given the possible benefits, we advocate for the study of CAR T cells in paediatric patients with rheumatic diseases who carry a lifelong risk of morbidity and mortality from chronic illness and medication toxicity. As this patient population is relatively small, consensus around definitions of success, robust study of predictors of response and uniform assessment and reporting of toxicities are critical to advancing the field. In this Perspective, the authors and the members of the Integrated Multidisciplinary Paediatric Autoimmunity and Cell Therapy (IMPACT) working group discuss specific considerations for the use of chimeric antigen receptor (CAR) T cell therapies in paediatric patients with rheumatic diseases.
{"title":"CAR T cell therapy for children with rheumatic disease: the time is now","authors":"Holly Wobma, Stacy P. Ardoin, Challice L. Bonifant, Jennifer C. Cooper, Hanna Kim, Rebecca E. Sadun, Laura Lewandowski, Michael Keller, Robert A. Colbert, Cuoghi Edens, Kimberly DeQuattro, Kyla Driest, Julia Shalen, Ivana Stojkic, Andrea Knight, Colleen Annesley, Kathryn S. Torok, Caitlin W. Elgarten, Toshihiro Onishi, Shaun W. Jackson, Susan Prockop, Nirali N. Shah, Kaveh Ardalan, Margaret Lamb, on behalf of the Integrated Multidisciplinary Paediatric Autoimmunity and Cell Therapy (IMPACT) working group*","doi":"10.1038/s41584-025-01272-3","DOIUrl":"10.1038/s41584-025-01272-3","url":null,"abstract":"Initial success with B cell-targeted chimeric antigen receptor (CAR) T cells for the treatment of systemic lupus erythematosus and other rheumatic diseases has generated enthusiasm for the broad application of this technology outside of the field of oncology. Paediatric patients with severe rheumatic diseases require lifelong therapy with a substantial toxicity burden and a high cost of care. Paradigm-shifting treatments, including CAR T cells, are desperately needed. Although CAR T cell therapy shows promise for paediatric rheumatic diseases, there are unique aspects of care compared with adults, which require careful consideration and expertise. In response, we established the Integrated Multidisciplinary Paediatric Autoimmunity and Cell Therapy (IMPACT) working group, comprising international experts in the fields of paediatric rheumatology, oncology and cellular therapy, immunology and nephrology, to address the challenges of introducing cell therapies to patients with paediatric-onset autoimmune diseases. Given the possible benefits, we advocate for the study of CAR T cells in paediatric patients with rheumatic diseases who carry a lifelong risk of morbidity and mortality from chronic illness and medication toxicity. As this patient population is relatively small, consensus around definitions of success, robust study of predictors of response and uniform assessment and reporting of toxicities are critical to advancing the field. In this Perspective, the authors and the members of the Integrated Multidisciplinary Paediatric Autoimmunity and Cell Therapy (IMPACT) working group discuss specific considerations for the use of chimeric antigen receptor (CAR) T cell therapies in paediatric patients with rheumatic diseases.","PeriodicalId":18810,"journal":{"name":"Nature Reviews Rheumatology","volume":"21 8","pages":"494-506"},"PeriodicalIF":32.7,"publicationDate":"2025-07-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144547240","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}
Pub Date : 2025-06-25DOI: 10.1038/s41584-025-01278-x
Adrian Ciurea, Caroline Ospelt
Over the past decades, considerable progress has been made in the pharmacological treatment of immune-mediated joint diseases such as rheumatoid arthritis and psoriatic arthritis; however, treatment-resistant arthritis remains a major clinical challenge. To tackle insufficient treatment outcomes, further research into the underlying mechanisms and patterns of non-responsiveness is needed.
{"title":"Location-specific treatment of chronic inflammatory joint disease","authors":"Adrian Ciurea, Caroline Ospelt","doi":"10.1038/s41584-025-01278-x","DOIUrl":"10.1038/s41584-025-01278-x","url":null,"abstract":"Over the past decades, considerable progress has been made in the pharmacological treatment of immune-mediated joint diseases such as rheumatoid arthritis and psoriatic arthritis; however, treatment-resistant arthritis remains a major clinical challenge. To tackle insufficient treatment outcomes, further research into the underlying mechanisms and patterns of non-responsiveness is needed.","PeriodicalId":18810,"journal":{"name":"Nature Reviews Rheumatology","volume":"21 9","pages":"507-508"},"PeriodicalIF":32.7,"publicationDate":"2025-06-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144479043","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}
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