Lancet Rheumatology最新文献

{"title":"Vascular Ehlers-Danlos syndrome: a heritable condition with potentially fatal consequences.","authors":"Emily Woods, Jessica M Bowen, Diana S Johnson, Glenda J Sobey","doi":"10.1016/S2665-9913(25)00128-6","DOIUrl":"https://doi.org/10.1016/S2665-9913(25)00128-6","url":null,"abstract":"","PeriodicalId":48540,"journal":{"name":"Lancet Rheumatology","volume":" ","pages":""},"PeriodicalIF":15.0,"publicationDate":"2025-05-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144086892","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}

{"title":"Patient-reported outcomes: a new basis for prediction models?","authors":"Tanja Stamm , Preston Long , Valentin Ritschl , Erika Mosor , Peter Mandl","doi":"10.1016/S2665-9913(25)00066-9","DOIUrl":"10.1016/S2665-9913(25)00066-9","url":null,"abstract":"","PeriodicalId":48540,"journal":{"name":"Lancet Rheumatology","volume":"7 7","pages":"Pages e455-e457"},"PeriodicalIF":15.0,"publicationDate":"2025-05-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144053440","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}

{"title":"Long-term effect of anifrolumab on patient-reported outcomes in systemic lupus erythematosus (TULIP-LTE): a randomised, placebo-controlled, phase 3 long-term extension trial","authors":"Prof Vibeke Strand MD ,&nbsp;Prof Kenneth C Kalunian MD ,&nbsp;Kai Wai Lee MSc ,&nbsp;Caroline Seo BS ,&nbsp;Gabriel Abreu PhD ,&nbsp;Raj Tummala MD ,&nbsp;Hussein Al-Mossawi MD ,&nbsp;Elizabeth A Duncan MD ,&nbsp;Catharina Lindholm MD","doi":"10.1016/S2665-9913(25)00022-0","DOIUrl":"10.1016/S2665-9913(25)00022-0","url":null,"abstract":"<div><h3>Background</h3><div>In the TULIP-1 and TULIP-2 phase 3 trials, anifrolumab treatment was associated with clinical efficacy and clinically meaningful improvements in multiple patient-reported outcomes over 52 weeks in patients with moderate-to-severe systemic lupus erythematosus (SLE). At the end of TULIP-1 and TULIP-2 (week 52), eligible patients could reconsent to enter a 3-year long-term extension trial (TULIP-LTE). Here, we investigated changes in patient-reported outcomes during treatment with anifrolumab or placebo for up to 4 years in patients with SLE who were receiving standard therapy.</div></div><div><h3>Methods</h3><div>TULIP-LTE was a 3-year randomised, double-blind, placebo-controlled, phase 3 long-term extension of the 1-year TULIP-1 and TULIP-2 trials. Patients who were randomly assigned to receive anifrolumab 300 mg (n=257) or placebo (n=112) every 4 weeks in the TULIP-1 and TULIP-2 trials and who continued the same treatment in TULIP-LTE were assessed. Exploratory endpoints were changes from baseline in patient-reported outcomes (Short Form-36 version 2 acute recall [SF-36v2 (acute)], Patient Global Assessment of disease activity, EuroQoL 5 Dimensions-5 Levels [EQ-5D-5L], and Work Productivity and Activity Impairment-Lupus) and health utility indices, Short-Form 6-Dimension (SF-6D), derived from SF-36v2 (acute), and EQ-5D-5L. All analyses were done in the modified intention-to-treat population, which included all randomly assigned patients from TULIP-1 and TULIP-2 who received at least one dose of the same treatment during the long-term extension phase. Least-squares mean patient-reported outcome scores were adjusted using a repeated measures model. No people with lived experience were directly involved in the study design or implementation. This trial was registered with <span><span>ClinicalTrials.gov</span><svg><path></path></svg></span>, <span><span>NCT02794285</span><svg><path></path></svg></span>.</div></div><div><h3>Findings</h3><div>Between June 30, 2016, and Oct 12, 2018, 369 patients entered the long-term extension study and were included in this exploratory analysis (257 in the anifrolumab group and 112 in the placebo group). Mean age of patients was 42·8 years (SD 11·5), 340 (92%) of 369 patients were women and 29 (8%) were men, 250 (68%) patients were White and 82 (22%) self-identified as Hispanic or Latino. Patient-reported outcome scores improved from baseline in both groups during the 4-year treatment period. At week 208, improvement from baseline in SF-36v2 (acute) was numerically higher in the anifrolumab group than the placebo group for the bodily pain domain (least-squares mean difference 5·9 [95% CI −0·7 to 12·5]) and the mental health domain (3·7 [−1·2 to 8·6]). Improvements from baseline in SF-6D were generally greater in the anifrolumab group than the placebo group, with numerical differences between groups evident from as early as week 24 (least-squares mean difference 0·013 [−0·007 to 0·032]) and","PeriodicalId":48540,"journal":{"name":"Lancet Rheumatology","volume":"7 7","pages":"Pages e485-e494"},"PeriodicalIF":15.0,"publicationDate":"2025-05-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144054713","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}

{"title":"Local immune effector cell-associated toxicity syndrome in CAR T-cell treated patients with autoimmune disease: an observational study","authors":"Melanie Hagen MD ,&nbsp;Prof Fabian Müller MD ,&nbsp;Andreas Wirsching MD ,&nbsp;Soraya Kharboutli MD ,&nbsp;Prof Silvia Spoerl MD ,&nbsp;Christina Düsing MD ,&nbsp;Tobias Krickau MD ,&nbsp;Prof Markus Metzler MD ,&nbsp;Simon Völkl PhD ,&nbsp;Michael Aigner MD ,&nbsp;Sascha Kretschmann PhD ,&nbsp;Ingrid Vasova MD ,&nbsp;Prof Marc Saake MD ,&nbsp;Stefan Schliep MD ,&nbsp;Torsten Kubacki MD ,&nbsp;Prof Nicolas Hunzelmann MD ,&nbsp;Laura Bucci MD ,&nbsp;Jule Taubmann MD ,&nbsp;Christina Bergmann MD ,&nbsp;Andrea-Hermina Györfi MD ,&nbsp;Prof Georg Schett MD","doi":"10.1016/S2665-9913(25)00091-8","DOIUrl":"10.1016/S2665-9913(25)00091-8","url":null,"abstract":"<div><h3>Background</h3><div>CD19-targeting chimeric antigen receptor (CAR) T-cell therapy has advanced treatment strategies for severe autoimmune diseases such as systemic lupus erythematosus (SLE), systemic sclerosis, and idiopathic inflammatory myopathy. Data regarding side-effects are mostly generated from patients with malignancies, but little is known about autoimmune disease-specific adverse events. This study aimed to describe autoimmune disease-specific adverse events that occur with CAR T-cell therapy.</div></div><div><h3>Methods</h3><div>In this observational study, patients of any age with autoimmune disease receiving CD19-targeting CAR T-cell therapy in two centres in Germany with a follow-up of at least 30 days were assessed for local organ-specific reactions occurring after CAR T-cell infusion. Observed reactions were documented according to localisation, time of onset, and duration, and were graded for severity (grade 1: spontaneous resolution; grade 2: glucocorticoid treatment due to symptoms lasting &gt;1 week or presence of relevant inner organ involvement; grade 3: prolonged or new hospitalisation; grade 4: intensive care treatment). People with related lived experience were involved in the study design and implementation.</div></div><div><h3>Findings</h3><div>Between March 1, 2021, and Oct 31, 2024, 39 patients with autoimmune disease were treated with CD19-targeting CAR T cells (20 with SLE, 13 with systemic sclerosis, six with idiopathic inflammatory myopathy). 25 (64%) patients were female and 14 (36%) were male. Median age was 36 years (IQR 22–44). 54 local reactions, which we termed local immune effector cell-associated toxicity syndrome (LICATS), were recorded, affecting 30 (77%) patients with a median time of onset of 10 days (IQR 9–21) from CAR T-cell infusion and a median duration of 11 days (5–14). LICATS exclusively occurred during the B-cell aplasia phase and only involved organs previously affected by the respective autoimmune disease. The most frequently affected organs were the skin (19 [35%] of 54) and the kidneys (12 [22%]). Most cases of LICATS were mild (grade 1: 35 [65%]; grade 2: 16 [30%]). Only three cases were grade 3. All events of LICATS resolved without sequelae.</div></div><div><h3>Interpretation</h3><div>LICATS is a new form of toxicity in patients with autoimmune disease receiving CD19-targeting CAR T-cell therapy, most likely based on the cleansing of immune cells from the affected organs. It is self-limited, organ-specific, and usually mild in its intensity.</div></div><div><h3>Funding</h3><div>Deutsche Forschungsgemeinschaft (DFG), German Cancer Aid, Bundesministerium für Bildung und Forschung, European Union, Staedtler Foundation, Lupus Research Alliance, and donations from the Bendel family and the Bleyl family.</div></div>","PeriodicalId":48540,"journal":{"name":"Lancet Rheumatology","volume":"7 6","pages":"Pages e424-e433"},"PeriodicalIF":15.0,"publicationDate":"2025-04-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144006991","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}

{"title":"A new toxicity syndrome in patients with autoimmune disease treated with CAR T-cell therapy","authors":"Samuel D Good ,&nbsp;Elizabeth R Volkmann","doi":"10.1016/S2665-9913(25)00100-6","DOIUrl":"10.1016/S2665-9913(25)00100-6","url":null,"abstract":"","PeriodicalId":48540,"journal":{"name":"Lancet Rheumatology","volume":"7 6","pages":"Pages e382-e384"},"PeriodicalIF":15.0,"publicationDate":"2025-04-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144054859","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}

{"title":"Laura Coates: all about the people","authors":"Heather Van Epps","doi":"10.1016/S2665-9913(25)00107-9","DOIUrl":"10.1016/S2665-9913(25)00107-9","url":null,"abstract":"","PeriodicalId":48540,"journal":{"name":"Lancet Rheumatology","volume":"7 5","pages":"Page e313"},"PeriodicalIF":15.0,"publicationDate":"2025-04-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143873444","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}

{"title":"Research in Brief","authors":"Jennifer Thorley","doi":"10.1016/S2665-9913(25)00106-7","DOIUrl":"10.1016/S2665-9913(25)00106-7","url":null,"abstract":"","PeriodicalId":48540,"journal":{"name":"Lancet Rheumatology","volume":"7 5","pages":"Page e312"},"PeriodicalIF":15.0,"publicationDate":"2025-04-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143873443","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}

{"title":"A cold case of joint injury.","authors":"Clément Triaille, Marie-Claude Miron, Patricia Egerszegi, Jean Jacques De Bruycker","doi":"10.1016/S2665-9913(25)00078-5","DOIUrl":"https://doi.org/10.1016/S2665-9913(25)00078-5","url":null,"abstract":"","PeriodicalId":48540,"journal":{"name":"Lancet Rheumatology","volume":" ","pages":""},"PeriodicalIF":15.0,"publicationDate":"2025-04-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144035207","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}

{"title":"Tumour necrosis factor inhibitor safety in rheumatoid arthritis with early-stage cancer","authors":"Jeffrey A Sparks ,&nbsp;Namrata Singh","doi":"10.1016/S2665-9913(25)00099-2","DOIUrl":"10.1016/S2665-9913(25)00099-2","url":null,"abstract":"","PeriodicalId":48540,"journal":{"name":"Lancet Rheumatology","volume":"7 5","pages":"Pages e307-e308"},"PeriodicalIF":15.0,"publicationDate":"2025-04-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143873439","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}

{"title":"Reasoning large language models in rheumatology: a call for responsible action","authors":"Vincenzo Venerito ,&nbsp;Florenzo Iannone ,&nbsp;Latika Gupta","doi":"10.1016/S2665-9913(25)00101-8","DOIUrl":"10.1016/S2665-9913(25)00101-8","url":null,"abstract":"","PeriodicalId":48540,"journal":{"name":"Lancet Rheumatology","volume":"7 6","pages":"Pages e384-e386"},"PeriodicalIF":15.0,"publicationDate":"2025-04-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144025377","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}