Journal of Neurology最新文献

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Familial myasthenia gravis: characterization of an Israeli cohort and systematic review of the literature. 家族性重症肌无力:以色列队列的特征和文献的系统回顾。
IF 4.8 2区 医学
Journal of Neurology Pub Date : 2025-07-10 DOI: 10.1007/s00415-025-13236-4
Mark A Hellmann, Israel Steiner, Maor Mermelstein, Itzhak Friedman, Adi Wilf-Yarkoni, Itay Lotan
{"title":"Familial myasthenia gravis: characterization of an Israeli cohort and systematic review of the literature.","authors":"Mark A Hellmann, Israel Steiner, Maor Mermelstein, Itzhak Friedman, Adi Wilf-Yarkoni, Itay Lotan","doi":"10.1007/s00415-025-13236-4","DOIUrl":"https://doi.org/10.1007/s00415-025-13236-4","url":null,"abstract":"<p><strong>Background: </strong>Myasthenia gravis (MG) is an autoimmune disorder of the neuromuscular junction, most commonly associated with autoantibodies against the acetylcholine receptor (AChR). While familial clustering of autoimmune MG (fMG) has been described, its prevalence and clinical characteristics remain uncertain. This study aimed to characterize autoimmune fMG cases in an Israeli cohort and describe global data through a systematic literature review.</p><p><strong>Methods: </strong>We retrospectively analyzed the medical records of MG patients treated at Rabin Medical Center from 2000 to 2022. The clinical and demographic features of autoimmune fMG cases-defined by compatible clinical, serological, and electrophysiological features-were compared to those of sporadic MG. Additionally, a systematic review of published autoimmune fMG cases was performed according to PRISMA guidelines.</p><p><strong>Results: </strong>Among 281 MG patients, 16 patients (5.7%) from eight families met criteria for autoimmune fMG. Compared to sporadic MG, fMG cases had a significantly younger age of onset (median 44.5 vs. 58 years, p = 0.04) and more frequently presented with severe generalized disease (MGFA class IV-V, 43.8% vs. 15%, p = 0.008). The antibody profiles, sex distribution, ocular involvement, and comorbid autoimmune diseases did not differ significantly. All fMG patients responded to immunotherapy. The systematic review identified 73 additional fMG cases, with similar trends toward earlier onset and generalized presentation. Patients with fMG in our cohort had higher rates of severe initial presentation (43.8% vs. 16.4%, p = 0.03) and underwent thymectomy less frequently (19% vs. 49.3%, p = 0.03).</p><p><strong>Conclusions: </strong>Autoimmune familial MG occurs in 5-6% of MG cases and is associated with a younger onset and more severe initial presentation compared to sporadic MG, but shows similar long-term treatment response. These findings suggest that genetic factors may contribute to disease susceptibility and phenotypic expression in familial MG, highlighting the need for further research into the underlying genetic and immunological mechanisms.</p>","PeriodicalId":16558,"journal":{"name":"Journal of Neurology","volume":"272 8","pages":"498"},"PeriodicalIF":4.8,"publicationDate":"2025-07-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144600738","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"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
Temporal arteritis and vasculitic myopathy in polyarteritis nodosa. 结节性多动脉炎的颞动脉炎和血管性肌病。
IF 4.8 2区 医学
Journal of Neurology Pub Date : 2025-07-09 DOI: 10.1007/s00415-025-13199-6
Nikolas Ruffer, Isabell Haase, Tingting Xiong, Florian Prinz, Daniel Koehler, Ina Kötter, Martin Krusche
{"title":"Temporal arteritis and vasculitic myopathy in polyarteritis nodosa.","authors":"Nikolas Ruffer, Isabell Haase, Tingting Xiong, Florian Prinz, Daniel Koehler, Ina Kötter, Martin Krusche","doi":"10.1007/s00415-025-13199-6","DOIUrl":"10.1007/s00415-025-13199-6","url":null,"abstract":"","PeriodicalId":16558,"journal":{"name":"Journal of Neurology","volume":"272 8","pages":"496"},"PeriodicalIF":4.8,"publicationDate":"2025-07-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12241109/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144591478","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Théophile Alajouanine (1890-1980). Theophile Alajouanine(1890-1980)。
IF 4.8 2区 医学
Journal of Neurology Pub Date : 2025-07-09 DOI: 10.1007/s00415-025-13240-8
Bruno Kusznir Vitturi
{"title":"Théophile Alajouanine (1890-1980).","authors":"Bruno Kusznir Vitturi","doi":"10.1007/s00415-025-13240-8","DOIUrl":"10.1007/s00415-025-13240-8","url":null,"abstract":"","PeriodicalId":16558,"journal":{"name":"Journal of Neurology","volume":"272 8","pages":"497"},"PeriodicalIF":4.8,"publicationDate":"2025-07-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12241222/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144591479","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Clinical application of age-derived cut-offs for plasma neurofilament light chain in multiple sclerosis. 年龄源性血浆神经丝轻链切断在多发性硬化中的临床应用。
IF 4.8 2区 医学
Journal of Neurology Pub Date : 2025-07-09 DOI: 10.1007/s00415-025-13223-9
Valerio Nicolella, Marco Varelli, Stefania Fasano, Rosa Sirica, Carmela Polito, Aniello Saviano, Mariano Fiorenza, Federica Novarella, Davide Ranucci, Antonio Carotenuto, Maria Petracca, Roberta Lanzillo, Vincenzo Brescia Morra, Giuseppe Castaldo, Daniela Terracciano, Marcello Moccia
{"title":"Clinical application of age-derived cut-offs for plasma neurofilament light chain in multiple sclerosis.","authors":"Valerio Nicolella, Marco Varelli, Stefania Fasano, Rosa Sirica, Carmela Polito, Aniello Saviano, Mariano Fiorenza, Federica Novarella, Davide Ranucci, Antonio Carotenuto, Maria Petracca, Roberta Lanzillo, Vincenzo Brescia Morra, Giuseppe Castaldo, Daniela Terracciano, Marcello Moccia","doi":"10.1007/s00415-025-13223-9","DOIUrl":"https://doi.org/10.1007/s00415-025-13223-9","url":null,"abstract":"<p><strong>Objective: </strong>Clinical use of neurofilament light chain (NfL) requires cut-off values that reflect disease status independently of confounding factors, such as age, hemodilution, and cardiovascular risk factors. We compared the performance of different previously suggested cut-offs in separating MS cases and controls, and in identifying different MS clinical features, across age groups.</p><p><strong>Methods: </strong>In this cross-sectional study, we included people with MS (n = 312) and age-, sex-, and eGFR-matched controls (n = 236). For MS cases, we collected descriptor of disease progression (relapsing or progressive), EDSS, and evidence of disease activity in the previous year (including relapses, active MRI, and EDSS progression) and disease duration. Plasma NfL (pNfL) was evaluated using Lumipulse™ fully automated chemiluminescent enzyme immunoassay. We then classified both MS cases and controls based on pNfL suggested by Simrèn et al. (specific for different age ranges), Vermunt et al. (age-derived percentiles), and Benkert et al. (age- and BMI-derived percentiles).</p><p><strong>Results: </strong>In individuals aged 18-50 years, the three suggested pNfL cut-offs provided high specificity (> 85%) in discriminating MS cases and controls (AUC = 0.73; 95%CI = 0.67, 0.78; p = 0.028). In the MS population, the three suggested pNfL cut-offs provided high sensitivity (> 75%) in discriminating relapsing and progressive cases (AUC = 0.70; 95%CI = 0.63, 0.77; p = 0.034), patients with EDSS ≥ 4.0 and EDSS < 4.0 (AUC = 0.69; 95%CI = 0.63, 0.76; p = 0.032), and patients with EDSS ≥ 6.0 and EDSS < 6.0 (AUC = 0.70; 95%CI = 0.62, 0.78; p = 0.040). The three suggested pNfL cut-offs provided lower accuracy in age groups older than 50 years.</p><p><strong>Conclusions: </strong>Previously validated cut-offs provided similar sensitivity and specificity in separating MS cases and controls and in identifying MS clinical features across different age groups, with the best performance before 50 years.</p>","PeriodicalId":16558,"journal":{"name":"Journal of Neurology","volume":"272 8","pages":"495"},"PeriodicalIF":4.8,"publicationDate":"2025-07-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144591477","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"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
Central nervous system involvement and mimickers in ANCA associated vasculitis. ANCA相关血管炎的中枢神经系统受累及拟态物。
IF 4.8 2区 医学
Journal of Neurology Pub Date : 2025-07-08 DOI: 10.1007/s00415-025-13203-z
Yeliz Yagiz Ozogul, Sinem Nihal Esatoglu, Murat Ozogul, Osman Kizilkilic, Yesim Ozguler, Emire Seyahi, Serdal Ugurlu, Melike Melikoglu, Izzet Fresko, Vedat Hamuryudan, Ugur Uygunoglu, Gulen Hatemi
{"title":"Central nervous system involvement and mimickers in ANCA associated vasculitis.","authors":"Yeliz Yagiz Ozogul, Sinem Nihal Esatoglu, Murat Ozogul, Osman Kizilkilic, Yesim Ozguler, Emire Seyahi, Serdal Ugurlu, Melike Melikoglu, Izzet Fresko, Vedat Hamuryudan, Ugur Uygunoglu, Gulen Hatemi","doi":"10.1007/s00415-025-13203-z","DOIUrl":"10.1007/s00415-025-13203-z","url":null,"abstract":"<p><strong>Objective: </strong>Central nervous system (CNS) involvement is rare in ANCA-associated vasculitis (AAV). On the other hand, AAV patients may develop complications or other conditions that mimic CNS involvement. We aimed to present the clinical, laboratory and imaging features of our AAV patients with CNS involvement and conditions other than CNS involvement that caused neurologic symptoms.</p><p><strong>Method: </strong>We surveyed the charts of 430 AAV patients in order to identify patients who were evaluated for neurologic symptoms suggesting CNS involvement. We extracted data on their demographics, AAV features, neurologic symptoms, final diagnoses after neurologic work-up, and their outcomes.</p><p><strong>Results: </strong>Of 430 AAV patients, 61 patients (14%) (41 GPA, 11 MPA, 9 EGPA; 27 women, 34 men; mean age: 51.6 ± 15.4 years) with neurologic symptoms were identified. At the time of the occurrence of neurologic symptoms, all patients had active disease [median (IQR) BVAS=11.9 (7-15)]. The causes of neurologic symptoms were CNS involvement of AAV in 7 patients (meningeal involvement in 3, ischemic cerebrovascular accident in 2, intracranial hypertension in 1, and cerebral venous sinus thrombosis in 1), other AAV manifestations such as ocular, orbital and nasopharyngeal involvement in 30, and drug-related adverse events or comorbidities mimicking CNS involvement such as infections, atherosclerotic or thromboembolic events in 15 patients. Neurologic work-up did not lead to an underlying condition in 9.</p><p><strong>Conclusion: </strong>CNS involvement was uncommon, observed in only 1.6% of patients. AAV manifestations other than CNS involvement, as well as complications like infections and cardiovascular disease may mimic CNS involvement in patients with AAV.</p>","PeriodicalId":16558,"journal":{"name":"Journal of Neurology","volume":"272 8","pages":"492"},"PeriodicalIF":4.8,"publicationDate":"2025-07-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12238188/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144584198","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Vertigo and dizziness in genetic neurodevelopmental disorders: an international cross-sectional study. 遗传性神经发育障碍中的眩晕和头晕:一项国际横断面研究。
IF 4.8 2区 医学
Journal of Neurology Pub Date : 2025-07-08 DOI: 10.1007/s00415-025-13237-3
Christophe Lopez, Pauline Burger, Jean-Louis Mandel, Romain Coutelle
{"title":"Vertigo and dizziness in genetic neurodevelopmental disorders: an international cross-sectional study.","authors":"Christophe Lopez, Pauline Burger, Jean-Louis Mandel, Romain Coutelle","doi":"10.1007/s00415-025-13237-3","DOIUrl":"https://doi.org/10.1007/s00415-025-13237-3","url":null,"abstract":"<p><strong>Background: </strong>Neurodevelopmental disorders (NDDs) with a genetic basis are frequently associated with neurological and sensory impairments; yet vestibular symptoms remain underrecognized. Vertigo, dizziness, and imbalance can significantly impact daily functioning, but their prevalence and genetic associations in individuals with NDDs are poorly understood. This study aimed to assess the occurrence of vestibular symptoms in individuals with genetic NDDs.</p><p><strong>Methods: </strong>We conducted an international, prospective cross-sectional study using data from the GenIDA database, which includes individuals with confirmed genetic disorders, intellectual developmental disorders and/or other NDDs. Caregivers completed a survey assessing the occurrence of vertigo, dizziness, and balance issues, as well as emotional distress, psychiatric comorbidities, and associated conditions such as migraine and epilepsy. We analyzed the prevalence of vertigo in the past 12 months and explored potential genetic contributors.</p><p><strong>Results: </strong>The occurrence rate of vertigo (11.8%) and imbalance (27.7%) in the past 12 months was dramatically increased in individuals with genetic NDDs compared to that observed in the general population. However, less than 40% of patients had received a formal diagnosis for their vertigo. Notably, vertigo was significantly associated with an increased likelihood of feeling unhappy and depressed. Twenty genetic anomalies causally involved in NDDs were identified as potential contributors to vertigo, including 22q11.2 microdeletions and variants in ANKRD11, MED13L, PACS1 and SHANK3.</p><p><strong>Discussion: </strong>Vertigo appears highly prevalent, yet underdiagnosed, in individuals with genetic NDDs. The identified genetic anomalies suggest that (1) vertigo may be an intrinsic component of NDDs, reflecting shared genetic pathways leading to inner ear malformations, vestibular network dysfunction, cerebellar abnormalities, and epilepsy, alongside other neurodevelopmental features; and/or (2) early vestibular dysfunction may exacerbate neurodevelopmental impairments.</p>","PeriodicalId":16558,"journal":{"name":"Journal of Neurology","volume":"272 8","pages":"494"},"PeriodicalIF":4.8,"publicationDate":"2025-07-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144584200","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"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
Randomised feasibility study evaluating eye movement desensitisation and reprocessing therapy for functional neurological disorder (MODIFI). 评估眼动脱敏和再加工治疗功能性神经障碍(MODIFI)的随机可行性研究。
IF 4.8 2区 医学
Journal of Neurology Pub Date : 2025-07-08 DOI: 10.1007/s00415-025-13219-5
Sarah R Cope, Jared G Smith, Sharif El-Leithy, Serena Vanzan, Patricia Hogwood, Dawn Golder, Kati Jane Turner, Maeve Crowley, Jo Billings, Susannah Pick, Caitlin Pentland, Mark J Edwards
{"title":"Randomised feasibility study evaluating eye movement desensitisation and reprocessing therapy for functional neurological disorder (MODIFI).","authors":"Sarah R Cope, Jared G Smith, Sharif El-Leithy, Serena Vanzan, Patricia Hogwood, Dawn Golder, Kati Jane Turner, Maeve Crowley, Jo Billings, Susannah Pick, Caitlin Pentland, Mark J Edwards","doi":"10.1007/s00415-025-13219-5","DOIUrl":"10.1007/s00415-025-13219-5","url":null,"abstract":"<p><strong>Background: </strong>Functional neurological disorder (FND) is a common neurological presentation with symptoms such as seizures, walking difficulties, limb weakness and cognitive difficulties. Treatments for FND include physiotherapy and psychological therapy. Eye movement desensitisation and reprocessing therapy (EMDR) is a therapy designed to reduce disturbance associated with distressing or traumatic memories. Case report evidence suggests possible benefit for people with FND. This randomised feasibility study aimed to assess whether a large-scale trial evaluating EMDR for FND would be feasible and acceptable.</p><p><strong>Methods: </strong>Fifty participants with FND were randomised to either FND-focused EMDR plus standard neuropsychiatric care (NPC) or NPC alone. Feasibility criteria were recruitment rate, intervention adherence, and outcome measure completion. Assessment of safety was also examined, as well as therapy satisfaction. Participants completed questionnaires at baseline, 3 months, 6 months and 9 months. FND symptoms were assessed using Ecological Momentary Assessment at each time point.</p><p><strong>Results: </strong>Recruitment rate was 58%, intervention adherence was 88%, and outcome measure completion was 68% for Ecological Momentary Assessment and 76% for questionnaires at 9-month follow-up. Participants experienced functional motor symptoms (80%), functional seizures (64%), and cognitive symptoms (32%). Participants receiving EMDR + NPC reported greater satisfaction and greater FND improvement compared to NPC. Questionnaire data suggested greater reductions in PTSD, depression, anxiety, dissociation, disability and healthcare-use for EMDR + NPC.</p><p><strong>Discussion: </strong>The study demonstrated that an FND-specific protocol for EMDR was feasible and acceptable. Potential positive effects on FND symptoms, mental health, disability, and healthcare utilisation were found. A full-scale trial is warranted to establish efficacy.</p><p><strong>Trial registration: </strong>NCT05455450 ( www.</p><p><strong>Clinicaltrials: </strong>gov ).</p>","PeriodicalId":16558,"journal":{"name":"Journal of Neurology","volume":"272 8","pages":"493"},"PeriodicalIF":4.8,"publicationDate":"2025-07-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12238153/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144584199","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Therapeutic strategies targeting complement in myasthenia gravis patients. 重症肌无力患者补体靶向治疗策略。
IF 4.8 2区 医学
Journal of Neurology Pub Date : 2025-07-02 DOI: 10.1007/s00415-025-13225-7
Yinxiang Wang, Jianfei Nao, Yingjie Duan, Zijian Li, Juan Feng
{"title":"Therapeutic strategies targeting complement in myasthenia gravis patients.","authors":"Yinxiang Wang, Jianfei Nao, Yingjie Duan, Zijian Li, Juan Feng","doi":"10.1007/s00415-025-13225-7","DOIUrl":"10.1007/s00415-025-13225-7","url":null,"abstract":"<p><p>Myasthenia gravis (MG) is an acquired autoimmune disorder characterized by impaired neuromuscular junction transmission, leading to fluctuating muscle weakness. With the progressive identification of MG-related antibodies, such as acetylcholine receptor (AChR) antibodies and muscle-specific kinase (MuSK) antibodies, coupled with advancements in antibody detection technology, the use of these diagnostic markers has become widely accessible in clinical practice. This has facilitated the detection of key biomarkers and enabled the study of targeted therapeutic interventions aimed at addressing the underlying production of MG pathogenic antibodies and their various stages of pathogenicity. Among the emerging therapeutic strategies, complement-targeting drugs have garnered significant attention from medical researchers because of their ability to reduce complement activation and inhibit autoimmune-mediated tissue damage. Inhibition of the terminal complement cascade has demonstrated efficacy in reducing disease severity and improving clinical outcomes, particularly in refractory cases of MG. This review summarizes the underlying mechanisms of complement activation in MG, evaluates the current therapeutic landscape, including both approved and investigational complement inhibitors and discusses the safety considerations of their application.</p>","PeriodicalId":16558,"journal":{"name":"Journal of Neurology","volume":"272 8","pages":"489"},"PeriodicalIF":4.8,"publicationDate":"2025-07-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12222297/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144540582","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Comparative effectiveness of teriflunomide and ocrelizumab on smoldering activity in multiple sclerosis: an observational study in the Swiss Multiple Sclerosis Cohort. teri氟米特和ocrelizumab对多发性硬化症患者闷烧活性的比较疗效:一项瑞士多发性硬化症队列观察性研究
IF 4.8 2区 医学
Journal of Neurology Pub Date : 2025-07-02 DOI: 10.1007/s00415-025-13221-x
Alessandro Cagol, Sabine Schaedelin, Mario Ocampo-Pineda, Pascal Benkert, Lester Melie-Garcia, Ludovico Luchetti, Özgür Yaldizli, Johanna Oechtering, Marcus D'Souza, Bettina Fischer-Barnicol, Stefanie Müller, Sebastian Finkener, Jochen Vehoff, Giulio Disanto, Andrew Chan, Caroline Pot, Chiara Zecca, Tobias Derfuss, Johanna M Lieb, Michael Diepers, Franca Wagner, Renaud Du Pasquier, Patrice H Lalive, Emanuele Pravatà, Olaf Chan-Hi Kim, Robert Hoepner, Patrick Roth, Claudio Gobbi, David Leppert, Marco Battaglini, Ludwig Kappos, Maria Pia Sormani, Jens Kuhle, Cristina Granziera
{"title":"Comparative effectiveness of teriflunomide and ocrelizumab on smoldering activity in multiple sclerosis: an observational study in the Swiss Multiple Sclerosis Cohort.","authors":"Alessandro Cagol, Sabine Schaedelin, Mario Ocampo-Pineda, Pascal Benkert, Lester Melie-Garcia, Ludovico Luchetti, Özgür Yaldizli, Johanna Oechtering, Marcus D'Souza, Bettina Fischer-Barnicol, Stefanie Müller, Sebastian Finkener, Jochen Vehoff, Giulio Disanto, Andrew Chan, Caroline Pot, Chiara Zecca, Tobias Derfuss, Johanna M Lieb, Michael Diepers, Franca Wagner, Renaud Du Pasquier, Patrice H Lalive, Emanuele Pravatà, Olaf Chan-Hi Kim, Robert Hoepner, Patrick Roth, Claudio Gobbi, David Leppert, Marco Battaglini, Ludwig Kappos, Maria Pia Sormani, Jens Kuhle, Cristina Granziera","doi":"10.1007/s00415-025-13221-x","DOIUrl":"10.1007/s00415-025-13221-x","url":null,"abstract":"<p><strong>Background: </strong>This study aimed to compare the effects of teriflunomide and ocrelizumab on clinical and MRI endpoints related to smoldering activity in relapsing-remitting multiple sclerosis (RRMS).</p><p><strong>Methods: </strong>In this observational, longitudinal, multicenter study, we included 128 people with RRMS (pwRRMS) treated with teriflunomide and 495 treated with ocrelizumab. Outcomes included time to progression independent of relapse activity (PIRA). In a subset, we also assessed brain volume loss (BVL), longitudinal changes in diffusion tensor imaging (DTI) metrics, and the burden of paramagnetic rim lesions (PRLs). Propensity score matching was used for between-group comparisons.</p><p><strong>Results: </strong>Over a median follow-up of 3.1 years in the ocrelizumab group and 1.9 years in the teriflunomide group, there were no significant differences in the risk of PIRA (HR for teriflunomide vs. ocrelizumab: 0.80 [95%-CI:0.40-1.60]; p = 0.53). PwRRMS treated with teriflunomide exhibited lower annualized rates of BVL (-0.80 [95%-CI: -0.91; -0.69] vs. -1.06 [95%-CI: -1.25; -0.86]; p = 0.025) and gray matter volume loss (-0.92 [95%-CI: -1.05; -0.79] vs. -1.20 [95%-CI: -1.43; -0.97]; p = 0.035). No differences were observed in DTI metrics or PRL count.</p><p><strong>Conclusions: </strong>This real-world study suggests that teriflunomide shows similar efficacy to ocrelizumab on smoldering activity, with a potentially greater effect in reducing BVL. Further research is needed to confirm these findings and understand their long-term implications.</p>","PeriodicalId":16558,"journal":{"name":"Journal of Neurology","volume":"272 8","pages":"491"},"PeriodicalIF":4.8,"publicationDate":"2025-07-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12222313/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144553788","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Frailty diminishes functional outcome in patients with nonaneurysmal subarachnoid hemorrhage: a dual specialized neurovascular center analysis. 虚弱会降低非动脉瘤性蛛网膜下腔出血患者的功能结局:一项双专业神经血管中心分析。
IF 4.8 2区 医学
Journal of Neurology Pub Date : 2025-07-02 DOI: 10.1007/s00415-025-13227-5
Larissa Penner, Anna-Laura Potthoff, Tim Lampmann, Rebecca Heinz, Johannes Lemcke, Motaz Hamed, Florian Gessler, Hartmut Vatter, Patrick Schuss, Alexis Hadjiathanasiou, Matthias Schneider
{"title":"Frailty diminishes functional outcome in patients with nonaneurysmal subarachnoid hemorrhage: a dual specialized neurovascular center analysis.","authors":"Larissa Penner, Anna-Laura Potthoff, Tim Lampmann, Rebecca Heinz, Johannes Lemcke, Motaz Hamed, Florian Gessler, Hartmut Vatter, Patrick Schuss, Alexis Hadjiathanasiou, Matthias Schneider","doi":"10.1007/s00415-025-13227-5","DOIUrl":"10.1007/s00415-025-13227-5","url":null,"abstract":"<p><strong>Objective: </strong>Frailty is increasingly recognized as a significant prognostic factor in various conditions. However, its impact on outcomes following spontaneous, nonaneurysmal subarachnoid hemorrhage (naSAH) remains unclear. This study aimed to assess the association between pre-existing frailty and functional outcomes in patients with naSAH.</p><p><strong>Methods: </strong>The study cohort was made up of 257 patients treated for naSAH at two neurovascular centers between 2012 and 2021. Frailty prior to naSAH was assessed using the modified frailty index (mFI), with patients classified as nonfrail (mFI 0-1) or frail (mFI ≥ 2). Functional outcomes at 6 months were evaluated using the modified Rankin Scale (mRS), categorized as favorable (mRS 0-2) or unfavorable (mRS 3-6). A multivariable logistic regression analysis was performed to identify independent predictors of unfavorable outcomes.</p><p><strong>Results: </strong>Among 257 naSAH patients, 56 (22%) were classified as frail (mFI ≥ 2) before ictus. At the 6-month follow-up, unfavorable outcomes were observed in 17 of the 56 frail patients (30%) compared to 21 of 201 nonfrail patients (10%) (p = 0.001). In addition to established negative prognostic factors such as delayed cerebral ischemia (p < 0.001) and poor-grade naSAH (Hunt & Hess grades III-IV; p = 0.001), multivariable analysis identified frailty (p = 0.03) as an independent and significant predictor of unfavorable functional outcomes.</p><p><strong>Conclusions: </strong>Frailty prior to hemorrhage, as determined by an mFI of ≥ 2, was associated with poor functional outcomes at 6 months in patients with naSAH. These findings underscore the importance of incorporating frailty assessments into early prognostic evaluations to guide patient management and counseling.</p>","PeriodicalId":16558,"journal":{"name":"Journal of Neurology","volume":"272 8","pages":"488"},"PeriodicalIF":4.8,"publicationDate":"2025-07-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12222263/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144540580","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
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