Journal of Neurology最新文献

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Evaluating a motor progression connectivity model across Parkinson's disease stages. 评估帕金森病各阶段的运动进展连接模型。
IF 4.8 2区 医学
Journal of Neurology Pub Date : 2024-10-07 DOI: 10.1007/s00415-024-12703-8
Mallory L Hacker, David A Isaacs, Nanditha Rajamani, Kian Pazira, Eli Abdou, Sheffield Sharp, Thomas L Davis, Peter Hedera, Fenna T Phibbs, David Charles, Andreas Horn
{"title":"Evaluating a motor progression connectivity model across Parkinson's disease stages.","authors":"Mallory L Hacker, David A Isaacs, Nanditha Rajamani, Kian Pazira, Eli Abdou, Sheffield Sharp, Thomas L Davis, Peter Hedera, Fenna T Phibbs, David Charles, Andreas Horn","doi":"10.1007/s00415-024-12703-8","DOIUrl":"10.1007/s00415-024-12703-8","url":null,"abstract":"<p><strong>Background: </strong>Stimulation of a specific site in the dorsolateral subthalamic nucleus (STN) was recently associated with slower motor progression in Parkinson's Disease (PD), based on the deep brain stimulation (DBS) in early-stage PD pilot clinical trial. Here, subject-level visualizations are presented of this early-stage PD dataset to further describe the relationship between active contacts and motor progression. This study also evaluates whether stimulation of the sweet spot and connectivity model associated with slower motor progression is also associated with improvements in long-term motor outcomes in patients with advanced-stage PD.</p><p><strong>Methods: </strong>Active contacts of the early-stage PD cohort (N = 14) were analyzed alongside the degree of two-year motor progression. Sweet spot and connectivity models derived from the early-stage PD cohort were then used to determine how well they can estimate the variance in long-term motor outcomes in an independent STN-DBS cohort of advanced-stage PD patients (N = 29).</p><p><strong>Results: </strong>In early-stage PD, proximity of stimulation to the dorsolateral STN was associated with slower motor progression. In advanced-stage PD, stimulation proximity to the early PD connectivity model and sweet spot were associated with better long-term motor outcomes (R = 0.60, P < 0.001; R = 0.37, P = 0.046, respectively).</p><p><strong>Conclusions: </strong>Results suggest stimulation of a specific site in the dorsolateral STN is associated with both slower motor progression and long-term motor improvements in PD.</p>","PeriodicalId":16558,"journal":{"name":"Journal of Neurology","volume":null,"pages":null},"PeriodicalIF":4.8,"publicationDate":"2024-10-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142381088","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
Autoimmune and infectious encephalitis: development of a discriminative tool for early diagnosis and initiation of therapy. 自身免疫性脑炎和传染性脑炎:开发用于早期诊断和开始治疗的鉴别工具。
IF 4.8 2区 医学
Journal of Neurology Pub Date : 2024-10-05 DOI: 10.1007/s00415-024-12712-7
Tobias Moser, Joachim Gruber, Eirini Mylonaki, Vincent Böhm, Daniel Schwarzenhofer, Anna R Tröscher, Eva Lenzenweger, Ingomar Krehan, Eva Söllradl, Markus Leitinger, Raimund Helbok, Eugen Trinka, Tim J von Oertzen, Judith N Wagner
{"title":"Autoimmune and infectious encephalitis: development of a discriminative tool for early diagnosis and initiation of therapy.","authors":"Tobias Moser, Joachim Gruber, Eirini Mylonaki, Vincent Böhm, Daniel Schwarzenhofer, Anna R Tröscher, Eva Lenzenweger, Ingomar Krehan, Eva Söllradl, Markus Leitinger, Raimund Helbok, Eugen Trinka, Tim J von Oertzen, Judith N Wagner","doi":"10.1007/s00415-024-12712-7","DOIUrl":"10.1007/s00415-024-12712-7","url":null,"abstract":"<p><strong>Background: </strong>Encephalitis originates from diverse autoimmune and infectious etiologies. Diagnostic challenges arise due to the spectrum of presentation and the frequent absence of specific biomarkers. This study aimed to comprehensively characterize and differentiate autoimmune encephalitis (AE) from infectious encephalitis (IE) in adults, and disentangle clinical, paraclinical, and therapeutic differences.</p><p><strong>Methods: </strong>A cohort study spanning 10 years was conducted across three Austrian tertiary care hospitals. Inclusion criteria comprised adults with probable or definite encephalitis. Demographics, clinical features, technical findings, treatment modalities, and outcomes were collected from the electronic patient files. A follow-up was performed via telephone interviews and clinical visits.</p><p><strong>Results: </strong>Of 149 patients, 17% had AE, 73% IE, and 10% encephalitis of unknown etiology. Significant differences between AE and IE included the prevalence of acute symptomatic seizures (AE: 85% vs. IE: 20%, p < 0.001), fever (8% vs. 72%, p < 0.001), headache (15% vs. 61%, p < 0.001), and focal neurological deficits (56% vs. 23%, p = 0.004), respectively. Paraclinical differences comprised lower CSF pleocytosis in AE compared to IE (median 6 cells/µl vs. 125 cells/µl, p < 0.001). Epileptic discharges on EEG and MRI lesions were more prevalent in AE than IE (50% vs. 14%, p < 0.001; 50% vs. 28%, p = 0.037). The modified Rankin Scale scores at discharge and last follow-up (median duration 2304 days, IQR 1433-3274) indicated favorable outcomes in both groups.</p><p><strong>Conclusion: </strong>This comprehensive analysis provides insights into the epidemiology, clinical, paraclinical, and therapeutic aspects and the outcomes of AE and IE in adults. We developed a diagnostic tool that facilitates early differentiation between AE and IE, aiding in timely therapeutic decision-making.</p>","PeriodicalId":16558,"journal":{"name":"Journal of Neurology","volume":null,"pages":null},"PeriodicalIF":4.8,"publicationDate":"2024-10-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142377975","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
A 14-year longitudinal study of neurofilament light chain dynamics in premanifest and transitional Huntington's disease. 对显性亨廷顿氏病前期和过渡期神经丝蛋白轻链动态的 14 年纵向研究。
IF 4.8 2区 医学
Journal of Neurology Pub Date : 2024-10-03 DOI: 10.1007/s00415-024-12700-x
Z J Voysey, N E Owen, J A Holbrook, M Malpetti, C Le Draoulec, L R B Spindler, A O G Goodman, A S Lazar, R A Barker
{"title":"A 14-year longitudinal study of neurofilament light chain dynamics in premanifest and transitional Huntington's disease.","authors":"Z J Voysey, N E Owen, J A Holbrook, M Malpetti, C Le Draoulec, L R B Spindler, A O G Goodman, A S Lazar, R A Barker","doi":"10.1007/s00415-024-12700-x","DOIUrl":"https://doi.org/10.1007/s00415-024-12700-x","url":null,"abstract":"<p><strong>Background: </strong>Growing evidence supports the value of neurofilament light (NfL) as a prognostic biomarker in premanifest Huntington's disease (HD). To date, however, there has been no longitudinal study exceeding 3 years examining either its serial dynamics or predictive power in HD. We aimed to conduct the first such study.</p><p><strong>Methods: </strong>Serum NfL was sampled using ultrasensitive immunoassay at four timepoints across a 14-year period in a cohort of HD gene carriers (n = 21) and controls (n = 14). Gene carriers were premanifest at baseline. Clinical features of HD were evaluated by Unified Huntington's Disease Rating Scale (UHDRS TMS), Montreal Cognitive Assessment (MoCA), Trail A/B task, Symbol Digit Modalities Task and semantic/phonemic fluency tasks.</p><p><strong>Results: </strong>14/21 HD gene carriers converted to prodromal or manifest disease by the final timepoint (\"converters\"). At baseline and each subsequent timepoint, NfL levels were higher in converters than in non-converters and controls (p = < 0.001-0.03, η<sub>p</sub><sup>2</sup> = 0.25-0.66). The estimated rate of change in NfL was higher in converters than in non-converters (p = 0.03) and controls (p = 0.001). Baseline NfL was able to discriminate converters from non-converters (area under curve = 1.000, p = 0.003). A higher rate of change in NfL was predictive of more severe motor (UHDRS-TMS p = 0.007, β = 0.711, R<sup>2</sup> = 0.468) and cognitive deficits (MoCA p = 0.007, β = - 0.798, R<sup>2</sup> = 0.604; Trail B, p = 0.007, β = 0.772, R<sup>2</sup> = 0.567; phonemic fluency p = 0.035, β = - 0.632, R<sup>2</sup> = 0.345).</p><p><strong>Conclusions: </strong>Our data suggest that (1) NfL longitudinal dynamics in premanifest/transitional HD are non-constant; rising faster in those closer to disease onset, and (2) NfL can identify individuals at risk of conversion to manifest disease and predict clinical trajectory, > 10 years from disease onset.</p>","PeriodicalId":16558,"journal":{"name":"Journal of Neurology","volume":null,"pages":null},"PeriodicalIF":4.8,"publicationDate":"2024-10-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142365482","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
Physical activity and risk of Parkinson's disease: an updated systematic review and meta-analysis. 体育锻炼与帕金森病的风险:最新的系统综述和荟萃分析。
IF 4.8 2区 医学
Journal of Neurology Pub Date : 2024-10-03 DOI: 10.1007/s00415-024-12672-y
Yanjie Jiang, Shipeng Zhang, Yuecan Chen, Hanyu Wang, Xingyi He, Chengli Bin, Rui Fu, Huan Wang, Hanqi Zhu, Moshen Pan, Qinxiu Zhang, Yan Lu
{"title":"Physical activity and risk of Parkinson's disease: an updated systematic review and meta-analysis.","authors":"Yanjie Jiang, Shipeng Zhang, Yuecan Chen, Hanyu Wang, Xingyi He, Chengli Bin, Rui Fu, Huan Wang, Hanqi Zhu, Moshen Pan, Qinxiu Zhang, Yan Lu","doi":"10.1007/s00415-024-12672-y","DOIUrl":"https://doi.org/10.1007/s00415-024-12672-y","url":null,"abstract":"<p><strong>Background and objectives: </strong>Although recent meta-analyses have shown that the association between physical activity (PA) and the risk of developing Parkinson's disease (PD) is influenced by gender differences, a growing number of studies are revealing the general applicability of this association across genders. This study aimed to reassess the association and dose-response relationship between PA and PD risk in populations.</p><p><strong>Methods: </strong>A systematic search of PubMed, Embase, Cochrane Library, and Web of Science databases was conducted in this study from inception to February 1, 2024, without language restrictions. Stratified analyses were conducted to explore the association between PA and PD risk, combining multivariate-adjusted effect estimates via random-effects models, and to validate the dose-response relationship between the two.</p><p><strong>Results: </strong>This study included 21 observational studies, comprising 13 cohort studies and 8 case-control studies. The pooled analysis revealed that PA significantly reduced the risk of developing PD [relative risk (RR) = 0.77, 95% CI 0.70-0.85]. In addition, the dose-response analysis revealed both linear and nonlinear associations, with linear results indicating a 9% reduction in PD risk for every 10 MET-h/wk increase in PA. The study also demonstrated that the protective effect of PA against PD was significant for both sexes. Moreover, no statistically significant effects of PA on preventing PD were observed in individuals with a BMI > 26 (RR = 0.35, 95% CI 0.12-1.02) or in Asian populations (RR = 0.78, 95% CI 0.60-1.01); however, the trends suggest potential protective effects, warranting further investigation. Sensitivity analyses confirmed the robustness of these findings.</p><p><strong>Conclusion: </strong>This meta-analysis produced substantial evidence to reaffirm the protective effect of high PA on PD across various population groups and the inverse dose-response relationship with PD risk, and to validate the protective effect of PA among different demographic groups.</p>","PeriodicalId":16558,"journal":{"name":"Journal of Neurology","volume":null,"pages":null},"PeriodicalIF":4.8,"publicationDate":"2024-10-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142372122","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
Assessment of the reliability, responsiveness, and meaningfulness of the scale for the assessment and rating of ataxia (SARA) for lysosomal storage disorders. 评估溶酶体贮积症共济失调评估和评级量表(SARA)的可靠性、响应性和意义。
IF 4.8 2区 医学
Journal of Neurology Pub Date : 2024-10-01 Epub Date: 2024-09-03 DOI: 10.1007/s00415-024-12664-y
Julien Park, Tatiana Bremova-Ertl, Marion Brands, Tomas Foltan, Matthias Gautschi, Paul Gissen, Andreas Hahn, Simon Jones, Laila Arash-Kaps, Miriam Kolnikova, Marc Patterson, Susan Perlman, Uma Ramaswami, Stella Reichmannová, Marianne Rohrbach, Susanne A Schneider, Aasef Shaikh, Siyamini Sivananthan, Matthis Synofzik, Mark Walterfarng, Pierre Wibawa, Kyriakos Martakis, Mario Manto
{"title":"Assessment of the reliability, responsiveness, and meaningfulness of the scale for the assessment and rating of ataxia (SARA) for lysosomal storage disorders.","authors":"Julien Park, Tatiana Bremova-Ertl, Marion Brands, Tomas Foltan, Matthias Gautschi, Paul Gissen, Andreas Hahn, Simon Jones, Laila Arash-Kaps, Miriam Kolnikova, Marc Patterson, Susan Perlman, Uma Ramaswami, Stella Reichmannová, Marianne Rohrbach, Susanne A Schneider, Aasef Shaikh, Siyamini Sivananthan, Matthis Synofzik, Mark Walterfarng, Pierre Wibawa, Kyriakos Martakis, Mario Manto","doi":"10.1007/s00415-024-12664-y","DOIUrl":"10.1007/s00415-024-12664-y","url":null,"abstract":"<p><strong>Objective: </strong>To evaluate the reliability, responsiveness, and validity of the Scale for the Assessment and Rating of Ataxia (SARA) in patients with lysosomal storage disorders (LSDs) who present with neurological symptoms, and quantify the threshold for a clinically meaningful change.</p><p><strong>Methods: </strong>We analyzed data from three clinical trial cohorts (IB1001-201, IB1001-202, and IB1001-301) of patients with Niemann-Pick disease type C (NPC) and GM2 Gangliosidoses (Tay-Sachs and Sandhoff disease) comprising 122 patients and 703 visits. Reproducibility was described as re-test reliability between repeat baseline visits or baseline and post-treatment washout visits. Responsiveness was determined in relation to the Investigator's, Caregiver's, and Patient's Clinical Global Impression of Improvement (CGI-I). The CGI-I data was also used to quantify a threshold for a clinically meaningful improvement on the SARA scale. Using a qualitative methods approach, patient/caregiver interviews from the IB1001-301 trial were further used to assess a threshold of meaningful change as well as the breadth of neurological signs and symptoms captured and evaluated by the SARA scale.</p><p><strong>Results: </strong>The Inter-Class Correlation (ICC) was 0.95 or greater for all three trials, indicating a high internal consistency/reliability. The mean change in SARA between repeat baseline and post-treatment washout visit assessments in all trials was -0.05, SD 1.98, i.e., minimal, indicating no significant differences, learning effects or other systematic biases. For the CGI-I responses and change in SARA scores, Area Under the Curve (AUC) values were 0.82, 0.71, and 0.77 for the Investigator's, Caregiver's, and Patient's CGI-I respectively, indicating strong agreement. Further qualitative analyses of the patient/caregiver interviews demonstrated a 1-point or greater change on SARA to be a clinically meaningful improvement which is directly relevant to the patient's everyday functioning and quality of life. Changes captured by the SARA were also paralleled by improvement in a broad range of neurological signs and symptoms and beyond cerebellar ataxia.</p><p><strong>Conclusion: </strong>Qualitative and quantitative data demonstrate the reliability and responsiveness of the SARA score as a valid measure of neurological signs and symptoms in LSDs with CNS involvement, such as NPC and GM2 Gangliosidoses. A 1-point change represents a clinically meaningful transition reflecting the gain or loss of complex function.</p>","PeriodicalId":16558,"journal":{"name":"Journal of Neurology","volume":null,"pages":null},"PeriodicalIF":4.8,"publicationDate":"2024-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11447074/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142120080","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
Highly beneficial outcome in severe acute necrotizing encephalopathy with tocilizumab treatment. 托珠单抗治疗严重急性坏死性脑病疗效显著。
IF 4.8 2区 医学
Journal of Neurology Pub Date : 2024-10-01 Epub Date: 2024-09-06 DOI: 10.1007/s00415-024-12661-1
Alexander Balck, Lara M Lange, Alexander Neumann, Georg Royl, Philipp Jung, Jens Schaumberg, Norbert Brüggemann, Philipp J Koch
{"title":"Highly beneficial outcome in severe acute necrotizing encephalopathy with tocilizumab treatment.","authors":"Alexander Balck, Lara M Lange, Alexander Neumann, Georg Royl, Philipp Jung, Jens Schaumberg, Norbert Brüggemann, Philipp J Koch","doi":"10.1007/s00415-024-12661-1","DOIUrl":"10.1007/s00415-024-12661-1","url":null,"abstract":"","PeriodicalId":16558,"journal":{"name":"Journal of Neurology","volume":null,"pages":null},"PeriodicalIF":4.8,"publicationDate":"2024-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11447148/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142145812","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
Richard Gundry Rows (1866-1925). 理查德-冈德罗斯(1866-1925 年)。
IF 4.8 2区 医学
Journal of Neurology Pub Date : 2024-10-01 Epub Date: 2024-08-13 DOI: 10.1007/s00415-024-12626-4
Andrew J Larner
{"title":"Richard Gundry Rows (1866-1925).","authors":"Andrew J Larner","doi":"10.1007/s00415-024-12626-4","DOIUrl":"10.1007/s00415-024-12626-4","url":null,"abstract":"","PeriodicalId":16558,"journal":{"name":"Journal of Neurology","volume":null,"pages":null},"PeriodicalIF":4.8,"publicationDate":"2024-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141975930","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
Correction to: Moving towards a new era for the treatment of neuromyelitis optica spectrum disorders. 更正:迈向治疗神经脊髓炎视网膜谱系障碍的新时代。
IF 4.8 2区 医学
Journal of Neurology Pub Date : 2024-10-01 DOI: 10.1007/s00415-024-12628-2
Paolo Preziosa, Maria Pia Amato, Luca Battistini, Marco Capobianco, Diego Centonze, Eleonora Cocco, Antonella Conte, Claudio Gasperini, Matteo Gastaldi, Carla Tortorella, Massimo Filippi
{"title":"Correction to: Moving towards a new era for the treatment of neuromyelitis optica spectrum disorders.","authors":"Paolo Preziosa, Maria Pia Amato, Luca Battistini, Marco Capobianco, Diego Centonze, Eleonora Cocco, Antonella Conte, Claudio Gasperini, Matteo Gastaldi, Carla Tortorella, Massimo Filippi","doi":"10.1007/s00415-024-12628-2","DOIUrl":"10.1007/s00415-024-12628-2","url":null,"abstract":"","PeriodicalId":16558,"journal":{"name":"Journal of Neurology","volume":null,"pages":null},"PeriodicalIF":4.8,"publicationDate":"2024-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142132985","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
Risks of epilepsy. 癫痫的风险。
IF 4.8 2区 医学
Journal of Neurology Pub Date : 2024-10-01 Epub Date: 2024-09-11 DOI: 10.1007/s00415-024-12682-w
J Clarke, J A Johnston
{"title":"Risks of epilepsy.","authors":"J Clarke, J A Johnston","doi":"10.1007/s00415-024-12682-w","DOIUrl":"10.1007/s00415-024-12682-w","url":null,"abstract":"","PeriodicalId":16558,"journal":{"name":"Journal of Neurology","volume":null,"pages":null},"PeriodicalIF":4.8,"publicationDate":"2024-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142289402","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
Opicapone for the treatment of early wearing-off in levodopa-treated Parkinson's disease: pooled analysis of patient level data from two randomized open-label studies. 奥匹卡朋用于治疗左旋多巴治疗帕金森病的早期停药:两项随机开放标签研究的患者水平数据汇总分析。
IF 4.8 2区 医学
Journal of Neurology Pub Date : 2024-10-01 Epub Date: 2024-08-21 DOI: 10.1007/s00415-024-12614-8
Joaquim J Ferreira, Jee-Young Lee, Hyeo-Il Ma, Beomseok Jeon, Werner Poewe, Angelo Antonini, Fabrizio Stocchi, Daniela M Rodrigues, Miguel M Fonseca, Guillermo Castilla-Fernández, Joerg Holenz, José-Francisco Rocha, Olivier Rascol
{"title":"Opicapone for the treatment of early wearing-off in levodopa-treated Parkinson's disease: pooled analysis of patient level data from two randomized open-label studies.","authors":"Joaquim J Ferreira, Jee-Young Lee, Hyeo-Il Ma, Beomseok Jeon, Werner Poewe, Angelo Antonini, Fabrizio Stocchi, Daniela M Rodrigues, Miguel M Fonseca, Guillermo Castilla-Fernández, Joerg Holenz, José-Francisco Rocha, Olivier Rascol","doi":"10.1007/s00415-024-12614-8","DOIUrl":"10.1007/s00415-024-12614-8","url":null,"abstract":"<p><strong>Background: </strong>The wearing-off phenomenon is a key driver of medication change for patients with Parkinson's disease (PD) treated with levodopa. Common first-line options include increasing the levodopa dose or adding a catechol-O-methyltransferase (COMT) inhibitor, but there are no trials comparing the efficacy of these approaches. We evaluated the effectiveness of adjunct opicapone versus an additional 100 mg levodopa dose in PD patients with early wearing-off using pooled data from 2 randomized studies.</p><p><strong>Methods: </strong>The ADOPTION study program included two similarly designed 4-week, open-label studies conducted in South Korea (NCT04821687) and Europe (NCT04990284). Patients with PD, treated with 3-4 daily doses of levodopa therapy and with signs of early wearing-off were randomized (1:1) to adjunct opicapone 50 mg or an additional dose of levodopa 100 mg. Patient-level data from the two studies were pooled.</p><p><strong>Results: </strong>The adjusted mean [SE] change from baseline to week 4 in absolute OFF time (key endpoint) was - 62.8 min [8.8] in the opicapone group and - 33.8 min [9.0] in the levodopa 100 mg group, the difference significantly favoring opicapone (- 29.0 [- 53.8, - 4.2] min, p = 0.02). Significant differences in the Movement Disorder Society-Unified Parkinson's Disease Rating Scale Part III subscore (- 4.1 with opicapone vs - 2.5 with levodopa 100 mg), also favored opicapone (- 1.7 [- 3.3, - 0.04], p < 0.05). Dyskinesia was the most frequently reported adverse event (opicapone 7.2% vs. levodopa 100 mg 4.2%).</p><p><strong>Conclusions: </strong>In these short-term trials, introducing adjunct opicapone was more effective at reducing OFF time than adding another 100 mg levodopa dose in PD patients with early signs of wearing-off.</p>","PeriodicalId":16558,"journal":{"name":"Journal of Neurology","volume":null,"pages":null},"PeriodicalIF":4.8,"publicationDate":"2024-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11447072/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142008926","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
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