JAMA neurology最新文献

{"title":"Digital Biomarkers for Neurodegenerative Disease.","authors":"Claire M Erickson, Anna Wexler, Emily A Largent","doi":"10.1001/jamaneurol.2024.3533","DOIUrl":"10.1001/jamaneurol.2024.3533","url":null,"abstract":"","PeriodicalId":14677,"journal":{"name":"JAMA neurology","volume":" ","pages":"5-6"},"PeriodicalIF":20.4,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11729353/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142465719","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"JAMA Neurology.","authors":"","doi":"10.1001/jamaneurol.2024.3408","DOIUrl":"https://doi.org/10.1001/jamaneurol.2024.3408","url":null,"abstract":"","PeriodicalId":14677,"journal":{"name":"JAMA neurology","volume":"82 1","pages":"4"},"PeriodicalIF":20.4,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142970888","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":"Gantenerumab in Dominantly Inherited Alzheimer Disease-Reply.","authors":"Olivia Wagemann, Eric McDade, Randall J Bateman","doi":"10.1001/jamaneurol.2024.3828","DOIUrl":"10.1001/jamaneurol.2024.3828","url":null,"abstract":"","PeriodicalId":14677,"journal":{"name":"JAMA neurology","volume":" ","pages":"111-112"},"PeriodicalIF":20.4,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142620720","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":"Error in Results.","authors":"","doi":"10.1001/jamaneurol.2024.4836","DOIUrl":"10.1001/jamaneurol.2024.4836","url":null,"abstract":"","PeriodicalId":14677,"journal":{"name":"JAMA neurology","volume":"82 1","pages":"112"},"PeriodicalIF":20.4,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11731175/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142970885","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Deferiprone in Alzheimer Disease: A Randomized Clinical Trial.","authors":"Scott Ayton, David Barton, Bruce Brew, Amy Brodtmann, Roger Clarnette, Patricia Desmond, David Devos, Kathryn A Ellis, Amir Fazlollahi, Caroline Fradette, Anita M Y Goh, Pawel Kalinowski, Christopher Kyndt, Rosalyn Lai, Yen Ying Lim, Paul Maruff, Terence J O'Brien, Christopher Rowe, Olivier Salvado, Peter W Schofield, Michael Spino, Fernando Tricta, Aaron Wagen, Robert Williams, Michael Woodward, Ashley I Bush","doi":"10.1001/jamaneurol.2024.3733","DOIUrl":"10.1001/jamaneurol.2024.3733","url":null,"abstract":"<p><strong>Importance: </strong>Interventions that substantially slow neurodegeneration are needed to address the growing burden of Alzheimer disease (AD) to societies worldwide. Elevated brain iron observed in AD has been associated with accelerated cognitive decline and may be a tractable drug target.</p><p><strong>Objective: </strong>To investigate whether the brain-permeable iron chelator deferiprone slows cognitive decline in people with AD.</p><p><strong>Design, setting, and participants: </strong>This phase 2, double-masked, placebo-controlled randomized clinical trial of 12-month duration was conducted at 9 sites in Australia between August 2, 2018, and April 1, 2023. Patients older than 54 years with amyloid-confirmed mild cognitive impairment or early AD (a Mini-Mental State Examination score of 20 or higher) were screened. Randomization was 2:1 and masked to participants and all study staff.</p><p><strong>Interventions: </strong>Deferiprone 15 mg/kg twice a day or placebo administered orally for 12 months.</p><p><strong>Main outcomes and measures: </strong>The primary outcome was a composite cognitive measure assessed at baseline, 6 months, and 12 months using a neuropsychological test battery (NTB) of memory, executive function, and attention tasks. Secondary outcomes included change in brain iron burden measured by quantitative susceptibility mapping (QSM) magnetic resonance imaging (target engagement), brain volume changes (secondary efficacy measure), and adverse events (safety analysis).</p><p><strong>Results: </strong>Of 167 patients screened for eligibility, 81 were included, with 53 randomly assigned to the deferiprone group (mean [SD] age, 73.0 [8.0] years; 29 male [54.7%]) and 28 to the placebo group (mean [SD] age, 71.6 [7.2] years; 17 male [60.7%]); 54 participants completed the study (7 [25.0%] withdrew from the placebo group and 20 [37.7%] from the deferiprone group). In an intention-to-treat analysis, participants in the deferiprone group showed accelerated cognitive decline on the NTB primary outcome (β for interaction = -0.50; 95% CI, -0.80 to -0.20) compared with placebo (change in NTB composite z score for deferiprone, -0.80 [95% CI, -0.98 to -0.62]; for placebo, -0.30 [95% CI, -0.54 to -0.06]). Secondary analysis revealed that this result was driven by worsening performance on executive function tests. The QSM confirmed that deferiprone decreased iron in the hippocampus compared with placebo (change in hippocampal QSM for deferiprone, -0.36 ppb [95% CI, -0.76 to 0.04 ppb]; for placebo, 0.32 ppb [95% CI, -0.12 to 0.75 ppb]; β for interaction = -0.68 [95% CI, -1.27 to -0.09]). Longitudinal hippocampal volume loss was not affected by deferiprone, but exploratory analysis of other brain regions revealed increased volume loss with deferiprone in frontal areas. The frequency of the adverse effect of neutropenia (4 participants [7.5%] in the deferiprone group) was higher than in similar studies (1.6%-4.4%).</p><p><strong>C","PeriodicalId":14677,"journal":{"name":"JAMA neurology","volume":" ","pages":"11-18"},"PeriodicalIF":20.4,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11536302/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142568645","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Immediate or Delayed Statin in Acute Atherosclerotic Ischemia-Reply.","authors":"Ying Gao, Yuesong Pan, Yilong Wang","doi":"10.1001/jamaneurol.2024.3750","DOIUrl":"10.1001/jamaneurol.2024.3750","url":null,"abstract":"","PeriodicalId":14677,"journal":{"name":"JAMA neurology","volume":" ","pages":"110-111"},"PeriodicalIF":20.4,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142568650","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":"Remembering the Physician's Humanity-Physicians Are Humans Too.","authors":"Noriko Anderson","doi":"10.1001/jamaneurol.2024.3744","DOIUrl":"10.1001/jamaneurol.2024.3744","url":null,"abstract":"","PeriodicalId":14677,"journal":{"name":"JAMA neurology","volume":" ","pages":"9-10"},"PeriodicalIF":20.4,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142568653","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":"Gantenerumab in Dominantly Inherited Alzheimer Disease.","authors":"Nunzio Pomara, Bruno Pietro Imbimbo","doi":"10.1001/jamaneurol.2024.3825","DOIUrl":"10.1001/jamaneurol.2024.3825","url":null,"abstract":"","PeriodicalId":14677,"journal":{"name":"JAMA neurology","volume":" ","pages":"111"},"PeriodicalIF":20.4,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142620708","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":"Mood Alterations in the Prodromal Phase of Sporadic Creutzfeldt-Jakob Disease","authors":"Raphael Wurm, Sigrid Klotz, Astrid Erber, Felix Gruber, Stefan Leitner, Berthold Reichardt, Elisabeth Stögmann, Eva Schernhammer, Ellen Gelpi, Hakan Cetin","doi":"10.1001/jamaneurol.2024.4447","DOIUrl":"https://doi.org/10.1001/jamaneurol.2024.4447","url":null,"abstract":"ImportanceSporadic Creutzfeldt-Jakob disease (sCJD) is a rare, rapidly progressive and fatal neurodegenerative disease. Definite sCJD diagnosis can only be made post mortem, and little is known about the prodromal phase of the disease.ObjectiveTo compare drug prescription patterns before the clinical onset of sCJD between patients and matched controls for exploration of potential risk factors and to assess correlations between drug exposure and sCJD survival.Design, Setting, and ParticipantsThis retrospective analysis was designed as a case-control study, with data collected from January 2013 to December 2020 and analyzed in 2023. Follow-up was available until December 2020. Cases were collected from the Austrian Reference Centre for Human Prion Diseases, which receives all suspected cases at a national level in Austria. The analyses were conducted at a single center. Patients with autopsy-confirmed sCJD were linked with insurance claims data, and a minimum of 10 control individuals were matched by sex, age at onset, and area of residence for each patient with sCJD.ExposureMedication prescribed to 10% or more of the cohort with sCJD up to 5 years before symptom onset or the matching date in the control cohort.Main Outcomes and MeasuresDrug prescription before symptom onset or the matching date was compared between patients with sCJD and controls using conditional regression, and prescriptions in the cohort with sCJD were assessed for correlation with survival using Cox proportional hazard models.ResultsA total of 129 patients with sCJD (median [IQR] age, 68.9 [62.4-75.5] years; 67 female [51.9%]) and 1350 controls (median [IQR] age, 69.0 [62.2-75.3] years; 700 female [51.9%]) were included. As compared with controls, patients with sCJD were found to have significantly higher odds of being prescribed selective serotonin reuptake inhibitors (SSRIs) in the year preceding disease onset (odds ratio, 2.86; 95% CI, 1.63-4.95; <jats:italic>P</jats:italic> &amp;amp;lt; .001). SSRI prescription rates started to increase 3 years before symptom onset in the cohort with sCJD.Conclusions and RelevanceResults of this case-control study provide evidence for prodromal mood alterations as early as 3 years before symptom onset in patients with sCJD. Although sCJD remains an extremely rare cause of mood alterations, increased vigilance for neurodegenerative diseases in this setting could eventually help to extend the diagnostic window.","PeriodicalId":14677,"journal":{"name":"JAMA neurology","volume":"164 1","pages":""},"PeriodicalIF":29.0,"publicationDate":"2024-12-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142902145","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":"Blood-Based Biomarkers for Identifying Disease Activity in AQP4-IgG-Positive Neuromyelitis Optica Spectrum Disorder.","authors":"Su-Hyun Kim, Ana Beatriz Ayroza Galvão Ribeiro Gomes, Patrick Schindler, Jae-Won Hyun, Ki Hoon Kim, Dong-Eun Lee, Vinicius Andreoli Schoeps, Aline de Moura Brasil Matos, Natalia Trombini Mendes, Samira Luisa Dos Apóstolos-Pereira, Dagoberto Callegaro, Jasmine Lerner, Pascal Benkert, Jens Kuhle, Klemens Ruprecht, Friedemann Paul, Anne-Katrin Pröbstel, Ho Jin Kim","doi":"10.1001/jamaneurol.2024.4400","DOIUrl":"https://doi.org/10.1001/jamaneurol.2024.4400","url":null,"abstract":"<p><strong>Importance: </strong>The temporal dynamics of serum glial fibrillary acidic protein (sGFAP) and serum neurofilament light chain (sNfL) as biomarkers of disease activity for neuromyelitis optica spectrum disorder (NMOSD) remain underexplored.</p><p><strong>Objective: </strong>To determine optimal timing for assessing sGFAP and sNfL, establish cutoff values differentiating between attacks and remissions in NMOSD, and evaluate these findings across independent cohorts.</p><p><strong>Design, setting, and participants: </strong>This retrospective, longitudinal, multicenter cohort study was conducted among patients with aquaporin-4 antibody (AQP4-IgG)-positive NMOSD. Patients with available stored serum samples were included, totaling 181 patients with 625 samples. Discovery cohort samples were collected from February 2008 to October 2023 and validation cohort samples were collected from January 2013 to October 2023. A combined analysis of both cohorts was conducted from November 2023 to March 2024.</p><p><strong>Exposures: </strong>sNfL and sGFAP concentrations, measured by a single-molecule array assay.</p><p><strong>Main outcomes and measures: </strong>The primary outcomes were the optimal timing of assessing sGFAP and sNfL and the adjusted cutoff values for evaluating disease activity in NMOSD.</p><p><strong>Results: </strong>The discovery cohort consisted of 366 samples from 78 Korean patients (median [IQR] age, 35 [30-42] years; 73 female patients [95%]), while the validation cohort included 190 samples from 34 German patients (median [IQR] age, 54 [39-61] years; 32 female patients [94%]) and 69 samples from 69 Brazilian patients (median [IQR] age, 46 [35-55] years; 62 female patients [90%]). Six-month postattack temporal biomarker dynamics were analyzed in 202 samples from 74 patients in the discovery cohort: sGFAP levels peaked within the first week and sNfL levels peaked at 5 weeks postattack. The optimal time frames for evaluating attacks were within 1 week for sGFAP and from 1 to 8 weeks for sNfL, with remission defined as at least 6 months postattack. z Score cutoffs of 3.0 for sGFAP and 2.1 for sNfL effectively distinguished between attack and remission phases, indicated by area under the curve values of 0.95 (95% CI, 0.88-1.02) and 0.87 (95% CI, 0.82-0.91), respectively. The discovery cohort time frames and cutoff values were applied to the validation cohort, achieving 71% sensitivity and 94% specificity for sNfL and 100% sensitivity and specificity for sGFAP in the German and Brazilian cohorts.</p><p><strong>Conclusions and relevance: </strong>This longitudinal cohort study established optimal timing and thresholds for sGFAP and sNfL, which were consistent in independent cohorts, supporting these biomarkers' effectiveness in distinguishing NMOSD attacks from remission.</p>","PeriodicalId":14677,"journal":{"name":"JAMA neurology","volume":" ","pages":""},"PeriodicalIF":20.4,"publicationDate":"2024-12-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142876775","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}