JAMA neurology最新文献

{"title":"Modulation of Treatment Effect on Disability by Disease Activity in Progressive Multiple Sclerosis","authors":"Noemi Montobbio, Francesca Bovis, Douglas L. Arnold, Maria Pia Sormani","doi":"10.1001/jamaneurol.2025.2565","DOIUrl":"https://doi.org/10.1001/jamaneurol.2025.2565","url":null,"abstract":"This meta-analysis of individual patient data from 6 clinical trials confirms the efficacy of existing therapies in patients with progressive multiple sclerosis showing disease activity and assesses the role of lesion reduction as a mediator of treatment effect.","PeriodicalId":14677,"journal":{"name":"JAMA neurology","volume":"97 1","pages":""},"PeriodicalIF":29.0,"publicationDate":"2025-07-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144715288","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":"Life Expectancy for Patients With Sporadic Creutzfeldt-Jakob Disease","authors":"Amber Yaqub, A. Charlotte Menart, Bettina E. Hansen, M. Arfan Ikram","doi":"10.1001/jamaneurol.2025.2495","DOIUrl":"https://doi.org/10.1001/jamaneurol.2025.2495","url":null,"abstract":"This cohort study examines registry data to determine age-specific life expectancies for patients with sporadic Creutzfeldt-Jakob disease and analyze trends in median survival over the past 30 years.","PeriodicalId":14677,"journal":{"name":"JAMA neurology","volume":"12 1","pages":""},"PeriodicalIF":29.0,"publicationDate":"2025-07-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144715295","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":"Concordance Between Amyloid-PET Quantification and Real-World Visual Reads","authors":"Ehud Zeltzer, Daniel R. Schonhaut, Nidhi S. Mundada, Ganna Blazhenets, David N. Soleimani-Meigooni, Hanna Cho, Kamalini G. Ranasinghe, Charles Windon, Golnaz Yadollahikhales, Charles Apgar, Constantine Gatsonis, Maria C. Carrillo, Lucy Hanna, Justin Romanoff, Bruce E. Hillner, Robert A. Koeppe, Andrew March, Barry A. Siegel, Karen Smith, Rachel A. Whitmer, Leonardo Iaccarino, Gil D. Rabinovici, Renaud La Joie","doi":"10.1001/jamaneurol.2025.2218","DOIUrl":"https://doi.org/10.1001/jamaneurol.2025.2218","url":null,"abstract":"ImportanceWith increased payer coverage and the advent of antiamyloid therapies, clinical use of amyloid positron emission tomography (PET) is likely to increase to help guide the diagnosis and treatment of patients with cognitive impairment. However, unlike most previous research studies, in clinical practice, scan acquisition is less standardized, interpretation typically relies purely on visual reads rather than scan quantification, and patients have more frequent comorbidities, all of which might compromise test accuracy.ObjectiveTo compare visual interpretation of amyloid-PET in real-world clinical settings to scan interpretation based on central quantification, in order to assess the accuracy of clinical reads.Design, Setting, and ParticipantsThis cross-sectional quality improvement study used data from the Imaging Dementia—Evidence for Amyloid Scanning study, collected between February 2016 and January 2018 and analyzed between December 2021 and April 2023. The setting included 294 imaging facilities in the US. Medicare beneficiaries 65 years or older with cognitive decline for whom Alzheimer disease was a diagnostic consideration were recruited by dementia specialists from their clinical practices.ExposuresAmyloid-PET with [18F]florbetapir, [18F]florbetaben, or [18F]flutemetamol.Main Outcomes and MeasuresPET scans were visually interpreted as positive or negative by local radiologists or nuclear medicine physicians following approved guidelines. Independently, scans were centrally processed and quantified using the standardized Centiloid (CL) scale. We applied an a priori autopsy-based threshold of 24.4 CL to quantitatively define scan positivity.ResultsOf 18 293 participants included in the parent study, scan images were available for 10 774 (59%), of which Centiloids were successfully calculated for 10 361 (96%). Median (IQR) patient age was 75 (71-80) years; 5245 patients (51%) were female, 6500 (63%) had mild cognitive impairment, and 3861 (37%) had dementia). Participants self-reported the following races and ethnicities: 1 Alaska Native (0%), 23 American Indian (0.2%), 188 Asian (1.8%), 316 Black (3.1%), 449 Hispanic or Latino (4.3%), 8 Native Hawaiian or Other Pacific Islander (0.1%), and 9125 White (88.2%). A total of 6332 scans (61%) were visually read as positive, and 6121 (59%) were quantitatively positive. Agreement between visual reads and quantitative classification was 86.3% (95% CI, 85.7%-87.0%; Cohen κ = 0.72; 95% CI, 0.70-0.73). A total of 5519 (53%) scans were positive visually and quantitatively (V+/Q+), 3416 (33%) were negative by both (V−/Q−), 813 (8%) were V+/Q−, and 602 (6%) were V−/Q+. Female sex (female: 4581/5241 [87.4%]; male: 4354/5109 [85.2%]; <jats:italic>P</jats:italic> =.001), White race (White race: 7900/9125 [86.6%]; non-White race: 1035/1225 [84.5%]; <jats:italic>P</jats:italic> =.046), and use of [18F]flutemetamol and [18F]florbetaben ([18F]flutemetamol: 559/628 [89.0%]; [18F]florbetaben: 2664/3032 [87","PeriodicalId":14677,"journal":{"name":"JAMA neurology","volume":"57 1","pages":""},"PeriodicalIF":29.0,"publicationDate":"2025-07-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144715282","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":"Gene Therapy vs Cochlear Implantation in Restoring Hearing Function and Speech Perception for Individuals With Congenital Deafness.","authors":"Xiaoting Cheng,Jiake Zhong,Jiajia Zhang,Chong Cui,Luoying Jiang,Yang-Wenyi Liu,Yuxin Chen,Qi Cao,Daqi Wang,Guiqing Cheng,Yuxin Zong,Min Shen,Chunxin Xu,Jun Lv,Hui Wang,Longlong Zhang,Biyun Zhu,Honghai Tang,Jinghan Wang,Xintai Fan,Yanqing Fang,Luo Guo,Jiawei Guo,Liheng Chen,Yanbo Yin,Zijing Wang,Lei Han,Shaowei Hu,Shengyi Wang,Guoyou Qin,Xuezhong Liu,Jinqiu Sang,Fangang Zeng,Wuqing Wang,Bing Chen,Zheng-Yi Chen,Huawei Li,Yilai Shu","doi":"10.1001/jamaneurol.2025.2053","DOIUrl":"https://doi.org/10.1001/jamaneurol.2025.2053","url":null,"abstract":"ImportanceOTOF gene therapy (GT) has been shown to improve hearing and speech. The efficacy of GT remains to be compared against cochlear implantation (CI), the current gold standard for congenital deafness.ObjectiveTo evaluate treatment outcomes in auditory and speech perception between patients with congenital deafness treated with GT, CI, or both.Design, Setting, and ParticipantsThis nonblind cohort study was conducted between December 2022 and November 2024. GT patients received follow-up at 3, 6, and 12 months; CI patients received 1-time evaluation at the corresponding time intervals or longer (3, 6, or 12 months). The study was conducted at a single class A tertiary hospital in China. Participants with congenital severe to complete hearing loss, aged 1 to 18 years, who received GT or CI were enrolled. They were matched on duration of deafness, hearing thresholds, and speech ability at the presurgical baseline. Of 1568 participants screened, 72 participants enrolled. Participants were excluded if they had inner ear malformations or vestibular-cochlear nerve abnormalities.ExposuresGT only vs CI; bimodal (unilateral GT plus contralateral CI) vs bilateral CI; GT (CI turned off [CI-off]) vs unilateral CI.Main Outcomes and MeasuresThe primary outcomes were auditory and speech perception evaluated by questionnaires, including the Infant-Toddler Meaningful Auditory Integration Scale/Meaningful Auditory Integration Scale (IT-MAIS/MAIS), and tests, including audiometry, speech, and music tests. The main secondary outcome was auditory information processing ability assessed by mismatch negativity (MMN).ResultsA total of 11 GT patients (6 male [55%]; mean [SD] age at baseline, 3.7 [2.8] years) and 61 CI patients (34 male [56%]; mean [SD] age at baseline, 1.9 [1.5] years) were enrolled. The mean (SD) auditory brainstem response thresholds were restored from greater than 95.0 (0.0) decibels normalized hearing level (dB nHL) to 54.8 (15.9) dB nHL in 9 GT patients at 12 months. For GT-only vs CI in auditory and speech perception, GT patients performed better in IT-MAIS/MAIS at 6 months (median [IQR] score, 31.0 [30.0-32.0] vs 23.5 [19.0-26.3]; P = .01) and 12 months (median [IQR] score, 32.0 [31.0-32.0] vs 28.0 [24.5-30.5]; P = .007). GT patients showed shorter latencies of MMN at 6 months (median [IQR], 0.20 [0.05-0.21] seconds vs 0.23 [0.22-0.25] seconds; P = .006). For bimodal patients at 12 months, GT (CI-off) patients performed better than unilateral CI patients in speech in a noisy environment (median [IQR] disyllable, -1.0 [-3.0 to 2.4] dB sound pressure level (SPL) vs 5.3 [3.1 to 12.1] dB SPL; P = .03); GT plus CI patients performed better than bilateral CI patients in singing in-tune rates (median [IQR], 66.6% [53.7%-83.9%] vs 37.1% [30.3%-56.3%]; P = .04); GT plus CI patients showed shorter latencies of MMN at 12 months (median [IQR], 0.08 [0.07-0.10] seconds vs 0.21 [0.15-0.23] seconds, P = .01).Conclusions and RelevanceGT patients showed stabl","PeriodicalId":14677,"journal":{"name":"JAMA neurology","volume":"94 1","pages":""},"PeriodicalIF":29.0,"publicationDate":"2025-07-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144669517","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":"Concern About Predictive Performance of a Pain Sensitivity Biomarker.","authors":"Ole Goltermann,Tamas Spisak,Christian Büchel","doi":"10.1001/jamaneurol.2025.2351","DOIUrl":"https://doi.org/10.1001/jamaneurol.2025.2351","url":null,"abstract":"","PeriodicalId":14677,"journal":{"name":"JAMA neurology","volume":"26 1","pages":""},"PeriodicalIF":29.0,"publicationDate":"2025-07-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144669422","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":"Concern About Predictive Performance of a Pain Sensitivity Biomarker-Reply.","authors":"Nahian S Chowdhury,Chuan Bi,David A Seminowicz","doi":"10.1001/jamaneurol.2025.2354","DOIUrl":"https://doi.org/10.1001/jamaneurol.2025.2354","url":null,"abstract":"","PeriodicalId":14677,"journal":{"name":"JAMA neurology","volume":"14 1","pages":""},"PeriodicalIF":29.0,"publicationDate":"2025-07-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144669423","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":"Unusual Finding of Filling Defect During Mechanical Thrombectomy.","authors":"Yating Han,Zunjing Liu","doi":"10.1001/jamaneurol.2025.2227","DOIUrl":"https://doi.org/10.1001/jamaneurol.2025.2227","url":null,"abstract":"","PeriodicalId":14677,"journal":{"name":"JAMA neurology","volume":"103 1","pages":""},"PeriodicalIF":29.0,"publicationDate":"2025-07-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144669424","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":"The Escape Valve Phenomenon in Medical Education.","authors":"Joseph E Safdieh","doi":"10.1001/jamaneurol.2025.2292","DOIUrl":"https://doi.org/10.1001/jamaneurol.2025.2292","url":null,"abstract":"","PeriodicalId":14677,"journal":{"name":"JAMA neurology","volume":"658 1","pages":""},"PeriodicalIF":29.0,"publicationDate":"2025-07-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144669425","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":"GLP-1 RAs for Idiopathic Intracranial Hypertension-Time for a Trial.","authors":"Nancy J Newman,Beau B Bruce,Valérie Biousse","doi":"10.1001/jamaneurol.2025.2019","DOIUrl":"https://doi.org/10.1001/jamaneurol.2025.2019","url":null,"abstract":"","PeriodicalId":14677,"journal":{"name":"JAMA neurology","volume":"38 1","pages":""},"PeriodicalIF":29.0,"publicationDate":"2025-07-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144622151","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":"GLP-1 Receptor Agonists in Idiopathic Intracranial Hypertension.","authors":"Georgios S Sioutas,William Mualem,John Reavey-Cantwell,Dennis J Rivet","doi":"10.1001/jamaneurol.2025.2020","DOIUrl":"https://doi.org/10.1001/jamaneurol.2025.2020","url":null,"abstract":"ImportanceCurrent treatment options for idiopathic intracranial hypertension (IIH) are limited by efficacy, safety, and sustainability concerns. Glucagon-like peptide 1 receptor agonists (GLP-1 RAs), known for promoting weight loss and metabolic regulation, may offer a novel therapeutic approach.ObjectiveTo assess whether GLP-1 RA therapy is associated with improved clinical outcomes in patients with IIH compared with conventional therapies.Design, Setting, and ParticipantsThis retrospective cohort study was conducted using data from the TriNetX US Collaborative Network between 2005 and 2024, with a follow-up duration of 1 year. Electronic health records from 67 health care organizations across the United States were examined. Participants were patients 18 years and older who had IIH.ExposureInitiation of GLP-1 RA therapy within 6 months of IIH diagnosis. The control group included patients managed with conventional treatments, such as acetazolamide, topiramate, and dietary counseling, without GLP-1 RA exposure.Main Outcomes and MeasuresOutcomes included use of non-GLP-1 RA medication, symptoms and signs, procedures, and mortality over 1 year. Outcomes were expressed as risk ratios (RRs) with 95% CI.ResultsA total of 44 373 patients with IIH were identified. Before propensity score matching, the cohort included 603 GLP-1 RA users and 43 770 nonusers. The GLP-1 RA group was older (mean [SD] age, 43.2 [13.0] vs 35.5 [14.3] years; P < .001) with fewer male patients (n = 60 [10.0%] vs n = 5879 [13.5%]; P = .01) and a similar number of female patients (n = 522 [86.6%] vs n = 36 796 [84.3%]; P = .13). After matching, 555 GLP-1 RAs users were compared with 555 nonusers. GLP-1 RA use was associated with lower medication use (RR, 0.53; 95% CI, 0.46-0.61; P < .001) and reduced headaches (RR, 0.45; 95% CI, 0.35-0.58; P < .001), visual disturbances or blindness (RR, 0.60; 95% CI, 0.41-0.88; P = .007), and papilledema (RR, 0.19; 95% CI, 0.10-0.34; P < .001). Procedures (RR, 0.44; 95% CI, 0.30-0.63; P < .001) and mortality (RR, 0.36; 95% CI, 0.18-0.73; P = .003) were lower in the GLP-1 RA group, but mean (SD) body mass index (BMI) did not differ at follow-up (40.6 [9.2] vs 39.5 [8.7]; P = .10). Sensitivity analysis stratified by BMI (≥40 vs <40) showed similar associations. Bariatric surgery was associated with greater weight loss, but GLP-1 RA therapy was associated with better outcomes.Conclusions and RelevanceGLP-1 RA therapy in IIH is associated with significant reductions in medication use, symptoms/signs, and procedural interventions, suggesting its potential as a management strategy. Further prospective studies are warranted to confirm these findings.","PeriodicalId":14677,"journal":{"name":"JAMA neurology","volume":"275 1","pages":""},"PeriodicalIF":29.0,"publicationDate":"2025-07-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144622147","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}