JAMA neurology最新文献

{"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}

{"title":"Behavioral Compared With Drug Therapy for Overactive Bladder Symptoms in Parkinson Disease: A Randomized Noninferiority Trial.","authors":"Camille P Vaughan,James F Morley,Jessica Lehosit,Gerald McGwin,Lisa Muirhead,Anjali Khakharia,Theodore M Johnson,Marian L Evatt,Taressa Sergent,Kathryn L Burgio,Alayne D Markland","doi":"10.1001/jamaneurol.2025.1904","DOIUrl":"https://doi.org/10.1001/jamaneurol.2025.1904","url":null,"abstract":"ImportanceOveractive bladder (OAB) symptoms in Parkinson disease (PD) are burdensome, and the adverse effects of drug treatment can worsen PD-associated morbidity. Drug adverse effects are avoided with pelvic floor muscle exercise-based behavioral therapy.ObjectiveTo assess the noninferiority of behavioral compared with solifenacin drug therapy for OAB symptoms in persons with PD.Design, Setting, and ParticipantsThis 12-week randomized noninferiority trial of behavioral therapy compared with solifenacin was conducted between 2018 and 2023 within 4 US Veterans Affairs health care systems. Eligible participants were diagnosed with PD by a movement disorder neurologist and had an International Consultation on Incontinence Questionnaire OAB module (ICIQ-OAB) symptom score of 7 or higher (range, 0-16; higher score indicates worse symptoms) and Montreal Cognitive Assessment (MOCA) score of 18 or higher (range, 0-30). Participants were randomized 1:1 after stratification by sex, recruitment site, OAB severity, and PD motor symptom severity. Analyses were conducted from October 2023 to April 2024.InterventionsBehavioral therapy was implemented by a nurse practitioner and included pelvic floor muscle training and urge suppression strategies. Solifenacin therapy started at 5 mg daily, with titration to 10 mg daily if needed.Main Outcome and MeasuresThe primary outcome was the 12-week ICIQ-OAB score across groups within a 15% noninferiority margin. Adverse events were assessed every 2 weeks for 8 weeks and again at 12 weeks.ResultsA total of 77 persons with PD (65 [84%] male; mean [SD] age, 71.3 [8.9] years; mean [SD] years with PD, 6.6 [5.8]) were randomized to behavioral (n = 36) or drug therapy (n = 41). Seventy-three participants completed the study (4 dropped out in drug group). Baseline characteristics were balanced across groups, including MOCA score (mean [SD], drug, 23.9 [3.1]; behavioral, 24.8 [3.3]) and ICIQ-OAB score (mean [SD], drug, 9.1 [1.7]; behavioral, 8.5 [1.4]). At 12 weeks postrandomization, ICIQ-OAB scores across groups indicated clinically significant improvement and were within the a priori noninferiority margin of 15% (mean [SD] score, drug, 5.8 [2.4]; behavioral, 5.5 [2.0]; P = .02). Dry mouth and falls were reported more frequently in the drug compared with the behavioral group.Conclusions and RelevanceResults of this randomized noninferiority trial suggest that behavioral therapy is noninferior to drug therapy in improving OAB symptoms in PD. These findings may inform clinical guidelines for urinary symptoms in PD to consider behavioral therapy as an initial treatment option.Trial RegistrationClinicalTrials.gov Identifier: NCT03149809.","PeriodicalId":14677,"journal":{"name":"JAMA neurology","volume":"19 1","pages":""},"PeriodicalIF":29.0,"publicationDate":"2025-07-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144622146","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":"Isolated Hepatitis B Virus-A Rare Cause of Brain Damage.","authors":"Xiaoting Zheng,Huifang Shang,Ling Liu","doi":"10.1001/jamaneurol.2025.2201","DOIUrl":"https://doi.org/10.1001/jamaneurol.2025.2201","url":null,"abstract":"","PeriodicalId":14677,"journal":{"name":"JAMA neurology","volume":"9 1","pages":""},"PeriodicalIF":29.0,"publicationDate":"2025-07-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144568428","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":"Interstitial Thermal Therapy in Mesial Temporal Lobe Epilepsy.","authors":"Patrick Landazuri,Jennifer J Cheng,Eric Leuthardt,Albert H Kim,Derek G Southwell,Peter E Fecci,Joseph Neimat,David Sun,Bradley Lega,Fedor Panov,Veronica Chiang,Taylor Abel,Sharona Ben-Haim,David E Piccioni,Jerry J Shih,Viktoras Palys,Analiz Rodriguez,S Kathleen Bandt,Joseph Petronio,Michel Lacroix,James Baumgartner","doi":"10.1001/jamaneurol.2025.1897","DOIUrl":"https://doi.org/10.1001/jamaneurol.2025.1897","url":null,"abstract":"ImportanceLaser interstitial thermal therapy (LITT) is a surgical tool used to ablate epileptic foci and brain tumors. Understanding clinical and procedural outcomes of LITT for mesial temporal lobe epilepsy (MTLE) is relevant to clinicians and patients.ObjectiveTo describe seizure outcomes, procedural outcomes, and safety data of MTLE LITT.Design, Setting, and ParticipantsLaser Ablation of Abnormal Neurological Tissue Using Robotic NeuroBlate System (LAANTERN) is a prospective multicenter registry with up to 5 years of follow-up lasting from October 2015 to March 2023 at LAANTERN epilepsy sites, which are all level IV National Association of Epilepsy Centers in the US. Adult and pediatric LAANTERN enrollees undergoing LITT for drug-resistant MTLE with at least 6 months of follow-up were included. Those with epilepsy related to a malignant lesion were excluded.InterventionLITT for drug-resistant MTLE.Main Outcomes and MeasuresDemographic, epilepsy, and seizure characteristics; procedural data; postsurgical seizure outcomes; safety data; and quality of life (QOL) scores were prospectively collected.ResultsFifteen centers enrolled 145 patients (73 [50.3%] female) with MTLE undergoing LITT, with 77 reaching 2-year follow-up. The mean (SD) age was 39.2 (15.4) years at time of LITT with 14 of 145 in the pediatric range (younger than 22 years). The 2 most common etiologies were mesial temporal sclerosis (n = 74) and unknown etiology or magnetic resonance imaging normal (n = 31). Mean (SD) ablation volume was 28.2 (29.8) mL. Mean (SD) surgery duration was 4.3 (2.1) hours, and mean (SD) blood loss was 22 (17.6) mL. Median (IQR) length of stay was 1 (1-3) day, and 33 patients (23%) had no intensive care unit stay postprocedure. Median (IQR) intensive care unit time was 22 (19.2-28.8) hours. Mean (SD) discharge head pain score was 2.1 (2.6) on a 0-10 scale. Most patients (n = 140 [96.6%]) were discharged home. Two-year seizure outcomes were 45 of 77 (58.4%) and 44 of 77 (57.2%) for Engel 1 and International League Against Epilepsy 1/2, respectively. No clinical characteristics were associated with seizure outcome. Adverse events were seen in 24 patients (16.5%), most being mild and transient. Pediatric seizure outcomes were similar to adult outcomes. One-third of patients stopped or decreased their antiseizure medicines. Improvements in QOL scores were seen at almost all time points assessed.ConclusionsIn the largest prospective multicenter MTLE LITT cohort, LITT was found to be well tolerated with clinically meaningful seizure outcomes and QOL improvements. These findings indicate that LITT may be considered as a treatment option for drug-resistant MTLE.","PeriodicalId":14677,"journal":{"name":"JAMA neurology","volume":"31 1","pages":""},"PeriodicalIF":29.0,"publicationDate":"2025-07-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144568427","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}