Neurology and Therapy最新文献

{"title":"Profiles of Medicaid Beneficiaries with Treatment-Resistant Depression Initiated on Esketamine Nasal Spray.","authors":"Kristin Clemens, Maryia Zhdanava, Amanda Teeple, Arthur Voegel, Aditi Shah, Hannah E Bowrey, Anabelle Tardif-Samson, Dominic Pilon, Kruti Joshi","doi":"10.1007/s40120-025-00802-1","DOIUrl":"10.1007/s40120-025-00802-1","url":null,"abstract":"<p><strong>Introduction: </strong>While the literature on esketamine use in commercially insured patients with treatment-resistant depression (TRD) is growing, data on Medicaid beneficiaries remain limited. This study aimed to fill this gap by exploring characteristics of Medicaid beneficiaries with TRD who initiated esketamine treatment in the United States.</p><p><strong>Methods: </strong>Adults with TRD initiating esketamine on/after 03/05/2019 (index date) were selected from the Merative™ MarketScan^® Multi-State Medicaid Database (01/2016-06/2022). Baseline was 12 months pre-index date; follow-up spanned from the index date until the end of health plan insurance or data. Patient baseline characteristics and follow-up treatment patterns, healthcare resource utilization (HRU), and medical costs were reported.</p><p><strong>Results: </strong>A total of 151 patients were identified (mean age: 40.6 years; female: 70.2%; racial minorities: 31.1%). The most common comorbidities were anxiety (81.5%), sleep-wake disorders (47.0%), trauma-related disorders (43.0%), substance-related disorders (30.5%), and obesity (30.5%). Patients completed a mean of 22.5 esketamine sessions; 67.5% of patients completed the induction phase (≥ 8 sessions), 62.3% initiated the maintenance phase, and 53.0% completed ≥ 12 sessions. After esketamine initiation, patients had a mean of 0.02 inpatient admissions, 0.09 inpatient days, 0.31 emergency department visits, and 5.85 outpatient visits monthly. The mean follow-up medical costs were $2075 monthly, including inpatient costs of $97, emergency department costs of $51, and outpatient costs of $1902.</p><p><strong>Conclusion: </strong>Medicaid beneficiaries with TRD who initiated esketamine treatment had a high comorbidity burden and a higher proportion of racial minorities than the US population overall. Nonetheless, most patients progressed to maintenance phase of esketamine treatment. Findings suggest that esketamine may be a viable treatment option for this complex patient population.</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":"2125-2135"},"PeriodicalIF":4.8,"publicationDate":"2025-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12450134/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144855816","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Levodopa Intestinal Gel Infusion Therapies in Advanced Parkinson's Disease: A Swedish Study on Real-World Use and Costs.","authors":"Per Odin, Christoffer Tærud, Jenny Samuelsson, Emma Sabelström, Jeanette Lagerlund, Jonatan Freilich, M Natalia Stelmaszuk","doi":"10.1007/s40120-025-00766-2","DOIUrl":"10.1007/s40120-025-00766-2","url":null,"abstract":"<p><strong>Introduction: </strong>This study evaluated real-world cassette use and cost of levodopa-carbidopa intestinal gel (LCIG; 2000 mg levodopa equivalent dose [LED]) and levodopa-entacapone-carbidopa intestinal gel (LECIG; 1250 mg LED) pump treatments among patients with advanced Parkinson's disease (PD) in Sweden.</p><p><strong>Methods: </strong>This was a non-interventional, longitudinal, retrospective, comparative study of patients with PD using data from Swedish national registries (National Patient Register [NPR] and Prescribed Drug Register [PDR]). Patients were enrolled in the study from January 1, 2017, to May 31, 2022 (NPR) or June 30, 2022 (PDR). Patients had two or more dispensed prescription records for either LCIG (January 1, 2017, onward) or LECIG (January 1, 2019, onward) and at least one diagnosis of PD on or before their first dispensed prescription. Average daily cassette use, costs (Swedish krona [SEK]), treatment persistence, and treatment patterns were assessed.</p><p><strong>Results: </strong>Overall, 419 patients (LCIG, n = 276; LECIG, n = 180; both LCIG and LECIG, n = 37 [cohorts not mutually exclusive]) were included. Mean (SD) daily cassette use was significantly higher for LECIG than for LCIG: 1.28 (0.40) versus 1.09 (0.28) at 1-365 days (p < 0.0001). LECIG use was higher than LCIG at 365 days regardless of prior levodopa pump experience. The overall cost of LECIG was higher than that of LCIG; the highest cost difference between treatments was at 365 days (daily cost mean [SD], LCIG 991.23 [259.43] SEK vs. LECIG 1100.23 [341.04] SEK; p = 0.0036). Annual per-patient costs were approximately 40,000 SEK higher for LECIG versus LCIG. More patients discontinued LECIG than LCIG treatment, with 68.9% and 75.2% of those receiving LECIG and LCIG, respectively, still on treatment at 365 days.</p><p><strong>Conclusion: </strong>Real-world data show that the number of dispensed cassettes and overall treatment costs are higher for LECIG than LCIG treatment among patients with advanced PD in Sweden.</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":"1845-1860"},"PeriodicalIF":4.8,"publicationDate":"2025-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12450165/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144258546","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Retrospective Multicenter Chart Review Study of Adjunctive Cannabidiol for Seizures Associated with Lennox-Gastaut Syndrome, Dravet Syndrome and Tuberous Sclerosis Complex.","authors":"Adam Strzelczyk, Kerstin Alexandra Klotz, Thomas Mayer, Felix von Podewils, Susanne Knake, Gerhard Kurlemann, Luise Herold, Ilka Immisch, Elisa Buhleier, Felix Rosenow, Susanne Schubert-Bast","doi":"10.1007/s40120-025-00788-w","DOIUrl":"10.1007/s40120-025-00788-w","url":null,"abstract":"<p><strong>Introduction: </strong>Effectiveness and tolerability of plant-derived highly purified cannabidiol (CBD) in patients with Lennox-Gastaut syndrome (LGS), Dravet syndrome (DS), or tuberous sclerosis complex (TSC)-associated epilepsy in clinical practice in Germany were evaluated.</p><p><strong>Methods: </strong>This multicenter, retrospective, chart review study analyzed patients with LGS, DS, or TSC-associated epilepsy receiving ≥ 1 dose of adjunctive CBD (Epidyolex^® 100 mg/mL oral solution). Treatment characteristics, seizure outcomes, physician-rated Clinical Global Impression of Change (CGI-C), treatment retention rates, and adverse events (AEs) were analyzed ≤ 12 months.</p><p><strong>Results: </strong>Among 202 patients identified (159 LGS; 34 DS; 9 TSC), median (interquartile range; range) age was 18.0 (7.9-32.0; 0.3-72.0) years, and median (range) number of prior and concomitant antiseizure medications was 6 (1-24) and 3 (1-7), respectively. Median target CBD dose was 11.1 mg/kg/day (17.6, 15.2, and 9.9 mg/kg/day in the < 6, 6-17, and ≥ 18 years subgroups, respectively). Responder rates (≥ 50% seizure reduction) for total seizures at 3 (n = 194) and 12 (n = 168) months were 43.3% (37.0-50.0% across ages) and 44.0% (37.0-52.5% across ages), respectively, and for generalized tonic-clonic seizures 54.3% (n = 94) (50.0-66.7% across ages) and 47.7% (n = 88) (37.8-66.7% across ages), respectively. Median (range) number of seizure days per month significantly decreased from 30 (0.3-30) to 18 (0-30) in the 3 months before the last 3 months of CBD treatment (p < 0.001). Any improvement in CGI-C was observed in 62% of patients. Of those with available data at 3 and 12 months, 89.6% and 67.1% remained on CBD, respectively. Retention was similar across age groups. AEs reported in ≥ 5% of patients were sedation and diarrhea.</p><p><strong>Conclusions: </strong>In patients with LGS, DS, or TSC-associated epilepsy, adjunctive CBD was associated with a reduction in seizure frequency across age groups. CBD demonstrated tolerability consistent with its known profile, and 67% of patients remained on treatment at 12 months.</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":"1935-1959"},"PeriodicalIF":4.8,"publicationDate":"2025-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12450151/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144619470","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Neuroprotection in Parkinson Disease.","authors":"Cristina Gonzalez-Robles, Oliver Bandmann, Anthony H V Schapira","doi":"10.1007/s40120-025-00793-z","DOIUrl":"10.1007/s40120-025-00793-z","url":null,"abstract":"<p><p>Parkinson disease (PD) is a progressive neurodegenerative condition characterised by tremor, bradykinesia and rigidity, as well as other motor and non-motor symptoms, for which no effective disease-modifying treatments have been discovered. Neuroprotection in PD is limited by its clinical and biological heterogeneity, suboptimal preclinical models, lack of established disease progression biomarkers, complex pathophysiology, the existence of effective symptomatic therapies which hamper the detection of actual disease modification, and trial design. This review discusses the above issues and other important concepts in neuroprotection in PD. The main pathophysiological mechanisms in PD are classified into mitochondrial dysfunction, lysosomal dysfunction, inflammation, protein aggregation/propagation, and \"other\", and discussed briefly. The most relevant disease-modifying candidates in PD are classified into the aforementioned categories and reviewed. Finally, conclusions and recommendations for future improvements in the field of disease modification in PD are provided.</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":"1747-1767"},"PeriodicalIF":4.8,"publicationDate":"2025-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12450203/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144659710","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Clinical Performance of the Elecsys CSF pTau₁₈₁/Aβ₄₂ Ratio for Concordance with Tau-PET in Two Independent Cohorts.","authors":"Ruben Smith, Leslie Shaw, Sebastian Palmqvist, Niklas Mattsson-Carlgren, Gregory Klein, Matteo Tonietto, Clara Quijano-Rubio, Christopher M Rank, Myrto Andreadou, Samantha C Burnham, Erik Stomrud","doi":"10.1007/s40120-025-00798-8","DOIUrl":"10.1007/s40120-025-00798-8","url":null,"abstract":"<p><strong>Introduction: </strong>Amyloid- and tau-positron emission tomography (PET) are promising modalities for detecting pathological changes associated with Alzheimer's disease (AD); however, their application is limited. Although the use of cerebrospinal fluid (CSF) biomarkers as alternatives to amyloid-PET and tau-PET has been explored, no in vitro diagnostic-approved or commercial CSF biomarker assays are currently available for detecting tau pathology in clinical practice.</p><p><strong>Methods: </strong>In this study, we determined and validated the optimal cutoff value for the ratio of tau phosphorylated at a threonine residue at position 181 (pTau₁₈₁) to β-amyloid(1-42) (Aβ₄₂) in CSF, measured with the Elecsys^® Phospho-Tau (181P) CSF and β-Amyloid(1-42) CSF immunoassays (Roche Diagnostics International Ltd, Rotkreuz, Switzerland), based on its concordance with binary tau-PET status. Clinical performance was explored using CSF measurements and tau-PET scans retrospectively obtained from a subset of subjects with mild cognitive impairment and dementia due to AD in two independent cohorts, Alzheimer's Disease Neuroimaging Initiative-2/3 (ADNI-2/3; N = 133) and Swedish BioFINDER-2 (N = 62).</p><p><strong>Results: </strong>In the first part of this analysis (ADNI-2/3 pre-analytics), a CSF pTau₁₈₁/Aβ₄₂ cutoff value of 0.0395 was selected as the best compromise between positive percent agreement (PPA) and negative percent agreement (NPA) for the tau-PET visual read endpoint. After adjustment to account for differences between the ADNI-specific protocol and the manufacturer's recommended pre-analytical protocol used in BioFINDER-2, the optimal CSF pTau₁₈₁/Aβ₄₂ cutoff value was set at 0.037. The adjusted cutoff was validated in BioFINDER-2 and was associated with a PPA of 85.7% (95% confidence interval [CI] 70.6, 93.7), NPA of 70.4% (95% CI 51.5, 84.1), and overall percent agreement (OPA) of 79.0% (95% CI 67.4, 87.3); the positive and negative likelihood ratios were 2.89 and 0.203, respectively.</p><p><strong>Conclusion: </strong>The Elecsys CSF pTau₁₈₁/Aβ₄₂ ratio may be a reliable tool for identifying tau pathology in clinical practice.</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":"2011-2031"},"PeriodicalIF":4.8,"publicationDate":"2025-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12450182/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144668096","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Machine Learning for Predicting Thromboembolic Events Following Flow Diverter Treatment of Intracranial Aneurysms: A Multicenter Retrospective Study.","authors":"Yunpeng Lin, Xiaoning Liu, Bingcheng Ren, Jiwen Wang, Yang Li, Xiangbo Liu, Yidi Wang, Fushun Xiao, Shiqing Mu","doi":"10.1007/s40120-025-00808-9","DOIUrl":"10.1007/s40120-025-00808-9","url":null,"abstract":"<p><strong>Introduction: </strong>Flow diverters (FD) have gradually become the preferred treatment option for complex and large intracranial aneurysms. Postoperative thromboembolic events (TEEs) are among the most common complications associated with endovascular treatment. However, widely applicable predictive tools for the occurrence of TEEs are currently lacking.</p><p><strong>Methods: </strong>This retrospective study included clinical data from 377 patients (a total of 451 aneurysms) treated with flow diverters at two neurointerventional centers between June 2018 and September 2022. Thirty-nine baseline patient characteristics were included as clinical variables. The primary endpoint was the occurrence of postoperative ischemic events. The dataset was randomly divided into a training set (80%) and a testing set (20%). We performed fivefold cross-validation and applied Lasso regression to the training set to identify the most informative features. Multiple machine learning (ML) algorithms were employed to construct predictive models. Model performance was evaluated on the testing set using the area under the receiver operating characteristic curve (AUC-ROC), the area under the precision-recall curve (AUC-PR), and calibration plots. SHapley Additive exPlanations (SHAP) analysis was used to visualize feature contributions and to interpret individual case predictions.</p><p><strong>Results: </strong>Among 377 patients, 21 (5.6%) experienced TEEs. A machine learning model incorporating 10 variables was developed, with the support vector machine (SVM) model demonstrating the best performance-achieving an AUC-ROC of 0.96 and an AUC-PR of 0.88 in validation. The key predictive factors included aneurysm width, low-density lipoprotein (LDL) levels, hypertension, aneurysm location, triglycerides (TG), and diabetes. Additionally, a web-based tool was developed to assist clinicians in applying the model in practice.</p><p><strong>Conclusions: </strong>We developed a machine learning model to predict the risk of TEEs following FD implantation for intracranial aneurysms, and demonstrated its clinical potential through internal validation. This tool can assist neurointerventionalists in estimating the probability of TEE occurrence based on patient clinical data and aneurysm characteristics, enabling the development of personalized treatment strategies.</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":"2171-2185"},"PeriodicalIF":4.8,"publicationDate":"2025-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12450181/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144962848","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Perspective of Disease and Treatment in Patients with Multiple Sclerosis: A Polish Multi-center Study.","authors":"Justyna Chojdak-Łukasiewicz, Alina Kułakowska, Joanna Tarasiuk, Katarzyna Kapica-Topczewska, Joanna Siuda, Weronika Galus, Maja Patalong-Ogiewa, Aleksandra Kaczmarczyk, Anetta Lasek-Bal, Ewa Krzystanek, Arkadiusz Stęposz, Aleksandra Karuga, Elżbieta Jasińska, Katarzyna Gołuch, Waldemar Brola, Martyna Odzimek, Agnieszka Słowik, Klaudia Kaczówka, Marcin Wnuk, Monika Marona, Konrad Rejdak, Anna Jamroz-Wiśniewska, Mariusz Stasiołek, Bartosz Bielecki, Mariola Świderek-Matysiak, Małgorzata Siger, Andrzej Głąbiński, Marta Milewska-Jędrzejczak, Beata Łabuz-Roszak, Małgorzata Gardzińska, Maja Sakowska, Halina Bartosik-Psujek, Iwona Rościszewska-Żukowska, Małgorzata Popiel, Julia Rudnicka-Czerwiec, Andrzej Potemkowski, Monika Adamczyk-Sowa, Katarzyna Kubicka-Bączyk, Natalia Niedziela, Aleksandra Kołtuniuk, Sławomir Budrewicz, Anna Pokryszko-Dragan","doi":"10.1007/s40120-025-00817-8","DOIUrl":"https://doi.org/10.1007/s40120-025-00817-8","url":null,"abstract":"<p><strong>Introduction: </strong>Due to recent progress in multiple sclerosis (MS) research, a range of disease-modifying therapies (DMT) is increasingly available. According to the personalized therapeutic approach, the choice of DMT for a particular patient is based on complex analysis of disease-related and drug-related aspects, with emphasis on patient's preferences and shared decision-making. The aim of this study was to evaluate the perspective of the disease and various aspects of treatment in Polish patients with MS (pwMS), with reference to sociodemographic and clinical data.</p><p><strong>Methods: </strong>The nationwide survey was conducted, addressed to adult pwMS treated with DMT and undergoing regular follow-up in regional MS Centers. The questionnaire contained sociodemographic data, and questions about major troublesome and feared aspects of disease, and about the importance of various aspects of treatment. In addition, MS-related data were provided by neurologists. The responses have been summarized and analyzed for their relationships with sociodemographic and clinical data.</p><p><strong>Results: </strong>A total of 2032 pwMS (70% women; mean age 42.1 ± 10.8 years) were included from 14 MS Centers. Over 90% had relapsing-remitting MS, mean disease duration was 12 years and the median Expanded Disability Status Scale (EDSS) was 2.4 ± 1.5. Fatigue (50%), limb weakness (47%), and balance and gait disturbances (30%) were the most common and troublesome symptoms reported by the respondents. Their main concerns about disease consequences included disability (46%) and dependence on others (17%). All aspects of DMT efficacy were very important for more than 70% of patients, with preventing disability progression, maintaining social participation, and reduction of relapses as top priorities (86-95%). Treatment safety concerns were focused on risk of cancer (74%), effect on comorbidities (63%), and severe infections (60%). Drug efficacy (93%), modernity (60%), and mechanism of action (59%) were most commonly indicated factors influencing patients' preference for DMT. Significant relationships were found between pwMS opinion about aspects of treatment and their age, sex, family status and vocational activity, as well as type and duration of MS, EDSS score, and type of DMT used.</p><p><strong>Conclusion: </strong>The Polish pwMS perspective of disease is focused on emerging disability and its social context. Regarding aspects of treatment, sufferers are highly concerned about its efficacy (especially in preventing the mentioned disease consequences), followed by safety and convenience. Individual differences in patients' responses should be highlighted, associated with combined impact of demographic and clinical data. The study findings should inform complex and personalized therapeutic approaches to MS management in clinical practice.</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":""},"PeriodicalIF":4.8,"publicationDate":"2025-09-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145200491","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Safety and Effectiveness of Satralizumab in Japanese Patients with Neuromyelitis Optica Spectrum Disorder: A 30‑Month Interim Analysis of Post‑marketing Surveillance.","authors":"Takashi Yamamura, Noriko Isobe, Izumi Kawachi, Chiyoko Nohara, Yusei Miyazaki, Minami Tomita, Yuta Kamei, Katsuhisa Yamashita, Jin Nakahara, Ichiro Nakashima, Kazuo Fujihara","doi":"10.1007/s40120-025-00799-7","DOIUrl":"https://doi.org/10.1007/s40120-025-00799-7","url":null,"abstract":"<p><strong>Introduction: </strong>A large-scale post-marketing surveillance (PMS) study is ongoing to evaluate the safety and effectiveness of satralizumab over 6 years in Japanese patients with neuromyelitis optica spectrum disorder (NMOSD) in real-world settings. We present the results of a 30-month interim analysis of the study.</p><p><strong>Methods: </strong>This PMS is being conducted across 234 sites in Japan. In this 30-month interim analysis, the end of the observation period was defined as either the date of data lock for the 30-month case report form for all patients or the last observation date for patients with satralizumab discontinuation. Primary outcomes include proportion of patients experiencing adverse drug reactions (ADRs), event rate, and oral glucocorticoid use. Secondary outcomes include time to relapse and relapse rate.</p><p><strong>Results: </strong>Of 571 patients included (mean age: 52.4 years), 91.76% were female. At baseline, 85.98% of patients received oral glucocorticoids. ADRs were reported in 28.72% of patients (event rate: 27.69 events/100 person-years), with infections being most common (11.73%; 8.97 events/100 person-years). Univariate analysis showed that at 30 months, serious infections occurred in 6.83% of patients (5.00 events/100 person-years vs 8.13 events/100 person-years during 0-6 months) and were more frequent in patients aged ≥ 75 years, with diagnosis-to-treatment initiation duration ≥ 10 years, ≥ 3 relapses within 2 years, and Expanded Disability Status Scale score ≥ 6. Mean glucocorticoid dose decreased from 12.27 mg/day (baseline) to 3.48 mg/day (30 months). Kaplan-Meier cumulative relapse-free rate was 85.86% at 30 months. The annualized relapse rate was 0.08/person-year. Overall, 9.63% and 3.50% of patients discontinued treatment because of adverse events and relapses, respectively.</p><p><strong>Conclusion: </strong>Serious infections were more common during the satralizumab treatment period, occurring most frequently within the first 6 months, highlighting the need for continuous monitoring of infections throughout satralizumab treatment. Satralizumab was found to be safe, without new safety concerns over 30 months. A reduction in concomitant immunosuppressive therapy usage was observed. The study demonstrated the effectiveness of satralizumab in preventing relapses in Japanese patients with NMOSD.</p><p><strong>Trial registration: </strong>UMIN Clinical Trials Registry, UMIN000041047.</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":""},"PeriodicalIF":4.8,"publicationDate":"2025-09-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145150111","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Impact of Adjunctive Istradefylline on Dyskinesia Onset in Patients with Parkinson's Disease Exhibiting Wearing-Off: An Open-Label Randomized Controlled Trial.","authors":"Yoshio Tsuboi, Takafumi Hasegawa, Yasushi Shimo, Satoshi Kaneko, Masahiko Tomiyama, Kenichi Kashihara, Shih-Wei Chiu, Takuhiro Yamaguchi","doi":"10.1007/s40120-025-00809-8","DOIUrl":"https://doi.org/10.1007/s40120-025-00809-8","url":null,"abstract":"<p><strong>Introduction: </strong>Motor complications become major treatment challenges in patients with Parkinson's disease (PD). Istradefylline (IST) is used as an adjunct to levodopa in patients with PD exhibiting wearing-off (WO), but its impact on the onset of dyskinesia remains unclear. The objective of this study was to investigate the effect of IST on dyskinesia onset in patients with PD exhibiting WO.</p><p><strong>Methods: </strong>In this 3-year, multicenter, randomized, open-label, parallel-group, controlled study, 214 patients with levodopa-treated PD exhibiting WO without pre-existing dyskinesia were randomized (1:1) to either adjunctive IST (IST group) or to adding another or increasing the dose of other anti-PD drugs (non-IST group). The primary endpoint was the time to onset of dyskinesia.</p><p><strong>Results: </strong>Over 3 years, the incidence of dyskinesia was 37.9% and 41.1% in the IST and non-IST groups, respectively. The time to onset of dyskinesia was not significantly different between the IST versus non-IST groups (median: 1100.00 vs. 1082.00 days). When dividing patients by age, the rate of time to onset of dyskinesia was lower in the IST group in patients aged ≥ 68 years (median) and in the non-IST group for patients aged < 68 years and patients aged < 60 years at PD onset. The levodopa-equivalent daily dose (LEDD) was significantly lower throughout the study period, by 57 mg on average, in the IST group than in the non-IST group. Efficacy and safety indexes were not significantly different between the two groups.</p><p><strong>Conclusion: </strong>Adjunctive IST showed no difference in the onset of dyskinesia compared with adding another or increasing the dose of other anti-PD drugs, and displayed equivalent efficacy and tolerability, while maintaining a lower LEDD in this long-term study of patients with PD exhibiting WO. These findings support the use of adjunctive IST as a viable treatment option for patients with PD exhibiting WO.</p><p><strong>Trial registration: </strong>University hospital Medical Information Network clinical trials registry (UMIN000024536) and Japan Registry of Clinical Trials (jRCTs071180014).</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":""},"PeriodicalIF":4.8,"publicationDate":"2025-09-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145131400","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Neurofilament Light Chain Levels as Diagnostic and Prognostic Biomarkers in Guillain-Barré Syndrome: An Updated Systematic Review and Meta-Analysis.","authors":"Giovanni Siconolfi, Francesca Vitali, Maria Ausilia Sciarrone, Valeria Guglielmino, Guido Primiano, Marco Luigetti","doi":"10.1007/s40120-025-00830-x","DOIUrl":"https://doi.org/10.1007/s40120-025-00830-x","url":null,"abstract":"<p><strong>Introduction: </strong>Guillain-Barré syndrome (GBS) is an acute immune-mediated disorder of the peripheral nervous system, marked by rapid onset of neurological symptoms. Despite progress in understanding the etiology and improving clinical management, no validated biomarkers are currently available to predict disease severity or treatment response during the acute phase. This meta-analysis aims to evaluate the role of serum neurofilament light chain (NfL) as a biomarker of acute disease activity and prognostic outcomes in GBS.</p><p><strong>Methods: </strong>A systematic review and meta-analysis was conducted using PubMed, Scopus, and Cochrane Library databases to identify studies assessing NfL levels in patients with GBS. In addition, we included data from our own cohort of patients with GBS-whose NfL levels were measured at disease onset-and from healthy controls. The primary outcome was the difference in NfL levels-both in serum and cerebrospinal fluid (CSF)-between patients with GBS and controls. Secondary outcomes included the correlations between acute-phase NfL levels, clinical severity at admission as measured by the Guillain-Barré Disability Scale (GBDS) or the Hughes Functional Scale (HFS), and long-term outcomes such as the inability to walk or run 1 year after disease onset.</p><p><strong>Results: </strong>In this meta-analysis of nine studies, which also included data from our cohort, serum NfL levels were significantly higher in patients with GBS compared with controls (mean difference 143.17 pg/mL, 95% CI 67.7-218.6; p < 0.01; I² = 83%). In contrast, the difference in CSF NfL levels only approached statistical significance (mean difference 2091.1 pg/mL, 95% CI 171.2-4353.4; p = 0.07, I² = 92.1%). These findings were corroborated in our cohort, where median serum NfL concentrations were markedly higher in patients with GBS compared to controls (97 pg/mL, IQR 79-194 vs. 15 pg/mL, IQR 13-20; p < 0.05, Wilcoxon rank-sum test). Serum NfL levels were higher in patients with the acute motor axonal neuropathy (AMAN) compared to those with acute inflammatory demyelinating polyneuropathy (AIDP) (MD 531.9 pg/mL, 95% CI 32.8-1031.01; I² = 81.1%; p = 0.04). Moreover, NfL levels positively correlated with disease severity at admission (r = 0.38; p < 0.001) and poor long-term outcomes (OR 3.74, 95% CI 1.05-13.37; p < 0.001).</p><p><strong>Conclusion: </strong>Serum NfL is a promising biomarker for early diagnosis and prognosis in GBS and may support risk stratification at hospital admission.</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":""},"PeriodicalIF":4.8,"publicationDate":"2025-09-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145113843","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}