Neurology and Therapy最新文献

{"title":"Expert-Agreed Practical Recommendations on the Use of Fenfluramine in Developmental and Epileptic Encephalopathies Based on Clinical Experience and Literature Review.","authors":"Vicente Villanueva, Victor Soto-Insuga, Patricia Smeyers, Ángel Aledo-Serrano, Rocío Sánchez-Carpintero, Juan García-Peñas, Antonio Gil-Nagel","doi":"10.1007/s40120-025-00713-1","DOIUrl":"10.1007/s40120-025-00713-1","url":null,"abstract":"<p><strong>Introduction: </strong>Fenfluramine (FFA) represents the latest therapeutic option approved for seizure management in Dravet syndrome (DS) and Lennox-Gastaut syndrome (LGS) for patients aged ≥ 2 years. This article provides expert guidance for optimizing FFA therapy to support clinical decision-making in these populations.</p><p><strong>Methods: </strong>A panel of Spanish experts specialized in developmental epileptic encephalopathies (DEEs) has developed practical recommendations for the clinical use of FFA, focusing on key aspects of FFA management: mechanism of action, pharmacokinetics including drug interactions, titration, efficacy, safety and tolerability, contraindications, and considerations for its broader application in DEEs. The methodology adopted in this project was an expert-opinion, evidence-based approach.</p><p><strong>Results: </strong>The panel issued targeted recommendations, including a modified titration strategy slower than the product guidelines, adjusted for possible antiseizure concomitant medications, and management of other concomitant treatments. Key efficacy indicators, such as reductions in seizure frequency and severity of the most disabling seizures, were emphasized as core measures for treatment evaluation. Periodic assessments of non-seizure outcomes and daily life activities are recommended during follow-up to comprehensively capture treatment outcomes. The panel noted that their clinical observations align with positive findings from clinical trials, suggesting a potential role for FFA in other DEEs, tailored to individual electroclinical and etiological profiles.</p><p><strong>Conclusion: </strong>This article presents expert practical recommendations for the management and treatment optimization of FFA in patients with DEEs, supporting clinicians in achieving improved patient outcomes.</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":"447-465"},"PeriodicalIF":3.9,"publicationDate":"2025-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11906945/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143483591","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":"Measurement Properties of the Friedreich Ataxia Rating Scale in Patients with Spinocerebellar Ataxia.","authors":"Michele H Potashman, Evan Popoff, Lauren C Powell, Melissa Wolfe Beiner, Ainsley Mackenzie, Vlad Coric, Sub Subramony, Matthis Synofzik, Jeremy Schmahmann, Gilbert L'Italien","doi":"10.1007/s40120-024-00708-4","DOIUrl":"10.1007/s40120-024-00708-4","url":null,"abstract":"<p><strong>Introduction: </strong>The Friedreich Ataxia Rating Scale-Activities of Daily Living (FARS-ADL) is a valid, highly utilized measure for assessing ADL impacts in patients with Friedreich ataxia. We provide evidence of the psychometric validity of the FARS-ADL in two cohorts of patients with spinocerebellar ataxia (SCA).</p><p><strong>Methods: </strong>Using data from a cohort of real-world subjects with SCA (recruited at Massachusetts General Hospital [MGH]; n = 33) and a phase 3 trial of troriluzole in adults with SCA (NCT03701399 [Study 206]; n = 217), comprising a subset of patients with the SCA3 genotype (n = 89), the psychometric measurement properties and minimal change thresholds of the FARS-ADL were examined.</p><p><strong>Results: </strong>Ceiling effects for the FARS-ADL were absent within the MGH cohort while floor effects were observed for eight of nine items. Excellent internal consistency reliability was observed (α_total = 0.88; α_{items-removed} = 0.86-0.87), and item-to-total correlations were acceptable (r = 0.55-0.89 per item). Convergent and divergent validity were supported with strong correlations demonstrated between FARS-ADL and scales measuring similar concepts (Neuro-QOL [Upper], Neuro-QOL [Lower], PROM-ADL, PROM-PHYS, and FARS-FUNC; all P < 0.001) and weaker correlations shown between measures of differing constructs. A two- to three-point threshold for meaningful changes was supported as 0.5 × SD = 2.43, SEM = 2.19. Mean changes from baseline for subjects classified as \"improved,\" \"no change,\" or \"deteriorated\" were -0.54, 0.22, and 1.47, respectively. Similar trends were observed in the Study 206 all-SCA and SCA3 cohorts.</p><p><strong>Conclusion: </strong>Psychometric evaluation showed that the FARS-ADL performed well on analyses examining the reliability and validity of the measure and can detect meaningful changes in patients with SCA, including those with SCA3.</p><p><strong>Trial registration: </strong>ClinicalTrials.gov identifier, NCT03701399 (Study 206).</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":"527-545"},"PeriodicalIF":3.9,"publicationDate":"2025-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11906947/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142979326","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":"Efficacy and Safety of Various Intravenous Thrombolytics for Acute Ischemic Stroke (AIS) at Various Dosages: A Systematic Review and Network Meta-Analysis.","authors":"Yibin Hu, Shengxian Wu, Haixuan Zhang, Kangfeng Wang, Lijuan Zhang, Yizheng Ma, He Li","doi":"10.1007/s40120-024-00684-9","DOIUrl":"10.1007/s40120-024-00684-9","url":null,"abstract":"<p><strong>Background: </strong>Currently, there is limited evidence on the efficacy and safety of various thrombolytic drugs at different dosages for the treatment of acute ischemic stroke (AIS). From current randomized clinical trials, the optimal type and dosage of thrombolytics for patients with AIS are unclear.</p><p><strong>Methods: </strong>This systematic review was registered in PROSPERO (CRD42024563757). We searched four databases using a combination of keywords that contained various intravenous thrombolytics, as well as acute ischemic stroke. Only data from participants with AIS treated with various intravenous thrombolytics within the 4.5-h time window were included. Among initially identified studies, 16 met the selection criteria. Network meta-analysis was conducted for efficacy (90 day modified Rankin scale score) and safety (intracranial hemorrhage events, mortality at 90 days) using Stata 17.0 software, with a fixed-effects model. Cochrane risk of bias tool assessed all risk of bias domains, and the CINeMA Evidence Assessment Tool evaluated the level of evidence for each outcome.</p><p><strong>Results: </strong>A total of 9056 studies were retrieved through the literature search, and 12,792 patients screened from 16 randomized controlled trials were included in the network meta-analysis. The risk of bias in the included studies ranged from moderate to low. The network meta-analysis results indicated that reteplase at 18 + 18 mg ranked highest in efficacy, though its safety was lower compared to 0.25 mg/kg tenecteplase and alteplase. The dose of 0.25 mg/kg tenecteplase emerged as the optimal dose, demonstrating both superior efficacy and a lower risk of bleeding compared to alteplase, making it a potential alternative to alteplase. The dose of 50 mg prourokinase was associated with the highest risk of symptomatic intracranial hemorrhage and was inferior to reteplase in terms of both efficacy and safety. The CINeMA Evidence Assessment Tool identified one outcome with a high level of evidence, several with moderate levels, and the remainder with low levels.</p><p><strong>Conclusions: </strong>Reteplase at 18 + 18 mg may be more suitable for patients with lower incidence of adverse events evaluated by physicians. Compared to 0.9 mg/kg alteplase, 0.25 mg/kg tenecteplase is more effective, with the lowest risk of intracranial hemorrhage. However, as tenecteplase's dosages increase (0.32 mg/kg and 0.4 mg/kg), its efficacy in improving neurological deficits decreases, while the risk of intracranial hemorrhage and death (especially at 0.4 mg/kg) increases. Clinicians are supposed to carefully assess the needs of patients with AIS and the risks then choose decent thrombolytics.</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":"491-523"},"PeriodicalIF":3.9,"publicationDate":"2025-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11906929/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142807316","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":"Health-Related Quality of Life and Cognitive Performance During 12-Month Adjunctive Brivaracetam Treatment in Patients with Focal-Onset Seizures: A Prospective, Observational Study in Europe.","authors":"Eduardo Rubio-Nazabal, Marian Majoie, Anne-Liv Schulz, Fiona Brock, Iryna Leunikava, Dimitrios Bourikas, Bernhard J Steinhoff","doi":"10.1007/s40120-024-00698-3","DOIUrl":"10.1007/s40120-024-00698-3","url":null,"abstract":"<p><strong>Introduction: </strong>This analysis aimed to evaluate patient-related outcomes for health-related quality of life (HRQoL) and cognitive performance in patients (≥ 16 years) with focal-onset seizures (FOS), with/without focal to bilateral tonic-clonic seizures, after initiating adjunctive brivaracetam (BRV) in routine clinical practice.</p><p><strong>Methods: </strong>A 12-month, prospective, real-world, noninterventional study in nine European countries (EP0077/NCT02687711) was performed. BRV was prescribed per clinical practice and the European Summary of Product Characteristics. The outcomes evaluated were the Patient Weighted Quality of Life in Epilepsy Inventory-Form 31 (QOLIE-31-P), the Clinical and the Patient's Global Impression of Change (CGIC and PGIC, respectively), and EpiTrack^®. EpiTrack^® scores were categorized into cognitive performance categories (excellent: ≥ 39 points; average: 32-38 points; mildly impaired: 29-31 points; significantly impaired: ≤ 28 points). The change in EpiTrack^® score was evaluated [improvement: increase in score of ≥ 4 points; no change: change in score  of - 2 to 3 points (inclusive); worsening: change in score of at least - 3 points].</p><p><strong>Results: </strong>Full Analysis Set: 541 patients. 46.6% of patients reported a clinically meaningful improvement in QOLIE-31-P total score from baseline to 12 months; the mean change in total score was + 6.2 points (N = 103). Per CGIC (N = 142) and PGIC (N = 148), respectively, 69.0% and 62.8% of patients had improved in overall condition at 12 months versus baseline, while 3.5% and 8.1% had worsened. EpiTrack^® categories at 12 months versus baseline showed improved cognitive performance [baseline (N = 142): significantly impaired 49.3%, mildly impaired 14.8%, average 33.1%, excellent 2.8%; 12 months (N = 61): significantly impaired 36.1%, mildly impaired 4.9%, average 52.5%, excellent 6.6%]. At 12 months, 67.2% of patients showed no significant change from baseline in EpiTrack^® score, 23.0% had improved, and 9.8% had worsened (N = 61).</p><p><strong>Conclusion: </strong>In patients with predominantly difficult-to-treat FOS, BRV add-on was associated with good HRQoL and cognitive functioning. Cognitive functioning remained stable for 12 months after BRV initiation in most patients; nearly one-quarter experienced significant improvements. At 12 months, 46.6% of patients reported clinically meaningful HRQoL improvements, and most showed an improved overall condition.</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":"609-625"},"PeriodicalIF":3.9,"publicationDate":"2025-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11906959/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143458848","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":"Risk of Exacerbation and Level of Healthcare Resource Utilization in Myasthenia Gravis Assessed by Myasthenia Gravis Activities of Daily Living Score.","authors":"Angela Ting, Minjee Park, Oshin Sangha, Mohita Kumar, Jean-François Ricci, Edward Lee, Richard J Nowak","doi":"10.1007/s40120-025-00711-3","DOIUrl":"10.1007/s40120-025-00711-3","url":null,"abstract":"<p><strong>Introduction: </strong>Limited data are available on the relationship between myasthenia gravis (MG) severity and MG exacerbations and healthcare resource utilization (HCRU) following exacerbations. The objective of this study was to assess patient characteristics, exacerbation risk in relation to the MG Activities of Daily Living (MG-ADL) score, and HCRU following exacerbation.</p><p><strong>Methods: </strong>This was a retrospective, cross-sectional, observational study of the patient-reported Myasthenia Gravis Foundation of America Global MG Patient Registry (MGFAPR). Participants were based in the USA, aged ≥ 18 years, had a self-reported MG diagnosis and complete MG-ADL data, and were enrolled between July 1, 2013 and September 30, 2022. Patient demographics, disease characteristics, and HCRU were stratified by MG-ADL score. Negative binomial regression was used to assess the association between MG-ADL score and exacerbation. HCRU for those who had one exacerbation was calculated.</p><p><strong>Results: </strong>In total, 3416 patients (2092 [61.2%] females) were eligible; mean (standard deviation) age at diagnosis was 49.4 (17.4) years. Compared with patients in the groups with lower MG-ADL scores (≤ 7), more patients in the higher MG-ADL groups (> 7) were female, younger at the time of MG diagnosis, Black, unemployed, uninsured, had a greater comorbidity burden, and had a shorter disease duration. A positive association between the number of exacerbations and MG-ADL score was observed at enrollment. For each additional point on the MG-ADL score, the rate of exacerbations increased by 13% (incidence rate ratio: 1.13; 95% confidence interval: 1.11-1.15; p < 0.001). At enrollment, 49.6% (n = 386/778) of patients who had one exacerbation had HCRU.</p><p><strong>Conclusions: </strong>We found socio-demographic disparities in disease severity, a higher comorbidity burden, and an increased MG exacerbation risk with higher MG-ADL scores, with a significant impact of MG exacerbation on HCRU. These results highlight the relationship of MG-ADL score to clinical outcomes and the need for treatment optimization and personalized approaches to MG management, especially in socio-demographic groups with an increased risk of exacerbations.</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":"575-591"},"PeriodicalIF":3.9,"publicationDate":"2025-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11906949/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143441520","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":"A Multi-omics Framework Based on Machine Learning as a Predictor of Cognitive Impairment Progression in Early Parkinson's Disease.","authors":"Yang Luo, YaQin Xiang, JiaBin Liu, YuXuan Hu, JiFeng Guo","doi":"10.1007/s40120-025-00716-y","DOIUrl":"10.1007/s40120-025-00716-y","url":null,"abstract":"<p><strong>Introduction: </strong>Cognitive impairment (CI) is a common non-motor symptom of Parkinson's disease (PD). However, the diagnosis and prediction of CI progression in PD remain challenging. We aimed to explore a multi-omics framework based on machine learning integrating comprehensive radiomics, cerebrospinal fluid biomarkers, and genetics information to identify CI progression in early PD.</p><p><strong>Methods: </strong>Patients were first diagnosed with PD without CI at baseline. According to whether CI progressed within 5 years, patients were divided into two groups: PD without CI and PD with CI. Radiomics signatures were extracted from patients' T1-weighted MRI. We used machine learning methods to construct radiomics, hybrid, and multi-omics models in the training set and validated the models in the testing set.</p><p><strong>Result: </strong>In the two groups, we found 7, 23, and 25 radiomics signatures with significant differences in the parietal, temporal, and frontal lobes, respectively. The radiomics model using the 25 signatures of the frontal lobe had an accuracy of 0.833 and an AUC (area under the curve) of 0.879 to predict CI progression. In addition, the hybrid model fused with the cerebrospinal fluid Aβ level had an accuracy of 0.867 and an AUC of 0.916. In our study, the multi-omics model showed the best predictive performance. The accuracy of the multi-omics model was 0.900, and the average AUC value after five-fold cross-validation was 0.928.</p><p><strong>Conclusion: </strong>Radiomics signatures have a recognition effect in the CI progression in early PD. Multi-omics frameworks combining radiomics, cerebrospinal fluid biomarkers, and genetic information may be a potential predictor of CI progression in PD.</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":"643-658"},"PeriodicalIF":3.9,"publicationDate":"2025-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11906927/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143476847","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":"Letter to the Editor Regarding: Effects of Manual Acupuncture Versus Sham Acupuncture in Patients with Post-Stroke Depression: A Randomized Clinical Trial.","authors":"Benshu Chen, Ziyu Ye, Lihua Wu, Wenbin Fu","doi":"10.1007/s40120-024-00701-x","DOIUrl":"10.1007/s40120-024-00701-x","url":null,"abstract":"","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":"659-661"},"PeriodicalIF":3.9,"publicationDate":"2025-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11906924/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143033586","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":"A Response to: Letter to the Editor Regarding Effects of Manual Acupuncture Versus Sham Acupuncture in patients with Post-Stroke Depression: A Randomized Clinical Trial.","authors":"Conghui Wei, Jinling Chen, Yu Liu, Jun Luo","doi":"10.1007/s40120-024-00702-w","DOIUrl":"10.1007/s40120-024-00702-w","url":null,"abstract":"","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":"663-664"},"PeriodicalIF":3.9,"publicationDate":"2025-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11906937/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143033513","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":"Prolonged Hospital Length of Stay Does Not Improve Functional Outcome in Acute Ischemic Stroke.","authors":"Yuxuan Lu, Peng Sun, Haiqiang Jin, Zhaoxia Wang, Zhiyuan Shen, Wei Sun, Yongan Sun, Ran Liu, Fan Li, Junlong Shu, Zifeng Qiu, Zhongbing Lu, Weiping Sun, Sainan Zhu, Yining Huang","doi":"10.1007/s40120-025-00712-2","DOIUrl":"10.1007/s40120-025-00712-2","url":null,"abstract":"<p><strong>Introduction: </strong>Ischemic stroke is the second leading cause of mortality and a major contributor to disability worldwide. Hospital length of stay (LOS) is a marker of medical efficiency for stroke care. There is limited information on this issue in China. This study aimed to identify predictors of prolonged LOS and to assess whether a prolonged LOS benefits functional outcomes in patients with acute ischemic stroke (AIS) in China.</p><p><strong>Methods: </strong>This secondary analysis of the Chinese Acute Ischemic Stroke Treatment Outcome Registry, a multicenter, prospective, hospital-based registry study, included patients diagnosed as AIS and admitted to a hospital within 3 days after AIS onset from May 2015 to October 2017. Participants were dichotomized as the normal LOS group (LOS ≤ 14 days) and the prolonged LOS group (LOS > 14 days). The outcomes were the proportions of poor outcome at 3 months and 12 months after stroke onset. Poor outcome was defined as a mRS score of 3-6.</p><p><strong>Results: </strong>This study included 8171 patients (median age 64.0 years; 5367 male, 2804 female); 2968 (36.3%) patients had a prolonged LOS. Multivariable analysis identified independent predictors of prolonged LOS, including medical insurance, history of diabetes mellitus, stroke severity, use of anticoagulant agents, in-hospital infection complications, hemorrhagic events, and hospital region. A prolonged LOS was associated with a higher risk of poor outcome at 3 and 12 months after stroke, with this finding upheld in a propensity score-matched cohort as well as subgroup analyses stratified by stroke severity and age.</p><p><strong>Conclusion: </strong>In this study, approximately one-third of patients with AIS experienced a prolonged LOS over 14 days. Independent predictors of prolonged LOS included medical insurance, history of diabetes mellitus, stroke severity, use of anticoagulant agents, in-hospital infection complications, hemorrhagic events, and hospital region. Extending the LOS beyond 14 days did not enhance the prognosis for patients with AIS. As an observational study, our research provided the foundation for further interventional studies. Graphical abstract available for this article.</p><p><strong>Trial registration: </strong>ClinicalTrials.gov NCT02470624.</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":"593-607"},"PeriodicalIF":3.9,"publicationDate":"2025-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11906935/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143441519","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":"Effectiveness During 12-Month Adjunctive Brivaracetam Treatment in Patients with Focal-Onset Seizures in a Real-Life Setting: A Prospective, Observational Study in Europe.","authors":"Bernhard J Steinhoff, Jakob Christensen, Colin P Doherty, Marian Majoie, Anne-Liv Schulz, Fiona Brock, Dimitrios Bourikas, Iryna Leunikava, Anna Kelemen, Eduardo Rubio-Nazabal","doi":"10.1007/s40120-024-00697-4","DOIUrl":"10.1007/s40120-024-00697-4","url":null,"abstract":"<p><strong>Introduction: </strong>Efficacy/tolerability of adjunctive brivaracetam (BRV) for focal-onset seizures (FOS) in patients aged ≥ 16 years was established in randomized controlled trials. This study aimed to evaluate the effectiveness of adjunctive BRV in patients (≥ 16 years) with FOS with/without focal to bilateral tonic-clonic seizures in daily clinical practice.</p><p><strong>Methods: </strong>A 12-month, prospective, real-world, noninterventional study in nine European countries (EP0077/NCT02687711). BRV was prescribed per clinical practice and European Summary of Product Characteristics. Eligible patients had never received BRV before inclusion. Treating physicians made the decision to prescribe BRV, independently of study participation. Primary effectiveness outcome: BRV retention rate at 12 months; secondary effectiveness outcomes: 50% responder rate, seizure freedom.</p><p><strong>Results: </strong>A total of 544 patients received ≥ 1 BRV dose (mean age: 43.6 years; 52.8% female; mean time since diagnosis: 22.7 years). Patients had a mean of 7.3 lifetime antiseizure medications (ASMs) and median of 3.7 FOS/28 days during 3-month retrospective baseline. Median total ASM drug load (including BRV) was 3.0 at BRV initiation (n = 539) and 3.3 at study end (n = 314). At 12 months, 57.7% of 541 patients remained on BRV, 60.4% of 230 were responders (≥ 50% seizure reduction since baseline), and 13.8% of 269 were seizure-free since BRV initiation. Historical levetiracetam use appeared not to impact retention rate (56.6% of 320 and 59.3% of 221 patients with and without historical levetiracetam use, respectively). 36.0% of 544 patients had drug-related treatment-emergent adverse events (TEAEs), mostly (≥ 5% of patients) drug ineffective (11.4%) and seizure (6.3%). The three most common drug-related TEAEs leading to permanent BRV discontinuation (of 544 patients) were drug ineffective (10.1%), seizure (5.1%), and behavior disorder (3.3%).</p><p><strong>Conclusions: </strong>Adjunctive BRV was effective in clinical practice in patients with predominantly difficult-to-treat FOS, as shown by BRV retention rate of 57.7% at 12 months, which is in line with real-world retention rates for other new-generation ASMs.</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":"627-642"},"PeriodicalIF":3.9,"publicationDate":"2025-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11906953/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143458846","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}