Neurology and Therapy最新文献

{"title":"Therapies for Advanced Parkinson's Disease in Sweden: A Cost-Effectiveness Analysis Using Real-World Data.","authors":"Dag Nyholm, Simon Eggington, Astrid Holm","doi":"10.1007/s40120-025-00730-0","DOIUrl":"10.1007/s40120-025-00730-0","url":null,"abstract":"<p><strong>Introduction: </strong>The Swedish Parkinson's Disease (PD) Registry provides rich data on cost and quality of life for patients receiving device-aided therapies (DATs) for Parkinson's disease in Sweden. This study sought to use this real-world evidence to determine the cost-effectiveness profile of specific DATs in Sweden.</p><p><strong>Methods: </strong>We developed a state transition (Markov) model to represent disease progression over time and used published clinical data to represent short- and long-term disease outcomes. We used data from the Swedish PD Registry to assign costs and quality of life measures (utilities) to each health state and modelled total costs and outcomes over a 20-year time horizon. The four treatment groups modelled were: best medical therapy, deep brain stimulation, levodopa-carbidopa intestinal gel, and continuous subcutaneous apomorphine infusion. We calculated results from both payer and societal perspectives.</p><p><strong>Results: </strong>The most cost-effective intervention was deep brain stimulation (DBS), which dominated all other interventions. Sensitivity analysis indicated that the key drivers of uncertainty were the transition probabilities between successive Hoehn and Yahr stages on each treatment. Results were consistent from both payer and societal perspectives.</p><p><strong>Conclusion: </strong>DBS is a cost-effective use of resources in Sweden. However, uncertainty remains regarding long-term symptom control as well as quality of life within specific health states for all device-aided therapies, and further data are needed to fully validate the model projections and provide more insight into areas of future research.</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":"801-812"},"PeriodicalIF":3.9,"publicationDate":"2025-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12088999/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143673445","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":"The Mediating Effect of Intracranial Hemorrhage Status on the Relationship between the INR and Mortality in Patients with Ischemic Stroke.","authors":"Yapeng Guo, Lingshan Wu, Zhenxuan Tian, Xu Xu, Jinfu Ma, Changwei Guo, Linyu Li, Jie Yang, Wenjie Zi, Jiacheng Huang, Xianjun Huang","doi":"10.1007/s40120-025-00715-z","DOIUrl":"10.1007/s40120-025-00715-z","url":null,"abstract":"<p><strong>Introduction: </strong>The international normalized ratio (INR) is a biomarker of coagulopathy. The objective of this study was to assess the relationship between the INR and clinical outcomes in patients with large vessel occlusion (LVO) stroke who received endovascular therapy.</p><p><strong>Methods: </strong>The RESCUE BT trial was a multicenter, randomized, double-blind, placebo-controlled clinical trial involving 948 stroke patients from 55 centers across China. We extracted INR data and related data from the BT database, with outcome measures comprising intracranial hemorrhage (ICH) and 90-day mortality. Logistic regression analysis was conducted to examine the associations between the INR and clinical outcomes in the entire patient cohort and across different stratified subgroups.</p><p><strong>Results: </strong>A total of 885 patients met the study criteria, with 672 exhibiting a normal INR and 213 showing an elevated INR. Multivariable analysis indicated that an elevated INR was linked to an increased risk of ICH (OR 1.65, 95% confidence interval CI 1.17-2.33, P =0.005) and 90-day mortality (OR 1.78, 95% CI 1.17-2.70, P =0.007). Mediation analysis indicated that the association between the INR and 90-day mortality risk was partially mediated by ICH status, with the mediation effect contributing 11.4% to the overall relationship. Subgroup analyses revealed no significant differences between the different subgroups (P for interaction > 0.05). In patients receiving tirofiban, an elevated INR was more strongly associated with an increased 90-day mortality rate (OR 7.75, 95% CI 1.42-42.33, P =0.018).</p><p><strong>Conclusion: </strong>Our findings underscore the critical importance of INR monitoring in patients with LVO stroke undergoing endovascular treatment (EVT). The association between the INR and 90-day mortality was mediated through ICH status. The use of tirofiban strengthened the associated between an elevated INR and a higher 90-day mortality rate. These insights offer valuable guidance for optimizing patient outcomes.</p><p><strong>Trial registration: </strong>URL: http://www.chictr.org.cn ; ChiCTR-INR-17014167.</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":"881-894"},"PeriodicalIF":3.9,"publicationDate":"2025-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12089563/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144029533","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 Analysis of Repetitive Transcranial Magnetic Stimulation in Patients with Persistent Postural Perceptual Dizziness: A Single-Center, Single-Blind, Randomized Placebo-Controlled Trial.","authors":"Wenze Li, Chang Liu, Yuqing Zhang, Maolin Peng, Xiuhang Luo, Hongfa Zhang, Hairong Lan, Zhipeng Li, Yankun Chen, Zhen Li, Zhimin Xiao, Linyan Tong, Yangmei Chen","doi":"10.1007/s40120-025-00733-x","DOIUrl":"10.1007/s40120-025-00733-x","url":null,"abstract":"<p><strong>Introduction: </strong>Persistent postural-perceptual dizziness (PPPD) is a chronic functional dizziness often triggered by vestibular, psychological, or environmental factors. Current treatments, including pharmacological and cognitive therapies, show limitations. In recent years, transcranial magnetic stimulation (TMS) has been explored in other neuropsychiatric disorders but has not been studied extensively for PPPD.</p><p><strong>Objective: </strong>We aimed to evaluate the efficacy and safety of TMS of the left high-frequency dorsolateral prefrontal cortex (DLPFC) in improving dizziness and mood disorders in patients with PPPD in a single-blind, placebo-randomized controlled trial.</p><p><strong>Methods: </strong>This trial recruited patients from October 8, 2023, to June 30, 2024, with follow-up completed on September 30, 2024, of 80 patients screened from the second affiliated hospital of Chongqing Medical University in China. Totals of 4 patients were excluded and 66 patients were randomized. PPPD patients were randomized to receive either TMS (10 Hz, 20 min) or SHAM-TMS treatments to the left DLPFC over ten sessions within 2 weeks. Dizziness severity, anxiety, and depression quality were assessed at baseline, post-treatment, and 1 and 3 months. Adverse events were also monitored.</p><p><strong>Results: </strong>Of 66 eligible patients [median (IQR) age, 54.5 (49.8-67.0) years; Of 42 women (63.6%)], 33 were randomized to the TMS group, and 33 were randomized to the SHAM-TMS group. After three months, a total of 52 participants (TMS group [n = 27]; SHAM-TMS group [n = 25)] completed the follow-up. At 2 weeks, 1 month, and 3 months post-treatment, the TMS group exhibited significant reductions in the levels of dizziness and anxiety compared to both their baseline measurements and the SHAM-TMS group at the same time points (all, p < 0.05). In the SHAM-TMS group, dizziness showed a significant improvement only at 2 weeks post-treatment compared to baseline (p < 0.05). Additionally, in the TMS group, Hamilton Depression Rating Scale (HAMD) scores decreased at both 2 weeks and 1 month relative to baseline. In contrast, the SHAM-TMS group displayed no significant changes in HAMD scores during the 3-month follow-up.</p><p><strong>Conclusion: </strong>TMS targeting the DLPFC significantly alleviated symptoms of dizziness and anxiety in patients with PPPD. This non-invasive treatment may offer a safe and effective therapeutic alternative for managing PPPD symptoms. Further large-scale studies are recommended to confirm these findings.</p><p><strong>Trial registration: </strong>ClinicalTrials.gov identifier, CTR2400093690.</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":"849-863"},"PeriodicalIF":3.9,"publicationDate":"2025-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12089549/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143753641","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":"Cost-Effectiveness of Newborn Screening for Spinal Muscular Atrophy in Australian Hospitals.","authors":"Ian R Woodcock, Didu S Kariyawasam, Maina P Kava, Eppie M Yiu, Damian Clark, Jane Adams, Matthias Bischof, Adrian Peacock, Colman Taylor, Nicholas J C Smith","doi":"10.1007/s40120-025-00744-8","DOIUrl":"10.1007/s40120-025-00744-8","url":null,"abstract":"<p><strong>Introduction: </strong>This analysis evaluated the cost-effectiveness of newborn screening (NBS) for spinal muscular atrophy (SMA) from the perspective of Australian state hospital payers.</p><p><strong>Methods: </strong>A cost-utility analysis consisting of a decision tree and Markov cohort designed to calculate the difference in costs and health outcomes between two scenarios: (1) disease-modifying treatment (DMT) for SMA after diagnosis through NBS, and (2) DMT for SMA after diagnosis as symptoms appear. A population of 295,906 newborns was modeled, based on the total number of live births in Australia in 2023. Inputs included screening parameters, epidemiology inputs, SMA natural history data and DMT parameters (nusinersen and onasemnogene abeparvovec), costs, and health-related quality of life parameters. Assumed participation in NBS was 100%. A one-way sensitivity analysis and probabilistic sensitivity analysis were conducted to examine the impact of parameter uncertainty.</p><p><strong>Results: </strong>There were 30 patients identified with SMA, of whom 25 patients would be eligible for presymptomatic treatment. NBS for SMA was dominant compared with no NBS for SMA. On a population level, NBS demonstrated a lifetime gain of 267 quality-adjusted life years (QALY) and incremental costs of -AUD$3,983,263 (i.e., cost savings). Every dollar invested in NBS would save hospitals $3.69. Deterministic and probabilistic sensitivity analyses demonstrated the robustness of the base-case results.</p><p><strong>Conclusion: </strong>NBS for SMA was dominant compared with no NBS for SMA in Australia from a state and territory payer perspective. Universal implementation of NBS for SMA would support access equity, as well as early diagnosis and treatment in infants with SMA, potentially leading to improved outcomes.</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":"1007-1022"},"PeriodicalIF":3.9,"publicationDate":"2025-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12089012/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144028609","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":"Comparison of Factors Affecting Quality of Life in Patients with Acute Ischemic Stroke Across Different Stroke Severities.","authors":"Yuxuan Lu, Weiping Sun, Yining Huang, Zhaoxia Wang, Zhiyuan Shen, Wei Sun, Ran Liu, Fan Li, Junlong Shu, Qing Peng, Jingjing Jia, Peng Sun, Yijun Song, Haiqiang Jin","doi":"10.1007/s40120-025-00743-9","DOIUrl":"10.1007/s40120-025-00743-9","url":null,"abstract":"<p><strong>Introduction: </strong>Research investigating the differences in determinants affecting the quality of life (QoL) in patients with acute ischemic stroke (AIS) of varying severities remains limited, and how these factors influence QoL remains unclear. The aim of this study was to address this critical issue, refining treatments to enhance long-term QoL and optimize resource use.</p><p><strong>Methods: </strong>In this multicenter prospective study conducted in China, patients with AIS were assessed using the EuroQol-5 Dimension (EQ-5D) questionnaire at admission and 1 year later. Univariate and multivariate analyses were conducted to identify QoL determinants. Motor function-related outcomes (MFRO) and non-motor function-related outcomes (NMFRO) were defined based on the EQ-5D questionnaire to explore how factors influence outcomes through mediation analysis.</p><p><strong>Results: </strong>The study included 8598 patients with AIS (median age 64 years; 65.7% male), 3927 of whom had minor stroke severity (National Institutes of Health Stroke Scale [NIHSS] scores ≤ 3). The median QoL score improved from 0.597 at admission to 1.000 after 1 year. Compared to patients with minor stroke, more patients with non-minor stroke had non-motor function-related problems (NMFRP) at admission (78.2% vs. 56.6%). Age, stroke history, QoL at admission, infection, hospitalization costs, and discharge outcomes were found to be factors influencing QoL in both cohorts. In the non-minor stroke cohort, analysis revealed that additional factors include diabetes mellitus, geographical region, speech impairment, and thrombolysis, while in the minor stroke cohort, hypertension, coronary heart disease, cancer, prolonged length of stay, and hemorrhage were found to be relevant factors. The impact of most factors on QoL was mediated by MFRO, while the effects of age, speech impairment, and geographical region were also mediated by NMFRO. Hospitalization costs beyond 15,000 China Yuan (CNY) did not improve QoL for the patients with non-minor stroke, with a threshold of 10,000 CNY for the patients with minor stroke.</p><p><strong>Conclusions: </strong>Over half of the patients in the study population had NMFRP, necessitating greater medical attention. Patients with different stroke severities had distinct QoL determinants. Age, speech impairment, and geographical region may exert an impact partly mediated through NMFRO. Higher hospitalization costs did not consistently improve QoL beyond a certain threshold.</p><p><strong>Trial registration: </strong>ClinicalTrials.gov Identifier: NCT02470624.</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":"1023-1038"},"PeriodicalIF":3.9,"publicationDate":"2025-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12089634/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144042593","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":"Long-Term Management and Therapeutic Sequencing for Patients with Relapsing Multiple Sclerosis in France: A Vignette Study.","authors":"Patrick Vermersch, Xavier Moisset, Baptiste Roux, Anais Lecomte, Laura Luciani, Martine Paret, Jérôme de Sèze","doi":"10.1007/s40120-025-00726-w","DOIUrl":"10.1007/s40120-025-00726-w","url":null,"abstract":"<p><strong>Introduction: </strong>We have analysed prescribing decisions for relapsing multiple sclerosis (RMS) of 111 neurologists (\"participating physicians\") in France using hypothetical case vignettes.</p><p><strong>Methods: </strong>Six case vignettes were presented to participating physicians, each based on realistic, hypothetical clinical interactions between a neurologist and people with active or highly active RMS, with or without prior treatment with a disease-modifying therapy (DMT). \"Disruptive events\" are where the appearance of new MS disease activity, side-effects or other issues prompted the return of the hypothetical patients for a review of their care.</p><p><strong>Results: </strong>A population of 111 participating physicians reviewed the cases and recommended treatments. Our data suggested a willingness among participating physicians to treat with higher-efficacy DMTs early in the course of RMS, with platform agents given to only one-quarter of DMT-naïve cases. MS disease activity was the main driver of switches to higher-efficacy DMTs, although an escalation approach was common in response to either moderate MS disease activity or side-effects on platform agents. A desire for pregnancy drove high usage of cladribine tablets and natalizumab (especially for cases negative for John Cunningham virus).</p><p><strong>Conclusions: </strong>These findings suggest that the management of RMS in France has shifted in recent years towards a desire to achieve earlier and more effective control of disease activity for people with RMS. Better guidance on the sequencing of DMTs for different scenarios within the overall management of RMS may be warranted. This study offers valuable insights into the current practices of French neurologists in managing RMS, emphasizing the complexity of therapeutic decisions, the diversity of strategies, and the significance of an individualized approach in treatment management.</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":"813-827"},"PeriodicalIF":3.9,"publicationDate":"2025-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12089545/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143692476","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":"Understanding Depression in People Living with Multiple Sclerosis: A Narrative Review of Recent Literature.","authors":"Amy B Sullivan, Bryan Davis, Julie Kidd, Horacio Chiong-Rivero","doi":"10.1007/s40120-025-00728-8","DOIUrl":"10.1007/s40120-025-00728-8","url":null,"abstract":"<p><p>Multiple sclerosis (MS) is a chronic neurodegenerative and autoimmune disease that affects approximately 1 million adults in the US. Psychologic disorders are typical comorbidities in people with MS (pwMS), with depression being the most common. Clinical depression in pwMS can substantially impact quality of life and factor heavily in treatment adherence. Depression can surface early in MS, becoming more prevalent as the disease progresses and the severity of clinical disability increases. The etiology of comorbid depression in pwMS is not completely understood, but recent research has indicated that structural and functional brain abnormalities, along with genetic and immunologic factors, may contribute to the pathogenesis of depression in pwMS. Although depression has a significant impact on pwMS, it is often underdiagnosed and undertreated. Furthermore, the efficacy of pharmacologic and non-pharmacologic approaches for treating depression in pwMS has not been thoroughly investigated, with most studies showing minimal or no beneficial effect. Improved evaluation and diagnosis of depression and a better understanding of its pathophysiology may provide a stronger foundation for treatment and management of pwMS suffering from depression. This review discusses recent research on the potential causes of depression, the risk factors associated with developing depression, and the overall impact of depression in pwMS. It also reviews patient-reported outcomes utilized to assess depression in pwMS and the impact of disease-modifying therapies on depression in pwMS. Consideration is also given to management of depression in pwMS (both pharmacologic and non-pharmacologic) to better facilitate the patient journey.</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":"681-710"},"PeriodicalIF":3.9,"publicationDate":"2025-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12089588/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143720881","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":"Characterizing the Journey of Early Alzheimer's Disease in Patients Initiating Lecanemab Treatment in the United States: A Real-World Evidence Study.","authors":"Marwan N Sabbagh, Chenyue Zhao, Malena Mahendran, Se Ryeong Jang, François Laliberté, Hideki Toyosaki, Kaixin Zhang, Feride Frech, Kavita V Nair","doi":"10.1007/s40120-025-00756-4","DOIUrl":"10.1007/s40120-025-00756-4","url":null,"abstract":"<p><strong>Introduction: </strong>With the advent of disease-modifying therapies for early Alzheimer's disease (AD), a comprehensive characterization of patients initiating lecanemab in the USA is needed to understand its use in real-world settings.</p><p><strong>Methods: </strong>This retrospective observational study used administrative claims from the Komodo Research Database (1/1/2023-6/30/2024). Eligible patients had ≥ 1 lecanemab administration (first claim defined the index date) and ≥ 12 months of clinical activity/insurance eligibility before the index date. Patient characteristics, diagnostic process, and AD-related medications were evaluated within 12 months before the index date (baseline), whereas lecanemab treatment patterns and concomitant medications were evaluated on or after the index date (follow-up). Outcomes were reported using descriptive statistics and persistence to lecanemab was evaluated using Kaplan-Meier analysis.</p><p><strong>Results: </strong>Of 3155 patients included in the study, mean age was 75.0 years, 55.8% were female, 44.2% were male, and most (93.3%) received their index lecanemab administration in an urban setting. Diagnoses of AD (83.8%) and mild cognitive impairment (60.8%) were common at baseline, and 67.6% of patients used AD symptomatic medications. Average time from earliest diagnosis to first lecanemab administration was 4.9 months among patients with a diagnosis in January 2023 (accelerated approval date) or onwards. Over a mean follow-up of 138.8 days, the monthly mean number of administrations of lecanemab was 1.9, with an average of 16.5 days between consecutive administrations and 47.4 days to the first follow-up head magnetic resonance imaging. Persistence to lecanemab was 87.6% at 4 months after treatment initiation.</p><p><strong>Conclusion: </strong>Lecanemab was utilized in appropriate patient populations according to the prescribing information approved by the US Food and Drug Administration. Findings from our study provide first insights into the real-world use of lecanemab in the USA and shed light on the need for increased and timely lecanemab initiation for the long-term management of early AD.</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":"1115-1127"},"PeriodicalIF":3.9,"publicationDate":"2025-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12089008/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143991218","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":"U-Shaped Relationship of Serum Albumin and Neurological Functional Outcomes After Acute Ischemic Stroke: A Prospective Cohort Study.","authors":"Yuan Zhu, Gang Xue, Shufan Xu, Qi Qin, Peian Liu, Lianhong Ji, Huimin Wu, Minghua Wu, Zhuyuan Fang","doi":"10.1007/s40120-025-00729-7","DOIUrl":"10.1007/s40120-025-00729-7","url":null,"abstract":"<p><strong>Introduction: </strong>Several studies indicate that individuals with acute ischemic stroke (AIS) who have low levels of serum albumin (SA) have a dismal prognosis. However, intravenously administering albumin 25% at a dose of 2 g/kg did not lead to improved outcomes for patients with AIS after 90 days. Our objective was to examine the possible correlation between SA levels and stroke outcomes in a prospective cohort investigation.</p><p><strong>Methods: </strong>The research included a total of 5111 participants diagnosed with AIS. The correlation between SA level and modified Rankin Scale (mRS) scores 90 days after onset was examined via univariate and multivariate logistic analyses. The relationships were examined employing restricted cubic splines. An investigation was conducted to ascertain the connection between SA levels and neurological functional results by employing mediation analysis, with the mediation impact of low-density lipoprotein (LDL) taken into account. In addition, the subgroup analyses were performed using the logistic regression.</p><p><strong>Results: </strong>The connection between levels of SA and neurological functional outcomes following AIS exhibited a U-shaped pattern. The likelihood of a negative result dropped significantly with an elevation in SA (per g/L: OR (odds ratio) 0.88; 95% CI (confidence interval) 0.847-0.913) among individuals with SA levels below 42.2 g/L. Conversely, the likelihood of a negative outcome rose with an increase in SA (per g/L: OR 1.033, 95% CI 1.009-1.058) among people with SA levels of 42.2 g/L or above. Comparable findings were seen for mortality outcomes. A mediation study revealed that LDL had a mediating function in the statistical connection between SA levels and neurological functional outcomes, accounting for 12.3% of the connection. No significant interactions were seen in any of the groupings.</p><p><strong>Conclusion: </strong>Among patients with AIS, there was a U-shaped relationship between SA levels at admission and the likelihood of poor outcomes, which was partially mediated by LDL. There is a Graphical Abstract available for this article.</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":"949-964"},"PeriodicalIF":3.9,"publicationDate":"2025-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12089567/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144012509","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 of Stiripentol Beyond Dravet Syndrome: A Retrospective Medical Record Review of Patients with Drug-Resistant Epilepsies.","authors":"Víctor Soto-Insuga, Elena González-Alguacil, María Ballarà-Petitbò, Nuria Lamagrande-Casanova, Anna Duat-Rodríguez, Cristina Benítez-Provedo, Elena Cardenal-Muñoz, Juan José García-Peñas","doi":"10.1007/s40120-025-00755-5","DOIUrl":"10.1007/s40120-025-00755-5","url":null,"abstract":"<p><strong>Introduction: </strong>Stiripentol is approved as an add-on therapy with clobazam and/or valproate for seizures associated with Dravet syndrome (DS). Evidence of stiripentol efficacy in other drug-resistant epilepsies is limited.</p><p><strong>Methods: </strong>This was a single-centre, retrospective, observational study of patients aged ≤ 15 years with non-Dravet epilepsy or DS who initiated stiripentol treatment in Spain.</p><p><strong>Results: </strong>The study included 18 patients with DS and 17 with non-Dravet epilepsy; 76.5% of the latter had a developmental and epileptic encephalopathy. Median (range) age at stiripentol initiation was 52 (4-180) months. Three months of add-on stiripentol provided overall improvement in seizures (number, duration and/or intensity) for 76.5% of the non-Dravet and 61.1% of the DS patients (p = 0.30), all of whom were drug-resistant to prior antiseizure medications (ASMs). Stiripentol reduced seizure frequency by ≥ 50% in 58.8% of the non-Dravet patients and 44.4% of the DS cohort (p = 0.40); 20% of all patients became seizure-free. Stiripentol reduced all seizure types in both cohorts. Kaplan-Meier survival analysis found a higher probability of sustained stiripentol efficacy in the DS cohort (120 months) than the non-Dravet cohort (16 months; p = 0.012). Stiripentol improved cognition and Clinical Global Impression scale scores in approximately 60% of all patients; sleep improved for 19.2%. Acute stiripentol treatment (maximum dose 6.7-100 mg/kg/day) initiated in five patients (four with non-Dravet epilepsy and one with DS) during refractory status epilepticus (SE) successfully resolved SE over a median 0.5 days. Adverse events, mainly mild-to-moderate, occurred in 47.1% and 41.2% of patients in the non-Dravet and DS cohorts, respectively. Six patients (35.3%) with non-Dravet epilepsy discontinued ≥ 1 other ASMs after stiripentol initiation.</p><p><strong>Conclusion: </strong>Add-on stiripentol provides overall improvement in different seizure types and non-seizure manifestations for paediatric patients with drug-resistant epilepsy, including epileptic syndromes besides DS, and appeared effective in acute treatment of SE. Stiripentol was generally well tolerated.</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":"1129-1150"},"PeriodicalIF":3.9,"publicationDate":"2025-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12089635/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144002911","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}