Neurology and Therapy最新文献

{"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}

{"title":"The Difficult Journey of a Child with Dravet Syndrome: Perspectives from a Parent and the Neuropaediatrician.","authors":"Romain Reboux, Silvia Napuri","doi":"10.1007/s40120-025-00826-7","DOIUrl":"https://doi.org/10.1007/s40120-025-00826-7","url":null,"abstract":"<p><p>Dravet syndrome (DS) is a rare and severe form of epilepsy, characterised by recurrent seizures that begin during the first year of life, leading to motor, cognitive and behavioural impairments. This article provides the perspectives of a parent of a child with DS ('Ethan') and the treating neuropaediatrician. Ethan's seizures began when he was 9 months old, and were a mixture of focal seizures and status epilepticus. Numerous treatments were tried, including standard anti-seizure medications (such as levetiracetam, clobazam and fenfluramine), other medications (cannabidiol) and nonpharmacological approaches (ketogenic diet), with little success. When Ethan was 3 years old, a prolonged episode of status epilepticus precipitated by coronavirus disease 2019 (COVID-19) led to brain damage. Rehabilitation allowed Ethan to regain some of his previous functioning and, at the age of 38 months, combination therapy with clobazam, sodium valproate and stiripentol was begun and has successfully controlled Ethan's seizures. Ethan's father describes the stress that the diagnosis of DS, interactions with the healthcare system, and the search for effective treatment imposed on the family. Since Ethan's seizures have been better controlled, the family has been able to lead a more normal life, and is now focused on supporting Ethan and looking to the future. Ethan's neuropaediatrician outlines the approach she takes to the diagnosis and management of DS, including the importance of the clinician-parent relationship in imparting the diagnosis and making initial and ongoing treatment decisions. The preferred first-line treatment is sodium valproate, which is followed by sodium valproate-clobazam-stiripentol combination therapy, topiramate or a ketogenic diet as second-line options. In children > 2 years, cannabidiol and fenfluramine can also be considered. The aim of maintenance treatment (which will invariably be polytherapy) is to reduce the number of seizures, particularly status epilepticus, given the significant impact of this seizure type on patients and caregivers.</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":""},"PeriodicalIF":4.8,"publicationDate":"2025-09-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145086099","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":"Microglial Polarization and Therapeutic Strategies in Post-stroke Neuroinflammation.","authors":"Travis Yui Hei Chan, Brian De Yu Ma, Timothy Keith Hung, Jenny Sum Yee Wong, Benjamin Wai Yue Lo","doi":"10.1007/s40120-025-00825-8","DOIUrl":"https://doi.org/10.1007/s40120-025-00825-8","url":null,"abstract":"<p><p>Stroke remains a leading cause of global disability, perpetuated by maladaptive neuroinflammation that drives secondary injury and impairs recovery. An early reparative (M2) state rapidly transitions into a dominant destructive (M1) phenotype within days, worsening tissue damage through the release of cytokines [tumor necrosis factor alpha (TNFα), interleukin-6 (IL-6)], blood-brain barrier disruption, and amplified peripheral immune cell infiltration. Emerging pharmacological interventions, such as the free radical scavenger edaravone, the neurotrophic factor cerebrolysin, and the excitotoxicity modulator citicoline, demonstrate promising neuroprotective potential when strategically timed. Additionally, novel non-pharmacological approaches, including repetitive transcranial magnetic stimulation, stem cell therapy, and nanoparticle-based drug delivery, offer innovative pathways for targeting neuroinflammation. However, translational challenges persist, including narrow therapeutic windows, biomarker heterogeneity, and preclinical-to-clinical gaps. Future progress necessitates precision medicine paradigms integrating spatiotemporal drug delivery, biomarker-guided intervention timing, and synergistic combinatorial regimens targeting acute injury and chronic repair phases. By bridging mechanistic insights with clinical applications, this review delineates neuroinflammatory modulation as a pivotal frontier for redefining stroke recovery while outlining essential research trajectories to overcome existing barriers. Systematic search of electronic databases including PubMed, Web of Science, Embase, and Cochrane (1996-2025) was performed, with eligible studies assessed using PRISMA guidelines. Findings on neuroinflammation, mechanism, or interventions in ischemic stroke were narratively synthesized through thematic analysis. This review summarizes current insights into post-stroke neuroinflammatory mechanisms, with a focus on the dual role of microglial polarization.</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":""},"PeriodicalIF":4.8,"publicationDate":"2025-09-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145085496","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":"Predictors of Post-Alteplase Long-Term Unfavorable Outcome in Atrial Fibrillation Patients Presented with Embolic Stroke in the Middle East and North Africa Regions: A Multi-center, Longitudinal Study.","authors":"Mohamed G Zeinhom, Mohamed Fouad Elsayed Khalil, Ahmad Galal Elmesallami, Tamer Shaaban Zedan, Karam Dahy Hamdan Hassaneen, Hossam Mohamed Refat, Ahmed Ahmed Mohamed Kamal Ebied, Shady S Georgy, Ahmed Zaki Omar Akl, Mohamed Ismaiel, Salah Ibrahim Ahmed, Hesham Farouk Eissa, Asmaa Ibrahem Desouky Mostafa Ibrahem, Youssry Salah Shafiq Kerolos, Mohamed Elshafei, Amir Ahmed Elsaeed Egila, Enji Hamdy Elsawy Khalil, Emad Labib Abdelhamid Mahmoud, Sherihan Rezk Ahmed","doi":"10.1007/s40120-025-00814-x","DOIUrl":"https://doi.org/10.1007/s40120-025-00814-x","url":null,"abstract":"<p><strong>Introduction: </strong>Embolic stroke is connected to a higher risk of hemorrhagic transformation (HT), functional disability, and mortality. There is a gap of knowledge regarding the predictors of long-term post-alteplase functional outcomes, especially in patients with atrial fibrillation (AF), which limits delivering adequate care and support to stroke survivors. We aimed to assess the predictors of long-term post-thrombolysis unfavorable functional outcomes in patients with atrial fibrillation who presented with first-ever embolic stroke in the Middle East and North Africa (MENA).</p><p><strong>Methods: </strong>Our prospective cohort study was conducted between May 2021 and May 2025 and included patients with AF who presented with first-ever embolic stroke and received thrombolytic therapy, and who were recruited from Kafr Elsheikh University Hospital, Kafr Elsheikh General Hospital, Al-Sahel Teaching Hospital, NMC Royal Hospital, and Al-Obour Hospital in the period from May 2021 to May 2023. Our longitudinal study included two groups; the unfavorable outcomes group, and the favorable outcomes group.</p><p><strong>Results: </strong>A total of 580 patients completed the 2-year follow-up period. National institute of health stroke scale (NIHSS) at the time of admission [odds ratio (OR), 2.06; 95% CI, 1.86-4.39; P = 0.03], sustained atrial fibrillation (OR, 1.98; 95% CI, 1.42-3.80; P = 0.03), heart failure (OR, 1.79; 95% CI, 1.23-2.96; P = 0.03), HAS-BLED score (OR, 1.64; 95% CI, 1.41-3.65; P = 0.03), CHA₂DS₂VASc score (OR, 1.72; 95% CI, 1.72-3.53; P = 0.04), post-thrombolysis intracranial hemorrhage (OR, 2.89; 95% CI, 1.74-3.63; P = 0.02), and recurrent symptomatic stroke (OR, 1.98; 95% CI, 1.22-3.73; P = 0.04) were predictors of long-term post-thrombolysis unfavorable outcomes.</p><p><strong>Conclusion: </strong>Higher baseline NIHSS, heart failure, post-thrombolysis intracranial haemorrhage, and recurrent symptomatic stroke were independent predictors of long-term post-thrombolysis unfavorable functional outcome in embolic stroke patients. Novelly, higher HAS-BLED and CHA2DS2-VASc scores were independent predictors of long-term post-thrombolysis unfavorable functional outcome in Arabian patients. Moreover, sustained AF was an independent predictor of long-term post-thrombolysis unfavorable functional outcome.</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":""},"PeriodicalIF":4.8,"publicationDate":"2025-09-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145085541","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":"Real-World 24-Month Outcomes of Ofatumumab in Relapsing Multiple Sclerosis: Efficacy, Safety, and the Impact of Frailty.","authors":"Gina Ferrazzano, Roberta Fantozzi, Shalom Haggiag, Doriana Landi, Francesca Napoli, Maria Chiara Buscarinu, Leonardo Malimpensa, Assunta Bianco, Giovanna Borriello, Elena Barbuti, Fabiana Marinelli, Fabrizia Monteleone, Francesca Marchione, Nicola Falcone, Marta Altieri, Giorgio Leodori, Daniele Belvisi, Fabio Buttari, Valeria Pozzilli, Alessandra Cicia, Antonio Cortese, Francesco Sica, Anna Chiara Landi, Elisabetta Ferraro, Carlo Pozzilli, Massimiliano Mirabella, Carla Tortorella, Girolama Alessandra Marfia, Diego Centonze, Marco Salvetti, Antonella Conte","doi":"10.1007/s40120-025-00818-7","DOIUrl":"https://doi.org/10.1007/s40120-025-00818-7","url":null,"abstract":"<p><strong>Introduction: </strong>Ofatumumab (OFA) is a highly effective therapeutic option for multiple sclerosis (MS), but real-world data on its efficacy and safety remain limited. We evaluated the real-world efficacy and safety of OFA in patients with MS and explored the predictive value of frailty.</p><p><strong>Methods: </strong>We retrospectively collected clinical and MRI data from 12 MS centers in Central Italy, including patients who initiated OFA between April 2022 and January 2024. We assessed annualized relapse rate (ARR), clinical relapses, radiological activity, and safety. Frailty, defined as increased vulnerability due to age-related health deficits, was measured using a frailty index (FI). The study was approved by the local Ethics Committee (No. 6357).</p><p><strong>Results: </strong>A total of 242 patients with MS were included (66.8% female and 33.2% male; mean age: 38.9 ± 10.3 years; disease duration: 7.7 ± 7.6 years). Of these, 95 (39.2%) were treatment-naïve, and 147 (60.8%) had switched from another therapy, mostly a first switch. The mean follow-up was 15.4 ± 5.4 months; all patients completed 12-month follow-up, and 103 completed 24 months. ARR dropped from 0.9 to 0.02 (p < 0.001). Only 4 patients (1.6%) had a clinical relapse, all within 6 months (mean time: 3.0 ± 1.8 months). Expanded Disability Status Scale (EDSS) scores remained stable (p > 0.05). MRI activity occurred in 10 patients (4.1%) at 6 months and 3 (1.2%) at 12 months; none at 24 months. Adverse events included flu-like symptoms (34.3%), injection site reactions (8.2%), and infections (18.5%). Among 239 patients assessed for frailty (mean FI: 0.06 ± 0.08), 187 were relatively fit (FI ≤ 0.10), 30 least fit, and 22 frail. FI predicted 24-month confirmed disability progression (p = 0.0068), with significant variation by frailty level (p = 0.0009).</p><p><strong>Conclusion: </strong>This real-world study suggests that OFA is effective and safe for MS, offering rapid disease control. Lower frailty levels suggest preferential use in patients with lower baseline disability. Further large-scale, long-term studies are needed.</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":""},"PeriodicalIF":4.8,"publicationDate":"2025-09-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145085523","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":"Treatment Preferences of Neurologists in Generalized Myasthenia Gravis: A Conjoint Analysis Study.","authors":"Luis Querol, Rocío Gómez-Ballesteros, Gerardo Gutiérrez-Gutiérrez, Adrián Ares, Ramón Villaverde, Virginia Reyes, Thaís Armangué, Elisa Salas, Paola Díaz-Abós, Pablo Rebollo, Mònica Sarmiento, Neus Canal, Jorge Maurino, Elena Cortés-Vicente","doi":"10.1007/s40120-025-00821-y","DOIUrl":"https://doi.org/10.1007/s40120-025-00821-y","url":null,"abstract":"<p><strong>Introduction: </strong>Generalized myasthenia gravis (gMG) is a chronic autoimmune neuromuscular disorder for which treatment decision-making is increasingly complex due to the emergence of biologic targeted therapies. This study aimed to evaluate neurologists' treatment preferences in acetylcholine receptor seropositive gMG using conjoint analysis to simulate real-world clinical decision-making.</p><p><strong>Methods: </strong>We conducted a cross-sectional, web-based study among neurologists involved in the management of gMG in collaboration with the Spanish Society of Neurology. Participants were presented with eight hypothetical treatment scenarios comprising five key attributes: intensity of improvement, onset of action, duration of effect, adverse events, and route/frequency of administration. Utility values and importance of each attribute were estimated using ordinary least squares regression. Demographic, professional, and behavioral characteristics were assessed to explore variability in preference patterns.</p><p><strong>Results: </strong>A total of 149 neurologists participated in the study. The mean age was 39.0 (SD 9.4) years, and 54.4% were male. Participants had a mean of 9.9 years (8.4) of experience treating patients with MG. The most valued treatment attribute was intensity of improvement (mean relative importance: 38.6%), followed by onset of action (21.5%) and duration of effect (17.4%). At the individual level, route and frequency of administration accounted for up to 21.9% of decision weight in some participants. Preferences were consistent across neuromuscular specialists and general neurologists but differed based on empathy and conscientiousness. Model fit was robust (Pearson's R = 1.000, p < 0.001).</p><p><strong>Conclusions: </strong>Neurologists treating gMG placed the highest value on rapid and clinically meaningful symptom improvement, consistent with the therapeutic goals of emerging targeted therapies. However, substantial heterogeneity in preferences, especially regarding administration burden, emphasizes the need for individualized approaches and shared decision-making.</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":""},"PeriodicalIF":4.8,"publicationDate":"2025-09-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145086194","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}