Therapeutic Advances in Neurological Disorders最新文献

{"title":"Biochemical and nutritional changes during continuous subcutaneous apomorphine infusion in advanced Parkinson's disease: findings from an observational cohort.","authors":"Onanong Phokaewvarangkul, Naruemon Rattanapitak, Panthitra Kuwattanasuchati, Roongroj Bhidayasiri","doi":"10.1177/17562864261446737","DOIUrl":"10.1177/17562864261446737","url":null,"abstract":"<p><strong>Background: </strong>Patients with Parkinson's disease (PD) receiving device-aided therapies (DATs) frequently experience vitamin B depletion and nutritional challenges. However, the effects of continuous subcutaneous apomorphine infusion (CSAI) on vitamin B status and nutritional outcomes in advanced PD remain unclear.</p><p><strong>Objective: </strong>To evaluate the clinical effectiveness of CSAI and associated changes in vitamin B status, homocysteine (Hcy) levels and nutritional outcomes in patients with advanced PD.</p><p><strong>Design: </strong>A prospective observational cohort study in a single centre.</p><p><strong>Methods: </strong>Thirty patients with advanced PD were enrolled between January 2023 and March 2025. Clinical, biochemical and nutritional assessments were performed at baseline and after 3 months. Motor outcomes included daily OFF hours, dyskinesia hours, Hoehn and Yahr stage and the Unified PD Rating Scale (UPDRS). Biochemical measures comprised serum vitamins B₁, B₆, B₁₂, folate and plasma Hcy levels. Nutritional status was assessed using body weight, body mass index (BMI) and the Mini Nutritional Assessment-Short Form (MNA-SF).</p><p><strong>Results: </strong>Thirty participants (mean age 68.5 ± 9.2 years; disease duration 12.0 ± 3.9 years; Hoehn & Yahr 3.7 ± 0.7) completed follow-up. After 3 months of CSAI, daily OFF hours, dyskinesia hours and UPDRS Part 4 scores decreased significantly (<i>p</i> < 0.05 for all). The oral levodopa daily dose was significantly reduced (<i>p</i> < 0.001). Serum vitamin B₁ and B₁₂ levels increased significantly, while Hcy levels showed a modest but significant reduction (<i>p</i> < 0.05). Body weight, BMI and total MNA-SF score remained unchanged (<i>p</i> > 0.05). However, MNA-SF categories shifted toward improved nutritional classification (<i>p</i> = 0.008). Adjunctive antiparkinsonian medications were significantly reduced, while levodopa remained universally prescribed.</p><p><strong>Conclusion: </strong>CSAI significantly reduced motor complications and oral levodopa exposure. Secondary analyses showed selective biochemical improvements with stable overall nutritional status. Findings should be interpreted considering the observational design, lack of control group and short follow-up.</p>","PeriodicalId":22980,"journal":{"name":"Therapeutic Advances in Neurological Disorders","volume":"19 ","pages":"17562864261446737"},"PeriodicalIF":4.0,"publicationDate":"2026-05-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13167274/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147943033","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Meningococcal prophylaxis in neurological diseases treated with complement inhibitors: an expert consensus for Germany, Austria, and Switzerland.","authors":"Achim Berthele, Orhan Aktas, Ilya Ayzenberg, Heike Claus, Thiên-Trí Lâm, Wolfgang N Löscher, Anne-Katrin Pröbstel, Harald Prüss, Nina Rademacher, Marius Ringelstein, Paulus Rommer, Ulrike Schara-Schmidt, Benedikt Schoser, Jörg R Weber, Jana Zschüntzsch, Andreas Meisel, Jan D Lünemann","doi":"10.1177/17562864261442558","DOIUrl":"10.1177/17562864261442558","url":null,"abstract":"<p><p>Inhibition of terminal complement activation is an effective therapeutic strategy for acetylcholine receptor antibody-positive generalized myasthenia gravis (gMG) and aquaporin-4 antibody-positive neuromyelitis optica spectrum disorder (NMOSD), but it increases the risk of invasive meningococcal infections. Consequently, vaccination against meningococcal serogroups A, C, W, Y, and B is mandatory for all patients receiving complement inhibitors. This article provides expert consensus recommendations for managing meningococcal vaccination in patients with NMOSD and gMG receiving complement-inhibiting therapies in Germany, Austria, and Switzerland. The timing and procedures of vaccination should be adapted to the underlying diagnosis and the individual risk of disease exacerbation in this population. In NMOSD, treatment often must begin promptly, particularly after an acute attack; in such cases, vaccination should be administered at treatment initiation together with antibiotic prophylaxis. By contrast, treatment urgency in gMG is typically lower, allowing vaccination to be completed in advance and thereby avoiding antibiotic exposure, which may worsen gMG symptoms. For both diseases, ceftriaxone is recommended as first-line therapy for suspected infection, rifampicin for prophylaxis, and either rifampicin or intramuscular ceftriaxone for post-exposure chemoprophylaxis. Patients should also carry a standby dose of ciprofloxacin for emergency self-administration at the first signs of meningitis, followed by immediate clinical evaluation. These recommendations should be reviewed regularly and updated as necessary to reflect emerging evidence and new vaccine options.</p>","PeriodicalId":22980,"journal":{"name":"Therapeutic Advances in Neurological Disorders","volume":"19 ","pages":"17562864261442558"},"PeriodicalIF":4.0,"publicationDate":"2026-05-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13167371/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147943008","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Rituximab and IVIG added to plasma exchange improves the outcome of severe CNS demyelinating attacks: a retrospective study.","authors":"Juan Luis Chico-Garcia, Moisés Morales de la Prida, Almudena Boix Lago, José Manuel García-Domínguez, María Sepúlveda, Eugenia Martínez-Hernández, Mar Guasp, Elianet Fonseca, Alberto Calvi, María Teresa Alba-Isasi, Blanca Albertí, Joan Cid, Miquel Lozano, Paola Charry, Fernando Rodríguez-Jorge, Raquel Sainz-Amo, Susana Sainz de la Maza, Enric Monreal, Luisa Maria Villar, Jaime Masjuan, Gabriel Valero-López, Jorge Millán-Pascual, Juan Pablo Cuello, Michael Palacios, Ariadna Gifreu Fraixinó, María Luisa Martínez-Gins, Gary Álvarez Bravo, José E Meca-Lallana, Lucienne Costa-Frossard, Yolanda Blanco, Jose Maria Cabrera-Maqueda, Albert Saiz, Sara Llufriu","doi":"10.1177/17562864261440665","DOIUrl":"10.1177/17562864261440665","url":null,"abstract":"<p><strong>Background: </strong>Plasma exchange (PE) is a therapeutic option for CNS demyelinating attacks unresponsive to corticosteroids, but the potential benefit of adding rituximab and IV immunoglobulins to PE (RTX-IVIG-PE), which has shown its efficacy and safety in other immune-mediated conditions, has not been investigated.</p><p><strong>Objective: </strong>We aimed to determine the overall effectiveness of PE in severe demyelinating attacks, identify predictors of response, and compare outcomes between standard PE and RTX-IVIG-PE protocols.</p><p><strong>Design: </strong>Retrospective analysis of patients with CNS demyelinating attacks treated with PE between 2012 and 2023 across five Spanish hospitals.</p><p><strong>Methods: </strong>Clinical response was measured as an improvement or a complete recovery at discharge and 6 months. Uni- and multivariable logistic regression models were developed to identify predictors of PE response. A logistic regression analysis, after inverse probability of treatment weighting (IPTW), was performed to compare the outcome of attacks treated with standard PE and RTX-IVIG-PE.</p><p><strong>Results: </strong>A total of 162 attacks in 150 patients (81 multiple sclerosis; 37 others acquired demyelinating syndromes; 27 neuromyelitis optica spectrum disorder; 5 myelin oligodendrocyte glycoprotein antibody-associated disease; 73% female; median age 43 years (interquartile range 33-52.75)) were analyzed. At discharge, 60% of all attacks showed clinical improvement, and 15% achieved complete recovery. At 6 months, these rates increased to 77% and 31%, respectively. Younger age, lower preattack Expanded Disability Status Scale, and treatment with RTX-IVIG-PE were independent predictors of complete recovery at 6 months. IPTW analysis showed that attacks treated with RTX-IVIG-PE (33) were associated with improvement (adjusted odds ratio (aOR) 5.4, <i>p</i> < 0.001) and complete recovery (aOR 4.55, <i>p</i> < 0.001) at 6 months compared to those treated with standard PE (129), without significant differences in the adverse event profile between the two protocols.</p><p><strong>Conclusion: </strong>PE improves the outcome of steroid-refractory CNS demyelinating attacks, and the addition of rituximab and IVIG to PE may increase the likelihood of long-term recovery.</p>","PeriodicalId":22980,"journal":{"name":"Therapeutic Advances in Neurological Disorders","volume":"19 ","pages":"17562864261440665"},"PeriodicalIF":4.0,"publicationDate":"2026-05-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13167357/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147943031","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Brivaracetam monotherapy in juvenile myoclonic epilepsy: a safety and efficacy evaluation.","authors":"Jing-Wen Zuo, Ying-Yue Dai, Wen-Jing Liu, Jia-Ying Zhang, Xiao-Qiu Shao, Qun Wang, Rui-Juan Lv","doi":"10.1177/17562864261440217","DOIUrl":"10.1177/17562864261440217","url":null,"abstract":"&lt;p&gt;&lt;strong&gt;Background: &lt;/strong&gt;The treatment of juvenile myoclonic epilepsy (JME) is limited, with most patients requiring long-term medication and over half experiencing seizure recurrence upon drug withdrawal. As a third-generation antiseizure medication, brivaracetam (BRV) has emerged as a promising therapeutic option. Its efficacy has been investigated in focal epilepsies and genetic generalized epilepsies (GGEs), with promising results.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Objectives: &lt;/strong&gt;This study aims to evaluate the safety and preliminary efficacy of BRV as an off-label initial monotherapy in patients with newly diagnosed JME, with a specific focus on the control of myoclonic seizures.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Design: &lt;/strong&gt;A prospective, single-center, and observational study.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Methods: &lt;/strong&gt;This study prospectively enrolled drug-naïve patients with JME. All participants received BRV monotherapy. Clinical data were collected at baseline and after a 6-month follow-up period, including demographic characteristics, electroencephalography (EEG), cranial magnetic resonance imaging (MRI), and comprehensive neuropsychological assessments. Changes in seizure frequency, cognitive function, levels of anxiety and depression, sleep quality, and quality of life from baseline to the 6-month follow-up were analyzed and compared.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Results: &lt;/strong&gt;A total of 19 patients were included with a mean age of 20.26 ± 6.88 years (median: 18, interquartile range: 8), and a male-to-female ratio of 12:7. The average age of onset was 14.58 ± 3.42 years, and the average duration of epilepsy prior to BRV treatment was 5.71 ± 7.40 years (median: 2, interquartile range: 5). The mean frequency of myoclonic seizures at baseline was 38.79 ± 45.60 times per month (median: 10, interquartile range: 86). Eighteen patients (94.73%) experienced both generalized tonic-clonic seizures (GTCS) and myoclonic seizures, one patient only experienced myoclonic seizures, The MRI findings were negative in all patients (100%). The EEG of all patients at baseline was abnormal, revealing 3-5.5 Hz generalized spike-and-wave or polyspike-and-wave discharges. At the 6-month evaluation, all patients achieved seizure-free status (&lt;i&gt;p&lt;/i&gt; &lt; 0.001), neuropsychological assessments also demonstrated significant improvement, including Montreal Cognitive Assessment (MoCA; &lt;i&gt;p&lt;/i&gt; &lt; 0.001), Hamilton Anxiety Scale (HAMA; &lt;i&gt;p&lt;/i&gt; &lt; 0.001), Hamilton Depression Scale (HAMD; &lt;i&gt;p&lt;/i&gt; &lt; 0.001), Pittsburgh Sleep Quality Index (PSQI; &lt;i&gt;p&lt;/i&gt; &lt; 0.001), and Quality of Life in Epilepsy-31 (QOLIE-31; &lt;i&gt;p&lt;/i&gt; &lt; 0.001). Only one patient complained of poor sleep after BRV administration.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Conclusion: &lt;/strong&gt;This study suggests that BRV may offer promising efficacy, specifically in controlling myoclonic seizures and favorable tolerability as an off-label initial monotherapy for JME patients. While the evaluation of efficacy against GTCS requires longer follow-up, our findings support the pote","PeriodicalId":22980,"journal":{"name":"Therapeutic Advances in Neurological Disorders","volume":"19 ","pages":"17562864261440217"},"PeriodicalIF":4.0,"publicationDate":"2026-04-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13125810/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147821142","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Natalizumab for pediatric multiple sclerosis: a systematic review and meta-analysis.","authors":"Yasin Ebne-Ali-Heydari, Aryana Ramezani, Amirmohammad Jozayi, Mahdi Norouzi, Parto Zohrabi, Hasan Kaveyee","doi":"10.1177/17562864261440657","DOIUrl":"10.1177/17562864261440657","url":null,"abstract":"<p><strong>Background: </strong>Pediatric-onset multiple sclerosis (POMS) is the onset of MS before the age of 18 and accounts for 3%-5% of all multiple sclerosis (MS) cases. Natalizumab (NTZ) is among the higher-efficacy disease-modifying treatments (HETs) in MS and is increasingly used for POMS.</p><p><strong>Objectives: </strong>In this systematic review and meta-analysis, we aimed to discuss the debate on the efficacy and safety of natalizumab use in POMS, providing quantitative results on relapse rate, disability progression, adverse events (AEs), and JC virus seropositivity.</p><p><strong>Design: </strong>The primary endpoint for meta-analysis was the mean difference (MD) in annualized relapse rate (ARR) after natalizumab compared to before treatment. Secondary outcomes were the MD of Expanded Disability Status Scale (EDSS) and the proportion of POMS patients experiencing AEs and JC virus seropositivity after natalizumab treatment.</p><p><strong>Data sources and methods: </strong>We performed a comprehensive search of PubMed, Embase, Web of Science, and Scopus between January 1, 1991 and May 1, 2025.</p><p><strong>Results: </strong>In this systematic review, 18 non-randomized interventional studies including 922 patients with POMS were included. Natalizumab therapy was associated with a mean reduction in ARR of -1.962 relapses per patient-year from baseline (95% confidence interval (CI): -2.449 to -1.475; <i>p</i> < 0.001). The drug was also associated with a statistically significant improvement in disability, with a mean change in EDSS of -0.807 from baseline (95% CI: -1.078 to -0.536; <i>p</i> < 0.001). After treatment, 18% of patients experienced AEs (95% CI: 0.11-0.25), and JC virus seropositivity was observed in 12% (95% CI: 0.07-0.17). No case of progressive multifocal leukoencephalopathy was reported among the 922 natalizumab-treated patients.</p><p><strong>Conclusion: </strong>Natalizumab may represent a viable therapeutic option for POMS patients exhibiting highly active disease or serves as an effective alternative in those with inadequate response to initial treatment. The safety profile remains acceptable, with most AEs being manageable.</p><p><strong>Registration: </strong>PROSPERO (CRD42024583911). This study follows the Preferred Reporting Items for Systematic Reviews and Meta-Analyses reporting guidelines.</p>","PeriodicalId":22980,"journal":{"name":"Therapeutic Advances in Neurological Disorders","volume":"19 ","pages":"17562864261440657"},"PeriodicalIF":4.0,"publicationDate":"2026-04-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13100434/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147781803","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"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 apomorphine in the treatment of Parkinson's disease: a systematic review and meta-analysis of randomized controlled trials.","authors":"Moaz Elsayed Abouelmagd, Abdallah Abbas, Obai Yousef, Atef Abdelrahman Hassan, Omar Kassar, Khalid Sarhan, Asmaa Zakria Alnajjar, Haneen Sabet, Mohamed Ahmed Zanaty, Hoda Awad, Muataz Kashbour, Fakhruddin Almuzghi, Matthew J Barrett, Brian Berman, Ahmed Negida","doi":"10.1177/17562864261436541","DOIUrl":"10.1177/17562864261436541","url":null,"abstract":"<p><strong>Background: </strong>Motor fluctuations and OFF episodes are common complications of long-term levodopa therapy in Parkinson's disease (PD) and significantly impair quality of life. Apomorphine, a short-acting dopamine agonist, is available in multiple formulations for on-demand symptom relief; however, comparative evidence across administration routes remains limited.</p><p><strong>Objectives: </strong>To evaluate the efficacy and safety of apomorphine across various routes of administration in patients with PD.</p><p><strong>Design: </strong>Systematic review and meta-analysis of randomized controlled trials and randomized crossover trials.</p><p><strong>Data sources and methods: </strong>A comprehensive search of PubMed, Scopus, Web of Science, CENTRAL, and Embase was conducted from inception to December 28, 2025. Eligible studies included randomized controlled or crossover trials assessing apomorphine versus placebo in PD. Primary outcomes were motor improvement measured by Unified Parkinson's Disease Rating Scale III (UPDRS-III) or Movement Disorder Society-UPDRS-III (MDS-UPDRS-III) and OFF time. Safety outcomes included treatment-related adverse events. Risk of bias was assessed using the Cochrane RoB 2 tool. Pooled analyses were conducted using standardized mean differences (SMD), mean differences (MD), and risk ratios (RR) with 95% confidence intervals (CIs).</p><p><strong>Results: </strong>Thirteen studies involving 557 patients were included. Apomorphine significantly improved motor symptoms across multiple administration routes. Intermittent subcutaneous (SC) injection showed the greatest efficacy (SMD: -2.19, 95% CI: (-3.32, -1.05), <i>p</i> < 0.0001). The inhalation and sublingual routes also showed significant improvement (SMD: -1.11, 95% CI: (-1.52, -0.7), <i>p</i> < 0.0001, <i>I</i> ² = 0%), (SMD: -1.69, 95% CI: (-1.99, -1.38), <i>p</i> < 0.0001, <i>I</i> ² = 0%) respectively. Intermittent SC injections also significantly reduced OFF time (MD = -1.62 h; 95% CI = (-2.59, -0.65); <i>p</i> < 0.00001). Adverse events were more frequent with apomorphine (RR 1.50, 95% CI = 1.09-2.06), particularly nausea, vomiting, dyskinesia, somnolence, yawning, and rhinorrhea.</p><p><strong>Conclusion: </strong>Apomorphine is an effective on-demand therapy for reducing motor symptoms and OFF time in PD, particularly when administered as intermittent SC injection. Although associated with increased mild-to-moderate adverse events, treatment choice should be individualized.</p><p><strong>Trial registration: </strong>PROSPERO (CRD42024548330).</p>","PeriodicalId":22980,"journal":{"name":"Therapeutic Advances in Neurological Disorders","volume":"19 ","pages":"17562864261436541"},"PeriodicalIF":4.0,"publicationDate":"2026-04-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13100369/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147781755","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Cerebrovascular fibromuscular dysplasia.","authors":"Marion Boulanger, Ahmad Nehme, Emmanuel Touzé","doi":"10.1177/17562864251386246","DOIUrl":"10.1177/17562864251386246","url":null,"abstract":"<p><strong>Background: </strong>Cerebrovascular fibromuscular dysplasia (FMD), that is, involving the cervical or intracranial arteries, is frequently overlooked despite its association with several neurological manifestations.</p><p><strong>Objectives: </strong>To describe cerebrovascular FMD.</p><p><strong>Design: </strong>Systematic review.</p><p><strong>Data sources and methods: </strong>We searched PubMed, Embase, and Medline up to December 2024. We identified additional studies by handsearching the bibliographies of relevant studies. We included articles describing at least one aspect of epidemiology, manifestations, prognosis, and management of cerebrovascular FMD and preferentially focused on studies including ⩾15 patients. The quality of evidence was rated according to the Oxford Centre for Evidence-Based Medicine levels of evidence.</p><p><strong>Results: </strong>We included 119 studies. Cerebrovascular FMD predominantly affects women around 50 years of age and most frequently involves the cervical arteries. Cerebrovascular FMD is frequently asymptomatic and incidentally diagnosed. The most common presenting symptoms are headache (8%-52%) and pulsatile tinnitus (17%-23%). Patients with FMD have a higher prevalence of cervical artery dissection (7%-17%) and intracranial saccular aneurysm (7%) compared to the general population. The main complications of cerebrovascular FMD are ischemic stroke (7%-10%), transient ischemic attack (5%-12%), subarachnoid hemorrhage (2%-3%), and intracerebral hemorrhage (0.01%). Webs are the predominant form of FMD in Black populations. Consensus-based recommendations have been summarized in the first international consensus statement on the diagnosis and management of FMD. Imaging of all arteries from brain-to-pelvis is recommended to detect asymptomatic lesions. Antiplatelet therapy is reasonable for patients with cerebrovascular FMD to prevent thromboembolic complications. Endovascular treatment can be considered in patients with risk factors for rupture of an unruptured intracranial aneurysm and in those with recurrent ischemic events despite medical management.</p><p><strong>Conclusion: </strong>Existing cross-sectional studies have improved the understanding of the manifestations of cerebrovascular FMD. However, knowledge gaps remain regarding the genetics, pathophysiology, management, and prognosis of cerebrovascular FMD, which should be addressed in future research.</p>","PeriodicalId":22980,"journal":{"name":"Therapeutic Advances in Neurological Disorders","volume":"19 ","pages":"17562864251386246"},"PeriodicalIF":4.0,"publicationDate":"2026-04-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13100393/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147781798","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Preliminary observations on the efficacy of efgartigimod in anti-LGI1-associated autoimmune encephalitis.","authors":"Jing-Wen Zuo, Ying-Yue Dai, Wen-Jing Liu, Jia-Ying Zhang, Xiao-Qiu Shao, Qun Wang, Rui-Juan Lv","doi":"10.1177/17562864261437004","DOIUrl":"10.1177/17562864261437004","url":null,"abstract":"<p><strong>Background: </strong>Leucine-rich glioma-inactivated 1 (LGI-1) antibodies are the second most common cause of autoimmune encephalitis, which is characterized by frequent seizures, memory loss, and psychiatric symptoms. Although most patients respond well to immunotherapy, treatment is often delayed or insufficient, resulting in a 14%-35% relapse rate and persistent neurological deficits. Therefore, exploring fast-acting and targeted therapies is crucial.</p><p><strong>Objectives: </strong>To present a series of cases involving anti-LGI1 encephalitis treated with efgartigimod, and to assess the efficacy and safety of this therapeutic approach in managing this condition.</p><p><strong>Design: </strong>A prospective, multicenter, and observational study.</p><p><strong>Methods: </strong>This study prospectively enrolled patients with anti-LGI1 encephalitis treated with efgartigimod and conducted a systematic review of the literature on the use of efgartigimod for anti-LGI1 encephalitis. The clinical outcomes before and 2 weeks after treatment were then compared.</p><p><strong>Results: </strong>A total of eight patients and two additional cases from the literature were included in the study, with a mean age of 55.6 ± 15.88 years and a male-to-female ratio of 6:4. The predominant clinical manifestations among the patients included seizures, psychiatric and behavioral abnormalities, and memory decline. Following treatment with efgartigimod, the modified Rankin scale (<i>p</i> = 0.006; 95% confidence interval (CI) 2-2.5), Clinical Assessment Scale in Autoimmune Encephalitis (<i>p</i> = 0.008; 95% CI 2.5-9.5), and Mini-Mental State Examination scores (<i>p</i> = 0.042; 95% CI 0-14) significantly improved. In addition, a significant reduction was observed in both serum IgG levels (<i>p</i> < 0.001; 95% CI 4.66-6.81) and antibody titers (<i>p</i> = 0.004) post-treatment, indicating that the degree of antibody decline is negatively correlated with clinical severity. No adverse events were reported for any of the patients in this study.</p><p><strong>Conclusion: </strong>This study suggests that efgartigimod may offer promising efficacy and safety for patients with anti-LGI1 encephalitis. Further randomized controlled trials involving larger cohorts and extended follow-up periods are required to validate these findings.</p>","PeriodicalId":22980,"journal":{"name":"Therapeutic Advances in Neurological Disorders","volume":"19 ","pages":"17562864261437004"},"PeriodicalIF":4.0,"publicationDate":"2026-04-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13100446/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147781849","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Real-world effectiveness and safety of ofatumumab in multiple sclerosis: a longitudinal study integrating clinical, cognitive, and MRI outcomes.","authors":"Stefano Ziccardi, Damiano Marastoni, Pietro Biasi, Agnese Tamanti, Francesca Benedetta Pizzini, Milena Calderone, Valentina Camera, Maddalena Bertolazzo, Alice Daldosso, Bianca Milocco, Roberta Magliozzi, Massimiliano Calabrese","doi":"10.1177/17562864261434351","DOIUrl":"10.1177/17562864261434351","url":null,"abstract":"<p><strong>Background: </strong>Despite the availability of several disease-modifying therapies for multiple sclerosis (MS) patients, the optimal strategy remains debated. High-efficacy therapies may better prevent subclinical disease activity and long-term disability; however, escalation remains frequent in real-world practice. Ofatumumab, an anti-CD20 monoclonal antibody, demonstrated robust efficacy and safety in clinical trials; however, real-world data are also essential.</p><p><strong>Objectives: </strong>To evaluate the effectiveness and safety of ofatumumab in relapsing MS (RMS), comparing outcomes between treatment-naïve and previously treated patients, and identify predictors of suboptimal response.</p><p><strong>Design: </strong>Prospective longitudinal observational study of RMS patients followed at the Verona MS Center.</p><p><strong>Methods: </strong>Clinical assessments, annual 3.0T brain MRI, and comprehensive neuropsychological testing were performed throughout follow-up. Treatment effectiveness was evaluated using no evidence of disease activity (NEDA), progression independent of relapse activity (PIRA), and cognitive PIRA. Safety was assessed by recording adverse events and treatment discontinuations.</p><p><strong>Results: </strong>Eighty-nine RMS patients (68.5% female, mean age 38.0 ± 10.4 years) were followed for a mean of 3 years. Overall, 91% achieved NEDA-3, with one relapse and one case of MRI activity. Seven patients (7.9%) developed PIRA, six of whom also fulfilled criteria for cognitive PIRA. Among 46 NEDA-3 patients who underwent longitudinal cognitive assessment, 37 (80.4%) did not exhibit cognitive worsening during follow-up and were classified as NEDA-4, indicating stability across clinical, radiological, and cognitive domains. In general, among patients with longitudinal cognitive data, 37 of 54 (68.5%) were NEDA-4. Naïve and previously treated patients showed comparable outcomes. Patients who failed to maintain NEDA-3 were older and had higher baseline disability, whereas in multivariable analyses, baseline Expanded Disability Status Scale (EDSS) was the only factor independently associated with NEDA-3 loss. When cognitive outcomes were included, associations between baseline clinical variables and NEDA-4 loss were attenuated. Ofatumumab was well-tolerated, with mostly mild transient injection-related reactions and 2 (2.2%) treatment discontinuations due to adverse events.</p><p><strong>Conclusion: </strong>Ofatumumab provided sustained multidimensional disease control, high NEDA-4 rates, and excellent tolerability. Baseline EDSS was the primary predictor of suboptimal response, underscoring the importance of early intervention. These findings support early anti-CD20 therapy as an effective strategy to preserve neurological function and limit long-term progression.</p>","PeriodicalId":22980,"journal":{"name":"Therapeutic Advances in Neurological Disorders","volume":"19 ","pages":"17562864261434351"},"PeriodicalIF":4.0,"publicationDate":"2026-04-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13058188/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147646476","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Pathophysiology of chronic subdural hematoma-new insights.","authors":"Misa Trieu, Ajith J Thomas","doi":"10.1177/17562864261435399","DOIUrl":"10.1177/17562864261435399","url":null,"abstract":"<p><p>Chronic subdural hematoma (CSDH) is an increasingly common disorder characterized by persistent accumulation of blood products and inflammatory exudate within the dural border cell (DBC) layer. Its pathogenesis represents a self-sustaining cycle of inflammation, pathological angiogenesis, and impaired resolution. Injury of the head, which may be traumatic or nontraumatic, initiates cleavage of the DBC layer, leading to fibroproliferative membrane formation mediated by collagen synthesis and TGF-β1/SMAD signaling. The resulting outer membrane develops fragile neovasculature, driven primarily by vascular endothelial growth factor, facilitating recurrent microhemorrhage and fibrinolysis perpetuating hematoma expansion. Chronic inflammation sustains disease progression through macrophage polarization, cytokine release, and increased vascular permeability, processes amplified by age-related immune dysregulation and hypoxia-induced factor-1alpha signaling. Impaired tissue repair due to metabolic deficits further limits resolution. Concurrent dysfunction of meningeal lymphatic drainage and fibrotic arachnoid granulations compromises clearance of blood degradation products and inflammatory mediators, while matrix remodeling and cerebrospinal fluid ingress contribute to hematoma persistence. This narrative review presents a pathophysiologic framework highlighting CSDH as a dynamic inflammatory-angiogenic disorder. Pharmacological strategies targeting inflammation, angiogenesis, fibrinolysis, hypoxia, and matrix remodeling hold potential as complements or alternatives to established treatments, including surgical drainage and middle meningeal artery embolization. As the burden of CSDH on healthcare systems rises, translational research and controlled clinical trials will be critical to developing mechanism-driven, multimodal management paradigms.</p>","PeriodicalId":22980,"journal":{"name":"Therapeutic Advances in Neurological Disorders","volume":"19 ","pages":"17562864261435399"},"PeriodicalIF":4.0,"publicationDate":"2026-04-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13053969/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147639951","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}