Therapeutic Advances in Neurological Disorders最新文献

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Optimized use of safinamide as an add-on therapy in Asian patients with Parkinson's disease: a narrative review and expert opinion. 沙非胺作为亚洲帕金森病患者附加治疗的优化使用:叙述性回顾和专家意见
IF 4.7 2区 医学
Therapeutic Advances in Neurological Disorders Pub Date : 2025-04-17 eCollection Date: 2025-01-01 DOI: 10.1177/17562864251329099
Jong Sam Baik, Young Hee Sung, Ruey-Meei Wu, Chin-Song Lu, Roongroj Bhidayasiri
{"title":"Optimized use of safinamide as an add-on therapy in Asian patients with Parkinson's disease: a narrative review and expert opinion.","authors":"Jong Sam Baik, Young Hee Sung, Ruey-Meei Wu, Chin-Song Lu, Roongroj Bhidayasiri","doi":"10.1177/17562864251329099","DOIUrl":"https://doi.org/10.1177/17562864251329099","url":null,"abstract":"<p><p>Parkinson's disease (PD) imposes a large burden on Asian countries and threatens to grow rapidly as Asian populations age. PD phenotypes in Asian patients differ from those reported in the West, yet management generally follows a similar approach. Levodopa (l-dopa) is a mainstay of therapy and is typically followed by the addition of a catechol-<i>O</i>-methyltransferase inhibitor or a monoamine oxidase-B (MAO-B) inhibitor to address the wearing-off effect. There is little guidance on switching between MAO-B inhibitors or other adjunct therapies that consider the newer evidence for safinamide as an add-on PD therapy in Asian patients. Therefore, a group of PD experts in Asia evaluated the evidence supporting safinamide for the treatment of PD with a focus on integrating this treatment option into local clinical practice. A narrative review was conducted to identify supportive evidence for the formulation of summary statements on key topics. The efficacy and safety of safinamide added to l-dopa in Asian patients with PD are supported by both clinical trials and observational data, including two randomized trials enrolling exclusively Asian patients (<i>n</i> = 406; <i>n</i> = 307) and an Asian subpopulation analysis from another randomized trial (<i>n</i> = 173). Safinamide reduces wear-off duration and has beneficial effects on motor symptoms of PD, with good tolerability outcomes. Safinamide may also have beneficial effects on non-motor symptoms of PD such as urinary symptoms, apathy and sleep disturbances, and it is a suitable treatment for older patients. Overall, safinamide is an effective and well-tolerated treatment for the wear-off effect of l-dopa in Asian patients and, during long-term treatment, might reduce the risk of dyskinesia in patients without pre-existing dyskinesia. Additional research is needed to better understand the role of safinamide for patients with fluctuating pain, the dose-effect relationship of safinamide in Asian patients and the efficacy of safinamide in Asian patients with early-onset PD.</p>","PeriodicalId":22980,"journal":{"name":"Therapeutic Advances in Neurological Disorders","volume":"18 ","pages":"17562864251329099"},"PeriodicalIF":4.7,"publicationDate":"2025-04-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12033687/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144044026","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}
引用次数: 0
Shifts in treatment initiation patterns among newly diagnosed multiple sclerosis patients in Germany: a claims data analysis from 2017 to 2022. 德国新诊断多发性硬化症患者治疗开始模式的转变:2017年至2022年的索赔数据分析
IF 4.7 2区 医学
Therapeutic Advances in Neurological Disorders Pub Date : 2025-04-17 eCollection Date: 2025-01-01 DOI: 10.1177/17562864251328576
Ann-Sophie Stratil, Steffeni Papukchieva, Natalie Joschko, Sven Guenther Meuth, Benjamin Friedrich
{"title":"Shifts in treatment initiation patterns among newly diagnosed multiple sclerosis patients in Germany: a claims data analysis from 2017 to 2022.","authors":"Ann-Sophie Stratil, Steffeni Papukchieva, Natalie Joschko, Sven Guenther Meuth, Benjamin Friedrich","doi":"10.1177/17562864251328576","DOIUrl":"https://doi.org/10.1177/17562864251328576","url":null,"abstract":"<p><strong>Background: </strong>Early use of high-efficacy therapies (EHT) in multiple sclerosis (MS) is a promising but novel treatment strategy. Its adoption in Germany's MS care warrants further research.</p><p><strong>Objectives: </strong>This study assessed treatment initiation patterns among newly diagnosed MS patients in Germany (2017-2022).</p><p><strong>Design: </strong>This is a retrospective observational study.</p><p><strong>Methods: </strong>Claims data from 4.5 million individuals insured by German statutory health insurance included 1448 newly diagnosed MS patients from 2017 to 2022. Patients were identified by International Statistical Classification of Diseases and Related Health Problems, 10th revision, German modification (ICD-10-GM) code G35 - in two different quarters of the same year, and a disease-modifying therapy (DMT) prescription, with no prior MS diagnosis or DMT prescription in the preceding 2 years. DMTs were categorized according to German Society of Neurology S2k guidelines: category 1 (low-efficacy), category 2 (moderate-efficacy) and category 3 (high-efficacy).</p><p><strong>Results: </strong>Of patients initiating treatment, 77.1% started with category 1, 8.1% with category 2 and 14.8% with category 3 DMTs. From 2017 to 2022, category 1 initiations declined by 7.6% points (pp), while categories 2 and 3 initiations increased by 2.8 and 4.8 pp, respectively. Escalation to category 3 occurred in 10.5% of category 1/2 starters, with 31.5% taking over 2 years. De-escalation to category 1/2 occurred in 3.3% of category 3 starters.</p><p><strong>Conclusion: </strong>Real-world data highlight a shift towards EHT in MS care.</p>","PeriodicalId":22980,"journal":{"name":"Therapeutic Advances in Neurological Disorders","volume":"18 ","pages":"17562864251328576"},"PeriodicalIF":4.7,"publicationDate":"2025-04-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12033395/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144047001","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}
引用次数: 0
The therapeutic effects and mechanisms of glucagon-like peptide-1 receptor agonists in neurocognitive disorders. 胰高血糖素样肽-1受体激动剂治疗神经认知障碍的疗效及机制。
IF 4.7 2区 医学
Therapeutic Advances in Neurological Disorders Pub Date : 2025-04-17 eCollection Date: 2025-01-01 DOI: 10.1177/17562864251332035
Junchen Si, Kai Yu, Jiheng Hao, Jiyue Wang, Liyong Zhang
{"title":"The therapeutic effects and mechanisms of glucagon-like peptide-1 receptor agonists in neurocognitive disorders.","authors":"Junchen Si, Kai Yu, Jiheng Hao, Jiyue Wang, Liyong Zhang","doi":"10.1177/17562864251332035","DOIUrl":"https://doi.org/10.1177/17562864251332035","url":null,"abstract":"<p><p>Chronic cerebral hypoperfusion (CCH) represents a key pathogenic contributor to neurocognitive disorders. It can lead to multifaceted pathological alterations including neuroinflammation, neuronal apoptosis, blood-brain barrier disruption, synaptic plasticity deficits, and mitochondrial dysfunction. The glucagon-like peptide-1 receptor (GLP-1R), ubiquitously expressed across multiple organ systems, exerts neuroprotective effects by maintaining intracellular homeostasis and mitigating neuronal damage triggered by oxidative stress, inflammatory cascades, apoptotic signaling, and ischemic insults. Furthermore, GLP-1R activity is modulated by gut microbiota composition and short-chain fatty acid abundance, implicating the gut-brain axis in its regulatory influence on neurological function. This review systematically examines the pathophysiological mechanisms underlying CCH and highlights the therapeutic potential of GLP-1R activation. Specifically, GLP-1R-targeted interventions attenuate hypoperfusion-induced damage through pleiotropic pathways and gut-brain crosstalk, thereby offering novel perspectives for advancing both fundamental research and clinical management of neurocognitive disorders.</p>","PeriodicalId":22980,"journal":{"name":"Therapeutic Advances in Neurological Disorders","volume":"18 ","pages":"17562864251332035"},"PeriodicalIF":4.7,"publicationDate":"2025-04-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12033604/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144019798","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}
引用次数: 0
Predictors of acute symptomatic seizure in cerebral venous thrombosis patients-a multicenter cohort study. 脑静脉血栓患者急性症状性发作的预测因素——一项多中心队列研究。
IF 4.7 2区 医学
Therapeutic Advances in Neurological Disorders Pub Date : 2025-04-17 eCollection Date: 2025-01-01 DOI: 10.1177/17562864251330864
Naaem Simaan, Jeremy Molad, Hen Hallevi, Issa Metanis, Rom Mendel, Rani Barnea, Eitan Auriel, Jonathan Naftali, Ruth Eliahou, Shorooq Aladdin, David Orion, Shlomi Peretz, Ronen R Leker, Asaf Honig
{"title":"Predictors of acute symptomatic seizure in cerebral venous thrombosis patients-a multicenter cohort study.","authors":"Naaem Simaan, Jeremy Molad, Hen Hallevi, Issa Metanis, Rom Mendel, Rani Barnea, Eitan Auriel, Jonathan Naftali, Ruth Eliahou, Shorooq Aladdin, David Orion, Shlomi Peretz, Ronen R Leker, Asaf Honig","doi":"10.1177/17562864251330864","DOIUrl":"https://doi.org/10.1177/17562864251330864","url":null,"abstract":"<p><strong>Background: </strong>Acute symptomatic seizures (ASYS) is a common presentation in cerebral sinus venous thrombosis (CSVT) patients.</p><p><strong>Objectives: </strong>We aimed to characterize CSVT patients experiencing ASYS within 7 days from presentation. Additionaly, we aimed to find predictors for ASYS within CSVT patients.</p><p><strong>Methods: </strong>Prospective CSVT databases from six academic centers (January 2010-December 2023) were retrospectively analyzed. Clinical outcomes at the 90-day follow-up included seizure occurrence and the modified-Rankin-Scale (mRS).</p><p><strong>Results: </strong>From 529 included patients (mean age 42.4 ± 18.6 years, 64.3% females), 106 (20%) had ASYS. ASYS patients were more often males (47.2% vs 20.1%, <0.001), and presented more often with focal neurological deficits (50% vs 22%, <i>p</i> < 0.001) but less often with papilledema (13.2% vs 29.3%, <i>p</i> < 0.001). On multivariate analysis cortical-vein thrombosis (odds ratio (OR) 4.17, <i>p</i> < 0.001), intracerebral hemorrhage (ICH; OR 3.06, <i>p</i> = 0.002), any superior-sagittal-sinus (SSS) thrombosis (OR 2.49, <i>p</i> = 0.006), predicted ASYS. Conversely, presentation with papilledema (OR 0.39, <i>p</i> = 0.03) negatively predicted ASYS. ASYS patients had lower rates of 90-day-mRS-0-1 (51.9% vs 83.9%, <i>p</i> < 0.001). Patients who experienced seizures between the second and seventh day (n = 58) had similar baseline characteristics to those with seizures only on the day of presentation (n = 48) but were less likely to achieve a good functional outcome by day 90 (42.6% vs 58.9%, <i>p</i> < 0.05) and had a lower rate of complete recanalization on follow-up venous imaging (25.5% vs 57.5%, <i>p</i> = 0.02). Status-epilepticus in comparison to non-ASYS patients achieved lower rates of 90-day-mRS-0-1 (11% vs 84%, <i>p</i> < 0.001) and higher 90-day-mortality (44% vs 5.6%, <i>p</i> < 0.001). In a multivariate analysis ASYS was a negative predictor for 90-day-mRS-0-1 (OR 3.3, 95% confidence interval 1.43-7.5, <i>p</i> = 0.005).</p><p><strong>Conclusion: </strong>CSVT patients experiencing ASYS, and to a greater degree patients with either status epilepticus or ASYS between second and seventh day achieved less often 90-day-mRS-0-1. Possibly, they epitomize a different course of disease that may require a more suitable treatment strategy.</p>","PeriodicalId":22980,"journal":{"name":"Therapeutic Advances in Neurological Disorders","volume":"18 ","pages":"17562864251330864"},"PeriodicalIF":4.7,"publicationDate":"2025-04-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12033526/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144054438","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}
引用次数: 0
Advances in the treatment of neuromyelitis optic spectrum disorder. 视谱障碍神经脊髓炎的治疗进展。
IF 4.7 2区 医学
Therapeutic Advances in Neurological Disorders Pub Date : 2025-04-12 eCollection Date: 2025-01-01 DOI: 10.1177/17562864251328276
Xiaolin Yang, Shaoru Zhang, Jinzhou Feng, Xinyue Qin
{"title":"Advances in the treatment of neuromyelitis optic spectrum disorder.","authors":"Xiaolin Yang, Shaoru Zhang, Jinzhou Feng, Xinyue Qin","doi":"10.1177/17562864251328276","DOIUrl":"https://doi.org/10.1177/17562864251328276","url":null,"abstract":"<p><p>Neuromyelitis optic spectrum disorder (NMOSD) is a rare autoimmune disease characterized by recurrent episodes and severe debilitation. It primarily involves the central nervous system and is associated with the presence of aquaporin-4 antibodies. Effective management of NMOSD necessitates long-term therapeutic strategies that focus on alleviating symptoms during acute episodes and preventing relapse. In recent years, the approval of emerging biologics targeting B cells, interleukin-6 receptors, and the complement pathway has marked a transformative development in NMOSD treatment. This article provides a comprehensive review of therapeutic advances in NMOSD, integrating the current literature to serve as a theoretical basis for clinical decision-making of NMOSD patients.</p>","PeriodicalId":22980,"journal":{"name":"Therapeutic Advances in Neurological Disorders","volume":"18 ","pages":"17562864251328276"},"PeriodicalIF":4.7,"publicationDate":"2025-04-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12033445/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144013793","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}
引用次数: 0
Secondary stroke prevention beyond antiplatelets: The role of colchicine and GLP-1RA - an ounce of prevention is worth a pound of cure. 抗血小板之外的二次中风预防:秋水仙碱和GLP-1RA的作用——一盎司的预防胜过一磅的治疗。
IF 4.7 2区 医学
Therapeutic Advances in Neurological Disorders Pub Date : 2025-04-12 eCollection Date: 2025-01-01 DOI: 10.1177/17562864251326769
Milija D Mijajlović, Natan M Bornstein, Vuk Aleksić
{"title":"Secondary stroke prevention beyond antiplatelets: The role of colchicine and GLP-1RA - an ounce of prevention is worth a pound of cure.","authors":"Milija D Mijajlović, Natan M Bornstein, Vuk Aleksić","doi":"10.1177/17562864251326769","DOIUrl":"https://doi.org/10.1177/17562864251326769","url":null,"abstract":"<p><p>Stroke remains a major global health concern, ranking as the second most common cause of death and the third leading cause of disability worldwide. Despite advances in therapy and management, ischemic stroke patients continue to face high risks of recurrence, cardiovascular events, and mortality. Effective secondary stroke prevention is critical, encompassing antithrombotic therapy, management of vascular risk factors such as hypertension, dyslipidemia, and diabetes mellitus, and conducting healthy lifestyle. Approximately 80% of strokes are ischemic, with a significant proportion attributable to large-artery atherosclerosis of the extra- and intracranial arteries, particularly in the internal carotid artery. Atherothrombotic strokes, linked to plaque rupture and thrombus formation, present a notably high risk of recurrence. Inflammatory and immune mechanisms play pivotal roles in both the initiation and progression of atherosclerosis and stroke. Colchicine, an anti-inflammatory agent, has shown potential in managing cardiovascular disease, though its effects on stroke reduction and prevention have been inconsistent across studies. Its possible protective role against stroke is attributed to its anti-inflammatory actions, which include disrupting microtubule dynamics, inhibiting immune cell movement, and lowering inflammatory markers like L-Selectin and E-Selectin, while also suppressing interleukin release. Glucagon-like peptide-1 receptor agonists (GLP-1RA) agents have emerged as effective therapies for type 2 diabetes with notable cardiovascular benefits. These agents enhance glucose control while also providing protective effects against atherosclerosis and stroke. GLP-1RA drugs work by mimicking the effects of GLP-1, a peptide that regulates insulin release and glucose metabolism. They also exhibit anti-inflammatory properties, potentially reducing stroke risk through mechanisms such as improved endothelial function and reduced plaque formation. Clinical trials have indicated that GLP-1RA agents can significantly lower the incidence of nonfatal strokes and major adverse events. This narrative review underscores the importance of targeting inflammation to reduce the risk of recurrent stroke, emphasizing recent studies on colchicine and GLP-1RA. It consolidates evidence regarding the efficacy of these agents in secondary stroke prevention; however, future studies are needed to further explore their mechanisms and roles in comprehensive stroke management strategies.</p>","PeriodicalId":22980,"journal":{"name":"Therapeutic Advances in Neurological Disorders","volume":"18 ","pages":"17562864251326769"},"PeriodicalIF":4.7,"publicationDate":"2025-04-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12033550/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143987609","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}
引用次数: 0
Walking reduces the risk of dementia in patients with Parkinson's disease: a longitudinal follow-up study. 行走可降低帕金森病患者患痴呆的风险:一项纵向随访研究。
IF 4.7 2区 医学
Therapeutic Advances in Neurological Disorders Pub Date : 2025-04-12 eCollection Date: 2025-01-01 DOI: 10.1177/17562864251330251
Cheng-Yu Wei, Ray-Chang Tzeng, Hsu-Chih Tai, Chun-Hsien Su, Pai-Yi Chiu
{"title":"Walking reduces the risk of dementia in patients with Parkinson's disease: a longitudinal follow-up study.","authors":"Cheng-Yu Wei, Ray-Chang Tzeng, Hsu-Chih Tai, Chun-Hsien Su, Pai-Yi Chiu","doi":"10.1177/17562864251330251","DOIUrl":"https://doi.org/10.1177/17562864251330251","url":null,"abstract":"<p><strong>Background: </strong>Physical activity, particularly regular aerobic exercise, is effective in preventing dementia. However, such activities are less feasible for patients with Parkinson's disease (PD) or other motor dysfunctions.</p><p><strong>Objectives: </strong>In this study, we investigated whether the minimal amount of exercise (MAE) through walking, which is practical for individuals with motor dysfunction, can reduce the risk of dementia in patients with PD.</p><p><strong>Design: </strong>For this retrospective longitudinal study, we enrolled 470 patients with PD without dementia from 3 centers in Taiwan.</p><p><strong>Methods: </strong>In total, 187 (39.8%) subsequently developed dementia, whereas 283 (60.2%) did not; the mean follow-up periods for these cohorts were 3.1 (range 0.3-6.1) and 2.4 (range 0.3-6.0) years, respectively. MAE was defined as walking approximately 1500-3000 steps or for 15-30 min. The patients were further stratified by the weekly frequency of MAE into MAE-no (frequency: 0), MAE-weekly (frequency: 1 or 2), and MAE-daily (frequency: ⩾3) groups, respectively. The incidence rates of dementia were compared among the three groups. Cox proportional-hazards analyses were performed to measure the effect of MAE on the incidence of dementia. The statistical model was adjusted for age, sex, education level, cognition level, activities of daily living, neuropsychiatric symptoms, vascular risk factors, and relevant medications.</p><p><strong>Results: </strong>The MAE-weekly and MAE-daily groups were 0.69 (95% confidence interval (CI): 0.41-1.17) and 0.59 (95% CI: 0.41-0.84) times, respectively, less likely to develop dementia than the MAE-no group. When the MAE-weekly and MAE-daily groups were combined, the hazard ratio for dementia was 0.62 (95% CI: 0.45-0.85). Cox regression revealed that older age, female sex, atrial fibrillation, antidiabetic drug use, and poor daily function were associated with an increased incidence of dementia.</p><p><strong>Conclusion: </strong>MAE may help prevent dementia in patients with PD. This finding highlights the benefits of walking for patients with PD and, potentially, older adults with motor dysfunction due to various disorders.</p>","PeriodicalId":22980,"journal":{"name":"Therapeutic Advances in Neurological Disorders","volume":"18 ","pages":"17562864251330251"},"PeriodicalIF":4.7,"publicationDate":"2025-04-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12033631/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144064713","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}
引用次数: 0
Atypical adverse events in a real-world study of long-term immunomodulation for multiple sclerosis and neuromyelitis optica spectrum disorder. 在多发性硬化症和视神经脊髓炎谱系障碍长期免疫调节的现实世界研究中的非典型不良事件。
IF 4.7 2区 医学
Therapeutic Advances in Neurological Disorders Pub Date : 2025-04-04 eCollection Date: 2025-01-01 DOI: 10.1177/17562864251320206
Amelie Kirschbaum, Felix Luessi, Arda Civelek, Stefan Bittner, Johannes Piepgras, Frauke Zipp
{"title":"Atypical adverse events in a real-world study of long-term immunomodulation for multiple sclerosis and neuromyelitis optica spectrum disorder.","authors":"Amelie Kirschbaum, Felix Luessi, Arda Civelek, Stefan Bittner, Johannes Piepgras, Frauke Zipp","doi":"10.1177/17562864251320206","DOIUrl":"https://doi.org/10.1177/17562864251320206","url":null,"abstract":"<p><strong>Background: </strong>Immunotherapies are integral in managing multiple sclerosis (MS) and related demyelinating diseases, but adverse drug reactions significantly affect the tolerability of disease-modifying therapies (DMTs).</p><p><strong>Objectives: </strong>This study aims to assess the safety profile of DMTs within a real-world cohort affected by MS and related diseases and to identify atypical adverse events (AEs) and those of exceptional severity.</p><p><strong>Methods: </strong>A retrospective analysis was conducted on 3850 patients with MS, neuromyelitis optica spectrum disorder (NMOSD), and related conditions (2009-2022). Demographic and clinical data were analyzed for patients treated with DMTs. Parameters included prior treatments, AEs, treatment durations, and reasons for discontinuation.</p><p><strong>Results: </strong>Of the cohort, 1989 patients (71.1% female) with a median follow-up of 46.3 months during DMT use were included. Monotherapy was employed in 987 patients, while 1002 received sequential DMTs, totaling 3850 treatments. Adverse reactions led to discontinuation in 24.2% of cases, while disease progression accounted for 22.9%. Among 1878 AEs, 31 (1.7%) were atypical, and 59 (3.1%) were unusually severe, which was systematically categorized based on type, timing, and remission.</p><p><strong>Conclusion: </strong>Within the confines of this real-world study, DMT administration emerged as generally well tolerated in MS, related demyelinating diseases and NMOSD. The identification of a limited number of atypical AEs, nevertheless, broadens the spectrum of potential complications associated with DMTs. Although weaker evidence for causal associations between drug exposure and observed AEs remains a limitation in observational studies without comparable control groups, this study underscores the value of real-world investigations in offering insights into the long-term safety of DMTs, particularly for rare events.</p>","PeriodicalId":22980,"journal":{"name":"Therapeutic Advances in Neurological Disorders","volume":"18 ","pages":"17562864251320206"},"PeriodicalIF":4.7,"publicationDate":"2025-04-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12032468/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144011624","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}
引用次数: 0
Integrated management of atypical parkinsonism: a home-based patient-centered healthcare delivery based on telenursing-the IMPACT study protocol. 非典型帕金森病的综合管理:基于远程护理的以患者为中心的家庭医疗服务--IMPACT 研究方案。
IF 4.7 2区 医学
Therapeutic Advances in Neurological Disorders Pub Date : 2025-04-03 eCollection Date: 2025-01-01 DOI: 10.1177/17562864241299347
Roberto Cilia, Fabiana Colucci, Antonio Suppa, Francesca Valentino, Carmen Terranova, Catia Leuzzi, Jessica Cordasco, Giulia Fusi, Simona Floridia, Francesca De Giorgi, Roberta Telese, Arianna Braccia, Alessandro Zampogna, Giulia Pinola, Martina Patera, Giorgio Belluscio, Sara Crivellari, Elisa Antoniazzi, Simona Cascino, Antonio Giaco, Alessio Masaracchio, Giacomina Clara Moreschi, Marisa Catotti, Roberto Eleopra
{"title":"Integrated management of atypical parkinsonism: a home-based patient-centered healthcare delivery based on telenursing-the IMPACT study protocol.","authors":"Roberto Cilia, Fabiana Colucci, Antonio Suppa, Francesca Valentino, Carmen Terranova, Catia Leuzzi, Jessica Cordasco, Giulia Fusi, Simona Floridia, Francesca De Giorgi, Roberta Telese, Arianna Braccia, Alessandro Zampogna, Giulia Pinola, Martina Patera, Giorgio Belluscio, Sara Crivellari, Elisa Antoniazzi, Simona Cascino, Antonio Giaco, Alessio Masaracchio, Giacomina Clara Moreschi, Marisa Catotti, Roberto Eleopra","doi":"10.1177/17562864241299347","DOIUrl":"10.1177/17562864241299347","url":null,"abstract":"<p><strong>Background: </strong>People with atypical parkinsonism, such as multiple system atrophy and progressive supranuclear palsy, experience a wide range of motor and non-motor symptoms associated with the increasing complexity of care delivery and the increased risk of complications and hospital admissions.</p><p><strong>Objectives: </strong>To investigate the efficacy and cost-effectiveness of a 12-month remote home-based integrated program aiming to improve healthcare delivery coordinated by a nurse specialized in the management of individuals with atypical parkinsonism (parkinsonism nurse specialist, PKNS) compared to the standard-of-care model.</p><p><strong>Design: </strong>Multicenter, randomized, single-blind, controlled clinical trial involving 164 individuals with atypical parkinsonism.</p><p><strong>Methods and analysis: </strong>Participants will be randomized 1:1 in intervention (PKNS) and control (standard-of-care) arms. Assessments will be undertaken at baseline and after 6 and 12 months. Primary outcome measure is the Parkinson's Disease Questionnaire 39-items scale total score. Secondary measures include the clinical scales testing motor and non-motor symptoms, caregiver burden, adherence to therapy, cumulative disease burden and the number of unplanned hospital visits/admissions during the study period. The cost-effectiveness of this method will be evaluated by using the EuroQoL-5, which estimates the incremental cost per quality-adjusted life-years gain. Real-life motor autonomy will be objectively measured by collecting waist-worn wearable data on gait parameters (automatically detecting motor patterns indicative of freezing of gait and falls) in all subjects for five consecutive days each month during the 12-month duration of the study.</p><p><strong>Ethic: </strong>Study protocol has been approved by the ethics committee of all participating centers. The study is conducted according to good clinical practice and the Declaration of Helsinki.</p><p><strong>Discussion: </strong>An integrated remote care model at home coordinated by a specialized nurse in the management of parkinsonism (Telenursing) could offer significant benefits to patients and healthcare professionals through better health education, continuity of care, and careful monitoring of complications.</p><p><strong>Trial registration: </strong>ClinicalTrials.gov identifier NCT05792332.</p>","PeriodicalId":22980,"journal":{"name":"Therapeutic Advances in Neurological Disorders","volume":"18 ","pages":"17562864241299347"},"PeriodicalIF":4.7,"publicationDate":"2025-04-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11970091/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143796175","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}
引用次数: 0
Anterior nucleus of thalamus deep brain stimulation for medication refractory epilepsy modulates theta and low-frequency gamma activity: a case study. 药物治疗难治性癫痫的丘脑前核深部脑刺激调节θ和低频γ活动:一个案例研究。
IF 4.7 2区 医学
Therapeutic Advances in Neurological Disorders Pub Date : 2025-03-24 eCollection Date: 2025-01-01 DOI: 10.1177/17562864251323052
Zachary T Sanger, Xinbing Zhang, Ilo E Leppik, Thomas Lisko, Theoden I Netoff, Robert A McGovern
{"title":"Anterior nucleus of thalamus deep brain stimulation for medication refractory epilepsy modulates theta and low-frequency gamma activity: a case study.","authors":"Zachary T Sanger, Xinbing Zhang, Ilo E Leppik, Thomas Lisko, Theoden I Netoff, Robert A McGovern","doi":"10.1177/17562864251323052","DOIUrl":"10.1177/17562864251323052","url":null,"abstract":"<p><p>A 35-year-old gentleman with a traumatic brain injury was diagnosed with refractory epilepsy with electroencephalogram and imaging findings supporting a broad seizure onset pattern in bilateral frontotemporal regions. He therefore received a Medtronic Percept PC Deep Brain Stimulator (DBS) placed bilaterally in the anterior nucleus of the thalamus (ANT). While most refractory epilepsy patients' stimulation parameters use the SANTE trial standard clinical settings of 145 Hz, 90 μs, with cycling 1-min stimulation on and 5 min stimulation off, this participant underwent 7 different stimulation parameter tests at home following testing in the clinic of 24 different stimulation parameters across 12 neurologist visits. This device allows for simultaneous stimulation of the ANT while recording the ANT local field potential (LFP) response under different stimulation parameters. Slepian multitaper analysis, modified Fitting Oscillations, and One Over F method for detrending the aperiodic component were performed to analyze neural oscillations in the frequency domain captured in the clinic. This participant was participating in a clinical study examining the effectiveness of nonstandard DBS settings to minimize broadband neural activity in the ANT. Statistically significant neuromodulatory suppression of gamma oscillations was observed in the clinic under multiple stimulation settings. We compared the ability of these research stimulation parameters to suppress at-home ANT neural activity against the standard clinical settings and examined the effects of both sets of parameters on LFP power nonstationarity. At home, theta/alpha LFP power suppression was statistically significantly reduced under the 125 Hz, 50 μs setting as opposed to the clinical setting of 145 Hz, 90 μs. The participant has achieved greater than 50% seizure reduction for over 1 year since the last neurology visit. Suppression of gamma in the clinic in the right hemisphere and suppression of theta at home in the left hemisphere show promise as quantitative feedback biomarkers for ANT-DBS. Understanding the local and network relationships of theta and slow gamma oscillations in the thalamus would further explain how these modulated oscillations may relate to the onset and propagation of seizures.</p>","PeriodicalId":22980,"journal":{"name":"Therapeutic Advances in Neurological Disorders","volume":"18 ","pages":"17562864251323052"},"PeriodicalIF":4.7,"publicationDate":"2025-03-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11938492/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143721503","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}
引用次数: 0
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