Therapeutic Advances in Neurological Disorders最新文献

{"title":"Shaping the role of plasma exchange in autoimmune neurology: lessons learned from 67 years and over 90,000 procedures.","authors":"Bhupendra O Khatri, Regina Berkovich, Jay S Raval, Mary Dukic, Lisa Sershon","doi":"10.1177/17562864251401510","DOIUrl":"10.1177/17562864251401510","url":null,"abstract":"<p><p>Therapeutic plasma exchange (PLEX) is a powerful and fast-acting immunomodulating therapy that is underutilized for autoimmune neurological disorders. Here, we present the largest collection of real-world experiences with PLEX procedures to date in the treatment of autoimmune neurological conditions, supporting its safety and clinical benefits with patient cases and corresponding patient videos. Our collective real-world experience with PLEX spans over 67 years, 90,210 procedures, and includes nine double-blind randomized controlled and unblinded studies serving as principal investigators. Case histories and videos of our patients demonstrate when and how PLEX should be used, identify barriers to using PLEX, and ways to overcome these barriers. Specific protocol details are shared of how to treat an acute or chronic phase of a disease. If used appropriately and early in the disease course for both acute and chronic progressive phases, PLEX can safely change the trajectory of many autoimmune neurological disorders in both outpatient and inpatient settings.</p>","PeriodicalId":22980,"journal":{"name":"Therapeutic Advances in Neurological Disorders","volume":"18 ","pages":"17562864251401510"},"PeriodicalIF":4.1,"publicationDate":"2025-12-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12744008/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145857788","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 of horizontal switching between disease-modifying therapies in multiple sclerosis: a retrospective analysis of the MSBase Registry.","authors":"Enrique Gomez-Figueroa, Patricia Orozco-Puga, Cynthia Patricia Corona-Vázquez, Carlos Moreno-Bernardino, Graciela Elizabeth De la Mora-Landín, Amado Jiménez-Ruiz, Christian García-Estrada, Lizeth Zertuche-Ortuño, Sergio Saldívar-Dávila, Roberto Rodríguez-Rivas, Lisette Bazán-Rodríguez, José Flores-Rivera, Tomas Kalincik, Katherine Buzzard, Samia Khoury, Pierre Duquette, Matteo Foschi, Andrea Surcinelli, Bianca Weinstock-Guttman, Riadh Gouider, Saloua Mrabet, Jeannette Lechner-Scott, Helmut Butzkueven, Raed Alroughani, Izanne Roos, Francesco Patti, Bassem Yamout, François Grand'Maison, Daniele Spitaleri, Pamela McCombe, José Luis Sanchez-Menoyo, Serkan Ozakbas, Abdullah Al-Asmi, Nevin John, Elisabetta Cartechini, Anneke Van der Walt, Justin Garber, Emmanuelle Lapointe, Aysun Soysal, Eduardo Aguera-Morales, Joana Guimarães, José Luis Ruiz-Sandoval","doi":"10.1177/17562864251399595","DOIUrl":"10.1177/17562864251399595","url":null,"abstract":"<p><strong>Background: </strong>Switching disease-modifying therapies (DMTs) is common in relapsing-remitting multiple sclerosis (RRMS). Vertical switching to higher-efficacy agents generally outperforms horizontal switching within the same efficacy tier, yet horizontal switches remain frequent where escalation is impractical.</p><p><strong>Objectives: </strong>To compare real-world outcomes after horizontal versus vertical DMT switches and to identify predictors of successful horizontal switching.</p><p><strong>Design: </strong>Retrospective, registry-based observational study.</p><p><strong>Methods: </strong>Adults with RRMS who switched DMTs in the MSBase Registry (2010-2023) were analyzed. Horizontal switches were defined as transitions within efficacy tiers, and vertical switches as transitions to a higher tier. Propensity score matching (1:1) generated balanced cohorts. Multivariable mixed-effects models with a random intercept for patients were used to evaluate associations with outcomes. The primary outcome was no evidence of disease activity (NEDA-3) during the treatment period; secondary outcomes included annualized relapse rate (ARR), Expanded Disability Status Scale (EDSS) change, confirmed disability worsening (CDW), confirmed disability improvement (CDI), and progression independent of relapse activity (PIRA). Predictors of successful horizontal switching were explored using logistic regression.</p><p><strong>Results: </strong>A total of 4934 matched switches (2467 pairs) were analyzed. Vertical switching achieved higher NEDA-3 rates than horizontal switching (45.8% vs 33.7%) and was associated with lower ARR, reduced CDW risk, and more frequent CDI; differences in EDSS progression and PIRA were not significant. Among horizontal switchers, 33.7% achieved NEDA-3. Success was associated with lower baseline EDSS, fewer prior relapses, and later-line switching. Outcomes varied by destination therapy: anti-CD20 agents had the highest success (≈50%), followed by cladribine (≈43%) and natalizumab (≈41%), whereas interferon and glatiramer acetate performed the poorest. Switches toward anti-CD20 therapies generally yielded better outcomes than other within-tier changes.</p><p><strong>Conclusion: </strong>Vertical switching should be preferred when treatment modification is required, particularly for patients with active disease. However, a subset of patients can achieve disease stability after horizontal switching, especially those with lower disability and fewer prior relapses. The dynamics of horizontal switching may further influence outcomes, warranting prospective validation.</p>","PeriodicalId":22980,"journal":{"name":"Therapeutic Advances in Neurological Disorders","volume":"18 ","pages":"17562864251399595"},"PeriodicalIF":4.1,"publicationDate":"2025-12-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12744030/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145857768","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":"Advancements and challenges of adherence to secondary prevention medications among patients with ischemic stroke or transient ischemic attack: the healthcare quality improvement in China, 2007-2018.","authors":"Yuan Shen, Xinya Li, Xue Xia, Meng Gao, Xue Tian, Qin Xu, Xiaoli Zhang, Ruobing Tian, Xia Meng, Anxin Wang","doi":"10.1177/17562864251406061","DOIUrl":"10.1177/17562864251406061","url":null,"abstract":"<p><strong>Background: </strong>Despite significant achievements in healthcare quality improvement for ischemic stroke and transient ischemic attack (TIA), the advancements and challenges of patient adherence to secondary prevention medications remain unclear.</p><p><strong>Objectives: </strong>We aimed to investigate adherence rates of secondary prevention medication and identified the key determinants in Chinese patients with ischemic stroke or TIA.</p><p><strong>Design: </strong>This is an observational study.</p><p><strong>Methods: </strong>Using the China National Stroke Registry (CNSR) database from 2007 to 2018, this observational study included patients with ischemic stroke or TIA who were admitted to the hospital within 7 days of symptom onset. The study outcome was the patient adherence to secondary prevention medications, which was defined as the consistent use of prescribed antithrombotic, lipid-modulating, antidiabetic, and antihypertensive medications post-discharge using an \"all-or-none\" approach. We calculated adherence rates in 3 and 12 months. Logistic regression models were used to evaluate influencing factor patterns and challenges in the improvement of patient adherence.</p><p><strong>Results: </strong>A total of 12,873 patients from CNSR I and 15,099 patients from CNSR III were included. Patient adherence rates for secondary prevention medications increased from 66.97% in CNSR I to 80.76% in CNSR III (<i>p</i> < 0.0001) in 3 months, and from 35.08% to 59.81% (<i>p</i> < 0.0001) in 12 months. Patient age, the National Institute of Health stroke scale score at admission, disease diagnosis, the Trial of Org 10172 in Acute Stroke Treatment classification, family income per capita, alcohol consumption, dyslipidemia history, hypertension history, diabetes history, and heart disease history appeared to exhibit a significant association with adherence.</p><p><strong>Conclusion: </strong>In spite of the remarkable progress in patient adherence to secondary prevention of stroke from 2007 to 2018, challenges remain in sustaining quality improvement initiatives, necessitating further improvements by addressing disease severity, lifestyle, medical history, and socioeconomic factors.</p>","PeriodicalId":22980,"journal":{"name":"Therapeutic Advances in Neurological Disorders","volume":"18 ","pages":"17562864251406061"},"PeriodicalIF":4.1,"publicationDate":"2025-12-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12743152/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145850923","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":"Effectiveness and tolerability of lasmiditan in the acute treatment of migraine: a real-world, prospective, multicentric study (DART study).","authors":"Gloria Vaghi, Luigi Francesco Iannone, Michele Corrado, Federico De Santis, Marina Romozzi, Gabriele Sebastianelli, Giorgio Dalla Volta, Marco Bolchini, Andrea Burgalassi, Francesco De Cesaris, Maria Albanese, Nicola Mercuri Biagio, Raffaele Ornello, Simona Sacco, Francesca Pistoia, Gennaro Saporito, Francesco Casillo, Gianluca Avino, Antonio Granato, Antonio Russo, Marcello Silvestro, Catello Vollono, Michele Trimboli, Alberto Doretti, Mariarosaria Valente, Sabina Cevoli, Edoardo Mampreso, Cristina Tassorelli, Roberto De Icco","doi":"10.1177/17562864251381886","DOIUrl":"10.1177/17562864251381886","url":null,"abstract":"<p><strong>Background: </strong>Lasmiditan, an oral 5-HT_1F receptor agonist, has been recently approved for acute migraine treatment. While its efficacy was confirmed in randomized clinical trials, scarce data is available regarding effectiveness and tolerability in the real-world setting.</p><p><strong>Objectives: </strong>To evaluate lasmiditan effectiveness and tolerability in the real-world setting in 16 Italian headache centers.</p><p><strong>Design: </strong>LasmiDitan as Acute migRaine Treatment (DART) study is a prospective, multicentric, observational study.</p><p><strong>Methods: </strong>We enrolled 58 participants with migraine (84.5% females, age 49.0 (45.2-52.9) years, 24.1% with chronic migraine) reporting 9.4 (7.4-11.3) monthly migraine days. Participants were instructed to treat their migraine attacks with oral lasmiditan 50 or 100 mg. Using an ad hoc electronic diary, participants prospectively collected migraine attack features at baseline and every 30 min after lasmiditan administration, up to 2 h post-dose. The primary outcome was 2-h pain freedom for the first-treated attack after lasmiditan intake. We also collected the occurrence of treatment-emergent adverse events (AE) after administration.</p><p><strong>Results: </strong>Overall, participants treated 100 attacks, of which 58 first-treated attacks. Regarding first-treated attacks, 44.8% of subjects rated migraine intensity as severe at lasmiditan intake. Pain freedom 2-h post-dosing was reported in 32.8% (19/58) of individuals and was associated with baseline pain intensity, being higher in subjects treating a mild/moderate attack (<i>p</i> = 0.044). Conversely, it was not influenced by timing of intake (<i>p</i> = 0.375), dosage (<i>p</i> = 0.727), or previous triptan failure (<i>p</i> = 0.351). Regarding all-treated attacks, pain freedom 2-h post-dosing was 37.0% (37/100). At least one AE was reported by 53.4% of participants (31/58), predominantly asthenia, dizziness, somnolence, anxiety or agitation, and paresthesia. Tolerability was rated as good-to-excellent by 51.8% of subjects.</p><p><strong>Conclusion: </strong>Our study supports clinical effectiveness of oral lasmiditan 50 and 100 mg for the treatment of acute migraine attacks. Lasmiditan effectiveness was not associated with the previous triptan failure and may therefore represent a valuable therapeutic option in subjects who did not benefit from, or have contraindications to, triptans.</p><p><strong>Trail registration: </strong>The study was preregistered on clinicaltrial.gov, NCT05903040 (https://clinicaltrials.gov/study/NCT05903040?cond=migraine&intr=lasmiditan&rank=5).</p>","PeriodicalId":22980,"journal":{"name":"Therapeutic Advances in Neurological Disorders","volume":"18 ","pages":"17562864251381886"},"PeriodicalIF":4.1,"publicationDate":"2025-12-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12722647/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145828459","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 of intravenous immunoglobulin in patients with chronic inflammatory demyelinating polyneuropathy with or without diabetes: insights from a multicenter prospective comparative study.","authors":"Samir P Macwan, Shalini Mahajan, Peter Novak, Khosro Farhad, Ericka Wong, Thomas H Brannagan, Sohail Masood, Fawad Piracha, Marinos C Dalakas","doi":"10.1177/17562864251401496","DOIUrl":"10.1177/17562864251401496","url":null,"abstract":"<p><strong>Background: </strong>Chronic inflammatory demyelinating polyradiculoneuropathy (CIDP) is the most frequently observed autoimmune neuropathy in patients with diabetes mellitus (DM). While intravenous immune globulin (IVIG) is a well-established treatment for CIDP, its efficacy in diabetic patients remains uncertain due to their exclusion from prior randomized trials, largely because of concerns about confounding diabetic axonal neuropathy.</p><p><strong>Objectives: </strong>To evaluate the effectiveness of IVIG therapy in CIDP patients with diabetes mellitus (CIDP-DM) compared to those without diabetes (CIDP).</p><p><strong>Design: </strong>Multi-center, prospective, observational study after at least 3 monthly infusions of IVIG therapy.</p><p><strong>Methods: </strong>Thirty-six patients meeting diagnostic criteria for CIDP were enrolled and stratified into CIDP or CIDP-DM. All patients were followed for a minimum of 3 months after initiating IVIG therapy. Clinical outcomes were assessed at baseline (visit #1) and after 3 monthly IVIG infusions (visit #4) using the adjusted Inflammatory Neuropathy Cause and Treatment Disability Score, the Rasch-built Overall Disability Scale, and the Chronic Acquired Polyneuropathy Patient-reported Index, measured at baseline and at the point of maximal improvement.</p><p><strong>Results: </strong>No significant differences were observed in clinical outcomes, treatment-related adverse events, or tolerance between CIDP and CIDP-DM groups, indicating comparable effectiveness of IVIG therapy. However, subgroup analyses revealed that longer duration of diabetes and elevated HbA1c levels were associated with delayed response to IVIG, likely due to cumulative axonal degeneration.</p><p><strong>Conclusion: </strong>Despite the small number of enrolled patients, IVIG appears equally effective in CIDP patients with and without diabetes. Earlier initiation of IVIG treatment should be considered in CIDP-DM patients to mitigate potential delays in therapeutic response associated with a possibly chronic diabetic neuropathy-related component.</p>","PeriodicalId":22980,"journal":{"name":"Therapeutic Advances in Neurological Disorders","volume":"18 ","pages":"17562864251401496"},"PeriodicalIF":4.1,"publicationDate":"2025-12-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12722652/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145828381","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":"Regional perfusion parameters as potential indicators of parenchymal hematoma risk following reperfusion therapy for acute ischemic stroke in the extended time window.","authors":"Xinyu Liu, Lan Hong, Guangjian Zhao, Zhijiao He, Xinru Wang, Juehua Zhu, Siyuan Li, Anqi Zhang, Nan Cao, Yifeng Ling, Xiangdi Chen, Ying Guo, Qi Fang, Ziran Wang, Qiang Dong, Xin Cheng","doi":"10.1177/17562864251406032","DOIUrl":"10.1177/17562864251406032","url":null,"abstract":"<p><strong>Background: </strong>Parenchymal hematoma (PH) is a common complication of acute ischemic stroke, particularly following reperfusion therapy.</p><p><strong>Objective: </strong>This study aimed to explore the relationship between regional perfusion parameters and PH outcomes in stroke patients treated beyond the conventional time window.</p><p><strong>Design: </strong>This retrospective cohort study included patients from the CHinese Acute tissue-Based imaging selection for Lysis In Stroke-Tenecteplase (CHABLIS-T) trials and the Huashan Hospital stroke registry.</p><p><strong>Methods: </strong>Regional perfusion parameters were calculated within Alberta Stroke Program Early CT Score (ASPECTS)-defined regions of interest (ROIs). Mirror indices of cerebral blood flow (CBFmi), cerebral blood volume (CBVmi), and mean transit time were derived as the ratios of median perfusion values within ASPECTS-ROIs in the lesion and its contralateral hemisphere. Absolute time to maximum values for symptomatic ASPECTS-ROIs were also recorded. Logistic regression evaluated associations between perfusion parameters and PH outcomes, with predictive performance assessed using receiver operating characteristic (ROC) curves and area under the curve (AUC). Sensitivity analysis was conducted in patients receiving endovascular treatment (EVT) and in the trial-only population.</p><p><strong>Results: </strong>Of 1010 patients screened, 313 met the inclusion criteria, and 54 developed PH. Multivariable stepwise logistic regression identified reduced CBFmi (adjusted odds ratios (aOR) = 0.07, 95% confidence interval (CI), 0.02-0.30, <i>p</i> < 0.001) and CBVmi (aOR = 0.11, 95% CI, 0.03-0.45, <i>p</i> = 0.002) in the lentiform nucleus as significant predictors of PH. ROC analysis showed good discriminative performance (AUC: CBFmi 0.71 (95% CI, 0.62-0.80), CBVmi 0.70 (95% CI, 0.61-0.79)). Sensitivity analysis in patients undergoing EVT and trial-only patients drew similar results.</p><p><strong>Conclusion: </strong>Decreased CBFmi and CBVmi in the lentiform nucleus were independently associated with an elevated risk of PH, highlighting their potential utility in predicting hemorrhagic complications.</p><p><strong>Trial registration: </strong>NCT04086147, NCT04516993.</p>","PeriodicalId":22980,"journal":{"name":"Therapeutic Advances in Neurological Disorders","volume":"18 ","pages":"17562864251406032"},"PeriodicalIF":4.1,"publicationDate":"2025-12-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12722669/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145828411","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":"Safety and efficacy of oral anticoagulation in patients with intracranial hemorrhage and atrial fibrillation: a systematic review and meta-analysis of randomized controlled clinical trials.","authors":"Nikolaos M Papageorgiou, Lina Palaiodimou, Aikaterini Theodorou, Eleni Bakola, Maria Chondrogianni, Georgia Papagiannopoulou, Apostolos Safouris, Eleni Anagnou, Panagiota-Eleni Tsalouchidou, Effrosyni Koutsouraki, Theodore Karapanayiotides, Efstathios Boviatsis, Christos Krogias, Sotirios Giannopoulos, Diana Aguiar de Sousa, Mira Katan, Thorsten Steiner, Georgios Tsivgoulis","doi":"10.1177/17562864251406065","DOIUrl":"10.1177/17562864251406065","url":null,"abstract":"<p><strong>Background: </strong>Patients with atrial fibrillation (AF) who survive spontaneous intracerebral hemorrhage (ICH) face competing risks of thromboembolism and recurrent bleeding.</p><p><strong>Objectives: </strong>To evaluate the safety and efficacy of initiating oral anticoagulants versus avoiding anticoagulation in adults with AF after spontaneous ICH.</p><p><strong>Design: </strong>Systematic review and meta-analysis of randomized-controlled clinical trials (RCTs).</p><p><strong>Data sources and methods: </strong>We searched MEDLINE, Scopus, and ClinicalTrials.gov up to August 28, 2025, for eligible RCTs randomizing adults with AF and prior spontaneous ICH to start oral anticoagulation versus avoid anticoagulation. Efficacy outcomes included the occurrence of new ischemic stroke (primary) and ischemic major adverse cardiovascular events (MACE; secondary). Safety outcomes included recurrent ICH (primary), hemorrhagic-MACE, all-cause mortality at follow-up, and cardiovascular death (secondary). Risk ratios (RRs) with 95% confidence intervals (CIs) were pooled using random-effects meta-analysis.</p><p><strong>Results: </strong>Six RCTs were included, comprising 403 patients in the anticoagulation group and 395 in the avoid-anticoagulation group. Anticoagulants reduced the rates of new ischemic stroke (RR = 0.20; 95% CI: 0.06-0.72; <i>I</i> ² = 60%; number needed to treat = 9) and ischemic-MACE (RR = 0.41; 95% CI: 0.23-0.75; <i>I</i> ² = 32%). Anticoagulants were associated with higher rates of recurrent ICH (RR = 3.14; 95% CI: 1.41-7.01; <i>I</i> ² = 0%; number needed to harm = 19) and hemorrhagic-MACE (RR = 2.35; 95% CI: 1.32-4.21; <i>I</i> ² = 1%). All-cause mortality at 90 days (RR = 1.06; 95% CI: 0.69-1.64; <i>I</i> ² = 28%) and cardiovascular death (RR = 0.98; 95% CI: 0.34-2.87; <i>I</i> ² = 63%) did not differ between the two groups. Leave-one-out sensitivity analyses supported the overall direction of effects, with some attenuation when individual trials were omitted.</p><p><strong>Conclusion: </strong>In AF survivors of spontaneous ICH, restarting oral anticoagulation lowers ischemic events but raises risks of recurrent ICH and major bleeding, without a clear early mortality difference. Potential benefits may outweigh risks in selected patients within a multidisciplinary framework. Adequately powered RCTs are needed to refine agent choice, timing, and patient selection.</p><p><strong>Trial registration: </strong>PROSPERO CRD420251135299 (registered August 27, 2025).</p>","PeriodicalId":22980,"journal":{"name":"Therapeutic Advances in Neurological Disorders","volume":"18 ","pages":"17562864251406065"},"PeriodicalIF":4.1,"publicationDate":"2025-12-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12722659/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145828428","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":"Long-term globus pallidus internus deep brain stimulation in a young patient with spinocerebellar ataxia type 3 initially presenting with levodopa-responsive parkinsonism: a 6-year follow-up case report and literature review.","authors":"Jing Zhao, Shaochen Ma, Yunlei Gao, Jia Chen, Jiaqi Chen, Chong Shi, Peifu Wang, Jilai Li, Jichen Du, Zhirong Wan","doi":"10.1177/17562864251396525","DOIUrl":"10.1177/17562864251396525","url":null,"abstract":"<p><p>Spinocerebellar ataxia type 3 (SCA3) is an inherited neurodegenerative disorder. Some of its clinical features resemble those of primary Parkinson's disease (PD), which can easily lead to misdiagnosis. There is currently no disease-modifying therapy available for SCA3, treatment is mainly symptomatic. Herein, we report a case of a young female patient with SCA3 who presented with Parkinsonian as the main manifestation and underwent globus pallidus internus (GPi) deep brain stimulation (DBS). This is a 36-year-old female patient. Her first symptoms occurred at the age of 28 in 2009, manifesting as gait abnormalities in the right lower limb. She was misdiagnosed with early-onset PD in 2011. Genetic testing showed abnormal numbers of CAG repeats (15/70) within the coding region of the ATXN3 genes. She was diagnosed with SCA3. The patient initially responded well to levodopa-based medication, but the treatment effects gradually attenuated over time, with the development of severe symptom fluctuations and dyskinesia in 2018. The patient underwent GPi-DBS surgery in the absence of cerebellar signs, cognitive, and mood disorders. Six-year postoperative follow-up results suggest that long-term GPi-DBS is effective for the control of dyskinesia, but the residual motor symptoms (parkinsonism and ataxia) had progressively worsened in the patient. Various targets have been reported to be selected for DBS treatment of SCA3, with substantial individual differences in treatment outcomes. This case emphasizes the importance of genetic testing for the diagnosis of SCA3 and provides a basis for personalized treatment of patients with SCA3.</p>","PeriodicalId":22980,"journal":{"name":"Therapeutic Advances in Neurological Disorders","volume":"18 ","pages":"17562864251396525"},"PeriodicalIF":4.1,"publicationDate":"2025-12-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12712318/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145805493","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":"Appropriate use of steroids for patients with generalized Myasthenia Gravis: an international Delphi study.","authors":"Heinz Wiendl, Hiroyuki Murai, Pushpa Narayanaswami, Francesco Saccà, Nicholas J Silvestri, James F Howard","doi":"10.1177/17562864251398375","DOIUrl":"10.1177/17562864251398375","url":null,"abstract":"<p><strong>Background: </strong>Oral corticosteroids (OCS) are commonly used in the management of autoimmune Myasthenia Gravis (MG), often in high doses for prolonged periods. Exposure to OCS is associated with significant and cumulative adverse effects. There is currently no universal consensus on the approach to OCS use in MG.</p><p><strong>Objectives: </strong>To reach a multinational consensus on the appropriate use of OCS in MG, including the role of treatment approaches to minimize dose and support tapering.</p><p><strong>Design: </strong>This modified Delphi study established consensus among a panel of 70 general neurologists/neuromuscular specialists from eight countries over two rounds of survey to establish best practice principles regarding the use of OCS in MG.</p><p><strong>Methods: </strong>The current literature on OCS use in MG was reviewed. Topics of interest were identified, and a modified Delphi consensus process was created. A steering committee of six experts in MG proposed statements for testing with the wider panel over two rounds of surveys. Consensus was reached if at least 75% agreed or strongly agreed on a 4-point Likert scale.</p><p><strong>Results: </strong>Consensus was achieved for all 37 final statements. These statements covered principles of OCS use, including a target for long-term use of ⩽5 mg/day; the role of biologic treatments in minimizing required OCS dose; the need for individualization of approach according to patient factors; and the need for detailed guidance regarding how and when to taper OCS dose.</p><p><strong>Conclusion: </strong>These findings reinforce a shift in the management of MG, advocating for the judicious and sparing use of OCS against the backdrop of expanding therapeutic options. As additional evidence-based data emerge, these recommendations should be updated.</p>","PeriodicalId":22980,"journal":{"name":"Therapeutic Advances in Neurological Disorders","volume":"18 ","pages":"17562864251398375"},"PeriodicalIF":4.1,"publicationDate":"2025-12-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12709019/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145782860","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":"Alterations of fecal short-chain fatty acids solely in the course of multiple sclerosis: rethinking the gut-brain axis in the early stages of MS.","authors":"Jakob Stögbauer, Niklas Kämpfer, Anouck Becker-Dorison, Andreas Schwiertz, Sergiu Groppa, Marcus M Unger, Mathias Fousse","doi":"10.1177/17562864251396028","DOIUrl":"10.1177/17562864251396028","url":null,"abstract":"<p><strong>Background: </strong>The role of gut microbiota in multiple sclerosis (MS) has become increasingly important, intestinal dysbiosis with reduced production of short-chain fatty acids (SCFA) being the prevailing paradigm. However, the direction of causality, that is, whether intestinal changes are cause or consequence of chronic central nervous system inflammation, remains to be elucidated. Previous studies have focused on long-term MS patients. Alteration in fecal SCFA concentrations in early MS, particularly during relapses, remains to be extensively studied.</p><p><strong>Objectives: </strong>To compare fecal SCFA concentrations in patients with a first diagnosis of MS with those in patients with long-term MS and in healthy controls (HCs).</p><p><strong>Design: </strong>Prospective cohort study.</p><p><strong>Methods: </strong>The prospective case-control study was conducted on relapsing-remitting MS (RRMS) patients at the time of first, acute relapse without ongoing immunotherapy (Early-RRMS). Clinical and demographic parameters, as well as fecal SCFA concentrations (measured by gas chromatography) were collected. The parameters were compared with those of matched RRMS patients under different, long-term immunotherapy (Late-RRMS) and HCs.</p><p><strong>Results: </strong>SCFA concentrations of propionate, butyrate, isobutyrate, valerate, and isovalerate were not significantly different between the early-RRMS cohort and HCs, but were lower in the late-RRMS cohort.</p><p><strong>Conclusion: </strong>The findings indicate that reduction in SCFA levels is exclusively observed in patients with RRMS during the further course of the disease and not at the onset. Decrease in SCFA concentration may be rather consequence or related to neurodegeneration than linked to the first demyelinating event. Further investigation related to disease trajectories of immunomodulatory or neuroprotective treatments are required.</p>","PeriodicalId":22980,"journal":{"name":"Therapeutic Advances in Neurological Disorders","volume":"18 ","pages":"17562864251396028"},"PeriodicalIF":4.1,"publicationDate":"2025-12-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12701217/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145757615","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}