Therapeutic Advances in Neurological Disorders最新文献

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Serum neurofilament light chain correlations in patients with a first clinical demyelinating event in the REFLEX study: a post hoc analysis. REFLEX研究中首次临床脱髓鞘事件患者血清神经丝蛋白轻链的相关性:事后分析。
IF 5.9 2区 医学
Therapeutic Advances in Neurological Disorders Pub Date : 2024-03-29 eCollection Date: 2024-01-01 DOI: 10.1177/17562864241239101
Jens Kuhle, David Leppert, Giancarlo Comi, Nicola de Stefano, Ludwig Kappos, Mark S Freedman, Andrea Seitzinger, Sanjeev Roy
{"title":"Serum neurofilament light chain correlations in patients with a first clinical demyelinating event in the REFLEX study: a <i>post hoc</i> analysis.","authors":"Jens Kuhle, David Leppert, Giancarlo Comi, Nicola de Stefano, Ludwig Kappos, Mark S Freedman, Andrea Seitzinger, Sanjeev Roy","doi":"10.1177/17562864241239101","DOIUrl":"10.1177/17562864241239101","url":null,"abstract":"<p><strong>Background: </strong>In REFLEX, subcutaneous interferon beta-1a (sc IFN β-1a) delayed the onset of multiple sclerosis (MS) in patients with a first clinical demyelinating event (FCDE).</p><p><strong>Objectives: </strong>This <i>post hoc</i> analysis aimed to determine whether baseline serum neurofilament light (sNfL) chain can predict conversion to MS and whether correlations exist between baseline sNfL and magnetic resonance imaging (MRI) metrics.</p><p><strong>Methods: </strong>sNfL was measured for 494 patients who received sc IFN β-1a 44 μg once weekly (qw; <i>n</i> = 168), three times weekly (tiw; <i>n</i> = 161), or placebo (<i>n</i> = 165) over 24 months. Median baseline sNfL (26.1 pg/mL) was used to define high/low sNfL subgroups. Hazard ratios (HRs) and 95% confidence intervals (CIs) were calculated using Cox's proportional hazard model to determine factors influencing the risk of conversion to MS. Kaplan-Meier estimates calculated median time-to-conversion to MS (McDonald 2005 criteria) or clinically definite MS (CDMS; Poser criteria). Correlations between sNfL and MRI findings were assessed using Spearman's rank correlation coefficient (<i>r</i>).</p><p><strong>Results: </strong>Multivariable models indicated that high baseline sNfL was associated with the likelihood of converting to MS and inversely to time-to-conversion (HR = 1.3, 95% CI: 1.03-1.64; <i>p</i> = 0.024). Significant additional factors affecting conversion to McDonald MS were on-study treatment (sc IFN β-1a/placebo; qw: HR = 0.59, 95% CI: 0.46-0.76; tiw: HR = 0.45, 95% CI: 0.34-0.59), classification of FCDE (monofocal/multifocal; HR = 0.69, 95% CI: 0.55-0.85), and most baseline imaging findings (T2 and T1 gadolinium-enhancing [Gd+] lesions; HR = 1.02, 95% CI: 1.01-1.03 and HR = 1.07, 95% CI: 1.03-1.11); all <i>p</i> ⩽ 0.001. Conversion to CDMS showed similar results. At month 24, sNfL was strongly correlated with a mean number of combined unique active (<i>r</i> = 0.71), new T2 (<i>r</i> = 0.72), and new T1 Gd+ (<i>r</i> = 0.60) lesions; weak correlations were observed between sNfL and clinical outcomes for all treatment groups.</p><p><strong>Conclusion: </strong>Higher baseline sNfL was associated with an increased risk of MS conversion, a risk that was mitigated by treatment with sc IFN β-1a tiw.</p><p><strong>Trial registration: </strong>ClinicalTrials.gov identifier: NCT00404352. Date registered: 28 November 2006.</p>","PeriodicalId":22980,"journal":{"name":"Therapeutic Advances in Neurological Disorders","volume":"17 ","pages":"17562864241239101"},"PeriodicalIF":5.9,"publicationDate":"2024-03-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10981258/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140336946","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
Sex differences in multiple sclerosis relapse presentation and outcome: a retrospective, monocentric study of 134 relapse events. 多发性硬化症复发表现和预后的性别差异:对 134 例复发事件的回顾性单中心研究。
IF 5.9 2区 医学
Therapeutic Advances in Neurological Disorders Pub Date : 2024-03-26 eCollection Date: 2024-01-01 DOI: 10.1177/17562864241237853
Pauline Thränhardt, Admirim Veselaj, Christoph Friedli, Franca Wagner, Stefanie Marti, Lara Diem, Helly Hammer, Piotr Radojewski, Roland Wiest, Andrew Chan, Robert Hoepner, Anke Salmen
{"title":"Sex differences in multiple sclerosis relapse presentation and outcome: a retrospective, monocentric study of 134 relapse events.","authors":"Pauline Thränhardt, Admirim Veselaj, Christoph Friedli, Franca Wagner, Stefanie Marti, Lara Diem, Helly Hammer, Piotr Radojewski, Roland Wiest, Andrew Chan, Robert Hoepner, Anke Salmen","doi":"10.1177/17562864241237853","DOIUrl":"10.1177/17562864241237853","url":null,"abstract":"<p><strong>Background: </strong>Reporting of sex-specific analyses in multiple sclerosis (MS) is sparse. Disability accrual results from relapses (relapse-associated worsening) and independent thereof (progression independent of relapses).</p><p><strong>Objectives: </strong>A population of MS patients during relapse treated per standard of care was analyzed for sex differences and short-term relapse outcome (3-6 months) as measured by Expanded Disability Status Scale (EDSS) change.</p><p><strong>Design: </strong>Single-center retrospective study.</p><p><strong>Methods: </strong>We analyzed 134 MS relapses between March 2016 and August 2020. All events required relapse treatment (steroids and/or plasma exchange). Demographic, disease, and paraclinical characteristics [cerebrospinal fluid (CSF) and magnetic resonance imaging (MRI)] were displayed separated by sex. Multivariable linear regression was run to identify factors associated with short-term EDSS change.</p><p><strong>Results: </strong>Mean age at relapse was 38.4 years (95% confidence interval: 36.3-40.4) with a proportion of 71.6% women in our cohort. Smoking was more than twice as prevalent in men (65.8%) than women (32.3%). In- and after-relapse EDSSs were higher in men [men: 3.3 (2.8-3.9), women: 2.7 (2.4-3.0); men: 3.0 (1.3-3.6); women: 1.8 (1.5-2.1)] despite similar relapse intervention. Paraclinical parameters revealed no sex differences. Our primary model identified female sex, younger age, and higher EDSS at relapse to be associated with EDSS improvement. A higher immunoglobulin G (IgG) quotient (CSF/serum) was associated with poorer short-term outcome [mean days between first relapse treatment and last EDSS assessment 130.2 (79.3-181.0)].</p><p><strong>Conclusion: </strong>Sex and gender differences are important in outcome analyses of MS relapses. Effective treatment regimens need to respect putative markers for a worse outcome to modify long-term prognosis such as clinical and demographic variables, complemented by intrathecal IgG synthesis. Prospective trials should be designed to address these differences and confirm our results.</p>","PeriodicalId":22980,"journal":{"name":"Therapeutic Advances in Neurological Disorders","volume":"17 ","pages":"17562864241237853"},"PeriodicalIF":5.9,"publicationDate":"2024-03-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10964455/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140294638","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
Improved visualization of median, ulnar nerves, and small branches in the wrist and palm using contrast-enhanced magnetic resonance neurography. 利用造影剂增强磁共振神经显像技术提高腕部和掌部正中神经、尺神经以及小分支的可视化。
IF 5.9 2区 医学
Therapeutic Advances in Neurological Disorders Pub Date : 2024-03-26 eCollection Date: 2024-01-01 DOI: 10.1177/17562864241239739
Jiamin Kang, Wenjun Wu, Xiangchuang Kong, Yu Su, Dingxi Liu, Chungao Li, Nan Gao, Youzhi Wang, Chuansheng Zheng, Yuxiong Weng, Lixia Wang
{"title":"Improved visualization of median, ulnar nerves, and small branches in the wrist and palm using contrast-enhanced magnetic resonance neurography.","authors":"Jiamin Kang, Wenjun Wu, Xiangchuang Kong, Yu Su, Dingxi Liu, Chungao Li, Nan Gao, Youzhi Wang, Chuansheng Zheng, Yuxiong Weng, Lixia Wang","doi":"10.1177/17562864241239739","DOIUrl":"10.1177/17562864241239739","url":null,"abstract":"<p><strong>Background: </strong>Magnetic resonance imaging of peripheral nerves in the wrist and palm is challenging due to the small size, tortuous course, complex surrounding tissues, and accompanying blood vessels. The occurrence of carpal palmar lesions leads to edema, swelling, and mass effect, which may further interfere with the display and identification of nerves.</p><p><strong>Objective: </strong>To evaluate whether contrast-enhanced magnetic resonance neurography (ceMRN) improves the visualization of the morphology and pathology of the median, ulnar nerves, and their small branches in the wrist and palm.</p><p><strong>Design: </strong>An observational study.</p><p><strong>Methods: </strong>In total 57 subjects, including 36 volunteers and 21 patients with carpal palmar lesions, were enrolled and underwent ceMRN and non-contrast MRN (ncMRN) examination at 3.0 Tesla. The degree of vascular suppression, nerve visualization, diagnostic confidence, and lesion conspicuity was qualitatively assessed by two radiologists. Kappa statistics were obtained for inter-reader agreement. The signal-to-noise ratio, contrast ratio (CR), and contrast-to-noise ratio (CNR) of the median nerve were measured. The subjective ratings and quantitative measurements were compared between ncMRN and ceMRN.</p><p><strong>Results: </strong>The inter-reader agreement was excellent (<i>k</i> > 0.8) for all qualitative assessments and visualization assessment of each nerve segment. Compared with ncMRN, ceMRN significantly improved vascular suppression in volunteers and patients (both <i>p</i> < 0.001). The ceMRN significantly enhanced nerve visualization of each segment (all <i>p</i> < 0.05) and diagnostic confidence in volunteers and patients (both <i>p</i> < 0.05). The ceMRN improved lesion conspicuity (<i>p</i> = 0.003) in patients. Quantitatively, ceMRN had significantly higher CRs of nerve <i>versus</i> subcutaneous fat, bone marrow, and vessels and CNR of nerve <i>versus</i> vessel than ncMRN (all <i>p</i> < 0.05).</p><p><strong>Conclusion: </strong>The ceMRN significantly improves the visualization of peripheral nerves and pathology in the wrist and palm by robustly suppressing the signals of fat, bone marrow, and especially vessels in volunteers and patients.</p>","PeriodicalId":22980,"journal":{"name":"Therapeutic Advances in Neurological Disorders","volume":"17 ","pages":"17562864241239739"},"PeriodicalIF":5.9,"publicationDate":"2024-03-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10964438/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140294636","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
Patisiran exposure in early pregnancy: a case report. 妊娠早期接触帕替西兰:病例报告。
IF 5.9 2区 医学
Therapeutic Advances in Neurological Disorders Pub Date : 2024-03-26 eCollection Date: 2024-01-01 DOI: 10.1177/17562864241239755
Valentin Loser, Thomas Baumgartner, Hélène Legardeur, Alice Panchaud, Marie Théaudin
{"title":"Patisiran exposure in early pregnancy: a case report.","authors":"Valentin Loser, Thomas Baumgartner, Hélène Legardeur, Alice Panchaud, Marie Théaudin","doi":"10.1177/17562864241239755","DOIUrl":"10.1177/17562864241239755","url":null,"abstract":"<p><p>We describe here the first case of exposure to patisiran treatment, a small interfering RNA molecule, during early pregnancy of a 36-year-old woman with symptomatic hereditary transthyretin-related amyloidosis. There were no major complications during pregnancy and delivery, except for a postpartum hemorrhage due to uterine atony. Vitamin A levels had to be closely monitored during pregnancy, and vitamin A substitution adapted accordingly. There was no sign of minor or major congenital abnormalities of the baby. One month after delivery, the patient showed slight clinical and electrophysiological signs of neuropathy progression due to patisiran treatment withdrawal. Patisiran infusions were resumed 3 months after delivery. Due to the unknown teratogenic potential of patisiran, the risk of neuropathy worsening associated with withholding treatment must of course be weighed against a potential teratogenic risk of treatment during pregnancy. Vitamin A levels need to be closely assessed, and substitution must be adapted accordingly, to avoid embryofetal adverse outcome due to vitamin A deficiency or toxicity.</p>","PeriodicalId":22980,"journal":{"name":"Therapeutic Advances in Neurological Disorders","volume":"17 ","pages":"17562864241239755"},"PeriodicalIF":5.9,"publicationDate":"2024-03-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10964446/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140294637","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
Treatment access and satisfaction on disease-modifying therapies of neuromyelitis optica spectrum disorder patients in China: a cross-sectional survey. 中国神经脊髓炎视网膜谱系障碍患者的治疗机会和对疾病改变疗法的满意度:横断面调查。
IF 5.9 2区 医学
Therapeutic Advances in Neurological Disorders Pub Date : 2024-03-23 eCollection Date: 2024-01-01 DOI: 10.1177/17562864241239105
Yue Yu, Mingkang Zhong, Chao Quan, Chunlai Ma
{"title":"Treatment access and satisfaction on disease-modifying therapies of neuromyelitis optica spectrum disorder patients in China: a cross-sectional survey.","authors":"Yue Yu, Mingkang Zhong, Chao Quan, Chunlai Ma","doi":"10.1177/17562864241239105","DOIUrl":"10.1177/17562864241239105","url":null,"abstract":"<p><strong>Background: </strong>Neuromyelitis optica spectrum disorder (NMOSD) is a rare and debilitating disease that has become more widely recognized in China. Legislative measures have been implemented by the government to improve treatment access for rare diseases.</p><p><strong>Objectives: </strong>To investigate the diagnostic journey, treatment status, treatment accessibility, and treatment satisfaction of the NMOSD patients on disease-modifying therapies (DMTs) in China.</p><p><strong>Design: </strong>A patient online survey.</p><p><strong>Methods: </strong>This cross-sectional online survey was conducted between November 2022 and January 2023. Patients over 18 years old and diagnosed with NMOSD were included. The questionnaire consisted of five sections covering demographics, diagnostic and treatment experiences, DMTs availability, cost and affordability, and treatment satisfaction using the Treatment Satisfaction Questionnaire for Medication (version II). Patient opinions and demands were also collected at the end of the survey.</p><p><strong>Results: </strong>A total of 375 patients diagnosed with NMOSD were recruited, of which 321 patients used DMTs. It required 1.22 ± 3.22 years and 3.58 ± 4.24 hospital visits for a definitive diagnosis. One-third of the patients still needed to travel for over 2 h to access DMTs. The total treatment expenditure was estimated to be CNY 59,827.00 (USD 8315.95) a year. Drug expenses alone accounted for 52.22% of the average annual household income. The most common challenges perceived were the inability to afford treatment and a lack of effective options. No significant difference was found in treatment satisfaction among DMTs, except that rituximab scored lowest in convenience compared to other DMTs. Patients' age and travel time required to obtain medications were negatively associated with global treatment satisfaction.</p><p><strong>Conclusion: </strong>In China, patients with NMOSD face challenges in obtaining proper treatment due to diagnostic difficulties, distant medication access, and high costs. Policies should prioritize improving disease education and alleviating financial burdens for the patients.</p>","PeriodicalId":22980,"journal":{"name":"Therapeutic Advances in Neurological Disorders","volume":"17 ","pages":"17562864241239105"},"PeriodicalIF":5.9,"publicationDate":"2024-03-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10960978/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140207593","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
Shifting from the treat-to-target to the early highly effective treatment approach in patients with multiple sclerosis - real-world evidence from Germany. 多发性硬化症患者从 "靶向治疗 "转向 "早期高效治疗"--来自德国的真实证据。
IF 5.9 2区 医学
Therapeutic Advances in Neurological Disorders Pub Date : 2024-03-23 eCollection Date: 2024-01-01 DOI: 10.1177/17562864241237857
Steffeni Papukchieva, Ann-Sophie Stratil, Maria Kahn, Nils-Henning Neß, Maike Hollnagel-Schmitz, Vivien Gerencser, Julia Rustemeier, Markus Eberl, Benjamin Friedrich, Tjalf Ziemssen
{"title":"Shifting from the treat-to-target to the early highly effective treatment approach in patients with multiple sclerosis - real-world evidence from Germany.","authors":"Steffeni Papukchieva, Ann-Sophie Stratil, Maria Kahn, Nils-Henning Neß, Maike Hollnagel-Schmitz, Vivien Gerencser, Julia Rustemeier, Markus Eberl, Benjamin Friedrich, Tjalf Ziemssen","doi":"10.1177/17562864241237857","DOIUrl":"10.1177/17562864241237857","url":null,"abstract":"<p><strong>Background: </strong>While evidence highlights the effectiveness of initiating disease-modifying therapy with a high-efficacy medication for multiple sclerosis (MS) patients with poor prognostic factors, it remains unclear whether this approach has been adopted by a broad range of MS providers in Germany yet.</p><p><strong>Objective: </strong>To assess the adoption of the early highly effective treatment (EHT) compared to the treat-to-target treatment approach with the option of escalating treatment efficacy over time in Germany based on real-world evidence data.</p><p><strong>Design: </strong>Patient-level pharmacy dispensing data from the Permea platform were analysed from 2020 to 2022.</p><p><strong>Methods: </strong>In total, 29,529 therapy beginners (>18 years) were included to analyse shifts in treatment approaches over time and switching behaviour. Medication classification adhered to the German Society of Neurology guidelines and designated fumarates, glatiramer acetate, teriflunomide and interferons as low-efficacy category 1 medications; cladribine and S1P-modulators as medium-efficacy category 2 medications; and alemtuzumab, natalizumab, ocrelizumab, ofatumumab and rituximab (off-label) as high-efficacy category 3 medications.</p><p><strong>Results: </strong>Our results show that 70.0% of patients redeemed their first prescription for category 1 medication, 16.3% for category 2 and 13.7% for category 3 medications. The proportion of prescriptions filled shifted from 2020 to 2022 with a decrease of 14.7% for category 1 drugs and an increase of 12.5% for category 3 drugs. 93.2% of patients stayed on their initially prescribed medication category. 3.2% of category 1 and 3.7% of category 2 therapy beginners escalated to category 3 medication. 3.4% of category 3 medication users de-escalated their treatment to category 1 or category 2.</p><p><strong>Conclusion: </strong>While most individuals started their treatment according to the treat-to-target approach and remained on their initially prescribed medication category, there has been a steadily increasing shift towards the EHT approach since 2020. These insights demonstrate that, while not officially recommended by German guidelines, MS providers increasingly adopt the EHT approach.</p>","PeriodicalId":22980,"journal":{"name":"Therapeutic Advances in Neurological Disorders","volume":"17 ","pages":"17562864241237857"},"PeriodicalIF":5.9,"publicationDate":"2024-03-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10960977/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140207592","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
Comparative efficacy of ofatumumab versus oral therapies for relapsing multiple sclerosis patients using propensity score analyses and simulated treatment comparisons. 使用倾向评分分析和模拟治疗比较法比较复发性多发性硬化症患者使用奥妥木单抗和口服疗法的疗效。
IF 5.9 2区 医学
Therapeutic Advances in Neurological Disorders Pub Date : 2024-03-23 eCollection Date: 2024-01-01 DOI: 10.1177/17562864241239453
Nicholas Riley, Christopher Drudge, Morag Nelson, Anja Haltner, Michael Barnett, Simon Broadley, Helmut Butzkueven, Pamela McCombe, Anneke Van der Walt, Erin O Y Wong, Martin Merschhemke, Nicholas Adlard, Rob Walker, Imtiaz A Samjoo
{"title":"Comparative efficacy of ofatumumab <i>versus</i> oral therapies for relapsing multiple sclerosis patients using propensity score analyses and simulated treatment comparisons.","authors":"Nicholas Riley, Christopher Drudge, Morag Nelson, Anja Haltner, Michael Barnett, Simon Broadley, Helmut Butzkueven, Pamela McCombe, Anneke Van der Walt, Erin O Y Wong, Martin Merschhemke, Nicholas Adlard, Rob Walker, Imtiaz A Samjoo","doi":"10.1177/17562864241239453","DOIUrl":"10.1177/17562864241239453","url":null,"abstract":"<p><strong>Background: </strong>Evidence from network meta-analyses (NMAs) and real-world propensity score (PS) analyses suggest monoclonal antibodies (mAbs) offer a therapeutic advantage over currently available oral therapies and, therefore, warrant consideration as a distinct group of high-efficacy disease-modifying therapies (DMTs) for patients with relapsing multiple sclerosis (RMS). This is counter to the current perception of these therapies by some stakeholders, including payers.</p><p><strong>Objectives: </strong>A multifaceted indirect treatment comparison (ITC) approach was undertaken to clarify the relative efficacy of mAbs and oral therapies.</p><p><strong>Design: </strong>Two ITC methods that use individual patient data (IPD) to adjust for between-trial differences, PS analyses and simulated treatment comparisons (STCs), were used to compare the mAb ofatumumab <i>versus</i> the oral therapies cladribine, fingolimod, and ozanimod.</p><p><strong>Data sources and methods: </strong>As IPD were available for trials of ofatumumab and fingolimod, PS analyses were conducted. Given summary-level data were available for cladribine, fingolimod, and ozanimod trials, STCs were conducted between ofatumumab and each of these oral therapies. Three efficacy outcomes were compared: annualized relapse rate (ARR), 3-month confirmed disability progression (3mCDP), and 6-month CDP (6mCDP).</p><p><strong>Results: </strong>The PS analyses demonstrated ofatumumab was statistically superior to fingolimod for ARR and time to 3mCDP but not time to 6mCDP. In STCs, ofatumumab was statistically superior in reducing ARR and decreasing the proportion of patients with 3mCDP compared with cladribine, fingolimod, and ozanimod and in decreasing the proportion with 6mCP compared with fingolimod and ozanimod. These findings were largely consistent with recently published NMAs that identified mAb therapies as the most efficacious DMTs for RMS.</p><p><strong>Conclusion: </strong>Complementary ITC methods showed ofatumumab was superior to cladribine, fingolimod, and ozanimod in lowering relapse rates and delaying disability progression among patients with RMS. Our study supports the therapeutic superiority of mAbs over currently available oral DMTs for RMS and the delineation of mAbs as high-efficacy therapies.</p>","PeriodicalId":22980,"journal":{"name":"Therapeutic Advances in Neurological Disorders","volume":"17 ","pages":"17562864241239453"},"PeriodicalIF":5.9,"publicationDate":"2024-03-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10960976/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140207591","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
Clinical characteristics and surgical outcomes of low-grade epilepsy-associated brain tumors. 低度癫痫相关脑肿瘤的临床特征和手术效果。
IF 5.9 2区 医学
Therapeutic Advances in Neurological Disorders Pub Date : 2024-03-22 eCollection Date: 2024-01-01 DOI: 10.1177/17562864241237851
Suhui Kuang, Shaohui Zhang, Zhiqiang Cui, Ming Ge, Liu Yuan, Jiaqi Wang, Zhirong Wei, Jinshan Xu, Feng Zhai, Shuli Liang
{"title":"Clinical characteristics and surgical outcomes of low-grade epilepsy-associated brain tumors.","authors":"Suhui Kuang, Shaohui Zhang, Zhiqiang Cui, Ming Ge, Liu Yuan, Jiaqi Wang, Zhirong Wei, Jinshan Xu, Feng Zhai, Shuli Liang","doi":"10.1177/17562864241237851","DOIUrl":"10.1177/17562864241237851","url":null,"abstract":"<p><strong>Background: </strong>Low-grade epilepsy-associated brain tumors (LEATs) are found to be the second most common lesion-related epilepsy. Malignant potential of LEATs is very low and the overall survival is good, so the focus of treatment is focused more on seizure outcome rather than oncological prognosis.</p><p><strong>Objectives: </strong>This study was conducted to evaluate the risk factors of seizure outcomes after resection in patients with LEATs.</p><p><strong>Design: </strong>A retrospective study.</p><p><strong>Methods: </strong>A retrospective analysis of patients with LEATs who underwent resective surgery in our three epilepsy centers between October 2010 and April 2023 with a minimum follow-up of 1 year. Demography, clinical characters, neurophysiology, and molecular neuropathology were assessed for association with postoperative seizure outcomes at 1-, 2-, and 5-year follow-up. Synthetic minority oversampling technique (SMOTE) algorithm model was performed to handle the imbalance of data distribution. Gaussian Naïve Bayes (GNB) algorithms were created as a basis for classifying outcomes according to observation indicators.</p><p><strong>Results: </strong>A total of 111 patients were enrolled in the cohort. The most common pathology was ganglioglioma (<i>n</i> = 37, 33.3%). The percentage of patients with seizure freedom was 91.0% (101/111) at 1-year follow-up, 87.5% (77/88) at 2-year follow-up, and 79.1% (53/67) at 5-year follow-up. Partial resection had a significantly poor seizure outcome compared to total resection and supratotal resection (<i>p</i> < 0.05). The epileptiform discharge on post-resective intraoperative electrocorticography (ECoG) or postoperative scalp electroencephalography (EEG) were negative factors on postoperative seizure freedom at 1-, 2-, or 5-year follow-ups (<i>p</i> < 0.05). The area under the receiver-operating characteristic curve value of the GNB-SMOTE model was 0.95 (95% CI, 0.876-1.000), 0.892 (95% CI, 0.656-0.934), and 0.786 (95% CI, 0.491-0.937) at 1-, 2-, and 5-year follow-up, respectively.</p><p><strong>Conclusion: </strong>The partial resection, post-resective intraoperative ECoG, and postoperative scalp EEG were valuable indicators of poor seizure outcomes. The utilization of post-resective intraoperative ECoG is beneficial to improve seizure outcomes. Based on the data diversity and completeness of three medical centers, a multivariate correlation analysis model was established based on GNB algorithm.</p>","PeriodicalId":22980,"journal":{"name":"Therapeutic Advances in Neurological Disorders","volume":"17 ","pages":"17562864241237851"},"PeriodicalIF":5.9,"publicationDate":"2024-03-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10958794/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140207590","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
Concurrent acute sensorimotor axonal neuropathy and disseminated encephalitis associated with Chlamydia pneumoniae in an adult patient with anti-MOG and anti-sulfatide antibodies: a case report. 抗 MOG 和抗硫化物抗体成人患者并发急性感觉运动轴索神经病和肺炎衣原体播散性脑炎:病例报告。
IF 5.9 2区 医学
Therapeutic Advances in Neurological Disorders Pub Date : 2024-03-16 eCollection Date: 2024-01-01 DOI: 10.1177/17562864241237850
Michail Papantoniou, Grigorios Panagopoulos
{"title":"Concurrent acute sensorimotor axonal neuropathy and disseminated encephalitis associated with <i>Chlamydia pneumoniae</i> in an adult patient with anti-MOG and anti-sulfatide antibodies: a case report.","authors":"Michail Papantoniou, Grigorios Panagopoulos","doi":"10.1177/17562864241237850","DOIUrl":"10.1177/17562864241237850","url":null,"abstract":"<p><p>Acute disseminated encephalomyelitis and Guillain-Barré syndrome refer to post-infectious or post-vaccination inflammatory demyelinating disorders of central and peripheral nervous system, respectively. We report the case of a 60-year-old male patient presenting with irritability, gait difficulty, asymmetric quadriparesis (mostly in his left extremities), distal sensory loss for pain and temperature in left limbs, and reduced tendon reflexes in his upper limbs and absent in his lower limbs, following an upper respiratory tract infection, 3 weeks earlier. Brain magnetic resonance imaging revealed abnormal T2 signal and peripherally enhancing lesions in hemispheres, brainstem, and cerebellum. Nerve conduction studies were compatible with acute motor and sensory axonal neuropathy. Serology revealed positive IgM and IgG antibodies for <i>Chlamydia pneumoniae</i>, and he also tested positive for myelin oligodendrocyte glycoprotein (MOG) and sulfatide antibodies. Treatment with intravenous immunoglobulin and methylprednisolone led to clinical and radiological recovery within weeks. Even though several cases of combined central and peripheral demyelination have been reported before, it is the first case report with seropositive anti-sulfatide and anti-MOG acute sensorimotor axonal neuropathy and disseminated encephalitis associated with <i>C. pneumoniae.</i></p>","PeriodicalId":22980,"journal":{"name":"Therapeutic Advances in Neurological Disorders","volume":"17 ","pages":"17562864241237850"},"PeriodicalIF":5.9,"publicationDate":"2024-03-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10944586/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140144072","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
Disease-modifying therapy initiation patterns in multiple sclerosis in three large MS populations. 三个大型多发性硬化症群体中多发性硬化症的疾病修饰疗法启动模式。
IF 5.9 2区 医学
Therapeutic Advances in Neurological Disorders Pub Date : 2024-03-15 eCollection Date: 2024-01-01 DOI: 10.1177/17562864241233044
Alexander Stahmann, Elaine Craig, David Ellenberger, Firas Fneish, Niklas Frahm, Ruth Ann Marrie, Rod Middleton, Richard Nicholas, Jeff Rodgers, Clemens Warnke, Amber Salter
{"title":"Disease-modifying therapy initiation patterns in multiple sclerosis in three large MS populations.","authors":"Alexander Stahmann, Elaine Craig, David Ellenberger, Firas Fneish, Niklas Frahm, Ruth Ann Marrie, Rod Middleton, Richard Nicholas, Jeff Rodgers, Clemens Warnke, Amber Salter","doi":"10.1177/17562864241233044","DOIUrl":"10.1177/17562864241233044","url":null,"abstract":"<p><strong>Background: </strong>Treatment guidelines recommend early disease-modifying therapy (DMT) initiation after diagnosis of multiple sclerosis (MS). Multinational comparative studies that assess time to DMT initiation in MS may allow detection of barriers inherent to healthcare systems to explain potential adverse systematic delays in commencing DMTs.</p><p><strong>Objectives: </strong>To investigate and compare the time to first DMT and its association with sociodemographic and clinical variables after MS diagnosis in three large MS registries.</p><p><strong>Design: </strong>This observational study was conducted using data from the German MS Registry (GMSR), the North American Research Committee on MS Registry (NARCOMS, US data only), and the United Kingdom MS Registry (UKMSR, both self- and clinician-reported).</p><p><strong>Methods: </strong>Data from relapsing people with MS (PwMS), with a diagnosis of MS between 2014 and 2019, and available DMT and disability status were pooled using a meta-analytic approach.</p><p><strong>Results: </strong>A total of 5395 PwMS were included in the analysis (GMSR: <i>n</i> = 2658; NARCOMS: <i>n</i> = 447; UKMSR: <i>n</i> = 2290). Kaplan-Meier estimates for the time to first DMT [median months (95% CI)] were 2.0 (1.9-2.0), 3.0 (2-4), and 9.0 (7.7-10.6) for GMSR, NARCOMS, and UKMSR, respectively. Pooled multivariable Cox regression demonstrated shorter time to first DMT for PwMS diagnosed after 2017 [1.65 (1.42-1.92), <i>p</i> < 0.01], and longer time to DMT when a higher-efficacy DMT was selected (0.69 (0.54-0.90), <i>p</i> < 0.0001].</p><p><strong>Conclusion: </strong>Time to DMT initiation differs across the populations studied, indicating that barriers may exist in early access to DMT, particularly in the United Kingdom. However, a consistent decrease in time to DMT initiation was noted since 2017 across all registries. Further studies are warranted comparing the effects of time to DMT and time to higher-efficacy DMT on long-term outcome.</p>","PeriodicalId":22980,"journal":{"name":"Therapeutic Advances in Neurological Disorders","volume":"17 ","pages":"17562864241233044"},"PeriodicalIF":5.9,"publicationDate":"2024-03-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10943712/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140144114","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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