Canadian Journal of Neurological Sciences / Journal Canadien des Sciences Neurologiques最新文献_第5页

D.1 Efficacy, safety, and tolerability of subcutaneous efgartigimod in chronic inflammatory demyelinating polyneuropathy: results from the ADHERE trial D.1 皮下注射依加替莫德治疗慢性炎症性脱髓鞘性多发性神经病的疗效、安全性和耐受性：ADHERE 试验的结果

Canadian Journal of Neurological Sciences / Journal Canadien des Sciences Neurologiques Pub Date : 2024-05-24 DOI: 10.1017/cjn.2024.92

Z. Siddiqi, JA Allen, I Basta, C Eggers, J Guptill, K Gwathmey, C. Hewamadduma, E Hofman, Y. Hussain, S Kuwabara, F Leypoldt, J. Lin, M Lipowska, M Lowe, G Lauria Pinter, L Querol, N Suresh, T Chang, A Tse, P Ulrichts, PA van Doorn, B Van Hoorick, R. Yamasaki, RA Lewis

{"title":"D.1 Efficacy, safety, and tolerability of subcutaneous efgartigimod in chronic inflammatory demyelinating polyneuropathy: results from the ADHERE trial","authors":"Z. Siddiqi, JA Allen, I Basta, C Eggers, J Guptill, K Gwathmey, C. Hewamadduma, E Hofman, Y. Hussain, S Kuwabara, F Leypoldt, J. Lin, M Lipowska, M Lowe, G Lauria Pinter, L Querol, N Suresh, T Chang, A Tse, P Ulrichts, PA van Doorn, B Van Hoorick, R. Yamasaki, RA Lewis","doi":"10.1017/cjn.2024.92","DOIUrl":"https://doi.org/10.1017/cjn.2024.92","url":null,"abstract":"Background: Efgartigimod, a human immunoglobulin G (IgG)1 antibody Fc fragment, blocks the neonatal Fc receptor, decreasing IgG recycling and reducing pathogenic IgG autoantibody levels. ADHERE assessed the efficacy and safety of efgartigimod PH20 subcutaneous (SC; co-formulated with recombinant human hyaluronidase PH20) in chronic inflammatory demyelinating polyneuropathy (CIDP). Methods: ADHERE enrolled participants with CIDP (treatment naive or on standard treatments withdrawn during run-in period) and consisted of open-label Stage A (efgartigimod PH20 SC once weekly [QW]), and randomized (1:1) Stage B (efgartigimod or placebo QW). Primary outcomes were clinical improvement (assessed with aINCAT, I-RODS, or mean grip strength; Stage A) and time to first aINCAT score deterioration (relapse; Stage B). Secondary outcomes included treatment-emergent adverse events (TEAEs) incidence. Results: 322 participants entered Stage A. 214 (66.5%) were considered responders, randomized, and treated in Stage B. Efgartigimod significantly reduced the risk of relapse (HR: 0.394; 95% CI: 0.25–0.61) versus placebo (p=0.000039). Reduced risk of relapse occurred in participants receiving corticosteroids, intravenous or SC immunoglobulin, or no treatment before study entry. Most TEAEs were mild to moderate; 3 deaths occurred, none related to efgartigimod. Conclusions: Participants treated with efgartigimod PH20 SC maintained a clinical response and remained relapse-free longer than those treated with placebo.","PeriodicalId":9571,"journal":{"name":"Canadian Journal of Neurological Sciences / Journal Canadien des Sciences Neurologiques","volume":"25 10","pages":""},"PeriodicalIF":0.0,"publicationDate":"2024-05-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141102822","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

P.020 Shifts in daytime functioning items on the insomnia severity scale with lemborexant after 6 months of treatment P.020 LEMBOREXANT 治疗 6 个月后失眠严重程度量表中日间功能项目的变化

Canadian Journal of Neurological Sciences / Journal Canadien des Sciences Neurologiques Pub Date : 2024-05-24 DOI: 10.1017/cjn.2024.127

C. Chepke, K. Cote, K. Pinner, J. Yardley, M. Moline

{"title":"P.020 Shifts in daytime functioning items on the insomnia severity scale with lemborexant after 6 months of treatment","authors":"C. Chepke, K. Cote, K. Pinner, J. Yardley, M. Moline","doi":"10.1017/cjn.2024.127","DOIUrl":"https://doi.org/10.1017/cjn.2024.127","url":null,"abstract":"Background: Improvements in daytime functioning ideally accompany improvements in insomnia. Scores on the Insomnia Severity Index (ISI) daytime-related items were analyzed following treatment with lemborexant (LEM), a dual orexin receptor antagonist, or placebo (PBO), based on baseline severity. Methods: Participants (≥18 y) with insomnia disorder in E2006-G000-303, a 12-month, randomized, double-blind, PBO-controlled study (first 6 months: Treatment Period 1 [TP1]), were randomized to PBO or LEM 5 mg (LEM5) or 10 mg (LEM10) for 6 months. ISI items are rated 0 (no problem) to 4 (very severe problem); daytime-related ISI items have a maximum score of 16. Results: Of 949 participants, 749 (78.9%) completed the ISI at baseline and end of TP1. Baseline daytime ISI total score distributions were similar between groups. More participants with baseline scores of 9-12 and 13-16 shifted to 0-4 with LEM5 (49.7% and 39.1%, respectively) and LEM10 (46.2% and 46.3%) versus PBO (26.6% and 29.6%). Overall shift distributions were significantly different, favoring both LEM groups (P<0.01). LEM was well tolerated. Conclusions: More LEM-treated participants had improved daytime functioning, evidenced by the significantly larger number of participants whose scores moved into lower categories (ie, better sleep) versus PBO-treated participants, demonstrating additional value beyond improved sleep parameters.","PeriodicalId":9571,"journal":{"name":"Canadian Journal of Neurological Sciences / Journal Canadien des Sciences Neurologiques","volume":"55 9","pages":""},"PeriodicalIF":0.0,"publicationDate":"2024-05-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141102353","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

P.108 Rate and clinical utility of early postoperative CT head in adult craniotomy P.108 成人开颅手术术后早期头部 CT 的应用率和临床实用性

Canadian Journal of Neurological Sciences / Journal Canadien des Sciences Neurologiques Pub Date : 2024-05-24 DOI: 10.1017/cjn.2024.211

IE Harmsen, I. Fatokun, C. Elliott

{"title":"P.108 Rate and clinical utility of early postoperative CT head in adult craniotomy","authors":"IE Harmsen, I. Fatokun, C. Elliott","doi":"10.1017/cjn.2024.211","DOIUrl":"https://doi.org/10.1017/cjn.2024.211","url":null,"abstract":"Background: Postoperative cranial neurosurgical imaging practices are highly variable. We evaluated the rate and utility of early postoperative computed tomography (EPCT, defined as a CT head scan within 24h of surgery) in consecutive adult craniotomies. Methods: We retrospectively reviewed consecutive adult craniotomies at the University of Alberta Hospital over a 45-day period (17/09/2022 to 01/11/2022). Electronic medical records were reviewed to extract data on the rate, timing, and utility of EPCT as well as the rate of neurologic deterioration and repeat surgical intervention. Results: A total of 56 patients (27 female; 55.5 ± 2.1 yrs, range: 19-84 years) were identified. All patients underwent EPCT, including 10/56 (17.9%) on POD0 and 46/56 (82.1%) on POD1. Surgical complications (bleeding, extensive pneumocephalus, edema, ischemia) were identified in 8/56 (14.3%) of the EPCT, of which 6 (10.7%) were reported to have neurologic deterioration and 2 (3.6%) underwent further surgical intervention (hematoma evacuation). Clinical and radiological postoperative changes were highly related (p=5.16e-06), and the rate of EPCT being adverse without neurologic deficit, managed surgically, was 1/56 (1.8%). Conclusions: EPCT is routine practice. Given the low rate (1.8%) of repeat surgical intervention in the absence of neurologic deficit despite abnormal EPCT, omitting EPCT in neurologically intact patients may be warranted.","PeriodicalId":9571,"journal":{"name":"Canadian Journal of Neurological Sciences / Journal Canadien des Sciences Neurologiques","volume":"13 11","pages":""},"PeriodicalIF":0.0,"publicationDate":"2024-05-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141098764","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

P.106 Using optic nerve sheath diameter over ventricular size to assess elevated intracranial pressure in pediatric patients with pineal region tumors P.106 使用视神经鞘直径大于脑室大小来评估患有松果体区肿瘤的儿科患者颅内压升高的情况

Canadian Journal of Neurological Sciences / Journal Canadien des Sciences Neurologiques Pub Date : 2024-05-24 DOI: 10.1017/cjn.2024.209

J. Zipfel, SR Kerscher, K. Dhillon, KP Ferraris, D. Feucht, A. Weir, MU Schuhmann, A. Singhal

{"title":"P.106 Using optic nerve sheath diameter over ventricular size to assess elevated intracranial pressure in pediatric patients with pineal region tumors","authors":"J. Zipfel, SR Kerscher, K. Dhillon, KP Ferraris, D. Feucht, A. Weir, MU Schuhmann, A. Singhal","doi":"10.1017/cjn.2024.209","DOIUrl":"https://doi.org/10.1017/cjn.2024.209","url":null,"abstract":"Background: Pineal region tumors are a heterogenous group of pathologies often symptomatic due to occlusive hydrocephalus leading to elevated intracranial pressure (ICP). High ICP may not always be associated with clinical signs. A non-invasive technique for assessment of ICP is measuring the optic nerve sheath diameter (ONSD). The goal of this study was to determine the utility of preoperative and postoperative ONSD measurements for assessment of elevated ICP in children with pineal region tumors. Methods: Retrospective data analysis was performed in patients operated for pineal region tumors at our tertiary care center between 2003 and 2022. Preoperative and postoperative MRI scans were reviewed. Clinical data and ONSD at multiple time points were analyzed and correlated. Results: Thirty-four patients with forty operative cases met the inclusion criteria. Hydrocephalus was seen in 80% of patients preoperatively (n=32/40). Presence of hydrocephalus was associated with significantly elevated ONSD preoperatively (p=0.006) and postoperatively (p=0.017). There was significant decrease in ONSD immediately postoperatively (p<0.001), at 3 months (p<0.001) and 12 months (p<0.001). In patients without hydrocephalus, no significant changes in ONSD were observed (p=0.369). Conclusions: ONSD is a useful adjunct for the identification of high ICP preoperatively and evaluation of treatment response postoperatively in patients presenting with pineal region tumors.","PeriodicalId":9571,"journal":{"name":"Canadian Journal of Neurological Sciences / Journal Canadien des Sciences Neurologiques","volume":"3 3","pages":""},"PeriodicalIF":0.0,"publicationDate":"2024-05-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141098936","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

P.074 Assessing the emergence and evolution of artificial intelligence and machine learning research in neuroradiology P.074 评估神经放射学中人工智能和机器学习研究的出现与发展

Canadian Journal of Neurological Sciences / Journal Canadien des Sciences Neurologiques Pub Date : 2024-05-24 DOI: 10.1017/cjn.2024.180

SS Haile, A Boutet, AZ Wang, H. Son, M Malik, V Pai, M Nasralla, J. Germann, A. Vetkas, F. Khalvati, BB Ertl-Wagner

{"title":"P.074 Assessing the emergence and evolution of artificial intelligence and machine learning research in neuroradiology","authors":"SS Haile, A Boutet, AZ Wang, H. Son, M Malik, V Pai, M Nasralla, J. Germann, A. Vetkas, F. Khalvati, BB Ertl-Wagner","doi":"10.1017/cjn.2024.180","DOIUrl":"https://doi.org/10.1017/cjn.2024.180","url":null,"abstract":"Background: Interest in artificial intelligence (AI) and machine learning (ML) has been growing in neuroradiology, but there is limited knowledge on how this interest has manifested into research and the field’s trends, challenges, and future directions. Methods: The American Journal of Neuroradiology was queried for original research articles published since inception (Jan. 1, 1980) to Sept. 19, 2022 that contained any of the following key terms: “machine learning”, “artificial intelligence”, or “radiomics”. Articles were screened, categorized into Statistical Modelling (Type 1), AI/ML Development (Type 2), or End-user Application (Type 3) and then bibliometrically analyzed. Results: A total of 124 articles were identified with 85% being non-integration focused (Type 1 n = 41, Type 2 n = 65) and the remaining (n = 18) being Type 3. The total number of articles published grew two-fold in the last five years, with Type 2 articles mainly driving this growth. While most (66%) Type 2 articles were led by a radiologist with 55% possessing a postgraduate degree, a minority of Type 2 articles addressed bias (15%) and explainability (20%). Conclusions: The results of this study highlight areas for improvement but also strengths that stakeholders can consider when promoting the shift towards integrating practical AI/ML solutions in neuroradiology.","PeriodicalId":9571,"journal":{"name":"Canadian Journal of Neurological Sciences / Journal Canadien des Sciences Neurologiques","volume":"14 5","pages":""},"PeriodicalIF":0.0,"publicationDate":"2024-05-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141099744","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

P.022 Gender disparity in canadian institutes of health research funding within neurology P.022 加拿大卫生研究所神经学研究经费中的性别差异

Canadian Journal of Neurological Sciences / Journal Canadien des Sciences Neurologiques Pub Date : 2024-05-24 DOI: 10.1017/cjn.2024.129

C Tsai, B. Tao, C Wang, AR Vosoughi, E. Bui, KM Chapman, SH Fox, F. Khosa

{"title":"P.022 Gender disparity in canadian institutes of health research funding within neurology","authors":"C Tsai, B. Tao, C Wang, AR Vosoughi, E. Bui, KM Chapman, SH Fox, F. Khosa","doi":"10.1017/cjn.2024.129","DOIUrl":"https://doi.org/10.1017/cjn.2024.129","url":null,"abstract":"Background: Despite efforts to advance equity, women face gender-based barriers in research, including fewer senior authorship and grant opportunities. We examined gender disparities in Canadian Institutes of Health Research (CIHR) funding for Canadian neurology divisions and departments. Methods: Data on CIHR grant recipients and metrics (duration, quantity, and contribution) within Canadian neurology divisions and departments (2008-2022) were acquired from the CIHR Funding Decisions Database. Gender-based differences in grant prevalence, duration, and contribution amount within neurology were calculated with subgroup analysis for Canadian neurologists and Project Grant awards. Results: 1604 grants were awarded to Canadian neurology divisions and departments between 2008-2022. Women received fewer grants (41.46%), less funding (p<0.0001), and shorter grant durations (p<0.0001) than men annually. Women comprised the minority of recipients (45.47%) and were less likely to be awarded grants (p<0.001) annually relative to men. Differences were consistent in subgroup analyses, except grant durations were equal across genders in Project Grant awards. Conclusions: Gender disparities persist in CIHR grant funding to Canadian neurology divisions and departments. Women receive fewer grants, lower contribution amounts, and are less likely to be recipients compared to men. Future work includes addressing gender differences and continuing to evaluate CIHR funding to provide equitable opportunities for women.","PeriodicalId":9571,"journal":{"name":"Canadian Journal of Neurological Sciences / Journal Canadien des Sciences Neurologiques","volume":"9 5","pages":""},"PeriodicalIF":0.0,"publicationDate":"2024-05-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141100136","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

P.133 Neurons in the lateral prefrontal cortex encode task features during virtual navigation P.133 外侧前额叶皮层神经元在虚拟导航过程中编码任务特征

Canadian Journal of Neurological Sciences / Journal Canadien des Sciences Neurologiques Pub Date : 2024-05-24 DOI: 10.1017/cjn.2024.234

M. Abbass, B. Corrigan, R. Johnston, R. Gulli, A. Sachs, JC Lau, J. Martinez-Trujillo

{"title":"P.133 Neurons in the lateral prefrontal cortex encode task features during virtual navigation","authors":"M. Abbass, B. Corrigan, R. Johnston, R. Gulli, A. Sachs, JC Lau, J. Martinez-Trujillo","doi":"10.1017/cjn.2024.234","DOIUrl":"https://doi.org/10.1017/cjn.2024.234","url":null,"abstract":"Background: The lateral prefrontal cortex (LPFC) is uniquely found in primates and has been associated with contextual learning. This function is thought to be subserved by neurons that are tuned to abstract concepts and the combination of those concepts. LPFC neuron tuning remains to be fully investigated in naturalistic conditions. Methods: Two macaques were trained to perform a context-colour association task while using a joystick to navigate in an X-shaped maze. They were implanted with two 96-channel microelectrode arrays, targeting the LPFC. Mean firing rates were computed and multivariate linear regressions were used to determine tuning. Results: LPFC neurons were tuned to context (12.4%), color position (6.2%), target side (17.2%), and were selective to more than one feature (21.2%). LPFC neurons acquired tuning to task features in an ordered manner, starting with context (130.1±27.4ms), followed by the colour position (296.2±21.4ms) and then target side (493.3±19.3ms). Furthermore, most neurons (54%) changed their tuning over time. Conclusions: We demonstrate that single neurons can encode relevant features embedded in a naturalistic virtual environment. Our results support previous observations that LPFC neurons combine individual features and suggest that these features are also combined temporally. These findings contribute towards understanding the LPFC and have potential practical implications.","PeriodicalId":9571,"journal":{"name":"Canadian Journal of Neurological Sciences / Journal Canadien des Sciences Neurologiques","volume":"93 6","pages":""},"PeriodicalIF":0.0,"publicationDate":"2024-05-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141101137","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

P.051 Parkinson’s disease tremor can show entrainment and distractibility with tapping test P.051 帕金森病震颤可通过拍击试验显示出缠绕性和分心性

Canadian Journal of Neurological Sciences / Journal Canadien des Sciences Neurologiques Pub Date : 2024-05-24 DOI: 10.1017/cjn.2024.158

NC Sheth, T. Grippe, N. Raies, M. Ding, R Chen

{"title":"P.051 Parkinson’s disease tremor can show entrainment and distractibility with tapping test","authors":"NC Sheth, T. Grippe, N. Raies, M. Ding, R Chen","doi":"10.1017/cjn.2024.158","DOIUrl":"https://doi.org/10.1017/cjn.2024.158","url":null,"abstract":"Background: Electrophysiological tests such as the tapping test are used to distinguish functional and organic tremors, in which patients with functional tremor commonly show entrainment and amplitude reduction (>50% decrease relative to baseline) of contralateral tremor during tapping. While these features are suggested to be specific to functional tremor, the tapping test in Parkinson’s disease (PD) tremor has not been tested. Methods: We evaluated 18 PD patients (2F, age 64.17±7.30 [mean±SD] years) with rest and postural tremors using surface electromyography and triaxial accelerometry. Patients were recorded while tapping at 1, 3 and 5 Hz with the contralateral arm at rest or outstretched. Tremor amplitude and frequency were calculated using power spectrum analysis from accelerometer recordings. Results: Reduction of rest tremor amplitude was observed in 3/18 patients during 1 and 3 Hz tapping. Reduction was seen in 3/16 and 1/16 patients with postural tremors at 1 and 3 Hz tapping, respectively. Frequency shifts (>1.5 Hz) were observed in 3/18 rest tremors and 6/16 postural tremors. Seven patients exhibited rest and/or postural tremor entrainment during 3 or 5 Hz tapping. Conclusions: Distractibility and entrainment can be found in PD tremor. The tapping test may not reliably distinguish between PD tremor and functional tremor.","PeriodicalId":9571,"journal":{"name":"Canadian Journal of Neurological Sciences / Journal Canadien des Sciences Neurologiques","volume":"17 4","pages":""},"PeriodicalIF":0.0,"publicationDate":"2024-05-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141102443","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

P.054 Long-term safety and efficacy of zilucoplan in myasthenia gravis: additional interim analyses of RAISE-XT P.054 齐鲁霉素对重症肌无力的长期安全性和疗效：RAISE-XT 的额外中期分析

Canadian Journal of Neurological Sciences / Journal Canadien des Sciences Neurologiques Pub Date : 2024-05-24 DOI: 10.1017/cjn.2024.161

A. Genge, J. Howard, M. Freimer, C. Hewamadduma, Y. Hussain, A. Maniaol, R. Mantegazza, M. Śmiłowski, K. Utsugisawa, T. Vu, MD Weiss, PW Duda, B. Boroojerdi, M. Vanderkelen, G. de la Borderie, MI Leite

{"title":"P.054 Long-term safety and efficacy of zilucoplan in myasthenia gravis: additional interim analyses of RAISE-XT","authors":"A. Genge, J. Howard, M. Freimer, C. Hewamadduma, Y. Hussain, A. Maniaol, R. Mantegazza, M. Śmiłowski, K. Utsugisawa, T. Vu, MD Weiss, PW Duda, B. Boroojerdi, M. Vanderkelen, G. de la Borderie, MI Leite","doi":"10.1017/cjn.2024.161","DOIUrl":"https://doi.org/10.1017/cjn.2024.161","url":null,"abstract":"Background: Zilucoplan, a macrocyclic peptide complement component 5 inhibitor, sustained efficacy for up to 60 weeks of treatment, with a favourable safety profile in patients with acetylcholine receptor autoantibody-positive generalised myasthenia gravis in an interim analysis of RAISE-XT (NCT04225871). We evaluate the safety and efficacy of zilucoplan up to 96 weeks. Methods: RAISE-XT, a Phase 3, multicentre, open-label extension study, included patients who participated in the double-blind Phase 2 (NCT03315130) and Phase 3 (NCT04115293) zilucoplan studies. Patients self-administered daily subcutaneous zilucoplan 0.3mg/kg injections. Primary outcome was incidence of treatment-emergent adverse events (TEAEs). Secondary outcomes included change from baseline in Myasthenia Gravis Activities of Daily Living (MG-ADL) score. Results: At data cut-off (11 May 2023), median (range) exposure to zilucoplan was 1.8 (0.11–5.1) years (N=200). TEAEs occurred in 191 (95.5%) patients; the most common TEAE was COVID-19 (n=64; 32.0%). At Week 96, mean (standard error) change in MG-ADL score from double-blind study baseline was –6.33 (0.49) and –7.83 (0.60) for patients who received zilucoplan 0.3mg/kg and placebo in the double-blind studies, respectively. Conclusions: Zilucoplan demonstrated a favourable long-term safety profile. Efficacy was sustained for 96 weeks in patients who had previously received zilucoplan and who switched from placebo.","PeriodicalId":9571,"journal":{"name":"Canadian Journal of Neurological Sciences / Journal Canadien des Sciences Neurologiques","volume":"60 13","pages":""},"PeriodicalIF":0.0,"publicationDate":"2024-05-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141102723","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

P.045 Trofinetide for the treatment of Rett syndrome: long-term safety and efficacy results from the open-label LILAC and LILAC-2 studies P.045 治疗雷特综合征的曲非奈德：开放标签 LILAC 和 LILAC-2 研究的长期安全性和有效性结果

Canadian Journal of Neurological Sciences / Journal Canadien des Sciences Neurologiques Pub Date : 2024-05-24 DOI: 10.1017/cjn.2024.152

AK Percy, JL Neul, TA Benke, EM Berry-Kravis, DG Glaze, ED Marsh, A. Lamontagne, D. An, KM Bishop, JM Youakim

{"title":"P.045 Trofinetide for the treatment of Rett syndrome: long-term safety and efficacy results from the open-label LILAC and LILAC-2 studies","authors":"AK Percy, JL Neul, TA Benke, EM Berry-Kravis, DG Glaze, ED Marsh, A. Lamontagne, D. An, KM Bishop, JM Youakim","doi":"10.1017/cjn.2024.152","DOIUrl":"https://doi.org/10.1017/cjn.2024.152","url":null,"abstract":"Background: Trofinetide significantly improved core symptoms of Rett syndrome (RTT) with an acceptable safety profile in LAVENDER. Here, we report the safety and efficacy results of LILAC and LILAC-2, open-label extension studies of LAVENDER. Methods: Females with RTT, aged 5–21 years, received twice-daily, oral trofinetide in LILAC for 40 weeks. Participants who completed LAVENDER and LILAC continued trofinetide in LILAC-2, a 32-month extension study. Safety assessments included the incidence of adverse events (AEs). Efficacy endpoints included the Rett Syndrome Behaviour Questionnaire (RSBQ) and the Clinical Global Impression–Improvement (CGI-I) scale. Results: Overall, 154 patients were enrolled in LILAC. The most common AEs were diarrhea (74.7%) and vomiting (28.6%). The mean (standard error [SE]) change from the LAVENDER baseline to Week 40 in the LILAC study in RSBQ was -7.3 (1.62) and -7.0 (1.61) for participants treated with trofinetide and placebo in LAVENDER, respectively. Mean (SE) CGI-I scores compared with the LILAC baseline at Week 40 were 3.1 (0.11) and 3.2 (0.14) for patients treated with trofinetide and placebo in LAVENDER, respectively. Similar safety and efficacy trends were observed in LILAC-2. Conclusions: Trofinetide continued to improve symptoms of RTT in LILAC and LILAC-2 with a safety profile consistent with LAVENDER.","PeriodicalId":9571,"journal":{"name":"Canadian Journal of Neurological Sciences / Journal Canadien des Sciences Neurologiques","volume":"7 30","pages":""},"PeriodicalIF":0.0,"publicationDate":"2024-05-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141099028","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0