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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
IE Harmsen, I. Fatokun, C. Elliott
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.
背景:头颅神经外科术后成像方法千差万别。我们评估了连续性成人开颅手术中术后早期计算机断层扫描(EPCT,定义为手术后 24 小时内的头部 CT 扫描)的使用率和效用。方法:我们回顾性审查了阿尔伯塔大学医院在 45 天内(2022 年 9 月 17 日至 2022 年 11 月 1 日)连续进行的成人开颅手术。我们回顾了电子病历,以提取有关 EPCT 的使用率、时间和效用以及神经功能恶化和重复手术干预率的数据。结果:共发现 56 名患者(27 名女性;55.5 ± 2.1 岁,年龄范围:19-84 岁)。所有患者均接受了 EPCT,其中 10/56 人(17.9%)在 POD0 接受了 EPCT,46/56 人(82.1%)在 POD1 接受了 EPCT。8/56(14.3%)例 EPCT 患者出现了手术并发症(出血、广泛性气胸、水肿、缺血),其中 6 例(10.7%)患者的神经系统状况恶化,2 例(3.6%)患者接受了进一步的手术干预(血肿清除)。临床和放射学术后变化高度相关(p=5.16e-06),EPCT不良但无神经功能缺损并接受手术治疗的比例为1/56(1.8%)。结论:EPCT 是常规做法。鉴于EPCT异常但无神经功能缺损的重复手术干预率较低(1.8%),神经功能完好的患者可能有必要省略EPCT。
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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
J. Zipfel, SR Kerscher, K. Dhillon, KP Ferraris, D. Feucht, A. Weir, MU Schuhmann, A. Singhal
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.
背景:松果体区肿瘤是一组异质性病变,通常因闭塞性脑积水导致颅内压(ICP)升高而出现症状。高 ICP 并不总是与临床症状相关联。测量视神经鞘直径(ONSD)是评估 ICP 的一种非侵入性技术。本研究旨在确定术前和术后测量视神经鞘直径对评估松果体区肿瘤患儿ICP升高的实用性。方法:对 2003 年至 2022 年期间在本三级医疗中心接受松果体区肿瘤手术的患者进行回顾性数据分析。对术前和术后的磁共振成像扫描进行了回顾。对多个时间点的临床数据和ONSD进行分析和关联。结果:34名患者的40例手术符合纳入标准。80%的患者术前出现脑积水(32/40)。术前(p=0.006)和术后(p=0.017),脑积水的存在与 ONSD 的显著升高有关。术后即刻(p<0.001)、3 个月(p<0.001)和 12 个月(p<0.001)时 ONSD 均明显下降。在无脑积水的患者中,未观察到 ONSD 有明显变化(p=0.369)。结论ONSD 是松果体区肿瘤患者术前识别高 ICP 和术后评估治疗反应的有效辅助手段。
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引用次数: 0
P.091 Synthetic data reliably reproduces brain tumor primary research data P.091 合成数据可靠再现脑肿瘤原始研究数据
R. Khalaf, W. Davalan, A. Mohammad, RJ Diaz
Background: Synthetic data has garnered heightened attention in contemporary research due to confidentiality barriers and its capacity to simulate variables challenging to obtain. This study aimed to evaluate the reliability and validity of synthetic data in the context of neuro-oncology research, comparing findings from two published studies with results from synthetic datasets. Methods: Two published neuro-oncology studies focusing on prognostic factors such as serum albumin and systemic inflammation scores were selected, and their methodologies were replicated using MDClone Platform to generate five synthetic datasets for each. We used Chi-Square test to assess inter-variability between synthetic datasets. Survival outcomes were evaluated using Kaplan-Meier and t-test was used to determine statistical significance. Results: Findings from synthetic data consistently matched outcomes from both original articles, with serum albumin and systemc inflammation scores correlating with survival prognosis in glioblastoma and metastasis patients (p<0.05) Reported findings, demographic trends and survival outcomes showed significant similarity (P > 0.05) with synthetic datasets. Conclusions: Synthetic data consistently reproduced the statistical attributes of real patient data. Integrating synthetic data into clinical research offers excellent potential for providing accurate predictive insights without compromising patient privacy. In neuro-oncology, where patient follow-up pose challenges, the adoption of synthetic datasets can be transformative.
背景:合成数据因其保密性和模拟难以获得的变量的能力而在当代研究中备受关注。本研究旨在评估神经肿瘤学研究中合成数据的可靠性和有效性,将两项已发表的研究结果与合成数据集的结果进行比较。研究方法我们选取了两项已发表的神经肿瘤学研究,重点关注血清白蛋白和全身炎症评分等预后因素,并使用 MDClone 平台复制了它们的方法,为每项研究生成了五个合成数据集。我们使用 Chi-Square 检验来评估合成数据集之间的变异性。我们使用 Kaplan-Meier 法评估生存结果,并使用 t 检验确定统计显著性。结果合成数据的结果与两篇原始文章的结果一致,血清白蛋白和系统炎症评分与胶质母细胞瘤和转移瘤患者合成数据集的生存预后相关(P 0.05)。结论:合成数据一致再现了真实患者数据的统计属性。将合成数据整合到临床研究中可在不损害患者隐私的情况下提供准确的预测见解。在神经肿瘤学领域,患者随访是一项挑战,采用合成数据集可以带来变革。
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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
C Tsai, B. Tao, C Wang, AR Vosoughi, E. Bui, KM Chapman, SH Fox, F. Khosa
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.
背景:尽管在促进公平方面做出了努力,但女性在研究中仍面临着基于性别的障碍,包括较少的高级作者和资助机会。我们研究了加拿大卫生研究院(CIHR)对加拿大神经病学分部和科室资助的性别差异。研究方法我们从加拿大卫生研究院资助决策数据库(CIHR Funding Decisions Database)中获取了加拿大卫生研究院(CIHR)资助获得者的数据以及加拿大神经病学分部和部门的指标(持续时间、数量和贡献)(2008-2022 年)。通过对加拿大神经病学家和项目补助金进行分组分析,计算了神经病学中补助金获得率、持续时间和贡献金额方面的性别差异。结果:2008-2022 年间,加拿大神经病学分部和科室共获得 1604 项资助。与男性相比,女性每年获得的拨款较少(41.46%),资金较少(p<0.0001),拨款期限较短(p<0.0001)。女性在受助者中占少数(45.47%),与男性相比,女性每年获得资助的可能性较低(p<0.001)。亚组分析中的差异是一致的,但在项目补助金的授予中,不同性别的补助金持续时间是相同的。结论:加拿大神经病学分部和科室获得的 CIHR 基金资助仍然存在性别差异。与男性相比,女性获得的补助金更少,捐款金额更低,成为补助金获得者的可能性也更小。今后的工作包括解决性别差异问题,并继续评估加拿大高级研究中心的资助情况,以便为妇女提供公平的机会。
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引用次数: 0
P.019 A shared decision-model toolkit for pregnancy related care in neurology P.019 神经内科妊娠相关护理的共享决策模型工具包
Y. Iyengar, S. Ng, S Chan, N. Sultan, H. Thornton, T. Patel, K. Grindrod, K. Krysko, G. Moores, A. Pikula, E. Bui
Background: Shared decision-making (SDM) is a dynamic, patient-engaged approach to collaborative medical care. Limited SDM tools exist in pregnancy. We aimed to examine the need and usability of a novel SDM tool for pharmaco-therapeutic treatment of neurological conditions in pregnancy. Methods: This is an exploratory mixed-methods study. Non-pregnant women of any age were recruited using convenience, purposive sampling from an academic neurology clinic in Toronto. Participants reported the user friendliness of the SDM by completing the systems usability (SUS) questionnaire and participated in a focus group to further elaborate on their experience. Results: Eleven participants completed the survey 45% each between age 31-40, and 51-60. Median time spent on the tool was 17.2 minutes, and median SUS score 70 (<68 being not usable). Thematic data analysis from 2 focus groups, identified technical and content improvements: use of inclusive language, simplified design, and importance of patient engagement in SDM. Conclusions: Based on our preliminary results, a SDM web-tool for medication-related concerns of pregnant patients with neurological conditions is needed and usable. With integration of patients’ lived experiences, this novel tool may serve as an anchor point for future work in this field.
背景:共同决策(SDM)是一种动态的、患者参与的合作医疗方法。妊娠期 SDM 工具有限。我们旨在研究一种新型 SDM 工具在妊娠期神经疾病药物治疗中的必要性和可用性。方法:这是一项探索性混合方法研究。研究人员从多伦多的一家神经病学学术诊所中采用方便、有目的的抽样方式招募了任何年龄段的非孕妇。参与者通过填写系统可用性 (SUS) 问卷来报告 SDM 的用户友好性,并参加焦点小组进一步阐述她们的体验。结果:11 名参与者完成了调查,年龄在 31-40 岁和 51-60 岁之间的各占 45%。使用该工具的时间中位数为 17.2 分钟,SUS 分数中位数为 70 分(低于 68 分为不可用)。通过对两个焦点小组的专题数据分析,确定了技术和内容方面的改进:使用包容性语言、简化设计以及患者参与 SDM 的重要性。结论:根据我们的初步研究结果,针对神经系统疾病孕妇用药相关问题的 SDM 网络工具是需要的,也是可用的。结合患者的生活经验,这种新颖的工具可作为该领域未来工作的立足点。
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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
AK Percy, JL Neul, TA Benke, EM Berry-Kravis, DG Glaze, ED Marsh, A. Lamontagne, D. An, KM Bishop, JM Youakim
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.
研究背景在LAVENDER研究中,曲非奈德明显改善了雷特综合征(RTT)的核心症状,且安全性可接受。在此,我们报告 LILAC 和 LILAC-2 的安全性和疗效结果,这两项研究是 LAVENDER 的开放标签扩展研究。研究方法年龄在 5-21 岁的女性 RTT 患者在 LILAC 中接受了为期 40 周的每日两次口服特罗芬太尼治疗。完成 LAVENDER 和 LILAC 研究的参与者在为期 32 个月的 LILAC-2 扩展研究中继续服用特罗菲肽。安全性评估包括不良事件(AE)的发生率。疗效终点包括雷特综合征行为问卷(RSBQ)和临床总体印象改善量表(CGI-I)。结果LILAC共招募了154名患者。最常见的不良反应是腹泻(74.7%)和呕吐(28.6%)。在LILAC研究中,从LAVENDER基线到第40周的RSBQ平均变化(标准误差[SE])分别为-7.3(1.62)和-7.0(1.61);在LAVENDER研究中,接受特罗芬肽治疗的患者和接受安慰剂治疗的患者的RSBQ平均变化(标准误差[SE])分别为-7.3(1.62)和-7.0(1.61)。在第40周时,与LILAC基线相比,接受特罗芬肽治疗的患者和接受安慰剂治疗的患者的CGI-I评分平均值(SE)分别为3.1(0.11)和3.2(0.14)。在 LILAC-2 中也观察到了类似的安全性和疗效趋势。结论在 LILAC 和 LILAC-2 中,特罗菲奈肽继续改善了 RTT 的症状,其安全性与 LAVENDER 一致。
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引用次数: 0
P.151 A critical appraisal of the application of frailty and sarcopenia in the spinal oncology population P.151 对脊柱肿瘤人群中虚弱和肌肉疏松症应用的批判性评估
MA MacLean, AJ Charles, M. Georgiopoulos, J. Phinney, R. Charest-Morin, C. Goodwin, MH Weber
Background: Frailty and sarcopenia predict worse surgical outcomes among spinal degenerative and deformity-related populations; this association is less clear in the context of spinal oncology. Here, we identified frailty and sarcopenia tools applied in spinal oncology and appraised their clinimetric properties. Methods: A systematic review was conducted from January 1st, 2000, until June 2022. Study characteristics, frailty tools, measures of sarcopenia, component domains, individual items, cut-off values, and measurement techniques were collected. Clinimetric assessment was performed according to Consensus-based Standards for Health Measurement Instruments. Results: Twenty-two studies were included (42,514 patients). The three most employed frailty tools were the Metastatic Spine tumor Frailty Index (MSTFI), Modified Frailty Index-11 (mFI-11), and the mFI-5. The three most common sarcopenia measures were the L3-Total Psoas Area (TPA)/Vertebral Body Area (VBA), L3-TPA/Height2, and L3-Spinal Muscle Index (L3-Cross-Sectional Muscle Area/Height2). Frailty and sarcopenia measures lacked content and construct validity. Positive predictive validity was observed in select studies employing the HFRS, mFI-5, MSTFI, and L3-TPA/VBA. All frailty tools had floor or ceiling effects. Conclusions: Existing tools for evaluating frailty and sarcopenia in surgical spine oncology have poor clinimetric properties. Here, we provide a pragmatic approach to utilizing existing frailty and sarcopenia tools, until more clinimetrically robust instruments are developed.
背景:在脊柱退行性病变和畸形相关人群中,虚弱和肌肉疏松症预示着较差的手术效果;而在脊柱肿瘤学中,这种关联并不明显。在此,我们确定了应用于脊柱肿瘤学的虚弱和肌肉疏松症工具,并评估了其临床测量特性。方法:我们对 2000 年 1 月 1 日至 2022 年 6 月期间的研究进行了系统回顾。收集了研究特征、虚弱工具、肌肉疏松症测量方法、组成域、单个项目、临界值和测量技术。临床评估根据基于共识的健康测量工具标准进行。结果:共纳入 22 项研究(42514 名患者)。使用最多的三种虚弱工具是转移性脊柱肿瘤虚弱指数(MSTFI)、改良虚弱指数-11(mFI-11)和 mFI-5。三种最常见的肌肉疏松测量方法是 L3-腰肌总面积(TPA)/椎体面积(VBA)、L3-TPA/身高2 和 L3-脊髓肌肉指数(L3-横断面肌肉面积/身高2)。虚弱和肌肉疏松测量缺乏内容有效性和结构有效性。在采用 HFRS、mFI-5、MSTFI 和 L3-TPA/VBA 的部分研究中观察到了积极的预测有效性。所有的虚弱工具都存在最低或最高效应。结论:现有的脊柱肿瘤外科虚弱和肌肉疏松症评估工具的临床测量属性较差。在此,我们提供了一种实用的方法来利用现有的虚弱和肌肉疏松工具,直到开发出临床测量性能更强的工具。
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引用次数: 0
P.076 Long-term outcomes of radiofrequency ablation for temporal lobe epilepsy P.076 射频消融治疗颞叶癫痫的长期疗效
R. Wang, A. Parrent, D. Steven, J. Burneo, A. Suller-Martí, JC Lau
Background: Radiofrequency ablation (RFA) is a minimally-invasive procedure that has been used to treat temporal lobe epilepsy (TLE), however its long-term efficacy is unknown. We aim to characterize the long-term outcomes of patients from the original series by Parrent and Blume (1999). Methods: Consecutive patients who underwent stereotactic RFA for TLE were retrospectively reviewed. Demographics, procedural details, and seizure outcomes until last follow-up were abstracted. Seizure-freedom after initial RFA treatment was estimated with Kaplan-Meier analysis. Results: 27 patients underwent RFA from 1994 to 2002. There were 14 female (52%) patients. 24 (89%) had mesial temporal sclerosis. Mean age at time of RFA was 33.1 years (range 12-45 years). 17 (63%) patients underwent left-sided RFA. 15 (56%) patients had further interventions: 4 (15%) underwent only repeat RFA, 1 (4%) had repeat RFA and anterior temporal lobectomy (ATL), and 10 (37%) underwent subsequent ATL only. Mean follow-up was 9.0 years (range 0.5-22.7 years). At last follow-up, 16 (59%) patients were seizure-free: 5 (19%) received one RFA treatment and 11 (41%) underwent additional procedures. Conclusions: Based on the original series describing the technique, stereotactic RFA for TLE is a safe, minimally-invasive procedure. The role of stereotactic RFA in the treatment of TLE remains to be determined.
背景:射频消融术(RFA)是一种微创手术,已被用于治疗颞叶癫痫(TLE),但其长期疗效尚不清楚。我们的目的是了解 Parrent 和 Blume(1999 年)最初系列患者的长期疗效。方法:对接受立体定向 RFA 治疗 TLE 的连续患者进行回顾性研究。摘录了患者的人口统计学特征、手术细节以及直至最后一次随访的癫痫发作结果。通过 Kaplan-Meier 分析估计了首次 RFA 治疗后的癫痫发作自由度。结果:1994年至2002年期间,27名患者接受了RFA治疗。其中有 14 名女性患者(52%)。24人(89%)患有颞中叶硬化症。接受 RFA 时的平均年龄为 33.1 岁(12-45 岁不等)。17(63%)名患者接受了左侧 RFA。15(56%)名患者接受了进一步干预:4人(15%)仅接受了重复RFA治疗,1人(4%)接受了重复RFA治疗和前颞叶切除术(ATL),10人(37%)仅接受了后续的ATL治疗。平均随访时间为 9.0 年(0.5-22.7 年不等)。最后一次随访时,16 名(59%)患者无癫痫发作:5人(19%)接受了一次RFA治疗,11人(41%)接受了其他治疗。结论:根据描述该技术的原始系列,立体定向 RFA 治疗 TLE 是一种安全的微创手术。立体定向 RFA 在治疗 TLE 中的作用仍有待确定。
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引用次数: 0
P.012 A global, long-term, prospective, observational registry of patients with AQP4+ NMOSD treated with complement component 5 inhibitor therapies eculizumab or ravulizumab P.012 对接受补体成分 5 抑制剂疗法 eculizumab 或 ravulizumab 治疗的 AQP4+ NMOSD 患者进行全球、长期、前瞻性观察登记
S. Fam, L. Przybyl, T. Azad, Jn Stankowski, K. Moy, D. Rotstein
Background: The complement component 5 inhibitor therapies (C5ITs) eculizumab and ravulizumab have been approved or submitted for regulatory approval in several regions for AQP4+ NMOSD. Methods: This global, long-term, prospective, multicenter, observational registry will enroll adult patients with AQP4+ NMOSD being treated with eculizumab or ravulizumab and who have received ≥1 dose of eculizumab or ravulizumab within 4 or 12 weeks prior to enrollment, respectively. Inclusion criteria include available historical data on C5IT dosing since initiation and the number and types of relapses from 1 year prior to C5IT initiation through enrollment. The primary outcome is annualized relapse rate. Safety outcomes will include serious adverse events, meningococcal infections, and pregnancy, breastfeeding, and neonatal outcomes. Data will be collected prospectively for up to 5 years. Approximately 130 patients will be enrolled, with a maximum of around 200 patients in up to 10 countries globally. Results: N/A Conclusions: This registry will collect data to characterize the long-term effectiveness and safety of the C5ITs eculizumab and ravulizumab in patients with AQP4+ NMOSD to provide evidence on the real-world impact of C5ITs in this patient population.
背景:补体成分 5 抑制剂疗法(C5ITs)eculizumab 和 ravulizumab 已在多个地区获批或提交监管审批,用于治疗 AQP4+ NMOSD。研究方法这项全球性、长期、前瞻性、多中心、观察性登记将纳入正在接受依库珠单抗或雷武利珠单抗治疗的 AQP4+ NMOSD 成年患者,这些患者在入组前 4 周或 12 周内分别接受过≥1 次依库珠单抗或雷武利珠单抗治疗。纳入标准包括自开始使用C5IT以来的可用历史数据,以及从开始使用C5IT前1年到入组前的复发次数和类型。主要结果为年复发率。安全性结果将包括严重不良事件、脑膜炎球菌感染以及妊娠、哺乳和新生儿结局。数据将进行长达 5 年的前瞻性收集。将在全球多达 10 个国家招募约 130 名患者,最多招募约 200 名患者。结果:不适用 结论:该登记处将收集数据,以确定 C5ITs eculizumab 和 ravulizumab 在 AQP4+ NMOSD 患者中的长期有效性和安全性,从而为 C5ITs 在该患者群体中的实际影响提供证据。
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引用次数: 0
C.3 Retrospective study of sulthiame in treatment of pediatric epilepsy C.3 苏合香治疗小儿癫痫的回顾性研究
A. Laliberté, KA Myers
Background: This retrospective study assessed the efficacy and tolerability of sulthiame as a treatment in children with epilepsy. In Canada, sulthiame is only available through Health Canada’s Special Access Program. Methods: Patients who received sulthiame at the Montreal Children’s Hospital from April 2012 to March 2023 were included. Patients’ medical charts were reviewed, and clinical data was extracted from neurology clinic notes and electroencephalogram (EEG) reports. Efficacy was assessed by comparing seizure frequency and frequency of EEG epileptiform abnormalities before and after initiating sulthiame, while also noting any reported changes in cognition or behaviour. Results: Sixteen patients were included (10 males, 6 females), all of whom had drug-resistant epilepsy and continuous spike-wave in sleep (CSWS) on EEG. Sulthiame starting dose ranged from 0.74 to 6.75 mg/kg/day. Improvement, either in terms of seizure control, cognition, or reduction in EEG epileptiform abnormalities, was reported in 8/16 children (50%). Two patients (13%) became seizure free, while three more (19%) had reduced seizure frequency. Three other patients (19%) had reported improvements in concentration, learning abilities or behaviour. No serious adverse event was reported. Conclusions: These data indicate that sulthiame is effective and well-tolerated in children with CSWS, regardless of the etiology and type of epilepsy.
研究背景这项回顾性研究评估了舒思安(sulthiame)治疗儿童癫痫的疗效和耐受性。在加拿大,舒思安只能通过加拿大卫生部的 "特别获取计划 "获得。研究方法纳入2012年4月至2023年3月期间在蒙特利尔儿童医院接受舒思安治疗的患者。研究人员查阅了患者的病历,并从神经科门诊记录和脑电图(EEG)报告中提取了临床数据。评估疗效的方法是比较开始使用舒思安前后的癫痫发作频率和脑电图癫痫样异常的频率,同时记录患者报告的认知或行为方面的任何变化。结果共纳入 16 名患者(10 名男性,6 名女性),他们都患有耐药性癫痫,脑电图显示睡眠中出现连续性棘波(CSWS)。舒思安的起始剂量为 0.74 至 6.75 毫克/千克/天。据报告,8/16 名患儿(50%)在癫痫发作控制、认知能力或脑电图癫痫样异常减少方面有所改善。两名患者(13%)癫痫不再发作,另有三名患者(19%)癫痫发作频率降低。另有三名患者(19%)报告注意力、学习能力或行为有所改善。无严重不良事件报告。结论这些数据表明,无论癫痫的病因和类型如何,舒利迭对CSWS患儿均有效且耐受性良好。
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Canadian Journal of Neurological Sciences / Journal Canadien des Sciences Neurologiques
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