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GR.3 Distinct longitudinal brain atrophy trajectories in parkinson’s disease clinical subtypes: insight towards precision medicine GR.3 帕金森病临床亚型的不同纵向脑萎缩轨迹:对精准医疗的启示
S Fereshtehnejad, R Moqadam, R Postuma, M Dadar, A Lang, Y Zeighami
Background: Parkinson’s disease (PD) varies widely across individuals in terms of clinical manifestations and course of progression. We aimed to compare patterns of brain atrophy between PD clinical subtypes using longitudinally acquired brain MRIs. Methods: We used T1-weighted MRIs from Parkinson’s Progression Markers Initiative (PPMI) on 134 PD individuals and 60 healthy controls with at least two MRIs. Patients were classified into three clinical subtypes at de novo stage using validated subtyping criteria based on major motor and non-motor classifiers (early cognitive impairment, RBD, dysautonomia): mild-motor predominant (n=74), intermediate (n=44), and diffuse-malignant (n=16). Deformation-based morphometry (DBM) maps were calculated and mixed effect models were used to examine the interaction between PD subtypes and rate of atrophy across brain regions over time, controlling for sex and age at baseline. Results: Individuals with ‘diffuse malignant’ PD showed a significantly higher rate of atrophy across multiple brain regions, including lateral nucleus of the forebrain, precuneus, paracentral lobule, inferior temporal gyrus, fusiform gyrus, and lateral hemisphere of the cerebellum (FDR corrected p<0.05). Conclusions: We demonstrated an accelerated atrophy pattern within several brain regions in ‘diffuse malignant’ PD subtype. These findings suggest the presence of a more diffuse multidomain neurodegenerative process in a subgroup of people with PD, favoring the existence of diverse underlying pathophysiologies.
背景:帕金森病(PD)的临床表现和进展过程因人而异。我们旨在利用纵向获得的脑磁共振成像比较帕金森病临床亚型之间的脑萎缩模式。研究方法我们使用帕金森病进展标志物倡议(PPMI)中的 T1 加权 MRI,对 134 名帕金森病患者和 60 名健康对照者进行了至少两次 MRI 检查。利用基于主要运动和非运动分类器(早期认知障碍、RBD、自主神经功能障碍)的有效亚型标准,将患者在新发期分为三种临床亚型:轻度运动主导型(74人)、中度(44人)和弥漫恶性型(16人)。计算基于形变的形态测量(DBM)图,并使用混合效应模型研究帕金森病亚型与脑区萎缩率随时间变化的交互作用,同时控制基线时的性别和年龄。研究结果弥漫性恶性 "帕金森氏症患者在多个脑区的萎缩率明显较高,包括前脑外侧核、楔前叶、旁中心小叶、颞下回、纺锤形回和小脑外侧半球(FDR校正后P<0.05)。结论我们发现 "弥漫性恶性 "帕金森病亚型的多个脑区存在加速萎缩模式。这些研究结果表明,在帕金森氏症亚型患者中存在一种更为弥漫的多域神经退行性过程,这有利于多种潜在病理生理机制的存在。
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引用次数: 0
C.2 An in-depth analysis of pediatric inflammatory myopathies: findings from a comprehensive tertiary care hospital C.2 深入分析儿科炎症性肌病:一家综合性三级医院的研究结果
E. Mostofi, H. McMillan, R. Jurencak, L. Hamilton, A. Yaworski
Background: Pediatric inflammatory myopathies (PIM) are a rare, heterogenous group of disorders requiring prompt diagnosis and treatment to reduce complications and improve long-term outcome. This study reviews the clinical characteristics, management, and outcomes in PIM. Methods: A retrospective analysis of pediatric patients diagnosed with PIM at CHEO from January 2009 to December 2023 was performed. Patient data, including age at symptom onset, diagnostic testing performed, treatment, and follow-up durations, were evaluated. Results: A total of 25 patients with juvenile dermatomyositis (JDM), overlap syndromes, and necrotizing myopathy (HMG-CoA reductase and anti-SRP myositis) were identified. Symptoms began at an average age of 8.37 years (1.10-14.11), with formal diagnosis occurring at 8.57 years (2.02-16.11). Initial symptoms included skin changes, muscle weakness, joint pain, and fatigue. Diagnosis involved laboratory testing (CK, myositis antibodies), muscle MRI, electromyography, and/or muscle biopsy. Treatments included corticosteroids, IVIG, and steroid-sparing agents (methotrexate, mycophenolate mofetil, rituximab, hydroxychloroquine). Follow-up averaged 4.23 years (range: 0.5 to 13). Most patients displayed only mild residual symptoms with the exception of an anti-SRP myositis patient who became wheelchair-dependent, requiring ventilatory support. Conclusions: Inflammatory myopathies require prompt treatment to prevent complications. Most patients require multiple treatment modalities, however with early diagnosis and treatment the majority of patients’ symptoms resolve.
背景:小儿炎症性肌病(PIM)是一种罕见的异质性疾病,需要及时诊断和治疗,以减少并发症并改善长期预后。本研究回顾了小儿炎症性肌病的临床特点、管理和预后。方法:对 2009 年 1 月至 2023 年 12 月期间在 CHEO 诊断为 PIM 的儿科患者进行回顾性分析。对患者的数据进行了评估,包括发病年龄、所进行的诊断测试、治疗和随访时间。结果:共发现25名幼年皮肌炎(JDM)、重叠综合征和坏死性肌病(HMG-CoA还原酶和抗SRP肌炎)患者。患者平均 8.37 岁(1.10-14.11 岁)开始出现症状,8.57 岁(2.02-16.11 岁)正式确诊。最初的症状包括皮肤变化、肌肉无力、关节疼痛和疲劳。诊断涉及实验室检测(肌酸激酶、肌炎抗体)、肌肉核磁共振成像、肌电图和/或肌肉活检。治疗方法包括皮质类固醇、IVIG 和类固醇替代药物(甲氨蝶呤、霉酚酸酯、利妥昔单抗、羟氯喹)。随访时间平均为 4.23 年(范围:0.5 至 13 年)。大多数患者仅表现出轻微的残留症状,只有一名抗SRP肌炎患者例外,该患者变得需要依赖轮椅,需要呼吸机支持。结论炎症性肌病需要及时治疗以预防并发症。大多数患者需要多种治疗方法,但只要早期诊断和治疗,大多数患者的症状都能得到缓解。
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引用次数: 0
P.116 Days at home after traumatic brain injury: moving beyond mortality to evaluate patient-centered outcomes using population health data P.116 脑外伤后在家的日子:超越死亡率,利用人口健康数据评估以患者为中心的结果
AK Malhotra, AB Nathens, H. Shakil, RH Jaffe, A. Essa, F. Mathieu, J. Badhiwala, K. Thorpe, AV Kulkarni, CD Witiw, JR Wilson
Background: Despite the utility of administrative health data, there remains a lack of patient-centered outcome measures to meaningfully capture morbidity after traumatic brain injury (TBI). We sought to characterize and validate days at home (DAH) as a feasible measure to assess population-level moderate to severe TBI (msTBI) outcomes and health resource utilization. Methods: We utilized linked health administrative data sources to identify adults with msTBI patients presenting to trauma centers in Ontario injured between 2009-2021. DAH at 180 days reflects the total number of days spent alive and at home excluding the days spent institutionalized in acute care, rehabilitation, inpatient mental health settings or post-acute readmissions. Construct and predictive validity were determined; we additionally estimated minimally important difference (MID) in DAH180days. Results: There were 6340 patients that met inclusion criteria. Median DAH180days were 70 days (interquartile range 0-144). Increased health resource utilization at baseline, older age, increasing cranial injury severity and major extracranial injuries were significantly associated with fewer DAH180days. DAH180days was correlated to DAH counts at 1-3 years. The average MID estimate from anchor-based and distribution-based methods was 18 days. Conclusions: We introduce DAH180days as a feasible and sufficiently responsive patient-centered outcome measure with construct, predictive and face validity in an msTBI population.
背景:尽管健康管理数据非常有用,但仍然缺乏以患者为中心的结果测量方法来有意义地反映创伤性脑损伤(TBI)后的发病率。我们试图对在家天数(DAH)进行特征描述和验证,将其作为评估人群中度至重度创伤性脑损伤(msTBI)结果和医疗资源利用率的可行指标。方法:我们利用关联的健康管理数据源来识别 2009-2021 年间前往安大略省创伤中心就诊的中重度创伤性脑损伤(msTBI)成人患者。180 天的 DAH 反映了患者在家中存活的总天数,但不包括在急症护理、康复、住院精神疾病治疗机构或急性期后再入院治疗的天数。我们还估算了DAH180天的最小重要差异(MID)。结果共有 6340 名患者符合纳入标准。DAH180天的中位数为70天(四分位距为0-144)。基线医疗资源利用率增加、年龄增大、颅脑损伤严重程度增加和主要颅外损伤与 DAH180 天数减少显著相关。DAH180天与1-3年的DAH计数相关。基于锚的方法和基于分布的方法估计的平均 MID 为 18 天。结论:我们介绍的 DAH180 天是一种以患者为中心的可行结果测量方法,在毫秒创伤性脑损伤人群中具有构造、预测和表面有效性。
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引用次数: 0
P.029 Endovascular therapy for cerebral venous thrombosis: an international survey P.029 脑静脉血栓的血管内治疗:一项国际调查
BA Brakel, A. Rebchuk, J. Ospel, M. Heran, M. Goyal, M. Hill, Z. Miao, Y Chen, S. Sacco, S. Yaghi, M. Ton, G. Thomalla, G. Boulouis, H. Yamagami, BC Campbell, J. Raymond, G. Saposnik, TN Nguyen, TS Field
Background: Cerebral venous thrombosis (CVT) is a rare cause of stroke, with 10–15% of patients experiencing dependence or death. The role of endovascular therapy (EVT) in the management of CVT remains controversial and practice patterns are not well-known. Methods: We distributed a comprehensive 53-question survey to neurologists, neuro-interventionalists, neurosurgeons and other relevant clinicians globally from May 2023 to October 2023. The survey asked about practice patterns and perspectives on EVT for CVT and assessed opinions regarding future clinical trials. Results: The overall response rate was 31% (863 respondents from 2744 invited participants) across 61 countries. A majority (74%) supported use of EVT for certain CVT cases. Key considerations for EVT included worsening level of consciousness (86%) and other clinical deficits (76%). Mechanical thrombectomy with aspiration (22%) and stent retriever (19%) were the most utilized techniques, with regional variations. Post-procedurally, low molecular weight heparin was the predominant anticoagulant administered (40%), although North American respondents favored unfractionated heparin. Most respondents supported future trials of EVT (90%). Conclusions: Our survey reveals significant heterogeneity in approaches to EVT for CVT, highlighting the necessity for adequately powered clinical trials to guide standard-of-care practices.
背景:脑静脉血栓(CVT)是中风的罕见病因,10%-15% 的患者会出现依赖性或死亡。血管内治疗(EVT)在治疗 CVT 中的作用仍存在争议,实践模式也不为人所知。方法:我们在 2023 年 5 月至 2023 年 10 月期间向全球的神经科医生、神经介入医生、神经外科医生和其他相关临床医生发放了一份包含 53 个问题的综合调查问卷。调查询问了有关 EVT 治疗 CVT 的实践模式和观点,并评估了有关未来临床试验的意见。调查结果显示61个国家的总回复率为31%(2744名受邀参与者中的863名回复者)。大多数人(74%)支持在某些 CVT 病例中使用 EVT。EVT的主要考虑因素包括意识水平恶化(86%)和其他临床缺陷(76%)。抽吸式机械血栓切除术(22%)和支架回取术(19%)是最常用的技术,但地区间存在差异。术后使用的抗凝剂主要是低分子量肝素(40%),但北美的受访者更倾向于使用非分叶肝素。大多数受访者支持未来进行 EVT 试验(90%)。结论:我们的调查显示,CVT 的 EVT 治疗方法存在明显的异质性,这凸显了进行充分有效的临床试验以指导标准护理实践的必要性。
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引用次数: 0
P.103 Automated pituitary adenoma segmentation for radiosurgery with deep learning-based model P.103 利用基于深度学习的模型为放射外科手术自动分割垂体腺瘤
N. Balasubramaniam, M. Cerny, J. May, L. Hamackova, J. Novotnyml, D. Barucic, J. Kybic, M. Majovsky, H. Hallak, R. Liscak, D. Netuka
Background: Pituitary adenomas are treated with endoscopic surgery, while stereotactic radiosurgery addresses complex cases. Our study highlights AI’s role in accurate segmentation, improving treatment planning workflow efficiency Methods: In a retrospective study at Na Homolce Hospital (January 2010 to October 2022), SRS for pituitary adenomas was analyzed. Data were split 80:20 for training and validation. Using nnU-net, a medical image segmentation tool, a model predicted precise tumor, optic nerve, and pituitary gland segmentation. Accuracy was evaluated quantitatively with Dice similarity coefficient and qualitatively by human experts. The study explored the impact of tumor volume and hormonal activity status on segmentation accuracy. Results: The study comprised 582 and 146 patients in training and validation sets, respectively. The model achieved Dice similarity coefficients of 83.1% (tumor), 62.9% (normal gland), and 78.0% (optic nerve). Expert assessments deemed 41% directly applicable, 31.5% needing minor adjustments, and 27.4% unsuitable for clinical use. Larger tumor volume and non-functioning adenomas correlated with higher accuracy. Including T2 weighted scans improved DSC for optic nerve and normal gland. Conclusions: The study showcases deep learning’s potential in automating pituitary adenoma segmentation from MRI data, particularly excelling in large, hormonally inactive macroadenomas. Encourages collaborative use with clinicians for improved neurosurgical patient care.
背景:垂体腺瘤可通过内窥镜手术治疗,而立体定向放射外科手术则可治疗复杂病例。我们的研究强调了人工智能在准确分割、提高治疗计划工作流程效率方面的作用:在Na Homolce医院进行的一项回顾性研究(2010年1月至2022年10月)中,对垂体腺瘤的SRS进行了分析。数据按 80:20 的比例分成训练和验证两部分。利用医学影像分割工具 nnU-net,一个模型预测了精确的肿瘤、视神经和垂体分割。用 Dice 相似性系数对准确性进行了定量评估,并由人类专家对准确性进行了定性评估。研究还探讨了肿瘤体积和激素活动状态对分割准确性的影响。研究结果研究分别将 582 名和 146 名患者纳入训练集和验证集。模型的 Dice 相似系数分别为 83.1%(肿瘤)、62.9%(正常腺体)和 78.0%(视神经)。专家评估认为,41%的模型可直接用于临床,31.5%的模型需要稍作调整,27.4%的模型不适合临床使用。较大的肿瘤体积和无功能腺瘤与较高的准确性相关。纳入T2加权扫描可提高视神经和正常腺体的DSC。结论该研究展示了深度学习在从磁共振成像数据中自动分割垂体腺瘤方面的潜力,尤其是在大体积、激素不活跃的大腺瘤方面表现出色。鼓励与临床医生合作使用,以改善神经外科患者的护理。
{"title":"P.103 Automated pituitary adenoma segmentation for radiosurgery with deep learning-based model","authors":"N. Balasubramaniam, M. Cerny, J. May, L. Hamackova, J. Novotnyml, D. Barucic, J. Kybic, M. Majovsky, H. Hallak, R. Liscak, D. Netuka","doi":"10.1017/cjn.2024.206","DOIUrl":"https://doi.org/10.1017/cjn.2024.206","url":null,"abstract":"Background: Pituitary adenomas are treated with endoscopic surgery, while stereotactic radiosurgery addresses complex cases. Our study highlights AI’s role in accurate segmentation, improving treatment planning workflow efficiency Methods: In a retrospective study at Na Homolce Hospital (January 2010 to October 2022), SRS for pituitary adenomas was analyzed. Data were split 80:20 for training and validation. Using nnU-net, a medical image segmentation tool, a model predicted precise tumor, optic nerve, and pituitary gland segmentation. Accuracy was evaluated quantitatively with Dice similarity coefficient and qualitatively by human experts. The study explored the impact of tumor volume and hormonal activity status on segmentation accuracy. Results: The study comprised 582 and 146 patients in training and validation sets, respectively. The model achieved Dice similarity coefficients of 83.1% (tumor), 62.9% (normal gland), and 78.0% (optic nerve). Expert assessments deemed 41% directly applicable, 31.5% needing minor adjustments, and 27.4% unsuitable for clinical use. Larger tumor volume and non-functioning adenomas correlated with higher accuracy. Including T2 weighted scans improved DSC for optic nerve and normal gland. Conclusions: The study showcases deep learning’s potential in automating pituitary adenoma segmentation from MRI data, particularly excelling in large, hormonally inactive macroadenomas. Encourages collaborative use with clinicians for improved neurosurgical patient care.","PeriodicalId":9571,"journal":{"name":"Canadian Journal of Neurological Sciences / Journal Canadien des Sciences Neurologiques","volume":"55 2","pages":""},"PeriodicalIF":0.0,"publicationDate":"2024-05-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141102360","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
B.1 CT Or MRI protocol for acute stroke reperfusion with EVT (COMPARE): an international retrospective cohort study B.1 使用 EVT 进行急性脑卒中再灌注的 CT 或 MRI 方案(COMPARE):一项国际回顾性队列研究
C Ducroux, W Boisseau, R Fahed, G Stotts, M Kyheng, J Labreuche, D. Roy, B Lapergue, A. Poppe
Background: Patients with an acute ischemic stroke (AIS) are selected to receive reperfusion therapy using either computed tomography (CT-CTA) or magnetic brain imaging (MRI). The aim of this study was to compare CT and MRI as the primary imaging modality for AIS patients undergoing EVT. Methods: Data for AIS patients between January 2018 and January 2021 were extracted from two prospective multicenter EVT cohorts: the ETIS registry in France (MRI) and the OPTIMISE registry in Canada (CT). Demographics, procedural data and outcomes were collected. We assessed the association of qualifying imaging (CT vs. MRI) with time metrics and functional outcome. Results: From January 2018 to January 2021, 4059 patients selected by MRI and 1324 patients selected by CT were included in the study. Demographics were similar between the two groups. The median imaging-to-arterial puncture time was 37 minutes longer in the MRI group. Patients selected by CT had more favorable 90-day functional outcomes (mRS 0-2) as compared to patients selected by MRI (48.5% vs 44.4%; adjusted OR (aOR), 1.54, 95%CI 1.31 to 1.80, p<0.001). Conclusions: Patients with AIS undergoing EVT who were selected with MRI as opposed to CT had longer imaging-to-arterial-puncture delays and worse functional outcomes at 90 days.
背景:急性缺血性脑卒中(AIS)患者可通过计算机断层扫描(CT-CTA)或脑磁成像(MRI)选择接受再灌注治疗。本研究旨在比较 CT 和 MRI 作为接受 EVT 的 AIS 患者的主要成像方式。方法:从两个前瞻性多中心EVT队列中提取了2018年1月至2021年1月期间AIS患者的数据:法国的ETIS登记(MRI)和加拿大的OPTIMISE登记(CT)。我们收集了人口统计学、手术数据和结果。我们评估了合格成像(CT 与 MRI)与时间指标和功能结果的关联。结果:从 2018 年 1 月到 2021 年 1 月,4059 名通过 MRI 筛选的患者和 1324 名通过 CT 筛选的患者被纳入研究。两组患者的人口统计学特征相似。核磁共振成像组从成像到动脉穿刺的中位时间比CT组长37分钟。与 MRI 选择的患者相比,CT 选择的患者 90 天功能预后(mRS 0-2)更佳(48.5% vs 44.4%;调整 OR (aOR),1.54,95%CI 1.31 至 1.80,p<0.001)。结论接受EVT治疗的AIS患者如果是通过MRI而非CT选择的,那么成像到动脉穿刺的延迟时间更长,90天后的功能预后更差。
{"title":"B.1 CT Or MRI protocol for acute stroke reperfusion with EVT (COMPARE): an international retrospective cohort study","authors":"C Ducroux, W Boisseau, R Fahed, G Stotts, M Kyheng, J Labreuche, D. Roy, B Lapergue, A. Poppe","doi":"10.1017/cjn.2024.80","DOIUrl":"https://doi.org/10.1017/cjn.2024.80","url":null,"abstract":"Background: Patients with an acute ischemic stroke (AIS) are selected to receive reperfusion therapy using either computed tomography (CT-CTA) or magnetic brain imaging (MRI). The aim of this study was to compare CT and MRI as the primary imaging modality for AIS patients undergoing EVT. Methods: Data for AIS patients between January 2018 and January 2021 were extracted from two prospective multicenter EVT cohorts: the ETIS registry in France (MRI) and the OPTIMISE registry in Canada (CT). Demographics, procedural data and outcomes were collected. We assessed the association of qualifying imaging (CT vs. MRI) with time metrics and functional outcome. Results: From January 2018 to January 2021, 4059 patients selected by MRI and 1324 patients selected by CT were included in the study. Demographics were similar between the two groups. The median imaging-to-arterial puncture time was 37 minutes longer in the MRI group. Patients selected by CT had more favorable 90-day functional outcomes (mRS 0-2) as compared to patients selected by MRI (48.5% vs 44.4%; adjusted OR (aOR), 1.54, 95%CI 1.31 to 1.80, p<0.001). Conclusions: Patients with AIS undergoing EVT who were selected with MRI as opposed to CT had longer imaging-to-arterial-puncture delays and worse functional outcomes at 90 days.","PeriodicalId":9571,"journal":{"name":"Canadian Journal of Neurological Sciences / Journal Canadien des Sciences Neurologiques","volume":"56 2","pages":""},"PeriodicalIF":0.0,"publicationDate":"2024-05-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141102204","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.126 An unlikely impersonator of primary brain tumours: Illustrative case report and literature review of primary angiitis of the central nervous system P.126 原发性脑肿瘤的假冒者:中枢神经系统原发性血管炎的说明性病例报告和文献综述
MW Elder, K. Chornenka, S. Marzoughi, MF Hassanabad, M. Rizzuto
Background: Primary angiitis of the central nervous system (PACNS) is a rare inflammatory condition affecting the parenchymal and leptomeningeal vessels of the CNS. PACNS presenting as a solitary mass lesion (ML-PACNS) constitutes a rare subtype of this pathology. Herein we present the first case reported in Canada of ML-PACNS, presenting with clinical and radiographic findings consistent with a high grade glial neoplasm, as well as a review of the literature on ML-PACNS. Methods: Review of the literature from 1987-2023 was conducted using PubMed to identify features of ML-PACNS and possible treatment paradigms. Results: A number of case reports of ML-PACNS were identified, as well as 6 retrospective analyses of a total of 67 patients. Features such as faster rate of symptom onset, and investigations such as MRI vessel-wall imaging and MR spectroscopy were suggested for identification of ML-PACNS. Treatment was highly variable, but followed guidelines for other neuroinflammatory disorders. Conclusions: Preoperative differentiation between ML-PACNS and CNS neoplasms is difficult due to their similar clinical and radiographic features. However, making this distinction is crucial as PACNS mass lesions can regress entirely with immunosuppressive therapy, potentially obviating the requirement for surgical intervention. Beyond diagnostics, further research is required to establish and validate a treatment paradigm.
背景:中枢神经系统原发性血管炎(PACNS)是一种影响中枢神经系统实质血管和脑膜血管的罕见炎症。以单发肿块病变(ML-PACNS)形式出现的原发性中枢神经系统血管炎是该病症的一种罕见亚型。在此,我们介绍了加拿大报告的首例 ML-PACNS 病例,该病例的临床和影像学表现与高级别胶质肿瘤一致,我们还回顾了有关 ML-PACNS 的文献。研究方法使用PubMed对1987-2023年的文献进行回顾,以确定ML-PACNS的特征和可能的治疗模式。结果:研究发现了多篇关于 ML-PACNS 的病例报告,以及对 67 名患者进行的 6 项回顾性分析。研究人员提出了一些识别 ML-PACNS 的特征,如症状出现的速度较快,以及核磁共振血管壁成像和核磁共振波谱成像等检查方法。治疗方法千差万别,但都遵循其他神经炎症性疾病的治疗指南。结论由于 ML-PACNS 和中枢神经系统肿瘤具有相似的临床和影像学特征,因此术前很难将两者区分开来。然而,由于 PACNS 肿块病变在接受免疫抑制治疗后可完全消退,从而有可能避免手术干预的要求,因此这种区分至关重要。除了诊断,还需要进一步的研究来建立和验证治疗模式。
{"title":"P.126 An unlikely impersonator of primary brain tumours: Illustrative case report and literature review of primary angiitis of the central nervous system","authors":"MW Elder, K. Chornenka, S. Marzoughi, MF Hassanabad, M. Rizzuto","doi":"10.1017/cjn.2024.227","DOIUrl":"https://doi.org/10.1017/cjn.2024.227","url":null,"abstract":"Background: Primary angiitis of the central nervous system (PACNS) is a rare inflammatory condition affecting the parenchymal and leptomeningeal vessels of the CNS. PACNS presenting as a solitary mass lesion (ML-PACNS) constitutes a rare subtype of this pathology. Herein we present the first case reported in Canada of ML-PACNS, presenting with clinical and radiographic findings consistent with a high grade glial neoplasm, as well as a review of the literature on ML-PACNS. Methods: Review of the literature from 1987-2023 was conducted using PubMed to identify features of ML-PACNS and possible treatment paradigms. Results: A number of case reports of ML-PACNS were identified, as well as 6 retrospective analyses of a total of 67 patients. Features such as faster rate of symptom onset, and investigations such as MRI vessel-wall imaging and MR spectroscopy were suggested for identification of ML-PACNS. Treatment was highly variable, but followed guidelines for other neuroinflammatory disorders. Conclusions: Preoperative differentiation between ML-PACNS and CNS neoplasms is difficult due to their similar clinical and radiographic features. However, making this distinction is crucial as PACNS mass lesions can regress entirely with immunosuppressive therapy, potentially obviating the requirement for surgical intervention. Beyond diagnostics, further research is required to establish and validate a treatment paradigm.","PeriodicalId":9571,"journal":{"name":"Canadian Journal of Neurological Sciences / Journal Canadien des Sciences Neurologiques","volume":"48 11","pages":""},"PeriodicalIF":0.0,"publicationDate":"2024-05-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141102292","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.119 NIRS regional oxygen saturation based cerebrovascular reactivity in the recovery from moderate/severe TBI P.119 中度/重度创伤性脑损伤恢复过程中基于区域血氧饱和度的近红外光谱脑血管反应能力
A Gomez, L. Froese, T Bergmann, AS Sainbhi, N Vakitbilir, A Islam, KY Stein, I Marquez, Y Ibrahim, F. Zeiler
Background: Near-infrared spectroscopy (NIRS) regional cerebral oxygen saturation (rSO2) based cerebrovascular reactivity (CVR) indicies have enable the entirely non-invasive continuous monitoring. This study aims to compare CVR in those recovering from moderate/severe TBI to a health control group. Methods: In this prospective cohort study the cerebral oxygen CVR index, COx_a (using rSO2 and arterial blood pressure), was measured in subjects with moderate/severe TBI at follow-up. COx_a was also measured in a group of healthy controls. CVR was compared within and between these groups using conventional statistics. Results: A total of 101 heathy subject were recruited for this study along with 29 TBI patients. In the health cohort COx_a was not statistically different between males and females or in the dominate and non-dominate hemisphere. The TBI cohort, COx_a was not statistically different between first and last available follow up. Surprisingly, CVR as measured by COx_a was statistically better in those recovering from TBI than in the healthy cohort. Conclusions: In the prospective cohort study, CVR as measured by NIRS based methods, was found to be more active in those recovering from TBI than in a healthy cohort. This study may indicate that, in those that survive TBI, CVR may be enhanced as a neuroprotective measure.
背景:基于近红外光谱(NIRS)区域脑氧饱和度(rSO2)的脑血管反应性(CVR)指标实现了完全无创的连续监测。本研究旨在比较中度/重度创伤性脑损伤康复者与健康对照组的 CVR。方法:在这项前瞻性队列研究中,对中度/重度创伤性脑损伤患者进行了随访,测量了脑氧CVR指数COx_a(使用rSO2和动脉血压)。同时还测量了一组健康对照组的 COx_a。采用常规统计方法对这些组内和组间的 CVR 进行了比较。结果:本研究共招募了 101 名健康受试者和 29 名创伤性脑损伤患者。在健康组中,COx_a 在男性和女性之间、优势半球和非优势半球之间没有统计学差异。在 TBI 群体中,COx_a 在第一次和最后一次随访之间没有统计学差异。令人惊讶的是,根据 COx_a 测量的心血管恢复能力,创伤性脑损伤康复者在统计学上优于健康人群。结论:在前瞻性队列研究中,通过基于近红外光谱的方法测量发现,创伤性脑损伤康复者的 CVR 比健康人群更活跃。这项研究可能表明,在创伤性脑损伤幸存者中,CVR 作为一种神经保护措施可能会得到加强。
{"title":"P.119 NIRS regional oxygen saturation based cerebrovascular reactivity in the recovery from moderate/severe TBI","authors":"A Gomez, L. Froese, T Bergmann, AS Sainbhi, N Vakitbilir, A Islam, KY Stein, I Marquez, Y Ibrahim, F. Zeiler","doi":"10.1017/cjn.2024.221","DOIUrl":"https://doi.org/10.1017/cjn.2024.221","url":null,"abstract":"Background: Near-infrared spectroscopy (NIRS) regional cerebral oxygen saturation (rSO2) based cerebrovascular reactivity (CVR) indicies have enable the entirely non-invasive continuous monitoring. This study aims to compare CVR in those recovering from moderate/severe TBI to a health control group. Methods: In this prospective cohort study the cerebral oxygen CVR index, COx_a (using rSO2 and arterial blood pressure), was measured in subjects with moderate/severe TBI at follow-up. COx_a was also measured in a group of healthy controls. CVR was compared within and between these groups using conventional statistics. Results: A total of 101 heathy subject were recruited for this study along with 29 TBI patients. In the health cohort COx_a was not statistically different between males and females or in the dominate and non-dominate hemisphere. The TBI cohort, COx_a was not statistically different between first and last available follow up. Surprisingly, CVR as measured by COx_a was statistically better in those recovering from TBI than in the healthy cohort. Conclusions: In the prospective cohort study, CVR as measured by NIRS based methods, was found to be more active in those recovering from TBI than in a healthy cohort. This study may indicate that, in those that survive TBI, CVR may be enhanced as a neuroprotective measure.","PeriodicalId":9571,"journal":{"name":"Canadian Journal of Neurological Sciences / Journal Canadien des Sciences Neurologiques","volume":"1 10","pages":""},"PeriodicalIF":0.0,"publicationDate":"2024-05-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141099380","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.109 Machine learning based approach to improving the prediction of neurological deterioration in mild Degenerative Cervical Myelopathy P.109 基于机器学习的方法改进对轻度颈椎退行性病变神经功能恶化的预测
A. Al-Shawwa, M. Craig, K. Ost, S. Tripathy, D. Cadotte
Background: Degenerative cervical myelopathy (DCM) is the most common form of atraumatic spinal cord injury globally, yet clinical guidelines remain unclear on surgical recommendations for patients with mild forms of DCM. This is in part due to limitations in current MR imaging interpretation and complex mechanisms of neurological deterioration. Supervised machine learning (ML) models can help to identify clinical and imaging indicators of deterioration within mild DCM patients. Methods: 127 MRI scans (T2w, Diffusion Tensor Imaging, and Magnetization transfer scans) accompanied by a series of clinical tests underwent a semi-automated analysis to derive quantitative metrics. Random forest classifier, Support Vector Machine, and Logistic Regression models were trained and tested to predict 6-month neurological deterioration within patients. Results: The ML models performed, on average, better than previous studies with a balanced accuracy ranging between 70-75%. “Advanced” imaging metrics such as diffusion tensor imaging and magnetization transfer scans played an important role in improving model accuracy but only when used near the maximally compressed disc level, suggesting that limited yet targetted imaging metrics support ML model performance. Conclusions: The inclusion of specific, targeted imaging and clinical metrics support ML model performance in predicting neurological deterioration within mild DCM patients.
背景:退行性颈椎脊髓病(DCM)是全球最常见的创伤性脊髓损伤,但临床指南对轻度 DCM 患者的手术建议仍不明确。这部分是由于目前磁共振成像解读的局限性和神经功能衰退的复杂机制造成的。有监督的机器学习(ML)模型有助于识别轻度 DCM 患者病情恶化的临床和影像学指标。方法:127 例核磁共振成像扫描(T2w、弥散张量成像和磁化转移扫描)以及一系列临床测试经过半自动化分析,得出定量指标。对随机森林分类器、支持向量机和逻辑回归模型进行了训练和测试,以预测患者 6 个月后的神经功能恶化情况。结果显示ML 模型的平均准确率在 70-75% 之间,表现优于以往的研究。弥散张量成像和磁化转移扫描等 "高级 "成像指标在提高模型准确性方面发挥了重要作用,但只有在最大压缩椎间盘水平附近使用时才能提高准确性。结论:纳入特定的、有针对性的成像和临床指标有助于提高 ML 模型在预测轻度 DCM 患者神经功能恶化方面的性能。
{"title":"P.109 Machine learning based approach to improving the prediction of neurological deterioration in mild Degenerative Cervical Myelopathy","authors":"A. Al-Shawwa, M. Craig, K. Ost, S. Tripathy, D. Cadotte","doi":"10.1017/cjn.2024.212","DOIUrl":"https://doi.org/10.1017/cjn.2024.212","url":null,"abstract":"Background: Degenerative cervical myelopathy (DCM) is the most common form of atraumatic spinal cord injury globally, yet clinical guidelines remain unclear on surgical recommendations for patients with mild forms of DCM. This is in part due to limitations in current MR imaging interpretation and complex mechanisms of neurological deterioration. Supervised machine learning (ML) models can help to identify clinical and imaging indicators of deterioration within mild DCM patients. Methods: 127 MRI scans (T2w, Diffusion Tensor Imaging, and Magnetization transfer scans) accompanied by a series of clinical tests underwent a semi-automated analysis to derive quantitative metrics. Random forest classifier, Support Vector Machine, and Logistic Regression models were trained and tested to predict 6-month neurological deterioration within patients. Results: The ML models performed, on average, better than previous studies with a balanced accuracy ranging between 70-75%. “Advanced” imaging metrics such as diffusion tensor imaging and magnetization transfer scans played an important role in improving model accuracy but only when used near the maximally compressed disc level, suggesting that limited yet targetted imaging metrics support ML model performance. Conclusions: The inclusion of specific, targeted imaging and clinical metrics support ML model performance in predicting neurological deterioration within mild DCM patients.","PeriodicalId":9571,"journal":{"name":"Canadian Journal of Neurological Sciences / Journal Canadien des Sciences Neurologiques","volume":"87 2","pages":""},"PeriodicalIF":0.0,"publicationDate":"2024-05-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141101527","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
A.1 Repurposing Ambroxol as a disease-modifying treatment for Parkinson’s disease dementia: A phase 2, randomized, double blind placebo-controlled trial A.1 将氨溴索重新用作帕金森病痴呆症的疾病调节疗法:2期随机双盲安慰剂对照试验
SH Pasternak, C. Silveira, K. Coleman, M. Borrie, J. Wells, E. Finger, R. Bartha, M. Jog, M. Jenkins, P. MacDonald, G. Zou, S. Stukas, C. Wellington, R. Tirona, T. Rupar
Background: Currently there are no disease modifying treatment for Synucleinopathies including Parkinson’s disease Dementia (PDD). Carrying a mutation in the GBA gene (beta-glucocerebrosidase/ GCAse) is a leading risk factor for synucleinopathies. Raising activity GCAse lowers α-synuclein levels in cells and animal models. Ambroxol is a pharmacological chaperone for GCAse and can raise GCAse levels. Our goal is to test Ambroxol as a disease-modifying treatment in PDD. Methods: We randomized fifty-five individuals with PDD to Ambroxol 1050mg/day, 525mg/day, or placebo for 52 weeks. Primary outcome measures included safety, Alzheimer’s disease Assessment Scale-cognitive (ADAS-Cog) subscale and the Clinician’s Global Impression of Change (CGIC). Secondary outcomes included pharmacokinetics, cognitive and motor outcomes and and plasma and CSF biomarkers. Results: Ambroxol was well tolerated. There were 7 serious adverse events (SAEs) none deemed related to Ambroxol. GCase activity was increased in white blood cells by ~1.5 fold. There were no differences between groups on primary outcome measures. Patients receiving high dose Ambroxol appeared better on the Neuropsychiatric Inventory. GBA carriers appeared to improve on some cognitive tests. pTau 181 was reduced in CSF. Conclusions: Ambroxol was safe and well-tolerated in PDD. Ambroxol may improve biomarkers and cognitive outcomes in GBA1 mutation carrie.rs Ambroxol improved some biomarkerss. ClinicalTrials.gov NCT02914366
背景:目前还没有针对包括帕金森病痴呆症(PDD)在内的突触核蛋白病的治疗方法。携带 GBA 基因(β-葡糖脑苷脂/GCAse)突变是突触核蛋白病的主要风险因素。提高 GCAse 的活性可降低细胞和动物模型中的α-突触核蛋白水平。氨溴索是 GCAse 的药理伴侣,可以提高 GCAse 的水平。我们的目标是测试氨溴索是否可作为治疗 PDD 的疾病调节剂。方法:我们将 55 名 PDD 患者随机分配到氨溴索 1050 毫克/天、525 毫克/天或安慰剂中,为期 52 周。主要结果指标包括安全性、阿尔茨海默病评估量表-认知(ADAS-Cog)分量表和临床医生总体变化印象(CGIC)。次要结果包括药代动力学、认知和运动结果以及血浆和脑脊液生物标志物。研究结果氨溴索耐受性良好。共发生了 7 起严重不良事件 (SAE),均与氨溴索无关。白细胞中的 GCase 活性增加了约 1.5 倍。各组在主要结果指标上没有差异。接受大剂量氨溴索治疗的患者在神经精神量表方面表现较好。脑脊液中的 pTau 181 有所降低。结论氨溴索对PDD患者安全且耐受性良好。氨溴索可改善GBA1突变携带者的生物标志物和认知结果。ClinicalTrials.gov NCT02914366
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Canadian Journal of Neurological Sciences / Journal Canadien des Sciences Neurologiques
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