Canadian Journal of Neurological Sciences / Journal Canadien des Sciences Neurologiques最新文献

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

Canadian Journal of Neurological Sciences / Journal Canadien des Sciences Neurologiques Pub Date : 2024-05-24 DOI: 10.1017/cjn.2024.70

S Fereshtehnejad, R Moqadam, R Postuma, M Dadar, A Lang, Y Zeighami

{"title":"GR.3 Distinct longitudinal brain atrophy trajectories in parkinson’s disease clinical subtypes: insight towards precision medicine","authors":"S Fereshtehnejad, R Moqadam, R Postuma, M Dadar, A Lang, Y Zeighami","doi":"10.1017/cjn.2024.70","DOIUrl":"https://doi.org/10.1017/cjn.2024.70","url":null,"abstract":"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.","PeriodicalId":9571,"journal":{"name":"Canadian Journal of Neurological Sciences / Journal Canadien des Sciences Neurologiques","volume":"1 3","pages":""},"PeriodicalIF":0.0,"publicationDate":"2024-05-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141099926","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

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

Canadian Journal of Neurological Sciences / Journal Canadien des Sciences Neurologiques Pub Date : 2024-05-24 DOI: 10.1017/cjn.2024.87

E. Mostofi, H. McMillan, R. Jurencak, L. Hamilton, A. Yaworski

{"title":"C.2 An in-depth analysis of pediatric inflammatory myopathies: findings from a comprehensive tertiary care hospital","authors":"E. Mostofi, H. McMillan, R. Jurencak, L. Hamilton, A. Yaworski","doi":"10.1017/cjn.2024.87","DOIUrl":"https://doi.org/10.1017/cjn.2024.87","url":null,"abstract":"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.","PeriodicalId":9571,"journal":{"name":"Canadian Journal of Neurological Sciences / Journal Canadien des Sciences Neurologiques","volume":"7 23","pages":""},"PeriodicalIF":0.0,"publicationDate":"2024-05-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141099189","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.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

Canadian Journal of Neurological Sciences / Journal Canadien des Sciences Neurologiques Pub Date : 2024-05-24 DOI: 10.1017/cjn.2024.219

AK Malhotra, AB Nathens, H. Shakil, RH Jaffe, A. Essa, F. Mathieu, J. Badhiwala, K. Thorpe, AV Kulkarni, CD Witiw, JR Wilson

{"title":"P.116 Days at home after traumatic brain injury: moving beyond mortality to evaluate patient-centered outcomes using population health data","authors":"AK Malhotra, AB Nathens, H. Shakil, RH Jaffe, A. Essa, F. Mathieu, J. Badhiwala, K. Thorpe, AV Kulkarni, CD Witiw, JR Wilson","doi":"10.1017/cjn.2024.219","DOIUrl":"https://doi.org/10.1017/cjn.2024.219","url":null,"abstract":"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.","PeriodicalId":9571,"journal":{"name":"Canadian Journal of Neurological Sciences / Journal Canadien des Sciences Neurologiques","volume":"1 5","pages":""},"PeriodicalIF":0.0,"publicationDate":"2024-05-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141099246","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.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

Canadian Journal of Neurological Sciences / Journal Canadien des Sciences Neurologiques Pub Date : 2024-05-24 DOI: 10.1017/cjn.2024.136

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

{"title":"P.029 Endovascular therapy for cerebral venous thrombosis: an international survey","authors":"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","doi":"10.1017/cjn.2024.136","DOIUrl":"https://doi.org/10.1017/cjn.2024.136","url":null,"abstract":"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.","PeriodicalId":9571,"journal":{"name":"Canadian Journal of Neurological Sciences / Journal Canadien des Sciences Neurologiques","volume":"61 23","pages":""},"PeriodicalIF":0.0,"publicationDate":"2024-05-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141102164","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.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

Canadian Journal of Neurological Sciences / Journal Canadien des Sciences Neurologiques Pub Date : 2024-05-24 DOI: 10.1017/cjn.2024.206

N. Balasubramaniam, M. Cerny, J. May, L. Hamackova, J. Novotnyml, D. Barucic, J. Kybic, M. Majovsky, H. Hallak, R. Liscak, D. Netuka

{"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

Canadian Journal of Neurological Sciences / Journal Canadien des Sciences Neurologiques Pub Date : 2024-05-24 DOI: 10.1017/cjn.2024.80

C Ducroux, W Boisseau, R Fahed, G Stotts, M Kyheng, J Labreuche, D. Roy, B Lapergue, A. Poppe

{"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

Canadian Journal of Neurological Sciences / Journal Canadien des Sciences Neurologiques Pub Date : 2024-05-24 DOI: 10.1017/cjn.2024.227

MW Elder, K. Chornenka, S. Marzoughi, MF Hassanabad, M. Rizzuto

{"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

Canadian Journal of Neurological Sciences / Journal Canadien des Sciences Neurologiques Pub Date : 2024-05-24 DOI: 10.1017/cjn.2024.221

A Gomez, L. Froese, T Bergmann, AS Sainbhi, N Vakitbilir, A Islam, KY Stein, I Marquez, Y Ibrahim, F. Zeiler

{"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

Canadian Journal of Neurological Sciences / Journal Canadien des Sciences Neurologiques Pub Date : 2024-05-24 DOI: 10.1017/cjn.2024.212

A. Al-Shawwa, M. Craig, K. Ost, S. Tripathy, D. Cadotte

{"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

Canadian Journal of Neurological Sciences / Journal Canadien des Sciences Neurologiques Pub Date : 2024-05-24 DOI: 10.1017/cjn.2024.74

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

{"title":"A.1 Repurposing Ambroxol as a disease-modifying treatment for Parkinson’s disease dementia: A phase 2, randomized, double blind placebo-controlled trial","authors":"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","doi":"10.1017/cjn.2024.74","DOIUrl":"https://doi.org/10.1017/cjn.2024.74","url":null,"abstract":"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","PeriodicalId":9571,"journal":{"name":"Canadian Journal of Neurological Sciences / Journal Canadien des Sciences Neurologiques","volume":"9 10","pages":""},"PeriodicalIF":0.0,"publicationDate":"2024-05-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141100827","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