Couto Blas, Fox Susan, Tartaglia Maria Carmela, Rogaeva Ekaterina, Antwi Jeffrey, Bhakta Puja, Kovacs Gabor G, Lang Anthony E
Edmond J. Safra Program in Parkinson's Disease, Rossy Progressive Supranuclear Palsy Centre and the Morton and Gloria Shulman Movement Disorders Clinic, Toronto Western Hospital, Toronto, Ontario, Canada.
Department of Medicine, Division of Neurology, University Health Network and the University of Toronto, Toronto, Ontario, Canada.
Can J Neurol Sci. 2023 Nov;50(6):845-852. doi: 10.1017/cjn.2022.332. Epub 2023 Jan 5.
To describe the development and initial experience of a clinical research program in progressive supranuclear palsy (PSP) and corticobasal syndrome (CBS) in Canada: The Rossy PSP Centre, to share the data acquisition tools adopted, and to report preliminary results.
Extensive demographic and longitudinal clinical information is collected every 6 months using standardized forms. Biofluids are collected for biobanking and genetic analysis, and many patients are enrolled in neuroimaging research protocols. Brain donation is an important component of the program, and standardized processing protocols have been established, including very short death to autopsy times in patients undergoing medical assistance in dying.
Between Oct 2019 and Dec 2021, 132 patients were screened, 91 fulfilling criteria for PSP and 19 for CBS; age 71 years; 41% female; duration 5 years, age-of-onset 66 years. The most common symptoms at onset were postural instability and falls (45%), cognitive-behavioral changes (22%), and Parkinsonism (9%). The predominant clinical phenotype was Richardson syndrome (82%). Levodopa and amantadine resulted in partial and short-lasting benefit.
The Rossy PSP Centre has been established to advance clinical and basic research in PSP and related tauopathies. The extent of the clinical data collected permits deep phenotyping of patients and allows for future clinical and basic research. Preliminary results showed expected distribution of phenotypes, demographics, and response to symptomatic treatments in our cohort. Longitudinal data will provide insight into the early diagnosis and management of PSP. Future steps include enrollment of patients in earlier stages, development of biomarkers, and fast-tracking well-characterized patients into clinical trials.
描述加拿大进行的一项关于进行性核上性麻痹（PSP）和皮质基底节综合征（CBS）的临床研究项目——罗西PSP中心的发展情况及初步经验，分享所采用的数据采集工具，并报告初步结果。
每6个月使用标准化表格收集广泛的人口统计学和纵向临床信息。采集生物流体用于生物样本库储存和基因分析，许多患者参与神经影像学研究方案。脑捐赠是该项目的一个重要组成部分，已制定标准化处理方案，包括对接受临终医疗援助的患者从死亡到尸检的时间间隔非常短。
在2019年10月至2021年12月期间，共筛选了132例患者，其中91例符合PSP标准，19例符合CBS标准；年龄71岁；41%为女性；病程5年，发病年龄66岁。发病时最常见的症状是姿势不稳和跌倒（45%）、认知行为改变（22%）和帕金森综合征（9%）。主要临床表型为理查森综合征（82%）。左旋多巴和金刚烷胺带来部分且持续时间短的益处。
已设立罗西PSP中心以推进PSP及相关tau蛋白病的临床和基础研究。所收集临床数据的广度允许对患者进行深入表型分析，并为未来的临床和基础研究提供支持。初步结果显示了我们队列中表型、人口统计学特征以及对对症治疗反应的预期分布情况。纵向数据将为PSP的早期诊断和管理提供见解。未来的步骤包括纳入更早期阶段的患者、开发生物标志物，以及将特征明确的患者快速纳入临床试验。
