Respondek Gesine, Höglinger Günter U
Department of Neurology, Hannover Medical School, Hanover, Germany.
German Center for Neurodegenerative Diseases, Munich, Germany.
Front Neurol. 2021 May 25;12:644064. doi: 10.3389/fneur.2021.644064. eCollection 2021.
The German research networks and prospectively collect comprehensive clinical data, imaging data and biomaterials of patients with a clinical diagnosis of progressive supranuclear palsy. Progressive supranuclear palsy is a rare, adult-onset, neurodegenerative disease with striking clinical heterogeneity. Since now, prospective natural history data are largely lacking. Clinical research into treatment strategies has been limited due to delay in clinical diagnosis and lack of natural history data on distinct clinical phenotypes. The network is organized by the German Center for Neurodegenerative Diseases. is embedded in a larger network with parallel cohorts of other neurodegenerative diseases and healthy controls. The network is directly linked to other cohorts with other primary diagnoses of the neurodegenerative and vascular disease spectrums and also to an autopsy program for clinico-pathological correlation. The network is organized by the German Parkinson and Movement Disorders Society. Both networks follow the same core protocol for patient recruitment and collection of data, imaging and biomaterials. Both networks host a web-based data registry and a central biorepository. Inclusion/exclusion criteria follow the 2017 Movement Disorder Society criteria for the clinical diagnosis of progressive supranuclear palsy. Both networks started recruitment of patients by the end of 2015. As of November 2020, = 354 and 269 patients were recruited into the and the studies, respectively, and = 131 and 87 patients received at least one follow-up visit. The and networks are ideal resources for comprehensive natural history data of PSP, including imaging data and biological samples. In contrast to previous natural history studies, and include not only patients with Richardson's syndrome, but also variant PSP phenotypes as well as patients at very early disease stages, before a diagnosis of possible or probable PSP can be made. This will allow for identification and evaluation of early biomarkers for diagnosis, prognosis, and progression.
德国的研究网络前瞻性地收集临床诊断为进行性核上性麻痹患者的全面临床数据、影像数据和生物材料。进行性核上性麻痹是一种罕见的、成年起病的神经退行性疾病，具有显著的临床异质性。到目前为止，前瞻性自然史数据非常缺乏。由于临床诊断延迟以及缺乏不同临床表型的自然史数据，治疗策略的临床研究受到限制。该网络由德国神经退行性疾病中心组织，嵌入到一个包含其他神经退行性疾病平行队列和健康对照的更大网络中。该网络还直接与具有神经退行性和血管疾病谱其他主要诊断的其他队列以及一个用于临床病理相关性研究的尸检项目相联系。该网络由德国帕金森病和运动障碍协会组织。两个网络都遵循相同的核心方案进行患者招募以及数据、影像和生物材料的收集。两个网络都设有基于网络的数据登记处和中央生物样本库。纳入/排除标准遵循2017年运动障碍协会关于进行性核上性麻痹临床诊断的标准。两个网络均于2015年底开始招募患者。截至2020年11月，分别有354例和269例患者被纳入到两个研究中，并且分别有131例和87例患者接受了至少一次随访。这两个网络是获取进行性核上性麻痹全面自然史数据（包括影像数据和生物样本）的理想资源。与以往的自然史研究不同，这两个网络不仅纳入了理查森综合征患者，还纳入了进行性核上性麻痹变异型表型患者以及处于疾病极早期（在可能或很可能诊断为进行性核上性麻痹之前）的患者。这将有助于识别和评估用于诊断、预后和病情进展的早期生物标志物。
