Clinical Core

摘要- ARTFL LEFFTDS纵向FTLD：临床核心 ARTFL LEFFTDS纵向额颞叶变性（ALLFTD）方案代表了 扩大和整合的国际研究联盟，涉及七个核心和两个项目; ALLFTD是 旨在大幅度和全面增加纵向数据量， 生物标本样本和MRI扫描，可公开用于FTLD的研究，以最终促进 疾病修饰疗法的发展。以前，研究和治疗的进展， 额颞叶变性（ARTFL; U 54 NS 092089）和家族性纵向评估 额颞叶痴呆受试者（LEFFTDS; U 01 AG 045390）方案集体招募>1100 来自18个地点的参与者，包括>380名受MAPT，GRN和C9 orf 72突变影响的家庭成员 （超过最初LEFFTDS目标300例），其余参与者为散发性FTLD患者 通过ARTFL注册。详细的临床和神经心理学数据，包括（但不限于） 国家阿尔茨海默氏症协调中心（NACC）统一数据集版本3和FTLD模块已被 完成，到目前为止有近1200个数据包上传到NACC。生物流体样本，包括DNA、PBMC、 收集了绝大多数参与者的mRNA、血浆和血清，以及子集的CSF 在ARTFL/LEFFTDS研究期间，在国家集中储存库转移和处理， 阿尔茨海默氏症和相关痴呆症。超过900个使用标准化协议的MRI扫描已经被 在585名参与者中进行，这些数据已经过质量控制检查并转移到 神经影像学实验室。所有数据、样本和扫描都是可用的，并已被研究人员使用 在ARTFL/LEFFTDS联盟内外。ALLFTD临床核心将继续建立在 ARTFL和LEFFTDS方案通过招募额外的参与者， 临床核心每年将招募和评估多达1100名参与者。 在纵向臂中，将包括500名无症状和轻度症状的运动员， 家族性FTLD和600例轻度症状的散发性FTLD患者。约1000个额外FTLD 患者将被招募到生物流体集中组。与FTD疾病登记处合作， 两组的参与者都将被远程跟踪，要求填写问卷，并提供 定期更新FTLD研究机会的信息。临床核心中的研究中心将提供 整合高质量的临床数据、生物流体样本和MRI扫描，用于所有核心和项目，以及 与科学界分享。

ABSTRACT – ARTFL LEFFTDS Longitudinal FTLD: CLINICAL CORE The ARTFL LEFFTDS Longitudinal Frontotemporal Lobar Degeneration (ALLFTD) protocol represents an expanded and integrated international research consortium involving seven cores and two projects; ALLFTD is designed to substantially and comprehensively increase the amount of longitudinal data, numbers of biospecimen samples and MRI scans that are publicly available for research in FTLD, to ultimately foster development of disease-modifying therapies. Previously, the Advancement of Research and Treatment in Frontotemporal Lobar Degeneration (ARTFL; U54 NS092089) and Longitudinal Evaluation of Familial Frontotemporal Dementia Subjects (LEFFTDS; U01 AG045390) protocols collectively enrolled >1100 participants at 18 sites, including >380 members of families affected by MAPT, GRN and C9orf72 mutations (surpassing the original LEFFTDS target of 300), with the remaining participants being sporadic FTLD patients enrolled through ARTFL. Detailed clinical and neuropsychological data, including (but not limited to) the National Alzheimer's Coordinating Center (NACC) Uniform Data Set Version 3 and FTLD Module has been completed, with almost 1200 data packets uploaded to NACC to date. Biofluid samples, including DNA, PBMC, mRNA, plasma and serum on the vast majority of participants, and CSF on a subset, have been collected during the ARTFL/LEFFTDS studies, transferred and processed at the National Centralized Repository for Alzheimer's Disease and Related Dementias. Over 900 MRI scans using a standardized protocol have been performed in 585 participants, and these data have undergone quality control inspection and transferal to the Laboratory of NeuroImaging. All data, samples and scans are available and have been used by investigators within and outside of the ARTFL/LEFFTDS Consortium. The ALLFTD Clinical Core will continue to build on the obvious success of the ARTFL and LEFFTDS protocols by recruiting additional participants in either the longitudinal or biofluid-focused arm. The Clinical Core will enroll and evaluate up to 1100 participants annually in the longitudinal arm, and will include 500 asymptomatic and mildly symptomatic members of kindreds with familial FTLD and 600 mildly symptomatic sporadic FTLD patients. Approximately 1000 additional FTLD patients will be recruited into the biofluid focused arm. In partnership with the FTD Disorders Registry, participants in both arms will be followed remotely, requested to complete questionnaires and provided with periodic updates and information of FTLD research opportunities. The sites in the Clinical Core will provide high quality clinical data, biofluid samples and MRI scans to be integrated for use in all cores and projects, and shared with the scientific community.