Kang Un Jung, Goldman Jennifer G, Alcalay Roy N, Xie Tao, Tuite Paul, Henchcliffe Claire, Hogarth Penelope, Amara Amy W, Frank Samuel, Rudolph Alice, Casaceli Cynthia, Andrews Howard, Gwinn Katrina, Sutherland Margaret, Kopil Catherine, Vincent Lona, Frasier Mark
Division of Movement Disorders, Department of Neurology, Columbia University Medical Center, New York, New York, USA.
Section of Parkinson Disease and Movement Disorders, Department of Neurological Sciences, Rush University Medical Center, Chicago, Illinois, USA.
Mov Disord. 2016 Jun;31(6):924-32. doi: 10.1002/mds.26613. Epub 2016 Apr 26.
Identifying PD-specific biomarkers in biofluids will greatly aid in diagnosis, monitoring progression, and therapeutic interventions. PD biomarkers have been limited by poor discriminatory power, partly driven by heterogeneity of the disease, variability of collection protocols, and focus on de novo, unmedicated patients. Thus, a platform for biomarker discovery and validation in well-characterized, clinically typical, moderate to advanced PD cohorts is critically needed.
BioFIND (Fox Investigation for New Discovery of Biomarkers in Parkinson's Disease) is a cross-sectional, multicenter biomarker study that established a repository of clinical data, blood, DNA, RNA, CSF, saliva, and urine samples from 118 moderate to advanced PD and 88 healthy control subjects. Inclusion criteria were designed to maximize diagnostic specificity by selecting participants with clinically typical PD symptoms, and clinical data and biospecimen collection utilized standardized procedures to minimize variability across sites.
We present the study methodology and data on the cohort's clinical characteristics. Motor scores and biospecimen samples including plasma are available for practically defined off and on states and thus enable testing the effects of PD medications on biomarkers. Other biospecimens are available from off state PD assessments and from controls.
Our cohort provides a valuable resource for biomarker discovery and validation in PD. Clinical data and biospecimens, available through The Michael J. Fox Foundation for Parkinson's Research and the National Institute of Neurological Disorders and Stroke, can serve as a platform for discovering biomarkers in clinically typical PD and comparisons across PD's broad and heterogeneous spectrum. © 2016 The Authors. Movement Disorders published by Wiley Periodicals, Inc. on behalf of International Parkinson and Movement Disorder Society.
在生物流体中识别帕金森病(PD)特异性生物标志物将极大地有助于诊断、监测疾病进展和进行治疗干预。PD生物标志物一直受到鉴别能力差的限制,部分原因是疾病的异质性、采集方案的变异性以及对初发、未用药患者的关注。因此,迫切需要一个在特征明确、临床典型的中晚期PD队列中发现和验证生物标志物的平台。
BioFIND(帕金森病生物标志物新发现的福克斯研究)是一项横断面、多中心生物标志物研究,建立了一个包含118例中晚期PD患者和88例健康对照受试者的临床数据、血液、DNA、RNA、脑脊液、唾液和尿液样本的储存库。纳入标准旨在通过选择具有临床典型PD症状的参与者来最大化诊断特异性,并且临床数据和生物样本采集采用标准化程序以最小化各研究点之间的变异性。
我们展示了该队列临床特征的研究方法和数据。运动评分和包括血浆在内的生物样本可用于实际定义的关期和开期状态,从而能够测试PD药物对生物标志物的影响。其他生物样本可从关期PD评估和对照中获得。
我们的队列提供了一个在PD中发现和验证生物标志物的宝贵资源。通过迈克尔·J·福克斯帕金森病研究基金会和美国国立神经疾病与中风研究所可获得的临床数据和生物样本,可作为在临床典型PD中发现生物标志物以及在PD广泛而异质的范围内进行比较的平台。© 2016作者。《运动障碍》由威利期刊公司代表国际帕金森病和运动障碍协会出版。