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熊猫研究:帕金森病的亚型分类——队列研究方案。

PANDA study: subtyping of Parkinson's disease - cohort study protocol.

作者信息

Yang Rui, He Chentao, Rong Siming, Gao Ziqi, Li Zihao, Luo Qibing, Cui Xilie, Li Yan, Zhang Piao, Cai Mengfei, Zhang Yuhu

机构信息

Department of Neurology, Guangdong Neuroscience Institute, Guangdong Provincial People's Hospital (Guangdong Academy of Medical Sciences), Southern Medical University, Guangzhou, Guangdong, China.

School of Medicine, South China University of Technology, Guangzhou, Guangdong, China.

出版信息

BMJ Open. 2025 Jul 30;15(7):e102417. doi: 10.1136/bmjopen-2025-102417.

Abstract

INTRODUCTION

Parkinson's disease (PD) is a progressive neurodegenerative disorder characterised by heterogeneous motor and non-motor symptoms. The classification of PD into distinct subtypes remains a major challenge, limiting the development of precision medicine approaches. Existing subtyping methods have relied on clinical features, genetic markers or isolated biomarkers, but an integrated classification system incorporating neuroimaging and fluid biomarkers is lacking. The Prospective Assessments with Neuroimaging and fluid Data Analysis in PD (PANDA) study is a single-centre, longitudinal cohort study designed to explore PD heterogeneity by integrating multimodal neuroimaging, fluid biomarkers and comprehensive clinical assessments.

METHODS AND ANALYSIS

The PANDA study aims to recruit 500 PD patients and 100 healthy controls (HC) at Guangdong Provincial People's Hospital from March 2021 to July 2026. Participants undergo three study visits: baseline, 2-year and 4-year follow-ups. Data collection includes brain MRI scans, blood and cerebrospinal fluid sampling, clinical scales, gait assessment, ambulatory blood pressure monitoring and eye-tracking evaluations. A standardised electronic data capture system ensures data accuracy and completeness. The primary objective is to identify biologically and neuroimaging-defined PD subtypes, track disease progression and explore potential biomarkers for stratification. Statistical analyses will include data-driven clustering techniques and longitudinal modelling to assess subtype stability over time.

ETHICS AND DISSEMINATION

The PANDA study has been approved by the Institutional Review Board of Guangdong Provincial People's Hospital (No KY2024-719-01) and is conducted in accordance with the Declaration of Helsinki. Written informed consent is obtained from all participants or their legal guardians prior to study enrolment. The findings from this study will be disseminated through peer-reviewed journals and conference presentations, ensuring accessibility to researchers, clinicians and stakeholders involved in PD research and treatment.

TRIAL REGISTRATION NUMBER

ChiCTR2400091709.

摘要

引言

帕金森病(PD)是一种进行性神经退行性疾病,其特征为运动和非运动症状的异质性。将PD分为不同亚型仍然是一项重大挑战,限制了精准医学方法的发展。现有的亚型分类方法依赖于临床特征、基因标记或单一生物标志物,但缺乏一个整合神经影像学和体液生物标志物的综合分类系统。帕金森病神经影像学和体液数据分析前瞻性评估(PANDA)研究是一项单中心纵向队列研究,旨在通过整合多模态神经影像学、体液生物标志物和全面的临床评估来探索PD的异质性。

方法与分析

PANDA研究旨在从2021年3月至2026年7月在广东省人民医院招募500例PD患者和100例健康对照(HC)。参与者需接受三次研究访视:基线访视、2年随访和4年随访。数据收集包括脑部MRI扫描、血液和脑脊液采样、临床量表、步态评估、动态血压监测和眼动追踪评估。标准化的电子数据采集系统确保数据的准确性和完整性。主要目标是识别生物学和神经影像学定义的PD亚型,跟踪疾病进展并探索分层的潜在生物标志物。统计分析将包括数据驱动的聚类技术和纵向建模,以评估亚型随时间的稳定性。

伦理与传播

PANDA研究已获得广东省人民医院机构审查委员会的批准(编号KY2024-719-01),并按照《赫尔辛基宣言》进行。在研究入组前,从所有参与者或其法定监护人处获得书面知情同意。本研究的结果将通过同行评审期刊和会议报告进行传播,确保参与PD研究和治疗的研究人员、临床医生和利益相关者能够获取。

试验注册号

ChiCTR2400091709。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/0a27/12314949/ff08d66177fd/bmjopen-15-7-g001.jpg

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