The Edmond J Safra Program in PD, Toronto Western Hospital, University of Toronto, Toronto, ON, Canada.
Department of Neurology, Columbia University Irving Medical Center, New York, NY, United States; Division of Movement Disorders, Neurological Institute, Tel Aviv Sourasky Medical Center, Tel Aviv, Israel.
Handb Clin Neurol. 2023;192:219-229. doi: 10.1016/B978-0-323-85538-9.00014-6.
Identifying individuals at high risk for developing neurodegenerative disease opens the possibility of conducting clinical trials that intervene at an earlier stage of neurodegeneration than has been possible to date, and in doing so hopefully improves the odds of efficacy for interventions aimed at slowing or stopping the disease process. The long prodromal phase of Parkinson disease presents opportunities and challenges to establishing cohorts of at-risk individuals. Recruiting people with genetic variants conferring increased risk and people with REM sleep behavior disorder currently constitutes the most promising strategies, but multistage screening of the general population may also be feasible capitalizing on known risk factors and prodromal features. This chapter discusses the challenges involved in identifying, recruiting, and retaining these individuals, and provides insights into possible solutions using examples from studies to date.
识别有发展为神经退行性疾病风险的个体为开展临床试验提供了可能，这种临床试验可以在神经退行性病变的早期阶段进行干预，从而有望提高针对减缓或阻止疾病进程的干预措施的疗效。帕金森病的漫长前驱期为建立高危个体队列提供了机会和挑战。招募具有增加风险的遗传变异的个体和 REM 睡眠行为障碍的个体目前构成了最有前途的策略，但利用已知的危险因素和前驱特征对一般人群进行多阶段筛查也可能是可行的。本章讨论了在识别、招募和保留这些个体时所涉及的挑战，并通过迄今为止的研究示例提供了可能的解决方案的见解。
