Edmond J. Safra Program in Parkinson's Disease and the Morton and Gloria Shulman Movement Disorders Clinic, Toronto Western Hospital, University Health Network, Toronto, Ontario, Canada.
Division of Neurology, Department of Medicine, The Ottawa Hospital, University of Ottawa, Ottawa, Ontario, Canada.
Mov Disord. 2024 Mar;39(3):462-471. doi: 10.1002/mds.29708. Epub 2024 Jan 20.
The International Parkinson and Movement Disorder Society (MDS) created a task force (TF) to provide a critical overview of the Parkinson's disease (PD) subtyping field and develop a guidance on future research in PD subtypes. Based on a literature review, we previously concluded that PD subtyping requires an ultimate alignment with principles of precision medicine, and consequently novel approaches were needed to describe heterogeneity at the individual patient level. In this manuscript, we present a novel purpose-driven framework for subtype research as a guidance to clinicians and researchers when proposing to develop, evaluate, or use PD subtypes. Using a formal consensus methodology, we determined that the key purposes of PD subtyping are: (1) to predict disease progression, for both the development of therapies (use in clinical trials) and prognosis counseling, (2) to predict response to treatments, and (3) to identify therapeutic targets for disease modification. For each purpose, we describe the desired product and the research required for its development. Given the current state of knowledge and data resources, we see purpose-driven subtyping as a pragmatic and necessary step on the way to precision medicine. © 2024 The Authors. Movement Disorders published by Wiley Periodicals LLC on behalf of International Parkinson and Movement Disorder Society.
国际帕金森病和运动障碍学会（MDS）成立了一个特别工作组（TF），对帕金森病（PD）亚型领域进行了批判性的综述，并就 PD 亚型的未来研究制定了指导方针。基于文献回顾，我们之前得出的结论是，PD 亚型需要与精准医学的原则最终保持一致，因此需要新的方法来描述个体患者水平的异质性。在本文中，我们提出了一个新的以目的为导向的亚型研究框架，为临床医生和研究人员在提出开发、评估或使用 PD 亚型时提供指导。我们使用正式的共识方法，确定了 PD 亚型研究的主要目的是：（1）预测疾病进展，包括治疗方法的开发（临床试验中使用）和预后咨询，（2）预测治疗反应，以及（3）确定疾病修正的治疗靶点。对于每个目的，我们描述了所需的产品和开发所需的研究。鉴于目前的知识和数据资源状况，我们认为以目的为导向的亚型研究是迈向精准医学的务实且必要的一步。© 2024 作者。运动障碍由 Wiley Periodicals LLC 代表国际帕金森病和运动障碍学会出版。
