Department of Clinical and Movement Neurosciences, UCL Queen Square Institute of Neurology, University College London, London, United Kingdom.
Expert by experience, Guildford, United Kingdom.
Mov Disord. 2024 Feb;39(2):433-438. doi: 10.1002/mds.29691. Epub 2023 Dec 22.
Clinical trials of disease-modifying therapies in PD require valid and responsive primary outcome measures that are relevant to patients.
The objective is to select a patient-centered primary outcome measure for disease-modification trials over three or more years.
Experts in Parkinson's disease (PD), statistics, and health economics and patient and public involvement and engagement (PPIE) representatives reviewed and discussed potential outcome measures. A larger PPIE group provided input on their key considerations for such an endpoint. Feasibility, clinimetric properties, and relevance to patients were assessed and synthesized.
Although initial considerations favored the Movement Disorder Society-sponsored revision of the Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Part III in Off, feasibility, PPIE input, and clinimetric properties supported the MDS-UPDRS Part II. However, PPIE input also highlighted the importance of nonmotor symptoms, especially in the longer term, leading to the selection of the MDS-UPDRS Parts I + II sum score.
The MDS-UPDRS Parts I + II sum score was chosen as the primary outcome for large 3-year disease-modification trials. © 2023 The Authors. Movement Disorders published by Wiley Periodicals LLC on behalf of International Parkinson and Movement Disorder Society.
在 PD 中进行疾病修饰疗法的临床试验需要有效的、有反应的、与患者相关的主要结局测量指标。
旨在为超过三年的疾病修饰试验选择以患者为中心的主要结局测量指标。
帕金森病（PD）专家、统计学专家、健康经济学专家以及患者和公众参与和参与（PPIE）代表审查并讨论了潜在的结局测量指标。更大的 PPIE 群体提供了对此类终点的关键考虑因素的意见。评估并综合了可行性、临床计量特性和与患者的相关性。
尽管最初的考虑因素倾向于支持由运动障碍协会赞助的统一帕金森病评定量表（MDS-UPDRS）第三部分停用期的修订版，但可行性、PPIE 意见和临床计量特性支持 MDS-UPDRS 第二部分。然而，PPIE 的意见也强调了非运动症状的重要性，尤其是在长期内，这导致选择 MDS-UPDRS 第一部分加第二部分的总和评分。
MDS-UPDRS 第一部分加第二部分的总和评分被选为 3 年大型疾病修饰试验的主要结局。© 2023 作者。运动障碍协会代表国际帕金森病和运动障碍协会由 Wiley 期刊出版公司出版。
