Outcome Measures for Disease-Modifying Trials in Parkinson's Disease: Consensus Paper by the EJS ACT-PD Multi-Arm Multi-Stage Trial Initiative.
Outcome Measures for Disease-Modifying Trials in Parkinson's Disease: Consensus Paper by the EJS ACT-PD Multi-Arm Multi-Stage Trial Initiative.
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DOI：
10.3233/jpd-230051
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发表时间：
2023
影响因子：
5.2
通讯作者：
Schrag, Anette
中科院分区：
文献类型：
--
作者：
Gonzalez-Robles, Cristina;Weil, Rimona S.;van Wamelen, Daniel;Bartlett, Michele;Burnell, Matthew;Clarke, Caroline S.;Hu, Michele T.;Huxford, Brook;Jha, Ashwani;Lambert, Christian;Lawton, Michael;Mills, Georgia;Noyce, Alastair;Piccini, Paola;Pushparatnam, Kuhan;Rochester, Lynn;Siu, Carroll;Williams-Gray, Caroline H.;Zeissler, Marie-Louise;Zetterberg, Henrik;Carroll, Camille B.;Foltynie, Thomas;Schrag, Anette
多臂多阶段（MAMS）平台试验可以加速帕金森病（PD）的疾病改善治疗的确定，但目前对这种方法的最佳结局指标（OM）尚未达成共识。为改善疾病的PD试验提供OM的最新库存，并为此类试验未来选择OM提供框架。作为Edmond J Safra加速帕金森病临床试验（EJS ACT-PD）倡议的一部分，一个专家组与患者和公众参与和参与（PPIE）代表的投入审查和评估了OM的现有证据，以用于延迟PD进展的试验。根据有效性、对变化的敏感性、参与者负担和多中心试验的实用性等方面对每个OM进行排名。对证据和专家意见的审查促成了本清单。创建了一个广泛的OM清单，分为：一般、运动和非运动量表、日记和波动问卷、认知、残疾和健康相关的生活质量、能力、定量运动、可穿戴和数字、组合、资源使用、成像和湿生物标志物以及基于里程碑的。提出了一个评价OM的框架，以在未来更新库存。PPIE输入强调了OM的必要性，OM反映了他们的疾病进展经验，适用于不同的人群和疾病阶段。我们提出了一系列OM，根据一个透明的框架进行分类，以帮助选择OM进行疾病修饰PD试验，同时允许在出现新证据时纳入或重新分类相关OM。
Multi-arm, multi-stage (MAMS) platform trials can accelerate the identification of disease-modifying treatments for Parkinson’s disease (PD) but there is no current consensus on the optimal outcome measures (OM) for this approach. To provide an up-to-date inventory of OM for disease-modifying PD trials, and a framework for future selection of OM for such trials. As part of the Edmond J Safra Accelerating Clinical Trials in Parkinson Disease (EJS ACT-PD) initiative, an expert group with Patient and Public Involvement and Engagement (PPIE) representatives’ input reviewed and evaluated available evidence on OM for potential use in trials to delay progression of PD. Each OM was ranked based on aspects such as validity, sensitivity to change, participant burden and practicality for a multi-site trial. Review of evidence and expert opinion led to the present inventory. An extensive inventory of OM was created, divided into: general, motor and non-motor scales, diaries and fluctuation questionnaires, cognitive, disability and health-related quality of life, capability, quantitative motor, wearable and digital, combined, resource use, imaging and wet biomarkers, and milestone-based. A framework for evaluation of OM is presented to update the inventory in the future. PPIE input highlighted the need for OM which reflect their experience of disease progression and are applicable to diverse populations and disease stages. We present a range of OM, classified according to a transparent framework, to aid selection of OM for disease-modifying PD trials, whilst allowing for inclusion or re-classification of relevant OM as new evidence emerges.
影响因子：
3.4
作者：
Pavel, Dan G.;Henderson, Theodore A.;DeBruin, Simon;Cohen, Philip F.
通讯作者：
Cohen, Philip F.
影响因子：
3.5
作者：
Brown, Carlos A.;Cheng, Eric M.;Hays, Ron D.;Vassar, Stefanie D.;Vickrey, Barbara G.
通讯作者：
Vickrey, Barbara G.
影响因子：
6
作者：
Waggan, Imran;Rissanen, Eero;Tuisku, Jouni;Joutsa, Juho;Helin, Semi;Parkkola, Riitta;Rinne, Juha O.;Airas, Laura
通讯作者：
Airas, Laura