Why a clinical trial is as good as its outcome measure: A framework for the selection and use of cognitive outcome measures for clinical trials of Alzheimer's disease.
Why a clinical trial is as good as its outcome measure: A framework for the selection and use of cognitive outcome measures for clinical trials of Alzheimer's disease.
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DOI：
10.1002/alz.12773
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发表时间：
2023-03
期刊：
影响因子：
--
通讯作者：
Sikkes SAM
中科院分区：
文献类型：
--
作者：
Jutten RJ;Papp KV;Hendrix S;Ellison N;Langbaum JB;Donohue MC;Hassenstab J;Maruff P;Rentz DM;Harrison J;Cummings J;Scheltens P;Sikkes SAM
任何临床试验的一个关键方面是使用正确的结果指标来评估治疗效果。与阿尔茨海默病（AD）临床前和早期症状阶段的病理生理学的快速发展的理解和测量相比，这些阶段的临床结局评估（COA）的进展相对较小。本文旨在为早期AD试验中使用的COA的设计和评价提供基准。我们讨论了在AD痴呆前期阶段捕获认知变化的经验教训，包括验证这些早期阶段的新COA时的挑战以及在临床试验中实施的必要证据。展望未来，我们提出了一个多步骤的框架，以推进使用更有效的COA来评估早期AD的临床意义变化，这将有助于围绕更适当的结局指标达成急需的共识，以评估推定治疗的临床疗效。
A crucial aspect of any clinical trial is using the right outcome measure to assess treatment efficacy. Compared to the rapidly evolved understanding and measurement of pathophysiology in preclinical and early symptomatic stages of Alzheimer’s disease (AD), relatively less progress has been made in the evolution of clinical outcome assessments (COAs) for those stages. The current paper aims to provide a benchmark for the design and evaluation of COAs for use in early AD trials. We discuss lessons learned on capturing cognitive changes in predementia stages of AD, including challenges when validating novel COAs for those early stages and necessary evidence for their implementation in clinical trials. Moving forward, we propose a multi-step framework to advance the use of more effective COAs to assess clinically meaningful changes in early AD, which will hopefully contribute to the much-needed consensus around more appropriate outcome measures to assess clinical efficacy of putative treatments.
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DOI：
10.1002/dad2.12012
发表时间：
2020
期刊：
Alzheimer's & dementia (Amsterdam, Netherlands)
影响因子：
--
作者：
Rentz DM;Papp KV
通讯作者：
Papp KV
影响因子：
29
作者：
Donohue, Michael C.;Sperling, Reisa A.;Salmon, David P.;Rentz, Dorene M.;Raman, Rema;Thomas, Ronald G.;Weiner, Michael;Aisen, Paul S.
通讯作者：
Aisen, Paul S.
影响因子：
4
作者：
Cloutier S;Chertkow H;Kergoat MJ;Gélinas I;Gauthier S;Belleville S
通讯作者：
Belleville S
影响因子：
14.5
作者：
Dong, Aoyan;Toledo, Jon B.;Davatzikos, Christos
通讯作者：
Davatzikos, Christos
影响因子：
2.4
作者：
Dowling, N. Maritza;Hermann, Bruce;La Rue, Asenath;Sager, Mark A.
通讯作者：
Sager, Mark A.