Clinical Trial Endpoints and Their Clinical Meaningfulness in Early Stages of Alzheimer's Disease.
Clinical Trial Endpoints and Their Clinical Meaningfulness in Early Stages of Alzheimer's Disease.
复制标题
DOI：
10.14283/jpad.2022.41
复制
发表时间：
2022
影响因子：
--
通讯作者：
中科院分区：
文献类型：
--
作者：
随着阿尔茨海默病 (AD) 治疗开发的重点转向疾病的早期阶段，药物试验中使用的临床终点以及如何将其转化为临床实践变得越来越重要。试验结果指标的临床意义通常尚不清楚，缺乏确凿的证据表明这些指标与疾病进展和治疗反应的变化有何关联。澄清这一点将使所有人受益，包括患者、护理合作伙伴、初级保健提供者、监管机构和付款人，并将增强我们对临床试验终点与日常实践中使用的评估之间关系的理解。目前，AD 临床试验中使用了多种评估工具，并且这些试验中选择的终点指标存在很大差异。本综述的目的是总结 AD 早期阶段最常用的评估工具，描述它们在临床试验和临床实践中的使用，并讨论这些措施中与疾病进展和治疗反应相关的、可能构成临床意义变化的内容。
As the focus of Alzheimer’s disease (AD) therapeutic development shifts to the early stages of the disease, the clinical endpoints used in drug trials, and how these might translate into clinical practice, are of increasing importance. The clinical meaningfulness of trial outcome measures is often unclear, with a lack of conclusive evidence as to how these measures correlate to changes in disease progression and treatment response. Clarifying this would benefit all, including patients, care partners, primary care providers, regulators, and payers, and would enhance our understanding of the relationship between clinical trial endpoints and assessments used in everyday practice. At present, there is a wide range of assessment tools used in clinical trials for AD and substantial variability in measures selected as endpoints across these trials. The aim of this review is to summarize the most commonly used assessment tools for early stages of AD, describe their use in clinical trials and clinical practice, and discuss what might constitute clinically meaningful change in these measures in relation to disease progression and treatment response.
影响因子：
2.3
作者：
Li F;Takechi H;Kokuryu A;Takahashi R
通讯作者：
Takahashi R