Defining benefit: Clinically and biologically meaningful outcomes in the next-generation Alzheimer's disease clinical care pathway

IF 13 1区医学 Q1 CLINICAL NEUROLOGY

Alzheimer's & Dementia Pub Date : 2024-12-19 DOI:10.1002/alz.14425

Aya Elhage, Sharon Cohen, Jeffrey Cummings, Wiesje M. van der Flier, Paul Aisen, Min Cho, Joanne Bell, Harald Hampel

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引用次数: 0

Abstract

To understand the potential benefits of emerging Alzheimer's disease (AD) therapies within and beyond clinical trial settings, there is a need to advance current outcome measurements into meaningful information relevant to all stakeholders. The relationship between the impact on disease biology and clinically measurable outcomes in cognition, function, and behavior must be considered when defining the meaningful benefit of early AD therapies. In this review, we discuss: (1) the lack of consideration for biomarkers in the current concept of meaningfulness in AD; (2) the lack of gold standards for determining minimal biologically and clinically important differences (MBCIDs) in AD trials; (3) how the treatment benefits of disease-modifying treatments are cumulative and increase over time; and (4) the different concepts of meaningfulness among key stakeholders. This review utilizes the future clinical biological framework of AD and aims to further integrate and expand the parameters of meaningful benefits toward a precision medicine framework.

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定义获益：下一代阿尔茨海默病临床护理路径中具有临床和生物学意义的成果

为了了解新出现的阿尔茨海默病（AD）疗法在临床试验环境内外可能带来的益处，有必要将目前的结果测量方法提升为与所有利益相关者相关的有意义的信息。在定义早期阿兹海默症疗法的实际获益时，必须考虑对疾病生物学的影响与认知、功能和行为方面的临床可测量结果之间的关系。在本综述中，我们将讨论(1) 在当前的 AD 有意义概念中缺乏对生物标记物的考虑；(2) 在 AD 试验中缺乏确定最小生物和临床重要差异 (MBCID) 的黄金标准；(3) 改变疾病疗法的治疗获益是如何随着时间的推移而累积和增加的；以及 (4) 主要利益相关者之间不同的有意义概念。本综述采用了未来的 AD 临床生物学框架，旨在进一步整合和扩展有意义获益的参数，以实现精准医学框架。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

Alzheimer's & Dementia 医学-临床神经学

CiteScore

14.50

自引率

5.00%

发文量

299

审稿时长

3 months

期刊介绍： Alzheimer's & Dementia is a peer-reviewed journal that aims to bridge knowledge gaps in dementia research by covering the entire spectrum, from basic science to clinical trials to social and behavioral investigations. It provides a platform for rapid communication of new findings and ideas, optimal translation of research into practical applications, increasing knowledge across diverse disciplines for early detection, diagnosis, and intervention, and identifying promising new research directions. In July 2008, Alzheimer's & Dementia was accepted for indexing by MEDLINE, recognizing its scientific merit and contribution to Alzheimer's research.