Which endpoints should be applied in interventional trials? – From single uni-dimensional assessment tailored to a drug's mechanism of action to multi-component measures and multi-domain composites

Felix Eckstein , Tanja Stamm , Jamie Collins
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Abstract

Objective

A vast array of structural/imaging and clinical endpoints/outcomes are available today to osteoarthritis epidemiologists or trialists. Which assessments are best suited for which studies remains unsettled. When several assessments are available, these may be analyzed together (simultaneously or hierarchically), using statistical modeling and adjustment. Or, alternatively, they may be combined to form more complex multi-component or composite (potentially multi-domain) endpoints/outcomes. This review describes such concepts and their challenges, using examples from current osteoarthritis imaging research.

Design

A narrative, non-systematic literature search (PubMed and others) was conducted, and informal consultations were held with experts in the field. The identified concepts and experimental findings were then organized to present an integrated framework.

Results

Single imaging assessments can encompass one (uni-dimensional) or more (multi-dimensional) structures. Integration of image assessments of one structure/tissue across anatomical locations provides aggregate measures. This can also be created across heterogeneous (multi-dimensional) types of assessments (multi-component/composite), either within an area (such as imaging - single domain) or across broader areas of health and well-being (multi-domain). Weighting, standardization, and (clinical) usefulness are crucial characteristics of multi-component/composite endpoints. Examples of these concepts are here provided in the context of osteoarthritis imaging.

Conclusions

Options for multi-component/composite endpoints in osteoarthritis research are virtually infinite. Smart research strategies are required to explore and validate these vast possibilities, with appropriate statistical treatment being paramount. A one-size/endpoint-fits-all approach will likely fail in observational and interventional studies. Imaging assessment needs to be tailored to both the drug's unique mechanism of action, and to the participants’ morpho-type.
介入试验应采用哪些终点?-从针对药物作用机制的单一单维评估到多组分测量和多域复合评估
目前,骨关节炎流行病学家或临床试验学家可以获得大量的结构/成像和临床终点/结果。哪种评估最适合哪种研究仍未确定。当有几个评估可用时,可以使用统计建模和调整将这些评估一起分析(同时或分层)。或者,它们可以组合起来形成更复杂的多组件或复合(可能是多域)端点/结果。这篇综述描述了这些概念和他们的挑战,使用实例从目前的骨关节炎成像研究。进行了一次叙述性、非系统的文献检索(PubMed等),并与该领域的专家进行了非正式磋商。然后将确定的概念和实验结果组织成一个综合框架。结果单一影像学评估可包括一个(单维)或多个(多维)结构。跨解剖位置的一个结构/组织的图像评估集成提供了汇总措施。这也可以跨异质(多维)类型的评估(多成分/复合)创建,无论是在一个领域内(例如成像-单一领域)还是跨更广泛的健康和福祉领域(多领域)。权重、标准化和(临床)有用性是多组分/复合终点的关键特征。这些概念的例子在这里提供骨关节炎成像的背景下。结论骨关节炎研究中多组分/复合终点的选择几乎是无限的。需要聪明的研究策略来探索和验证这些巨大的可能性,适当的统计处理是至关重要的。在观察性和干预性研究中,单一尺寸/终点适合所有方法可能会失败。成像评估需要根据药物的独特作用机制和参与者的形态类型进行调整。
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
Osteoarthritis imaging
Osteoarthritis imaging Radiology and Imaging
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