将群体水平的试验分析为 N-of-1 试验:步态应用

IF 1.4 Q4 MEDICINE, RESEARCH & EXPERIMENTAL
Lin Zhou , Juliana Schneider , Bert Arnrich , Stefan Konigorski
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引用次数: 0

摘要

只要干预效果在研究参与者之间存在差异,研究健康干预措施的个体因果效应就非常重要。进行 N-of-1 试验(即单人随机对照试验)是对其进行分析的黄金标准。作为一种替代方法,我们建议将现有的人群水平研究重新分析为 N-of-1 试验,并以步态为例进行说明。我们收集了 16 名年轻健康参与者在疲劳和非疲劳以及单一任务(仅行走)和双重任务(行走的同时执行认知任务)条件下的步态数据。参照N-of-1试验方法,我们首先计算了标准的群体水平方差分析模型,以评估不同条件下步态参数(步幅和步幅时间)的差异。然后,我们通过贝叶斯重复测量模型估算干预措施对个体步态参数的影响,将每个参与者视为各自的试验,并比较结果。结果表明,虽然总体层面的影响不明显,但个体层面的分析显示了参与者之间的差异。所有参与者的步态参数基线值差异很大,疲劳和认知任务的影响也不尽相同,有些人的影响方向相反。与认知任务干预相比,疲劳干预在群体层面和个体层面的分析差异更为明显。在我们的实证分析之后,我们将更广泛地讨论通过N-of-1试验的视角重新分析人群研究,并强调重要的注意事项和要求。我们的工作鼓励未来的研究利用人群数据调查个体效应。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Analyzing population-level trials as N-of-1 trials: An application to gait

Studying individual causal effects of health interventions is important whenever intervention effects are heterogeneous between study participants. Conducting N-of-1 trials, which are single-person randomized controlled trials, is the gold standard for their analysis. As an alternative method, we propose to re-analyze existing population-level studies as N-of-1 trials, and use gait as a use case for illustration. Gait data were collected from 16 young and healthy participants under fatigued and non-fatigued, as well as under single-task (only walking) and dual-task (walking while performing a cognitive task) conditions. As a reference to the N-of-1 trials approach, we first computed standard population-level ANOVA models to evaluate differences in gait parameters (stride length and stride time) across conditions. Then, we estimated the effect of the interventions on gait parameters on the individual level through Bayesian repeated-measures models, viewing each participant as their own trial, and compared the results. The results illustrated that while few overall population-level effects were visible, individual-level analyses revealed differences between participants. Baseline values of the gait parameters varied largely among all participants, and the effects of fatigue and cognitive task were also heterogeneous, with some individuals showing effects in opposite directions. These differences between population-level and individual-level analyses were more pronounced for the fatigue intervention compared to the cognitive task intervention. Following our empirical analysis, we discuss re-analyzing population studies through the lens of N-of-1 trials more generally and highlight important considerations and requirements. Our work encourages future studies to investigate individual effects using population-level data.

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来源期刊
Contemporary Clinical Trials Communications
Contemporary Clinical Trials Communications Pharmacology, Toxicology and Pharmaceutics-Pharmacology
CiteScore
2.70
自引率
6.70%
发文量
146
审稿时长
20 weeks
期刊介绍: Contemporary Clinical Trials Communications is an international peer reviewed open access journal that publishes articles pertaining to all aspects of clinical trials, including, but not limited to, design, conduct, analysis, regulation and ethics. Manuscripts submitted should appeal to a readership drawn from a wide range of disciplines including medicine, life science, pharmaceutical science, biostatistics, epidemiology, computer science, management science, behavioral science, and bioethics. Contemporary Clinical Trials Communications is unique in that it is outside the confines of disease specifications, and it strives to increase the transparency of medical research and reduce publication bias by publishing scientifically valid original research findings irrespective of their perceived importance, significance or impact. Both randomized and non-randomized trials are within the scope of the Journal. Some common topics include trial design rationale and methods, operational methodologies and challenges, and positive and negative trial results. In addition to original research, the Journal also welcomes other types of communications including, but are not limited to, methodology reviews, perspectives and discussions. Through timely dissemination of advances in clinical trials, the goal of Contemporary Clinical Trials Communications is to serve as a platform to enhance the communication and collaboration within the global clinical trials community that ultimately advances this field of research for the benefit of patients.
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