Lin Zhou , Juliana Schneider , Bert Arnrich , Stefan Konigorski
{"title":"将群体水平的试验分析为 N-of-1 试验:步态应用","authors":"Lin Zhou , Juliana Schneider , Bert Arnrich , Stefan Konigorski","doi":"10.1016/j.conctc.2024.101282","DOIUrl":null,"url":null,"abstract":"<div><p>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.</p></div>","PeriodicalId":37937,"journal":{"name":"Contemporary Clinical Trials Communications","volume":null,"pages":null},"PeriodicalIF":1.4000,"publicationDate":"2024-02-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S2451865424000292/pdfft?md5=531ced59354f9bc2fa73f904b652031b&pid=1-s2.0-S2451865424000292-main.pdf","citationCount":"0","resultStr":"{\"title\":\"Analyzing population-level trials as N-of-1 trials: An application to gait\",\"authors\":\"Lin Zhou , Juliana Schneider , Bert Arnrich , Stefan Konigorski\",\"doi\":\"10.1016/j.conctc.2024.101282\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<div><p>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.</p></div>\",\"PeriodicalId\":37937,\"journal\":{\"name\":\"Contemporary Clinical Trials Communications\",\"volume\":null,\"pages\":null},\"PeriodicalIF\":1.4000,\"publicationDate\":\"2024-02-29\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"https://www.sciencedirect.com/science/article/pii/S2451865424000292/pdfft?md5=531ced59354f9bc2fa73f904b652031b&pid=1-s2.0-S2451865424000292-main.pdf\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Contemporary Clinical Trials Communications\",\"FirstCategoryId\":\"1085\",\"ListUrlMain\":\"https://www.sciencedirect.com/science/article/pii/S2451865424000292\",\"RegionNum\":0,\"RegionCategory\":null,\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"Q4\",\"JCRName\":\"MEDICINE, RESEARCH & EXPERIMENTAL\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Contemporary Clinical Trials Communications","FirstCategoryId":"1085","ListUrlMain":"https://www.sciencedirect.com/science/article/pii/S2451865424000292","RegionNum":0,"RegionCategory":null,"ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q4","JCRName":"MEDICINE, RESEARCH & EXPERIMENTAL","Score":null,"Total":0}
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.
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.