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应用贝叶斯可信亚组分析方法识别治疗获益人群:以 LOOK AHEAD 试验为例。

Using the Bayesian credible subgroups method to identify populations benefiting from treatment: An application to the Look AHEAD trial.

机构信息

Graduate Entry Medical School, The University of Limerick, Limerick, Ireland.

Division of Biostatistics, College of Public Health, The Ohio State University, Columbus, OH, United States of America.

出版信息

PLoS One. 2020 Apr 21;15(4):e0231241. doi: 10.1371/journal.pone.0231241. eCollection 2020.

DOI:10.1371/journal.pone.0231241
PMID:32315340
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC7173866/
Abstract

Traditionally, subgroup analyses are used to assess whether patient characteristics moderate treatment effectiveness with general disregard for issues of multiplicity. Using data from The Action for Health in Diabetes (Look AHEAD) trial in the United States, we aim to identify a subgroup where all of its types of members experience a treatment benefit defined as reducing the likelihood of a major cardiovascular event under an intensive lifestyle and weight-loss intervention. We apply the credible subgroups method to a Bayesian logistic model with a conservative prior that is sceptical of large treatment effect heterogeneity. The covariate profiles for which there is sufficient evidence of treatment benefit are, coarsely, middle-aged women, in poor subjective general health and with moderately to poorly controlled diabetes. There is at least 80% posterior probability that the conditional average treatment effect is positive for all covariate profiles fitting this description, which account for 0.5% of trial participants. Conversely, the covariate profiles that are likely to be associated with no benefit are middle aged and older men in excellent subjective general health, with well-controlled diabetes. These profiles apply to less than 2% of trial participants. More information is required to determine treatment benefit or no benefit for the remainder of the trial population.

摘要

传统上,亚组分析用于评估患者特征是否会影响治疗效果,而通常忽略多重性问题。我们使用来自美国糖尿病行动研究(Look AHEAD)试验的数据,旨在确定一个亚组,其所有类型的成员都能从强化生活方式和减肥干预中获得治疗益处,定义为降低主要心血管事件的可能性。我们将可信亚组方法应用于贝叶斯逻辑模型,该模型具有保守的先验概率,对大的治疗效果异质性持怀疑态度。有足够证据表明治疗有益的协变量特征大致为:中年女性,主观总体健康状况较差,且糖尿病控制中等或较差。对于符合这种描述的所有协变量特征,条件平均治疗效果有至少 80%的后验概率为正,这些特征占试验参与者的 0.5%。相反,不太可能受益的协变量特征是主观总体健康状况良好的中老年男性,且糖尿病控制良好。这些特征适用于不到 2%的试验参与者。需要更多信息来确定试验人群中其余部分的治疗益处或无益处。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/216e/7173866/0a51b684cb83/pone.0231241.g005.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/216e/7173866/fd5b049c9103/pone.0231241.g001.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/216e/7173866/ec64529d463a/pone.0231241.g002.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/216e/7173866/174680741afc/pone.0231241.g003.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/216e/7173866/bddf9728279f/pone.0231241.g004.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/216e/7173866/0a51b684cb83/pone.0231241.g005.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/216e/7173866/fd5b049c9103/pone.0231241.g001.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/216e/7173866/ec64529d463a/pone.0231241.g002.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/216e/7173866/174680741afc/pone.0231241.g003.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/216e/7173866/bddf9728279f/pone.0231241.g004.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/216e/7173866/0a51b684cb83/pone.0231241.g005.jpg

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A Bayesian credible subgroups approach to identifying patient subgroups with positive treatment effects.一种用于识别具有积极治疗效果的患者亚组的贝叶斯可信亚组方法。
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ESTIMATING PERSON-CENTERED TREATMENT (PeT) EFFECTS USING INSTRUMENTAL VARIABLES: AN APPLICATION TO EVALUATING PROSTATE CANCER TREATMENTS.使用工具变量估计以患者为中心的治疗(PeT)效果:在评估前列腺癌治疗中的应用。
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