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临床试验中比例的校准近似可信区间的样本量要求。

Sample Size Requirements for Calibrated Approximate Credible Intervals for Proportions in Clinical Trials.

机构信息

Dipartimento di Scienze Statistiche, Sapienza University of Rome, Piazzale Aldo Moro n. 5, 00185 Rome, Italy.

出版信息

Int J Environ Res Public Health. 2021 Jan 12;18(2):595. doi: 10.3390/ijerph18020595.

Abstract

In Bayesian analysis of clinical trials data, credible intervals are widely used for inference on unknown parameters of interest, such as treatment effects or differences in treatments effects. Highest Posterior Density (HPD) sets are often used because they guarantee the shortest length. In most of standard problems, closed-form expressions for exact HPD intervals do not exist, but they are available for intervals based on the normal approximation of the posterior distribution. For small sample sizes, approximate intervals may be not calibrated in terms of posterior probability, but for increasing sample sizes their posterior probability tends to the correct credible level and they become closer and closer to exact sets. The article proposes a predictive analysis to select appropriate sample sizes needed to have approximate intervals calibrated at a pre-specified level. Examples are given for interval estimation of proportions and log-odds.

摘要

在临床试验数据的贝叶斯分析中,可信区间被广泛用于对感兴趣的未知参数(如治疗效果或治疗效果差异)进行推断。高后验密度(HPD)集通常被使用,因为它们保证了最短的长度。在大多数标准问题中,不存在精确 HPD 区间的闭式表达式,但对于基于后验分布正态逼近的区间是可用的。对于小样本量,近似区间在后置概率方面可能没有校准,但随着样本量的增加,它们的后置概率趋于正确的可信水平,并且它们越来越接近精确集。本文提出了一种预测分析方法,以选择适当的样本量,以使近似区间在预定水平上得到校准。给出了比例和对数优势的区间估计示例。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/58b5/7827664/e02bf7766b7f/ijerph-18-00595-g001.jpg

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