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用于单病例试验的贝叶斯模型。

Bayesian Models for N-of-1 Trials.

作者信息

Schmid Christopher, Yang Jiabei

机构信息

Department of Biostatistics, School of Public Health, Brown University, Providence, Rhode Island, United States of America.

出版信息

Harv Data Sci Rev. 2022;2022(SI3). doi: 10.1162/99608f92.3f1772ce. Epub 2022 Sep 8.

Abstract

We describe Bayesian models for data from N-of-1 trials, reviewing both the basics of Bayesian inference and applications to data from single trials and collections of trials sharing the same research questions and data structures. Bayesian inference is natural for drawing inferences from N-of-1 trials because it can incorporate external and subjective information to supplement trial data as well as give straightforward interpretations of posterior probabilities as an individual's state of knowledge about their own condition after their trial. Bayesian models are also easily augmented to incorporate specific characteristics of N-of-1 data such as trend, carryover, and autocorrelation and offer flexibility of implementation. Combining data from multiple N-of-1 trials using Bayesian multilevel models leads naturally to inferences about population and subgroup parameters such as average treatment effects and treatment effect heterogeneity and to improved inferences about individual parameters. Data from a trial comparing different diets for treating children with inflammatory bowel disease are used to illustrate the models and inferences that may be drawn. The analysis shows that certain diets were better on average at reducing pain, but that benefits were restricted to a subset of patients and that withdrawal from the study was a good marker for lack of benefit.

摘要

我们描述了针对单病例试验数据的贝叶斯模型,回顾了贝叶斯推断的基础知识以及其在来自单个试验和具有相同研究问题及数据结构的试验集合的数据中的应用。贝叶斯推断对于从单病例试验中得出推断很自然,因为它可以纳入外部和主观信息来补充试验数据,并且能将后验概率直接解释为个体在试验后对自身状况的了解状态。贝叶斯模型也很容易扩展以纳入单病例数据的特定特征,如趋势、残留效应和自相关,并提供实施的灵活性。使用贝叶斯多层次模型组合来自多个单病例试验的数据自然会得出关于总体和亚组参数的推断,如平均治疗效果和治疗效果异质性,并能改进对个体参数的推断。一项比较不同饮食治疗炎症性肠病儿童的试验数据被用于说明可能得出的模型和推断。分析表明,某些饮食平均而言在减轻疼痛方面效果更好,但益处仅限于一部分患者,并且退出研究是缺乏益处的一个良好指标。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/568c/10817775/683a5412a0d7/nihms-1883199-f0001.jpg

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