n(1)次试验的贝叶斯模型

Harvard data science review Pub Date : 2022-01-01 Epub Date: 2022-09-08 DOI:10.1162/99608f92.3f1772ce
Christopher Schmid, Jiabei Yang
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引用次数: 0

摘要

我们介绍了 N-of-1 试验数据的贝叶斯模型,回顾了贝叶斯推断的基本原理以及在单个试验数据和具有相同研究问题和数据结构的试验集合中的应用。贝叶斯推断法是从 N-of-1 试验中得出推论的自然方法,因为它可以结合外部和主观信息来补充试验数据,并将后验概率直接解释为个体在试验后对自身情况的了解程度。贝叶斯模型也很容易进行扩展,以纳入 N-of-1 数据的特定特征,如趋势、结转和自相关性,并提供实施的灵活性。使用贝叶斯多层次模型将来自多个 N-of-1 试验的数据结合起来,自然可以推断出群体和亚组参数,如平均治疗效果和治疗效果异质性,并改进对个体参数的推断。本研究使用了一项比较不同饮食治疗炎症性肠病患儿的试验数据来说明模型和可能得出的推论。分析表明,某些饮食在减轻疼痛方面平均效果较好,但获益者仅限于部分患者,退出研究是缺乏获益的良好标志。
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Bayesian Models for N-of-1 Trials.

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.

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