Combining single patient (N-of-1) trials to estimate population treatment effects and to evaluate individual patient responses to treatment
Combining single patient (N-of-1) trials to estimate population treatment effects and to evaluate individual patient responses to treatment
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DOI：
10.1016/s0895-4356(96)00429-5
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发表时间：
1997-04-01
影响因子：
7.2
通讯作者：
Lau, J
中科院分区：
文献类型：
--
作者：
Zucker, DR;Schmid, CH;Lau, J
在治疗个别患者时，由于这些研究的普遍性有限，医生在使用来自中心的随机对照试验 (RCT) 的证据时可能会遇到困难。因此，他们经常对治疗效果进行自己的“非正式”测试。单患者（“N-of-1”）试验提供了一种结构化设计，可以更严格地评估慢性病的药物治疗，但仅适用于指标患者。我们提出了一个分层贝叶斯随机效应模型，结合 N-of-1 研究来获得对人群治疗效果的估计，并使用该人群信息来帮助评估个体患者的试验结果。该模型的治疗效果估计是总体估计和个体观察结果之间的调整。这种调整基于患者内部和患者之间的异质性。我们使用来自 23 项 N-of-1 试验结果的已发表数据证明了这种以患者为中心的方法，比较阿米替林和安慰剂治疗纤维肌痛的效果。 (C) 1997 爱思唯尔科学公司。
When treating individual patients, physicians may fare difficulties using the evidence from center-based randomized control trials (RCTs) due to limitations in these studies' generalizability. Therefore, they often perform their own ''informal'' tests of treatment effectiveness. Single patient (''N-of-1'') trials provide a structured design for more rigorous assessment of medical treatments of chronic diseases, but are applied only to the index patient. We present a hierarchical Bayesian random effects model to combine N-of-1 studies to obtain an estimate of treatment effectiveness for the population and to use this population information to aid in the evaluation of an individual patient's trial results. The model's treatment effect estimates are adjustments between the population estimate and the individual's observed results. This adjustment is based upon the within-patient and between patient heterogeneity. We demonstrate this patient-focused method using published data from 23 N-of-1 trial results comparing amitriptyline and placebo for the treatment of fibromyalgia. (C) 1997 Elsevier Science Inc.