A mixed-effects quintile-stratified propensity adjustment for effectiveness analyses of ordered categorical doses
A mixed-effects quintile-stratified propensity adjustment for effectiveness analyses of ordered categorical doses
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DOI：
10.1002/sim.2042
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发表时间：
2005-02-28
影响因子：
2
通讯作者：
Hedeker, D
中科院分区：
文献类型：
--
作者：
Leon, AC;Hedeker, D
观察性研究可用于评估疾病严重程度范围比随机对照临床试验中通常所见更广的患者的治疗有效性。然而，观察性评价存在一些困难，包括非等效比较组、治疗剂量和持续时间差异很大，以及在纵向研究中，每例受试者的多个疗程。一个混合效应的方法来调整非等效比较组的倾向，可以解释这些扰动。该战略包括两个阶段。首先，在使用混合效应有序逻辑回归的治疗强度倾向模型中检查接受不同水平治疗的受试者之间的区别特征。其次，在直至恢复的时间的混合效应分组时间生存模型中比较有序分类剂量的倾向分层有效性。该模型应用于抗抑郁药有效性的纵向观察性研究。蒙特卡洛模拟研究表明，该策略具有可接受的I类错误率和最小的偏差估计的治疗效果。当N = 250和500时，优势比为1.5时，统计功效超过0.90，当N = 100时，优势比为2.0时，统计功效是可以接受的。然而，在N = 100的情况下，具有高组内相关系数的模型具有更大的不收敛趋势。这种方法是治疗有效性观察性研究的有用策略。它能够调整选择偏差。合并每个受试者的多个观察结果，并比较顺序剂量的有效性。版权所有（C）2005约翰威利父子有限公司。
Observational studies can be used to evaluate treatment effectiveness among patients with a broader range of illness severity than typically seen in randomized controlled clinical trials. However, there are several difficulties with observational evaluations including non-equivalent comparison groups, treatment doses and durations that vary widely, and, in longitudinal studies, multiple courses of treatment per subject. A mixed-effects approach to the propensity adjustment for non-equivalent comparison groups is described that can account for each of these perturbations. The strategy involves two stages. First, characteristics that distinguish among subjects who receive various levels of treatment are examined in a model of propensity for treatment intensity using mixed-effects ordinal logistic regression. Second, the propensity-stratified effectiveness of ordered categorical doses is compared in a mixed-effects grouped time survival model of time until recovery. The model is applied in a longitudinal, observational study of antidepressant effectiveness. Then a Monte Carlo simulation study indicates that the strategy has acceptable type I error rates and minimal bias in the estimates of treatment effectiveness. Statistical power exceeds 0.90 for an odds ratio of 1.5 with N = 250 and 500, and is acceptable for an odds ratio of 2.0 with N = 100. Nevertheless, with N = 100, the models that had high intraclass correlation coefficients had greater tendency towards non-convergence. This approach is a useful strategy for observational studies of treatment effectiveness. It is capable of adjusting for selection bias.. incorporating multiple observations per subject, and comparing effectiveness of ordinal doses. Copyright (C) 2005 John Wiley Sons, Ltd.