Observational data for comparative effectiveness research: An emulation of randomised trials of statins and primary prevention of coronary heart disease
Observational data for comparative effectiveness research: An emulation of randomised trials of statins and primary prevention of coronary heart disease
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DOI：
10.1177/0962280211403603
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发表时间：
2013-02-01
影响因子：
2.3
通讯作者：
Hernan, Miguel A.
中科院分区：
文献类型：
--
作者：
Danaei, Goodarz;Garcia Rodriguez, Luis A.;Hernan, Miguel A.
本文综述了使用观察数据进行比较有效性研究的方法。其基本思想是使用观察性研究来模拟一个假设的随机试验，通过比较治疗的启动者与非启动者。在对测量的基线混杂因素进行调整后，可以进行意向治疗分析的观察模拟。我们还解释了两种方法，以进行类似的每协议和治疗分析后，进一步调整测量的时变混杂和选择偏倚，使用逆概率加权。作为一个例子，我们实施了这些方法来估计他汀类药物的效果，用于冠心病（CHD）的一级预防，使用的数据来自英国的电子病历。尽管适应症有很强的混杂因素，但我们的方法检测到他汀类药物治疗的潜在益处。他汀类药物启动者与非启动者的CHD意向治疗风险比（HR）模拟值为0.89（0.73，1.09）。在符合方案分析中，CHD的HR为0.84（0.54，1.30），在2年使用与未使用的实际治疗分析中为0.79（0.41，1.41）。相比之下，目前使用者与从未使用过他汀类药物治疗者的常规比较导致HR为1.31（1.04，1.66）。我们提供了一个灵活的和注释的SAS程序来实现所提出的分析。
This article reviews methods for comparative effectiveness research using observational data. The basic idea is using an observational study to emulate a hypothetical randomised trial by comparing initiators versus non-initiators of treatment. After adjustment for measured baseline confounders, one can then conduct the observational analogue of an intention-to-treat analysis. We also explain two approaches to conduct the analogues of per-protocol and as-treated analyses after further adjusting for measured time-varying confounding and selection bias using inverse-probability weighting. As an example, we implemented these methods to estimate the effect of statins for primary prevention of coronary heart disease (CHD) using data from electronic medical records in the UK. Despite strong confounding by indication, our approach detected a potential benefit of statin therapy. The analogue of the intention-to-treat hazard ratio (HR) of CHD was 0.89 (0.73, 1.09) for statin initiators versus non-initiators. The HR of CHD was 0.84 (0.54, 1.30) in the per-protocol analysis and 0.79 (0.41, 1.41) in the as-treated analysis for 2 years of use versus no use. In contrast, a conventional comparison of current users versus never users of statin therapy resulted in a HR of 1.31 (1.04, 1.66). We provide a flexible and annotated SAS program to implement the proposed analyses.