Division of Pharmacoepidemiology and Pharmacoeconomics, Department of Medicine, Brigham and Women's Hospital and Harvard Medical School, Boston, Massachusetts, USA.
Clin Pharmacol Ther. 2018 Nov;104(5):1008-1015. doi: 10.1002/cpt.1045. Epub 2018 Mar 8.
In the presence of heterogeneity of treatment effect (HTE), the average treatment effect from a randomized controlled trial (RCT) may not be applicable to different patients, such as those in observational settings. Our objective was to develop a novel approach that uses individual-level simulation to expand RCT results to target patient populations in the presence of HTE. For this purpose, we compared the results of the Randomized Evaluation of Long-Term Anticoagulation Therapy (RE-LY) trial, and two observational studies that compared benefits and risks of dabigatran to warfarin in patients with atrial fibrillation. We developed a simulation model that replicates the rates of ischemic stroke and major bleeding observed in RE-LY using published outcome risk models and participants' baseline characteristics. We used our validated simulation model to predict what the results of the RCT would have been had it been conducted in populations similar to those in the observational studies.
在存在治疗效果异质性（HTE）的情况下，随机对照试验（RCT）的平均治疗效果可能不适用于不同的患者，例如观察性环境中的患者。我们的目的是开发一种新方法，该方法使用个体水平模拟将 RCT 结果扩展到存在 HTE 的目标患者人群。为此，我们比较了随机评估长期抗凝治疗（RE-LY）试验的结果，以及两项观察性研究的结果，这些研究比较了达比加群酯与华法林在房颤患者中的获益和风险。我们开发了一个模拟模型，该模型使用已发表的结局风险模型和参与者的基线特征来复制 RE-LY 中观察到的缺血性卒中和大出血发生率。我们使用经过验证的模拟模型来预测如果在类似于观察性研究的人群中进行 RCT，其结果将会是什么。
