Treatment benefit and treatment harm rate to characterize heterogeneity in treatment effect.
Treatment benefit and treatment harm rate to characterize heterogeneity in treatment effect.
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治疗获益率和治疗危害率来表征治疗效果的异质性。
DOI：
10.1111/biom.12038
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发表时间：
2013
期刊：
影响因子：
1.9
通讯作者：
Buxton,Alfred
中科院分区：
文献类型：
--
作者：
Shen,Changyu;Jeong,Jaesik;Li,Xiaochun;Chen,Peng-Sheng;Buxton,Alfred
众所周知，通过对个体患者进行平均来总结治疗效果的传统方法有其局限性，因为它忽略了目标人群对治疗的异质反应。然而，文献中很少有旨在捕捉这种异质性的替代指标。我们提出了治疗获益率（TBR）和治疗危害率（THR）的概念，它们表征了总体治疗效果和异质性程度。我们讨论了一种估计 TBR 和 THR 的方法，该方法可以轻松地结合敏感性分析方案，并通过对评估植入式心律转复除颤器 (ICD) 降低死亡率的随机试验的分析来说明这一想法。提出了一项模拟研究来评估所提出方法的性能。
It is well recognized that the conventional summary of treatment effect by averaging across individual patients has its limitation in ignoring the heterogeneous responses to the treatment in the target population. However, there are few alternative metrics in the literature that are designed to capture such heterogeneity. We propose the concept of treatment benefit rate (TBR) and treatment harm rate (THR) that characterize both the overall treatment effect and the magnitude of heterogeneity. We discuss a method to estimate TBR and THR that easily incorporates a sensitivity analysis scheme, and illustrate the idea through analysis of a randomized trial that evaluates the implantable cardioverter-defibrillator (ICD) in reducing mortality. A simulation study is presented to assess the performance of the proposed method.