Assessing the heterogeneity of treatment effects via potential outcomes of individual patients.
Assessing the heterogeneity of treatment effects via potential outcomes of individual patients.
复制标题
DOI：
10.1111/rssc.12012
复制
发表时间：
2013-11
期刊：
影响因子：
--
通讯作者：
Soon G
中科院分区：
文献类型：
--
作者：
Zhang Z;Wang C;Nie L;Soon G
治疗效果的异质性对治疗方案的评价和选择具有重要意义，人们对治疗效果异质性的研究日益受到重视。评估HTE的标准方法（即基于已知效应调节剂的亚组分析）可提供关于亚组之间异质性的信息，但不能提供亚组内异质性的信息。可以说，根据个体治疗效果评估HTE更能提供信息，这可以通过使用潜在结果来定义。然而，基于潜在结果的HTE估计受到缺乏完全可识别性的挑战。本文提出了利用基线协变量和重复测量中的相关信息来处理可辨识性问题的方法。如果一组协变量足以解释潜在结局之间的依赖性，则将在条件独立性假设下确定潜在结局的联合分布以及所有HTE指标。可能违反此假设的情况可以通过包含随机效应来解释剩余依赖或直接指定条件依赖结构来解决。所提出的方法被证明是有效地减少在人类免疫缺陷病毒的试验HTE的不确定性。
There is growing interest in understanding the heterogeneity of treatment effects (HTE), which has important implications in treatment evaluation and selection. The standard approach to assessing HTE (i.e. subgroup analyses based on known effect modifiers) is informative about the heterogeneity between subpopulations but not within. It is arguably more informative to assess HTE in terms of individual treatment effects, which can be defined by using potential outcomes. However, estimation of HTE based on potential outcomes is challenged by the lack of complete identifiability. The paper proposes methods to deal with the identifiability problem by using relevant information in baseline covariates and repeated measurements. If a set of covariates is sufficient for explaining the dependence between potential outcomes, the joint distribution of potential outcomes and hence all measures of HTE will then be identified under a conditional independence assumption. Possible violations of this assumption can be addressed by including a random effect to account for residual dependence or by specifying the conditional dependence structure directly. The methods proposed are shown to reduce effectively the uncertainty about HTE in a trial of human immunodeficiency virus.
登录
查看更多内容
影响因子：
2.7
作者：
CARROLL, RJ;SPIEGELMAN, CH;ABBOTT, RD
通讯作者：
ABBOTT, RD
影响因子：
1.8
作者：
Stefanski, LA;Boos, DD
通讯作者：
Boos, DD
DOI：
10.1056/nejmoa0803152
发表时间：
2008-10-02
期刊：
The New England journal of medicine
影响因子：
--
作者：
Gulick RM;Lalezari J;Goodrich J;Clumeck N;DeJesus E;Horban A;Nadler J;Clotet B;Karlsson A;Wohlfeiler M;Montana JB;McHale M;Sullivan J;Ridgway C;Felstead S;Dunne MW;van der Ryst E;Mayer H;MOTIVATE Study Teams
通讯作者：
MOTIVATE Study Teams
DOI：
10.1080/00031305.2012.671724
发表时间：
2012
期刊：
The American statistician
影响因子：
--
作者：
Poulson RS;Gadbury GL;Allison DB
通讯作者：
Allison DB
影响因子：
158.5
作者：
Faetkenheuer, Gerd;Nelson, Mark;van der Ryst, Elna
通讯作者：
van der Ryst, Elna