论文标题

使用多个插补来分类潜在结果子组

Using Multiple Imputation to Classify Potential Outcomes Subgroups

论文作者

Li, Yun, Bondarenko, Irina, Elliott, Michael R., Hofer, Timothy P., Taylor, Jeremy M. G.

论文摘要

随着医疗测试的越来越多,对过度测试和过度治疗的担忧急剧增加。因此,重要的是要了解测试对一般实践中治疗选择的影响。大多数统计方法都集中在测试对治疗决策的平均影响。但是,这可能是不明智的,特别是对于往往不会从此类测试中受益的患者亚组。此外,丢失的数据很常见,代表了对统计方法有效性的大型且通常没有解决的威胁。最后,希望进行可因果关系解释的分析。我们建议将患者分为四个潜在结果亚组,这些结果是由患者的治疗结果以及测试结果如何改变治疗选择的方向而定义的。该亚组分类自然捕获了医疗测试对不同患者治疗选择的差异影响,这可以提出改善医疗测试利用的靶标。然后,我们可以检查与患者潜在结果亚组成员相关的患者特征。我们使用多种插补方法同时估算缺失的潜在结果以及常规的缺失值。这种方法还可以提供许多传统因果量的估计。我们发现,将因果推理假设明确地纳入多重归档过程可以提高某些因果关系估计的精度。我们还发现,当违反有条件的独立性假设的潜在结果时,可能会发生偏见。提出了敏感性分析来评估这种违规的影响。我们应用了所提出的方法来检查21基因测定最常用的基因组检验对乳腺癌患者化学疗法选择的影响。

With medical tests becoming increasingly available, concerns about over-testing and over-treatment dramatically increase. Hence, it is important to understand the influence of testing on treatment selection in general practice. Most statistical methods focus on average effects of testing on treatment decisions. However, this may be ill-advised, particularly for patient subgroups that tend not to benefit from such tests. Furthermore, missing data are common, representing large and often unaddressed threats to the validity of statistical methods. Finally, it is desirable to conduct analyses that can be interpreted causally. We propose to classify patients into four potential outcomes subgroups, defined by whether or not a patient's treatment selection is changed by the test result and by the direction of how the test result changes treatment selection. This subgroup classification naturally captures the differential influence of medical testing on treatment selections for different patients, which can suggest targets to improve the utilization of medical tests. We can then examine patient characteristics associated with patient potential outcomes subgroup memberships. We used multiple imputation methods to simultaneously impute the missing potential outcomes as well as regular missing values. This approach can also provide estimates of many traditional causal quantities. We find that explicitly incorporating causal inference assumptions into the multiple imputation process can improve the precision for some causal estimates of interest. We also find that bias can occur when the potential outcomes conditional independence assumption is violated; sensitivity analyses are proposed to assess the impact of this violation. We applied the proposed methods to examine the influence of 21-gene assay, the most commonly used genomic test, on chemotherapy selection among breast cancer patients.

扫码加入交流群

加入微信交流群

微信交流群二维码

扫码加入学术交流群,获取更多资源