A likely responder approach for the analysis of randomized controlled trials.
A likely responder approach for the analysis of randomized controlled trials.
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DOI：
10.1016/j.cct.2022.106688
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发表时间：
2022-03
影响因子：
2.2
通讯作者：
Lin Z
中科院分区：
文献类型：
--
作者：
Laska E;Siegel C;Lin Z
为了进一步实现根据个体患者特征定制医疗方案的精准医学目标，通过提供一种方法，分析测试治疗 T 与参考治疗 R 相比，RCT 参与者中可能对 T 产生反应的人。T 的可能反应者是指基线预期反应超过预先指定的最小值的个体。预后评分是根据基线协变量预测的预期反应，在试验完成时获得。它是一个平衡分数，可用于将随机分配到 T 的可能响应者与随机分配到 R 的可能响应者进行匹配；结果是具有共同协方差分布的可比较治疗组。根据该丰富样本中观察到的结果对治疗进行比较。该方法在一项随机对照试验中得到了阐述，该随机对照试验比较了阿片类药物使用障碍的两种治疗方法。阿片类药物使用障碍随机对照试验的标准统计分析发现，总样本中没有治疗差异。然而，确定了对 T 可能有反应的一个子集，并且在该组中，T 在统计上优于 R。在可能的反应者中，T 相对于 R 的因果治疗效果可能比整个目标人群中的效果更重要。预后评分函数提供定量信息，以支持患者有关 T 的特定治疗决策，从而进一步实现精准医疗的目标。
To further the precision medicine goal of tailoring medical treatment to individual patient characteristics by providing a method of analysis of the effect of test treatment, T, compared to a reference treatment, R, in participants in a RCT who are likely responders to T Likely responders to T are individuals whose expected response at baseline exceeds a prespecified minimum. A prognostic score, the expected response predicted as a function of baseline covariates, is obtained at trial completion. It is a balancing score that can be used to match likely responders randomized to T with those randomized to R; the result is comparable treatment groups that have a common covariance distribution. Treatments are compared based on observed outcomes in this enriched sample. The approach is illustrated in a RCT comparing two treatments for opioid use disorder. A standard statistical analysis of the opioid use disorder RCT found no treatment difference in the total sample. However, a subset of likely responders to T were identified and in this group, T was statistically superior to R. The causal treatment effect of T relative to R among likely responders may be more important than the effect in the whole target population. The prognostic score function provides quantitative information to support patient specific treatment decision regarding T furthering the goal of precision medicine.
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