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AI 根据所提供的论文材料归类:摘要研究随机对照试验平均处理效应的预后协变量调整,以及两阶段估计对效应推断方差的影响。

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关注这篇论文的作者
Nodoka Seya
Masataka Taguri

随机对照试验中预后协变量调整的第一阶段估计不确定性

研究问题
先用历史数据估计预后评分,再在随机试验中进行协方差分析时,忽略评分估计误差的方差估计是否仍然具有渐近有效性?
方法与思路
作者比较预后评分已知与估计两种情形的渐近方差,考虑具有 L₂ 一致性的机器学习评分,并构造计入或忽略评分误差的方差估计。
材料陈述的贡献
摘要称两种渐近方差相等,两类方差估计均渐近有效;通常建议使用较简单者,历史样本较小时可为保守推断考虑显式计入误差。
阅读关注点
应核对历史与试验样本的关系、渐近规模及评分一致性条件,以及方差相等的推导、小历史样本情形和保守性的证据。
阅读边界
这是 arXiv 预印本,同行评审或期刊接收未核实。摘要未列出完整正则条件,渐近方差相等并不保证两种估计在有限样本中等同。

原文摘要

Prognostic covariate adjustment (PROCOVA) is a two-sample two-stage estimation method for covariate adjustment in randomized controlled trials. In the first stage, a prognostic score, defined as the conditional expectation of an outcome given covariates under the control treatment, is estimated using historical data. In the second stage, analysis of covariance with the estimated prognostic score and treatment assignment as explanatory variables is performed, and the average treatment effect is estimated. Although the prognostic score is estimated in this procedure, the variance estimator, which treats the prognostic score as known, has been used. Furthermore, the difference in the asymptotic variance between cases where the prognostic score is known versus where it is estimated has not been previously clarified. In this study, we derived these two asymptotic variances and showed that they are equal. This result also holds when the prognostic score is estimated using machine learning with $L_2$ consistency. We also constructed two variance estimators: one that treats the prognostic score as known, and another that accounts for its estimation, and compared their performance through simulation studies and data applications. For PROCOVA, since both variance estimators are asymptotically valid, it is generally recommended to use a variance estimator that treats the prognostic score as known, as it is simpler to derive and implement. When historical data is small, a variance estimator that explicitly accounts for prognostic score estimation is recommended if conservative inference is preferred.

笔记与分组