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利用机器学习推断处理特定生存曲线AI 解读

In the absence of data from a randomized trial, researchers may aim to use observational data to draw causal inference about the effect of a treatment on a time-to-event outcome. In this context, interest often focuses on the treatment-specific survival curves, that is, the survival curves were the population under study to be assigned to receive the treatment or not. Under certain conditions, including that all confounders of the treatment-outcome relationship are observed,…

利用机器学习估计和检验疫苗筛效应AI 解读

When available, vaccines are an effective means of disease prevention. Unfortunately, efficacious vaccines have not yet been developed for several major infectious diseases, including HIV and malaria. Vaccine sieve analysis studies whether and how the efficacy of a vaccine varies with the genetics of the pathogen of interest, which can guide subsequent vaccine development and deployment. In sieve analyses, the effect of the vaccine on the cumulative incidence corresponding…

随机临床试验中依方案生存终点疗效的敏感性分析AI 解读

Assessing per-protocol treatment effcacy on a time-to-event endpoint is a common objective of randomized clinical trials. The typical analysis uses the same method employed for the intention-to-treat analysis (e.g., standard survival analysis) applied to the subgroup meeting protocol adherence criteria. However, due to potential post-randomization selection bias, this analysis may mislead about treatment efficacy. Moreover, while there is extensive literature on methods for…

HIV预防试验中时变主分层的部分隐马尔可夫模型AI 解读

It is frequently of interest to estimate the intervention effect that adjusts for post-randomization variables in clinical trials. In the recently completed HPTN 035 trial, there is differential condom use between the three microbicide gel arms and the No Gel control arm, so that intention to treat (ITT) analyses only assess the net treatment effect that includes the indirect treatment effect mediated through differential condom use. Various statistical methods in causal…

随机化后事件发生才观测结局时，给定基线协变量的治疗效应半参数估计AI 解读

Summary We consider estimation, from a double-blind randomized trial, of treatment effect within levels of base-line covariates on an outcome that is measured after a post-treatment event E has occurred in the subpopulation 𝒫E,E that would experience event E regardless of treatment. Specifically, we consider estimation of the parameters γ indexing models for the outcome mean conditional on treatment and base-line covariates in the subpopulation 𝒫E,E. Such parameters are…

半参数偏倚抽样模型中最大似然估计的大样本理论AI 解读

Vardi [Ann.Statist.13 178-203 (1985)] introduced an $s$-sample biased sampling model with known selection weight functions, gave a condition under which the common underlying probability distribution $G$ is uniquely estimable and developed simple procedure for computing the nonparametric maximum likelihood estimator (NPMLE) $\mathbb{G}_n$ of $G$. Gill, Vardi and Wellner thoroughly described the large sample properties of Vardi’s NPMLE, giving results on uniform consistency,…