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On the limitations of causal inference with current-treatment Cox models

仅含当前治疗的 Cox 模型在因果推断中的局限

Cox models with time-varying treatments often include only the current treatment level. Translating such a model into causally meaningful intervention-specific survival probabilities relies on the Markov property: that the hazard is independent of treatment history conditional on current treatment. For the Markov property to not be population-specific, it needs to hold also conditional on any unmeasured prognostic heterogeneity (frailty). Using a discrete-time argument,…

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Estimation of treatment effects in presence of differential use of post-randomization concomitant medication with time-to-event outcomes

随机化后伴随用药差异下的生存结局处理效应估计

In placebo-controlled randomized trials, the post-randomization use of concomitant medications may be higher in the placebo arm than in the treatment arm. This may dilute the full benefits of the randomized drug as estimated by the intention-to-treat analysis. We focus on cardiovascular outcomes trials in type-2 diabetes patients of glucose-lowering treatments where patients in the placebo arm are more likely to add other glucose-lowering agents with established…

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Improving precision of cumulative incidence estimates in randomized controlled trials with external controls

利用外部对照提高随机试验累积发生率估计的精度

Augmenting the control arm in clinical trials with external data can improve statistical power for demonstrating treatment effects. In many time-to-event outcome trials, participants are subject to truncation by death. Direct application of methods for competing risks analysis on the joint data may introduce bias, for example, due to covariate shifts between the populations. In this work, we consider transportability of the conditional cause-specific hazard of the event of…

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Proximal indirect comparison

近端间接比较

We consider the problem of indirect comparison, where a treatment arm of interest is absent by design in one randomized controlled trial but available in the other. The former is the target trial, and the latter is the source trial. The identifiability of the target population average treatment effect often relies on conditional transportability assumptions. However, it is a common concern whether all relevant effect modifiers are measured and controlled for. We give a new…

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Nonparametric efficient causal estimation of the intervention-specific expected number of recurrent events with continuous-time targeted maximum likelihood and highly adaptive lasso estimation

连续时间靶向极大似然与高度自适应 Lasso 的干预特定复发事件期望次数估计

Longitudinal settings involving outcome, competing risks and censoring events occurring and recurring in continuous time are common in medical research, but are often analyzed with methods that do not allow for taking post-baseline information into account. In this work, we define statistical and causal target parameters via the g-computation formula by carrying out interventions directly on the product integral representing the observed data distribution in a…

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Ranking of average treatment effects with generalized random forests for time-to-event outcomes

利用广义随机森林排序事件时间结果的平均处理效应

In this paper we present a data-adaptive estimation procedure for estimation of average treatment effects in a time-to-event setting based on generalized random forests. In these kinds of settings, the definition of causal effect parameters are complicated by competing risks; here we distinguish between treatment effects on the crude and the net probabilities, respectively. To handle right-censoring, and to switch between crude and net probabilities, we propose a two-step…

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