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Ellen C. Caniglia

Publications and source records attributed to Ellen C. Caniglia.

2 recordsLinked to original sources

Timescales in Time-to-Event Analyses in Pregnancy

The target trial framework is increasingly used to define the causal estimand for an observational analysis. However, designing time-to-event analyses that align with the causal estimand is challenging in pregnancy because the timescale on which outcomes are often defined, "gestational age", differs from the timescale on which outcomes are measured, "time since study entry". We argue that neither timescale is "best" but that each aligns with a different estimand. In this article, we use the target trial framework to define the causal estimands corresponding to each timescale. We consider a (hypothetical) target trial comparing randomization to 17-alpha hydroxyprogesterone caproate (17-OHPC) versus placebo between 16-20 weeks of gestation to prevent delivery before 37 weeks' gestation, whether by miscarriage, stillbirth, or live birth. We define the estimand of time-to-event analyses on the "time since study entry" timescale as the effect of randomization to 17-OHPC versus placebo between 16-20 weeks of gestation on delivery before 37 weeks'. In contrast, the causal estimand of time-to-event analyses using the "gestational age" timescale corresponds to the effect had, counter to fact, all participants been randomized at one gestational age (e.g., 16 weeks). We review specific challenges to estimation and possible solutions for each timescale.

stat.AP↗

Sensitivity Analysis when Generalizing Causal Effects from Multiple Studies to a Target Population: Motivation from the ECHO Program

Unobserved effect modifiers can induce bias when generalizing causal effect estimates to target populations. In this work, we extend a sensitivity analysis framework assessing the robustness of study results to unobserved effect modification that adapts to various generalizability scenarios, including multiple (conditionally) randomized trials, observational studies, or combinations thereof. This framework is interpretable and does not rely on distributional or functional assumptions about unknown parameters. We demonstrate how to leverage the multi-study setting to detect violation of the generalizability assumption through hypothesis testing, showing with simulations that the proposed test achieves high power under real-world sample sizes. Finally, we apply our sensitivity analysis framework to analyze the generalized effect estimate of secondhand smoke exposure on birth weight using cohort sites from the Environmental influences on Child Health Outcomes (ECHO) study.

stat.ME↗