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A recent cohort study revealed a positive correlate between major structural birth defects in infants and a certain medication taken by pregnant women. To draw valid causal inference, an outstanding problem to overcome was the missing birth…

应用统计 · 统计学 2022-06-09 Andrew Ying , Ronghui Xu , Christina D. Chambers , Kenneth Lyons Jones

Measurement error in observational datasets can lead to systematic bias in inferences based on these datasets. As studies based on observational data are increasingly used to inform decisions with real-world impact, it is critical that we…

机器学习 · 统计学 2019-01-29 Roy Adams , Yuelong Ji , Xiaobin Wang , Suchi Saria

We describe the Bedside Patient Rescue (BPR) project, the goal of which is risk prediction of adverse events for non-ICU patients using ~200 variables (vitals, lab results, assessments, ...). There are several missing predictor values for…

Missing data are ubiquitous in public health research. When estimating causal effects, there are well-established methods to address bias to due missing outcomes. Commonly, causal estimands are defined under hypothetical interventions to…

Standard methods for estimating average causal effects require complete observations of the exposure and confounders. In observational studies, however, missing data are ubiquitous. Motivated by a study on the effect of prescription opioids…

统计方法学 · 统计学 2025-06-30 Lan Wen , Glen McGee

Inferring causal effects of treatments is a central goal in many disciplines. The potential outcomes framework is a main statistical approach to causal inference, in which a causal effect is defined as a comparison of the potential outcomes…

统计方法学 · 统计学 2018-01-04 Peng Ding , Fan Li

Understanding how effective high-level NICUs (neonatal intensive care units that have the capacity for sustained mechanical assisted ventilation and high volume) are compared to low-level NICUs is important and valuable for both individual…

应用统计 · 统计学 2014-04-10 Fan Yang , Scott A. Lorch , Dylan S. Small

In observational studies, the identification of causal estimands depends on the no unmeasured confounding (NUC) assumption. As this assumption is not testable from observed data, sensitivity analysis plays an important role in observational…

统计方法学 · 统计学 2023-09-28 Md Abdul Basit , Mahbub A. H. M. Latif , Abdus S Wahed

Pregnancy loss is recognized as an important competing event in studies of prenatal medication use. However, a healthy live birth also precludes subsequent adverse pregnancy outcomes, yet these events are often censored. Using Monte Carlo…

Measurement error arises commonly in clinical research settings that rely on data from electronic health records or large observational cohorts. In particular, self-reported outcomes are typical in cohort studies for chronic diseases such…

统计方法学 · 统计学 2021-02-08 Lillian A. Boe , Lesley F. Tinker , Pamela A. Shaw

PURPOSE: Clinical examinations are performed on the basis of necessity. However, our decisions to investigate and document are influenced by various other factors, such as workload and preconceptions. Data missingness patterns may contain…

应用统计 · 统计学 2019-12-19 Robert O'Shea

Electronic health record (EHR)-linked biobank data hold tremendous promise for large-scale discoveries via genome-wide association study (GWAS) on diverse phenotypic traits and biomarkers routinely captured in the EHR. However,…

应用统计 · 统计学 2026-04-14 Xingran Chen , Cheng-Han Yang , Zhenke Wu , Bhramar Mukherjee

While observational data are routinely used to estimate causal effects of biomedical treatments, doing so requires special methods to adjust for observed confounding. These methods invariably rely on untestable statistical and causal…

统计方法学 · 统计学 2026-03-02 Arman Oganisian

The analysis of randomized trials is often complicated by the occurrence of intercurrent events and missing values. Even though there are different strategies to address missing values it is still common to require missing values…

统计方法学 · 统计学 2025-11-11 A. Ruiz de Villa , Ll. Badiella

The interventional effects approach to causal mediation analysis is increasingly common in epidemiologic research, given its potential to address policy-relevant questions about hypothetical mediator interventions. Multiple imputation (MI)…

Infant mortality remains a significant public health concern in the United States, with birth defects identified as a leading cause. Despite ongoing efforts to understand the causes of negative pregnancy outcomes like miscarriage,…

计算与语言 · 计算机科学 2025-12-30 Md Badsha Biswas

Evaluating treatment effects is critical in clinical trials but sometimes involves lengthy, invasive, or costly follow-up procedures. In these cases, surrogate markers, which provide intermediate measures of the long-term treatment effect,…

统计方法学 · 统计学 2026-03-24 Sarah C. Lotspeich , P. D. Anh. Nguyen , Layla Parast

Targeted Maximum Likelihood Estimation (TMLE) is increasingly used for doubly robust causal inference, but how missing data should be handled when using TMLE with data-adaptive approaches is unclear. Based on the Victorian Adolescent Health…

We evaluate the performance of targeted maximum likelihood estimation (TMLE) for estimating the average treatment effect in missing data scenarios under varying levels of positivity violations. We employ model- and design-based simulations,…

统计方法学 · 统计学 2026-05-12 Christoph Wiederkehr , Christian Heumann , Michael Schomaker

In causal inference, sensitivity analysis is important to assess the robustness of study conclusions to key assumptions. We perform sensitivity analysis of the assumption that missing outcomes are missing completely at random. We follow a…

统计理论 · 数学 2023-05-12 Bart Eggen , Stéphanie L. van der Pas , Aad W. van der Vaart
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