Open Access. Powered by Scholars. Published by Universities.®
- Discipline
- Institution
- Publication Year
- Publication
- Publication Type
Articles 1 - 22 of 22
Full-Text Articles in Biostatistics
Statistical Methods For Assessing Drug Interactions And Identifying Effect Modifiers Using Observational Data., Qian Xu
Electronic Theses and Dissertations
This dissertation consists of three projects related to causal inference based on observational data. In the first project, we propose a double robust to identify the effect modifiers and estimate optimal treatment. Observational studies differ from experimental studies in that assignment of subjects to treatments is not randomized but rather occurs due to natural mechanisms, which are usually hidden from the researchers. Many statistical methods to identify the treatment effect and select the optimal personalized treatment for experimental studies may not be suitable for observational studies any more. In this project, we propose a exible outcome model to select the …
Measurements Of Generalizability And Adjustment For Bias In Clinical Trials, Yuanyuan Lu
Measurements Of Generalizability And Adjustment For Bias In Clinical Trials, Yuanyuan Lu
USF Tampa Graduate Theses and Dissertations
While randomized controlled trials (RCTs) are widely used as a gold standard in clinical research and public health, they are criticized because of a potential lack of generalizability, as the trial patients may be unrepresentative of the target patient population. Few research addresses how to assess and evaluate the generalizability of RCTs. As we know, patients are rarely selected on a random basis from a well-defined patient population of interest into a clinical trial. Generalizing findings from the RCT samples to the patient population has begun to receive increasing attention. We simulate a patient population with treatment effect size of …
Modified-Half-Normal Distribution And Different Methods To Estimate Average Treatment Effect., Jingchao Sun
Modified-Half-Normal Distribution And Different Methods To Estimate Average Treatment Effect., Jingchao Sun
Electronic Theses and Dissertations
This dissertation consists of three projects related to Modified-Half-Normal distribution and causal inference. In my first project, a new distribution called Modified-Half-Normal distribution was introduced. I explored a few of its distributional properties, the procedures for generating random samples based on Bayesian approaches, and the parameter estimation based on the method of moments. The second project deals with the problem of selection bias of average treatment effect (ATE) if we use the observational data. I combined the propensity score based inverse probability of treatment weighting (IPTW) method and the directed acyclic graph (DAG) to solve this problem. The third project …
Aspects Of Causal Inference., John A. Craycroft
Aspects Of Causal Inference., John A. Craycroft
Electronic Theses and Dissertations
Observational studies differ from experimental studies in that assignment of subjects to treatments is not randomized but rather occurs due to natural mechanisms, which are usually hidden from researchers. Yet objectives of the two studies are frequently the same: identify the causal – rather than merely associational – relationship between some treatment or exposure and an outcome. The statistical issues that arise in properly analyzing observational data for this goal are numerous and fascinating, and these issues are encompassed in the domain of causal inference. The research presented in this dissertation explores several distinct aspects of causal inference. This dissertation …
Estimating Marginal Hazard Ratios By Simultaneously Using A Set Of Propensity Score Models: A Multiply Robust Approach, Di Shu, Peisong Han, Rui Wang, Sengwee Toh
Estimating Marginal Hazard Ratios By Simultaneously Using A Set Of Propensity Score Models: A Multiply Robust Approach, Di Shu, Peisong Han, Rui Wang, Sengwee Toh
Harvard University Biostatistics Working Paper Series
The inverse probability weighted Cox model is frequently used to estimate marginal hazard ratios. Its validity requires a crucial condition that the propensity score model is correctly specified. To provide protection against misspecification of the propensity score model, we propose a weighted estimation method rooted in empirical likelihood theory. The proposed estimator is multiply robust in that it is guaranteed to be consistent when a set of postulated propensity score models contains a correctly specified model. Our simulation studies demonstrate satisfactory finite sample performance of the proposed method in terms of consistency and efficiency. We apply the proposed method to …
Statistical Methods For Estimating And Testing Treatment Effect For Multiple Treatment Groups In Observational Studies., Xiaofang Yan
Statistical Methods For Estimating And Testing Treatment Effect For Multiple Treatment Groups In Observational Studies., Xiaofang Yan
Electronic Theses and Dissertations
Note: Abstract would not save due to an issue with some of the characters.
Controlling For Confounding Via Propensity Score Methods Can Result In Biased Estimation Of The Conditional Auc: A Simulation Study, Hadiza I. Galadima, Donna K. Mcclish
Controlling For Confounding Via Propensity Score Methods Can Result In Biased Estimation Of The Conditional Auc: A Simulation Study, Hadiza I. Galadima, Donna K. Mcclish
Community & Environmental Health Faculty Publications
In the medical literature, there has been an increased interest in evaluating association between exposure and outcomes using nonrandomized observational studies. However, because assignments to exposure are not random in observational studies, comparisons of outcomes between exposed and nonexposed subjects must account for the effect of confounders. Propensity score methods have been widely used to control for confounding, when estimating exposure effect. Previous studies have shown that conditioning on the propensity score results in biased estimation of conditional odds ratio and hazard ratio. However, research is lacking on the performance of propensity score methods for covariate adjustment when estimating the …
Using Sensitivity Analyses For Unobserved Confounding To Address Covariate Measurement Error In Propensity Score Methods, Kara E. Rudolph, Elizabeth A. Stuart
Using Sensitivity Analyses For Unobserved Confounding To Address Covariate Measurement Error In Propensity Score Methods, Kara E. Rudolph, Elizabeth A. Stuart
Johns Hopkins University, Dept. of Biostatistics Working Papers
Propensity score methods are a popular tool to control for confounding in observational data, but their bias-reduction properties are threatened by covariate measurement error. There are few easy-to-implement methods to correct for such bias. We describe and demonstrate how existing sensitivity analyses for unobserved confounding---propensity score calibration, Vanderweele and Arah's bias formulas, and Rosenbaum's sensitivity analysis---can be adapted to address this problem. In a simulation study, we examined the extent to which these sensitivity analyses can correct for several measurement error structures: classical, systematic differential, and heteroscedastic covariate measurement error. We then apply these approaches to address covariate measurement error …
Moving Towards Best Practice When Using Inverse Probability Of Treatment Weighting (Iptw) Using The Propensity Score To Estimate Causal Treatment Effects In Observational Studies, Peter Austin, Elizabeth Stuart
Moving Towards Best Practice When Using Inverse Probability Of Treatment Weighting (Iptw) Using The Propensity Score To Estimate Causal Treatment Effects In Observational Studies, Peter Austin, Elizabeth Stuart
Peter Austin
The propensity score is defined as a subject’s probability of treatment selection, conditional on observed baseline covariates.Weighting subjects by the inverse probability of treatment received creates a synthetic sample in which treatment assignment is independent of measured baseline covariates. Inverse probability of treatment weighting (IPTW) using the propensity score allows one to obtain unbiased estimates of average treatment effects. However, these estimates are only valid if there are no residual systematic differences in observed baseline characteristics between treated and control subjects in the sample weighted by the estimated inverse probability of treatment. We report on a systematic literature review, in …
Constrained Bayesian Estimation Of Inverse Probability Weights For Nonmonotone Missing Data, Baoluo Sun, Eric J. Tchetgen Tchetgen
Constrained Bayesian Estimation Of Inverse Probability Weights For Nonmonotone Missing Data, Baoluo Sun, Eric J. Tchetgen Tchetgen
Harvard University Biostatistics Working Paper Series
No abstract provided.
A Comparison Of 12 Algorithms For Matching On The Propensity Score, Peter C. Austin
A Comparison Of 12 Algorithms For Matching On The Propensity Score, Peter C. Austin
Peter Austin
Propensity-score matching is increasingly being used to reduce the confounding that can occur in observational studies examining the effects of treatments or interventions on outcomes. We used Monte Carlo simulations to examine the following algorithms for forming matched pairs of treated and untreated subjects: optimal matching, greedy nearest neighbor matching without replacement, and greedy nearest neighbor matching without replacement within specified caliper widths. For each of the latter two algorithms, we examined four different sub-algorithms defined by the order in which treated subjects were selected for matching to an untreated subject: lowest to highest propensity score, highest to lowest propensity …
The Use Of Propensity Score Methods With Survival Or Time-To-Event Outcomes: Reporting Measures Of Effect Similar To Those Used In Randomized Experiments, Peter C. Austin
The Use Of Propensity Score Methods With Survival Or Time-To-Event Outcomes: Reporting Measures Of Effect Similar To Those Used In Randomized Experiments, Peter C. Austin
Peter Austin
Propensity score methods are increasingly being used to estimate causal treatment effects in observational studies. In medical and epidemiological studies, outcomes are frequently time-to-event in nature. Propensity-score methods are often applied incorrectly when estimating the effect of treatment on time-to-event outcomes. This article describes how two different propensity score methods (matching and inverse probability of treatment weighting) can be used to estimate the measures of effect that are frequently reported in randomized controlled trials: (i) marginal survival curves, which describe survival in the population if all subjects were treated or if all subjects were untreated; and (ii) marginal hazard ratios. …
The Performance Of Different Propensity Score Methods For Estimating Absolute Effects Of Treatments On Survival Outcomes: A Simulation Study, Peter C. Austin
The Performance Of Different Propensity Score Methods For Estimating Absolute Effects Of Treatments On Survival Outcomes: A Simulation Study, Peter C. Austin
Peter Austin
Observational studies are increasingly being used to estimate the effect of treatments, interventions and exposures on outcomes that can occur over time. Historically, the hazard ratio, which is a relative measure of effect, has been reported. However, medical decision making is best informed when both relative and absolute measures of effect are reported. When outcomes are time-to-event in nature, the effect of treatment can also be quantified as the change in mean or median survival time due to treatment and the absolute reduction in the probability of the occurrence of an event within a specified duration of follow-up. We describe …
Analysis Of Subgroup Data Of Clinical Trials, Kao-Tai Tsai, Karl E. Peace
Analysis Of Subgroup Data Of Clinical Trials, Kao-Tai Tsai, Karl E. Peace
Biostatistics Faculty Publications
Large randomized controlled clinical trials are the gold standard to evaluate and compare the effects of treatments. It is common practice for investigators to explore and even attempt to compare treatments, beyond the first round of primary analyses, for various subsets of the study populations based on scientific or clinical interests to take advantage of the potentially rich information contained in the clinical database. Although subjects are randomized to treatment groups in clinical trials, this does not imply the same degree of randomization among sub-populations of the original trials. Therefore, comparisons of treatments in sub-populations may not produce fair and …
Balancing Score Adjusted Targeted Minimum Loss-Based Estimation, Samuel D. Lendle, Bruce Fireman, Mark J. Van Der Laan
Balancing Score Adjusted Targeted Minimum Loss-Based Estimation, Samuel D. Lendle, Bruce Fireman, Mark J. Van Der Laan
U.C. Berkeley Division of Biostatistics Working Paper Series
Adjusting for a balancing score is sufficient for bias reduction when estimating causal effects including the average treatment effect and effect among the treated. Estimators that adjust for the propensity score in a nonparametric way, such as matching on an estimate of the propensity score, can be consistent when the estimated propensity score is not consistent for the true propensity score but converges to some other balancing score. We call this property the balancing score property, and discuss a class of estimators that have this property. We introduce a targeted minimum loss-based estimator (TMLE) for a treatment specific mean with …
A Targeted Confounder Selection Strategy For Propensity Score Estimation, Susan Gruber
A Targeted Confounder Selection Strategy For Propensity Score Estimation, Susan Gruber
Susan Gruber
These slides provide an introduction to data-adaptive propensity score estimation, and the collaborative targeted maximum likelihood estimator (C-TMLE) of van der Laan and Gruber. The notation has been greatly simplified, which makes the work accessible to a more general audience, but loses a little in the translation.
Using Ensemble-Based Methods For Directly Estimating Causal Effects: An Investigation Of Tree-Based G-Computation, Peter C. Austin
Using Ensemble-Based Methods For Directly Estimating Causal Effects: An Investigation Of Tree-Based G-Computation, Peter C. Austin
Peter Austin
Researchers are increasingly using observational or nonrandomized data to estimate causal treatment effects. Essential to the production of high-quality evidence is the ability to reduce or minimize the confounding that frequently occurs in observational studies. When using the potential outcome framework to define causal treatment effects, one requires the potential outcome under each possible treatment. However, only the outcome under the actual treatment received is observed, whereas the potential outcomes under the other treatments are considered missing data. Some authors have proposed that parametric regression models be used to estimate potential outcomes. In this study, we examined the use of …
Comparing Paired Vs. Non-Paired Statistical Methods Of Analyses When Making Inferences About Absolute Risk Reductions In Propensity-Score Matched Samples., Peter C. Austin
Peter Austin
Propensity-score matching allows one to reduce the effects of treatment-selection bias or confounding when estimating the effects of treatments when using observational data. Some authors have suggested that methods of inference appropriate for independent samples can be used for assessing the statistical significance of treatment effects when using propensity-score matching. Indeed, many authors in the applied medical literature use methods for independent samples when making inferences about treatment effects using propensity-score matched samples. Dichotomous outcomes are common in healthcare research. In this study, we used Monte Carlo simulations to examine the effect on inferences about risk differences (or absolute risk …
Optimal Caliper Widths For Propensity-Score Matching When Estimating Differences In Means And Differences In Proportions In Observational Studies., Peter C. Austin
Optimal Caliper Widths For Propensity-Score Matching When Estimating Differences In Means And Differences In Proportions In Observational Studies., Peter C. Austin
Peter Austin
In a study comparing the effects of two treatments, the propensity score is the probability of assignment to one treatment conditional on a subject's measured baseline covariates. Propensity-score matching is increasingly being used to estimate the effects of exposures using observational data. In the most common implementation of propensity-score matching, pairs of treated and untreated subjects are formed whose propensity scores differ by at most a pre-specified amount (the caliper width). There has been a little research into the optimal caliper width. We conducted an extensive series of Monte Carlo simulations to determine the optimal caliper width for estimating differences …
A Tutorial And Case Study In Propensity Score Analysis: An Application To Estimating The Effect Of In-Hospital Smoking Cessation Counseling On Mortality, Peter C. Austin
A Tutorial And Case Study In Propensity Score Analysis: An Application To Estimating The Effect Of In-Hospital Smoking Cessation Counseling On Mortality, Peter C. Austin
Peter Austin
Propensity score methods allow investigators to estimate causal treatment effects using observational or nonrandomized data. In this article we provide a practical illustration of the appropriate steps in conducting propensity score analyses. For illustrative purposes, we use a sample of current smokers who were discharged alive after being hospitalized with a diagnosis of acute myocardial infarction. The exposure of interest was receipt of smoking cessation counseling prior to hospital discharge and the outcome was mortality with 3 years of hospital discharge. We illustrate the following concepts: first, how to specify the propensity score model; second, how to match treated and …
An Introduction To Propensity-Score Methods For Reducing Confounding In Observational Studies, Peter C. Austin
An Introduction To Propensity-Score Methods For Reducing Confounding In Observational Studies, Peter C. Austin
Peter Austin
The propensity score is the probability of treatment assignment conditional on observed baseline characteristics. The propensity score allows one to design and analyze an observational (non-randomized) study so that it mimics some of the particular characteristics of a randomized controlled trial. In particular, the propensity score is a balancing score: conditional on the propensity score, the distribution of observed baseline covariates will be similar between treated and untreated subjects. We describe four different propensity score methods: matching on the propensity score, stratification on the propensity score, inverse probability of treatment weighting using the propensity score, and covariate adjustment using the …
The Performance Of Different Propensity-Score Methods For Estimating Differences In Proportions (Risk Differences Or Absolute Risk Reductions) In Observational Studies, Peter C. Austin
Peter Austin
Propensity score methods are increasingly being used to estimate the effects of treatments on health outcomes using observational data. There are four methods for using the propensity score to estimate treatment effects: covariate adjustment using the propensity score, stratification on the propensity score, propensity-score matching, and inverse probability of treatment weighting (IPTW) using the propensity score. When outcomes are binary, the effect of treatment on the outcome can be described using odds ratios, relative risks, risk differences, or the number needed to treat. Several clinical commentators suggested that risk differences and numbers needed to treat are more meaningful for clinical …