Survival Analysis Commons^™

Causal Inference For The Effect Of Continuous Treatment On Time-To-Event Outcomes And Mediation Analysis On Health Disparities In Observational Studies., Triparna Poddar

Electronic Theses and Dissertations

The dissertation comprises two projects related to causal inference based on observational data. In healthcare research, where abundant observational data such as claims data and electronic records are available, researchers often aim to study the treatment effect and the pathway of that effect. However, estimating treatment effects in observational data presents challenges due to confounding factors. The first project focuses on estimating continuous treatment effects for survival outcomes, while the second concentrates on mediation analysis, allowing the exploration of the pathway of the causal effect. Both projects involve addressing confounding variables. In the first project, I investigate estimation of the …

Parameter Estimation For Patient Enrollment In Clinical Trials, Junyan Liu

Undergraduate Honors Theses

In this paper, we study the Poisson-gamma model for recruitment time in clinical trials. We proved several properties of this model that match our intuitions from a reliability perspective, did simulations on this model, and used different optimization methods to estimate the parameters. Although the behaviors of the optimization methods were unfavorable and unstable, we identified certain conditions and provided potential explanations for this phenomenon and further insights into the Poisson-gamma model.

Nonparametric Derivative Estimation Using Penalized Splines: Theory And Application, Bright Antwi Boasiako

Doctoral Dissertations

This dissertation is in the field of Nonparametric Derivative Estimation using
Penalized Splines. It is conducted in two parts. In the first part, we study the L2
convergence rates of estimating derivatives of mean regression functions using penalized splines. In 1982, Stone provided the optimal rates of convergence for estimating derivatives of mean regression functions using nonparametric methods. Using these rates, Zhou et. al. in their 2000 paper showed that the MSE of derivative estimators based on regression splines approach zero at the optimal rate of convergence. Also, in 2019, Xiao showed that, under some general conditions, penalized spline estimators …

Optimal Experimental Planning Of Reliability Experiments Based On Coherent Systems, Yang Yu

Statistical Science Theses and Dissertations

In industrial engineering and manufacturing, assessing the reliability of a product or system is an important topic. Life-testing and reliability experiments are commonly used reliability assessment methods to gain sound knowledge about product or system lifetime distributions. Usually, a sample of items of interest is subjected to stresses and environmental conditions that characterize the normal operating conditions. During the life-test, successive times to failure are recorded and lifetime data are collected. Life-testing is useful in many industrial environments, including the automobile, materials, telecommunications, and electronics industries.

There are different kinds of life-testing experiments that can be applied for different purposes. …

Empirical Likelihood Ratio Tests For Homogeneity Of Distributions Of Component Lifetimes From System Lifetime Data With Known System Structures, Jingjing Qu

Statistical Science Theses and Dissertations

In system reliability, practitioners may be interested in testing the homogeneity of the component lifetime distributions based on system lifetimes from multiple data sources for various reasons, such as identifying the component supplier that provides the most reliable components.

In the first part of the dissertation, we develop distribution-free hypothesis testing procedures for the homogeneity of the component lifetime distributions based on system lifetime data when the system structures are known. Several nonparametric testing statistics based on the empirical likelihood method are proposed for testing the homogeneity of two or more component lifetime distributions. The computational approaches to obtain the …

An Analysis Of All-Cause Mortality On Patients With Sickle Cell Disease And Kidney Disease Using Propensity Score Matching, Adam Garrison

Electronic Theses and Dissertations

In this work, we provide an overview of the Cox proportional hazards model for time to event or survival analysis and the notion of propensity score matching to deal with confounding factors. A full analysis is reported in Chapter 2 concerning mortality for in-center dialysis patients with sickle cell disease to demonstrate the application of a general analysis strategy that has some logistical benefits over more traditional approaches to accounting for confounding variables. We also provide some insight and discussions on the challenges and future research questions that will emerge when trying to implement this strategy as a monitoring tool …

Inverse Probability Weighting In Survival Analysis And Network Analysis, Yukun Lu

Doctoral Dissertations

Inverse probability weighting is a popular technique to accommodate selection bias due to non-random sampling and missing data. In the first chapter, we develop an inverse probability weighted estimator and an augmented inverse probability weighted estimator of regression coefficients for a linear model with randomly censored covariates, when the censoring mechanism may be dependent on the outcome. We investigate the asymptotic properties of both estimators and evaluate their finite sample performance through extensive simulation studies. We apply the proposed methods to an Alzheimer’s disease study. In the second chapter, we present an application of network analysis in a study of …

Striving For Appropriate Antibiotic Use: A Biomarker Initiative, And Outcomes Associated With Azithromycin Exposure, Amanda Gusovsky

Theses and Dissertations--Pharmacy

The introduction of antibiotics into clinical practice is considered the greatest medical breakthrough of the 20^thcentury. However, the use of antibiotics can contribute to the development of resistance. In the United States (U.S.), approximately 2.8 million people are infected with antibiotic-resistant bacteria each year, and more than 35,000 people die as a result. Moreover, some antibiotics are known to cause cardiac side effects including QT prolongation, hypotension, and ventricular arrythmias. The U.S. Centers for Disease Control and Prevention (CDC) defines appropriate antibiotic use as the effort to use “the right antibiotic, at the right dose, for the right …

Regression Modeling Of Complex Survival Data Based On Pseudo-Observations, Rong Rong

Statistical Science Theses and Dissertations

The restricted mean survival time (RMST) is a clinically meaningful summary measure in studies with survival outcomes. Statistical methods have been developed for regression analysis of RMST to investigate impacts of covariates on RMST, which is a useful alternative to the Cox regression analysis. However, existing methods for regression modeling of RMST are not applicable to left-truncated right-censored data that arise frequently in prevalent cohort studies, for which the sampling bias due to left truncation and informative censoring induced by the prevalent sampling scheme must be properly addressed. Meanwhile, statistical methods have been developed for regression modeling of the cumulative …

Dynamic Prediction For Alternating Recurrent Events Using A Semiparametric Joint Frailty Model, Jaehyeon Yun

Statistical Science Theses and Dissertations

Alternating recurrent events data arise commonly in health research; examples include hospital admissions and discharges of diabetes patients; exacerbations and remissions of chronic bronchitis; and quitting and restarting smoking. Recent work has involved formulating and estimating joint models for the recurrent event times considering non-negligible event durations. However, prediction models for transition between recurrent events are lacking. We consider the development and evaluation of methods for predicting future events within these models. Specifically, we propose a tool for dynamically predicting transition between alternating recurrent events in real time. Under a flexible joint frailty model, we derive the predictive probability of …

Statistical Methods For Personalized Treatment Selection And Survival Data Analysis Based On Observational Data With High-Dimensional Covariates., Don Ramesh Dinendra Sudaraka Tholkage

Electronic Theses and Dissertations

Due to the wide availability of functional data from multiple disciplines, the studies of functional data analysis have become popular in the recent literature. However, the related development in censored survival data has been relatively sparse. In Chapter 2, we consider the problem of analyzing time-to-event data in the presence of functional predictors. We develop a conditional generalized Kaplan Meier (KM) estimator that incorporates functional predictors using kernel weights and rigorously establishes its asymptotic properties. In addition, we propose to select the optimal bandwidth based on a time-dependent Brier score. We then carry out extensive numerical studies to examine the …

New Developments On The Estimability And The Estimation Of Phase-Type Actuarial Models, Cong Nie

Electronic Thesis and Dissertation Repository

This thesis studies the estimability and the estimation methods for two models based on Markov processes: the phase-type aging model (PTAM), which models the human aging process, and the discrete multivariate phase-type model (DMPTM), which can be used to model multivariate insurance claim processes.

The principal contributions of this thesis can be categorized into two areas. First, an objective measure of estimability is proposed to quantify estimability in the context of statistical models. Existing methods for assessing estimability require the subjective specification of thresholds, which potentially limits their usefulness. Unlike these methods, the proposed measure of estimability is objective. In …

Statistical Modeling Of Longitudinal Medical Cost Data, Shikun Wang

Dissertations & Theses (Open Access)

Projecting the future cancer care cost is critical in health economics research and policy making. An indispensable step is to estimate cost trajectories from an incident cohort of cancer patients using longitudinal medical cost data, accounting for terminal events such as death, and right censoring due to loss of follow-up. Since the cost of cancer care and survival are correlated, a scientifically meaningful quantity for inference in this context is the mean cost trajectory conditional on survival. Many standard approaches for longitudinal and survival analysis are not valid for the problem. The research for my Ph.D. dissertation consists of three …

Factors Affecting Time To Recovery: A Covid-19 Survival Analysis, Fernanda Montoya

Honors Capstones

This project is focused on the recovery rates of patients diagnosed with COVID-19 after different clinical trial drug treatments. Data for the clinical trial studied was obtained from the National Institute of Allergy and Infectious Diseases for the primary purpose of a survival analysis on patient time to recovery under a placebo and therapeutic drug treatment. Specifically, patients in this clinical trial were randomly selected to receive remdesivir, an antiviral drug, in combination with a placebo or baricitinib, a janus kinase inhibitor drug. Cox PH models were used to identify how the different treatment drugs affect time to recovery and …

Flexible Modelling Of Time-Dependent Covariate Effects With Correlated Competing Risks: Application To Hereditary Breast And Ovarian Cancer Families, Seungwoo Lee

Electronic Thesis and Dissertation Repository

This thesis aims to develop a flexible approach for modelling time-dependent covariate effects on event risk using B-splines in the presence of correlated competing risks. The performance of the proposed model was evaluated via simulation in terms of the bias and precision of the estimation of the parameters and penetrance functions. In addition, we extended the concordance index to account for time-dependent effects and competing events simultaneously and demonstrated its inference procedures. We applied our proposed methods to data rising from the BRCA1 mutation families from the breast cancer family registry to evaluate the time-dependent effects of mammographic screening and …

Predicting Tf33-Pw-100a Engine Failures Due To Oil Issues Using Survival Analyses, Anna M. Davis

Theses and Dissertations

In 2007, the Office of the Assistant Secretary of Defense for Sustainment pushed for the need to transition to a Condition Based Maintenance Plus (CBM ) initiative for weapon systems in the U.S. Department of Defense. The CBM initiative can help increase aircraft availability (AA) for the United States Air Force. There are many reasons where AA can be affected but one such issue is engine availability primarily due to oil issues. Within the CBM perspective, this study examines the risk of a jet engine failure due to an oil issue and attempts to predict an engines time until next …

Examining Failures Of Kc-135s Using Survival Analysis, Vanessa I. R. Unseth

Theses and Dissertations

The United States Air Force manages an inventory of 396 KC-135 Stratotanker aircraft. With mission capability rates falling and total non-mission capability supply rates increasing, it is necessary to take a deeper look at recurrent failures. The study applies non-parametric and semi-parametric survival models to a dataset retrieved from LIMS-EV to look at the duration(s) until failure for the KC-135. Results of non-parametric models show cumulative failure rates increase as sorties or flight hours increase. In addition, semi-parametric models or Cox proportional hazards models with frailty confirm that locations or air bases are not associated with recurrent failures.

Approximate Likelihood Based Estimations For Joint Models With Intractable Likelihoods, Karl Stessy M. Bisselou

Theses & Dissertations

This dissertation focuses on the development of approximation approaches for the joint modeling (JM) of repeated measures data and time-to-event data in the presence of analytically or numerically intractable likelihoods. Current likelihood-based inferences for JMs show several limitations including (i) intractability of integrals during marginal likelihood derivations due to the complexity in computations, and (ii) the large number of nuisance parameters (unobserved) posing a problem with convergence. The h-likelihood (HL) and synthetic likelihood (SL) are two computationally efficient estimation approaches that overcome these challenges.

In the presence of extremely high censoring rates, the HL can produce bias parameter estimates. We …

Addressing Bias In Non-Experimental Studies Assessing Treatment Outcomes In Prostate Cancer, David E. Guy

Electronic Thesis and Dissertation Repository

We evaluated the ability of matching techniques to balance baseline characteristics between treatment groups using non-experimental data. We identified a set of balance diagnostics that assessed key differences in baseline covariates with potential for confounding. These diagnostics were used in a novel systematic approach to developing and evaluating models for use in propensity score matching that optimized balance and data retention. We then compared the performance of propensity score and coarsened exact matching strategies in optimizing balance and data retention, using non-experimental data from a pan-Canadian prostate cancer database. Both matching techniques balanced baseline covariates adequately and retained approximately 70% …

Extension Of The Two-Step Approach For Informative Dropout In Survival Analysis, Cristina Murray-Krezan

Mathematics & Statistics ETDs

Chronic kidney disease (CKD) in children is known to result in poor growth and quality of life, and frequently results in kidney failure. The Chronic Kidney Disease in Children study (CKiD) is a prospective cohort study enrolling children ages 1 to 16 to assess health outcomes in children with CKD including the effects of declining glomerular filtration rate and the resulting consequences of growth failure on morbidity. Quantification of the magnitude of the risk for decreased kidney function and, ultimately, failure has been achieved through a variety of studies, often including cohort studies such as the CKiD study. Longitudinal studies …

Optimizing Critical Values And Combining Axes For Multi-Axial Neck Injury Criteria, Ethan J. Gaston

Theses and Dissertations

The Air Force employs ejection seats in its high-performance aircraft. While these systems are intended to ensure aircrew safety, the ejection process subjects the aircrew to potentially injurious forces. System validation includes evaluation of forces against a standard which is linked to the probability of injury. The Muti-Axial Neck Injury Criteria (MANIC) was developed to account for forces in all six degrees of freedom. Unfortunately, the MANIC is applied to each of the three linear input directions separately and applies different criterion values for each direction. These three separate criteria create a lack of clarity regarding acceptable neck loading, leading …

An Examination Into Retention Behavior Of Air Force Female Officers, Jessica M. Astudillo

Theses and Dissertations

Female retention rates in the US military have been considerably lower than that of their male counterparts for numerous years. In the Air Force, women represent 14 percent of officer ranks from O-5 level and above. Comparatively, the overall rate of women officers in service is 20 percent. Understanding the negative factors associated with the attrition rate of this group can help the Air Force leverage positive change. It may also influence adjustments that will increase the number of women serving, and improve diversity throughout both the officer and enlisted ranks. In this study, logistic regression and survival analysis are …

An Examination Of Civilian Retention In The United States Air Force, William F. Wilson

Theses and Dissertations

The backbone of the United States Air Force is undoubtedly the large civilian workforce that supplements the great work that is accomplished. Many research studies have been conducted on officer and enlisted personnel to ensure that the career fields are properly developed and managed to meet the ever growing demands of the military's varied missions, but no recent studies have focused on the civilian workforce. Striking a balance between new and experienced employees is paramount to success given the ever-changing economic and political landscapes where we find ourselves. The first part of the research uses logistic regression to determine the …

Innovative Statistical Models In Cancer Immunotherapy Trial Design, Jing Wei

Theses and Dissertations--Statistics

A challenge arising in cancer immunotherapy trial design is the presence of non-proportional hazards (NPH) patterns in survival curves. We considered three different NPH patterns caused by delayed treatment effect, cure rate and responder rate of treatment group in this dissertation. These three NPH patterns would violate the proportional hazard model assumption and ignoring any of them in an immunotherapy trial design will result in substantial loss of statistical power.

In this dissertation, four models to deal with NPH patterns are discussed. First, a piecewise proportional hazards model is proposed to incorporate delayed treatment effect into the trial design consideration. …

Improved Statistical Methods For Time-Series And Lifetime Data, Xiaojie Zhu

Statistical Science Theses and Dissertations

In this dissertation, improved statistical methods for time-series and lifetime data are developed. First, an improved trend test for time series data is presented. Then, robust parametric estimation methods based on system lifetime data with known system signatures are developed.

In the first part of this dissertation, we consider a test for the monotonic trend in time series data proposed by Brillinger (1989). It has been shown that when there are highly correlated residuals or short record lengths, Brillinger’s test procedure tends to have significance level much higher than the nominal level. This could be related to the discrepancy between …

Survival Analysis: An Exact Method For Rare Events, Kristina Reutzel

All Graduate Plan B and other Reports, Spring 1920 to Spring 2023

Conventional asymptotic methods for survival analysis work well when sample sizes are at least moderately sufficient. When dealing with small sample sizes or rare events, the results from these methods have the potential to be inaccurate or misleading. To handle such data, an exact method is proposed and compared against two other methods: 1) the Cox proportional hazards model and 2) stratified logistic regression for discrete survival analysis data.

Causal Inference And Prediction On Observational Data With Survival Outcomes, Xiaofei Chen

Statistical Science Theses and Dissertations

Infants with hypoplastic left heart syndrome require an initial Norwood operation, followed some months later by a stage 2 palliation (S2P). The timing of S2P is critical for the operation’s success and the infant’s survival, but the optimal timing, if one exists, is unknown. We attempt to estimate the optimal timing of S2P by analyzing data from the Single Ventricle Reconstruction Trial (SVRT), which randomized patients between two different types of Norwood procedure. In the SVRT, the timing of the S2P was chosen by the medical team; thus with respect to this exposure, the trial constitutes an observational study, and …

Statistical Models And Analysis Of Univariate And Multivariate Degradation Data, Lochana Palayangoda

Statistical Science Theses and Dissertations

For degradation data in reliability analysis, estimation of the first-passage time (FPT) distribution to a threshold provides valuable information on reliability characteristics. Recently, Balakrishnan and Qin (2019; Applied Stochastic Models in Business and Industry, 35:571-590) studied a nonparametric method to approximate the FPT distribution of such degradation processes if the underlying process type is unknown. In this thesis, we propose improved techniques based on saddlepoint approximation, which enhance upon their suggested methods. Numerical examples and Monte Carlo simulation studies are used to illustrate the advantages of the proposed techniques. Limitations of the improved techniques are discussed and some possible solutions …

Estimation Of The Treatment Effect With Bayesian Adjustment For Covariates, Li Xu

Theses and Dissertations--Statistics

The Bayesian adjustment for confounding (BAC) is a Bayesian model averaging method to select and adjust for confounding factors when evaluating the average causal effect of an exposure on a certain outcome. We extend the BAC method to time-to-event outcomes. Specifically, the posterior distribution of the exposure effect on a time-to-event outcome is calculated as a weighted average of posterior distributions from a number of candidate proportional hazards models, weighing each model by its ability to adjust for confounding factors. The Bayesian Information Criterion based on the partial likelihood is used to compare different models and approximate the Bayes factor. …

Robustness Of Semi-Parametric Survival Model: Simulation Studies And Application To Clinical Data, Isaac Nwi-Mozu

Electronic Theses and Dissertations

An efficient way of analyzing survival clinical data such as cancer data is a great concern to health experts. In this study, we investigate and propose an efficient way of handling survival clinical data. Simulation studies were conducted to compare performances of various forms of survival model techniques using an R package ``survsim". Models performance was conducted with varying sample sizes as small ($n5000$). For small and mild samples, the performance of the semi-parametric outperform or approximate the performance of the parametric model. However, for large samples, the parametric model outperforms the semi-parametric model. We compared the effectiveness and reliability …