Survival Analysis Commons^™

Bayesian Designs For Two-Arm Clinical Trials With Time-To-Event Endpoints: Incorporating Historical Data Through Power Priors, Sara Hajraf H. Almutiri

Mathematics & Statistics ETDs

Bayesian methods provide a flexible framework for time-to-event analysis by incorporating prior information. The power prior offers a systematic way to borrow information from historical data. This approach is especially valuable in clinical research, where historical data can enhance inference in early-phase trials with limited sample sizes. This dissertation develops Bayesian approaches for two-arm survival studies using both closed-form and simulation-based methods. The closed-form inference is derived under exponential and Weibull survival models. Under the proportional hazards framework, the posterior is derived through a normal approximation to the log hazard ratio, allowing inference on the treatment effect when the variance …

Survival Patterns Among Adult And Pediatric Bone Cancer Patients, Ethan Estes

Mathematical Sciences Undergraduate Honors Theses

Recently noted, Huang et al. (2023), machine learning (ML) models, while offering great advantages over traditional statistical predictive modeling methods, are less explored in the analysis of survival and other similar time-to-event predictive data modeling. ML methods such as neural networks offer a great deal of promise but need to be further explored to investigate their comparative power in predicting survival outcomes. Focusing specifically on survival analysis in adult and pediatric bone cancer patients, traditional methods, like shown in Emmert-Streib and Dehmer (2019), will be shown with machine learning models using methods in Hothorn, Hornik, and Zeileis (2006). In this …

A New Approach To Generate Combinatorial Patterns In Logical Analysis Of Data And Its Application To Predict College Retention, Salihah Ahmed E. Jaafari

Theses and Dissertations

Student retention and degree completion remain central challenges for higher-education institutions, with significant implications for student success, institutional effectiveness, and public accountability. While advances in predictive analytics have enabled earlier identification of students at risk of withdrawal, many commonly used machine learning approaches suffer from limited interpretability, constraining their practical usefulness for advising, intervention, and policy decision making. This dissertation addresses the problem of predicting student persistence by developing and evaluating optimization based, interpretable classification models within the Logical Analysis of Data (LAD) framework. Building on existing LAD formulations, this research introduces two novel pattern generation models, the Best Term …

Explainable Ai For Liver Transplant Survival Prediction: Integrating Immunological Mismatch Features, Sourab Shaik

Honors Projects

Liver Transplantations are crucial treatment for end-stage liver disease. However, a persistent deficit of donor organs necessitates maximizing the utility of each available graft to minimize failure rates. We evaluated whether donor–recipient molecular immunogenicity metrics - Electrostatic and Hydrophobic Mismatch Scores (HMS/EMS) and eplet-based counts - improve post–liver-transplant survival prediction. The analytic cohort comprised adult, first time, single-organ deceased-donor transplants drawn from Scientific Registry of Transplant Recipients; follow-up was truncated at five years, and the endpoint was all-cause graft failure (earliest of graft failure or death; otherwise, censored). HLA variables were derived via high- resolution conversion and molecular mismatch computations …

A Leslie System For A Demographic Simulation: From An Actuarial Point Of View, David Kings

Electronic Theses and Dissertations

This thesis develops a discrete stochastic linear systems interpretation of age–stage demographic evolution grounded in Leslie operators and realized in a discrete-event simulation implemented with salabim. The central claim is that one annual cycle of the simulation constitutes a cone-preserving, stochastic affine transformation on a high- dimensional population state vector indexed by age, sex, marital status, household type, employment, and education, and that the composition of yearly operators yields a random matrix product whose top Lyapunov exponent is the stochastic counterpart of the Perron–Frobenius growth rate (Caswell, 2001; Tuljapurkar, 1997)[1, 2]. The actuarial bridge is constructed by mapping simulated survival …

Statistical Methods For Joint Outcome Modeling And Dynamic Assessment Of Recurrent Events, Zifang Kong

Statistical Science Theses and Dissertations

Recurrent event data frequently arise in clinical studies where individuals experience repeated, possibly related, events over time. These data are often accompanied by sparse and irregular longitudinal measurements, creating challenges for traditional joint modeling approaches that struggle to account for time-dependent associations and within-subject correlations. We propose FRAILTY (Functional Regression with AutoRegressIve fraiLTY), a novel two-step framework that integrates functional principal component analysis (PACE) with a dynamic frailty model featuring autoregressive structure. FRAILTY accommodates both scalar and functional predictors and captures within-subject dependence across recurrent events. To further extend its utility, we develop a multivariate joint modeling framework that simultaneously …

Evaluating Alpha Spending Functions Applied To Observational Time-To-Event Analysis, Moses Torgbenu

Electronic Theses and Dissertations

This thesis explores the theoretical foundation of the alpha spending approach and extends its application beyond the conventional setting of randomized controlled trials (RCTs) to observational studies with time to event analyses. In these less structured environments, key design parameters such as the total number of events are often unknown, posing challenges for the standard implementation of sequential analysis methods.

Through simulation studies, this research delivers several important contributions. First, it presents a modified approach that uses calendar time to define the timing of interim analyses while relying on event-based information to estimate the correlation among test statistics. This adjustment …

Expressive And Interpretable User Engagement Prediction Using Multivariate Survival Processes, Akshay Aravamudan

Theses and Dissertations

The ability to characterize how information diffuses online is of paramount importance to stakeholders that are interested in tasks such as proposing solutions for mitigating and countering dis/misinformation, predicting user engagement of content in social media, planning marketing campaigns to roll-out products and planning dissemination of political campaign messaging among others. One such facet of learning the dynamics of information diffusion is the ability to predict user engagement or the popularity of a single piece of information as it spreads through an online medium. Existing works in this regard mainly either obfuscate user level information or utilize frameworks that are …

Innovative Methods For The Design And Analysis Of Phase Ii Clinical Trials, Feng Tian

Dissertations and Theses (Open Access)

Drug development has become increasingly time-consuming, costly, and risky in recent years. There is significant potential for improving clinical trial designs, particularly for phase II trials, which play a critical role in the drug development process. Innovative methods are especially necessary for addressing key challenges in phase II trials in terms of dose-ranging study, patient population selection, and decentralized clinical trials (DCTs). This dissertation presents a comprehensive set of methodologies that address these critical issues with three projects. The first project introduces a Bayesian adaptive dose-ranging design that integrates both efficacy and toxicity data to evaluate each dose comprehensively. The …

The Impact Of “Multiple Looks” When Performing Survival Analysis, Quentin Eloise

Electronic Theses and Dissertations

Survival analysis is a critical statistical method in healthcare to assess patient treatment effects and disease progression. Another critical area of statistical methodology in health care is the practice of adaptive designs. Adaptive designs allow for interim analyses to take place during a study and various decisions and actions can take place more ethically. This is beneficial for studies that take multiple years to complete and allows administrators and healthcare providers to make sound decisions as early as possible. A challenging aspect of adaptive designs is that the number of interim analyses is known in advance which is applicable in …

Bayesian Approaches In Multi-State Markov Models And High Dimensional Time-To-Event Data., Yuchen Han

Electronic Theses and Dissertations

This dissertation consists of two projects. The first one involves nonparametric methods on Continuous Time Markov Chains (CTMCs). The second one is centered around Bayesian shrinkage models for detecting prognostic and predictive biomarkers in high-dimensional clinical data. Both these projects build on methods from across the frequentist and Bayesian paradigm to offer novel solutions. In the first project, we aim to model the nonlinear effects of continuous variables within multistate framework in a non-parametrically by appealing to the rich mathematical framework of Reproducing Kernel Hilbert Spaces (RKHS). Then we adapted the classical Representer Theorem to penalized (squared norm) log-likelihood which …

Dynamic Prediction Of Disease Progression With Longitudinal Data, Wenhao Li

Dissertations and Theses (Open Access)

Dynamic prediction plays a pivotal role in clinical research, especially when forecasting time-to-event outcomes based on evolving longitudinal data. This process often leverages the integration of longitudinal and time-to-event data through joint modeling, a prevalent technique. Alongside joint modeling, landmark modeling stands as another key approach in the realm of longitudinal studies. These methodologies are instrumental in dynamically predicting clinical events by utilizing predictor variables measured over time, up until the moment predictions are made. Within this framework, Chapter 2 addresses the challenge of comparing joint modeling and landmark modeling for dynamic prediction in longitudinal studies, introducing a novel algorithm …

Evaluating Novel Approaches For Improving Anadromous Fish Passage In Coastal Rivers, Aaron Bunch

All Dissertations

Few of the world's rivers remain free-flowing for over 1000 km, significantly impacting anadromous fish populations due to dam-induced habitat fragmentation. Three partial migration barriers (low-head dams) on North Carolina's Cape Fear River (USA)—lock and dam 1 (LD1), lock and dam 2 (LD2), and lock and dam 3 (LD3)—impact American shad Alosa sapidissima, Atlantic sturgeon Acipenser oxyrinchus oxyrinchus, and striped bass Morone saxatilis populations. Mitigation strategies to pass fish upstream have included conservation locking, nature-like fishway construction, and environmental flows (e-flows). This study introduced a novel e-flow (dam submergence flow), tested acoustic double-tagging techniques for tracking American …

Examining The Effect Of Contractor Logistics Support On The Reliability Of Military Aircraft, Rodrigo S. Campos De Moura

Theses and Dissertations

This study utilizes survival analysis for examining the effect of Contractor Logistics Support (CLS) on the reliability of military aircraft before and after implementing CLS. The provider of CLS in this study is the original equipment manufacturer that designed and produced the target aircraft of this study, the Embraer A-29 Super Tucano.

Simulating Human-Autonomous Aircraft Teams In An Anti-Access Area Denial (A2ad) Environment, Michael Kaminski

Theses and Dissertations

The role of autonomy has evolved recently, demanding tighter integration between human and autonomous systems, particularly in highly contested A2AD environments. Near-peer adversaries have modernized their integrated air defense systems (IADS), diminishing the current advantages of the United States Air Force. To regain air dominance, efforts like the Collaborative Combat Aircraft (CCA) program are underway, aiming to deploy unmanned autonomous alongside manned next-generation fighter aircraft. This research assesses various operational concepts, focusing on autonomous tactics post-manned fighter loss, strike timing of independent teams, and weapon configuration observability. Using the Advanced Framework for Simulation, Integration and Modeling (AFSIM), an agent-based model …

Outpatient Fall Prevention In Ambulatory Adults 65 Years Old And Over, Dorothy L. Osborne-White

Doctor of Nursing Practice (DNP) Scholarly Projects - Archive

Background: In the United States (U.S.), falls are the leading cause of injury among adults 65 and over, resulting in 36 million falls yearly (Moreland et al., 2020). According to the Centers for Disease Control and Prevention (CDC, 2023), one in four older adults experiences a fall each year. Falls are the world's second most prominent cause of accidental deaths (World Health Organization [WHO], 2021). Falls are the leading cause of both fatal and non-fatal injuries among older adults (Moreland et al., 2020).

Methods: A quality improvement project that included a fall bundle was implemented in a primary clinic. A …

Applications Of Causal Inference Methods For The Estimation Of Effects Of Bone Marrow Transplant And Prescription Drugs On Survival Of Aplastic Anemia Patients, Yesha M. Patel

Computational and Data Sciences (PhD) Dissertations

This dissertation provides an in-depth exploration into the treatment effectiveness for aplastic anemia using causal inference methods, structured around three pivotal research papers. Each paper contributes to a nuanced understanding of treatment impacts, specifically focusing on bone marrow transplantation (BMT) and prescription drugs, and the identification of optimal treatment strategies.

The first paper, "Causal Inference Analysis for Assessing the Effect of Bone Marrow Transplantation on the One-Year Survival of Adult and Pediatric Aplastic Anemia Patients," sets the foundation. It examines the short-term effectiveness of BMT in both adult and pediatric patients, providing crucial insights into how this treatment affects survival …

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 …

Addressing The Analytical And Computational Challenges Using Machine Learning In Biomedical Research, Yizhuo Wang

Dissertations and Theses (Open Access)

In the contemporary healthcare field, professionals are confronted with an ever-growing volume of clinical data stored in electronic health records, alongside the genomic data stemming from laboratory experiments. As a response to this deluge of data, the application of machine learning (ML) techniques is gaining popularity since ML techniques have demonstrated an exceptional proficiency in processing big data and deciphering complex nonlinear patterns that are intrinsic to biomedical research.

My research leverages ML's capabilities to address the computational challenges spanning diverse areas, including adaptive clinical trial designs, survival analysis, and high-dimensional genetic data analysis. Specifically, Chapter 2 focused on the …

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 …

Examining Fuel Service System Failures Of The Usaf R11 Using Survival Analysis, Roed M.S. Mejia

Theses and Dissertations

Recent events show that fuel supply is a large contributor to the success or failure of a military operation in response to a contingency. Any future near-peer conflict will stress the supply chain and require fully operational vehicles to be ready for the primary mission sets they support. In the United States Air Force (USAF), the readiness of fuel distribution trucks is crucial to meeting those mission sets in global operations. Utilizing non-parametric and semi-parametric survival models, which do not assume specific probability distributions, this study analyzes maintenance data for R-11 trucks that refuel aircraft.

Examining Failures Of Kc-135 Boom Assemblies Using Survival Analysis, Benjamin D. Miller

Theses and Dissertations

The purposes of this study are to confirm the applicability of survival analysis for predicting recurrent failures of a component of a military aircraft and to provide practical insights to maintenance managers and mission planners. The results of this study also can help the United States Department of Defense improve the CBM+ program. This study was able to predict recurrent failures of the component using Nelson-Aalen cumulative estimates. In addition, this study used a Cox proportional hazards regression model with shared frailty for measuring the effect of covariates on recurrent failures and unidentified heterogeneity in the model, which warranted future …

Debris Survivability Study For Mega-Constellation Architectures, Joseph C. Canoy

Theses and Dissertations

The analysis for the overall theoretical debris survivabilty of mega-constellation architectures, with an emphasis on space-based ballistic missile defense constellation (SB-BMD), is explored via three extensive different Monte Carlo simulations: preliminary analysis of low Earth Orbit (LEO) mega-constellation survivabilty following a fragmentation event within the constellation, analysis of LEO mega-constellation survivability with a fragmentation event occurring on a satellite performing a maneuver to insert itself within the constellation, and the analysis of LEO mega-constellation survivabilty after a fragmentation event resulting from the destruction of a missile. The LEO mega-constellations represent the SB-BMD constellation. The first two analysis sections will include …

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 …

Infrastructure Development For Personalized Risk Prediction To Reduce Cardiovascular Disease In Childhood Cancer Survivors, Suman Shrestha

Dissertations and Theses (Open Access)

Although childhood cancer survivors have lengthy life expectancies, they run the risk of experiencing long-term health issues as a result of their treatment. The most frequent non-cancerous cause of morbidity and mortality for these survivors is cardiac disease. Radiation therapy (RT) has been linked in numerous cohort studies to a higher chance of developing a late cardiac disease in these survivors, and this risk rises with higher mean heart doses and increased RT exposure to larger cardiac volumes. Since, the heart is a heterogeneous organ made up of several distinct substructures, RT dose received by the entire heart does not …

Hypothesis Testing And Parameter Estimation In Mixture Cure Models For Cancer Survival Data, Mohammod Mahmudur Rahman

Graduate Theses and Dissertations

In oncology clinical trials, when a treatment is administered to the patient population, a certain subset of patients may respond to the treatment while the other does not. The positive responders with long-term survival are considered “statistically cured” and can be referred to as cured patients or long-term survivors. When a proportion of patients achieve long-term survival, the hazard functions of two arms (control vs. treatment) are no longer proportional. As a result, the traditional log-rank test, which is the most popular test to evaluate the effectiveness of a treatment in clinical trials, tends to lose its power. In this …

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 …