Statistics and Probability Commons^™

Flowgraph Models For Clustered Multistate Time To Event Data, Kristin Hall

USF Tampa Graduate Theses and Dissertations

Healthcare systems have multistate processes. Such processes may be modeled using flowgraphs, which are directed graphs. Flowgraph models support a variety of transition time distributions, easily handle reversibility between states and allow alternate paths to the event or state of interest to be taken. However, estimation of flowgraph and first passage time distribution parameters can lead to incorrect inferences when interdependent data are treated as independent.

In this dissertation, we expand the flowgraph model to accommodate nested and correlated data structures. We develop a framework to incorporate random effects into transition probability and transition time components of a flowgraph model. …

Survival Analysis: A Modified Kaplan-Meir Estimator, Justin A. Bancroft

Graduate Theses/Dissertations

The popular Kaplan-Meir estimator has traditionally been used to great effect as a survival function estimator. However, the Kaplan-Meir estimator is dependent upon a maximum likelihood parameter estimator which may not be the best estimator in all cases. We modify the Kaplan-Meir estimator, based on a Bayes parameter estimation, in hopes of providing a more accurate survival estimator for small sample sizes. Core elements of survival analysis are presented, acting as a foundation from which to construct and compare our modified Kaplan-Meir estimator. It is hypothesized that our modified Kaplan-Meir estimator is generally more accurate than the standard Kaplan-Meir estimator …

Diagnostics For Choosing Between Stratified Logrank And Stratified Wilcoxon, Jhoanne Marsh C. Gatpatan

Dissertations

Martinez and Naranjo (2010) proposed a pretest for choosing between Logrank or Wilcoxon test in a two - sample case. However, in the presence of covariates, comparing two populations without adjusting for covariates would yield misleading results. In this study, we propose several pretests that will help the analyst decide to use stratified Logrank or stratified Wilcoxon tests in comparing two survival curves after covariates have been taken into account. Power performance of each adaptive test was done through simulations under PH and non-PH cases.

Statistical Analysis And Modeling Of Stomach Cancer Data, Chao Gao

USF Tampa Graduate Theses and Dissertations

The objective of this study is to address some important questions associated with stomach cancer patients using the data from the Surveillance Epidemiology and End Results (SEER) program of the United States. To better understand the behavior of stomach cancer, we first perform parametric analysis for each patient group (white male, white female, African American male, African American female, other male and female) to identify the probability distribution function which can best characterize the behavior of the malignant stomach tumor sizes. We evaluate the effects of patients’ age, gender and race on the malignant stomach tumor sizes by developing quantile …

Statistical Analysis And Modeling Of Ovarian And Breast Cancer, Muditha V. Devamitta Perera

USF Tampa Graduate Theses and Dissertations

The objective of the present study is to investigate key aspects of ovarian and breast cancers, which are two main causes of mortality among women. Identification of the true behavior of survivorship and influential risk factors is essential in designing treatment protocols, increasing disease awareness and preventing possible causes of disease. There is a commonly held belief that African Americans have a higher risk of cancer mortality. We studied racial disparities of women diagnosed with ovarian cancer on overall and disease-free survival and found out that there is no significant difference in the survival experience among the three races: Whites, …

Implant Treatment In The Predoctoral Clinic: A Retrospective Database Study Of 1091 Patients, Soni Prasad, Christopher Hambrook, Eric Reigle, Katherine Sherman, Naveen K. Bansal, Arthur F. Hefti

Mathematics, Statistics and Computer Science Faculty Research and Publications

Purpose: This retrospective study was conducted at the Marquette University School of Dentistry to (1) characterize the implant patient population in a predoctoral clinic, (2) describe the implants inserted, and (3) provide information on implant failures.

Materials and Methods: The study cohort included 1091 patients who received 1918 dental implants between 2004 and 2012, and had their implants restored by a crown or a fixed dental prosthesis. Data were collected from patient records, entered in a database, and summarized in tables and figures. Contingency tables were prepared and analyzed by a chi-squared test. The cumulative survival probability of implants was …

Spatial Analysis Of Time Between Two Consecutive Dental And Two Consecutive Well-Child Visits For Foster Care Youth, Chenyang Shi

Dissertations

Foster care youth is a medically vulnerable population. Poor dental health and irregular well-child visits may cause serious health-related issues, such as mental disorder, nutrition imbalance, tooth damage, etc. Michigan requires all youth in foster care to receive annual dental and well-child visits. Usually, the study of foster care well-child and dental visits include two parts: time between two consecutive visits (gap time) and number of visits. For this study, a longitudinal-spatial model that has the flexibility to analyze the well-child/dental gap times and number of visits was developed. The longitudinal data (2009-2012) on Michigan foster care youth from 10 …

Efficiency Of Two Sample Tests Via The T-Mean Survival Time For Analyzing Event Time Observations, Lu Tian, Haoda Fu, Stephen J. Ruberg, Hajime Uno, Lj Wei

Harvard University Biostatistics Working Paper Series

In comparing two treatments with the event time observations, the hazard ratio (HR) estimate is routinely used to quantify the treatment difference. However, this model dependent estimate may be difficult to interpret clinically especially when the proportional hazards (PH) assumption is violated. An alternative estimation procedure for treatment efficacy based on the restricted means survival time or t-year mean survival time (t-MST) has been discussed extensively in the statistical and clinical literature. On the other hand, a statistical test 1 via the HR or its asymptotically equivalent counterpart, the logrank test, is asymptotically distribution-free. In this paper, we assess the …

Finding The Cutpoint Of A Continuous Covariate In A Parametric Survival Analysis Model, Kabita Joshi

Theses and Dissertations

In many clinical studies, continuous variables such as age, blood pressure and cholesterol are measured and analyzed. Often clinicians prefer to categorize these continuous variables into different groups, such as low and high risk groups. The goal of this work is to find the cutpoint of a continuous variable where the transition occurs from low to high risk group. Different methods have been published in literature to find such a cutpoint. We extended the methods of Contal and O’Quigley (1999) which was based on the log-rank test and the methods of Klein and Wu (2004) which was based on the …

Analytical Comparison Of Contrasting Approaches To Estimating Competing Risks Models, Brian Stephen Rickard

Graduate Theses and Dissertations

Survival analysis is a commonly used tool in many fields but has seen little use in education research despite a common number of research questions for which it is well suited. Researchers often use logistic regression instead; however, this omits useful information. In research on retention and graduation for example, the timing of the event is an important piece of information omitted when using logistic regression. A simulation study was conducted to evaluate four methods of analyzing competing risks survival data, Cox proportional hazards regression, Weibull regression, Fine and Gray's Method, and Cox proportional hazards regression with frailty. College student …

Characteristics Of Stem Success: A Survival Analysis Model Of Factors Influencing Time To Graduation Among Undergraduate Stem Majors, Riley K. Acton

Business and Economics Honors Papers

Producing more graduates in Science, Technology, Engineering, and Mathematics (STEM), as well as ensuring students complete college in a timely manner are both areas of national public policy interest. In order to improve these two outcomes, it is imperative to understand what factors lead undergraduate students to persist in, and ultimately graduate with STEM degrees. This paper uses data from the Beginning Postsecondary Students Longitudinal Study, provided by The National Center of Education Statistics, to model the time to baccalaureate degree among STEM majors using a Cox proportional hazard model.

Cox Regression Models With Functional Covariates For Survival Data, Jonathan E. Gellar, Elizabeth Colantuoni, Dale M. Needham, Ciprian M. Crainiceanu

Johns Hopkins University, Dept. of Biostatistics Working Papers

We extend the Cox proportional hazards model to cases when the exposure is a densely sampled functional process, measured at baseline. The fundamental idea is to combine penalized signal regression with methods developed for mixed effects proportional hazards models. The model is fit by maximizing the penalized partial likelihood, with smoothing parameters estimated by a likelihood-based criterion such as AIC or EPIC. The model may be extended to allow for multiple functional predictors, time varying coefficients, and missing or unequally-spaced data. Methods were inspired by and applied to a study of the association between time to death after hospital discharge …

Comparison Of Hazard, Odds And Risk Ratio In The Two-Sample Survival Problem, Benedict P. Dormitorio

Dissertations

Cox proportional hazards is the standard method for analyzing treatment efficacy when time-to-event data is available. In the absence of time-to-event, investigators may use logistic regression which only requires relative frequencies of events, or Poisson regression which requires only interval-summarized frequency tables of time-to-event. When event frequencies are used instead of time-to-events, does it always result in a loss in power?

We investigate the relative performance of the three methods. In particular, we compare the power of tests based on the respective effect-size estimates (1)hazard ratio (HR), (2)odds ratio (OR), and (3)risk ratio (RR). We use a variety of survival …

A Predictive Enrichment Procedure To Identify Potential Responders To A New Therapy For Randomized, Comparative, Controlled Clinical Studies, Junlong Li, Lihui Zhao, Lu Tian, Tianxi Cai, Brian Claggett, Andrea Callegaro, Benjamin Dizier, Bart Spiessens, Fernando Ulloa-Montoya, L. J. Wei

Harvard University Biostatistics Working Paper Series

No abstract provided.

Hypothesis Testing For An Extended Cox Model With Time-Varying Coefficients, Takumi Saegusa, Chongzhi Di, Ying Qing Chen

UW Biostatistics Working Paper Series

The log-rank test has been widely used to test a treatment effect under the Cox model for censored time-to-event outcomes, though it may lose power substantially when the model's proportional hazards assumption does not hold. In this paper, we consider an extended Cox model that uses B-splines or smoothing splines to model a time-varying treatment effect and propose score test statistics for the treatment effect. Our proposed new tests combine statistical evidence from both the magnitude and the shape of the time-varying hazard ratio function, and thus are omnibus and powerful against various types of alternatives. In addition, the new …

Penalized Smoothed Partial Rank Estimator For The Nonparametric Transformation Survival Model With High-Dimensional Covariates, Wei Dai, Yi Li

The University of Michigan Department of Biostatistics Working Paper Series

Microarray technology has the potential to lead to a better understanding of biological processes and diseases such as cancer. When failure time outcomes are also available, one might be interested in relating gene expression profiles to the survival outcome such as time to cancer recurrence or time to death. This is statistically challenging because the number of covariates greatly exceeds the number of observations. While the majority of work has focused on regularized Cox regression model and accelerated failure time model, they may be restrictive in practice. We relax the model assumption and and consider a nonparametric transformation model that …

A Frailty Approach For Survival Analysis With Error-Prone Covariate, Sehee Kim, Yi Li, Donna Spiegelman

The University of Michigan Department of Biostatistics Working Paper Series

This paper discovers an inherent relationship between the survival model with covariate measurement error and the frailty model. The discovery motivates our using a frailty-based estimating equation to draw inference for the proportional hazards model with error-prone covariates. Our established framework accommodates general distributional structures for the error-prone covariates, not restricted to a linear additive measurement error model or Gaussian measurement error. When the conditional distribution of the frailty given the surrogate is unknown, it is estimated through a semiparametric copula function. The proposed copula-based approach enables us to fit flexible measurement error models without the curse of dimensionality as …

Non-Likelihood Based Model Evaluation And Comparison With Application To Genetic And Clinical Hiv-1 Outcomes, Ashley Elise Giambrone

Legacy Theses & Dissertations (2009 - 2024)

Although treatment for human immunodeficiency virus type-1 (HIV-1) has undergone drastic change and morbidity and mortality has decreased over time, the development of drug-resistant HIV-1 is of concern for the long-term antiretroviral treatment of infected individuals. Drug-resistant virus is known to manifest with potentially complex mutational patterns in the HIV-1 genotype sequence and is associated with decreased response to therapy. Resistance occurs either as a result of development of mutations in the viral genome under selective drug pressure or as a result of naturally occurring polymorphisms. The most effective treatment methods are still debated at this time; however, current treatment …

A Monte Carlo Approach To Change Point Detection In A Liver Transplant, Alexia Melissa Makris

USF Tampa Graduate Theses and Dissertations

Patient survival post liver transplant (LT) is important to both the patient and the center's accreditation, but over the years physicians have noticed that distant patients struggle with post LT care. I hypothesized that patient's distance from the transplant center had a detrimental effect on post LT survival. I suspected Hepatitis C (HCV) and Hepatocellular Carcinoma (HCC) patients would deteriorate due to their recurrent disease and there is a need for close monitoring post LT. From the current literature it was not clear if patients' distance from a transplant center affects outcomes post LT. Firozvi et al. (Firozvi AA, 2008) …

Survival Prediction For Brain Tumor Patients Using Gene Expression Data, Vinicius Bonato

Dissertations and Theses (Open Access)

Brain tumor is one of the most aggressive types of cancer in humans, with an estimated median survival time of 12 months and only 4% of the patients surviving more than 5 years after disease diagnosis. Until recently, brain tumor prognosis has been based only on clinical information such as tumor grade and patient age, but there are reports indicating that molecular profiling of gliomas can reveal subgroups of patients with distinct survival rates. We hypothesize that coupling molecular profiling of brain tumors with clinical information might improve predictions of patient survival time and, consequently, better guide future treatment decisions. …

The Joint Distribution Of Bivariate Exponential Under Linearly Related Model, Norou Diawara, Kumer Pial Das

Mathematics & Statistics Faculty Publications

In this paper, fundamental results of the joint distribution of the bivariate exponential distributions are established. The positive support multivariate distribution theory is important in reliability and survival analysis, and we applied it to the case where more than one failure or survival is observed in a given study. Usually, the multivariate distribution is restricted to those with marginal distributions of a specified and familiar lifetime family. The family of exponential distribution contains the absolutely continuous and discrete case models with a nonzero probability on a set of measure zero. Examples are given, and estimators are developed and applied to …

Survival Analysis With High-Dimensional Covariates: An Application In Microarray Studies, David Engler, Yi Li

Faculty Publications

Use of microarray technology often leads to high-dimensional and low-sample size (HDLSS) data settings. A variety of approaches have been proposed for variable selection in this context. However, only a small number of these have been adapted for time-to-event data where censoring is present. Among standard variable selection methods shown both to have good predictive accuracy and to be computationally efficient is the elastic net penalization approach. In this paper, adaptations of the elastic net approach are presented for variable selection both under the Cox proportional hazards model and under an accelerated failure time (AFT) model. Assessment of the two …

A Note On Targeted Maximum Likelihood And Right Censored Data, Mark J. Van Der Laan, Daniel Rubin

U.C. Berkeley Division of Biostatistics Working Paper Series

A popular way to estimate an unknown parameter is with substitution, or evaluating the parameter at a likelihood based fit of the data generating density. In many cases, such estimators have substantial bias and can fail to converge at the parametric rate. van der Laan and Rubin (2006) introduced targeted maximum likelihood learning, removing these shackles from substitution estimators, which were made in full agreement with the locally efficient estimating equation procedures as presented in Robins and Rotnitzsky (1992) and van der Laan and Robins (2003). This note illustrates how targeted maximum likelihood can be applied in right censored data …

Empirical Efficiency Maximization, Daniel B. Rubin, Mark J. Van Der Laan

U.C. Berkeley Division of Biostatistics Working Paper Series

It has long been recognized that covariate adjustment can increase precision, even when it is not strictly necessary. The phenomenon is particularly emphasized in clinical trials, whether using continuous, categorical, or censored time-to-event outcomes. Adjustment is often straightforward when a discrete covariate partitions the sample into a handful of strata, but becomes more involved when modern studies collect copious amounts of baseline information on each subject.

The dilemma helped motivate locally efficient estimation for coarsened data structures, as surveyed in the books of van der Laan and Robins (2003) and Tsiatis (2006). Here one fits a relatively small working model …

Survival Point Estimate Prediction In Matched And Non-Matched Case-Control Subsample Designed Studies, Annette M. Molinaro, Mark J. Van Der Laan, Dan H. Moore, Karla Kerlikowske

U.C. Berkeley Division of Biostatistics Working Paper Series

Providing information about the risk of disease and clinical factors that may increase or decrease a patient's risk of disease is standard medical practice. Although case-control studies can provide evidence of strong associations between diseases and risk factors, clinicians need to be able to communicate to patients the age-specific risks of disease over a defined time interval for a set of risk factors.

An estimate of absolute risk cannot be determined from case-control studies because cases are generally chosen from a population whose size is not known (necessary for calculation of absolute risk) and where duration of follow-up is not …

Bias Of The Cox Model Hazard Ratio, Inger Persson, Harry Khamis

Journal of Modern Applied Statistical Methods

The hazard ratio estimated with the Cox model is investigated under proportional and five forms of nonproportional hazards. Results indicate that the highest bias occurs for diverging hazards with early censoring, and for increasing and crossing hazards under a high censoring rate.

Survival Ensembles, Torsten Hothorn, Peter Buhlmann, Sandrine Dudoit, Annette M. Molinaro, Mark J. Van Der Laan

U.C. Berkeley Division of Biostatistics Working Paper Series

We propose a unified and flexible framework for ensemble learning in the presence of censoring. For right-censored data, we introduce a random forest algorithm and a generic gradient boosting algorithm for the construction of prognostic models. The methodology is utilized for predicting the survival time of patients suffering from acute myeloid leukemia based on clinical and genetic covariates. Furthermore, we compare the diagnostic capabilities of the proposed censored data random forest and boosting methods applied to the recurrence free survival time of node positive breast cancer patients with previously published findings.

On A Simple Method For Analyzing Multivariate Survival Data Using Sample Survey Methods, Pingfu Fu, J. Sunil Rao

Journal of Modern Applied Statistical Methods

A simple technique is illustrated for analyzing multivariate survival data. The data situation arises when an individual records multiple survival events, or when individuals recording single survival events are grouped into clusters. Past work has focused on developing new methods to handle such data. Here, we use a connection between Poisson regression and survival modeling and a cluster sampling approach to adjust the variance estimates. The approach requires parametric assumption for the marginal hazard function, but avoids specification of a joint multivariate survival distribution. A simulation study demonstrates the proposed approach is a competing method of recent developed marginal approaches …

Semiparametric Quantitative-Trait-Locus Mapping: Ii. On Censored Age-At-Onset, Ying Qing Chen, Chengcheng Hu, Rongling Wu

U.C. Berkeley Division of Biostatistics Working Paper Series

In genetic studies, the variation in genotypes may not only affect different inheritance patterns in qualitative traits, but may also affect the age-at-onset as quantitative trait. In this article, we use standard cross designs, such as backcross or F2, to propose some hazard regression models, namely, the additive hazards model in quantitative trait loci mapping for age-at-onset, although the developed method can be extended to more complex designs. With additive invariance of the additive hazards models in mixture probabilities, we develop flexible semiparametric methodologies in interval regression mapping without heavy computing burden. A recently developed multiple comparison procedures is adapted …

Loss-Based Estimation With Cross-Validation: Applications To Microarray Data Analysis And Motif Finding, Sandrine Dudoit, Mark J. Van Der Laan, Sunduz Keles, Annette M. Molinaro, Sandra E. Sinisi, Siew Leng Teng

U.C. Berkeley Division of Biostatistics Working Paper Series

Current statistical inference problems in genomic data analysis involve parameter estimation for high-dimensional multivariate distributions, with typically unknown and intricate correlation patterns among variables. Addressing these inference questions satisfactorily requires: (i) an intensive and thorough search of the parameter space to generate good candidate estimators, (ii) an approach for selecting an optimal estimator among these candidates, and (iii) a method for reliably assessing the performance of the resulting estimator. We propose a unified loss-based methodology for estimator construction, selection, and performance assessment with cross-validation. In this approach, the parameter of interest is defined as the risk minimizer for a suitable …