Biostatistics Commons^™

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 …

Sars-Cov-2 Pandemic Analytical Overview With Machine Learning Predictability, Anthony Tanaydin, Jingchen Liang, Daniel W. Engels

SMU Data Science Review

Understanding diagnostic tests and examining important features of novel coronavirus (COVID-19) infection are essential steps for controlling the current pandemic of 2020. In this paper, we study the relationship between clinical diagnosis and analytical features of patient blood panels from the US, Mexico, and Brazil. Our analysis confirms that among adults, the risk of severe illness from COVID-19 increases with pre-existing conditions such as diabetes and immunosuppression. Although more than eight months into pandemic, more data have become available to indicate that more young adults were getting infected. In addition, we expand on the definition of COVID-19 test and discuss …

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. …