Statistical Models Commons^™

Forecasting Remission Time Of A Treatment Method For Leukemia As An Application To Statistical Inference Approach, Ahmed Galal Atia, Mahmoud Mansour, Rashad Mohamed El-Sagheer, B. S. El-Desouky

Basic Science Engineering

In this paper, Weibull-Linear Exponential distribution (WLED) has been investigated whether being it is a well-fit distribution to a clinical real data. These data represent the duration of remission achieved by a certain drug used in the treatment of leukemia for a group of patients. The statistical inference approach is used to estimate the parameters of the WLED through the set of the fitted data. The estimated parameters are utilized to evaluate the survival and hazard functions and hence assessing the treatment method through forecasting the duration of remission times of patients. A two-sample prediction approach has been applied to …

Unified Methods For Feature Selection In Large-Scale Genomic Studies With Censored Survival Outcomes, Lauren Spirko-Burns, Karthik Devarajan

COBRA Preprint Series

One of the major goals in large-scale genomic studies is to identify genes with a prognostic impact on time-to-event outcomes which provide insight into the disease's process. With rapid developments in high-throughput genomic technologies in the past two decades, the scientific community is able to monitor the expression levels of tens of thousands of genes and proteins resulting in enormous data sets where the number of genomic features is far greater than the number of subjects. Methods based on univariate Cox regression are often used to select genomic features related to survival outcome; however, the Cox model assumes proportional hazards …

Non Parametric Test For Testing Exponentiality Against Exponential Better Than Used In Laplace Transform Order, Mahmoud Mansour, M A W Mahmoud Prof.

Basic Science Engineering

In this paper, the test statistic for testing exponentiality against exponential better than used in Laplace transform order (EBUL) based on the Laplace transform technique is proposed. Pitman’s asymptotic efficiency of our test is calculated and compared with other tests. The percentiles of this test are tabulated. The powers of the test are estimated for famously used distributions in aging problems. In the case of censored data, our test is applied and the percentiles are also calculated and tabulated. Finally, real examples in different areas are utilized as practical applications for the proposed test.

Burden Of Atopic Dermatitis In The United States: Analysis Of Healthcare Claims Data In The Commercial, Medicare, And Medi-Cal Databases, Sulena Shrestha, Raymond Miao, Li Wang, Jingdong Chao, Huseyin Yuce, Wenhui Wei

Publications and Research

Comparative data on the burden of atopic dermatitis (AD) in adults relative to the general population are limited. We performed a large-scale evaluation of the burden of disease among US adults with AD relative to matched non-AD controls, encompassing comorbidities, healthcare resource utilization (HCRU), and costs, using healthcare claims data. The impact of AD disease severity on these outcomes was also evaluated.

Inference On The Stress-Strength Model From Weibull Gamma Distribution, Mahmoud Mansour, Rashad El-Sagheer, M. A. W. Mahmoud Prof.

Basic Science Engineering

No abstract provided.

Models For Hsv Shedding Must Account For Two Levels Of Overdispersion, Amalia Magaret

UW Biostatistics Working Paper Series

We have frequently implemented crossover studies to evaluate new therapeutic interventions for genital herpes simplex virus infection. The outcome measured to assess the efficacy of interventions on herpes disease severity is the viral shedding rate, defined as the frequency of detection of HSV on the genital skin and mucosa. We performed a simulation study to ascertain whether our standard model, which we have used previously, was appropriately considering all the necessary features of the shedding data to provide correct inference. We simulated shedding data under our standard, validated assumptions and assessed the ability of 5 different models to reproduce the …

Depicting Estimates Using The Intercept In Meta-Regression Models: The Moving Constant Technique, Blair T. Johnson Dr., Tania B. Huedo-Medina Dr.

CHIP Documents

In any scientific discipline, the ability to portray research patterns graphically often aids greatly in interpreting a phenomenon. In part to depict phenomena, the statistics and capabilities of meta-analytic models have grown increasingly sophisticated. Accordingly, this article details how to move the constant in weighted meta-analysis regression models (viz. “meta-regression”) to illuminate the patterns in such models across a range of complexities. Although it is commonly ignored in practice, the constant (or intercept) in such models can be indispensible when it is not relegated to its usual static role. The moving constant technique makes possible estimates and confidence intervals at …

Effectively Selecting A Target Population For A Future Comparative Study, Lihui Zhao, Lu Tian, Tianxi Cai, Brian Claggett, L. J. Wei

Harvard University Biostatistics Working Paper Series

When comparing a new treatment with a control in a randomized clinical study, the treatment effect is generally assessed by evaluating a summary measure over a specific study population. The success of the trial heavily depends on the choice of such a population. In this paper, we show a systematic, effective way to identify a promising population, for which the new treatment is expected to have a desired benefit, using the data from a current study involving similar comparator treatments. Specifically, with the existing data we first create a parametric scoring system using multiple covariates to estimate subject-specific treatment differences. …

On The Covariate-Adjusted Estimation For An Overall Treatment Difference With Data From A Randomized Comparative Clinical Trial, Lu Tian, Tianxi Cai, Lihui Zhao, L. J. Wei

Harvard University Biostatistics Working Paper Series

No abstract provided.

Estimating Subject-Specific Treatment Differences For Risk-Benefit Assessment With Competing Risk Event-Time Data, Brian Claggett, Lihui Zhao, Lu Tian, Davide Castagno, L. J. Wei

Harvard University Biostatistics Working Paper Series

No abstract provided.

Stratifying Subjects For Treatment Selection With Censored Event Time Data From A Comparative Study, Lihui Zhao, Tianxi Cai, Lu Tian, Hajime Uno, Scott D. Solomon, L. J. Wei

Harvard University Biostatistics Working Paper Series

No abstract provided.

Nonparametric Regression With Missing Outcomes Using Weighted Kernel Estimating Equations, Lu Wang, Andrea Rotnitzky, Xihong Lin

Harvard University Biostatistics Working Paper Series

No abstract provided.

Causal Comparisons In Randomized Trials Of Two Active Treatments: The Effect Of Supervised Exercise To Promote Smoking Cessation, Jason Roy, Joseph W. Hogan

COBRA Preprint Series

In behavioral medicine trials, such as smoking cessation trials, two or more active treatments are often compared. Noncompliance by some subjects with their assigned treatment poses a challenge to the data analyst. Causal parameters of interest might include those defined by subpopulations based on their potential compliance status under each assignment, using the principal stratification framework (e.g., causal effect of new therapy compared to standard therapy among subjects that would comply with either intervention). Even if subjects in one arm do not have access to the other treatment(s), the causal effect of each treatment typically can only be identified from …

Semiparametric Bayesian Modeling Of Multivariate Average Bioequivalence, Pulak Ghosh Dr., Mithat Gonen

Memorial Sloan-Kettering Cancer Center, Dept. of Epidemiology & Biostatistics Working Paper Series

Bioequivalence trials are usually conducted to compare two or more formulations of a drug. Simultaneous assessment of bioequivalence on multiple endpoints is called multivariate bioequivalence. Despite the fact that some tests for multivariate bioequivalence are suggested, current practice usually involves univariate bioequivalence assessments ignoring the correlations between the endpoints such as AUC and Cmax. In this paper we develop a semiparametric Bayesian test for bioequivalence under multiple endpoints. Specifically, we show how the correlation between the endpoints can be incorporated in the analysis and how this correlation affects the inference. Resulting estimates and posterior probabilities ``borrow strength'' from one another …

New Statistical Paradigms Leading To Web-Based Tools For Clinical/Translational Science, Knut M. Wittkowski

COBRA Preprint Series

As the field of functional genetics and genomics is beginning to mature, we become confronted with new challenges. The constant drop in price for sequencing and gene expression profiling as well as the increasing number of genetic and genomic variables that can be measured makes it feasible to address more complex questions. The success with rare diseases caused by single loci or genes has provided us with a proof-of-concept that new therapies can be developed based on functional genomics and genetics.

Common diseases, however, typically involve genetic epistasis, genomic pathways, and proteomic pattern. Moreover, to better understand the underlying biologi-cal …

The Optimal Confidence Region For A Random Parameter, Hajime Uno, Lu Tian, L.J. Wei

Harvard University Biostatistics Working Paper Series

Under a two-level hierarchical model, suppose that the distribution of the random parameter is known or can be estimated well. Data are generated via a fixed, but unobservable realization of this parameter. In this paper, we derive the smallest confidence region of the random parameter under a joint Bayesian/frequentist paradigm. On average this optimal region can be much smaller than the corresponding Bayesian highest posterior density region. The new estimation procedure is appealing when one deals with data generated under a highly parallel structure, for example, data from a trial with a large number of clinical centers involved or genome-wide …

Mean Response Models Of Repeated Measurements In Presence Of Varying Effectiveness Onset, Ying Qing Chen, Su-Chun Cheng

U.C. Berkeley Division of Biostatistics Working Paper Series

Repeated measurements are often collected over time to evaluate treatment efficacy in clinical trials. Most of the statistical models of the repeated measurements have been focusing on their mean response as function of time. These models usually assume that the treatment has persistent effect of constant additivity or multiplicity on the mean response functions throughout the observation period of time. In reality, however, such assumption may be confounded by the potential existence of the so-called effectiveness action onset, although they are often unobserved or difficult to obtain. Instead of including nonparametric time-varying coefficients in the mean response models, we propose …

Comparison Of The Inverse Probability Of Treatment Weighted (Iptw) Estimator With A Naïve Estimator In The Analysis Of Longitudinal Data With Time-Dependent Confounding: A Simulation Study, Thaddeus Haight, Romain Neugebauer, Ira B. Tager, Mark J. Van Der Laan

U.C. Berkeley Division of Biostatistics Working Paper Series

A simulation study was conducted to compare estimates from a naïve estimator, using standard conditional regression, and an IPTW (Inverse Probability of Treatment Weighted) estimator, to true causal parameters for a given MSM (Marginal Structural Model). The study was extracted from a larger epidemiological study (Longitudinal Study of Effects of Physical Activity and Body Composition on Functional Limitation in the Elderly, by Tager et. al [accepted, Epidemiology, September 2003]), which examined the causal effects of physical activity and body composition on functional limitation. The simulation emulated the larger study in terms of the exposure and outcome variables of interest-- physical …

A Nonparametric Comparison Of Conditional Distributions With Nonnegligible Cure Fractions, Yi Li, Jin Feng

Harvard University Biostatistics Working Paper Series

No abstract provided.

Measuring Treatment Effects Using Semiparametric Models, Zhuo Yu, Mark J. Van Der Laan

U.C. Berkeley Division of Biostatistics Working Paper Series

In order to estimate the causal effect of treatments on an outcome of interest, one has to account for the effect of confounding factors which covary with the treatments and also contribute to the outcome of interest. In this paper, we use the semiparametric regression model to estimate the causal parameters. We assume the causal effect of the treatments can be described by the parametric component of the semiparametric regression model. Following the general methodology which was developed in van der Laan and Robins (2002) we give the orthogonal complement of the nuisance tangent space which identifies all the estimating …

An Extended General Location Model For Causal Inference From Data Subject To Noncompliance And Missing Values, Yahong Peng, Rod Little, Trivellore E. Raghuanthan

The University of Michigan Department of Biostatistics Working Paper Series

Noncompliance is a common problem in experiments involving randomized assignment of treatments, and standard analyses based on intention-to treat or treatment received have limitations. An attractive alternative is to estimate the Complier-Average Causal Effect (CACE), which is the average treatment effect for the subpopulation of subjects who would comply under either treatment (Angrist, Imbens and Rubin, 1996, henceforth AIR). We propose an Extended General Location Model to estimate the CACE from data with non-compliance and missing data in the outcome and in baseline covariates. Models for both continuous and categorical outcomes and ignorable and latent ignorable (Frangakis and Rubin, 1999) …

Mixtures Of Varying Coefficient Models For Longitudinal Data With Discrete Or Continuous Non-Ignorable Dropout, Joseph W. Hogan, Xihong Lin, Benjamin A. Herman

The University of Michigan Department of Biostatistics Working Paper Series

The analysis of longitudinal repeated measures data is frequently complicated by missing data due to informative dropout. We describe a mixture model for joint distribution for longitudinal repeated measures, where the dropout distribution may be continuous and the dependence between response and dropout is semiparametric. Specifically, we assume that responses follow a varying coefficient random effects model conditional on dropout time, where the regression coefficients depend on dropout time through unspecified nonparametric functions that are estimated using step functions when dropout time is discrete (e.g., for panel data) and using smoothing splines when dropout time is continuous. Inference under the …

Estimating Causal Parameters In Marginal Structural Models With Unmeasured Confounders Using Instrumental Variables, Tanya A. Henneman, Mark Johannes Van Der Laan, Alan E. Hubbard

U.C. Berkeley Division of Biostatistics Working Paper Series

For statisticians analyzing medical data, a significant problem in determining the causal effect of a treatment on a particular outcome of interest, is how to control for unmeasured confounders. Techniques using instrumental variables (IV) have been developed to estimate causal parameters in the presence of unmeasured confounders. In this paper we apply IV methods to both linear and non-linear marginal structural models. We study a specific class of generalized estimating equations that is appropriate to these data, and compare the performance of the resulting estimator to the standard IV method, a two-stage least squares procedure. Our results are applied to …

Mixture Hazards Models With Additive Random Effects Accounting For Treatment Effectiveness Lag Time, Ying Qing Chen, C. A. Rohde, M.-C. Wang

U.C. Berkeley Division of Biostatistics Working Paper Series

In many clinical trials to evaluate treatment efficacy, it is believed that there may exist latent treatment effectiveness lag times after which medical treatment procedure or chemical compound would be in full effect. In this article, semiparametric regression models are proposed and studied for estimating the treatment effect accounting for such latent lag times. The new models take advantage of the invariance property of the additive hazards model in marginalising over an additive latent variable; parameters in the models are thus easily estimated and interpreted, while the flexibility of not having to specify the baseline hazard function is preserved. Monte …

A Class Of Semiparametric Scale-Change Hazards Regression Models And Its Adequacy For Censored Survival Data, Ying Qing Chen

U.C. Berkeley Division of Biostatistics Working Paper Series

A class of semiparametric hazards regression models called the accelerated hazards models was introduced to identify the covariate effect characterized by the scale-change between hazard functions. In this article, we compare the accelerated hazards models with several other popular classes of regression models in statistical literature for censored survival data. We also propose and study some test statistics to assess the models' adequacy. Simulation studies are conducted to evaluate the performance of the test statistics. Actual clinical trials data are analyzed to demonstrate the proposed models and test statistics.