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Articles 151 - 180 of 205
Full-Text Articles in Biostatistics
A Regularization Corrected Score Method For Nonlinear Regression Models With Covariate Error, David M. Zucker, Malka Gorfine, Yi Li, Donna Spiegelman
A Regularization Corrected Score Method For Nonlinear Regression Models With Covariate Error, David M. Zucker, Malka Gorfine, Yi Li, Donna Spiegelman
Harvard University Biostatistics Working Paper Series
No abstract provided.
Effectively Selecting A Target Population For A Future Comparative Study, Lihui Zhao, Lu Tian, Tianxi Cai, Brian Claggett, L. J. Wei
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
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.
Estimation And Testing In Targeted Group Sequential Covariate-Adjusted Randomized Clinical Trials, Antoine Chambaz, Mark J. Van Der Laan
Estimation And Testing In Targeted Group Sequential Covariate-Adjusted Randomized Clinical Trials, Antoine Chambaz, Mark J. Van Der Laan
U.C. Berkeley Division of Biostatistics Working Paper Series
This article is devoted to the construction and asymptotic study of adaptive group sequential covariate-adjusted randomized clinical trials analyzed through the prism of the semiparametric methodology of targeted maximum likelihood estimation (TMLE). We show how to build, as the data accrue group-sequentially, a sampling design which targets a user-supplied optimal design. We also show how to carry out a sound TMLE statistical inference based on such an adaptive sampling scheme (therefore extending some results known in the i.i.d setting only so far), and how group-sequential testing applies on top of it. The procedure is robust (i.e., consistent even if the …
Minimum Description Length Measures Of Evidence For Enrichment, Zhenyu Yang, David R. Bickel
Minimum Description Length Measures Of Evidence For Enrichment, Zhenyu Yang, David R. Bickel
COBRA Preprint Series
In order to functionally interpret differentially expressed genes or other discovered features, researchers seek to detect enrichment in the form of overrepresentation of discovered features associated with a biological process. Most enrichment methods treat the p-value as the measure of evidence using a statistical test such as the binomial test, Fisher's exact test or the hypergeometric test. However, the p-value is not interpretable as a measure of evidence apart from adjustments in light of the sample size. As a measure of evidence supporting one hypothesis over the other, the Bayes factor (BF) overcomes this drawback of the p-value but lacks …
Landmark Prediction Of Survival, Layla Parast, Tianxi Cai
Landmark Prediction Of Survival, Layla Parast, Tianxi Cai
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
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.
Principled Sure Independence Screening For Cox Models With Ultra-High-Dimensional Covariates, Sihai Dave Zhao, Yi Li
Principled Sure Independence Screening For Cox Models With Ultra-High-Dimensional Covariates, Sihai Dave Zhao, Yi Li
Harvard University Biostatistics Working Paper Series
No abstract provided.
Estimating Causal Effects In Trials Involving Multi-Treatment Arms Subject To Non-Compliance: A Bayesian Frame-Work, Qi Long, Roderick J. Little, Xihong Lin
Estimating Causal Effects In Trials Involving Multi-Treatment Arms Subject To Non-Compliance: A Bayesian Frame-Work, Qi Long, Roderick J. Little, Xihong Lin
Harvard University Biostatistics Working Paper Series
No abstract provided.
Super Learner In Prediction, Eric C. Polley, Mark J. Van Der Laan
Super Learner In Prediction, Eric C. Polley, Mark J. Van Der Laan
U.C. Berkeley Division of Biostatistics Working Paper Series
Super learning is a general loss based learning method that has been proposed and analyzed theoretically in van der Laan et al. (2007). In this article we consider super learning for prediction. The super learner is a prediction method designed to find the optimal combination of a collection of prediction algorithms. The super learner algorithm finds the combination of algorithms minimizing the cross-validated risk. The super learner framework is built on the theory of cross-validation and allows for a general class of prediction algorithms to be considered for the ensemble. Due to the previously established oracle results for the cross-validation …
Assessing Noninferiority In A Three-Arm Trial Using The Bayesian Approach, Pulak Ghosh, Farouk S. Nathoo, Mithat Gonen, Ram C. Tiwari
Assessing Noninferiority In A Three-Arm Trial Using The Bayesian Approach, Pulak Ghosh, Farouk S. Nathoo, Mithat Gonen, Ram C. Tiwari
Memorial Sloan-Kettering Cancer Center, Dept. of Epidemiology & Biostatistics Working Paper Series
Non-inferiority trials, which aim to demonstrate that a test product is not worse than a competitor by more than a pre-specified small amount, are of great importance to the pharmaceutical community. As a result, methodology for designing and analyzing such trials is required, and developing new methods for such analysis is an important area of statistical research. The three-arm clinical trial is usually recommended for non-inferiority trials by the Food and Drug Administration (FDA). The three-arm trial consists of a placebo, a reference, and an experimental treatment, and simultaneously tests the superiority of the reference over the placebo along with …
Survival Prediction For Brain Tumor Patients Using Gene Expression Data, Vinicius Bonato
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. …
Nonparametric Regression With Missing Outcomes Using Weighted Kernel Estimating Equations, Lu Wang, Andrea Rotnitzky, Xihong Lin
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.
Likelihood Ratio Testing For Admixture Models With Application To Genetic Linkage Analysis, Chong-Zhi Di, Kung-Yee Liang
Likelihood Ratio Testing For Admixture Models With Application To Genetic Linkage Analysis, Chong-Zhi Di, Kung-Yee Liang
Johns Hopkins University, Dept. of Biostatistics Working Papers
We consider likelihood ratio tests (LRT) and their modifications for homogeneity in admixture models. The admixture model is a special case of two component mixture model, where one component is indexed by an unknown parameter while the parameter value for the other component is known. It has been widely used in genetic linkage analysis under heterogeneity, in which the kernel distribution is binomial. For such models, it is long recognized that testing for homogeneity is nonstandard and the LRT statistic does not converge to a conventional 2 distribution. In this paper, we investigate the asymptotic behavior of the LRT for …
Graphical Procedures For Evaluating Overall And Subject-Specific Incremental Values From New Predictors With Censored Event Time Data, Hajime Uno, Tianxi Cai, Lu Tian, L. J. Wei
Graphical Procedures For Evaluating Overall And Subject-Specific Incremental Values From New Predictors With Censored Event Time Data, Hajime Uno, Tianxi Cai, Lu Tian, L. J. Wei
Harvard University Biostatistics Working Paper Series
No abstract provided.
Readings In Targeted Maximum Likelihood Estimation, Mark J. Van Der Laan, Sherri Rose, Susan Gruber
Readings In Targeted Maximum Likelihood Estimation, Mark J. Van Der Laan, Sherri Rose, Susan Gruber
U.C. Berkeley Division of Biostatistics Working Paper Series
This is a compilation of current and past work on targeted maximum likelihood estimation. It features the original targeted maximum likelihood learning paper as well as chapters on super (machine) learning using cross validation, randomized controlled trials, realistic individualized treatment rules in observational studies, biomarker discovery, case-control studies, and time-to-event outcomes with censored data, among others. We hope this collection is helpful to the interested reader and stimulates additional research in this important area.
Causal Inference For Nested Case-Control Studies Using Targeted Maximum Likelihood Estimation, Sherri Rose, Mark J. Van Der Laan
Causal Inference For Nested Case-Control Studies Using Targeted Maximum Likelihood Estimation, Sherri Rose, Mark J. Van Der Laan
U.C. Berkeley Division of Biostatistics Working Paper Series
A nested case-control study is conducted within a well-defined cohort arising out of a population of interest. This design is often used in epidemiology to reduce the costs associated with collecting data on the full cohort; however, the case control sample within the cohort is a biased sample. Methods for analyzing case-control studies have largely focused on logistic regression models that provide conditional and not marginal causal estimates of the odds ratio. We previously developed a Case-Control Weighted Targeted Maximum Likelihood Estimation (TMLE) procedure for case-control study designs, which relies on the prevalence probability q0. We propose the use of …
Comparing Risk Scoring Systems Beyond The Roc Paradigm In Survival Analysis, Hajime Uno, Lu Tian, Tianxi Cai, Isaac S. Kohane, L. J. Wei
Comparing Risk Scoring Systems Beyond The Roc Paradigm In Survival Analysis, Hajime Uno, Lu Tian, Tianxi Cai, Isaac S. Kohane, L. J. Wei
Harvard University Biostatistics Working Paper Series
No abstract provided.
Marginalized Frailty Models For Multivariate Survival Data, Megan Othus, Yi Li
Marginalized Frailty Models For Multivariate Survival Data, Megan Othus, Yi Li
Harvard University Biostatistics Working Paper Series
No abstract provided.
Estimating Subject-Specific Dependent Competing Risk Profile With Censored Event Time Observations, Yi Li, Lu Tian, L. J. Wei
Estimating Subject-Specific Dependent Competing Risk Profile With Censored Event Time Observations, Yi Li, Lu Tian, L. J. Wei
Harvard University Biostatistics Working Paper Series
No abstract provided.
A Class Of Semiparametric Mixture Cure Survival Models With Dependent Censoring, Megan Othus, Yi Li, Ram C. Tiwari
A Class Of Semiparametric Mixture Cure Survival Models With Dependent Censoring, Megan Othus, Yi Li, Ram C. Tiwari
Harvard University Biostatistics Working Paper Series
No abstract provided.
Joint Multiple Testing Procedures For Graphical Model Selection With Applications To Biological Networks, Houston N. Gilbert, Mark J. Van Der Laan, Sandrine Dudoit
Joint Multiple Testing Procedures For Graphical Model Selection With Applications To Biological Networks, Houston N. Gilbert, Mark J. Van Der Laan, Sandrine Dudoit
U.C. Berkeley Division of Biostatistics Working Paper Series
Gaussian graphical models have become popular tools for identifying relationships between genes when analyzing microarray expression data. In the classical undirected Gaussian graphical model setting, conditional independence relationships can be inferred from partial correlations obtained from the concentration matrix (= inverse covariance matrix) when the sample size n exceeds the number of parameters p which need to estimated. In situations where n < p, another approach to graphical model estimation may rely on calculating unconditional (zero-order) and first-order partial correlations. In these settings, the goal is to identify a lower-order conditional independence graph, sometimes referred to as a ‘0-1 graphs’. For either choice of graph, model selection may involve a multiple testing problem, in which edges in a graph are drawn only after rejecting hypotheses involving (saturated or lower-order) partial correlation parameters. Most multiple testing procedures applied in previously proposed graphical model selection algorithms rely on standard, marginal testing methods which do not take into account the joint distribution of the test statistics derived from (partial) correlations. We propose and implement a multiple testing framework useful when testing for edge inclusion during graphical model selection. Two features of our methodology include (i) a computationally efficient and asymptotically valid test statistics joint null distribution derived from influence curves for correlation-based parameters, and (ii) the application of empirical Bayes joint multiple testing procedures which can effectively control a variety of popular Type I error rates by incorpo- rating joint null distributions such as those described here (Dudoit and van der Laan, 2008). Using a dataset from Arabidopsis thaliana, we observe that the use of more sophisticated, modular approaches to multiple testing allows one to identify greater numbers of edges when approximating an undirected graphical model using a 0-1 graph. Our framework may also be extended to edge testing algorithms for other types of graphical models (e.g., for classical undirected, bidirected, and directed acyclic graphs).
Analysis Of Randomized Comparative Clinical Trial Data For Personalized Treatment Selections, Tianxi Cai, Lu Tian, Peggy H. Wong, L. J. Wei
Analysis Of Randomized Comparative Clinical Trial Data For Personalized Treatment Selections, Tianxi Cai, Lu Tian, Peggy H. Wong, L. J. Wei
Harvard University Biostatistics Working Paper Series
No abstract provided.
A Small Sample Correction For Estimating Attributable Risk In Case-Control Studies, Daniel B. Rubin
A Small Sample Correction For Estimating Attributable Risk In Case-Control Studies, Daniel B. Rubin
U.C. Berkeley Division of Biostatistics Working Paper Series
The attributable risk, often called the population attributable risk, is in many epidemiological contexts a more relevant measure of exposure-disease association than the excess risk, relative risk, or odds ratio. When estimating attributable risk with case-control data and a rare disease, we present a simple correction to the standard approach making it essentially unbiased, and also less noisy. As with analogous corrections given in Jewell (1986) for other measures of association, the adjustment often won't make a substantial difference unless the sample size is very small or point estimates are desired within fine strata, but we discuss the possible utility …
The Highest Confidence Density Region And Its Usage For Inferences About The Survival Function With Censored Data, Lu Tian, Rui Wang, Tianxi Cai, L. J. Wei
The Highest Confidence Density Region And Its Usage For Inferences About The Survival Function With Censored Data, Lu Tian, Rui Wang, Tianxi Cai, L. J. Wei
Harvard University Biostatistics Working Paper Series
No abstract provided.
Calibrating Parametric Subject-Specific Risk Estimation, Tianxi Cai, Lu Tian, Hajime Uno, Scott D. Solomon, L. J. Wei
Calibrating Parametric Subject-Specific Risk Estimation, Tianxi Cai, Lu Tian, Hajime Uno, Scott D. Solomon, L. J. Wei
Harvard University Biostatistics Working Paper Series
No abstract provided.
Evaluating Subject-Level Incremental Values Of New Markers For Risk Classification Rule, Tianxi Cai, Lu Tian, Donald M. Lloyd-Jones, L. J. Wei
Evaluating Subject-Level Incremental Values Of New Markers For Risk Classification Rule, Tianxi Cai, Lu Tian, Donald M. Lloyd-Jones, L. J. Wei
Harvard University Biostatistics Working Paper Series
No abstract provided.
Confidence Intervals For Negative Binomial Random Variables Of High Dispersion, David Shilane, Alan E. Hubbard, S N. Evans
Confidence Intervals For Negative Binomial Random Variables Of High Dispersion, David Shilane, Alan E. Hubbard, S N. Evans
U.C. Berkeley Division of Biostatistics Working Paper Series
This paper considers the problem of constructing confidence intervals for the mean of a Negative Binomial random variable based upon sampled data. When the sample size is large, we traditionally rely upon a Normal distribution approximation to construct these intervals. However, we demonstrate that the sample mean of highly dispersed Negative Binomials exhibits a slow convergence to the Normal in distribution as a function of the sample size. As a result, standard techniques (such as the Normal approximation and bootstrap) that construct confidence intervals for the mean will typically be too narrow and significantly undercover in the case of high …
Estimation And Testing For The Effect Of A Genetic Pathway On A Disease Outcome Using Logistic Kernel Machine Regression Via Logistic Mixed Models, Dawei Liu, Debashis Ghosh, Xihong Lin
Estimation And Testing For The Effect Of A Genetic Pathway On A Disease Outcome Using Logistic Kernel Machine Regression Via Logistic Mixed Models, Dawei Liu, Debashis Ghosh, Xihong Lin
Harvard University Biostatistics Working Paper Series
No abstract provided.
A Powerful And Flexible Multilocus Association Test For Quantitative Traits, Lydia Coulter Kwee, Dawei Liu, Xihong Lin, Debashis Ghosh, Michael P. Epstein
A Powerful And Flexible Multilocus Association Test For Quantitative Traits, Lydia Coulter Kwee, Dawei Liu, Xihong Lin, Debashis Ghosh, Michael P. Epstein
Harvard University Biostatistics Working Paper Series
No abstract provided.