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Articles 31 - 44 of 44

Full-Text Articles in Statistical Theory

Direct Effect Models, Mark J. Van Der Laan, Maya L. Petersen Aug 2005

Direct Effect Models, Mark J. Van Der Laan, Maya L. Petersen

U.C. Berkeley Division of Biostatistics Working Paper Series

The causal effect of a treatment on an outcome is generally mediated by several intermediate variables. Estimation of the component of the causal effect of a treatment that is mediated by a given intermediate variable (the indirect effect of the treatment), and the component that is not mediated by that intermediate variable (the direct effect of the treatment) is often relevant to mechanistic understanding and to the design of clinical and public health interventions. Under the assumption of no-unmeasured confounders for treatment and the intermediate variable, Robins & Greenland (1992) define an individual direct effect as the counterfactual effect of …


Causal Inference In Longitudinal Studies With History-Restricted Marginal Structural Models, Romain Neugebauer, Mark J. Van Der Laan, Ira B. Tager Apr 2005

Causal Inference In Longitudinal Studies With History-Restricted Marginal Structural Models, Romain Neugebauer, Mark J. Van Der Laan, Ira B. Tager

U.C. Berkeley Division of Biostatistics Working Paper Series

Causal Inference based on Marginal Structural Models (MSMs) is particularly attractive to subject-matter investigators because MSM parameters provide explicit representations of causal effects. We introduce History-Restricted Marginal Structural Models (HRMSMs) for longitudinal data for the purpose of defining causal parameters which may often be better suited for Public Health research. This new class of MSMs allows investigators to analyze the causal effect of a treatment on an outcome based on a fixed, shorter and user-specified history of exposure compared to MSMs. By default, the latter represents the treatment causal effect of interest based on a treatment history defined by the …


Data Adaptive Estimation Of The Treatment Specific Mean, Yue Wang, Oliver Bembom, Mark J. Van Der Laan Oct 2004

Data Adaptive Estimation Of The Treatment Specific Mean, Yue Wang, Oliver Bembom, Mark J. Van Der Laan

U.C. Berkeley Division of Biostatistics Working Paper Series

An important problem in epidemiology and medical research is the estimation of the causal effect of a treatment action at a single point in time on the mean of an outcome, possibly within strata of the target population defined by a subset of the baseline covariates. Current approaches to this problem are based on marginal structural models, i.e., parametric models for the marginal distribution of counterfactural outcomes as a function of treatment and effect modifiers. The various estimators developed in this context furthermore each depend on a high-dimensional nuisance parameter whose estimation currently also relies on parametric models. Since misspecification …


History-Adjusted Marginal Structural Models And Statically-Optimal Dynamic Treatment Regimes, Mark J. Van Der Laan, Maya L. Petersen Sep 2004

History-Adjusted Marginal Structural Models And Statically-Optimal Dynamic Treatment Regimes, Mark J. Van Der Laan, Maya L. Petersen

U.C. Berkeley Division of Biostatistics Working Paper Series

Marginal structural models (MSM) provide a powerful tool for estimating the causal effect of a treatment. These models, introduced by Robins, model the marginal distributions of treatment-specific counterfactual outcomes, possibly conditional on a subset of the baseline covariates. Marginal structural models are particularly useful in the context of longitudinal data structures, in which each subject's treatment and covariate history are measured over time, and an outcome is recorded at a final time point. However, the utility of these models for some applications has been limited by their inability to incorporate modification of the causal effect of treatment by time-varying covariates. …


Estimation Of Treatment Effects In Randomized Trials With Noncompliance And A Dichotomous Outcome , Mark J. Van Der Laan, Alan E. Hubbard, Nicholas P. Jewell Sep 2004

Estimation Of Treatment Effects In Randomized Trials With Noncompliance And A Dichotomous Outcome , Mark J. Van Der Laan, Alan E. Hubbard, Nicholas P. Jewell

U.C. Berkeley Division of Biostatistics Working Paper Series

We propose a class of estimators of the treatment effect on a dichotomous outcome among the treated subjects within covariate and treatment arm strata in randomized trials with non-compliance. Recent articles by Vansteelandt and Goethebeur (2003) and Robins and Rotnitzky (2004) have presented consistent and asymptotically linear estimators of a causal odds ratio, which rely, beyond correct specification of a model for the causal odds ratio, on a correctly specified model for a potentially high dimensional nuisance parameter. In this article we propose consistent, asymptotically linear and locally efficient estimators of a causal relative risk and a new parameter -- …


Estimation Of Direct And Indirect Causal Effects In Longitudinal Studies, Mark J. Van Der Laan, Maya L. Petersen Aug 2004

Estimation Of Direct And Indirect Causal Effects In Longitudinal Studies, Mark J. Van Der Laan, Maya L. Petersen

U.C. Berkeley Division of Biostatistics Working Paper Series

The causal effect of a treatment on an outcome is generally mediated by several intermediate variables. Estimation of the component of the causal effect of a treatment that is mediated by a given intermediate variable (the indirect effect of the treatment), and the component that is not mediated by that intermediate variable (the direct effect of the treatment) is often relevant to mechanistic understanding and to the design of clinical and public health interventions. Under the assumption of no-unmeasured confounders, Robins & Greenland (1992) and Pearl (2000), develop two identifiability results for direct and indirect causal effects. They define an …


Multiple Testing Methods For Chip-Chip High Density Oligonucleotide Array Data, Sunduz Keles, Mark J. Van Der Laan, Sandrine Dudoit, Simon E. Cawley Jun 2004

Multiple Testing Methods For Chip-Chip High Density Oligonucleotide Array Data, Sunduz Keles, Mark J. Van Der Laan, Sandrine Dudoit, Simon E. Cawley

U.C. Berkeley Division of Biostatistics Working Paper Series

Cawley et al. (2004) have recently mapped the locations of binding sites for three transcription factors along human chromosomes 21 and 22 using ChIP-Chip experiments. ChIP-Chip experiments are a new approach to the genome-wide identification of transcription factor binding sites and consist of chromatin (Ch) immunoprecipitation (IP) of transcription factor-bound genomic DNA followed by high density oligonucleotide hybridization (Chip) of the IP-enriched DNA. We investigate the ChIP-Chip data structure and propose methods for inferring the location of transcription factor binding sites from these data. The proposed methods involve testing for each probe whether it is part of a bound sequence …


On Corrected Score Approach For Proportional Hazards Model With Covariate Measurement Error, Xiao Song, Yijian Huang May 2004

On Corrected Score Approach For Proportional Hazards Model With Covariate Measurement Error, Xiao Song, Yijian Huang

UW Biostatistics Working Paper Series

In the presence of covariate measurement error with the proportional hazards model, several functional modeling methods have been proposed. These include the conditional score estimator (Tsiatis and Davidian, 2001), the parametric correction estimator (Nakamura, 1992) and the nonparametric correction estimator (Huang and Wang, 2000, 2003) in the order of weaker assumptions on the error. Although they are all consistent, each suffers from potential difficulties with small samples and substantial measurement error. In this article, upon noting that the conditional score and parametric correction estimators are asymptotically equivalent in the case of normal error, we investigate their relative finite sample performance …


Improved Confidence Intervals For The Sensitivity At A Fixed Level Of Specificity Of A Continuous-Scale Diagnostic Test, Xiao-Hua Zhou, Gengsheng Qin May 2003

Improved Confidence Intervals For The Sensitivity At A Fixed Level Of Specificity Of A Continuous-Scale Diagnostic Test, Xiao-Hua Zhou, Gengsheng Qin

UW Biostatistics Working Paper Series

For a continuous-scale test, it is an interest to construct a confidence interval for the sensitivity of the diagnostic test at the cut-off that yields a predetermined level of its specificity (eg. 80%, 90%, or 95%). IN this paper we proposed two new intervals for the sensitivity of a continuous-scale diagnostic test at a fixed level of specificity. We then conducted simulation studies to compare the relative performance of these two intervals with the best existing BCa bootstrap interval, proposed by Platt et al. (2000). Our simulation results showed that the newly proposed intervals are better than the BCa bootstrap …


A Bootstrap Confidence Interval Procedure For The Treatment Effect Using Propensity Score Subclassification, Wanzhu Tu, Xiao-Hua Zhou May 2003

A Bootstrap Confidence Interval Procedure For The Treatment Effect Using Propensity Score Subclassification, Wanzhu Tu, Xiao-Hua Zhou

UW Biostatistics Working Paper Series

In the analysis of observational studies, propensity score subclassification has been shown to be a powerful method for adjusting unbalanced covariates for the purpose of causal inferences. One practical difficulty in carrying out such an analysis is to obtain a correct variance estimate for such inferences, while reducing bias in the estimate of the treatment effect due to an imbalance in the measured covariates. In this paper, we propose a bootstrap procedure for the inferences concerning the average treatment effect; our bootstrap method is based on an extension of Efron’s bias-corrected accelerated (BCa) bootstrap confidence interval to a two-sample problem. …


The Analysis Of Placement Values For Evaluating Discriminatory Measures, Margaret S. Pepe, Tianxi Cai Sep 2002

The Analysis Of Placement Values For Evaluating Discriminatory Measures, Margaret S. Pepe, Tianxi Cai

UW Biostatistics Working Paper Series

The idea of using measurements such as biomarkers, clinical data, or molecular biology assays for classification and prediction is popular in modern medicine. The scientific evaluation of such measures includes assessing the accuracy with which they predict the outcome of interest. Receiver operating characteristic curves are commonly used for evaluating the accuracy of diagnostic tests. They can be applied more broadly, indeed to any problem involving classification to two states or populations (D = 0 or D = 1). We show that the ROC curve can be interpreted as a cumulative distribution function for the discriminatory measure Y in the …


Case-Control Current Status Data, Nicholas P. Jewell, Mark J. Van Der Laan Sep 2002

Case-Control Current Status Data, Nicholas P. Jewell, Mark J. Van Der Laan

U.C. Berkeley Division of Biostatistics Working Paper Series

Current status observation on survival times has recently been widely studied. An extreme form of interval censoring, this data structure refers to situations where the only available information on a survival random variable, T, is whether or not T exceeds a random independent monitoring time C, a binary random variable, Y. To date, nonparametric analyses of current status data have assumed the availability of i.i.d. random samples of the random variable (Y, C), or a similar random sample at each of a set of fixed monitoring times. In many situations, it is useful to consider a case-control sampling scheme. Here, …


Current Status Data: Review, Recent Developments And Open Problems, Nicholas P. Jewell, Mark J. Van Der Laan Sep 2002

Current Status Data: Review, Recent Developments And Open Problems, Nicholas P. Jewell, Mark J. Van Der Laan

U.C. Berkeley Division of Biostatistics Working Paper Series

Researchers working with survival data are by now adept at handling issues associated with incomplete data, particular those associated with various forms of censoring. An extreme form of interval censoring, known as current status observation, refers to situations where the only available information on a survival random variable T is whether or not T exceeds a random independent monitoring time C. This article contains a brief review of the extensive literature on the analysis of current status data, discussing the implications of response-based sampling on these methods. The majority of the paper introduces some recent extensions of these ideas to …


Assessing The Accuracy Of A New Diagnostic Test When A Gold Standard Does Not Exist, Todd A. Alonzo, Margaret S. Pepe Oct 1998

Assessing The Accuracy Of A New Diagnostic Test When A Gold Standard Does Not Exist, Todd A. Alonzo, Margaret S. Pepe

UW Biostatistics Working Paper Series

Often the accuracy of a new diagnostic test must be assessed when a perfect gold standard does not exist. Use of an imperfect test biases the accuracy estimates of the new test. This paper reviews existing approaches to this problem including discrepant resolution and latent class analysis. Deficiencies with these approaches are identified. A new approach is proposed that combines the results of several imperfect reference tests to define a better reference standard. We call this the composite reference standard (CRS). Using the CRS, accuracy can be assessed using multistage sampling designs. Maximum likelihood estimates of accuracy and expressions for …