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Semiparametric efficient

Qi Liu, Chun Li, Valentine Wanga, Bryan E Shepherd
It is desirable to adjust Spearman's rank correlation for covariates, yet existing approaches have limitations. For example, the traditionally defined partial Spearman's correlation does not have a sensible population parameter, and the conditional Spearman's correlation defined with copulas cannot be easily generalized to discrete variables. We define population parameters for both partial and conditional Spearman's correlation through concordance-discordance probabilities. The definitions are natural extensions of Spearman's rank correlation in the presence of covariates and are general for any orderable random variables...
November 13, 2017: Biometrics
Mireille E Schnitzer, Matthew Cefalu
Causal inference practitioners are routinely presented with the challenge of model selection and, in particular, reducing the size of the covariate set with the goal of improving estimation efficiency. Collaborative targeted minimum loss-based estimation (CTMLE) is a general framework for constructing doubly robust semiparametric causal estimators that data-adaptively limit model complexity in the propensity score to optimize a preferred loss function. This stepwise complexity reduction is based on a loss function placed on a strategically updated model for the outcome variable through which the error is assessed using cross-validation...
November 2, 2017: Statistics in Medicine
Yan-Yong Zhao, Jin-Guan Lin, Xu-Guo Ye, Hong-Xia Wang, Xing-Fang Huang
Semiparametric smoothing methods are usually used to model longitudinal data, and the interest is to improve efficiency for regression coefficients. This paper is concerned with the estimation in semiparametric varying-coefficient models (SVCMs) for longitudinal data. By the orthogonal projection method, local linear technique, quasi-score estimation, and quasi-maximum likelihood estimation, we propose a two-stage orthogonality-based method to estimate parameter vector, coefficient function vector, and covariance function...
October 26, 2017: Biometrical Journal. Biometrische Zeitschrift
Jianxuan Liu, Yanyuan Ma, Liping Zhu, Raymond J Carroll
We introduce a general single index semiparametric measurement error model for the case that the main covariate of interest is measured with error and modeled parametrically, and where there are many other variables also important to the modeling. We propose a semiparametric bias-correction approach to estimate the effect of the covariate of interest. The resultant estimators are shown to be root-n consistent, asymptotically normal and locally efficient. Comprehensive simulations and an analysis of an empirical data set are performed to demonstrate the finite sample performance and the bias reduction of the locally efficient estimators...
2017: Electronic Journal of Statistics
Baosheng Liang, Xingwei Tong, Donglin Zeng, Yuanjia Wang
In many clinical studies, patients may be asked to report their medication adherence, presence of side effects, substance use, and hospitalization information during the study period. However, the exact occurrence time of these recurrent events may not be available due to privacy protection, recall difficulty, or incomplete medical records. Instead, the only available information is whether the events of interest have occurred during the past period. In this paper, we call these incomplete recurrent events as repeated current status data...
July 2017: Statistica Sinica
Gongjun Xu, Sy Han Chiou, Chiung-Yu Huang, Mei-Cheng Wang, Jun Yan
Recurrent event data arise frequently in various fields such as biomedical sciences, public health, engineering, and social sciences. In many instances, the observation of the recurrent event process can be stopped by the occurrence of a correlated failure event, such as treatment failure and death. In this article, we propose a joint scale-change model for the recurrent event process and the failure time, where a shared frailty variable is used to model the association between the two types of outcomes. In contrast to the popular Cox-type joint modeling approaches, the regression parameters in the proposed joint scale-change model have marginal interpretations...
2017: Journal of the American Statistical Association
Hanze Zhang, Yangxin Huang, Wei Wang, Henian Chen, Barbara Langland-Orban
In longitudinal AIDS studies, it is of interest to investigate the relationship between HIV viral load and CD4 cell counts, as well as the complicated time effect. Most of common models to analyze such complex longitudinal data are based on mean-regression, which fails to provide efficient estimates due to outliers and/or heavy tails. Quantile regression-based partially linear mixed-effects models, a special case of semiparametric models enjoying benefits of both parametric and nonparametric models, have the flexibility to monitor the viral dynamics nonparametrically and detect the varying CD4 effects parametrically at different quantiles of viral load...
January 1, 2017: Statistical Methods in Medical Research
Qi Liu, Bryan E Shepherd, Chun Li, Frank E Harrell
We study the application of a widely used ordinal regression model, the cumulative probability model (CPM), for continuous outcomes. Such models are attractive for the analysis of continuous response variables because they are invariant to any monotonic transformation of the outcome and because they directly model the cumulative distribution function from which summaries such as expectations and quantiles can easily be derived. Such models can also readily handle mixed type distributions. We describe the motivation, estimation, inference, model assumptions, and diagnostics...
November 30, 2017: Statistics in Medicine
Fan Wu, Sehee Kim, Jing Qin, Rajiv Saran, Yi Li
Survival data collected from a prevalent cohort are subject to left truncation and the analysis is challenging. Conditional approaches for left-truncated data could be inefficient as they ignore the information in the marginal likelihood of the truncation times. Length-biased sampling methods may improve the estimation efficiency but only when the underlying truncation time is uniform; otherwise, they may generate biased estimates. We propose a semiparametric method for left-truncated data under the Cox model with no parametric distributional assumption about the truncation times...
August 29, 2017: Biometrics
Qingning Zhou, Jianwen Cai, Haibo Zhou
Epidemiologic studies and disease prevention trials often seek to relate an exposure variable to a failure time that suffers from interval-censoring. When the failure rate is low and the time intervals are wide, a large cohort is often required so as to yield reliable precision on the exposure-failure-time relationship. However, large cohort studies with simple random sampling could be prohibitive for investigators with a limited budget, especially when the exposure variables are expensive to obtain. Alternative cost-effective sampling designs and inference procedures are therefore desirable...
August 3, 2017: Biometrics
Lixuan Yin, Guoqing Diao, Aiyi Liu
Receiver operating characteristic curves and the area under the curves (AUC) are often used to compare the discriminatory ability of potentially correlated biomarkers. Many biomarkers are subject to limit of detection due to the instrumental limitation in measurements and may not be normally distributed. Standard parametric methods assuming normality can lead to biased results when the normality assumption is violated. We propose new estimation and inference procedures for the AUCs of biomarkers subject to limit of detection by using the semiparametric transformation model allowing for heteroscedasticity...
July 25, 2017: Statistics in Medicine
Steven B Kim, Nathan Sanders
For many dose-response studies, large samples are not available. Particularly, when the outcome of interest is binary rather than continuous, a large sample size is required to provide evidence for hormesis at low doses. In a small or moderate sample, we can gain statistical power by the use of a parametric model. It is an efficient approach when it is correctly specified, but it can be misleading otherwise. This research is motivated by the fact that data points at high experimental doses have too much contribution in the hypothesis testing when a parametric model is misspecified...
April 2017: Dose-response: a Publication of International Hormesis Society
Ying Huang
This article focuses on the evaluation of vaccine-induced immune responses as principal surrogate markers for predicting a given vaccine's effect on the clinical endpoint of interest. To address the problem of missing potential outcomes under the principal surrogate framework, we can utilize baseline predictors of the immune biomarker(s) or vaccinate uninfected placebo recipients at the end of the trial and measure their immune biomarkers. Examples of good baseline predictors are baseline immune responses when subjects enrolled in the trial have been previously exposed to the same antigen, as in our motivating application of the Zostavax Efficacy and Safety Trial (ZEST)...
June 26, 2017: Biometrics
Tamar Sofer, Marilyn C Cornelis, Peter Kraft, Eric J Tchetgen Tchetgen
Case-control studies are designed towards studying associations between risk factors and a single, primary outcome. Information about additional, secondary outcomes is also collected, but association studies targeting such secondary outcomes should account for the case-control sampling scheme, or otherwise results may be biased. Often, one uses inverse probability weighted (IPW) estimators to estimate population effects in such studies. IPW estimators are robust, as they only require correct specification of the mean regression model of the secondary outcome on covariates, and knowledge of the disease prevalence...
April 2017: Statistica Sinica
Giorgos Bakoyannis, Menggang Yu, Constantin T Yiannoutsos
Many biomedical and clinical studies with time-to-event outcomes involve competing risks data. These data are frequently subject to interval censoring. This means that the failure time is not precisely observed but is only known to lie between two observation times such as clinical visits in a cohort study. Not taking into account the interval censoring may result in biased estimation of the cause-specific cumulative incidence function, an important quantity in the competing risks framework, used for evaluating interventions in populations, for studying the prognosis of various diseases, and for prediction and implementation science purposes...
October 15, 2017: Statistics in Medicine
Da Xu, Hui Zhao, Jianguo Sun
Interval-censored failure time data and panel count data are two types of incomplete data that commonly occur in event history studies and many methods have been developed for their analysis separately (Sun in The statistical analysis of interval-censored failure time data. Springer, New York, 2006; Sun and Zhao in The statistical analysis of panel count data. Springer, New York, 2013). Sometimes one may be interested in or need to conduct their joint analysis such as in the clinical trials with composite endpoints, for which it does not seem to exist an established approach in the literature...
June 12, 2017: Lifetime Data Analysis
Guoqing Diao, Donglin Zeng, Kuolung Hu, Joseph G Ibrahim
In many biomedical studies, it is often of interest to model event count data over the study period. For some patients, we may not follow up them for the entire study period owing to informative dropout. The dropout time can potentially provide valuable insight on the rate of the events. We propose a joint semiparametric model for event count data and informative dropout time that allows for correlation through a Gamma frailty. We develop efficient likelihood-based estimation and inference procedures. The proposed nonparametric maximum likelihood estimators are shown to be consistent and asymptotically normal...
May 30, 2017: Statistics in Medicine
Laura Balzer, Jennifer Ahern, Sandro Galea, Mark van der Laan
Many of the secondary outcomes in observational studies and randomized trials are rare. Methods for estimating causal effects and associations with rare outcomes, however, are limited, and this represents a missed opportunity for investigation. In this article, we construct a new targeted minimum loss-based estimator (TMLE) for the effect or association of an exposure on a rare outcome. We focus on the causal risk difference and statistical models incorporating bounds on the conditional mean of the outcome, given the exposure and measured confounders...
December 2016: Epidemiologic Methods
Yifei Sun, Kwun Chuen Gary Chan, Jing Qin
Length-biased survival data subject to right-censoring are often collected from a prevalent cohort. However, informative right censoring induced by the sampling design creates challenges in methodological development. While certain conditioning arguments could circumvent the problem of informative censoring, related rank estimation methods are typically inefficient because the marginal likelihood of the backward recurrence time is not ancillary. Under a semiparametric accelerated failure time model, an overidentified set of log-rank estimating equations is constructed based on the left-truncated right-censored data and backward recurrence time...
May 15, 2017: Biometrics
Fei Gao, Donglin Zeng, Dan-Yu Lin
Partly interval-censored (PIC) data arise when some failure times are exactly observed while others are only known to lie within certain intervals. In this article, we consider efficient semiparametric estimation of the accelerated failure time (AFT) model with PIC data. We first generalize the Buckley-James estimator for right-censored data to PIC data. Then, we develop a one-step estimator by deriving and estimating the efficient score for the regression parameters. We show that under mild regularity conditions the generalized Buckley-James estimator is consistent and asymptotically normal and the one-step estimator is consistent and asymptotically normal with a covariance matrix that attains the semiparametric efficiency bound...
April 25, 2017: Biometrics
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