Modeling longitudinal data with nonparametric multiplicative random effects jointly with survival data.

Abstract:

:In clinical studies, longitudinal biomarkers are often used to monitor disease progression and failure time. Joint modeling of longitudinal and survival data has certain advantages and has emerged as an effective way to mutually enhance information. Typically, a parametric longitudinal model is assumed to facilitate the likelihood approach. However, the choice of a proper parametric model turns out to be more elusive than models for standard longitudinal studies in which no survival endpoint occurs. In this article, we propose a nonparametric multiplicative random effects model for the longitudinal process, which has many applications and leads to a flexible yet parsimonious nonparametric random effects model. A proportional hazards model is then used to link the biomarkers and event time. We use B-splines to represent the nonparametric longitudinal process, and select the number of knots and degrees based on a version of the Akaike information criterion (AIC). Unknown model parameters are estimated through maximizing the observed joint likelihood, which is iteratively maximized by the Monte Carlo Expectation Maximization (MCEM) algorithm. Due to the simplicity of the model structure, the proposed approach has good numerical stability and compares well with the competing parametric longitudinal approaches. The new approach is illustrated with primary biliary cirrhosis (PBC) data, aiming to capture nonlinear patterns of serum bilirubin time courses and their relationship with survival time of PBC patients.

journal_name

Biometrics

journal_title

Biometrics

authors

Ding J,Wang JL

doi

10.1111/j.1541-0420.2007.00896.x

subject

Has Abstract

pub_date

2008-06-01 00:00:00

pages

546-56

issue

2

eissn

0006-341X

issn

1541-0420

pii

BIOM896

journal_volume

64

pub_type

杂志文章
  • Some aspects of cancer epidemiology.

    abstract::Epidemiolgic studies have strongly suggested that a vast majority (80-90%) of cancers are caused by radiation, chemical and biologic agents; the remainder result from endogenous or genetic factors. Biologically, cancer is most probably the end result of a complex multistage process and therefore may be due to a sequen...

    journal_title:Biometrics

    pub_type: 杂志文章,评审

    doi:

    authors: Lilienfeld AM

    更新日期:1982-03-01 00:00:00

  • Bayesian inference for the causal effect of mediation.

    abstract::We propose a nonparametric Bayesian approach to estimate the natural direct and indirect effects through a mediator in the setting of a continuous mediator and a binary response. Several conditional independence assumptions are introduced (with corresponding sensitivity parameters) to make these effects identifiable f...

    journal_title:Biometrics

    pub_type: 杂志文章,随机对照试验

    doi:10.1111/j.1541-0420.2012.01781.x

    authors: Daniels MJ,Roy JA,Kim C,Hogan JW,Perri MG

    更新日期:2012-12-01 00:00:00

  • Order-restricted tests for stratified comparisons of binomial proportions.

    abstract::The data set presented relates a binomial response to ordered levels of an explanatory variable, representing doses of a drug, with data collected at several centers. A study goal is to test independence of the response and the ordinal factor, assuming under the alternative only that the binomial parameter is a monoto...

    journal_title:Biometrics

    pub_type: 杂志文章

    doi:

    authors: Agresti A,Coull BA

    更新日期:1996-09-01 00:00:00

  • Semiparametric methods for mapping quantitative trait loci with censored data.

    abstract::Statistical methods for the detection of genes influencing quantitative traits with the aid of genetic markers are well developed for normally distributed, fully observed phenotypes. Many experiments are concerned with failure-time phenotypes, which have skewed distributions and which are usually subject to censoring ...

    journal_title:Biometrics

    pub_type: 杂志文章

    doi:10.1111/j.1541-0420.2005.00346.x

    authors: Diao G,Lin DY

    更新日期:2005-09-01 00:00:00

  • Sieve estimation of Cox models with latent structures.

    abstract::This article considers sieve estimation in the Cox model with an unknown regression structure based on right-censored data. We propose a semiparametric pursuit method to simultaneously identify and estimate linear and nonparametric covariate effects based on B-spline expansions through a penalized group selection meth...

    journal_title:Biometrics

    pub_type: 杂志文章

    doi:10.1111/biom.12529

    authors: Cao Y,Huang J,Liu Y,Zhao X

    更新日期:2016-12-01 00:00:00

  • Gene-trait similarity regression for multimarker-based association analysis.

    abstract::We propose a similarity-based regression method to detect associations between traits and multimarker genotypes. The model regresses similarity in traits for pairs of "unrelated" individuals on their haplotype similarities, and detects the significance by a score test for which the limiting distribution is derived. Th...

    journal_title:Biometrics

    pub_type: 杂志文章

    doi:10.1111/j.1541-0420.2008.01176.x

    authors: Tzeng JY,Zhang D,Chang SM,Thomas DC,Davidian M

    更新日期:2009-09-01 00:00:00

  • Testing nonlinear regression parameters under heteroscedastic, normally distributed errors.

    abstract::Likelihood ratio tests for parameters estimated assuming normally distributed errors are examined under a variety of homoscedastic and heteroscedastic variance assumptions. It is assumed that gamma ij, the jth observation from the ith population, is distributed as N[mu(chi ij, beta i), (sigma i mu(chi ij, beta i)theta...

    journal_title:Biometrics

    pub_type: 杂志文章

    doi:

    authors: Kimura DK

    更新日期:1990-09-01 00:00:00

  • Adaptive decision making in a lymphocyte infusion trial.

    abstract::We describe an adaptive Bayesian design for a clinical trial of an experimental treatment for patients with hematologic malignancies who initially received an allogeneic bone marrow transplant but subsequently suffered a disease recurrence. Treatment consists of up to two courses of targeted immunotherapy followed by ...

    journal_title:Biometrics

    pub_type: 杂志文章

    doi:10.1111/j.0006-341x.2002.00560.x

    authors: Thall PF,Inoue LY,Martin TG

    更新日期:2002-09-01 00:00:00

  • Fisher's contributions to genetics and heredity, with special emphasis on the Gregor Mendel controversy.

    abstract::R. A. Fisher is widely respected for his contributions to both statistics and genetics. For instance, his 1930 text on The Genetical Theory of Natural Selection remains a watershed contribution in that area. Fisher's subsequent research led him to study the work of (Johann) Gregor Mendel, the 19th century monk who fir...

    journal_title:Biometrics

    pub_type: 传,历史文章,杂志文章,评审

    doi:

    authors: Piegorsch WW

    更新日期:1990-12-01 00:00:00

  • Sequential model selection-based segmentation to detect DNA copy number variation.

    abstract::Array-based CGH experiments are designed to detect genomic aberrations or regions of DNA copy-number variation that are associated with an outcome, typically a state of disease. Most of the existing statistical methods target on detecting DNA copy number variations in a single sample or array. We focus on the detectio...

    journal_title:Biometrics

    pub_type: 杂志文章

    doi:10.1111/biom.12478

    authors: Hu J,Zhang L,Wang HJ

    更新日期:2016-09-01 00:00:00

  • Analysis of ordered categorical data: two score-independent approaches.

    abstract:SUMMARY:A trend test is often employed to analyze ordered categorical data, in which a set of increasing scores is assigned a priori. There is a drawback in this approach, because how to choose a set of scores is not clear. There have been debates on which scores should be used (e.g., Graubard and Korn, 1987, Biometric...

    journal_title:Biometrics

    pub_type: 杂志文章

    doi:10.1111/j.1541-0420.2008.00992.x

    authors: Zheng G

    更新日期:2008-12-01 00:00:00

  • Estimation of completeness and adjustment of age-specific and age-standardized incidence rates.

    abstract::This note discusses the use of capture-mark-recapture methods and log-linear, linear, product models for incomplete tables (Espeland, 1986, Communications in Statistics--Simulation and Computing 15, 405-424) to estimate completeness of reporting in a disease registry and to estimate incompleteness-adjusted incidence r...

    journal_title:Biometrics

    pub_type: 杂志文章

    doi:

    authors: Hilsenbeck SG,Kurucz C,Duncan RC

    更新日期:1992-12-01 00:00:00

  • A statistical method for detecting differentially expressed SNVs based on next-generation RNA-seq data.

    abstract::In this article, we propose a new statistical method-MutRSeq-for detecting differentially expressed single nucleotide variants (SNVs) based on RNA-seq data. Specifically, we focus on nonsynonymous mutations and employ a hierarchical likelihood approach to jointly model observed mutation events as well as read count me...

    journal_title:Biometrics

    pub_type: 杂志文章

    doi:10.1111/biom.12548

    authors: Fu R,Wang P,Ma W,Taguchi A,Wong CH,Zhang Q,Gazdar A,Hanash SM,Zhou Q,Zhong H,Feng Z

    更新日期:2017-03-01 00:00:00

  • Semiparametric estimation of proportional mean residual life model in presence of censoring.

    abstract::A mean residual life function is the average remaining life of a surviving subject, as it varies with time. The proportional mean residual life model was proposed by Oakes and Dasu (1990, Biometrika77, 409-410) in regression analysis to study its association with related covariates in absence of censoring. In this art...

    journal_title:Biometrics

    pub_type: 杂志文章

    doi:10.1111/j.0006-341X.2005.030224.x

    authors: Chen YQ,Jewell NP,Lei X,Cheng SC

    更新日期:2005-03-01 00:00:00

  • The analysis of pair-matched case-control studies, a multivariate approach.

    abstract::In matched case-control studies one frequently must consider more than one variable in the analysis and in this paper a log-linear model is presented to meet this objective. A conditional argument yields a method for making inferences on the parameters measuring the association between the variables and disease. The r...

    journal_title:Biometrics

    pub_type: 杂志文章

    doi:

    authors: Holford TR

    更新日期:1978-12-01 00:00:00

  • A note on permutation tests for variance components in multilevel generalized linear mixed models.

    abstract::In many applications of generalized linear mixed models to multilevel data, it is of interest to test whether a random effects variance component is zero. It is well known that the usual asymptotic chi-square distribution of the likelihood ratio and score statistics under the null does not necessarily hold. In this no...

    journal_title:Biometrics

    pub_type: 杂志文章

    doi:10.1111/j.1541-0420.2007.00775.x

    authors: Fitzmaurice GM,Lipsitz SR,Ibrahim JG

    更新日期:2007-09-01 00:00:00

  • Objective Bayesian search of Gaussian directed acyclic graphical models for ordered variables with non-local priors.

    abstract::Directed acyclic graphical (DAG) models are increasingly employed in the study of physical and biological systems to model direct influences between variables. Identifying the graph from data is a challenging endeavor, which can be more reasonably tackled if the variables are assumed to satisfy a given ordering; in th...

    journal_title:Biometrics

    pub_type: 杂志文章

    doi:10.1111/biom.12018

    authors: Altomare D,Consonni G,La Rocca L

    更新日期:2013-06-01 00:00:00

  • Bayesian latent multi-state modeling for nonequidistant longitudinal electronic health records.

    abstract::Large amounts of longitudinal health records are now available for dynamic monitoring of the underlying processes governing the observations. However, the health status progression across time is not typically observed directly: records are observed only when a subject interacts with the system, yielding irregular and...

    journal_title:Biometrics

    pub_type: 杂志文章

    doi:10.1111/biom.13261

    authors: Luo Y,Stephens DA,Verma A,Buckeridge DL

    更新日期:2020-03-11 00:00:00

  • A disequilibrium coefficient approach to Hardy-Weinberg testing.

    abstract::A comparison was made of various tests for Hardy-Weinberg equilibrium, with emphasis on methods for multiple alleles. For an overall test of deviations from equilibrium, the classical chi-square goodness-of-fit test generally performs well, with continuity corrections needed for extreme gene frequencies or extreme dep...

    journal_title:Biometrics

    pub_type: 杂志文章

    doi:

    authors: Hernández JL,Weir BS

    更新日期:1989-03-01 00:00:00

  • A general noninteractive multiple toxicity model including probit, logit, and Weibull transformations.

    abstract::A multiple toxicity model for the quantal response of organisms is constructed based on an existing bivariate theory. The main assumption is that the tolerances follow a multivariate normal distribution function. However, any monotone tolerance distribution can be applied by mapping the integration region in the n-dim...

    journal_title:Biometrics

    pub_type: 杂志文章

    doi:

    authors: Christensen ER,Chen CY

    更新日期:1985-09-01 00:00:00

  • Pleiotropy informed adaptive association test of multiple traits using genome-wide association study summary data.

    abstract::Genetic variants associated with disease outcomes can be used to develop personalized treatment. To reach this precision medicine goal, hundreds of large-scale genome-wide association studies (GWAS) have been conducted in the past decade to search for promising genetic variants associated with various traits. They hav...

    journal_title:Biometrics

    pub_type: 杂志文章

    doi:10.1111/biom.13076

    authors: Masotti M,Guo B,Wu B

    更新日期:2019-12-01 00:00:00

  • Statistical inference for serial dilution assay data.

    abstract::Serial dilution assays are widely employed for estimating substance concentrations and minimum inhibitory concentrations. The Poisson-Bernoulli model for such assays is appropriate for count data but not for continuous measurements that are encountered in applications involving substance concentrations. This paper pre...

    journal_title:Biometrics

    pub_type: 杂志文章

    doi:10.1111/j.0006-341x.1999.01215.x

    authors: Lee ML,Whitmore GA

    更新日期:1999-12-01 00:00:00

  • Median regression models for longitudinal data with dropouts.

    abstract:SUMMARY:Recently, median regression models have received increasing attention. When continuous responses follow a distribution that is quite different from a normal distribution, usual mean regression models may fail to produce efficient estimators whereas median regression models may perform satisfactorily. In this ar...

    journal_title:Biometrics

    pub_type: 杂志文章

    doi:10.1111/j.1541-0420.2008.01105.x

    authors: Yi GY,He W

    更新日期:2009-06-01 00:00:00

  • Numerical discretization-based estimation methods for ordinary differential equation models via penalized spline smoothing with applications in biomedical research.

    abstract::Differential equations are extensively used for modeling dynamics of physical processes in many scientific fields such as engineering, physics, and biomedical sciences. Parameter estimation of differential equation models is a challenging problem because of high computational cost and high-dimensional parameter space....

    journal_title:Biometrics

    pub_type: 杂志文章

    doi:10.1111/j.1541-0420.2012.01752.x

    authors: Wu H,Xue H,Kumar A

    更新日期:2012-06-01 00:00:00

  • Bayesian modeling of multiple lesion onset and growth from interval-censored data.

    abstract::In studying rates of occurrence and progression of lesions (or tumors), it is typically not possible to obtain exact onset times for each lesion. Instead, data consist of the number of lesions that reach a detectable size between screening examinations, along with measures of the size/severity of individual lesions at...

    journal_title:Biometrics

    pub_type: 杂志文章

    doi:10.1111/j.0006-341X.2004.00217.x

    authors: Dunson DB,Holloman C,Calder C,Gunn LH

    更新日期:2004-09-01 00:00:00

  • Statistical methods in ophthalmology: an adjusted chi-square approach.

    abstract::Ophthalmologic studies often compare several groups of subjects for the presence or absence of some ocular finding, where each subject may contribute two eyes to the analysis, the values from the two eyes being highly correlated. Rosner (1982, Biometrics 38, 105-114) and Dallal (1988, Biometrics 44, 253-257) proposed ...

    journal_title:Biometrics

    pub_type: 杂志文章

    doi:

    authors: Donner A

    更新日期:1989-06-01 00:00:00

  • Optimally weighted L(2) distance for functional data.

    abstract::Many techniques of functional data analysis require choosing a measure of distance between functions, with the most common choice being L2 distance. In this article we show that using a weighted L2 distance, with a judiciously chosen weight function, can improve the performance of various statistical methods for funct...

    journal_title:Biometrics

    pub_type: 杂志文章

    doi:10.1111/biom.12161

    authors: Chen H,Reiss PT,Tarpey T

    更新日期:2014-09-01 00:00:00

  • An adaptive trial design to optimize dose-schedule regimes with delayed outcomes.

    abstract::This paper proposes a two-stage phase I-II clinical trial design to optimize dose-schedule regimes of an experimental agent within ordered disease subgroups in terms of the toxicity-efficacy trade-off. The design is motivated by settings where prior biological information indicates it is certain that efficacy will imp...

    journal_title:Biometrics

    pub_type: 杂志文章

    doi:10.1111/biom.13116

    authors: Lin R,Thall PF,Yuan Y

    更新日期:2020-03-01 00:00:00

  • Dose-finding designs for HIV studies.

    abstract::We present a class of simple designs that can be used in early dose-finding studies in HIV. Such designs, in contrast with Phase I designs in cancer, have a lot of the Phase II flavor about them. Information on efficacy is obtained during the trial and is as important as that relating to toxicity. The designs proposed...

    journal_title:Biometrics

    pub_type: 杂志文章

    doi:10.1111/j.0006-341x.2001.01018.x

    authors: O'Quigley J,Hughes MD,Fenton T

    更新日期:2001-12-01 00:00:00

  • Sample size determination for testing whether an identified treatment is best.

    abstract::Laska and Meisner (1989, Biometrics 45, 1139-1151) dealt with the problem of testing whether an identified treatment belonging to a set of k + 1 treatments is better than each of the other k treatments. They calculated sample size tables for k = 2 when using multiple t-tests or Wilcoxon-Mann-Whitney tests, both under ...

    journal_title:Biometrics

    pub_type: 杂志文章

    doi:10.1111/j.0006-341x.2000.00879.x

    authors: Horn M,Vollandt R,Dunnett CW

    更新日期:2000-09-01 00:00:00