Estimation methods for marginal and association parameters for longitudinal binary data with nonignorable missing observations.

Abstract:

:In longitudinal studies, missing observations occur commonly. It has been well known that biased results could be produced if missingness is not properly handled in the analysis. Authors have developed many methods with the focus on either incomplete response or missing covariate observations, but rarely on both. The complexity of modeling and computational difficulty would be the major challenges in handling missingness in both response and covariate variables. In this paper, we develop methods using the pairwise likelihood formulation to handle longitudinal binary data with missing observations present in both response and covariate variables. We propose a unified framework to accommodate various types of missing data patterns. We evaluate the performance of the methods empirically under a variety of circumstances. In particular, we investigate issues on efficiency and robustness. We analyze longitudinal data from the National Population Health Study with the use of our methods.

journal_name

Stat Med

journal_title

Statistics in medicine

authors

Li H,Yi GY

doi

10.1002/sim.5536

subject

Has Abstract

pub_date

2013-02-28 00:00:00

pages

833-48

issue

5

eissn

0277-6715

issn

1097-0258

journal_volume

32

pub_type

杂志文章
  • Exact test size and power of a Gaussian error linear model for an internal pilot study.

    abstract::Wittes and Brittain recommended using an 'internal pilot study' to adjust sample size. The approach involves five steps in testing a general linear hypothesis for a general linear univariate model, with Gaussian errors. First, specify the design, hypothesis, desired test size, power, a smallest 'clinically meaningful'...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/(sici)1097-0258(19990530)18:10<1199::aid-s

    authors: Coffey CS,Muller KE

    更新日期:1999-05-30 00:00:00

  • A sequential classification rule based on multiple quantitative tests in the absence of a gold standard.

    abstract::In many medical applications, combining information from multiple biomarkers could yield a better diagnosis than any single one on its own. When there is a lack of a gold standard, an algorithm of classifying subjects into the case and non-case status is necessary for combining multiple markers. The aim of this paper ...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.6780

    authors: Zhang J,Zhang Y,Chaloner K,Stapleton JT

    更新日期:2016-04-15 00:00:00

  • Multilevel modeling versus cross-sectional analysis for assessing the longitudinal tracking of cardiovascular risk factors over time.

    abstract::Correlated data are obtained in longitudinal epidemiological studies, where repeated measurements are taken on individuals or groups over time. Such longitudinal data are ideally analyzed using multilevel modeling approaches, which appropriately account for the correlations in repeated responses in the same individual...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.5880

    authors: Xanthakis V,Sullivan LM,Vasan RS

    更新日期:2013-12-10 00:00:00

  • Group sequential large sample T2-like chi2 tests for multivariate observations.

    abstract::In many studies, a K degree of freedom large sample chi2 test is used to assess the effect of treatment on a multivariate response, such as an omnibus T2-like test of a difference between two treatment groups in any of K repeated measures. Alternately, a K df chi2 test may be used to test the equality of K+1 groups in...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.1637

    authors: Lachin JM,Greenhouse SW,Bautista OM

    更新日期:2003-11-15 00:00:00

  • On the association between variables with lower detection limits.

    abstract::In this paper, we define a modified version τ(b) of Kendall's tau to measure the association in a pair (X,Y) of random variables subject to fixed left censoring due to known lower detection limits. We provide a nonparametric estimator of τ(b) and investigate its asymptotic properties. We then assume an Archimedean cop...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.4319

    authors: Romdhani H,Lakhal-Chaieb L

    更新日期:2011-11-20 00:00:00

  • Dynamic thresholds and a summary ROC curve: Assessing prognostic accuracy of longitudinal markers.

    abstract::Cancer patients, chronic kidney disease patients, and subjects infected with HIV are routinely monitored over time using biomarkers that represent key health status indicators. Furthermore, biomarkers are frequently used to guide initiation of new treatments or to inform changes in intervention strategies. Since key m...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.7675

    authors: Saha-Chaudhuri P,Heagerty PJ

    更新日期:2018-08-15 00:00:00

  • Heterogeneity in the probability of HIV transmission per sexual contact: the case of male-to-female transmission in penile-vaginal intercourse.

    abstract::Recent studies have indicated variation in the infectivity beta of HIV among heterosexual couples. We represent this heterogeneity by modelling beta as a random variable. Using data on the number of contacts and seroconversion of couples, we fit the model by maximum-likelihood estimation with a beta distribution and a...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.4780080110

    authors: Wiley JA,Herschkorn SJ,Padian NS

    更新日期:1989-01-01 00:00:00

  • Estimating net transition probabilities from cross-sectional data with application to risk factors in chronic disease modeling.

    abstract::A problem occurring in chronic disease modeling is the estimation of transition probabilities of moving from one state of a categorical risk factor to another. Transitions could be obtained from a cohort study, but often such data may not be available. However, under the assumption that transitions remain stable over ...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.4423

    authors: Kassteele Jv,Hoogenveen RT,Engelfriet PM,Baal PH,Boshuizen HC

    更新日期:2012-03-15 00:00:00

  • Monitoring clinical trials: issues and controversies regarding confidentiality.

    abstract::During phase III clinical trials in life-threatening disease settings, it is important to ensure that the Data Monitoring Committee (DMC) has exclusive access to the interim efficacy and safety data generated by the data analysis centre, in order to minimize the risk of widespread prejudgement of unreliable trial resu...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.1288

    authors: Fleming TR,Ellenberg S,DeMets DL

    更新日期:2002-10-15 00:00:00

  • Bayesian clinical trials in action.

    abstract::Although the frequentist paradigm has been the predominant approach to clinical trial design since the 1940s, it has several notable limitations. Advancements in computational algorithms and computer hardware have greatly enhanced the alternative Bayesian paradigm. Compared with its frequentist counterpart, the Bayesi...

    journal_title:Statistics in medicine

    pub_type: 杂志文章,评审

    doi:10.1002/sim.5404

    authors: Lee JJ,Chu CT

    更新日期:2012-11-10 00:00:00

  • A simple correction for ties when censoring times depend on covariates.

    abstract::We point out that the conventional methods for ties correction may be seriously biased when censoring times depend on covariates. A simple modification to the Efron correction method is suggested which works remarkably well in simulation studies. The method corresponds closely to breaking ties by random ordering. The ...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.2173

    authors: Samuelsen SO,Wisløff TF,Skrondal A

    更新日期:2005-10-30 00:00:00

  • Statistical issues in the assessment of the evidence for an interaction between factors in epilepsy trials.

    abstract::We examine the common clinical belief that there is an interaction between epilepsy type and the two standard anti-epileptic drugs, valproate and carbamazepine, using data from several randomized clinical trials. Epilepsy type is not always easy to define, and three possible reclassifications are investigated to see w...

    journal_title:Statistics in medicine

    pub_type: 杂志文章,meta分析

    doi:10.1002/sim.1044

    authors: Williamson PR,Clough HE,Hutton JL,Marson AG,Chadwick DW

    更新日期:2002-09-30 00:00:00

  • Composite sequential Monte Carlo test for post-market vaccine safety surveillance.

    abstract::Group sequential hypothesis testing is now widely used to analyze prospective data. If Monte Carlo simulation is used to construct the signaling threshold, the challenge is how to manage the type I error probability for each one of the multiple tests without losing control on the overall significance level. This paper...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.6805

    authors: Silva IR

    更新日期:2016-04-30 00:00:00

  • Variable selection with group structure in competing risks quantile regression.

    abstract::We study the group bridge and the adaptive group bridge penalties for competing risks quantile regression with group variables. While the group bridge consistently identifies nonzero group variables, the adaptive group bridge consistently selects variables not only at group level but also at within-group level. We all...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.7619

    authors: Ahn KW,Kim S

    更新日期:2018-04-30 00:00:00

  • Approximate multinormal probabilities applied to correlated multiple endpoints in clinical trials.

    abstract::Clinical trials with multiple endpoints incur increased familywise type I errors. The Bonferroni correction is a common method used to modify the p-values to account for multiple significance testing. For independent endpoints the Bonferroni method is slightly conservative whereas with high correlation the conservatis...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.4780100712

    authors: James S

    更新日期:1991-07-01 00:00:00

  • Bias in the one-step method for pooling study results.

    abstract::The one-step (Peto) method for obtaining pooled effect estimates can yield extremely biased results when applied to unbalanced data. Even for balanced studies, the one-step estimate may incorporate an unacceptable degree of bias. In place of the one-step estimate, we recommend use of ordinary Mantel-Haenszel, weighted...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.4780090307

    authors: Greenland S,Salvan A

    更新日期:1990-03-01 00:00:00

  • The use of baselines in clinical trials of bronchodilators.

    abstract::Two important qualities of controlled clinical trials are that they reduce dependence on historical standards for evaluating therapy and separate the effect of treatment from the confounding influence of time. Whatever the theory of the clinical trial, however, time has not easily been banished from the analysis of me...

    journal_title:Statistics in medicine

    pub_type: 杂志文章,评审

    doi:10.1002/sim.4780081106

    authors: Senn SJ

    更新日期:1989-11-01 00:00:00

  • Prospective epidemiological studies involving paired organs.

    abstract::Standard methods for analysing survival data or case-control data normally concern factors affecting a subject as a whole. However, in a study of a condition that might develop in one or both of a pair of bodily organs information on response and on covariates may be available for each separately. This information can...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.4780070509

    authors: Whitehead J,Dorse C

    更新日期:1988-05-01 00:00:00

  • An improved algorithm for outbreak detection in multiple surveillance systems.

    abstract::In England and Wales, a large-scale multiple statistical surveillance system for infectious disease outbreaks has been in operation for nearly two decades. This system uses a robust quasi-Poisson regression algorithm to identify abberrances in weekly counts of isolates reported to the Health Protection Agency. In this...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.5595

    authors: Noufaily A,Enki DG,Farrington P,Garthwaite P,Andrews N,Charlett A

    更新日期:2013-03-30 00:00:00

  • Redesign of trials under different enrollment mixes.

    abstract::A few large multi-centre male-only heart trials done in the 1970s and 1980s have been seen as ill-conceived because they did not include females. The purpose here is to revisit two of those trials and to consider consequences in terms of cost and power had they been designed to include females. ...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/(sici)1097-0258(19990215)18:3<241::aid-sim

    authors: Meinert CL

    更新日期:1999-02-15 00:00:00

  • Incorporating data from various trial designs into a mixed treatment comparison model.

    abstract::Estimates of relative efficacy between alternative treatments are crucial for decision making in health care. Bayesian mixed treatment comparison models provide a powerful methodology to obtain such estimates when head-to-head evidence is not available or insufficient. In recent years, this methodology has become wide...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.5764

    authors: Schmitz S,Adams R,Walsh C

    更新日期:2013-07-30 00:00:00

  • Coping with time and space in modelling malaria incidence: a comparison of survival and count regression models.

    abstract::To study the effect of a mega hydropower dam in southwest Ethiopia on malaria incidence, we have set up a longitudinal study. To gain insight in temporal and spatial aspects, that is, in time (period  =  year-season combination) and location (village), we need models that account for these effects. The frailty model w...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.5752

    authors: Getachew Y,Janssen P,Yewhalaw D,Speybroeck N,Duchateau L

    更新日期:2013-08-15 00:00:00

  • The Integrated Calibration Index (ICI) and related metrics for quantifying the calibration of logistic regression models.

    abstract::Assessing the calibration of methods for estimating the probability of the occurrence of a binary outcome is an important aspect of validating the performance of risk-prediction algorithms. Calibration commonly refers to the agreement between predicted and observed probabilities of the outcome. Graphical methods are a...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.8281

    authors: Austin PC,Steyerberg EW

    更新日期:2019-09-20 00:00:00

  • Biostatistical concepts and methods in the legal setting.

    abstract::Biostatistical concepts and methods apply to various problems arising in actual U.S. legal cases. These involve: measures of association, assessing the potential effect of omitted variables and the Peters-Belson approach to regression. In particular, we present the inapplicability of Fisher's exact test in the case wh...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.4780141505

    authors: Gastwirth JL,Greenhouse SW

    更新日期:1995-08-15 00:00:00

  • First steps in analysing NHS waiting times: avoiding the 'stationary and closed population' fallacy.

    abstract::The aim of this paper is to demonstrate the effect of excluding incomplete observations and competing events when calculating cross-sectional measures of NHS waiting times, and to obtain a more accurate estimate of the 'time-to-admission' of those listed on NHS waiting lists using life-table methods. The official 'tim...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/1097-0258(20000815)19:15<2037::aid-sim606>

    authors: Armstrong PW

    更新日期:2000-08-15 00:00:00

  • Variance estimation of a survival function for interval-censored survival data.

    abstract::Interval-censored survival data often occur in medical studies, especially in clinical trials. In this case, many authors have considered estimation of a survival function. There is, however, relatively little discussion on estimating the variance of estimated survival functions. For right-censored data, a special cas...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.719

    authors: Sun J

    更新日期:2001-04-30 00:00:00

  • A joint model for interval-censored functional decline trajectories under informative observation.

    abstract::Multi-state models are useful for modelling disease progression where the state space of the process is used to represent the discrete disease status of subjects. Often, the disease process is only observed at clinical visits, and the schedule of these visits can depend on the disease status of patients. In such situa...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.6582

    authors: Lesperance ML,Sabelnykova V,Nathoo FS,Lau F,Downing MG

    更新日期:2015-12-20 00:00:00

  • Bayesian random effects meta-analysis of trials with binary outcomes: methods for the absolute risk difference and relative risk scales.

    abstract::In a recent Statistics in Medicine paper, Warn, Thompson and Spiegelhalter (WTS) made a comparison between the Bayesian approach to the meta-analysis of binary outcomes and a popular Classical approach that uses summary (two-stage) techniques. They included approximate summary (two-stage) Bayesian techniques in their ...

    journal_title:Statistics in medicine

    pub_type: 评论,信件

    doi:10.1002/sim.2115

    authors: O'Rourke K,Altman DG

    更新日期:2005-09-15 00:00:00

  • A semi-parametric regression analysis of CD4 cell counts.

    abstract::This paper considers the regression analysis of CD4 cell counts, a commonly used indicator and prognostic factor of AIDS progression. For this purpose, a number of methods have been proposed and most of them are based on random effects models. We present an alternative that is based on a mean function regression model...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.945

    authors: Sun J

    更新日期:2001-11-15 00:00:00

  • A maximally selected test of symmetry about zero.

    abstract::The problem of testing symmetry about zero has a long and rich history in the statistical literature. We introduce a new test that sequentially discards observations whose absolute value is below increasing thresholds defined by the data. McNemar's statistic is obtained at each threshold and the largest is used as the...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.5384

    authors: Laska E,Meisner M,Wanderling J

    更新日期:2012-11-20 00:00:00