Item response models for longitudinal quality of life data in clinical trials.

Abstract:

:Assessment of quality of life is becoming standard in clinical trials. A popular method for measuring quality of life is with instruments which utilize multiple-item subscales, in which each item is scored on a Likert scale. Most statistical methods for the analysis of quality of life data in clinical trials do not explicity consider the properties and psychometric features which were of interest in scale development. In this regard, the measurement and statistical summarization of quality of life data, along with the clinical interpretation, can be somewhat disjoint from the psychometric concerns of the development process. The aim of this paper is to address the complicated issues present in analysing multiple-item ordinal quality of life data in clinical trials while maintaining fidelity to the psychometrical foundations upon which quality of life instruments are built. Accomplishing this will require the development of item response models which recognize the longitudinal aspects of clinical trial designs as well as the potential problem of informatively missing data. A general item response modeling approach is presented for longitudinal multiple-item quality of life data measured on ordinal scales with model components for missing data mechanisms and latent trait regression on treatment indicators and other covariates.

journal_name

Stat Med

journal_title

Statistics in medicine

authors

Douglas JA

doi

10.1002/(sici)1097-0258(19991115)18:21<2917::aid-s

subject

Has Abstract

pub_date

1999-11-15 00:00:00

pages

2917-31

issue

21

eissn

0277-6715

issn

1097-0258

pii

10.1002/(SICI)1097-0258(19991115)18:21<2917::AID-S

journal_volume

18

pub_type

杂志文章
  • New confidence bounds for QT studies.

    abstract::The proposed guidelines for the assessment of the effect of new pharmaceutical agents on the QT interval (beginning of QRS complex to end of T wave on the electrocardiogram) are based on the maximum of a series over time of simple one-sided 95 per cent upper confidence bounds. This procedure is typically very conserva...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.2826

    authors: Boos DD,Hoffman D,Kringle R,Zhang J

    更新日期:2007-09-10 00:00:00

  • The power of focused tests to detect disease clustering.

    abstract::Statistical tests have been proposed for determining whether incident cases of adverse health effects are 'clustered' together. Several procedures, termed 'focused', specifically analyse disease surveillance data around pre-specified putative sources of environmental hazard. Little has been done to compare the perform...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.4780142103

    authors: Waller LA,Lawson AB

    更新日期:1995-11-15 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

  • Multilevel time series models with applications to repeated measures data.

    abstract::The analysis of repeated measures data can be conducted efficiently using a two-level random coefficients model. A standard assumption is that the within-individual (level 1) residuals are uncorrelated. In some cases, especially where measurements are made close together in time, this may not be reasonable and this ad...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.4780131605

    authors: Goldstein H,Healy MJ,Rasbash J

    更新日期:1994-08-30 00:00:00

  • Nonparametric comparison of two survival functions with dependent censoring via nonparametric multiple imputation.

    abstract::When the event time of interest depends on the censoring time, conventional two-sample test methods, such as the log-rank and Wilcoxon tests, can produce an invalid test result. We extend our previous work on estimation using auxiliary variables to adjust for dependent censoring via multiple imputation, to the compari...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.3480

    authors: Hsu CH,Taylor JM

    更新日期:2009-02-01 00:00:00

  • Causal conclusions are most sensitive to unobserved binary covariates.

    abstract::There is a rich literature that considers whether an observed relation between treatment and response is due to an unobserved covariate. In order to quantify this unmeasured bias, an assumption is made about the distribution of this unobserved covariate; typically that it is either binary or at least confined to the u...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.2344

    authors: Wang L,Krieger AM

    更新日期:2006-07-15 00:00:00

  • Statistical models for longitudinal biomarkers of disease onset.

    abstract::We consider the analysis of serial biomarkers to screen and monitor individuals in a given population for onset of a specific disease of interest. The biomarker readings are subject to error. We survey some of the existing literature and concentrate on two recently proposed models. The first is a fully Bayesian hierar...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/(sici)1097-0258(20000229)19:4<617::aid-sim

    authors: Slate EH,Turnbull BW

    更新日期:2000-02-29 00:00:00

  • A pathway analysis method for genome-wide association studies.

    abstract::For genome-wide association studies, we propose a new method for identifying significant biological pathways. In this approach, we aggregate data across single-nucleotide polymorphisms to obtain summary measures at the gene level. We then use a hierarchical Bayesian model, which takes the gene-level summary measures a...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.4477

    authors: Shahbaba B,Shachaf CM,Yu Z

    更新日期:2012-05-10 00:00:00

  • Quantifying the bias due to observed individual confounders in causal treatment effect estimates.

    abstract::It is often of interest to use observational data to estimate the causal effect of a target exposure or treatment on an outcome. When estimating the treatment effect, it is essential to appropriately adjust for selection bias due to observed confounders using, for example, propensity score weighting. Selection bias du...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.8549

    authors: Parast L,Griffin BA

    更新日期:2020-08-15 00:00:00

  • Modelling the geographical distribution of co-infection risk from single-disease surveys.

    abstract:BACKGROUND:The need to deliver interventions targeting multiple diseases in a cost-effective manner calls for integrated disease control efforts. Consequently, maps are required that show where the risk of co-infection is particularly high. Co-infection risk is preferably estimated via Bayesian geostatistical multinomi...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.4243

    authors: Schur N,Gosoniu L,Raso G,Utzinger J,Vounatsou P

    更新日期:2011-06-30 00:00:00

  • Robust fitting for neuroreceptor mapping.

    abstract::Among many other uses, positron emission tomography (PET) can be used in studies to estimate the density of a neuroreceptor at each location throughout the brain by measuring the concentration of a radiotracer over time and modeling its kinetics. There are a variety of kinetic models in common usage and these typicall...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.3510

    authors: Chang C,Ogden RT

    更新日期:2009-03-15 00:00:00

  • A statistical methodology for postmarketing surveillance of adverse drug reaction reports.

    abstract::This paper presents a statistically optimal exact hypothesis testing procedure for detecting changes in sales adjusted adverse drug reaction (ADR) rates between historical and current periods, with a computer program that implements this test appended. We provide discussions and illustrations on how to monitor ADR rat...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.4780071004

    authors: Norwood PK,Sampson AR

    更新日期:1988-10-01 00:00:00

  • Easy and accurate variance estimation of the nonparametric estimator of the partial area under the ROC curve and its application.

    abstract::The receiver operating characteristic (ROC) curve is a popular technique with applications, for example, investigating an accuracy of a biomarker to delineate between disease and non-disease groups. A common measure of accuracy of a given diagnostic marker is the area under the ROC curve (AUC). In contrast with the AU...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.6863

    authors: Yu J,Yang L,Vexler A,Hutson AD

    更新日期:2016-06-15 00:00:00

  • A spatial scan statistic for ordinal data.

    abstract::Spatial scan statistics are widely used for count data to detect geographical disease clusters of high or low incidence, mortality or prevalence and to evaluate their statistical significance. Some data are ordinal or continuous in nature, however, so that it is necessary to dichotomize the data to use a traditional s...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.2607

    authors: Jung I,Kulldorff M,Klassen AC

    更新日期:2007-03-30 00:00:00

  • The power prior: theory and applications.

    abstract::The power prior has been widely used in many applications covering a large number of disciplines. The power prior is intended to be an informative prior constructed from historical data. It has been used in clinical trials, genetics, health care, psychology, environmental health, engineering, economics, and business. ...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.6728

    authors: Ibrahim JG,Chen MH,Gwon Y,Chen F

    更新日期:2015-12-10 00:00:00

  • Assessing the incremental predictive performance of novel biomarkers over standard predictors.

    abstract::It is unclear to what extent the incremental predictive performance of a novel biomarker is impacted by the method used to control for standard predictors. We investigated whether adding a biomarker to a model with a published risk score overestimates its incremental performance as compared to adding it to a multivari...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.6165

    authors: Xanthakis V,Sullivan LM,Vasan RS,Benjamin EJ,Massaro JM,D'Agostino RB Sr,Pencina MJ

    更新日期:2014-07-10 00:00:00

  • On prediction of future observation in growth curve model.

    abstract::Rao proposed and compared several approaches for predicting future observations in a growth curve model. The assessment of associated prediction efficiency for different prediction methods were evaluated by Cross-Validation Assessment Error (CVAE). He used three data sets, each with a limited number of subjects (13-27...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.4780132103

    authors: Tian JJ,Shukla R,Buncher CR

    更新日期:1994-11-15 00:00:00

  • A robust goodness-of-fit test statistic with application to ordinal regression models.

    abstract::We propose a goodness-of-fit test statistic for linear regression with heterogeneous variance, which is asymptotically chi-square if the given model is correct. The test statistic is computed as a quadratic form of observed minus predicted responses. We apply the method to a linear regression for an ordinal categorica...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.4780130205

    authors: Lipsitz SR,Buoncristiani JF

    更新日期:1994-01-30 00:00:00

  • Methodological considerations on the design and analysis of an equivalence stratified cluster randomization trial.

    abstract::The World Health Organization and collaborating institutions in four developing countries have conducted a multi-centre randomized controlled trial, in which clinics were allocated at random to two antenatal care (ANC) models. These were the standard 'Western' ANC model and a 'new' ANC model consisting of tests, clini...

    journal_title:Statistics in medicine

    pub_type: 临床试验,杂志文章,随机对照试验

    doi:10.1002/1097-0258(20010215)20:3<401::aid-sim801>3.

    authors: Piaggio G,Carroli G,Villar J,Pinol A,Bakketeig L,Lumbiganon P,Bergsjø P,Al-Mazrou Y,Ba'aqeel H,Belizán JM,Farnot U,Berendes H,WHO Antenatal Care Trial Research Group.

    更新日期:2001-02-15 00:00:00

  • Parameters of mortality in human populations with widely varying life spans.

    abstract::A three-component, competing-risk mortality model, developed for animal survival data, fits human life table data for all ages over a range of mean life spans from 16 to 74 years. The competing risks are a novel exponentially-decreasing hazard, dominant during immaturity; a constant hazard, dominant during adulthood; ...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.4780020309

    authors: Siler W

    更新日期:1983-07-01 00:00:00

  • Exact unconditional tables for significance testing in the 2 x 2 multinomial trial.

    abstract::This paper presents tables analogous to T-tables for use in the 2 x 2 multinomial trial, where the continuity corrected Z-statistic is used to make exact unconditional inference. This is the first solution of a discrete exact unconditional inference problem involving a multivariate nuisance parameter for which no anci...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.4780110708

    authors: Shuster JJ

    更新日期:1992-05-01 00:00:00

  • Pros and cons of permutation tests in clinical trials.

    abstract::Hypothesis testing, in which the null hypothesis specifies no difference between treatment groups, is an important tool in the assessment of new medical interventions. For randomized clinical trials, permutation tests that reflect the actual randomization are design-based analyses for such hypotheses. This means that ...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/(sici)1097-0258(20000530)19:10<1319::aid-s

    authors: Berger VW

    更新日期:2000-05-30 00:00:00

  • A Bayesian methodology for detecting targeted genes under two related experiments.

    abstract::Many gene expression data are based on two experiments where the gene expressions of the targeted genes under both experiments are correlated. We consider problems in which objectives are to find genes that are simultaneously upregulated/downregulated under both experiments. A Bayesian methodology is proposed based on...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.6555

    authors: Bansal NK,Jiang H,Pradeep P

    更新日期:2015-11-10 00:00:00

  • The analysis of contingency tables with ordinal data: an application to monitoring antibiotic resistance.

    abstract::Rationalization of antibiotic therapy in the management of infectious diseases is helped by a knowledge of the patterns of sensitivity and resistance of bacteria to antibiotics and their possible changes both in time and from one hospital unit to another. In this paper we present the results regarding the sensitivitie...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.2447

    authors: Bonetto C,Giannerini S,Giovagnoli A

    更新日期:2006-10-30 00:00:00

  • Exact logistic models for nested binary data.

    abstract::The use of logistic models for independent binary data has relied first on asymptotic theory and later on exact distributions for small samples. However, the use of logistic models for dependent analysis based on exact analysis is not as common. Moreover, attention is usually given to one-stage clustering. In this pap...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.4157

    authors: Troxler S,Lalonde T,Wilson JR

    更新日期:2011-04-15 00:00:00

  • Targeted maximum likelihood estimation for a binary treatment: A tutorial.

    abstract::When estimating the average effect of a binary treatment (or exposure) on an outcome, methods that incorporate propensity scores, the G-formula, or targeted maximum likelihood estimation (TMLE) are preferred over naïve regression approaches, which are biased under misspecification of a parametric outcome model. In con...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.7628

    authors: Luque-Fernandez MA,Schomaker M,Rachet B,Schnitzer ME

    更新日期:2018-07-20 00:00:00

  • A functional-model-adjusted spatial scan statistic.

    abstract::This paper introduces a new spatial scan statistic designed to adjust cluster detection for longitudinal confounding factors indexed in space. The functional-model-adjusted statistic was developed using generalized functional linear models in which longitudinal confounding factors were considered to be functional cova...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.8459

    authors: Ahmed MS,Genin M

    更新日期:2020-04-15 00:00:00

  • The use of an autoregressive model for the analysis of longitudinal data in epidemiologic studies.

    abstract::Korn and Whittemore have presented methods for analyzing longitudinal data where the number of observations per individual is large relative to the number of variables considered for each subject. However, this is often not the case in epidemiologic studies, since one usually collects data at relatively few time point...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.4780040407

    authors: Rosner B,Muñoz A,Tager I,Speizer F,Weiss S

    更新日期:1985-10-01 00:00:00

  • Application of the parallel line assay to assessment of biosimilar products based on binary endpoints.

    abstract::Biological drug products are therapeutic moieties manufactured by a living system or organisms. These are important life-saving drug products for patients with unmet medical needs. Because of expensive cost, only a few patients have access to life-saving biological products. Most of the early biological products will ...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.5565

    authors: Lin JR,Chow SC,Chang CH,Lin YC,Liu JP

    更新日期:2013-02-10 00:00:00

  • Proportional hazards models with frailties and random effects.

    abstract::We discuss some of the fundamental concepts underlying the development of frailty and random effects models in survival. One of these fundamental concepts was the idea of a frailty model where each subject has his or her own disposition to failure, their so-called frailty, additional to any effects we wish to quantify...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.1259

    authors: O'Quigley J,Stare J

    更新日期:2002-11-15 00:00:00