Joint modeling longitudinal semi-continuous data and survival, with application to longitudinal medical cost data.

abstract：:A statistical definition of surrogate endpoints as well as validation criteria was first presented by Prentice. Freedman et al. supplemented these criteria with the so-called proportion explained. Buyse and Molenberghs pointed to inadequacies of these criteria and suggested a new definition of surrogacy based on (i) t...

journal_title：Statistics in medicine

authors： Molenberghs G,Geys H,Buyse M

更新日期：2001-10-30 00:00:00

abstract：:The focus of this paper is dietary intervention trials. We explore the statistical issues involved when the response variable, intake of a food or nutrient, is based on self-report data that are subject to inherent measurement error. There has been little work on handling error in this context. A particular feature of...

journal_title：Statistics in medicine

authors： Keogh RH,Carroll RJ,Tooze JA,Kirkpatrick SI,Freedman LS

更新日期：2016-11-10 00:00:00

abstract：:Many epidemiologic investigations are designed to study the effects of multiple exposures. Most of these studies are analysed either by fitting a risk-regression model with all exposures forced in the model, or by using a preliminary-testing algorithm, such as stepwise regression, to produce a smaller model. Research ...

journal_title：Statistics in medicine

authors： Greenland S

更新日期：1993-04-30 00:00:00

abstract：:Correlation is always a concern in the analysis of clustered data. One area of interest is to develop a general correlation modelling approach for high dimensional data with unbalanced hierarchical and heterogeneous data structures, e.g. multilevel data. Commonly used correlation structures might have limitation for s...

journal_title：Statistics in medicine

authors： Chao EC

更新日期：2006-07-30 00:00:00

abstract：:In a phase I clinical trial in cancer patients, the drug involved had one known main adverse effect, which also occurs spontaneously in cancer patients with a fairly high frequency. Experiments in rats have shown marked effects of the drug on tumour growth in high doses, but also dose-dependent toxicity. Consequently,...

journal_title：Statistics in medicine

authors： Møller S

更新日期：1995-05-15 00:00:00

abstract：:The generalized estimating equations (GEE) approach is commonly used to model incomplete longitudinal binary data. When drop-outs are missing at random through dependence on observed responses (MAR), GEE may give biased parameter estimates in the model for the marginal means. A weighted estimating equations approach g...

journal_title：Statistics in medicine

authors： Preisser JS,Lohman KK,Rathouz PJ

更新日期：2002-10-30 00:00:00

abstract：:In biomedical literature, numerous prediction models for clinical outcomes have been developed based either on clinical data or, more recently, on high-throughput molecular data (omics data). Prediction models based on both types of data, however, are less common, although some recent studies suggest that a suitable c...

journal_title：Statistics in medicine

authors： De Bin R,Sauerbrei W,Boulesteix AL

更新日期：2014-12-30 00:00:00

abstract：:A new, intuitive method has recently been proposed to explore treatment-covariate interactions in survival data arising from two treatment arms of a clinical trial. The method is based on constructing overlapping subpopulations of patients with respect to one (or more) covariates of interest and in observing the patte...

journal_title：Statistics in medicine

authors： Bonetti M,Zahrieh D,Cole BF,Gelber RD

更新日期：2009-04-15 00:00:00

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

authors： Siler W

更新日期：1983-07-01 00:00:00

abstract：:We describe the usual statistical concepts and consequent appropriate simulations of a prospective study for the simple case of a single risk variable and an assumed logistic model. We examine the simulations of Lilienfeld and Pyne, and show that they are seriously flawed. Contrary to those authors' claims, the estima...

journal_title：Statistics in medicine

authors： Halperin M,Abbott RD,Blackwelder WC,Jacobowitz R,Lan KK,Verter JI,Wedel H

更新日期：1985-04-01 00:00:00

abstract：:In observational cohort studies we may wish to examine the associations between fixed patient characteristics and the longitudinal changes from baseline in a repeated outcome measure. Many biological and other outcome measures are known to be subject to measurement error and biological variation. In an initial analysi...

journal_title：Statistics in medicine

authors： Harrison L,Dunn DT,Green H,Copas AJ

更新日期：2009-11-20 00:00:00

abstract：:Instrumental variable is an essential tool for addressing unmeasured confounding in observational studies. Two-stage predictor substitution (2SPS) estimator and two-stage residual inclusion (2SRI) are two commonly used approaches in applying instrumental variables. Recently, 2SPS was studied under the additive hazards...

journal_title：Statistics in medicine

authors： Ying A,Xu R,Murphy J

更新日期：2019-05-10 00:00:00

abstract：:Differences between arm-based (AB) and contrast-based (CB) models for network meta-analysis (NMA) are controversial. We compare the CB model of Lu and Ades (2006), the AB model of Hong et al(2016), and two intermediate models, using hypothetical data and a selected real data set. Differences between models arise prima...

journal_title：Statistics in medicine

authors： White IR,Turner RM,Karahalios A,Salanti G

更新日期：2019-11-30 00:00:00

abstract：:Sample size planning should reflect the primary objective of a trial. If the primary objective is prediction, the sample size determination should focus on prediction accuracy instead of power. We present formulas for the determination of training set sample size for survival prediction. Sample size is chosen to contr...

journal_title：Statistics in medicine

authors： Götte H,Zwiener I

更新日期：2013-02-28 00:00:00

abstract：:Identification of treatment selection biomarkers has become very important in cancer drug development. Adaptive enrichment designs have been developed for situations where a unique treatment selection biomarker is not apparent based on the mechanism of action of the drug. With such designs, the eligibility rules may b...

journal_title：Statistics in medicine

authors： Simon R,Simon N

更新日期：2017-11-20 00:00:00

abstract：:For patients who were previously treated for prostate cancer, salvage hormone therapy is frequently given when the longitudinal marker prostate-specific antigen begins to rise during follow-up. Because the treatment is given by indication, estimating the effect of the hormone therapy is challenging. In a previous pape...

journal_title：Statistics in medicine

authors： Taylor JM,Shen J,Kennedy EH,Wang L,Schaubel DE

更新日期：2014-01-30 00:00:00

abstract：:Binary matched-pairs data occur commonly in longitudinal studies, such as in cross-over experiments. Many analyses for comparing the matched probabilities of a particular outcome do not utilize pairs having the same outcome for each observation. An example is McNemar's test. Some methodologists find this to be counter...

journal_title：Statistics in medicine

authors： Agresti A,Min Y

更新日期：2004-01-15 00:00:00

abstract：:We present a model for describing correlated binocular data from reader-based diagnostic studies, where the same group of readers evaluates the presence or absence of certain diseases on binocular organs (e.g., fellow eyes) of patients. Multiple random effects are incorporated to meaningfully delineate various associa...

journal_title：Statistics in medicine

authors： Withanage N,de Leon AR,Rudnisky CJ

更新日期：2015-12-20 00:00:00

abstract：:Writing the variance of the Mantel-Haenszel estimator under the null of homogeneity and inverting the corresponding test, we arrive at an improved confidence interval for the common risk difference in stratified 2 × 2 tables. This interval outperforms a variety of other intervals currently recommended in the literatur...

journal_title：Statistics in medicine

authors： Klingenberg B

更新日期：2014-07-30 00:00:00

abstract：:The usual methods for analyzing case-cohort studies rely on sometimes not fully efficient weighted estimators. Multiple imputation might be a good alternative because it uses all the data available and approximates the maximum partial likelihood estimator. This method is based on the generation of several plausible co...

journal_title：Statistics in medicine

authors： Marti H,Chavance M

更新日期：2011-06-15 00:00:00

abstract：:The percentile-finding experimental design known variously as 'forced-choice fixed-staircase', 'geometric up-and-down' or 'k-in-a-row' (KR) was introduced by Wetherill four decades ago. To date, KR has been by far the most widely used up-and-down (U&D) design for estimating non-median percentiles; it is implemented mo...

journal_title：Statistics in medicine

authors： Oron AP,Hoff PD

更新日期：2009-06-15 00:00:00

abstract：:Medical costs data with administratively censored observations often arise in cost-effectiveness studies of treatments for life-threatening diseases. Mean of medical costs incurred from the start of a treatment until death or a certain time point after the implementation of treatment is frequently of interest. In many...

journal_title：Statistics in medicine

authors： Jiang H,Zhou XH

更新日期：2004-11-15 00:00:00

abstract：:For the analysis of time to event data in contraceptive studies when individuals are subject to competing causes for discontinuation, some authors have recently advocated the use of the cumulative incidence rate as a more appropriate measure to summarize data than the complement of the Kaplan-Meier estimate of discont...

journal_title：Statistics in medicine

authors： Farley TM,Ali MM,Slaymaker E

更新日期：2001-12-15 00:00:00

abstract：:This paper investigates the design and analysis of dose-finding trials with two agents. The set of doses for each agent is fixed in advance. The goal of the trial is to find the set of dose combinations with probability of toxicity closest to a pre-specified value. For each of the two agents we assume that the probabi...

journal_title：Statistics in medicine

authors： Ivanova A,Wang K

更新日期：2004-06-30 00:00:00

abstract：:Maps of estimated disease rates over multiple time periods are useful tools for gaining etiologic insights regarding potential exposures associated with specific locations and times. In this paper, we describe an extension of the Gangnon-Clayton model for spatial clustering to spatio-temporal data. As in the purely sp...

journal_title：Statistics in medicine

authors： Gangnon RE

更新日期：2010-09-30 00:00:00

abstract：:We compared eight nominal 95 per cent confidence intervals for the ratio of two Poisson parameters, both assumed small, on their true coverage (the probability that the interval includes the ratio of Poisson parameters) and median width. The commonly used log-linear interval, justified by asymptotic considerations, pr...

journal_title：Statistics in medicine

authors： Barker L,Cadwell BL

更新日期：2008-09-10 00:00:00

abstract：:Quality of life (QOL) assessment is a key component of many clinical studies and frequently requires the use of single global summary measures that capture the overall balance of findings from a potentially wide-ranging assessment of QOL issues. We propose and evaluate an irregular multilevel latent variable model sui...

journal_title：Statistics in medicine

authors： Kifley A,Heller GZ,Beath KJ,Bulger D,Ma J,Gebski V

更新日期：2012-05-20 00:00:00

abstract：:Determination of the equation that relates an ordered dependent variable to ordered independent variables is sought. One solution, non-parametric discriminant analysis (NPD), involves obtaining the best monotonic step function by means of a computer search procedure. Although one can use alternative selection criteria...

journal_title：Statistics in medicine

authors： Johnston B,Seshia SS

更新日期：1992-06-15 00:00:00

abstract：:Assessing the QT prolongation potential of a drug is typically done based on pivotal safety studies called thorough QT studies. Model-based estimation of the drug-induced QT prolongation at the estimated mean maximum drug concentration could increase efficiency over the currently used intersection-union test. However,...

journal_title：Statistics in medicine

authors： Dosne AG,Bergstrand M,Karlsson MO,Renard D,Heimann G

更新日期：2017-10-30 00:00:00

abstract：:Case-control studies are usually defined to investigate risk factors for a single disease of interest. However, subsequent to data collection, investigators may wish to examine as an 'outcome' a variable that was an exposure in the original study. A naive analysis that disregards the sampling strategy that gave rise t...

journal_title：Statistics in medicine

authors： Reilly M,Torrång A,Klint A

更新日期：2005-12-30 00:00:00