Power analyses for longitudinal study designs with missing data.

abstract：:Surrogate endpoint validation has been well established by the meta-analytical correlation-based approach as outlined in the seminal work of Buyse et al. (Biostatistics, 2000). Surrogacy can be assumed if strong associations on individual and study levels can be demonstrated. Alternatively, if an effect on a true endp...

journal_title：Statistics in medicine

authors： Schürmann C,Sieben W

更新日期：2016-03-30 00:00:00

abstract：:This paper considers the effect of non-differential outcome misclassification on the population attributable fraction and the population prevented fraction. I examine the bias in the attributable and the prevented fraction derived from a risk ratio estimate as a function of the sensitivity and specificity of the outco...

journal_title：Statistics in medicine

authors： Hsieh CC

更新日期：1991-03-01 00:00:00

abstract：:Laboratory determinations on children aged 6 to 10 years obtained over a 5-year period are analysed by a method described in detail for differentiating between children from exposed and control areas of Seveso, Italy. In the analysis, stratification is employed to distinguish the separate days of laboratory measuremen...

journal_title：Statistics in medicine

authors： Mantel N,Mocarelli P,Marocchi A,Brambilla P,Baretta R

更新日期：1983-04-01 00:00:00

abstract：:The use of the last observation carried forward (LOCF) method for imputing missing outcome data in randomized clinical trials has been much criticized and its shortcomings are well understood. However, only recently have published studies widely started using more appropriate imputation methods. Consequently, meta-ana...

journal_title：Statistics in medicine

authors： Mavridis D,Salanti G,Furukawa TA,Cipriani A,Chaimani A,White IR

更新日期：2019-02-28 00:00:00

abstract：:This paper gives a standard error for Cohen's Kappa, conditional on the margins of the observed r x r table. An explicit formula is given for the 2 x 2 table, and a procedure for the more general situation. A parsimonious log-linear model is suggested for the general case and an approximate confidence interval for kap...

journal_title：Statistics in medicine

authors： Garner JB

更新日期：1991-05-01 00:00:00

abstract：:Although all clinical trials are designed and monitored using more than one endpoint, methods are needed to assure that decision criteria are chosen to reflect the clinically relevant tradeoffs that assure the trial's scientific integrity. This article presents a framework for the design and monitoring clinical trials...

journal_title：Statistics in medicine

authors： Hu J,Blatchford PJ,Goldenberg NA,Kittelson JM

更新日期：2020-11-20 00:00:00

abstract：:I derive the locally most powerful rank tests for acceleration against semi-parametric alternatives when some patients are cured of the disease. I consider some particular classes of alternatives and present simulation results to verify the validity of the proposed tests. Real data from clinical trials for childhood l...

journal_title：Statistics in medicine

authors： Lee JW

更新日期：1995-10-15 00:00:00

abstract：:We have used Monte Carlo methods to compare the type I error properties of the conditional and unconditional versions of the generalized t and the generalized rank-sum tests to those of the independent samples t and Wilcoxon rank-sum tests. Results showed inflated type I errors for the conditional generalized tests bu...

journal_title：Statistics in medicine

authors： Blair RC,Morel JG

更新日期：1992-02-28 00:00:00

abstract：:In multiple-record systems estimation it is usually assumed that all registration relate to the same population. In this paper, we develop a method which can be used when the registrations relate to different populations, in the sense that they cover, for example, different time periods or regions. We show that under ...

journal_title：Statistics in medicine

authors： Zwane EN,van der Pal-de Bruin K,van der Heijden PG

更新日期：2004-07-30 00:00:00

abstract：:This paper discusses design considerations and the role of randomization-based inference in randomized community intervention trials. We stress that longitudinal follow-up of cohorts within communities often yields useful information on the effects of intervention on individuals, whereas cross-sectional surveys can us...

journal_title：Statistics in medicine

authors： Gail MH,Mark SD,Carroll RJ,Green SB,Pee D

更新日期：1996-06-15 00:00:00

abstract：:We discuss the use of the trichotomous logistic model to discriminate between patients with gastrointestinal (GI) cancer, patients with benign GI disease and 'normal' subjects, using symptoms and the concentrations of some serum proteins that are potentially indicative of malignancy as covariates. A parsimonious model...

journal_title：Statistics in medicine

authors： Marshall RJ,Chisholm EM

更新日期：1985-07-01 00:00:00

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

authors： Wang L,Krieger AM

更新日期：2006-07-15 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：:We consider the problem of identifying a subgroup of patients who may have an enhanced treatment effect in a randomized clinical trial, and it is desirable that the subgroup be defined by a limited number of covariates. For this problem, the development of a standard, pre-determined strategy may help to avoid the well...

journal_title：Statistics in medicine

authors： Foster JC,Taylor JM,Ruberg SJ

更新日期：2011-10-30 00:00:00

abstract：:In this paper we study the problem of variable selection for the proportional odds model, which is a useful alternative to the proportional hazards model and might be appropriate when the proportional hazards assumption is not satisfied. We propose to fit the proportional odds model by maximizing the marginal likeliho...

journal_title：Statistics in medicine

authors： Lu W,Zhang HH

更新日期：2007-09-10 00:00:00

abstract：:Genetic networks for gene expression data are often built by graphical models, which in turn are built from pair-wise correlations of gene expression levels. A key feature of building graphical models is the evaluation of conditional independence of two traits, given other traits. When conditional independence can be ...

journal_title：Statistics in medicine

authors： Yu Z,Wang L,Hildebrandt MA,Schaid DJ

更新日期：2008-08-30 00:00:00

abstract：:This paper presents combinatorial (exact) methods that are useful in the analysis of disease cluster data obtained from small environments, such as buildings and neighbourhoods. Maxwell-Boltzmann and Fermi-Dirac occupancy models are compared in terms of appropriateness of representation of disease incidence patterns (...

journal_title：Statistics in medicine

authors： Grimson RC

更新日期：1993-10-01 00:00:00

abstract：:The goal of phase I cancer trials is to determine the highest dose of a treatment regimen with an acceptable toxicity rate. Traditional designs for phase I trials, such as the Continual Reassessment Method (CRM) and the 3 + 3 design, require each patient or a cohort of patients to be fully evaluated for the dose-limit...

journal_title：Statistics in medicine

authors： Polley MY

更新日期：2011-07-30 00:00:00

abstract：:We propose a new design for dose finding for cytotoxic agents in two ordered groups of patients. By ordered groups, we mean that prior to the study there is clinical information that would indicate that for a given dose one group would be more susceptible to toxicities than patients in the other group. The designs are...

journal_title：Statistics in medicine

authors： Conaway MR,Wages NA

更新日期：2017-01-30 00:00:00

abstract：:To summarize safety data such as clinical adverse experiences in clinical trials with a moderate to long-term follow-up, we may use a measurement which accounts for the potential differences in the follow-up duration between treatment groups. The incidence rate, which uses the total person-time follow-up in a treatmen...

journal_title：Statistics in medicine

authors： Liu GF,Wang J,Liu K,Snavely DB

更新日期：2006-04-30 00:00:00

abstract：:Toxicity probability interval designs have received increasing attention as a dose-finding method in recent years. In this study, we compared the two-stage, likelihood-based continual reassessment method (CRM), modified toxicity probability interval (mTPI), and the Bayesian optimal interval design (BOIN) in order to e...

journal_title：Statistics in medicine

authors： Horton BJ,Wages NA,Conaway MR

更新日期：2017-01-30 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：:Clinical trials studying treatments for rare diseases are challenging to design and conduct due to the limited number of patients eligible for the trial. One design used to address this challenge is the small n, sequential, multiple assignment, randomized trial (snSMART). We propose a new snSMART design that investiga...

journal_title：Statistics in medicine

authors： Fang F,Hochstedler KA,Tamura RN,Braun TM,Kidwell KM

更新日期：2021-02-20 00:00:00

abstract：:The construction, validation and updating of a prognostic model for kidney graft survival is reported using data from the Eurotransplant database. First, a model is constructed for data from transplantations in the period 1984 to 1987. The model is later updated for the 1988 1990 data. The first data set was randomly ...

journal_title：Statistics in medicine

authors： Van Houwelingen HC,Thorogood J

更新日期：1995-09-30 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：:Population heterogeneity is frequently observed among patients' treatment responses in clinical trials because of various factors such as clinical background, environmental, and genetic factors. Different subpopulations defined by those baseline factors can lead to differences in the benefit or safety profile of a the...

journal_title：Statistics in medicine

authors： Chiu YD,Koenig F,Posch M,Jaki T

更新日期：2018-12-20 00:00:00

abstract：:We review two recent trends: the emergence of evidence-based medicine and the growing use of Bayesian statistics in medical applications. Evidence-based medicine requires an integrated assessment of the available evidence, and associated uncertainty, but there is also an emphasis on decision-making, for individual pat...

journal_title：Statistics in medicine

authors： Ashby D,Smith AF

更新日期：2000-12-15 00:00:00

abstract：:It is often assumed that randomisation will prevent bias in estimation of treatment effects from clinical trials, but this is not true of the semiparametric Proportional Hazards model for survival data when there is underlying risk heterogeneity. Here, a new formula is proposed for estimation of this bias, improving o...

journal_title：Statistics in medicine

authors： McNamee R

更新日期：2017-09-20 00:00:00

abstract：:Clinical studies that involve the recording of two or more distinct and well-defined events on each subject give rise to multiple event data. Treatment comparisons are usually reported in univariate analyses of time to first event or number of events observed. However, this approach may not uncover the 'full story' of...

journal_title：Statistics in medicine

authors： Barai U,Teoh N

更新日期：1997-04-30 00:00:00

abstract：:In medical research, risk difference (RD) and number needed to treat (NNT) measures for survival times have been mainly proposed without consideration of covariates. In this paper, we develop adjusted RD and NNT measures for use in observational studies with survival time outcomes within the framework of the Cox propo...

journal_title：Statistics in medicine

authors： Laubender RP,Bender R

更新日期：2010-03-30 00:00:00