Analysis of phase II methodologies for single-arm clinical trials with multiple endpoints in rare cancers: An example in Ewing's sarcoma.

abstract：:Within paediatric populations, there may be distinct age groups characterised by different exposure-response relationships. Several regulatory guidance documents have suggested general age groupings. However, it is not clear whether these categorisations will be suitable for all new medicines and in all disease areas....

journal_title：Statistical methods in medical research

authors： Wadsworth I,Hampson LV,Bornkamp B,Jaki T

更新日期：2020-09-01 00:00:00

abstract：:A growing body of evidence suggests that genetic factors have an important influence on the onset and course of smoking. Here we review some of the statistical methods that have been used to test for genetic influences on smoking behaviour, with a particular focus on studies of large national twin samples. We show how...

journal_title：Statistical methods in medical research

authors： Heath AC,Madden PA,Martin NG

更新日期：1998-06-01 00:00:00

abstract：:In many applications of zero-inflated models, score tests are often used to evaluate whether the population heterogeneity as implied by these models is consistent with the data. The most frequently cited justification for using score tests is that they only require estimation under the null hypothesis. Because this es...

journal_title：Statistical methods in medical research

authors： Hsu WW,Todem D,Mawella NR,Kim K,Rosenkranz RR

更新日期：2020-12-01 00:00:00

abstract：:To evaluate the antiretroviral activity of antiretroviral agents and to compare the effects of two different antiretroviral agents, we propose a non-parametric mixed-effects model to investigate change of CD4+ counts. The proposed model and methods are applied to analyse the data from PACTG345 study. Population and in...

journal_title：Statistical methods in medical research

authors： Liang H

更新日期：2008-04-01 00:00:00

abstract：:There is debate whether clinical trials with suboptimal power are justified and whether results from large studies are more reliable than the (combined) results of smaller trials. We quantified the error rates for evaluations based on single conventionally powered trials (80% or 90% power) versus evaluations based on ...

journal_title：Statistical methods in medical research

authors： IntHout J,Ioannidis JP,Borm GF

更新日期：2016-04-01 00:00:00

abstract：:One purpose of a longitudinal study is to gain a better understanding of how an outcome of interest changes among a given population over time. In what follows, a trajectory will be taken to mean the series of measurements of the outcome variable for an individual. Group-based trajectory modelling methods seek to iden...

journal_title：Statistical methods in medical research

authors： Davies CE,Glonek GF,Giles LC

更新日期：2017-08-01 00:00:00

abstract：:For semi-continuous data which are a mixture of true zeros and continuously distributed positive values, the use of two-part mixed models provides a convenient modelling framework. However, deriving population-averaged (marginal) effects from such models is not always straightforward. Su et al. presented a model that ...

journal_title：Statistical methods in medical research

authors： Tom BD,Su L,Farewell VT

更新日期：2016-10-01 00:00:00

abstract：:Statistical models of breast cancer tumour progression have been used to further our knowledge of the natural history of breast cancer, to evaluate mammography screening in terms of mortality, to estimate overdiagnosis, and to estimate the impact of lead-time bias when comparing survival times between screen detected ...

journal_title：Statistical methods in medical research

authors： Isheden G,Humphreys K

更新日期：2019-03-01 00:00:00

abstract：:In genomic analysis, it is significant though challenging to identify markers associated with cancer outcomes or phenotypes. Based on the biological mechanisms of cancers and the characteristics of datasets, we propose a novel integrative interaction approach under a semiparametric model, in which genetic and environm...

journal_title：Statistical methods in medical research

authors： Li Y,Wang F,Li R,Sun Y

更新日期：2020-10-01 00:00:00

abstract：:We review the origins of backcalculation (or back projection) methods developed for the analysis of AIDS (acquired immunodeficiency syndrome) incidence data. These techniques have been used extensively for >15 years to deconvolute clinical case incidence, given knowledge of the incubation period distribution, to obtai...

journal_title：Statistical methods in medical research

authors： Donnelly CA,Ferguson NM,Ghani AC,Anderson RM

更新日期：2003-06-01 00:00:00

abstract：:In reliability theory, diagnostic accuracy, and clinical trials, the quantity P ( X > Y ) + ...

journal_title：Statistical methods in medical research

authors： Verbeeck J,Deltuvaite-Thomas V,Berckmoes B,Burzykowski T,Aerts M,Thas O,Buyse M,Molenberghs G

更新日期：2020-12-01 00:00:00

abstract：:This paper reviews the application of statistical models to outbreaks of two common respiratory viral diseases, measles and influenza. For each disease, we look first at its epidemiological characteristics and assess the extent to which these either aid or hinder modelling. We then turn to the models that have been de...

journal_title：Statistical methods in medical research

authors： Cliff AD,Haggett P

更新日期：1993-01-01 00:00:00

abstract：:In medical experiments with the objective of testing the equality of two means, data are often partially paired by design or because of missing data. The partially paired data represent a combination of paired and unpaired observations. In this article, we review and compare nine methods for analyzing partially paired...

journal_title：Statistical methods in medical research

authors： Guo B,Yuan Y

更新日期：2017-06-01 00:00:00

abstract：BACKGROUND:Recent literature on the comparison of machine learning methods has raised questions about the neutrality, unbiasedness and utility of many comparative studies. Reporting of results on favourable datasets and sampling error in the estimated performance measures based on single samples are thought to be the m...

journal_title：Statistical methods in medical research

authors： Khondoker M,Dobson R,Skirrow C,Simmons A,Stahl D

更新日期：2016-10-01 00:00:00

abstract：:In the field of diagnostic studies for tree or umbrella ordering, under which the marker measurement for one class is lower or higher than those for the rest unordered classes, there exist a few diagnostic measures such as the naive AUC ( NAUC), the umbrella volume ( UV), and the recently proposed TAUC, i.e. area unde...

journal_title：Statistical methods in medical research

authors： Feng Y,Tian L

更新日期：2019-05-01 00:00:00

abstract：:DNA methylation has been shown to play an important role in many complex diseases. The rapid development of high-throughput DNA methylation scan technologies provides great opportunities for genomewide DNA methylation-disease association studies. As methylation is a dynamic process involving time, it is quite plausibl...

journal_title：Statistical methods in medical research

authors： Wang C,Shen Q,Du L,Xu J,Zhang H

更新日期：2018-09-01 00:00:00

abstract：:Dependent binary response data arise frequently in practice due to repeated measurements in longitudinal studies or to subsampling primary sampling units as in fields such as teratology and ophthalmology. Several classes of approaches have recently been proposed to analyse such repeated binary outcome data. The differ...

journal_title：Statistical methods in medical research

authors： Neuhaus JM

更新日期：1992-01-01 00:00:00

abstract：:Common limitations of clustering methods include the slow algorithm convergence, the instability of the pre-specification on a number of intrinsic parameters, and the lack of robustness to outliers. A recent clustering approach proposed a fast search algorithm of cluster centers based on their local densities. However...

journal_title：Statistical methods in medical research

authors： Wang XF,Xu Y

更新日期：2017-12-01 00:00:00

abstract：:Mixed models estimated by maximum likelihood and marginal models estimated by generalized estimating equations are the standard methods for the analysis of longitudinal data. However, their use is highly debated when attrition may be due to death. While some authors consider that mixed model estimates are interpretabl...

journal_title：Statistical methods in medical research

authors： Rouanet A,Helmer C,Dartigues JF,Jacqmin-Gadda H

更新日期：2019-02-01 00:00:00

abstract：:To project national hepatitis C virus (HCV) burden, unbiased estimation of HCV progression to liver cirrhosis is required for the whole community of HCV-infected individuals. However, widely varying estimates of progression rates to cirrhosis have been produced. This disparity is partly associated with the statistical...

journal_title：Statistical methods in medical research

authors： Fu B,Tom BD,Bird SM

更新日期：2009-06-01 00:00:00

abstract：:Methods for the analysis of reliability of ordered categorical scales are discussed, focussing on the limitation of the single summary-weighted kappa coefficients. A symmetric matrix of kappa-type coefficients is suggested as an alternative. The method is proposed as being suitable for ordinal scale where there is no ...

journal_title：Statistical methods in medical research

authors： Roberts C,McNamee R

更新日期：2005-10-01 00:00:00

abstract：:Evaluation of medical imaging devices often involves clinical studies where multiple readers (MR) read images of multiple cases (MC) for a clinical task, which are often called MRMC studies. In addition to sizing patient cases as is required in most clinical trials, MRMC studies also require sizing readers, since both...

journal_title：Statistical methods in medical research

authors： Huang Z,Samuelson F,Tcheuko L,Chen W

更新日期：2020-06-01 00:00:00

abstract：:Variable selection in semiparametric mixed models for longitudinal data remains a challenge, especially in the presence of multiple correlated outcomes. In this paper, we propose a model selection procedure that simultaneously selects fixed and random effects using a maximum penalized likelihood method with the adapti...

journal_title：Statistical methods in medical research

authors： Li Z,Liu H,Tu W

更新日期：2018-10-01 00:00:00

abstract：:A bivariate generalised linear mixed model is often used for meta-analysis of test accuracy studies. The model is complex and requires five parameters to be estimated. As there is no closed form for the likelihood function for the model, maximum likelihood estimates for the parameters have to be obtained numerically. ...

journal_title：Statistical methods in medical research

authors： Willis BH,Baragilly M,Coomar D

更新日期：2020-04-01 00:00:00

abstract：:The maximal procedure is a restricted randomization method that maximizes the number of feasible allocation sequences under the constraints of the maximum tolerated imbalance and the allocation sequence length. It assigns an equal probability to all feasible sequences. However, its implementation is not easy due to th...

journal_title：Statistical methods in medical research

authors： Zhao W,Berger VW,Yu Z

更新日期：2018-07-01 00:00:00

abstract：:In many experiments and especially in translational and preclinical research, sample sizes are (very) small. In addition, data designs are often high dimensional, i.e. more dependent than independent replications of the trial are observed. The present paper discusses the applicability of max t-test-type statistics (mu...

journal_title：Statistical methods in medical research

authors： Konietschke F,Schwab K,Pauly M

更新日期：2020-11-24 00:00:00

abstract：:Estimation of the effect of a binary exposure on an outcome in the presence of confounding is often carried out via outcome regression modelling. An alternative approach is to use propensity score methodology. The propensity score is the conditional probability of receiving the exposure given the observed covariates a...

journal_title：Statistical methods in medical research

authors： Williamson E,Morley R,Lucas A,Carpenter J

更新日期：2012-06-01 00:00:00

abstract：:This paper reviews models for incomplete continuous and categorical longitudinal data. In terms of Rubin's classification of missing value processes we are specifically concerned with the problem of nonrandom missingness. A distinction is drawn between the classes of selection and pattern-mixture models and, using sev...

journal_title：Statistical methods in medical research

authors： Kenward MG,Molenberghs G

更新日期：1999-03-01 00:00:00

abstract：:Tree-based methods are very powerful and popular tools for analysing survival data with right-censoring. The existing methods assume that the true time-to-event and the censoring times are independent given the covariates. We propose different ways to build survival forests when dependent censoring is suspected, by us...

journal_title：Statistical methods in medical research

authors： Moradian H,Larocque D,Bellavance F

更新日期：2019-02-01 00:00:00

abstract：:Understanding the limitation of solely relying on statistical significance, researchers have proposed methods to draw biomedical conclusions based on clinical significance. The minimal clinically important significance is one of the most fundamental concepts to study clinical significance. Based on an anchor question ...

journal_title：Statistical methods in medical research

authors： Zhou Z,Zhao J,Bisson LJ

更新日期：2020-03-01 00:00:00