Obtaining evidence by a single well-powered trial or several modestly powered trials.

abstract：:Several epidemiological parameters have been introduced for quantifying the population impact of a certain exposure on morbidity on a population level, termed 'attributable risk' (AR). Of these definitions, the AR as suggested by Levin in 1953 or some algebraic transformations of it are most commonly used. A structure...

journal_title：Statistical methods in medical research

authors： Uter W,Pfahlberg A

更新日期：2001-06-01 00:00:00

abstract：:Two tests are proposed for checking the linearity of nonparametric function in partially linear models. The first one is based on a Crámer-von Mises statistic. This test can detect the local alternative converging to the null at the parametric rate 1/square root n. A bootstrap resample technique is provided to calcula...

journal_title：Statistical methods in medical research

authors： Liang H

更新日期：2006-06-01 00:00:00

abstract：:Stochastic transmission dynamic models are needed to quantify the uncertainty in estimates and predictions during outbreaks of infectious diseases. We previously developed a calibration method for stochastic epidemic compartmental models, called Multiple Shooting for Stochastic Systems (MSS), and demonstrated its comp...

journal_title：Statistical methods in medical research

authors： Zimmer C,Leuba SI,Cohen T,Yaesoubi R

更新日期：2019-12-01 00:00:00

abstract：:The probability model for periodic screening was extended to provide statistical inference for sensitivity depending on sojourn time, in which the sensitivity was modeled as a function of time spent in the preclinical state and the sojourn time. The likelihood function with the proposed sensitivity model was then eval...

journal_title：Statistical methods in medical research

authors： Kim S,Wu D

更新日期：2016-04-01 00:00:00

abstract：:Couples with diseases associated with the sexual chromosomes, as well as families in countries where the desire for a male is extreme, are interested in influencing the sex of the baby. We propose an original composite likelihood approach to analyse the relation between sex of the newborn and timing of the intercourse...

journal_title：Statistical methods in medical research

authors： Tiberi S,Scarpa B,Sartori N

更新日期：2018-11-01 00:00:00

abstract：:Clinical trials investigating the efficacy of two or more doses of an experimental treatment compared to a single reference arm are not uncommon. In such situations, if each dose is compared to the reference arm using an un-adjusted significance level, consideration of the Type I familywise error is likely to be requi...

journal_title：Statistical methods in medical research

authors： Fernandes N,Stone A

更新日期：2011-12-01 00:00:00

abstract：:This work presents a brief overview of Markov models in cancer screening evaluation and focuses on two specific models. A three-state model was first proposed to estimate jointly the sensitivity of the screening procedure and the average duration in the preclinical phase, i.e. the period when the cancer is asymptomati...

journal_title：Statistical methods in medical research

authors： Uhry Z,Hédelin G,Colonna M,Asselain B,Arveux P,Rogel A,Exbrayat C,Guldenfels C,Courtial I,Soler-Michel P,Molinié F,Eilstein D,Duffy SW

更新日期：2010-10-01 00:00:00

abstract：:Statistical methods for spatial health data to identify the significant covariates associated with the health outcomes are of critical importance. Most studies have developed variable selection approaches in which the covariates included appear within the spatial domain and their effects are fixed across space. Howeve...

journal_title：Statistical methods in medical research

authors： Choi J,Lawson AB

更新日期：2018-01-01 00:00:00

abstract：:In multi-regional trials, the underlying overall and region-specific accrual rates often do not hold constant over time and different regions could have different start-up times, which combined with initial jump in accrual within each region often leads to a discontinuous overall accrual rate, and these issues associa...

journal_title：Statistical methods in medical research

authors： Deng Y,Zhang X,Long Q

更新日期：2017-04-01 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：:Rapid Early Action for Coronary Treatment (REACT) was a multisite trial testing a community intervention to reduce the delay between onset of symptoms of acute myocardial infarction (MI) and patients' arrival at a hospital emergency department. The study employed a group-randomized trial design, with ten communities r...

journal_title：Statistical methods in medical research

authors： Murray DM,Feldman HA,McGovern PG

更新日期：2000-04-01 00:00:00

abstract：:Accelerated failure time model is a popular model to analyze censored time-to-event data. Analysis of this model without assuming any parametric distribution for the model error is challenging, and the model complexity is enhanced in the presence of large number of covariates. We developed a nonparametric Bayesian met...

journal_title：Statistical methods in medical research

authors： Zhang Z,Sinha S,Maiti T,Shipp E

更新日期：2018-04-01 00:00:00

abstract：:Serial measurements of prostate-specific antigen (PSA) are used as a biomarker for men diagnosed with prostate cancer following an active monitoring programme. Distinguishing pathological changes from natural age-related changes is not straightforward. Here, we compare four approaches to modelling age-related change i...

journal_title：Statistical methods in medical research

authors： Simpkin AJ,Metcalfe C,Martin RM,Lane JA,Donovan JL,Hamdy FC,Neal DE,Tilling K

更新日期：2016-10-01 00:00:00

abstract：:Monte Carlo evaluation of resampling-based tests is often conducted in statistical analysis. However, this procedure is generally computationally intensive. The pooling resampling-based method has been developed to reduce the computational burden but the validity of the method has not been studied before. In this arti...

journal_title：Statistical methods in medical research

authors： Fung WK,Yu K,Yang Y,Zhou JY

更新日期：2018-05-01 00:00:00

abstract：:Cluster analysis via a finite mixture model approach is considered. With this approach to clustering, the data can be partitioned into a specified number of clusters g by first fitting a mixture model with g components. An outright clustering of the data is then obtained by assigning an observation to the component to...

journal_title：Statistical methods in medical research

authors： McLachlan GJ,Chang SU

更新日期：2004-10-01 00:00:00

abstract：:Excessive zeros are common in practice and may cause overdispersion and invalidate inference when fitting Poisson regression models. There is a large body of literature on zero-inflated Poisson models. However, methods for testing whether there are excessive zeros are less well developed. The Vuong test comparing a Po...

journal_title：Statistical methods in medical research

authors： He H,Zhang H,Ye P,Tang W

更新日期：2019-04-01 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 cluster randomized trials, clusters of subjects are randomized rather than subjects themselves, and missing outcomes are a concern as in individual randomized trials. We assessed strategies for handling missing data when analysing cluster randomized trials with a binary outcome; strategies included complete case, a...

journal_title：Statistical methods in medical research

authors： Caille A,Leyrat C,Giraudeau B

更新日期：2016-12-01 00:00:00

abstract：:Statistical methods for carrying out asymptotic inferences (tests or confidence intervals) relative to one or two independent binomial proportions are very frequent. However, inferences about a linear combination of K independent proportions L = Σβ(i)p(i) (in which the first two are special cases) have had very little...

journal_title：Statistical methods in medical research

authors： Martín Andrés A,Alvarez Hernández M,Herranz Tejedor I

更新日期：2011-08-01 00:00:00

abstract：:Researchers and clinicians often need to know whether a new method of measurement is equivalent to an established one that is already in use. For this problem, the estimation of limits of agreement advocated by Bland and Altman is a widely used solution. However, this approach ignores two vital issues in method compar...

journal_title：Statistical methods in medical research

authors： Alanen E

更新日期：2012-08-01 00:00:00

abstract：:Data in many experiments arise as curves and therefore it is natural to use a curve as a basic unit in the analysis, which is termed functional data analysis (FDA). In longitudinal studies, recent developments in FDA have extended classical linear models and linear mixed effects models to functional linear models (als...

journal_title：Statistical methods in medical research

authors： Guo W

更新日期：2004-02-01 00:00:00

abstract：:In recent years, there has been a prominent discussion in the literature about the potential for overestimation of the treatment effect when a clinical trial stops at an interim analysis due to the experimental treatment showing a benefit over the control. However, there has been much less attention paid to the conver...

journal_title：Statistical methods in medical research

authors： Marschner IC,Schou IM

更新日期：2019-10-01 00:00:00

abstract：:Competing risks data often exist within a center in multi-center randomized clinical trials where the treatment effects or baseline risks may vary among centers. In this paper, we propose a subdistribution hazard regression model with multivariate frailty to investigate heterogeneity in treatment effects among centers...

journal_title：Statistical methods in medical research

authors： Ha ID,Christian NJ,Jeong JH,Park J,Lee Y

更新日期：2016-12-01 00:00:00

abstract：:This article discusses and extends statistical models to jointly analyse the spatial variation of rates of several diseases with common risk factors. We start with a review of methods for separate analyses of diseases, then move to ecological regression approaches, where the rates from one of the diseases enter as sur...

journal_title：Statistical methods in medical research

authors： Held L,Natário I,Fenton SE,Rue H,Becker N

更新日期：2005-02-01 00:00:00

abstract：:A central assumption in the design and conduct of non-inferiority trials is that the active-control therapy will have the same degree of effectiveness in the planned non-inferiority trial as in the prior placebo-controlled trials used to define the non-inferiority margin. This is referred to as the 'constancy' assumpt...

journal_title：Statistical methods in medical research

authors： Hanscom B,Hughes JP,Williamson BD,Donnell D

更新日期：2019-10-01 00:00:00

abstract：:The use of genetic variants as instrumental variables - an approach known as Mendelian randomization - is a popular epidemiological method for estimating the causal effect of an exposure (phenotype, biomarker, risk factor) on a disease or health-related outcome from observational data. Instrumental variables must sati...

journal_title：Statistical methods in medical research

authors： Bucur IG,Claassen T,Heskes T

更新日期：2020-04-01 00:00:00

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：: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：:The Cochran-Armitage (CA) test is commonly used in both epidemiology and genetics to test for linear trend in two-way tables with a binary outcome. There has been increasing interest in the power and size of the test and in determination of sample size, especially when there is potential misclassification in the 'expo...

journal_title：Statistical methods in medical research

authors： Buonaccorsi JP,Laake P,Veierød MB

更新日期：2014-06-01 00:00:00

abstract：:Propensity score methods are common for estimating a binary treatment effect when treatment assignment is not randomized. When exposure is measured on an ordinal scale (i.e. low-medium-high), however, propensity score inference requires extensions which have received limited attention. Estimands of possible interest w...

journal_title：Statistical methods in medical research

authors： Lopez MJ,Gutman R

更新日期：2017-04-01 00:00:00