Abstract:
:Interim analyses are conducted to allow for early termination of the trial, for ethical as well as economical reasons. Here we consider interim analyses in repeated measurements studies where the measurements are binary. Two methods for analysing this kind of data are compared according to their operating characteristics. A subject-specific approach based on the logistic random-effects model is compared with the population-averaged approach based on the generalized estimating equations. The comparison is illustrated with simulations using a randomized clinical trial for toenail fungal infection.
journal_name
Stat Medjournal_title
Statistics in medicineauthors
Spiessens B,Lesaffre E,Verbeke Gdoi
10.1002/sim.1361subject
Has Abstractpub_date
2003-02-28 00:00:00pages
501-15issue
4eissn
0277-6715issn
1097-0258journal_volume
22pub_type
杂志文章abstract::Continuous-time multistate survival models can be used to describe health-related processes over time. In the presence of interval-censored times for transitions between the living states, the likelihood is constructed using transition probabilities. Models can be specified using parametric or semiparametric shapes fo...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.7604
更新日期:2018-05-10 00:00:00
abstract::Risk prediction models play an important role in prevention and treatment of several diseases. Models that are in clinical use are often refined and improved. In many instances, the most efficient way to improve a successful model is to identify subgroups for which there is a specific biological rationale for improvem...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.6077
更新日期:2014-05-20 00:00:00
abstract::The sample size required for a cluster randomised trial is inflated compared with an individually randomised trial because outcomes of participants from the same cluster are correlated. Sample size calculations for longitudinal cluster randomised trials (including stepped wedge trials) need to take account of at least...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.7028
更新日期:2016-11-20 00:00:00
abstract::Data on alcohol availability and problems in all cities in Los Angeles County were collected from several different sources and linked together to form a Local Alcohol Availability Database (LAAD). The two major purposes of the project are to provide a city-level alcohol availability and alcohol-related problems datab...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.4780140517
更新日期:1995-03-15 00:00:00
abstract::We consider a latent variable hazard model for clustered survival data where clusters are a random sample from an underlying population. We allow interactions between the random cluster effect and covariates. We use a maximum pseudo-likelihood estimator to estimate the mean hazard ratio parameters. We propose a bootst...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/(sici)1097-0258(19970915)16:17<2009::aid-s
更新日期:1997-09-15 00:00:00
abstract::While there has been extensive research developing gene-environment interaction (GEI) methods in case-control studies, little attention has been given to sparse and efficient modeling of GEI in longitudinal studies. In a two-way table for GEI with rows and columns as categorical variables, a conventional saturated int...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.6281
更新日期:2014-12-20 00:00:00
abstract::We consider the design of both cohort and case-control studies in which an initial ('stage 1') sample of complete data on an error-free disease indicator (D), a correct ('gold standard') dichotomous exposure measurement (X) and an error-prone exposure measurement (Z) are available. We calculate the amount of additiona...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.4780142307
更新日期:1995-12-15 00:00:00
abstract::Combinatorial drugs have been widely applied in disease treatment, especially chemotherapy for cancer, due to its improved efficacy and reduced toxicity compared with individual drugs. The study of combinatorial drugs requires efficient experimental designs and proper follow-up statistical modeling techniques. Linear ...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.7971
更新日期:2019-01-30 00:00:00
abstract::Appropriate quantification of added usefulness offered by new markers included in risk prediction algorithms is a problem of active research and debate. Standard methods, including statistical significance and c statistic are useful but not sufficient. Net reclassification improvement (NRI) offers a simple intuitive w...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.4085
更新日期:2011-01-15 00:00:00
abstract::We consider recurrent events of the same type that occur during alternating restraint and non-restraint time periods. This research is motivated by a study on juvenile recidivism, where the probationers were followed for re-offenses during alternating placement periods and free-time periods. During the placement perio...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.7150
更新日期:2017-02-20 00:00:00
abstract::Among subjects who have experienced a biological event, such as menarche, menopause or a delivery, one cannot distinguish the effects of time since the event from age at the event due to the linear dependency among these time variables and age at study ('current age'). This is a well-known problem that also exists in ...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/(SICI)1097-0258(19960730)15:14<1545::AID-S
更新日期:1996-07-30 00:00:00
abstract::In drug-drug interaction (DDI) research, a two drug interaction is usually predicted by individual drug pharmacokinetics (PK). Although subject-specific drug concentration data from clinical PK studies on inhibitor/inducer or substrate's PK are not usually published, sample mean plasma drug concentrations and their st...
journal_title:Statistics in medicine
pub_type: 杂志文章,meta分析
doi:10.1002/sim.2837
更新日期:2007-09-10 00:00:00
abstract::Patient reported outcome and observer evaluative studies in clinical trials and post-hoc analyses often use instruments that measure responses on ordinal-rating or Likert scales. We propose a flexible distributional approach by modeling the change scores from the baseline to the end of the study using independent beta...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.4012
更新日期:2010-10-30 00:00:00
abstract::In observational studies with censored data, exposure-outcome associations are commonly measured with adjusted hazard ratios from multivariable Cox proportional hazards models. The difference in restricted mean survival times (RMSTs) up to a pre-specified time point is an alternative measure that offers a clinically m...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.8206
更新日期:2019-09-10 00:00:00
abstract::Our objective is to develop a model to estimate the relative risk of disease in each area, Ai, i=1, ... , n, of a region and to identify areas of unusually high or low risk. We use a product partition model (PPM) in which we assume that the true relative risks can be partitioned into a number of components or sets of ...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.3253
更新日期:2008-08-30 00:00:00
abstract::Incomplete and unbalanced multivariate data often arise in longitudinal studies due to missing or unequally-timed repeated measurements and/or the presence of time-varying covariates. A general approach to analysing such data is through maximum likelihood analysis using a linear model for the expected responses, and s...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.4780070132
更新日期:1988-01-01 00:00:00
abstract::Exposure to a carcinogen is likely to be reflected by temporal clustering of cancer cases among the community members. Detection of such clustering is a complicated task in general and more so in a small workplace community with low turnover rate. In this study we applied the CUSCORE test and the q-interval statistic ...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.1534
更新日期:2003-10-15 00:00:00
abstract::Cost data that arise in the evaluation of health care technologies usually exhibit highly skew, heavy-tailed and, possibly, multi-modal distributions. Distribution-free methods for analysing these data, such as the bootstrap, or those based on the asymptotic normality of sample means, may often lead to inefficient or ...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.2012
更新日期:2005-10-30 00:00:00
abstract::This paper, arising out of an event to honour the statistical and scientific contributions of Professor Peter Armitage, is concerned with research strategies and needs for chronic disease prevention. A few highlights from recent intervention trials for the prevention of cancer, cardiovascular disease, fractures and di...
journal_title:Statistics in medicine
pub_type:
doi:10.1002/sim.2045
更新日期:2004-11-30 00:00:00
abstract::We propose the use of Gaussian mixed models to analyse statistically 24 hour ambulatory blood pressure data from clinical trials. We develop specific models and apply them to data from a clinical study that compares two angiotensin-converting enzyme inhibitors. We investigate and discuss computing issues related to th...
journal_title:Statistics in medicine
pub_type: 临床试验,杂志文章,随机对照试验
doi:10.1002/sim.4780121803
更新日期:1993-09-30 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
pub_type: 杂志文章
doi:10.1002/sim.3590
更新日期:2009-06-15 00:00:00
abstract::Zero-inflated Poisson regression is a popular tool used to analyze data with excessive zeros. Although much work has already been performed to fit zero-inflated data, most models heavily depend on special features of the individual data. To be specific, this means that there is a sizable group of respondents who endor...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.5650
更新日期:2013-04-30 00:00:00
abstract::Flexible survival models are in need when modelling data from long term follow-up studies. In many cases, the assumption of proportionality imposed by a Cox model will not be valid. Instead, a model that can identify time varying effects of fixed covariates can be used. Although there are several approaches that deal ...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.7088
更新日期:2016-12-10 00:00:00
abstract::The matched case-control designs are commonly used to control for potential confounding factors in genetic epidemiology studies especially epigenetic studies with DNA methylation. Compared with unmatched case-control studies with high-dimensional genomic or epigenetic data, there have been few variable selection metho...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.5694
更新日期:2013-05-30 00:00:00
abstract::We analyse the effect of using prevalence rates based on populations with different sizes in the power of spatial independence tests. We compare the well known spatial correlation Moran's index to three indexes obtained after adjusting for population density, one proposed by Oden, another proposed by Waldhör, and a th...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/(sici)1097-0258(19990830)18:16<2147::aid-s
更新日期:1999-08-30 00:00:00
abstract::With increasingly abundant spatial data in the form of case counts or rates combined over areal regions (eg, ZIP codes, census tracts, or counties), interest turns to formal identification of difference "boundaries," or barriers on the map, in addition to the estimated statistical map itself. "Boundary" refers to a bo...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.7408
更新日期:2017-11-10 00:00:00
abstract::Reference ranges which take time (such as age) into account are often required in medicine, but simple, systematic and efficient statistical methods for constructing them are lacking. A method is described which is based on low order polynomial curves (linear, quadratic or occasionally cubic), together with guidelines...
journal_title:Statistics in medicine
pub_type: 临床试验,杂志文章,多中心研究
doi:10.1002/sim.4780100502
更新日期:1991-05-01 00:00:00
abstract::In the comparison of two or more treatment groups to a control group, consider a study with non-decreasing repeated measurements of the same characteristic taken over a common set of time points for each subject. Based on the vector of possibly incomplete responses from each subject, this paper considers asymptoticall...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/(SICI)1097-0258(19961215)15:23<2509::AID-S
更新日期:1996-12-15 00:00:00
abstract::Genome-wide association studies are characterized by a huge number of statistical tests performed to discover new disease-related genetic variants [in the form of single-nucleotide polymorphisms (SNPs)] in human DNA. Many SNPs have been identified for cross-sectionally measured phenotypes. However, there is a growing ...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.5517
更新日期:2013-01-15 00:00:00
abstract::The publication of Fisher's correspondence on statistics has shed new light on his views on randomization. Quotations from this correspondence and from other works of Fisher are used to illustrate the role of randomization in clinical trials. It is concluded that Fisher's views not only are coherent but, despite havin...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.4780130305
更新日期:1994-02-15 00:00:00