Abstract:
:The intraclass correlation coefficient rho plays a key role in the design of cluster randomized trials. Estimates of rho obtained from previous cluster trials and used to inform sample size calculation in planned trials may be imprecise due to the typically small numbers of clusters in such studies. It may be useful to quantify this imprecision. This study used simulation to compare different methods for assigning bootstrap confidence intervals to rho for continuous outcomes from a balanced design. Data were simulated for combinations of numbers of clusters (10, 30, 50), intraclass correlation coefficients (0.001, 0.01, 0.05, 0.3) and outcome distributions (normal, non-normal continuous). The basic, bootstrap-t, percentile, bias corrected and bias corrected accelerated bootstrap intervals were compared with new methods using the basic and bootstrap-t intervals applied to a variance stabilizing transformation of rho. The standard bootstrap methods provided coverage levels for 95 per cent intervals that were markedly lower than the nominal level for data sets with only 10 clusters, and only provided close to 95 per cent coverage when there were 50 clusters. Application of the bootstrap-t method to the variance stabilizing transformation of rho improved upon the performance of the standard bootstrap methods, providing close to nominal coverage.
journal_name
Stat Medjournal_title
Statistics in medicineauthors
Ukoumunne OC,Davison AC,Gulliford MC,Chinn Sdoi
10.1002/sim.1643subject
Has Abstractpub_date
2003-12-30 00:00:00pages
3805-21issue
24eissn
0277-6715issn
1097-0258journal_volume
22pub_type
杂志文章abstract::Comparative Poisson trials of prophylactic interventions, such as vaccines, can be lengthy and costly. We evaluate two easily implemented approaches to reduce numbers of disease cases and person years of follow up (N(u+t)) for comparative Poisson trials with fixed numbers of cases (T); (i) altering k the portion of N(...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.1724
更新日期:2004-04-30 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
pub_type: 杂志文章
doi:10.1002/sim.4322
更新日期:2011-10-30 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::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 induced smoothing technique overcomes the difficulties caused by the non-smoothness in rank-based estimating functions for accelerated failure time models, but it is only natural when the estimating function has Gehan's weight. For a general weight, the induced smoothing method does not provide smooth estimating f...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.6415
更新日期:2015-04-30 00:00:00
abstract::Substantial advances in Bayesian methods for causal inference have been made in recent years. We provide an introduction to Bayesian inference for causal effects for practicing statisticians who have some familiarity with Bayesian models and would like an overview of what it can add to causal estimation in practical s...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.8761
更新日期:2021-01-30 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
pub_type: 杂志文章
doi:10.1002/sim.1796
更新日期:2004-06-30 00:00:00
abstract::Conventional seamless phase 2/3 design with fixed sample size determination (SSD) has gained its popularity in oncology drug development due to attractive features such as significantly shortening the development timeline, minimizing sample size, as well as early decision making. However, this design is not immune to ...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.8543
更新日期:2020-08-15 00:00:00
abstract::We consider methods for selecting the joint specification of the mean and variance functions in statistical models for rates or counts. Based on analyses of diagnosis-specific hospital discharge rates in Michigan, we show that a Poisson model with an extra variance component for the systematic variation is superior to...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.4780100908
更新日期:1991-09-01 00:00:00
abstract::The relative concentration index (RCI) and the absolute concentration index (ACI) have been widely used for monitoring health disparities with ranked health determinants. The RCI has been extended to allow value judgments about inequality aversion by Pereira in 1998 and by Wagstaff in 2002. Previous studies of the ext...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.7952
更新日期:2019-01-15 00:00:00
abstract::In this work, we describe a two-stage sampling design to estimate the infection prevalence in a population. In the first stage, an imperfect diagnostic test was performed on a random sample of the population. In the second stage, a different imperfect test was performed in a stratified random sample of the first sampl...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.6545
更新日期:2015-11-10 00:00:00
abstract::The article is motivated by a nephrology study in Taiwan, which enrolled hemodialysis patients who suffered from vascular access thrombosis. After treatment, some patients were cured of thrombosis, while some may experience recurrence of either type (acute or nonacute) of vascular access thrombosis. Our major interest...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.8711
更新日期:2020-11-30 00:00:00
abstract::It is unclear to what extent the incremental predictive performance of a novel biomarker is impacted by the method used to control for standard predictors. We investigated whether adding a biomarker to a model with a published risk score overestimates its incremental performance as compared to adding it to a multivari...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.6165
更新日期:2014-07-10 00:00:00
abstract::A frequent objective in medical research is the investigation of differences in patient survival between several experimental treatments and one standard treatment. In order to assess these differences statistically, we have to apply adjustments for multiple comparisons to prevent an increased number of false-positive...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.4403
更新日期:2012-01-13 00:00:00
abstract:BACKGROUND:Multiple imputation is becoming increasingly popular for handling missing data, with Markov chain Monte Carlo assuming multivariate normality (MVN) a commonly used approach. Imputing categorical variables (which are clearly non-normal) using MVN imputation is challenging, and several approaches have been sug...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.5445
更新日期:2012-12-30 00:00:00
abstract::Statistical methods for analyzing recurrent events have attracted significant attention. The majority of existing works consider situations in which subjects are observed over time periods and events of interest that occurred during the course of follow-up are recorded. In some applications, a subject may leave the st...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.8042
更新日期:2019-04-15 00:00:00
abstract::Prevalent sampling is frequently a convenient and economical sampling technique for the collection of time-to-event data and thus is commonly used in studies of the natural history of a disease. However, it is biased by design because it tends to recruit individuals with longer survival times. This paper considers est...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.7184
更新日期:2017-04-15 00:00:00
abstract::We explore the 'reassessment' design in a logistic regression setting, where a second wave of sampling is applied to recover a portion of the missing data on a binary exposure and/or outcome variable. We construct a joint likelihood function based on the original model of interest and a model for the missing data mech...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.6456
更新日期:2015-05-20 00:00:00
abstract::We consider a general model for anomaly detection in a longitudinal cohort mortality pattern based on logistic joinpoint regression with unknown joinpoints. We discuss backward and forward sequential procedures for selecting both the locations and the number of joinpoints. Estimation of the model parameters and the se...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.3017
更新日期:2008-04-30 00:00:00
abstract::Slow recruitment in clinical trials leads to increased costs and resource utilization, which includes both the clinic staff and patient volunteers. Careful planning and monitoring of the accrual process can prevent the unnecessary loss of these resources. We propose two hierarchical extensions to the existing Bayesian...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.6359
更新日期:2015-02-20 00:00:00
abstract::In behavioral, biomedical, and social-psychological sciences, it is common to encounter latent variables and heterogeneous data. Mixture structural equation models (SEMs) are very useful methods to analyze these kinds of data. Moreover, the presence of missing data, including both missing responses and missing covaria...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.3915
更新日期:2010-08-15 00:00:00
abstract::Multistate models are increasingly being used to model complex disease profiles. By modelling transitions between disease states, accounting for competing events at each transition, we can gain a much richer understanding of patient trajectories and how risk factors impact over the entire disease pathway. In this arti...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.7448
更新日期:2017-12-20 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
pub_type: 杂志文章
doi:10.1002/sim.7043
更新日期:2017-01-30 00:00:00
abstract::Immigration-death models are proposed to analyse the infection dynamics in longitudinal studies of panels of heavily parasitized human hosts where parasites have been typed at regular intervals by PCR. Immigration refers to the acquisition of a new parasitic genotype, occurring at rate lambda, and death refers to the ...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.2189
更新日期:2005-11-15 00:00:00
abstract::This paper presents a statistically optimal exact hypothesis testing procedure for detecting changes in sales adjusted adverse drug reaction (ADR) rates between historical and current periods, with a computer program that implements this test appended. We provide discussions and illustrations on how to monitor ADR rat...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.4780071004
更新日期:1988-10-01 00:00:00
abstract::Paired count data usually arise in medicine when before and after treatment measurements are considered. In the present paper we assume that the correlated paired count data follow a bivariate Poisson distribution in order to derive the distribution of their difference. The derived distribution is shown to be the same...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.2382
更新日期:2006-06-15 00:00:00
abstract::In this paper, we consider fitting a flexible and interpretable additive regression model in a data-rich setting. We wish to avoid pre-specifying the functional form of the conditional association between each covariate and the response, while still retaining interpretability of the fitted functions. A number of recen...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.7859
更新日期:2019-02-20 00:00:00
abstract::Motivated by a previously published study of HIV treatment, we simulated data subject to time-varying confounding affected by prior treatment to examine some finite-sample properties of marginal structural Cox proportional hazards models. We compared (a) unadjusted, (b) regression-adjusted, (c) unstabilized, and (d) s...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.5317
更新日期:2012-08-30 00:00:00
abstract::The stability-controlled quasi-experiment (SCQE) is an approach to study the effects of nonrandomized, newly adopted treatments. While covariate adjustment techniques rely on a "no unobserved confounding" assumption, SCQE imposes an assumption on the change in the average nontreatment outcome between successive cohort...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.8717
更新日期:2020-12-10 00:00:00
abstract::To update the British growth reference, anthropometric data for weight, height, body mass index (weight/height2) and head circumference from 17 distinct surveys representative of England, Scotland and Wales (37,700 children, age range 23 weeks gestation to 23 years) were analysed by maximum penalized likelihood using ...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:
更新日期:1998-02-28 00:00:00