Abstract:
:The estimation of population parameters using complex survey data requires careful statistical modelling to account for the design features. This is further complicated by unit and item nonresponse for which a number of methods have been developed in order to reduce estimation bias. In this paper, we address some issues that arise when the target of the inference (i.e. the analysis model or model of interest) is the conditional quantile of a continuous outcome. Survey design variables are duly included in the analysis and a bootstrap variance estimation approach is proposed. Missing data are multiply imputed by means of chained equations. In particular, imputation of continuous variables is based on their empirical distribution, conditional on all other variables in the analysis. This method preserves the distributional relationships in the data, including conditional skewness and kurtosis, and successfully handles bounded outcomes. Our motivating study concerns the analysis of birthweight determinants in a large UK-based cohort of children. A novel finding on the parental conflict theory is reported. R code implementing these procedures is provided.
journal_name
Stat Methods Med Resjournal_title
Statistical methods in medical researchauthors
Geraci Mdoi
10.1177/0962280213484401subject
Has Abstractpub_date
2016-08-01 00:00:00pages
1393-421issue
4eissn
0962-2802issn
1477-0334pii
0962280213484401journal_volume
25pub_type
杂志文章abstract::We often seek to estimate the impact of an exposure naturally occurring or randomly assigned at the cluster-level. For example, the literature on neighborhood determinants of health continues to grow. Likewise, community randomized trials are applied to learn about real-world implementation, sustainability, and popula...
journal_title:Statistical methods in medical research
pub_type: 杂志文章
doi:10.1177/0962280218774936
更新日期:2019-06-01 00:00:00
abstract::Clinical trials are expensive and time-consuming and so should also be used to study how treatments work, allowing for the evaluation of theoretical treatment models and refinement and improvement of treatments. These treatment processes can be studied using mediation analysis. Randomised treatment makes some of the a...
journal_title:Statistical methods in medical research
pub_type: 杂志文章
doi:10.1177/0962280216666111
更新日期:2018-06-01 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
pub_type: 杂志文章
doi:10.1177/0962280220937324
更新日期:2020-12-01 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
pub_type: 杂志文章
doi:10.1177/0962280215598806
更新日期:2017-08-01 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
pub_type: 杂志文章
doi:10.1177/0962280217734583
更新日期:2019-03-01 00:00:00
abstract::In many longitudinal studies, evaluating the effect of a binary or continuous predictor variable on the rate of change of the outcome, i.e. slope, is often of primary interest. Sample size determination of these studies, however, is complicated by the expectation that missing data will occur due to missed visits, earl...
journal_title:Statistical methods in medical research
pub_type: 杂志文章
doi:10.1177/0962280212437452
更新日期:2015-12-01 00:00:00
abstract::Most diagnostic accuracy measures and criteria for selecting optimal cut-points are only applicable to diseases with binary or three stages. Currently, there exist two diagnostic measures for diseases with general k stages: the hypervolume under the manifold and the generalized Youden index. While hypervolume under th...
journal_title:Statistical methods in medical research
pub_type: 杂志文章
doi:10.1177/0962280215611631
更新日期:2017-12-01 00:00:00
abstract::The ability to evaluate effects of factors on outcomes is increasingly important for studies that control some but not all of the factors. Although important advances have been made in methods of analysis for such partially controlled studies, work on designs has been limited. To help understand why, we review the mai...
journal_title:Statistical methods in medical research
pub_type: 杂志文章
doi:10.1191/0962280205sm405oa
更新日期:2005-08-01 00:00:00
abstract::The simple yet subtle concept of regression towards the mean is reviewed historically. Verbal, geometric, and mathematical expressions of the concept date to the discoverer of the concept, Francis Galton. That discovery and subsequent understanding (and misunderstanding) of the concept are surveyed. ...
journal_title:Statistical methods in medical research
pub_type: 传,历史文章,杂志文章,评审
doi:10.1177/096228029700600202
更新日期:1997-06-01 00:00:00
abstract::Comparison of sequences that have descended from a common ancestor based on an explicit stochastic model of substitutions, insertions and deletions has risen to prominence in the last decade. Making statements about the positions of insertions-deletions (abbr. indels) is central in sequence and genome analysis and is ...
journal_title:Statistical methods in medical research
pub_type: 杂志文章
doi:10.1177/0962280208099500
更新日期:2009-10-01 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
pub_type: 杂志文章
doi:10.1177/0962280219851817
更新日期:2020-04-01 00:00:00
abstract:BACKGROUND:Dyspepsia diagnoses and treatment decisions are made in situations in which multiple factors must be taken into account. Evolving from neuro-biological insights, artificial neural networks (ANNs) can employ multiple factors in resolving medical prediction, classification, pattern recognition, and pattern com...
journal_title:Statistical methods in medical research
pub_type: 杂志文章
doi:10.1177/0962280206071839
更新日期:2007-08-01 00:00:00
abstract::This paper focuses on inferential tools in the logistic regression model fitted by the Firth penalized likelihood. In this context, the Likelihood Ratio statistic is often reported to be the preferred choice as compared to the 'traditional' Wald statistic. In this work, we consider and discuss a wider range of test st...
journal_title:Statistical methods in medical research
pub_type: 杂志文章
doi:10.1177/0962280216661213
更新日期:2018-05-01 00:00:00
abstract::One of the main advantages of Bayesian analyses of clinical trials is their ability to formally incorporate skepticism about large treatment effects through the use of informative priors. We conducted a simulation study to assess the performance of informative normal, Student- t, and beta distributions in estimating r...
journal_title:Statistical methods in medical research
pub_type: 杂志文章
doi:10.1177/0962280215620828
更新日期:2018-01-01 00:00:00
abstract::Optimal therapeutic decisions can be made according to disease prognosis, where the residual lifetime is extensively used because of its straightforward interpretation and formula. To predict the residual lifetime in a dynamic manner, a longitudinal biomarker that is repeatedly measured during the post-baseline follow...
journal_title:Statistical methods in medical research
pub_type: 杂志文章
doi:10.1177/0962280217753466
更新日期:2019-04-01 00:00:00
abstract::Many non-experimental studies use propensity-score methods to estimate causal effects by balancing treatment and control groups on a set of observed baseline covariates. Full matching on the propensity score has emerged as a particularly effective and flexible method for utilizing all available data, and creating well...
journal_title:Statistical methods in medical research
pub_type: 杂志文章
doi:10.1177/0962280215601134
更新日期:2017-12-01 00:00:00
abstract::Simple mechanistic epidemic models are widely used for forecasting and parameter estimation of infectious diseases based on noisy case reporting data. Despite the widespread application of models to emerging infectious diseases, we know little about the comparative performance of standard computational-statistical fra...
journal_title:Statistical methods in medical research
pub_type: 杂志文章
doi:10.1177/0962280217747054
更新日期:2018-07-01 00:00:00
abstract::The threat of pandemics has made influenza surveillance systems a priority in epidemiology services around the world. The emergence of A-H1N1 influenza has required accurate surveillance systems in order to undertake specific actions only when and where they are necessary. In that sense, the main goal of this article ...
journal_title:Statistical methods in medical research
pub_type: 杂志文章
doi:10.1177/0962280210370265
更新日期:2011-04-01 00:00:00
abstract::Semicompeting risks and interval censoring are frequent in medical studies, for instance when a disease may be diagnosed only at times of visit and disease onset is in competition with death. To evaluate the ability of markers to predict disease onset in this context, estimators of discrimination measures must account...
journal_title:Statistical methods in medical research
pub_type: 杂志文章
doi:10.1177/0962280214531691
更新日期:2016-12-01 00:00:00
abstract::We propose a semiparametric multi-state frailty model to analyze clustered event-history data subject to interval censoring. The proposed model is motivated by an attempt to study the life course of dental caries at the tooth level, taking into account the multiplicity of caries states and the intra-oral clustering of...
journal_title:Statistical methods in medical research
pub_type: 杂志文章
doi:10.1177/0962280218788383
更新日期:2019-09-01 00:00:00
abstract::Measurement error is a serious problem in the analysis of epidemiological data. In the past 20 years, a large number of methods for the correction of measurement error have been developed. While at the beginning mostly methods for cohort studies were considered, recently more attention has been paid to case-control st...
journal_title:Statistical methods in medical research
pub_type: 杂志文章,评审
doi:10.1177/096228020000900504
更新日期:2000-10-01 00:00:00
abstract::This article aims to develop a probability-based model involving the use of direct likelihood formulation and generalised linear modelling (GLM) approaches useful in estimating important disease parameters from longitudinal or repeated measurement data. The current application is based on infection with respiratory sy...
journal_title:Statistical methods in medical research
pub_type: 杂志文章
doi:10.1177/0962280210385749
更新日期:2011-10-01 00:00:00
abstract::Recently, the joint analysis of longitudinal and survival data has been an active research area. Most joint models focus on survival data with only one type of failure. The research on joint modeling of longitudinal and competing risks survival data is sparse. Even so, many joint models for this type of data assume pa...
journal_title:Statistical methods in medical research
pub_type: 杂志文章
doi:10.1177/0962280215597939
更新日期:2017-10-01 00:00:00
abstract::Pediatric cardiac surgery may lead to poor outcomes such as acute kidney injury (AKI) and prolonged hospital length of stay (LOS). Plasma and urine biomarkers may help with early identification and prediction of these adverse clinical outcomes. In a recent multi-center study, 311 children undergoing cardiac surgery we...
journal_title:Statistical methods in medical research
pub_type: 杂志文章
doi:10.1177/0962280214530608
更新日期:2016-12-01 00:00:00
abstract::Repeated measures and multivariate outcomes are an increasingly common feature of trials. Their joint analysis by means of random effects and latent variable models is appealing but patterns of heterogeneity in outcome profile may not conform to standard multivariate normal assumptions. In addition, there is much inte...
journal_title:Statistical methods in medical research
pub_type: 杂志文章,评审
doi:10.1177/0962280209105016
更新日期:2010-06-01 00:00:00
abstract::In biomedical research, various longitudinal markers measuring different quantities are often collected over time. For example, repeated measures of psychometric scores are very informative about the degradation process toward dementia. These trajectories are generally nonlinear with an acceleration of the decline a f...
journal_title:Statistical methods in medical research
pub_type: 杂志文章
doi:10.1177/0962280219898719
更新日期:2020-09-01 00:00:00
abstract::Dependent censoring arises in biomedical studies when the survival outcome of interest is censored by competing risks. In survival data with microarray gene expressions, gene selection based on the univariate Cox regression analyses has been used extensively in medical research, which however, is only valid under the ...
journal_title:Statistical methods in medical research
pub_type: 杂志文章
doi:10.1177/0962280214533378
更新日期:2016-12-01 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
pub_type: 杂志文章,评审
doi:10.1177/096228029900800105
更新日期:1999-03-01 00:00:00
abstract::Most statistical developments in the joint modelling area have focused on the shared random-effect models that include characteristics of the longitudinal marker as predictors in the model for the time-to-event. A less well-known approach is the joint latent class model which consists in assuming that a latent class s...
journal_title:Statistical methods in medical research
pub_type: 杂志文章,评审
doi:10.1177/0962280212445839
更新日期:2014-02-01 00:00:00
abstract::Non-parametric linkage analysis examines similarities among affected relatives in alleles of one or more genetic markers (pieces of DNA at known locations on a chromosome). The objective is to evaluate departures from the null hypothesis that the markers are not near a disease gene. Under the null hypothesis, Mendel's...
journal_title:Statistical methods in medical research
pub_type: 杂志文章,评审
doi:10.1177/096228020101000103
更新日期:2001-02-01 00:00:00