Abstract:
:Using both simulated and real datasets, we compared two approaches for estimating absolute risk from nested case-control (NCC) data and demonstrated the feasibility of using the NCC design for estimating absolute risk. In contrast to previously published results, we successfully demonstrated not only that data from a matched NCC study can be used to unbiasedly estimate absolute risk but also that matched studies give better statistical efficiency and classify subjects into more appropriate risk categories. Our result has implications for studies that aim to develop or validate risk prediction models. In addition to the traditional full cohort study and case-cohort study, researchers designing these studies now have the option of performing a NCC study with huge potential savings in cost and resources. Detailed explanations on how to obtain the absolute risk estimates under the proposed approach are given. Copyright © 2016 John Wiley & Sons, Ltd.
journal_name
Stat Medjournal_title
Statistics in medicineauthors
Salim A,Delcoigne B,Villaflores K,Koh WP,Yuan JM,van Dam RM,Reilly Mdoi
10.1002/sim.7143subject
Has Abstractpub_date
2017-02-10 00:00:00pages
455-465issue
3eissn
0277-6715issn
1097-0258journal_volume
36pub_type
杂志文章abstract::I derive the locally most powerful rank tests for acceleration against semi-parametric alternatives when some patients are cured of the disease. I consider some particular classes of alternatives and present simulation results to verify the validity of the proposed tests. Real data from clinical trials for childhood l...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.4780141905
更新日期:1995-10-15 00:00:00
abstract::Recent studies have indicated variation in the infectivity beta of HIV among heterosexual couples. We represent this heterogeneity by modelling beta as a random variable. Using data on the number of contacts and seroconversion of couples, we fit the model by maximum-likelihood estimation with a beta distribution and a...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.4780080110
更新日期:1989-01-01 00:00:00
abstract::The National Children's Study is a national household probability sample designed to identify 100,000 children at birth and follow the sampled children for 21 years. Data from the study will support examining numerous hypotheses concerning genetic and environmental effects on the health and development of children. Th...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.3891
更新日期:2010-06-15 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::Omitted variable bias is discussed in the context of linear models. It is shown that the effect of omitted variables can be controlled in linear models for metric dependent variables by using data from follow-up studies. Two different models for analysing such data are proposed. In the first model the omitted variable...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.4780110906
更新日期:1992-06-30 00:00:00
abstract::The risk that a drug newly introduced into medical use will occasionally cause adverse reactions is neither negligible nor totally avoidable. Only well organized systems of monitoring can bring early detection and appropriate action. These in turn require either detailed supervision or spontaneous reporting. The paper...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.4780010208
更新日期:1982-04-01 00:00:00
abstract::We consider the estimation of parameters in a particular segmented generalized linear model with additive measurement error in predictors, with a focus on linear and logistic regression. In epidemiologic studies segmented regression models often occur as threshold models, where it is assumed that the exposure has no i...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/(sici)1097-0258(19970130)16:2<169::aid-sim
更新日期:1997-01-15 00:00:00
abstract::We consider counting process methods for analysing time-to-event data with multiple or recurrent outcomes, using the models developed by Anderson and Gill, Wei, Lin and Weissfeld and Prentice, Williams and Peterson. We compare the methods, and show how to implement them using popular statistical software programs. By ...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/(sici)1097-0258(19970930)16:18<2029::aid-s
更新日期:1997-09-30 00:00:00
abstract::Although radical changes in drug regulation are rare (e.g., the Federal Food, Drug and Cosmetic Act of 1938 and the 1962 amendment to the Act creating an effectiveness requirement), regulations and guidance do evolve significantly in the face of new problems and accumulating experience. Recent changes have been driven...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.1298
更新日期:2002-10-15 00:00:00
abstract::In this paper, an approach to estimating the cumulative mean function for history process with time dependent covariates and right censored time-to-event variable is developed using the combined technique of joint modeling and inverse probability weighting method. The consistency of proposed estimator is derived. Theo...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.6998
更新日期:2016-11-10 00:00:00
abstract::Prevention studies, as distinguished from studies investigating treatments for established disease, present some distinct challenges. Perhaps the most extensive experience with preventive agents is in the area of infectious diseases; vaccines have been extremely effective in preventing many such diseases. Vaccines hav...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.1717
更新日期:2004-01-30 00:00:00
abstract::We use a mixed Poisson regression model with extra variation to analyse mortality data cross-classified by age and geographic region. We use estimates of dispersion parameter and fixed effects parameters, obtained by maximizing a marginal quasi-likelihood function, to estimate mortality rates in an empirical Bayes man...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/(SICI)1097-0258(19960715)15:13<1397::AID-S
更新日期:1996-07-15 00:00:00
abstract::Case-control studies are usually defined to investigate risk factors for a single disease of interest. However, subsequent to data collection, investigators may wish to examine as an 'outcome' a variable that was an exposure in the original study. A naive analysis that disregards the sampling strategy that gave rise t...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.2398
更新日期:2005-12-30 00:00:00
abstract::Technologic advances give rise to new tests for detecting disease in many fields, including cancer and sexually transmitted disease. Before a new disease screening test is approved for public use, its accuracy should be shown to be better than or at least not inferior to an existing test. Standards do not yet exist fo...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.1058
更新日期:2002-03-30 00:00:00
abstract::Many epidemiologic investigations are designed to study the effects of multiple exposures. Most of these studies are analysed either by fitting a risk-regression model with all exposures forced in the model, or by using a preliminary-testing algorithm, such as stepwise regression, to produce a smaller model. Research ...
journal_title:Statistics in medicine
pub_type: 杂志文章,评审
doi:10.1002/sim.4780120802
更新日期:1993-04-30 00:00:00
abstract::In many medical applications, combining information from multiple biomarkers could yield a better diagnosis than any single one on its own. When there is a lack of a gold standard, an algorithm of classifying subjects into the case and non-case status is necessary for combining multiple markers. The aim of this paper ...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.6780
更新日期:2016-04-15 00:00:00
abstract::One Sentence Summary: A Bayesian repeated measures model based on quantitative muscle strength data from a prospective Natural History Study was developed to determine disease progression and design clinical trials for GNE myopathy, a rare and slowly progressive muscle disease. GNE myopathy is a rare muscle disease ch...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.8050
更新日期:2019-04-15 00:00:00
abstract::In medical research, a two-phase study is often used for the estimation of the area under the receiver operating characteristic curve (AUC) of a diagnostic test. However, such a design introduces verification bias. One of the methods to correct verification bias is inverse probability weighting (IPW). Since the probab...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.8700
更新日期:2020-11-30 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::A model is developed to estimate the effect of covariate imbalance on the size of a test of treatment efficacy in randomized clinical trials comparing two treatments when dispersion parameters are known. It is concluded that tests of homogeneity on the covariates should not be performed, that covariate imbalance is ju...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.4780080410
更新日期:1989-04-01 00:00:00
abstract::We consider the use of the assurance method in clinical trial planning. In the assurance method, which is an alternative to a power calculation, we calculate the probability of a clinical trial resulting in a successful outcome, via eliciting a prior probability distribution about the relevant treatment effect. This i...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.5916
更新日期:2014-01-15 00:00:00
abstract::A survey is conducted at w of K selection units or lists, e.g. health care institutions or weeks in a year, to estimate N, the total number of individuals with particular characteristics. Our estimator utilizes two items determined for each survey participant: the number, u, among the w lists in S and the number, j, a...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.3614
更新日期:2009-07-30 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::When sample size is recalculated using unblinded interim data, use of the usual t-test at the end of a study may lead to an elevated type I error rate. This paper describes a numerical quadrature investigation to calculate the true probability of rejection as a function of the time of the recalculation, the magnitude ...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/(sici)1097-0258(19991230)18:24<3481::aid-s
更新日期:1999-12-30 00:00:00
abstract::In time-to-event analysis, the traditional summary measures have been based on the hazard function, survival function, quantile event time, restricted mean event time, and residual lifetime. Under competing risks, furthermore, typical summary measures have been the cause-specific hazard function and cumulative inciden...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.8871
更新日期:2021-01-06 00:00:00
abstract::There is considerable public concern about health disparities among different cultural/racial/ethnic groups. Important process measures that might reflect inequities are treated prevalence and the service utilization rate in a defined period of time. We have previously described a method for estimating N, the distinct...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.3904
更新日期:2010-07-20 00:00:00
abstract::For many HIV-infected patients, use of antiretroviral therapy (ART) results in a sustained suppression of plasma viral load to undetectable levels. However, due to lack of antigenic stimulation, this may also result in a gradual loss of cell-mediated immune (CMI) responses that help control HIV infection. In concept, ...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.2555
更新日期:2007-02-10 00:00:00
abstract::The focus of this paper is dietary intervention trials. We explore the statistical issues involved when the response variable, intake of a food or nutrient, is based on self-report data that are subject to inherent measurement error. There has been little work on handling error in this context. A particular feature of...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.7011
更新日期:2016-11-10 00:00:00
abstract::In conventional survival analysis there is an underlying assumption that all study subjects are susceptible to the event. In general, this assumption does not adequately hold when investigating the time to an event other than death. Owing to genetic and/or environmental etiology, study subjects may not be susceptible ...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.5845
更新日期:2013-10-30 00:00:00
abstract::A number of variance formulae for the attributable fraction have been presented, but none is consistent in sparse data, such as found in individually matched case-control studies. This paper employs Mantel-Haenszel estimation to derive variance estimators for attributable fractions that are dually consistent, that is,...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.4780060607
更新日期:1987-09-01 00:00:00