Comparisons of risk prediction methods using nested case-control data.

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 Med

journal_title

Statistics in medicine

authors

Salim A,Delcoigne B,Villaflores K,Koh WP,Yuan JM,van Dam RM,Reilly M

doi

10.1002/sim.7143

subject

Has Abstract

pub_date

2017-02-10 00:00:00

pages

455-465

issue

3

eissn

0277-6715

issn

1097-0258

journal_volume

36

pub_type

杂志文章
  • Two-sample rank tests for acceleration in cure models.

    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

    authors: Lee JW

    更新日期:1995-10-15 00:00:00

  • Heterogeneity in the probability of HIV transmission per sexual contact: the case of male-to-female transmission in penile-vaginal intercourse.

    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

    authors: Wiley JA,Herschkorn SJ,Padian NS

    更新日期:1989-01-01 00:00:00

  • Statistical and practical issues in the design of a national probability sample of births for the Vanguard Study of the National Children's Study.

    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

    authors: Montaquila JM,Brick JM,Curtin LR

    更新日期:2010-06-15 00:00:00

  • Development and applications of a city-level alcohol availability and alcohol problems database.

    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

    authors: MacKinnon DP,Scribner R,Taft KA

    更新日期:1995-03-15 00:00:00

  • Using follow-up data to avoid omitted variable bias: an application to cardiovascular epidemiology.

    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

    authors: Rehm J,Arminger G,Kohlmeier L

    更新日期:1992-06-30 00:00:00

  • The detection of adverse reactions to therapeutic drugs.

    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

    authors: Finney DJ

    更新日期:1982-04-01 00:00:00

  • Segmented regression with errors in predictors: semi-parametric and parametric methods.

    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

    authors: Küchenhoff H,Carroll RJ

    更新日期:1997-01-15 00:00:00

  • rhDNase as an example of recurrent event analysis.

    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

    authors: Therneau TM,Hamilton SA

    更新日期:1997-09-30 00:00:00

  • Policy developments in regulatory approval.

    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

    authors: Temple R

    更新日期:2002-10-15 00:00:00

  • Estimating the cumulative mean function for history process with time-dependent covariates and censoring mechanism.

    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

    authors: Deng D

    更新日期:2016-11-10 00:00:00

  • Analytical, practical and regulatory issues in prevention studies.

    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

    authors: Ellenberg SS

    更新日期:2004-01-30 00:00:00

  • Analysis of mortality rates via marginal extended quasi-likelihood.

    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

    authors: Lu WS,Tsutakawa RK

    更新日期:1996-07-15 00:00:00

  • Re-use of case-control data for analysis of new outcome variables.

    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

    authors: Reilly M,Torrång A,Klint A

    更新日期:2005-12-30 00:00:00

  • Sample size calculations for comparative studies of medical tests for detecting presence of disease.

    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

    authors: Alonzo TA,Pepe MS,Moskowitz CS

    更新日期:2002-03-30 00:00:00

  • Methods for epidemiologic analyses of multiple exposures: a review and comparative study of maximum-likelihood, preliminary-testing, and empirical-Bayes regression.

    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

    authors: Greenland S

    更新日期:1993-04-30 00:00:00

  • A sequential classification rule based on multiple quantitative tests in the absence of a gold standard.

    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

    authors: Zhang J,Zhang Y,Chaloner K,Stapleton JT

    更新日期:2016-04-15 00:00:00

  • Bayesian model of disease progression in GNE myopathy.

    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

    authors: Quintana M,Shrader J,Slota C,Joe G,McKew JC,Fitzgerald M,Gahl WA,Berry S,Carrillo N

    更新日期:2019-04-15 00:00:00

  • True verification probabilities should not be used in estimating the area under receiver operating characteristic curve.

    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

    authors: Wu Y

    更新日期:2020-11-30 00:00:00

  • Estimation of infection prevalence and sensitivity in a stratified two-stage sampling design employing highly specific diagnostic tests when there is no gold standard.

    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

    authors: Miller E,Huppert A,Novikov I,Warburg A,Hailu A,Abbasi I,Freedman LS

    更新日期:2015-11-10 00:00:00

  • Covariate imbalance and random allocation in clinical trials.

    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

    authors: Senn SJ

    更新日期:1989-04-01 00:00:00

  • Assurance calculations for planning clinical trials with time-to-event outcomes.

    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

    authors: Ren S,Oakley JE

    更新日期:2014-01-15 00:00:00

  • Model-based multiplicity estimation of population size.

    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

    authors: Laska EM,Meisner M,Wanderling J

    更新日期:2009-07-30 00:00:00

  • Determining the value of additional surrogate exposure data for improving the estimate of an odds ratio.

    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

    authors: Dahm PF,Gail MH,Rosenberg PS,Pee D

    更新日期:1995-12-15 00:00:00

  • Internal pilot studies I: type I error rate of the naive t-test.

    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

    authors: Wittes J,Schabenberger O,Zucker D,Brittain E,Proschan M

    更新日期:1999-12-30 00:00:00

  • Cause-specific quantile regression on inactivity time.

    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

    authors: Jia Y,Jeong JH

    更新日期:2021-01-06 00:00:00

  • Estimating treated prevalence and service utilization rates: assessing disparities in mental health.

    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

    authors: Laska EM,Meisner M,Wanderling J,Siegel C

    更新日期:2010-07-20 00:00:00

  • Analysis of antiretroviral immunotherapy trials with potentially non-normal and incomplete longitudinal data.

    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

    authors: Mogg R,Mehrotra DV

    更新日期:2007-02-10 00:00:00

  • Statistical issues related to dietary intake as the response variable in intervention trials.

    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

    authors: Keogh RH,Carroll RJ,Tooze JA,Kirkpatrick SI,Freedman LS

    更新日期:2016-11-10 00:00:00

  • Logistic-AFT location-scale mixture regression models with nonsusceptibility for left-truncated and general interval-censored data.

    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

    authors: Chen CH,Tsay YC,Wu YC,Horng CF

    更新日期:2013-10-30 00:00:00

  • Variance estimators for attributable fraction estimates consistent in both large strata and sparse data.

    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

    authors: Greenland S

    更新日期:1987-09-01 00:00:00