Analysis of the ratio of marginal probabilities in a matched-pair setting.

Abstract:

:Statistical methods for testing and interval estimation of the ratio of marginal probabilities in the matched-pair setting are considered in this paper. We are especially interested in the situation where the null value is not one, as in one-sided equivalence trials. We propose a Fieller-type statistic based on constrained maximum likelihood (CML) estimation of nuisance parameters. For a series of examples, the significance level of the CML test is satisfactorily close to the nominal level, while a Wald-type test is anticonservative for reasonable sample sizes. We present formulae for approximate power and sample size for the CML and Wald tests. The matched design is seen to have a clear advantage over the unmatched design in terms of asymptotic efficiency when the two responses of the pair are highly positively correlated. We recommend the CML method over the Wald method, especially for small or moderate sample sizes.

journal_name

Stat Med

journal_title

Statistics in medicine

authors

Nam JM,Blackwelder WC

doi

10.1002/sim.1017

subject

Has Abstract

pub_date

2002-03-15 00:00:00

pages

689-99

issue

5

eissn

0277-6715

issn

1097-0258

pii

10.1002/sim.1017

journal_volume

21

pub_type

杂志文章
  • A unified inference procedure for a class of measures to assess improvement in risk prediction systems with survival data.

    abstract::Risk prediction procedures can be quite useful for the patient's treatment selection, prevention strategy, or disease management in evidence-based medicine. Often, potentially important new predictors are available in addition to the conventional markers. The question is how to quantify the improvement from the new ma...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.5647

    authors: Uno H,Tian L,Cai T,Kohane IS,Wei LJ

    更新日期:2013-06-30 00:00:00

  • Adjusted Kaplan-Meier estimator and log-rank test with inverse probability of treatment weighting for survival data.

    abstract::Estimation and group comparison of survival curves are two very common issues in survival analysis. In practice, the Kaplan-Meier estimates of survival functions may be biased due to unbalanced distribution of confounders. Here we develop an adjusted Kaplan-Meier estimator (AKME) to reduce confounding effects using in...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.2174

    authors: Xie J,Liu C

    更新日期:2005-10-30 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

  • Comparison of methods for the analysis of longitudinal interval count data.

    abstract::Longitudinal studies are often concerned with estimating the recurrence rate of a non-fatal event. In many cases, only the total number of events occurring during successive time intervals is known. We compared a mixed Poisson-gamma regression method proposed by Thall and a quasi-likelihood method proposed by Zeger an...

    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.4780121406

    authors: Stukel TA

    更新日期:1993-07-30 00:00:00

  • Stochastic approximation EM for large-scale exploratory IRT factor analysis.

    abstract::A stochastic approximation EM algorithm (SAEM) is described for exploratory factor analysis of dichotomous or ordinal variables. The factor structure is obtained from sufficient statistics that are updated during iterations with the Robbins-Monro procedure. Two large-scale simulations are reported that compare accurac...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.8217

    authors: Camilli G,Geis E

    更新日期:2019-09-20 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

  • Prognostic modelling with logistic regression analysis: a comparison of selection and estimation methods in small data sets.

    abstract::Logistic regression analysis may well be used to develop a prognostic model for a dichotomous outcome. Especially when limited data are available, it is difficult to determine an appropriate selection of covariables for inclusion in such models. Also, predictions may be improved by applying some sort of shrinkage in t...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/(sici)1097-0258(20000430)19:8<1059::aid-si

    authors: Steyerberg EW,Eijkemans MJ,Harrell FE Jr,Habbema JD

    更新日期:2000-04-30 00:00:00

  • A large class of models derived from generalized linear models.

    abstract::Generalized linear models may be extended in several ways. This paper describes five such extensions: (i) generalized additive models; (ii) the use of quasi-likelihood; (iii) joint modelling of mean and dispersion; (iv) introduction of extra random components to give hierarchical generalized linear models; (v) modelli...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/(sici)1097-0258(19981215)17:23<2747::aid-s

    authors: Nelder JA

    更新日期:1998-12-15 00:00:00

  • Regression analysis applied to PVC histories: a statistical procedure for evaluating antiarrhythmic drug efficacy.

    abstract::Suppression of premature ventricular contractions (PVCs) is one of the goals of antiarrhythmic therapy. In a clinical trial, however, it may be difficult to distinguish antiarrhythmic drug effect from spontaneous variation in PVCs. We propose the application of linear regression to PVC histories to ascertain drug effe...

    journal_title:Statistics in medicine

    pub_type: 临床试验,杂志文章

    doi:10.1002/sim.4780020305

    authors: Berry DA,Fox TL

    更新日期:1983-07-01 00:00:00

  • Formulae and tables for the determination of sample sizes and power in clinical trials for testing differences in proportions for the two-sample design: a review.

    abstract::This paper is a compendium of exact and asymptotic formulae and tables for estimating the sample size in a clinical trial with two treatment groups and a dichotomous outcome. The paper provides separate formulae for equal and unequal treatment group sizes, formulae for the calculation of power given the sample size, a...

    journal_title:Statistics in medicine

    pub_type: 杂志文章,评审

    doi:10.1002/(SICI)1097-0258(19960115)15:1<1::AID-SIM13

    authors: Sahai H,Khurshid A

    更新日期:1996-01-15 00:00:00

  • Network-based regularization for matched case-control analysis of high-dimensional DNA methylation data.

    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

    authors: Sun H,Wang S

    更新日期:2013-05-30 00:00:00

  • Group sequential large sample T2-like chi2 tests for multivariate observations.

    abstract::In many studies, a K degree of freedom large sample chi2 test is used to assess the effect of treatment on a multivariate response, such as an omnibus T2-like test of a difference between two treatment groups in any of K repeated measures. Alternately, a K df chi2 test may be used to test the equality of K+1 groups in...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.1637

    authors: Lachin JM,Greenhouse SW,Bautista OM

    更新日期:2003-11-15 00:00:00

  • Probabilistic cause-of-disease assignment using case-control diagnostic tests: A latent variable regression approach.

    abstract::Optimal prevention and treatment strategies for a disease of multiple causes, such as pneumonia, must be informed by the population distribution of causes among cases, or cause-specific case fractions (CSCFs). CSCFs may further depend on additional explanatory variables. Existing methodological literature in disease e...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.8804

    authors: Wu Z,Chen I

    更新日期:2021-02-20 00:00:00

  • Comparison of methods for imputing ordinal data using multivariate normal imputation: a case study of non-linear effects in a large cohort study.

    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

    authors: Lee KJ,Galati JC,Simpson JA,Carlin JB

    更新日期:2012-12-30 00:00:00

  • Extensions of net reclassification improvement calculations to measure usefulness of new biomarkers.

    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

    authors: Pencina MJ,D'Agostino RB Sr,Steyerberg EW

    更新日期:2011-01-15 00:00:00

  • Dynamic clustering detection through multi-valued descriptors of dermoscopic images.

    abstract::This paper introduces a dynamic clustering methodology based on multi-valued descriptors of dermoscopic images. The main idea is to support medical diagnosis to decide if pigmented skin lesions belonging to an uncertain set are nearer to malignant melanoma or to benign nevi. Melanoma is the most deadly skin cancer, an...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.4285

    authors: Cozza V,Guarracino MR,Maddalena L,Baroni A

    更新日期:2011-09-10 00:00:00

  • Modification of the sample size and the schedule of interim analyses in survival trials based on data inspections.

    abstract::A method is presented which allows us to adapt the sample size as well as the number and time points of interim analyses to the treatment difference observed at an interim look during the course of a clinical trial with censored survival time as the endpoint. The method allows the inclusion of data inspections during ...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.1136

    authors: Schäfer H,Müller HH

    更新日期:2001-12-30 00:00:00

  • Drug treatment of mild hypertension to reduce the risk of CHD: is it worth-while?

    abstract::Although hypertension is regarded as a causal factor for coronary heart disease (CHD) a reduction in the risk of CHD as a result of lowering blood pressure in mild hypertension could not be demonstrated. This conclusion is based on an overview analysis of all published randomized trials in mild hypertension, including...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.4780071104

    authors: Holme I

    更新日期:1988-11-01 00:00:00

  • Measuring spatial effects in time to event data: a case study using months from angiography to coronary artery bypass graft (CABG).

    abstract::The application of Bayesian hierarchical models to measure spatial effects in time to event data has not been widely reported. This case study aims to estimate the effect of area of residence on waiting times to coronary artery bypass graft (CABG) and to assess the role of important individual specific covariates (age...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.1535

    authors: Crook AM,Knorr-Held L,Hemingway H

    更新日期:2003-09-30 00:00:00

  • The analysis of continuous outcomes in multi-centre trials with small centre sizes.

    abstract::The standard analysis of clinical trials stratified by centre is to include centres as fixed effects, but if many centres contribute small numbers of patients, this approach results in a loss of power. Assuming no treatment by centre interaction, we used simulation to examine power and coverage of confidence intervals...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.3068

    authors: Pickering RM,Weatherall M

    更新日期:2007-12-30 00:00:00

  • Assessing local influence in principal component analysis with application to haematology study data.

    abstract::In many medical and health studies, high-dimensional data are often encountered. Principal component analysis (PCA) is a commonly used technique to reduce such data to a few components that includes most of the information provided by the original data. However, PCA is known to be very sensitive to some abnormal obser...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.2747

    authors: Fung WK,Gu H,Xiang L,Yau KK

    更新日期:2007-06-15 00:00:00

  • Multivariate test power approximations for balanced linear mixed models in studies with missing data.

    abstract::Multilevel and longitudinal studies are frequently subject to missing data. For example, biomarker studies for oral cancer may involve multiple assays for each participant. Assays may fail, resulting in missing data values that can be assumed to be missing completely at random. Catellier and Muller proposed a data ana...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.6811

    authors: Ringham BM,Kreidler SM,Muller KE,Glueck DH

    更新日期:2016-07-30 00:00:00

  • Measuring vaccine efficacy from epidemics of acute infectious agents.

    abstract::A good measure of field vaccine efficacy should evaluate the direct protective effect of vaccination on the person who receives the vaccine. The conventional estimator for vaccine efficacy depends on population level factors that are either unrelated or indirectly related to the direct biological action of the vaccine...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.4780120309

    authors: Longini IM Jr,Halloran ME,Haber M,Chen RT

    更新日期:1993-02-01 00:00:00

  • Statistical inferences of extended concentration indices for directly standardized rates.

    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

    authors: Yu M,Liu B,Li Y,Zou ZJ,Breen N

    更新日期:2019-01-15 00:00:00

  • Design evaluation and optimisation in crossover pharmacokinetic studies analysed by nonlinear mixed effects models.

    abstract::Bioequivalence or interaction trials are commonly studied in crossover design and can be analysed by nonlinear mixed effects models as an alternative to noncompartmental approach. We propose an extension of the population Fisher information matrix in nonlinear mixed effects models to design crossover pharmacokinetic t...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.4390

    authors: Nguyen TT,Bazzoli C,Mentré F

    更新日期:2012-05-20 00:00:00

  • Randomization tests for multiarmed randomized clinical trials.

    abstract::We examine the use of randomization-based inference for analyzing multiarmed randomized clinical trials, including the application of conditional randomization tests to multiple comparisons. The view is taken that the linkage of the statistical test to the experimental design (randomization procedure) should be recogn...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.8418

    authors: Wang Y,Rosenberger WF,Uschner D

    更新日期:2020-02-20 00:00:00

  • Power and sample size calculation for log-rank test with a time lag in treatment effect.

    abstract::The log-rank test is the most powerful non-parametric test for detecting a proportional hazards alternative and thus is the most commonly used testing procedure for comparing time-to-event distributions between different treatments in clinical trials. When the log-rank test is used for the primary data analysis, the s...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.3501

    authors: Zhang D,Quan H

    更新日期:2009-02-28 00:00:00

  • Estimation of the time-dependent vaccine efficacy from a measles epidemic.

    abstract::We present a method to estimate the time-dependent vaccine efficacy from the cohort-specific vaccination coverage and from data on the vaccination status of cases and apply it to a measles epidemic in Germany which involved 529 cases, 88 of whom were vaccinated and 370 unvaccinated (for the remaining 71 cases the vacc...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.1043

    authors: Eichner M,Diebner HH,Schubert C,Kreth HW,Dietz K

    更新日期:2002-08-30 00:00:00

  • Automated time series forecasting for biosurveillance.

    abstract::For robust detection performance, traditional control chart monitoring for biosurveillance is based on input data free of trends, day-of-week effects, and other systematic behaviour. Time series forecasting methods may be used to remove this behaviour by subtracting forecasts from observations to form residuals for al...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.2835

    authors: Burkom HS,Murphy SP,Shmueli G

    更新日期:2007-09-30 00:00:00

  • Methods for comparing cumulative hazard functions in a semi-proportional hazard model.

    abstract::Graphical methods based on the analysis of differences between log cumulative hazard functions are considered for a two-group semi-proportional hazard model which allows for interaction between treatments and covariates. Confidence procedures and test statistics that can be used to test for interaction and for main ef...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.4780111105

    authors: Dabrowska DM,Doksum KA,Feduska NJ,Husing R,Neville P

    更新日期:1992-08-01 00:00:00