A simple test for synergy for a small number of combinations.

abstract：:Small but important therapeutic effects of new treatments can be most efficiently detected through the study of large randomized prospective series of patients. Such large scale clinical trials are nowadays commonplace. The alternative is years of polemic and debate surrounding several trials each too small to detect ...

journal_title：Statistics in medicine

authors： McPherson K

更新日期：1982-01-01 00:00:00

abstract：:Orthogonal polynomial scores (OPS) is a simple, biologically meaningful approach to characterize longitudinal data in phase I and II clinical pharmacology trials. It describes average, linear, quadratic and higher order polynomial characteristics of each subject's response over time with use of composite scores comput...

journal_title：Statistics in medicine

authors： Bradstreet TE

更新日期：1993-04-15 00:00:00

abstract：:In record linkage studies, unique identifiers are often not available, and therefore, the linkage procedure depends on combinations of partially identifying variables with low discriminating power. As a consequence, wrongly linked covariate and outcome pairs will be created and bias further analysis of the linked data...

journal_title：Statistics in medicine

authors： Hof MH,Zwinderman AH

更新日期：2012-12-30 00:00:00

abstract：:Matched-pair designs have been commonly employed in diagnostic, epidemiologic and laboratory studies. For estimation of a ratio of two marginal probabilities in matched-pair data, a Wald-type logarithmic method is computationally simple, but an actual coverage rate is known to be smaller than a nominal one and a lengt...

journal_title：Statistics in medicine

authors： Nam JM

更新日期：2009-10-15 00:00:00

abstract：:There is a rich literature that considers whether an observed relation between treatment and response is due to an unobserved covariate. In order to quantify this unmeasured bias, an assumption is made about the distribution of this unobserved covariate; typically that it is either binary or at least confined to the u...

journal_title：Statistics in medicine

authors： Wang L,Krieger AM

更新日期：2006-07-15 00:00:00

abstract：:Assessing the calibration of methods for estimating the probability of the occurrence of a binary outcome is an important aspect of validating the performance of risk-prediction algorithms. Calibration commonly refers to the agreement between predicted and observed probabilities of the outcome. Graphical methods are a...

journal_title：Statistics in medicine

authors： Austin PC,Steyerberg EW

更新日期：2019-09-20 00:00:00

abstract：:Estimating the causal effect of quitting smoking on time to death or first myocardial infarction requires that one control for the differences in risk factors between individuals who elect to quite at each time t versus those who elect to continue smoking at time t. In this paper we examine the limitations of standard...

journal_title：Statistics in medicine

authors： Mark SD,Robins JM

更新日期：1993-09-15 00:00:00

abstract：:We present a method to determine the number of subjects, k, and number of repeated measurements, n, that minimize the overall cost of conducting a reliability study, while providing acceptable power for tests of hypotheses concerning the reliability coefficient rho. Tables showing optimal choices of k and n under vari...

journal_title：Statistics in medicine

authors： Eliasziw M,Donner A

更新日期：1987-09-01 00:00:00

abstract：:It is not uncommon for a continuous outcome variable Y to be dichotomized and analysed using logistic regression. Moser and Coombs (Statist. Med. 2004; 23:1843-1860) provide a method for converting the output from a standard linear regression analysis using the original continuous outcome Y to give much more efficient...

journal_title：Statistics in medicine

authors： Jiang Y,Scott A,Wild CJ

更新日期：2009-01-30 00:00:00

abstract：:In observational studies, many continuous or categorical covariates may be related to an outcome. Various spline-based procedures or the multivariable fractional polynomial (MFP) procedure can be used to identify important variables and functional forms for continuous covariates. This is the main aim of an explanatory...

journal_title：Statistics in medicine

authors： Binder H,Sauerbrei W,Royston P

更新日期：2013-06-15 00:00:00

abstract：:Various aspects of portfolio management and project prioritization within the pharmaceutical industry are examined. It is shown that the cost and probability architecture of a project is a crucial aspect of its value. An appropriate simple tool for ranking projects is the Pearson index. Various difficulties are consid...

journal_title：Statistics in medicine

authors： Senn S

更新日期：1996-12-30 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

authors： Sun H,Wang S

更新日期：2013-05-30 00:00:00

abstract：:Relative survival is used to estimate patient survival excluding causes of death not related to the disease of interest. Rather than using cause of death information from death certificates, which is often poorly recorded, relative survival compares the observed survival to that expected in a matched group from the ge...

journal_title：Statistics in medicine

authors： Lambert PC,Smith LK,Jones DR,Botha JL

更新日期：2005-12-30 00:00:00

abstract：:Studies in health research are commonly carried out in clustered settings, where the individual response data are correlated within clusters. Estimation and modelling of the extent of between-cluster variation contributes to understanding of the current study and to design of future studies. It is common to express be...

journal_title：Statistics in medicine

authors： Turner RM,Omar RZ,Thompson SG

更新日期：2006-05-15 00:00:00

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 constr...

journal_title：Statistics in medicine

authors： Nam JM,Blackwelder WC

更新日期：2002-03-15 00:00:00

abstract：:In phase 3 clinical trials, ethical and financial concerns motivate sequential analyses in which the data are analyzed prior to completion of the entire planned study. Existing group sequential software accounts for the effects of these interim analyses on the sampling density by assuming that the contribution of subs...

journal_title：Statistics in medicine

authors： Shoben AB,Emerson SS

更新日期：2014-12-20 00:00:00

abstract：:We construct nonparametric regression estimators of a number of temporal functions in a multistate system based on a continuous univariate baseline covariate. These estimators include state occupation probabilities, state entry, exit, and waiting (sojourn) time distribution functions of a general progressive (e.g., ac...

journal_title：Statistics in medicine

authors： Mostajabi F,Datta S

更新日期：2013-07-30 00:00:00

abstract：:A technique is presented which provides guidance on the spacing of follow-up waves in a multiwave study. Only information from the baseline wave is needed, as well as rough parameter estimates for the survival distribution. The computations use the expected Fisher information; a new method for its calculation is given...

journal_title：Statistics in medicine

authors： Chappell R

更新日期：1991-12-01 00:00:00

abstract：:Although the frequentist paradigm has been the predominant approach to clinical trial design since the 1940s, it has several notable limitations. Advancements in computational algorithms and computer hardware have greatly enhanced the alternative Bayesian paradigm. Compared with its frequentist counterpart, the Bayesi...

journal_title：Statistics in medicine

authors： Lee JJ,Chu CT

更新日期：2012-11-10 00:00:00

abstract：:Assessment of quality of life is becoming standard in clinical trials. A popular method for measuring quality of life is with instruments which utilize multiple-item subscales, in which each item is scored on a Likert scale. Most statistical methods for the analysis of quality of life data in clinical trials do not ex...

journal_title：Statistics in medicine

authors： Douglas JA

更新日期：1999-11-15 00:00:00

abstract：:Although the literature on contraceptive failure is vast and is expanding rapidly, our understanding of the relative efficacy of methods is quite limited because of defects in the research design and in the analytical tools used by investigators. Errors in the literature range from simple arithmetical mistakes to outr...

journal_title：Statistics in medicine

authors： Trussell J

更新日期：1991-02-01 00:00:00

abstract：:A preference trial is a special form of cross-over trial where clinical conditions determine when patients change treatment, in a prescribed order. This can be modelled using a geometric distribution. The model can be simply fitted using standard logistic regression methodology. The procedure is applied to a trial stu...

journal_title：Statistics in medicine

authors： Lindsey JK,Jones B

更新日期：1996-02-28 00:00:00

abstract：:Multilevel mixed effects survival models are used in the analysis of clustered survival data, such as repeated events, multicenter clinical trials, and individual participant data (IPD) meta-analyses, to investigate heterogeneity in baseline risk and covariate effects. In this paper, we extend parametric frailty model...

journal_title：Statistics in medicine

authors： Crowther MJ,Look MP,Riley RD

更新日期：2014-09-28 00:00:00

abstract：:Estimates of relative efficacy between alternative treatments are crucial for decision making in health care. Bayesian mixed treatment comparison models provide a powerful methodology to obtain such estimates when head-to-head evidence is not available or insufficient. In recent years, this methodology has become wide...

journal_title：Statistics in medicine

authors： Schmitz S,Adams R,Walsh C

更新日期：2013-07-30 00:00:00

abstract：:Screening for a disease such as cancer is often regarded as a beneficial and successful strategy for reducing mortality. However, as with any clinical treatment or intervention, benefit cannot be assumed, and screening can entail both costs and harms, so the screening as a 'treatment' must undergo evaluation. An evalu...

journal_title：Statistics in medicine

authors： Kafadar K,Prorok PC

更新日期：2005-02-28 00:00:00

abstract：:The use of outcome-dependent sampling with longitudinal data analysis has previously been shown to improve efficiency in the estimation of regression parameters. The motivating scenario is when outcome data exist for all cohort members but key exposure variables will be gathered only on a subset. Inference with outcom...

journal_title：Statistics in medicine

authors： Zelnick LR,Schildcrout JS,Heagerty PJ

更新日期：2018-06-15 00:00:00

abstract：:Randomized clinical trial designs commonly include one or more planned interim analyses. At these times an external monitoring committee reviews the accumulated data and determines whether it is scientifically and ethically appropriate for the study to continue. With failure-time endpoints, it is common to schedule an...

journal_title：Statistics in medicine

authors： Bagiella E,Heitjan DF

更新日期：2001-07-30 00:00:00

abstract：:In a typical bioequivalence trial, summary measures of the plasma concentration versus time profile are used to compare two formulations of a drug product. Commonly used measures include area under the curve (AUC), maximum plasma concentration (C(max)) and time to maximum concentration (T(max)). Equivalence of these s...

journal_title：Statistics in medicine

authors： Mauger DT,Chinchilli VM

更新日期：2000-10-30 00:00:00

abstract：:We present an estimate of the kappa-coefficient of agreement between two methods of rating based on matched pairs of binary responses and show that the estimate depends on the common intraclass correlation coefficient between the pairs. Via Monte Carlo simulation, we investigate power of the test of significance on ka...

journal_title：Statistics in medicine

authors： Shoukri MM,Martin SW,Mian IU

更新日期：1995-01-15 00:00:00

abstract：:We have formulated the problem of determining whether there has been an upturn in HIV-1 seroconversion incidence over the first five years of follow-up in the Multicenter AIDS Cohort Study (MACS) as that of locating the minimum of a quadratic regression or examination of two-knot piecewise spline models. Under a quadr...

journal_title：Statistics in medicine

authors： Zhou SY,Kingsley LA,Taylor JM,Chmiel JS,He DY,Hoover DR

更新日期：1993-01-30 00:00:00