Methods for proper handling of overrunning and underrunning in phase II designs for oncology trials.

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

authors： Greenland S

更新日期：1987-09-01 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：:To construct a confidence interval for the mean of a log-normal distribution in small samples, we propose likelihood-based approaches - the signed log-likelihood ratio and modified signed log-likelihood ratio methods. Extensive Monte Carlo simulation results show the advantages of the modified signed log-likelihood ra...

journal_title：Statistics in medicine

authors： Wu J,Wong AC,Jiang G

更新日期：2003-06-15 00:00:00

abstract：:Paired count data usually arise in medicine when before and after treatment measurements are considered. In the present paper we assume that the correlated paired count data follow a bivariate Poisson distribution in order to derive the distribution of their difference. The derived distribution is shown to be the same...

journal_title：Statistics in medicine

authors： Karlis D,Ntzoufras I

更新日期：2006-06-15 00:00:00

abstract：:One may encounter the application of the two independent samples t-test to ordinal scaled data (for example, data that assume only the values 0, 1, 2, 3) from small samples. This situation clearly violates the underlying normality assumption for the t-test and one cannot appeal to large sample theory for validity. In ...

journal_title：Statistics in medicine

authors： Heeren T,D'Agostino R

更新日期：1987-01-01 00:00:00

abstract：:This paper concerns using modified weighted Schoenfeld residuals to test the proportionality of subdistribution hazards for the Fine-Gray model, similar to the tests proposed by Grambsch and Therneau for independently censored data. We develop a score test for the time-varying coefficients based on the modified Schoen...

journal_title：Statistics in medicine

authors： Zhou B,Fine J,Laird G

更新日期：2013-09-30 00:00:00

abstract：:A measure of similarity for response curves is presented and its potential use is discussed. The distribution of a suitable test statistic for testing the independence of the course of two curves is derived. The method proposed is compared with other proposals in the literature for the analysis of paired response curv...

journal_title：Statistics in medicine

authors： Schulgen G

更新日期：1989-11-01 00:00:00

abstract：:The objective of a phase II cancer clinical trial is to screen a treatment that can produce a similar or better response rate compared to the current treatment results. This screening is usually carried out in two stages as proposed by Simon. For ineffective treatment, the trial should terminate at the first stage. En...

journal_title：Statistics in medicine

authors： Chen TT

更新日期：1997-12-15 00:00:00

abstract：:In this paper, we discuss family-based association test (FBATs) relating genetic data to survival and time-to-onset data. We show how the standard logrank and Wilcoxon statistics can be used with family data to develop tests of association. We prove that the FBAT-logrank approach can be identical to the proportional h...

journal_title：Statistics in medicine

authors： Lange C,Blacker D,Laird NM

更新日期：2004-01-30 00:00:00

abstract：:Dependence between observations on a dichotomous variable renders invalid the usual chi-square tests of independence and inflates the variances of parameter estimates. Such a situation occurs, for example, when subjects consist of members of the same family or with repeated observations on the same person. In this pap...

journal_title：Statistics in medicine

authors： Donald A,Donner A

更新日期：1987-06-01 00:00:00

abstract：:We point out that the conventional methods for ties correction may be seriously biased when censoring times depend on covariates. A simple modification to the Efron correction method is suggested which works remarkably well in simulation studies. The method corresponds closely to breaking ties by random ordering. The ...

journal_title：Statistics in medicine

authors： Samuelsen SO,Wisløff TF,Skrondal A

更新日期：2005-10-30 00:00:00

abstract：:The relative importance of prognostic factors in regression can be measured either by standardized regression coefficients or by percentages of explained variation in a dependent variable. One advantage of using explained variation is the direct comparability of qualitative prognostic factors with others, or of groups...

journal_title：Statistics in medicine

authors： Schemper M

更新日期：1993-12-30 00:00:00

abstract：:In this paper, we consider pooling schemes in which samples are to be tested in two-stages. We show that when batch size is limited as well as pool size, selection schemes tend to be more efficient and flexible. Formulae for the efficiencies of square arrays in all dimensions and for all selection schemes are given in...

journal_title：Statistics in medicine

authors： Sudbury A

更新日期：2010-09-20 00:00:00

abstract：:In recent years, quality control charts have been increasingly applied in the healthcare environment, for example, to monitor surgical performance. Risk-adjusted cumulative (CUSUM) charts that utilize risk scores like the Parsonnet score to estimate the probability of death of a patient from an operation turn out to b...

journal_title：Statistics in medicine

authors： Knoth S,Wittenberg P,Gan FF

更新日期：2019-05-30 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：: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

authors： Ellenberg SS

更新日期：2004-01-30 00:00:00

abstract：:A popular method to control multiplicity in confirmatory clinical trials is to use a so-called hierarchical, or fixed sequence, test procedure. This requires that the null hypotheses are ordered a priori, for example, in order of clinical importance. The procedure tests the hypotheses in this order using alpha-level t...

journal_title：Statistics in medicine

authors： Edwards D,Madsen J

更新日期：2007-12-10 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：:This paper examines several methods for deriving standardized morbidity ratios (SMR) and attributable fraction (attributable risk percentage) estimates. We show that some of the proposed methods will, in general, produce biased estimators, although the low variance of certain estimators sometimes compensates for their...

journal_title：Statistics in medicine

authors： Greenland S

更新日期：1984-04-01 00:00:00

abstract：:Epidermal nerve fibre (ENF) density and morphology are used to study small fibre involvement in diabetic, HIV, chemotherapy induced and other neuropathies. ENF density and summed length of ENFs per epidermal surface area are reduced, and ENFs may appear more clustered within the epidermis in subjects with small fibre ...

journal_title：Statistics in medicine

authors： Andersson C,Guttorp P,Särkkä A

更新日期：2016-10-30 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

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

更新日期：1999-12-30 00:00:00

abstract：:A method has been developed for simultaneously comparing the usefulness of many treatments of established value for symptomatic medical conditions. Medical assessment of outcome is not employed. Instead patients are required to assess treatments prescribed during the course of ordinary general practice rather than und...

journal_title：Statistics in medicine

authors： Charlton JR,D'Souza MF,Tooley M,Silver R

更新日期：1985-01-01 00:00:00

abstract：:A few large multi-centre male-only heart trials done in the 1970s and 1980s have been seen as ill-conceived because they did not include females. The purpose here is to revisit two of those trials and to consider consequences in terms of cost and power had they been designed to include females. ...

journal_title：Statistics in medicine

authors： Meinert CL

更新日期：1999-02-15 00:00:00

abstract：:This work is motivated by a longitudinal study of women and their ectopic pregnancy outcomes in Lund, Sweden. In this article, we review and apply the Liang-Zeger methodology to the Lund ectopic pregnancy data set. We further analyse the ectopic pregnancy data using conditional modelling approaches suggested by Rosner...

journal_title：Statistics in medicine

authors： Hadgu A,Koch G,Westrom L

更新日期：1997-11-15 00:00:00

abstract：:Correlated data are obtained in longitudinal epidemiological studies, where repeated measurements are taken on individuals or groups over time. Such longitudinal data are ideally analyzed using multilevel modeling approaches, which appropriately account for the correlations in repeated responses in the same individual...

journal_title：Statistics in medicine

authors： Xanthakis V,Sullivan LM,Vasan RS

更新日期：2013-12-10 00:00:00

abstract：:It is often of interest to use observational data to estimate the causal effect of a target exposure or treatment on an outcome. When estimating the treatment effect, it is essential to appropriately adjust for selection bias due to observed confounders using, for example, propensity score weighting. Selection bias du...

journal_title：Statistics in medicine

authors： Parast L,Griffin BA

更新日期：2020-08-15 00:00:00

abstract：:We study the competing risks model and show that the cause j cumulative incidence function integrated from 0 to τ has a natural interpretation as the expected number of life years lost due to cause j before time τ. This is analogous to the τ-restricted mean lifetime, which is the survival function integrated from 0 to...

journal_title：Statistics in medicine

authors： Andersen PK

更新日期：2013-12-30 00:00:00

abstract：:This paper considers the problem of selecting a set of regressors when the response variable is distributed according to a specified parametric model and observations are censored. Under a Bayesian perspective, the most widely used tools are Bayes factors (BFs), which are undefined when improper priors are used. In or...

journal_title：Statistics in medicine

authors： Cabras S,Castellanos ME,Perra S

更新日期：2014-11-20 00:00:00

abstract：:Paired data from matched case-control studies are commonly used to estimate the association between the exposure to a risk factor and the occurrence of a disease. The odds ratio is typically used to quantify this association. Difficulties in estimating the true odds ratio with matched pairs arise, however, when the ex...

journal_title：Statistics in medicine

authors： Miller KM,Looney SW

更新日期：2012-11-30 00:00:00

abstract：:Meta-analyses of clinical trials are increasingly seeking to go beyond estimating the effect of a treatment and may also aim to investigate the effect of other covariates and how they alter treatment effectiveness. This requires the estimation of treatment-covariate interactions. Meta-regression can be used to estimat...

journal_title：Statistics in medicine

authors： Simmonds MC,Higgins JP

更新日期：2007-07-10 00:00:00