Pros and cons of permutation tests in clinical trials.

abstract：:This article deals with determination of a sample size that guarantees the success of a trial. We follow a Bayesian approach and we say an experiment is successful if it yields a large posterior probability that an unknown parameter of interest (an unknown treatment effect or an effects-difference) is greater than a c...

journal_title：Statistics in medicine

authors： Brutti P,De Santis F,Gubbiotti S

更新日期：2008-06-15 00:00:00

abstract：:The percentile-finding experimental design known variously as 'forced-choice fixed-staircase', 'geometric up-and-down' or 'k-in-a-row' (KR) was introduced by Wetherill four decades ago. To date, KR has been by far the most widely used up-and-down (U&D) design for estimating non-median percentiles; it is implemented mo...

journal_title：Statistics in medicine

authors： Oron AP,Hoff PD

更新日期：2009-06-15 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

authors： Küchenhoff H,Carroll RJ

更新日期：1997-01-15 00:00:00

abstract：:We propose a new design for dose finding for cytotoxic agents in two ordered groups of patients. By ordered groups, we mean that prior to the study there is clinical information that would indicate that for a given dose one group would be more susceptible to toxicities than patients in the other group. The designs are...

journal_title：Statistics in medicine

authors： Conaway MR,Wages NA

更新日期：2017-01-30 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：:In longitudinal studies with incomplete data, where the number of time points can become numerous, it is often advantageous to model the covariance matrix. We describe several covariance models (for example, mixed models, compound symmetry, AR(1)-type models, and combination models) that offer parsimonious alternative...

journal_title：Statistics in medicine

authors： Grady JJ,Helms RW

更新日期：1995-07-15 00:00:00

abstract：:Generalized relative and absolute risk models, in which various functions of time and age modify the excess relative or absolute risk of radiation-induced cancer, are fitted to the Japanese atomic bomb survivor cancer incidence data set. Among generalized relative risk models, those in which a product of powers of tim...

journal_title：Statistics in medicine

authors： Little MP,Muirhead CR,Charles MW

更新日期：1999-01-15 00:00:00

abstract：:Crossover studies have been successfully conducted in the case of continuous responses. Existing procedures of analysis for ordinal responses, on the other hand, are rarely satisfactory unless strict, usually unrealistic, assumptions are made. In this paper we investigate a random effects model and show that the model...

journal_title：Statistics in medicine

authors： Ezzet F,Whitehead J

更新日期：1991-06-01 00:00:00

abstract：:When the event time of interest depends on the censoring time, conventional two-sample test methods, such as the log-rank and Wilcoxon tests, can produce an invalid test result. We extend our previous work on estimation using auxiliary variables to adjust for dependent censoring via multiple imputation, to the compari...

journal_title：Statistics in medicine

authors： Hsu CH,Taylor JM

更新日期：2009-02-01 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：:A decision-theoretic framework is proposed for designing sequential dose-finding trials with multiple outcomes. The optimal strategy is solvable theoretically via backward induction. However, for dose-finding studies involving k doses, the computational complexity is the same as the bandit problem with k-dependent arm...

journal_title：Statistics in medicine

authors： Fan SK,Wang YG

更新日期：2006-05-30 00:00:00

abstract：:R2 has been criticized as a measure of model performance when predicting a dichotomous outcome, both because its value is often low and because it is sensitive to the prevalence of the event of interest. The C statistic is more widely used to measure model performance in a 0/1 setting. We use a simple parametric famil...

journal_title：Statistics in medicine

authors： Ash A,Shwartz M

更新日期：1999-02-28 00:00:00

abstract：:In observational cohort studies we may wish to examine the associations between fixed patient characteristics and the longitudinal changes from baseline in a repeated outcome measure. Many biological and other outcome measures are known to be subject to measurement error and biological variation. In an initial analysi...

journal_title：Statistics in medicine

authors： Harrison L,Dunn DT,Green H,Copas AJ

更新日期：2009-11-20 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

authors： Lu WS,Tsutakawa RK

更新日期：1996-07-15 00:00:00

abstract：:Joint models initially dedicated to a single longitudinal marker and a single time-to-event need to be extended to account for the rich longitudinal data of cohort studies. Multiple causes of clinical progression are indeed usually observed, and multiple longitudinal markers are collected when the true latent trait of...

journal_title：Statistics in medicine

authors： Proust-Lima C,Dartigues JF,Jacqmin-Gadda H

更新日期：2016-02-10 00:00:00

abstract：:There are inconsistencies between the formulas for the variance of standardized mean difference (SMD) in the Cochrane Handbook for Systematic Reviews and the variance reported in other sources. Instead of the variance appropriate for the SMD of a crossover experiment, the Cochrane Handbook uses the variance appropriat...

journal_title：Statistics in medicine

authors： Kitchenham B,Madeyski L

更新日期：2020-11-30 00:00:00

abstract：:Juvenile dermatomyositis (JDM) is a rare autoimmune disease that may lead to serious complications, even to death. We develop a 2-state Markov regression model in a Bayesian framework to characterise disease progression in JDM over time and gain a better understanding of the factors influencing disease risk. The trans...

journal_title：Statistics in medicine

authors： De Iorio M,Gallot N,Valcarcel B,Wedderburn L

更新日期：2018-05-10 00:00:00

abstract：:A trial of Duchenne muscular dystrophy involved tracking boys of all ages through a one-year baseline period, followed by a one-year trial of leucine versus placebo treatment. In this paper we develop a model for a total-muscle-strength score that uses the data of the extended baseline period in the evaluation of the ...

journal_title：Statistics in medicine

authors： Madsen KS,Miller JP,Province MA

更新日期：1986-05-01 00:00:00

abstract：:The goal of phase I cancer trials is to determine the highest dose of a treatment regimen with an acceptable toxicity rate. Traditional designs for phase I trials, such as the Continual Reassessment Method (CRM) and the 3 + 3 design, require each patient or a cohort of patients to be fully evaluated for the dose-limit...

journal_title：Statistics in medicine

authors： Polley MY

更新日期：2011-07-30 00:00:00

abstract：:Novel methodology is implemented to assess the predictive power of covariate information associated with sequential binary events. Logistic models are first fitted on the basis of a subset of the observations and then evaluated sequentially on the rest. The probabilistic forecasts are compared to the outcomes via a sc...

journal_title：Statistics in medicine

authors： Seillier-Moiseiwitsch F

更新日期：1996-10-30 00:00:00

abstract：:Detecting safety signals in clinical trial safety data is known to be challenging due to high dimensionality, rare occurrence, weak signal, and complex dependence. We propose a new hierarchical testing approach for analyzing safety data from a typical randomized clinical trial. This approach accounts for the hierarchi...

journal_title：Statistics in medicine

authors： Tan X,Chen BE,Sun J,Patel T,Ibrahim JG

更新日期：2020-05-15 00:00:00

abstract：:Quantifying socioeconomic disparities and understanding the roots of inequalities are growing topics in cancer research. However, socioeconomic differences are challenging to investigate mainly due to the lack of accurate data at individual-level, while aggregate indicators are only partially informative. We implement...

journal_title：Statistics in medicine

authors： Mezzetti M,Palli D,Dominici F

更新日期：2020-01-15 00:00:00

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

authors： Lachin JM,Greenhouse SW,Bautista OM

更新日期：2003-11-15 00:00:00

abstract：:A new method is proposed for the surveillance of Down's syndrome among newborn. Despite the strong dependence of overall risk of Down's syndrome on maternal age, it has been suggested that an environmentally induced increase in risk may be additive over all maternal ages. The surveillance method introduced here is spe...

journal_title：Statistics in medicine

authors： Lie RT,Heuch I,Irgens LM

更新日期：1993-01-15 00:00:00

abstract：:Clinical trials studying treatments for rare diseases are challenging to design and conduct due to the limited number of patients eligible for the trial. One design used to address this challenge is the small n, sequential, multiple assignment, randomized trial (snSMART). We propose a new snSMART design that investiga...

journal_title：Statistics in medicine

authors： Fang F,Hochstedler KA,Tamura RN,Braun TM,Kidwell KM

更新日期：2021-02-20 00:00:00

abstract：:The lifetime risk of a clinical condition is the probability of onset of the condition during one's lifespan. Recent advances in Alzheimer's disease (AD) research have identified screening tests for biomarkers that can identify persons who are in the earliest stages of the AD process but who do not yet have any clinic...

journal_title：Statistics in medicine

authors： Brookmeyer R,Abdalla N

更新日期：2019-04-30 00:00:00

abstract：:The analysis of multivariate time-to-event (TTE) data can become complicated due to the presence of clustering, leading to dependence between multiple event times. For a long time, (conditional) frailty models and (marginal) copula models have been used to analyze clustered TTE data. In this article, we propose a gene...

journal_title：Statistics in medicine

authors： Tran TMP,Abrams S,Braekers R

更新日期：2020-05-30 00:00:00

abstract：:Epidemiologic and clinical studies routinely collect longitudinal measures of multiple outcomes, including biomarker measures, cognitive functions, and clinical symptoms. These longitudinal outcomes can be used to establish the temporal order of relevant biological processes and their association with the onset of cli...

journal_title：Statistics in medicine

authors： Yang L,Gao S

更新日期：2013-03-15 00:00:00

abstract：:Some clinical trials aim to demonstrate therapeutic equivalence on multiple primary endpoints. For example, therapeutic equivalence studies of agents for the treatment of osteoarthritis use several primary endpoints including investigator's global assessment of disease activity, patient's global assessment of response...

journal_title：Statistics in medicine

authors： Quan H,Bolognese J,Yuan W

更新日期：2001-11-15 00:00:00

abstract：:Identification of treatment selection biomarkers has become very important in cancer drug development. Adaptive enrichment designs have been developed for situations where a unique treatment selection biomarker is not apparent based on the mechanism of action of the drug. With such designs, the eligibility rules may b...

journal_title：Statistics in medicine

authors： Simon R,Simon N

更新日期：2017-11-20 00:00:00