Bayesian clinical trials in action.

abstract：:Standard randomization-based tests of sharp null hypotheses in randomized clinical trials, that is, intent-to-treat analyses, are valid without extraneous assumptions, but generally can be appropriately powerful only with alternative hypotheses that involve treatment assignment having an effect on outcome. In the cont...

journal_title：Statistics in medicine

authors： Rubin DB

更新日期：1998-02-15 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：:The Box-Cox power exponential (BCPE) distribution, developed in this paper, provides a model for a dependent variable Y exhibiting both skewness and kurtosis (leptokurtosis or platykurtosis). The distribution is defined by a power transformation Y(nu) having a shifted and scaled (truncated) standard power exponential ...

journal_title：Statistics in medicine

authors： Rigby RA,Stasinopoulos DM

更新日期：2004-10-15 00:00:00

abstract：:Multivariate random length data occur when we observe multiple measurements of a quantitative variable and the variable number of these measurements is also an observed outcome for each experimental unit. For example, for a patient with coronary artery disease, we may observe a number of lesions in that patient's coro...

journal_title：Statistics in medicine

authors： Barnhart HX,Kosinski AS,Sampson AR

更新日期：1999-01-30 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：:The 'landmark' and 'Simon and Makuch' non-parametric estimators of the survival function are commonly used to contrast the survival experience of time-dependent treatment groups in applications such as stem cell transplant versus chemotherapy in leukemia. However, the theoretical survival functions corresponding to th...

journal_title：Statistics in medicine

authors： Bernasconi DP,Rebora P,Iacobelli S,Valsecchi MG,Antolini L

更新日期：2016-03-30 00:00:00

abstract：:Prognostic models are used in medicine for investigating patient outcome in relation to patient and disease characteristics. Such models do not always work well in practice, so it is widely recommended that they need to be validated. The idea of validating a prognostic model is generally taken to mean establishing tha...

journal_title：Statistics in medicine

authors： Altman DG,Royston P

更新日期：2000-02-29 00:00:00

abstract：:An observed confidence distribution is proposed as a measure of strength of evidence for practically equivalent efficacies of two treatments. The concept is independent of prior opinions about relevant sizes of a difference in efficacy. It also avoids retrospective power calculations for trials with missed recruitment...

journal_title：Statistics in medicine

authors： Mau J

更新日期：1988-12-01 00:00:00

abstract：:Interval-censored data are commonly found in studies of diseases that progress without symptoms, which require clinical evaluation for detection. Several techniques have been suggested with independent assumption. However, the assumption will not be valid if observations come from clusters. Furthermore, when the clust...

journal_title：Statistics in medicine

authors： Kim YJ

更新日期：2010-12-10 00:00:00

abstract：:Despite our best efforts, missing outcomes are common in randomized controlled clinical trials. The National Research Council's Committee on National Statistics panel report titled The Prevention and Treatment of Missing Data in Clinical Trials noted that further research is required to assess the impact of missing da...

journal_title：Statistics in medicine

authors： Cook T,Zea R

更新日期：2020-01-30 00:00:00

abstract：:The mutation and natural selection phenomenon can and often does cause the failure of antimicrobial, herbicidal, pesticide and cancer treatments selection pressures. This phenomenon operates in a mathematically predictable behavior, which when understood leads to approaches to reduce and prevent the failure of the use...

journal_title：Statistics in medicine

authors： Kleinman A

更新日期：2014-12-20 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：:In a recent Statistics in Medicine paper, Warn, Thompson and Spiegelhalter (WTS) made a comparison between the Bayesian approach to the meta-analysis of binary outcomes and a popular Classical approach that uses summary (two-stage) techniques. They included approximate summary (two-stage) Bayesian techniques in their ...

journal_title：Statistics in medicine

authors： O'Rourke K,Altman DG

更新日期：2005-09-15 00:00:00

abstract：:Several large-sample confidence intervals for the ratio of independent binomial proportions are compared in terms of exact coverage probability and width. A non-iterative approximate Bayesian interval is derived and its frequency properties are superior to all of the non-iterative confidence intervals considered. The ...

journal_title：Statistics in medicine

authors： Price RM,Bonett DG

更新日期：2008-11-20 00:00:00

abstract：:Our objective is to develop a model to estimate the relative risk of disease in each area, Ai, i=1, ... , n, of a region and to identify areas of unusually high or low risk. We use a product partition model (PPM) in which we assume that the true relative risks can be partitioned into a number of components or sets of ...

journal_title：Statistics in medicine

authors： Hegarty A,Barry D

更新日期：2008-08-30 00:00:00

abstract：:In modern observational studies using electronic health records or other routinely collected data, both the outcome and covariates of interest can be error-prone and their errors often correlated. A cost-effective solution is the two-phase design, under which the error-prone outcome and covariates are observed for all...

journal_title：Statistics in medicine

authors： Tao R,Lotspeich SC,Amorim G,Shaw PA,Shepherd BE

更新日期：2021-02-10 00:00:00

abstract：:Although there are many models which are used to calculate the health benefits (and thus the cost-effectiveness) of vaccination programmes, they can be divided into two groups: those which assume a constant force of infection, that is a constant per-susceptible rate of infection; and those which assume that the force ...

journal_title：Statistics in medicine

authors： Edmunds WJ,Medley GF,Nokes DJ

更新日期：1999-12-15 00:00:00

abstract：:We describe how trends in the vaccination coverage at 19 months of age vary by race/ethnicity; explore the extent to which data required to evaluate a child's up-to-date vaccination status is missing as a result of the scattering of vaccination records among many vaccination providers; evaluate how the prevalence of t...

journal_title：Statistics in medicine

authors： Smith PJ,Stevenson J

更新日期：2008-09-10 00:00:00

abstract：:Geocoding a study population as completely as possible is an important data assimilation component of many spatial epidemiologic studies. Unfortunately, complete geocoding is rare in practice. The failure of a substantial proportion of study subjects' addresses to geocode has consequences for spatial analyses, some of...

journal_title：Statistics in medicine

authors： Zimmerman DL,Fang X,Mazumdar S

更新日期：2008-09-20 00:00:00

abstract：:Estimates of transmission probabilities for sexually transmitted diseases historically come from studies of uninfected individuals exposed to those with a high disease prevalence (for example, prostitutes). However, changes in sexual behaviour, much of which relates to concerns about AIDS, has made identification of p...

journal_title：Statistics in medicine

authors： Katz BP

更新日期：1992-03-01 00:00:00

abstract：:Wittes and Brittain recommended using an 'internal pilot study' to adjust sample size. The approach involves five steps in testing a general linear hypothesis for a general linear univariate model, with Gaussian errors. First, specify the design, hypothesis, desired test size, power, a smallest 'clinically meaningful'...

journal_title：Statistics in medicine

authors： Coffey CS,Muller KE

更新日期：1999-05-30 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：:Methods for multiple informants help to estimate the marginal effect of each multiple source predictor and formally compare the strength of their association with an outcome. We extend multiple informant methods to the case of hierarchical data structures to account for within cluster correlation. We apply the propose...

journal_title：Statistics in medicine

authors： Baek J,Sánchez BN,Sanchez-Vaznaugh EV

更新日期：2014-02-20 00:00:00

abstract：:In this paper, we investigate the impact of time-invariant covariates when fitting transition mixed models. This is carried out by emphasizing on the role of baseline responses on the estimation process. Transition models are allowed for two cases of exogenous and endogenous baseline responses. We illustrate these con...

journal_title：Statistics in medicine

authors： Rikhtehgaran R,Kazemi I,Verbeke G

更新日期：2014-11-30 00:00:00

abstract：:Providing personalized treatments designed to maximize benefits and minimizing harms is of tremendous current medical interest. One problem in this area is the evaluation of the interaction between the treatment and other predictor variables. Treatment effects in subgroups having the same direction but different magni...

journal_title：Statistics in medicine

authors： Chen W,Ghosh D,Raghunathan TE,Norkin M,Sargent DJ,Bepler G

更新日期：2012-12-10 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：:This paper reviews several emerging and recurrent issues relating to the drug development process. These emerging issues include changes to the FDA regulatory environment, internationalization of drug development, advances in computer technology and visualization tools, and efforts to incorporate meta-analysis methodo...

journal_title：Statistics in medicine

authors： Anello C

更新日期：1999-09-15 00:00:00

abstract：:Graphical methods are often used to check goodness-of-fit of models to data. It is common to plot residuals against a reference distribution so that when the model fits the data, the configuration should be close to a straight line. Since the resemblance to a straight line is often unclear, it has been suggested to ad...

journal_title：Statistics in medicine

authors： Cohen A,Barnett O

更新日期：1995-08-30 00:00:00

abstract：:Analysis of panel data is often challenged by the presence of heterogeneity and state misclassification. In this paper, we propose a hidden mover-stayer model to facilitate heterogeneity for a population that consists of two subpopulations each of movers or of stayers and to simultaneously account for state misclassif...

journal_title：Statistics in medicine

authors： Yi GY,He W,He F

更新日期：2017-09-10 00:00:00

abstract：:Evaluating treatment effects within different subsets of patients is a common practice in the analysis of individual randomized clinical trials. Such analyses are limited, however, by the number of patients available. Overviews, by providing evidence based on large numbers of patients, can be useful for overcoming the...

journal_title：Statistics in medicine

authors： Gelber RD,Goldhirsch A

更新日期：1987-04-01 00:00:00