Power and sample size determination for group comparison of patient-reported outcomes using polytomous Rasch models.

abstract：:This paper presents a procedure for obtaining short-term projections and lower bounds on the size of the acquired immunodeficiency syndrome (AIDS) epidemic. The method is similar to that proposed by Brookmeyer and Gail but adapted to the situation where individual dates of AIDS diagnosis are available. It gives result...

journal_title：Statistics in medicine

authors： Tango T

更新日期：1989-12-01 00:00:00

abstract：:In many epidemiological studies it is common to resort to regression models relating incidence of a disease and its risk factors. The main goal of this paper is to consider inference on such models with error-prone observations and variances of the measurement errors changing across observations. We suppose that the o...

journal_title：Statistics in medicine

authors： de Castro M,Galea M,Bolfarine H

更新日期：2008-11-10 00:00:00

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

authors： Zhang D,Quan H

更新日期：2009-02-28 00:00:00

abstract：:Risk prediction models play an important role in prevention and treatment of several diseases. Models that are in clinical use are often refined and improved. In many instances, the most efficient way to improve a successful model is to identify subgroups for which there is a specific biological rationale for improvem...

journal_title：Statistics in medicine

authors： Biswas S,Arun B,Parmigiani G

更新日期：2014-05-20 00:00:00

abstract：:Exploring the possible reasons for heterogeneity between studies is an important aspect of conducting a meta-analysis. This paper compares a number of methods which can be used to investigate whether a particular covariate, with a value defined for each study in the meta-analysis, explains any heterogeneity. The main ...

journal_title：Statistics in medicine

authors： Thompson SG,Sharp SJ

更新日期：1999-10-30 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：:Lower urinary tract symptoms can indicate the presence of urinary tract infection (UTI), a condition that if it becomes chronic requires expensive and time consuming care as well as leading to reduced quality of life. Detecting the presence and gravity of an infection from the earliest symptoms is then highly valuable...

journal_title：Statistics in medicine

authors： Barcella W,Iorio MD,Baio G,Malone-Lee J

更新日期：2016-04-15 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：:Weak measurement of epidemiologic exposures is an impediment to appreciation of the effects of those exposures. This paper discusses two strategies to assess the true effects of weakly measured exposure. The first is to use external information about the extent of mismeasurement to adjust estimates of the effects of e...

journal_title：Statistics in medicine

authors： Marshall JR

更新日期：1989-09-01 00:00:00

abstract：:Intraclass correlation coefficient (ICC) estimates must be provided when reporting the results of a cluster randomized trial. This study demonstrates that estimating this parameter with one-way ANOVA and an underlying mixed-effects statistical model leads to biased estimates. The bias depends on the effect size of the...

journal_title：Statistics in medicine

authors： Giraudeau B

更新日期：2006-03-30 00:00:00

abstract：:An important problem in oncology is comparing chemotherapy (chemo) agents in terms of their effects on survival or progression-free survival time. When the goal is to evaluate individual agents, a difficulty commonly encountered with observational data is that many patients receive a chemo combination including two or...

journal_title：Statistics in medicine

authors： Thall PF,Liu DD,Berrak SG,Wolff JE

更新日期：2011-07-10 00:00:00

abstract：:Two correction methods are considered for multiple logistic regression models with some covariates measured with error. Both methods are based on approximating the complicated regression model between the response and the observed covariates with simpler models. The first model is the logistic approximation proposed b...

journal_title：Statistics in medicine

authors： Kuha J

更新日期：1994-06-15 00:00:00

abstract：:The problem for assessing biosimilarity and drug interchangeability of follow-on biologics (biosimilar products) is studied. Unlike the generic products, the development of biosimilar products is much more complicated because of fundamental differences in functional structures and manufacturing processes. As a result,...

journal_title：Statistics in medicine

authors： Chow SC,Wang J,Endrenyi L,Lachenbruch PA

更新日期：2013-02-10 00:00:00

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

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

更新日期：2002-08-30 00:00:00

abstract：:We extend the method proposed in a recent work by the Authors for trial-level general surrogate evaluation to allow combinations of biomarkers and provide a procedure for finding the "best" combination of biomarkers based on the absolute prediction error summary of surrogate quality. We use a nonparametric Bayesian mo...

journal_title：Statistics in medicine

authors： Gabriel EE,Sachs MC,Daniels MJ,Halloran ME

更新日期：2019-03-30 00:00:00

abstract：:We give up-to-date methods for estimating the age-specific incidence of a disease and for estimating the effect of risk factors. We recommend taking age as the basic time scale of the analysis; then, the hazard function can be interpreted as the age-specific incidence of the disease. This choice raises a delayed entry...

journal_title：Statistics in medicine

authors： Commenges D,Letenneur L,Joly P,Alioum A,Dartigues JF

更新日期：1998-09-15 00:00:00

abstract：:Correlation is inherent in longitudinal studies due to the repeated measurements on subjects, as well as due to time-dependent covariates in the study. In the National Longitudinal Study of Adolescent to Adult Health (Add Health), data were repeatedly collected on children in grades 7-12 across four waves. Thus, obser...

journal_title：Statistics in medicine

authors： Irimata KM,Broatch J,Wilson JR

更新日期：2019-05-30 00:00:00

abstract：:Slow recruitment in clinical trials leads to increased costs and resource utilization, which includes both the clinic staff and patient volunteers. Careful planning and monitoring of the accrual process can prevent the unnecessary loss of these resources. We propose two hierarchical extensions to the existing Bayesian...

journal_title：Statistics in medicine

authors： Jiang Y,Simon S,Mayo MS,Gajewski BJ

更新日期：2015-02-20 00:00:00

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

authors： Nguyen TT,Bazzoli C,Mentré F

更新日期：2012-05-20 00:00:00

abstract：:Composite endpoints are frequently used in clinical trials, but simple approaches, such as the time to first event, do not reflect any ordering among the endpoints. However, some endpoints, such as mortality, are worse than others. A variety of procedures have been proposed to reflect the severity of the individual en...

journal_title：Statistics in medicine

authors： Follmann D,Fay MP,Hamasaki T,Evans S

更新日期：2020-02-28 00:00:00

abstract：:This paper investigates the design and analysis of dose-finding trials with two agents. The set of doses for each agent is fixed in advance. The goal of the trial is to find the set of dose combinations with probability of toxicity closest to a pre-specified value. For each of the two agents we assume that the probabi...

journal_title：Statistics in medicine

authors： Ivanova A,Wang K

更新日期：2004-06-30 00:00:00

abstract：:It has been well known that ignoring measurement error may result in substantially biased estimates in many contexts including linear and nonlinear regressions. For survival data with measurement error in covariates there has been extensive discussion in the literature with the focus being on the Cox proportional haza...

journal_title：Statistics in medicine

authors： He W,Yi GY,Xiong J

更新日期：2007-11-20 00:00:00

abstract：:In failure time studies involving a chronic disease such as cancer, several competing causes of mortality may be operating. Commonly, the conventional statistical technique of Kaplan-Meier, which is only meaningfully interpreted by assuming independence of failure types and the censoring mechanism, is employed in clin...

journal_title：Statistics in medicine

authors： Tai BC,Machin D,White I,Gebski V,EOI (The European Osteosarcoma Intergroup).

更新日期：2001-03-15 00:00:00

abstract：:In the pharmaceutical industry, some tests for quality assurance before, during and after the manufacture of a drug product involve a two-stage sampling technique. We propose statistical testing procedures in a two-stage sampling problem with large within-class sample sizes. We also derive a two-stage sampling plan by...

journal_title：Statistics in medicine

authors： Shao J,Chow SC

更新日期：1993-11-15 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：:Longitudinal studies collect information on a sample of individuals which is followed over time to analyze the effects of individual and time-dependent characteristics on the observed response. These studies often suffer from attrition: individuals drop out of the study before its completion time and thus present inco...

journal_title：Statistics in medicine

authors： Alfò M,Maruotti A

更新日期：2009-08-30 00:00:00

abstract：:Meta-analysis to investigate the joint effect of multiple factors in the aetiology of a disease is of increasing importance in epidemiology. This task is often challenging in practice, because studies typically concentrate on studying the effect of only one exposure, sometimes may report the interaction between two ex...

journal_title：Statistics in medicine

authors： Salanti G,Higgins JP,White IR

更新日期：2006-12-30 00:00:00

abstract：:Novel therapies are challenging the standards of drug development. Agents with specific biologic targets, unknown dose-efficacy curves, and limited toxicity mandate novel designs to identify biologically optimal doses. We review two model-based designs that utilize either a proportional odds model or a continuation ra...

journal_title：Statistics in medicine

authors： Mandrekar SJ,Qin R,Sargent DJ

更新日期：2010-05-10 00:00:00

abstract：:Diagnostic problems in medicine are sometimes polytomous, meaning that the outcome has more than two distinct categories. For example, ovarian tumors can be benign, borderline, primary invasive, or metastatic. Extending the main measure of binary discrimination, the c-statistic or area under the ROC curve, to nominal ...

journal_title：Statistics in medicine

authors： Van Calster B,Van Belle V,Vergouwe Y,Timmerman D,Van Huffel S,Steyerberg EW

更新日期：2012-10-15 00:00:00

abstract：:Bayesian methods are proposed for analysing matched case-control studies in which a binary exposure variable is sometimes measured with error, but whose correct values have been validated for a random sample of the matched case-control sets. Three models are considered. Model 1 makes few assumptions other than randomn...

journal_title：Statistics in medicine

authors： Prescott GJ,Garthwaite PH

更新日期：2005-02-15 00:00:00