A general statistical principle for changing a design any time during the course of a trial.

abstract：:Many reactions in enzymology are governed by the Michaelis-Menten equation. Characterising these reactions requires the estimation of the parameters K(M) and V(max) which determine the Michaelis-Menten equation and this is done by observing rates of reactions at a set of substrate concentrations. The choice of substra...

journal_title：Statistics in medicine

authors： Matthews JN,Allcock GC

更新日期：2004-02-15 00:00:00

abstract：:When estimating the average effect of a binary treatment (or exposure) on an outcome, methods that incorporate propensity scores, the G-formula, or targeted maximum likelihood estimation (TMLE) are preferred over naïve regression approaches, which are biased under misspecification of a parametric outcome model. In con...

journal_title：Statistics in medicine

authors： Luque-Fernandez MA,Schomaker M,Rachet B,Schnitzer ME

更新日期：2018-07-20 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：:We present an approach for inference on contrasts of clinically meaningful functionals of a survivor distribution (e.g., restricted mean, quantiles) that can avoid strong parametric or semiparametric assumptions on the underlying structure of the data. In this multistage approach, we first use an adaptive predictive m...

journal_title：Statistics in medicine

authors： Rudser KD,LeBlanc ML,Emerson SS

更新日期：2012-07-20 00:00:00

abstract：:Missing values in the covariates are a widespread complication in the statistical inference of regression models. The maximum likelihood principle requires specification of the distribution of the covariates, at least in part. For categorical covariates, log-linear models can be used. Additionally, the missing at rand...

journal_title：Statistics in medicine

authors： Vach W,Blettner M

更新日期：1995-06-30 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：:The application of Bayesian hierarchical models to measure spatial effects in time to event data has not been widely reported. This case study aims to estimate the effect of area of residence on waiting times to coronary artery bypass graft (CABG) and to assess the role of important individual specific covariates (age...

journal_title：Statistics in medicine

authors： Crook AM,Knorr-Held L,Hemingway H

更新日期：2003-09-30 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：:In biomedical literature, numerous prediction models for clinical outcomes have been developed based either on clinical data or, more recently, on high-throughput molecular data (omics data). Prediction models based on both types of data, however, are less common, although some recent studies suggest that a suitable c...

journal_title：Statistics in medicine

authors： De Bin R,Sauerbrei W,Boulesteix AL

更新日期：2014-12-30 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：:We consider statistical procedures for hypothesis testing of real valued functionals of matched pairs with missing values. In order to improve the accuracy of existing methods, we propose a novel multiplication combination procedure. Dividing the observed data into dependent (completely observed) pairs and independent...

journal_title：Statistics in medicine

authors： Amro L,Konietschke F,Pauly M

更新日期：2019-07-30 00:00:00

abstract：:It is well established that neural activity is stochastically modulated over time. Therefore, direct comparisons across experimental conditions and determination of change points or maximum firing rates are not straightforward. This study sought to compare temporal firing probability curves that may vary across groups...

journal_title：Statistics in medicine

authors： Roca-Pardiñas J,Cadarso-Suárez C,Pardo-Vazquez JL,Leboran V,Molenberghs G,Faes C,Acuña C

更新日期：2011-06-30 00:00:00

abstract：:Studies involving longitudinal binary responses are widely applied in the health and biomedical sciences research and frequently analyzed by generalized estimating equations (GEE) method. This article proposes an alternative goodness-of-fit test based on the nonparametric smoothing approach for assessing the adequacy ...

journal_title：Statistics in medicine

authors： Lin KC,Chen YJ,Shyr Y

更新日期：2008-09-30 00:00:00

abstract：:It is unclear to what extent the incremental predictive performance of a novel biomarker is impacted by the method used to control for standard predictors. We investigated whether adding a biomarker to a model with a published risk score overestimates its incremental performance as compared to adding it to a multivari...

journal_title：Statistics in medicine

authors： Xanthakis V,Sullivan LM,Vasan RS,Benjamin EJ,Massaro JM,D'Agostino RB Sr,Pencina MJ

更新日期：2014-07-10 00:00:00

abstract：:Correct specification of the inverse probability weighting (IPW) model is necessary for consistent inference from a marginal structural Cox model (MSCM). In practical applications, researchers are typically unaware of the true specification of the weight model. Nonetheless, IPWs are commonly estimated using parametric...

journal_title：Statistics in medicine

authors： Karim ME,Platt RW,BeAMS study group.

更新日期：2017-06-15 00:00:00

abstract：:Effective statistical help to biological and medical research demands thorough involvement of the statistician. The breadth of his activities can be illustrated by considering the questions he needs to discuss with his scientific colleagues in the course of planning a comparative experiment. The paper presents and com...

journal_title：Statistics in medicine

authors： Finney DJ

更新日期：1982-01-01 00:00:00

abstract：:Testing the equality of 2 proportions for a control group versus a treatment group is a well-researched statistical problem. In some settings, there may be strong historical data that allow one to reliably expect that the control proportion is one, or nearly so. While one-sample tests or comparisons to historical cont...

journal_title：Statistics in medicine

authors： Gabriel EE,Nason M,Fay MP,Follmann DA

更新日期：2018-03-30 00:00:00

abstract：:Meta-analyses pooling continuous outcomes can use mean differences (MD), standardized MD (MD in pooled standard deviation units, SMD), or ratio of arithmetic means (RoM). Recently, ratio of geometric means using ad hoc (RoGM (ad hoc) ) or Taylor series (RoGM (Taylor) ) methods for estimating variances have been propos...

journal_title：Statistics in medicine

authors： Friedrich JO,Adhikari NK,Beyene J

更新日期：2012-07-30 00:00:00

abstract：:Correlated response data arise often in biomedical studies. The generalized estimation equation (GEE) approach is widely used in regression analysis for such data. However, there are few methods available to check the adequacy of regression models in GEE. In this paper, a graphical method is proposed based on Cook and...

journal_title：Statistics in medicine

authors： Pan W,Connett JE,Porzio GC,Weisberg S

更新日期：2001-10-15 00:00:00

abstract：:In many countries, the monitoring of child growth does not occur in a regular manner, and instead, we may have to rely on sporadic observations that are subject to substantial measurement error. In these countries, it can be difficult to identify patterns of poor growth, and faltering children may miss out on essentia...

journal_title：Statistics in medicine

authors： Anderson C,Xiao L,Checkley W

更新日期：2019-08-30 00:00:00

abstract：:Genome-wide association studies are characterized by a huge number of statistical tests performed to discover new disease-related genetic variants [in the form of single-nucleotide polymorphisms (SNPs)] in human DNA. Many SNPs have been identified for cross-sectionally measured phenotypes. However, there is a growing ...

journal_title：Statistics in medicine

authors： Sikorska K,Rivadeneira F,Groenen PJ,Hofman A,Uitterlinden AG,Eilers PH,Lesaffre E

更新日期：2013-01-15 00:00:00

abstract：:In lifetime data, like cancer studies, there may be long term survivors, which lead to heavy censoring at the end of the follow-up period. Since a standard survival model is not appropriate to handle these data, a cure model is needed. In the literature, covariate hypothesis tests for cure models are limited to parame...

journal_title：Statistics in medicine

authors： López-Cheda A,Jácome MA,Van Keilegom I,Cao R

更新日期：2020-07-30 00:00:00

abstract：:Knee replacement is the most commonly used surgical treatment for knee arthritis. It has been reported that knee replacement rates vary across both regions and counties. This paper used data from Medicare patients to develop explanations for the variation. One problem with our data is that we do not have patient level...

journal_title：Statistics in medicine

authors： Zhou XH,Katz BP,Holleman E,Melfi CA,Dittus R

更新日期：1996-09-15 00:00:00

abstract：:We study the group bridge and the adaptive group bridge penalties for competing risks quantile regression with group variables. While the group bridge consistently identifies nonzero group variables, the adaptive group bridge consistently selects variables not only at group level but also at within-group level. We all...

journal_title：Statistics in medicine

authors： Ahn KW,Kim S

更新日期：2018-04-30 00:00:00

abstract：:In genetic association studies, mixed effects models have been widely used in detecting the pleiotropy effects which occur when one gene affects multiple phenotype traits. In particular, bivariate mixed effects models are useful for describing the association of a gene with a continuous trait and a binary trait. Howev...

journal_title：Statistics in medicine

authors： Zhang Q,Yi GY

更新日期：2020-11-20 00:00:00

abstract：:Exposure to a carcinogen is likely to be reflected by temporal clustering of cancer cases among the community members. Detection of such clustering is a complicated task in general and more so in a small workplace community with low turnover rate. In this study we applied the CUSCORE test and the q-interval statistic ...

journal_title：Statistics in medicine

authors： Chen R,Froom P

更新日期：2003-10-15 00:00:00

abstract：:We consider evaluation and comparison of the diagnostic accuracy of biomarkers with continuous test outcomes, possibly correlated due to repeated measurements. We develop nonparametric group sequential testing procedures to evaluate and compare the area of biomarkers under their receiver operating characteristic curve...

journal_title：Statistics in medicine

authors： Liu A,Wu C,Schisterman EF

更新日期：2008-05-10 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：:In this study, 401 cases of childhood cancer in four California counties in 1980-1988 were analysed with the innovative methodology of density equalizing map projections. The data were originally collected and analysed by the California State Department of Health Services (DHS). In addition to the new analytic techniq...

journal_title：Statistics in medicine

authors： Merrill DW

更新日期：2001-05-15 00:00:00

abstract：:In vaccine safety studies, subjects are considered at increased risk for adverse events for a period of time after vaccination known as risk window. To our knowledge, risk windows for vaccine safety studies have tended to be pre-defined and not to use information from the current study. Inaccurate specification of the...

journal_title：Statistics in medicine

authors： Xu S,Zhang L,Nelson JC,Zeng C,Mullooly J,McClure D,Glanz J

更新日期：2011-03-30 00:00:00