Assurance calculations for planning clinical trials with time-to-event outcomes.

abstract：:When data are dichotomous, this paper notes the utility of inverse sampling in establishing equivalence with respect to the risk ratio. This paper develops an exact equivalence test that accounts for the risk ratio under inverse sampling and further discusses the relationship between the exact equivalence test and the...

journal_title：Statistics in medicine

authors： Lui KJ

更新日期：1997-08-15 00:00:00

abstract：:Biomarkers that predict the efficacy of treatment can potentially improve clinical outcomes and decrease medical costs by allowing treatment to be provided only to those most likely to benefit. We consider the design of a randomized clinical trial in which one objective is to evaluate a treatment selection marker. The...

journal_title：Statistics in medicine

authors： Janes H,Brown MD,Pepe MS

更新日期：2015-11-30 00:00:00

abstract：:This paper is a compendium of exact and asymptotic formulae and tables for estimating the sample size in a clinical trial with two treatment groups and a dichotomous outcome. The paper provides separate formulae for equal and unequal treatment group sizes, formulae for the calculation of power given the sample size, a...

journal_title：Statistics in medicine

authors： Sahai H,Khurshid A

更新日期：1996-01-15 00:00:00

abstract：:Unmeasured confounding is a common concern when researchers attempt to estimate a treatment effect using observational data or randomized studies with nonperfect compliance. To address this concern, instrumental variable methods, such as 2-stage predictor substitution (2SPS) and 2-stage residual inclusion (2SRI), have...

journal_title：Statistics in medicine

authors： Wan F,Small D,Mitra N

更新日期：2018-05-30 00:00:00

abstract：:Frequently, multiple doses of an active treatment and multiple endpoints are simultaneously considered in the designs of clinical trials. For these trials, traditional multiplicity adjustment procedures such as Bonferroni, Hochberg and Hommel procedures can be applied when treating the comparisons of different doses t...

journal_title：Statistics in medicine

authors： Quan H,Luo X,Capizzi T

更新日期：2005-07-30 00:00:00

abstract：:There is an increasing interest in the use of propensity score methods to estimate causal effects in observational studies. However, recent systematic reviews have demonstrated that propensity score methods are inconsistently used and frequently poorly applied in the medical literature. In this study, we compared the ...

journal_title：Statistics in medicine

authors： Austin PC,Mamdani MM

更新日期：2006-06-30 00:00:00

abstract：:In medical and health studies, heterogeneities in clustered count data have been traditionally modeled by positive random effects in Poisson mixed models; however, excessive zeros often occur in clustered medical and health count data. In this paper, we consider a three-level random effects zero-inflated Poisson model...

journal_title：Statistics in medicine

authors： Ma R,Hasan MT,Sneddon G

更新日期：2009-08-15 00:00:00

abstract：:Korn and Whittemore have presented methods for analyzing longitudinal data where the number of observations per individual is large relative to the number of variables considered for each subject. However, this is often not the case in epidemiologic studies, since one usually collects data at relatively few time point...

journal_title：Statistics in medicine

authors： Rosner B,Muñoz A,Tager I,Speizer F,Weiss S

更新日期：1985-10-01 00:00:00

abstract：:The principles and historical development of clinical trials are outlined briefly. Some issues that extend the basic philosophy first put forward by Sir Austin Bradford Hill 40 years ago and that will need consideration in the future application of clinical trials are discussed. ...

journal_title：Statistics in medicine

authors： Holland WW,Breeze E,Swan AV

更新日期：1982-10-01 00:00:00

abstract：:Equivalence trials aim to show that two treatments have equivalent therapeutic effects. The approach is to define, in advance, a range of equivalence -d to +d for the treatment difference such that any value in the range is clinically unimportant. If the confidence interval for the difference, calculated after the tri...

journal_title：Statistics in medicine

authors： Ebbutt AF,Frith L

更新日期：1998-08-15 00:00:00

abstract：:Much has been published on various aspects of data analysis and reporting from clinical trials within the biopharmaceutical environment. This ranges from regulatory guidelines on the format and content of registration dossiers to recommendations on data presentation and the statistical methodologies that are appropria...

journal_title：Statistics in medicine

authors： Huster WJ,Enas GG

更新日期：1998-08-15 00:00:00

abstract：:Recently, Wu and Follmann developed summary measures to adjust for informative drop-out in longitudinal studies where drop-out depends on the underlying true value of the response. In this paper we evaluate these procedures in the common situation where drop-out depends on the observed responses. We also discuss vario...

journal_title：Statistics in medicine

authors： Wu MC,Albert PS,Wu BU

更新日期：2001-01-15 00:00:00

abstract：:Interpretation of the Mantoux test for tuberculous infection can be complicated by cross-reactions caused by infection with non-specific mycobacteria. Thus, the distribution of positive indurations is a mixture of two distributions. To estimate tuberculous infection prevalence, the marginal distribution of indurations...

journal_title：Statistics in medicine

authors： Nagelkerke NJ,Borgdorff MW,Kim SJ

更新日期：2001-04-15 00:00:00

abstract：:Non-tumor cell-based model systems have recently gained interest in pharmacogenetic research as a hypothesis generating tool. The hypotheses generated from these model systems can be followed up in functional studies, or tested in individuals taking the same investigational agents. The current cellular phenotypes (e.g...

journal_title：Statistics in medicine

authors： Fridley BL,Jenkins G,Schaid DJ,Wang L

更新日期：2009-09-20 00:00:00

abstract：:This paper models monthly AIDS diagnosis counts in terms of smooth secular trend, calendar month effects, and the number of workdays per month. A parameterization of month effects allows separation of true seasonal effects from a linear trend over the calendar year and an arbitrary June effect. There is strong evidenc...

journal_title：Statistics in medicine

authors： Bacchetti P

更新日期：1994-10-15 00:00:00

abstract：:Results from an analysis of traffic accidents from a study of the police records of four police stations in the Bangkok metropolis are presented. The main emphasis in this study was put on the development of a measure for traffic accident density. The traffic flow was estimated at the various study locations by traine...

journal_title：Statistics in medicine

authors： Ayuthya RS,Böhning D

更新日期：1995-11-15 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：:The most popular model used for survival analysis is the proportional hazards regression model proposed by Cox. This is mainly due to its exceptional simplicity. Nevertheless the fundamental assumption of the Cox model is the proportionality of the hazards. For many applications, however, this assumption is doubtful. ...

journal_title：Statistics in medicine

authors： Berger U,Schäfer J,Ulm K

更新日期：2003-04-15 00:00:00

abstract：:When a cancer patient develops a new tumor it is necessary to determine if it is a recurrence (metastasis) of the original cancer, or an entirely new occurrence of the disease. This is accomplished by assessing the histo-pathology of the lesions. However, there are many clinical scenarios in which this pathological di...

journal_title：Statistics in medicine

authors： Ostrovnaya I,Olshen AB,Seshan VE,Orlow I,Albertson DG,Begg CB

更新日期：2010-07-10 00:00:00

abstract：:A frequent objective in medical research is the investigation of differences in patient survival between several experimental treatments and one standard treatment. In order to assess these differences statistically, we have to apply adjustments for multiple comparisons to prevent an increased number of false-positive...

journal_title：Statistics in medicine

authors： Herberich E,Hothorn T

更新日期：2012-01-13 00:00:00

abstract：:In this paper, we construct a partial additive regression (PAR) model to predict the survival times of cancer patients based on microarray gene expression data with right censoring. The area under time-dependent receiver operating characteristic curve is used as a model evaluation criterion. We conduct a simulation st...

journal_title：Statistics in medicine

authors： Zhao Y,Zhou Y,Zhao M

更新日期：2009-01-30 00:00:00

abstract：:Although the frequentist paradigm has been the predominant approach to clinical trial design since the 1940s, it has several notable limitations. Advancements in computational algorithms and computer hardware have greatly enhanced the alternative Bayesian paradigm. Compared with its frequentist counterpart, the Bayesi...

journal_title：Statistics in medicine

authors： Lee JJ,Chu CT

更新日期：2012-11-10 00:00:00

abstract：:In this paper we examine the efficiency of a generalization of the traditional normal linear (LDA) or quadratic (QDA) discriminant analysis. This procedure (the generalized discriminant analysis, GDA) replaces each normal density used in the traditional classification rule by a Fourier series density estimator which '...

journal_title：Statistics in medicine

authors： Odom-Maryon T,Langholz B,Niland J,Azen S

更新日期：1991-03-01 00:00:00

abstract：:We simulated multinomial AIDS incidence counts from 27 'representative' AIDS epidemics that spanned a period corresponding to previous applications of backcalculation (1 January 1977 to 1 July 1987) and assessed mean square error for several back-calculated estimators of HIV prevalence and short-term AIDS projections....

journal_title：Statistics in medicine

authors： Rosenberg PS,Gail MH,Pee D

更新日期：1991-08-01 00:00:00

abstract：:Clinical studies that involve the recording of two or more distinct and well-defined events on each subject give rise to multiple event data. Treatment comparisons are usually reported in univariate analyses of time to first event or number of events observed. However, this approach may not uncover the 'full story' of...

journal_title：Statistics in medicine

authors： Barai U,Teoh N

更新日期：1997-04-30 00:00:00

abstract：:In this paper we give an informal introduction to a robust method for survival analysis which is based on a modification of the usual partial likelihood estimator (PLE). Large sample results lead us to expect reduced bias for this robust estimator compared with the PLE whenever there are even slight violations of the ...

journal_title：Statistics in medicine

authors： Minder CE,Bednarski T

更新日期：1996-05-30 00:00:00

abstract：:A method is presented which allows us to adapt the sample size as well as the number and time points of interim analyses to the treatment difference observed at an interim look during the course of a clinical trial with censored survival time as the endpoint. The method allows the inclusion of data inspections during ...

journal_title：Statistics in medicine

authors： Schäfer H,Müller HH

更新日期：2001-12-30 00:00:00

abstract：:The serial dilution assay (for example, an in vitro antimicrobic susceptibility test or a serum antibody titer assay) is an important technique in biomedical research. The structure of the experiment forces grouping of the threshold concentrations into intervals. Statistical methods to analyse threshold concentrations...

journal_title：Statistics in medicine

authors： Hamilton MA,Rinaldi MG

更新日期：1988-04-01 00:00:00

abstract：:The two tailed Fisher's exact P value is extremely sensitive to small perturbations in 2 x 2 contingency tables. An example indicates that a 1 per cent increase in the denominator of one treatment group results in a 32 per cent drop in the exact P value, but a mere 0.1 per cent decrease in the treatment success rate. ...

journal_title：Statistics in medicine

authors： Dupont WD

更新日期：1986-11-01 00:00:00

abstract：:In biomedical studies and clinical trials, repeated measures are often subject to some upper and/or lower limits of detection. Hence, the responses are either left or right censored. A complication arises when more than one series of responses is repeatedly collected on each subject at irregular intervals over a perio...

journal_title：Statistics in medicine

authors： Lachos VH,A Matos L,Castro LM,Chen MH

更新日期：2019-03-15 00:00:00