Utility-based optimization of phase II/III programs.

abstract：:Flexible survival models are in need when modelling data from long term follow-up studies. In many cases, the assumption of proportionality imposed by a Cox model will not be valid. Instead, a model that can identify time varying effects of fixed covariates can be used. Although there are several approaches that deal ...

journal_title：Statistics in medicine

authors： Perperoglou A

更新日期：2016-12-10 00:00:00

abstract：:The use of classification and regression tree (CART) methodology is explored for the diagnosis of patients complaining of anterior chest pain. The results are compared with those previously obtained using correspondence analysis and independent Bayes classification. The technique is shown to be of potential value for ...

journal_title：Statistics in medicine

authors： Crichton NJ,Hinde JP,Marchini J

更新日期：1997-04-15 00:00:00

abstract：:Cross-sectional designs are often used to monitor the proportion of infections and other post-surgical complications acquired in hospitals. However, conventional methods for estimating incidence proportions when applied to cross-sectional data may provide estimators that are highly biased, as cross-sectional designs t...

journal_title：Statistics in medicine

authors： Fluss R,Mandel M,Freedman LS,Weiss IS,Zohar AE,Haklai Z,Gordon ES,Simchen E

更新日期：2013-06-30 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：:The validity and practical utility of observational medical research depends critically on good study design, excellent data quality, appropriate statistical methods and accurate interpretation of results. Statistical methodology has seen substantial development in recent times. Unfortunately, many of these methodolog...

journal_title：Statistics in medicine

authors： Sauerbrei W,Abrahamowicz M,Altman DG,le Cessie S,Carpenter J,STRATOS initiative.

更新日期：2014-12-30 00:00:00

abstract：:Systematic reviews often provide recommendations for further research. When meta-analyses are inconclusive, such recommendations typically argue for further studies to be conducted. However, the nature and amount of future research should depend on the nature and amount of the existing research. We propose a method ba...

journal_title：Statistics in medicine

authors： Roloff V,Higgins JP,Sutton AJ

更新日期：2013-01-15 00:00:00

abstract：:New combination regimens evaluated in phase II cancer clinical trials often show promising results compared to the standard therapy for a disease system. Selection of patients with a better prognosis can be a prominent factor for this optimism. For most disease systems, prognostic variables that are related to the out...

journal_title：Statistics in medicine

authors： Mazumdar M,Fazzari M,Panageas KS

更新日期：2001-03-30 00:00:00

abstract：:In randomised trials, continuous endpoints are often measured with some degree of error. This study explores the impact of ignoring measurement error and proposes methods to improve statistical inference in the presence of measurement error. Three main types of measurement error in continuous endpoints are considered:...

journal_title：Statistics in medicine

authors： Nab L,Groenwold RHH,Welsing PMJ,van Smeden M

更新日期：2019-11-30 00:00:00

abstract：:We consider recurrent events of the same type that occur during alternating restraint and non-restraint time periods. This research is motivated by a study on juvenile recidivism, where the probationers were followed for re-offenses during alternating placement periods and free-time periods. During the placement perio...

journal_title：Statistics in medicine

authors： Li X,Chen Y,Li R

更新日期：2017-02-20 00:00:00

abstract：:Equal allocation of patients to treatment in a randomized clinical trial may have disadvantages ethically if the new treatment is believed to be at least as beneficial as the standard treatment. Others have considered, in a non-sequential setting, unbalanced randomized designs which allocate fewer patients to the pote...

journal_title：Statistics in medicine

authors： Lesser ML

更新日期：1982-07-01 00:00:00

abstract：:The Rvachev-Baroyan-Longini model is a space-time predictive model of the spread of influenza epidemics. It has been applied to 128 cities of the USSR, and more recently, to forecasting the spread of the pandemic of 1968-1969 throughout 52 large cities. It is a deterministic, mass-action, space and time continuous mod...

journal_title：Statistics in medicine

authors： Flahault A,Letrait S,Blin P,Hazout S,Ménarés J,Valleron AJ

更新日期：1988-11-01 00:00:00

abstract：:We propose a joint modeling approach to investigating the observed and latent risk factors of mixed types of outcomes. The proposed model comprises three parts. The first part is an exploratory factor analysis model that summarizes latent factors through multiple observed variables. The second part is a proportional h...

journal_title：Statistics in medicine

authors： Pan D,Wei Y,Song X

更新日期：2020-12-09 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：:The case-control study is a simple and an useful method to characterize the effect of a gene, the effect of an exposure, as well as the interaction between the two. The control-free case-only study is yet an even simpler design, if interest is centered on gene-environment interaction only. It requires the sometimes pl...

journal_title：Statistics in medicine

authors： Lee WC,Wang LY,Cheng KF

更新日期：2010-10-30 00:00:00

abstract：:We have used Monte Carlo methods to compare the type I error properties of the conditional and unconditional versions of the generalized t and the generalized rank-sum tests to those of the independent samples t and Wilcoxon rank-sum tests. Results showed inflated type I errors for the conditional generalized tests bu...

journal_title：Statistics in medicine

authors： Blair RC,Morel JG

更新日期：1992-02-28 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：: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：:Maps of estimated disease rates over multiple time periods are useful tools for gaining etiologic insights regarding potential exposures associated with specific locations and times. In this paper, we describe an extension of the Gangnon-Clayton model for spatial clustering to spatio-temporal data. As in the purely sp...

journal_title：Statistics in medicine

authors： Gangnon RE

更新日期：2010-09-30 00:00:00

abstract：:The stability-controlled quasi-experiment (SCQE) is an approach to study the effects of nonrandomized, newly adopted treatments. While covariate adjustment techniques rely on a "no unobserved confounding" assumption, SCQE imposes an assumption on the change in the average nontreatment outcome between successive cohort...

journal_title：Statistics in medicine

authors： Hazlett C,Maokola W,Wulf DA

更新日期：2020-12-10 00:00:00

abstract：:Most statistical methodology for phase III clinical trials focuses on the comparison of a single experimental treatment with a control. An increasing desire to reduce the time before regulatory approval of a new drug is sought has led to development of two-stage or sequential designs for trials that combine the defini...

journal_title：Statistics in medicine

authors： Stallard N,Todd S

更新日期：2003-03-15 00:00:00

abstract：:A common problem encountered in many medical applications is the comparison of survival curves. Often, rather than comparison of the entire survival curves, interest is focused on the comparison at a fixed point in time. In most cases, the naive test based on a difference in the estimates of survival is used for this ...

journal_title：Statistics in medicine

authors： Klein JP,Logan B,Harhoff M,Andersen PK

更新日期：2007-10-30 00:00:00

abstract：:Orthogonal polynomial scores (OPS) is a simple, biologically meaningful approach to characterize longitudinal data in phase I and II clinical pharmacology trials. It describes average, linear, quadratic and higher order polynomial characteristics of each subject's response over time with use of composite scores comput...

journal_title：Statistics in medicine

authors： Bradstreet TE

更新日期：1993-04-15 00:00:00

abstract：:In Part I we presented a covariance structure model for analysing measurement error in the assessment of nitrogen intake. In this paper we include data on urine nitrogen excretion which allows a critical assessment of the model proposed. Inclusion of urine nitrogen data produces more pessimistic estimates of the quali...

journal_title：Statistics in medicine

authors： Plummer M,Clayton D

更新日期：1993-05-30 00:00:00

abstract：:Important differences between explanatory and pragmatic studies were originally argued by Schwartz and Lellouch. Three important differences between the two types of study involve study control, study violators and inclusion criteria. It was originally argued that explanatory studies are highly controlled, and pragmat...

journal_title：Statistics in medicine

authors： McMahon AD

更新日期：2002-05-30 00:00:00

abstract：:We investigate population-averaged (PA) and cluster-specific (CS) associations for clustered binary logistic regression in the context of a longitudinal clinical trial that investigated the association between tooth-specific visual elastase kit results and periodontal disease progression within 26 weeks of follow-up. ...

journal_title：Statistics in medicine

authors： Ten Have TR,Landis JR,Weaver SL

更新日期：1995-02-28 00:00:00

abstract：:In the presence of confounding, the consistency assumption required for identification of causal effects may be violated due to misclassification of the outcome variable. We introduce an inverse probability weighted approach to rebalance covariates across treatment groups while mitigating the influence of differential...

journal_title：Statistics in medicine

authors： Gravel CA,Platt RW

更新日期：2018-02-10 00:00:00

abstract：:This paper discusses regression analysis of multivariate current status failure time data (The Statistical Analysis of Interval-censoring Failure Time Data. Springer: New York, 2006), which occur quite often in, for example, tumorigenicity experiments and epidemiologic investigations of the natural history of a diseas...

journal_title：Statistics in medicine

authors： Chen MH,Tong X,Sun J

更新日期：2009-11-30 00:00:00

abstract：:In this commentary, we revisit Sir Austin Bradford Hill's seminal Alfred Watson Memorial Lecture in 1962 through the eyes of two practicing biostatisticians of the current era. We summarize some eternal takeaway messages from Hill's lecture regarding observations and experiments translated through the modern lexicon o...

journal_title：Statistics in medicine

authors： Dempsey W,Mukherjee B

更新日期：2021-01-15 00:00:00

abstract：:We describe the usual statistical concepts and consequent appropriate simulations of a prospective study for the simple case of a single risk variable and an assumed logistic model. We examine the simulations of Lilienfeld and Pyne, and show that they are seriously flawed. Contrary to those authors' claims, the estima...

journal_title：Statistics in medicine

authors： Halperin M,Abbott RD,Blackwelder WC,Jacobowitz R,Lan KK,Verter JI,Wedel H

更新日期：1985-04-01 00:00:00

abstract：:Motivated by a study about prompt coronary angiography in myocardial infarction, we propose a method to estimate the causal effect of a treatment in two-arm experimental studies with possible noncompliance in both treatment and control arms. We base the method on a causal model for repeated binary outcomes (before and...

journal_title：Statistics in medicine

authors： Bartolucci F,Farcomeni A

更新日期：2013-11-10 00:00:00