An extension of the continual reassessment method using decision theory.

abstract：:One Sentence Summary: A Bayesian repeated measures model based on quantitative muscle strength data from a prospective Natural History Study was developed to determine disease progression and design clinical trials for GNE myopathy, a rare and slowly progressive muscle disease. GNE myopathy is a rare muscle disease ch...

journal_title：Statistics in medicine

authors： Quintana M,Shrader J,Slota C,Joe G,McKew JC,Fitzgerald M,Gahl WA,Berry S,Carrillo N

更新日期：2019-04-15 00:00:00

abstract：:We extend the shared frailty model of recurrent events and a dependent terminal event to allow for a nonparametric covariate function. We include a Gaussian random effect (frailty) in the intensity functions of both the recurrent and terminal events to capture correlation between the two processes. We employ the penal...

journal_title：Statistics in medicine

authors： Zhangsheng Y,Liu L

更新日期：2011-09-30 00:00:00

abstract：:There are inconsistencies between the formulas for the variance of standardized mean difference (SMD) in the Cochrane Handbook for Systematic Reviews and the variance reported in other sources. Instead of the variance appropriate for the SMD of a crossover experiment, the Cochrane Handbook uses the variance appropriat...

journal_title：Statistics in medicine

authors： Kitchenham B,Madeyski L

更新日期：2020-11-30 00:00:00

abstract：:Multiple imputation by chained equations is a flexible and practical approach to handling missing data. We describe the principles of the method and show how to impute categorical and quantitative variables, including skewed variables. We give guidance on how to specify the imputation model and how many imputations ar...

journal_title：Statistics in medicine

authors： White IR,Royston P,Wood AM

更新日期：2011-02-20 00:00:00

abstract：:Comparative Poisson trials of prophylactic interventions, such as vaccines, can be lengthy and costly. We evaluate two easily implemented approaches to reduce numbers of disease cases and person years of follow up (N(u+t)) for comparative Poisson trials with fixed numbers of cases (T); (i) altering k the portion of N(...

journal_title：Statistics in medicine

authors： Hoover DR

更新日期：2004-04-30 00:00:00

abstract：:The relative importance of prognostic factors in regression can be measured either by standardized regression coefficients or by percentages of explained variation in a dependent variable. One advantage of using explained variation is the direct comparability of qualitative prognostic factors with others, or of groups...

journal_title：Statistics in medicine

authors： Schemper M

更新日期：1993-12-30 00:00:00

abstract：:The goal of phase I cancer trials is to determine the highest dose of a treatment regimen with an acceptable toxicity rate. Traditional designs for phase I trials, such as the Continual Reassessment Method (CRM) and the 3 + 3 design, require each patient or a cohort of patients to be fully evaluated for the dose-limit...

journal_title：Statistics in medicine

authors： Polley MY

更新日期：2011-07-30 00:00:00

abstract：:Biomarkers, increasingly used in biomedical studies for the diagnosis and prognosis of acute and chronic diseases, provide insight into the effectiveness of treatments and potential pathways that can be used to guide future treatment targets. The measurement of these markers is often limited by the sensitivity of the ...

journal_title：Statistics in medicine

authors： Lee M,Kong L,Weissfeld L

更新日期：2012-07-30 00:00:00

abstract：:Although radical changes in drug regulation are rare (e.g., the Federal Food, Drug and Cosmetic Act of 1938 and the 1962 amendment to the Act creating an effectiveness requirement), regulations and guidance do evolve significantly in the face of new problems and accumulating experience. Recent changes have been driven...

journal_title：Statistics in medicine

authors： Temple R

更新日期：2002-10-15 00:00:00

abstract：:In this article, we investigate procedures for comparing two independent Poisson variates that are observed over unequal sampling frames (i.e. time intervals, populations, areas or any combination thereof). We consider two statistics (with and without the logarithmic transformation) for testing the equality of two Poi...

journal_title：Statistics in medicine

authors： Ng HK,Tang ML

更新日期：2005-03-30 00:00:00

abstract：:In biomedical research such as the development of vaccines for infectious diseases or cancer, study outcomes measured by an assay or device are often collected from multiple sources or laboratories. Measurement error that may vary between laboratories needs to be adjusted for when combining samples across data sources...

journal_title：Statistics in medicine

authors： Huang Y,Huang Y,Moodie Z,Li S,Self S

更新日期：2012-12-10 00:00:00

abstract：:This paper, arising out of an event to honour the statistical and scientific contributions of Professor Peter Armitage, is concerned with research strategies and needs for chronic disease prevention. A few highlights from recent intervention trials for the prevention of cancer, cardiovascular disease, fractures and di...

journal_title：Statistics in medicine

authors： Prentice RL

更新日期：2004-11-30 00:00:00

abstract：:We analyse the effect of using prevalence rates based on populations with different sizes in the power of spatial independence tests. We compare the well known spatial correlation Moran's index to three indexes obtained after adjusting for population density, one proposed by Oden, another proposed by Waldhör, and a th...

journal_title：Statistics in medicine

authors： Assunção RM,Reis EA

更新日期：1999-08-30 00:00:00

abstract：:Methods for addressing multiplicity in clinical trials have attracted much attention during the past 20 years. They include the investigation of new classes of multiple test procedures, such as fixed sequence, fallback and gatekeeping procedures. More recently, sequentially rejective graphical test procedures have bee...

journal_title：Statistics in medicine

authors： Maurer W,Bretz F

更新日期：2013-05-10 00:00:00

abstract：:Assay sensitivity has been proposed as a criterion for including psychiatric clinical outcome studies in meta-analyses. The authors assess the performance of assay sensitivity as a method for determining study appropriateness for meta-analysis by calculating expected standard drug vs placebo effect sizes for various c...

journal_title：Statistics in medicine

authors： Gelfand LA,Strunk DR,Tu XM,Noble RE,Derubeis RJ

更新日期：2006-03-30 00:00:00

abstract：:In some randomized controlled trials, subjects with a better prognosis may be diverted into the treatment group. This subverting of randomization results in an unobserved non-compliance with the originally intended treatment assignment. Consequently, the estimate of treatment effect from these trials may be biased. Th...

journal_title：Statistics in medicine

authors： Marcus SM

更新日期：2001-02-28 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：:We consider the problem of comparing cumulative incidence functions of non-mortality events in the presence of informative coarsening and the competing risk of death. We extend frequentist-based hypothesis tests previously developed for non-informative coarsening and propose a novel Bayesian method based on comparing ...

journal_title：Statistics in medicine

authors： Shardell M,Scharfstein DO,Vlahov D,Galai N

更新日期：2008-12-10 00:00:00

abstract：:Hot-deck imputation is an intuitively simple and popular method of accommodating incomplete data. Users of the method will often use the usual multiple imputation variance estimator which is not appropriate in this case. However, no variance expression has yet been derived for this easily implemented method applied to...

journal_title：Statistics in medicine

authors： Reilly M,Pepe M

更新日期：1997-01-15 00:00:00

abstract：:This paper considers the regression analysis of CD4 cell counts, a commonly used indicator and prognostic factor of AIDS progression. For this purpose, a number of methods have been proposed and most of them are based on random effects models. We present an alternative that is based on a mean function regression model...

journal_title：Statistics in medicine

authors： Sun J

更新日期：2001-11-15 00:00:00

abstract：:Population heterogeneity is frequently observed among patients' treatment responses in clinical trials because of various factors such as clinical background, environmental, and genetic factors. Different subpopulations defined by those baseline factors can lead to differences in the benefit or safety profile of a the...

journal_title：Statistics in medicine

authors： Chiu YD,Koenig F,Posch M,Jaki T

更新日期：2018-12-20 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：:Screening for a disease such as cancer is often regarded as a beneficial and successful strategy for reducing mortality. However, as with any clinical treatment or intervention, benefit cannot be assumed, and screening can entail both costs and harms, so the screening as a 'treatment' must undergo evaluation. An evalu...

journal_title：Statistics in medicine

authors： Kafadar K,Prorok PC

更新日期：2005-02-28 00:00:00

abstract：:We discuss a sample size calculation for a pair-matched case-control study to test for interaction between a specific exposure and a second risk factor. The second risk factor could be either binary or continuous. An algorithm for the calculation of sample size is suggested which is based on a logistic regression mode...

journal_title：Statistics in medicine

authors： Qiu P,Moeschberger ML,Cooke GE,Goldschmidt-Clermont PJ

更新日期：2000-04-15 00:00:00

abstract：:Propensity score analysis is widely used in observational studies to adjust for confounding and estimate the causal effect of a treatment on the outcome. When the outcome is survival time, there are special considerations on the definition of the causal estimand, point, and variance estimation that have not been thoro...

journal_title：Statistics in medicine

authors： Mao H,Li L,Yang W,Shen Y

更新日期：2018-11-20 00:00:00

abstract：:One may encounter the application of the two independent samples t-test to ordinal scaled data (for example, data that assume only the values 0, 1, 2, 3) from small samples. This situation clearly violates the underlying normality assumption for the t-test and one cannot appeal to large sample theory for validity. In ...

journal_title：Statistics in medicine

authors： Heeren T,D'Agostino R

更新日期：1987-01-01 00:00:00

abstract：:Sam Greenhouse joined the Census Bureau as a clerk at an interesting time period for the agency. The first use of sampling in the decennial census occurred in 1940. There was a major expansion of the amount of data collected. The organization of the Census Bureau underwent radical changes, including the growth of the ...

journal_title：Statistics in medicine

authors： Keller J,Clark CZ

更新日期：2003-11-15 00:00:00

abstract：:In observational studies, many continuous or categorical covariates may be related to an outcome. Various spline-based procedures or the multivariable fractional polynomial (MFP) procedure can be used to identify important variables and functional forms for continuous covariates. This is the main aim of an explanatory...

journal_title：Statistics in medicine

authors： Binder H,Sauerbrei W,Royston P

更新日期：2013-06-15 00:00:00

abstract：:The randomized clinical trial, LU19, conducted by the Medical Research Council Lung Cancer Working Party, was designed to compare ACE (doxorubicin, cyclophosphamide and etoposide) chemotherapy plus G-CSF (granulocyte colony-stimulating factor) at 2-week intervals versus ACE chemotherapy alone at standard 3-week interv...

journal_title：Statistics in medicine

authors： Qian W,Parmar MK,Sambrook RJ,Fayers PM,Girling DJ,Stephens RJ

更新日期：2000-10-15 00:00:00

abstract：:We present a method to determine the number of subjects, k, and number of repeated measurements, n, that minimize the overall cost of conducting a reliability study, while providing acceptable power for tests of hypotheses concerning the reliability coefficient rho. Tables showing optimal choices of k and n under vari...

journal_title：Statistics in medicine

authors： Eliasziw M,Donner A

更新日期：1987-09-01 00:00:00