An evaluation of phase I clinical trial designs in the continuous dose-response setting.

abstract：:Service on the Data Monitoring Committee of the CPEP (Calcium for Pre-eclampsia Prevention) has led us to four conclusions about clinical trials which we should like to present to this gathering of biostatisticians for their reactions: (i) meta-analyses of the pertinent published trials of the same therapy should alwa...

journal_title：Statistics in medicine

authors： Chalmers TC,Lau J

更新日期：1996-06-30 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：:The National Children's Study is a national household probability sample designed to identify 100,000 children at birth and follow the sampled children for 21 years. Data from the study will support examining numerous hypotheses concerning genetic and environmental effects on the health and development of children. Th...

journal_title：Statistics in medicine

authors： Montaquila JM,Brick JM,Curtin LR

更新日期：2010-06-15 00:00:00

abstract：:The receiver operating characteristic (ROC) curve can be utilized to evaluate the performance of diagnostic tests. The area under the ROC curve (AUC) is a widely used summary index for comparing multiple ROC curves. Both parametric and nonparametric methods have been developed to estimate and compare the AUCs. However...

journal_title：Statistics in medicine

authors： Yao W,Li Z,Graubard BI

更新日期：2015-04-15 00:00:00

abstract：:Statistical inference based on correlated count measurements are frequently performed in biomedical studies. Most of existing sample size calculation methods for count outcomes are developed under the Poisson model. Deviation from the Poisson assumption (equality of mean and variance) has been widely documented in pra...

journal_title：Statistics in medicine

authors： Li D,Zhang S,Cao J

更新日期：2019-12-10 00:00:00

abstract：:Methodology for the meta-analysis of individual patient data with survival end-points is proposed. Motivated by questions about the reliance on hazard ratios as summary measures of treatment effects, a parametric approach is considered and percentile ratios are introduced as an alternative to hazard ratios. The genera...

journal_title：Statistics in medicine

authors： Siannis F,Barrett JK,Farewell VT,Tierney JF

更新日期：2010-12-20 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：:Estimates of analyte concentrations, obtained by immunoassay, have error distributions which are generally underestimated. Better estimates, which take into account the distribution of the response metameter of the calibration curve and uncertainties associated with the location of the fitted curve, have been obtained...

journal_title：Statistics in medicine

authors： Kay C,Nix AB,Kemp KW,Rowlands RJ,Richards G,Groom GV,Griffiths K,Wilson DW

更新日期：1986-03-01 00:00:00

abstract：:To update the British growth reference, anthropometric data for weight, height, body mass index (weight/height2) and head circumference from 17 distinct surveys representative of England, Scotland and Wales (37,700 children, age range 23 weeks gestation to 23 years) were analysed by maximum penalized likelihood using ...

journal_title：Statistics in medicine

authors： Cole TJ,Freeman JV,Preece MA

更新日期：1998-02-28 00:00:00

abstract：:In record linkage studies, unique identifiers are often not available, and therefore, the linkage procedure depends on combinations of partially identifying variables with low discriminating power. As a consequence, wrongly linked covariate and outcome pairs will be created and bias further analysis of the linked data...

journal_title：Statistics in medicine

authors： Hof MH,Zwinderman AH

更新日期：2012-12-30 00:00:00

abstract：:In an individually matched case-control study, effects of potential risk factors are ascertained through conditional logistic regression (CLR). Extension of CLR to situations with multiple disease or reference categories has been made through polychotomous CLR and is shown to be more efficient than carrying out separa...

journal_title：Statistics in medicine

authors： Mukherjee B,Liu I,Sinha S

更新日期：2007-07-30 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：:This field study sought to measure the effects of dental delivery and school-based, dental health education on use of dental health care by children in grades K-6. We attempted to control for two potential confounding factors by an approximate randomization of children into treatment groups with stratification on grad...

journal_title：Statistics in medicine

authors： Oler J,Bentley JM

更新日期：1983-01-01 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：:Graphical methods based on the analysis of differences between log cumulative hazard functions are considered for a two-group semi-proportional hazard model which allows for interaction between treatments and covariates. Confidence procedures and test statistics that can be used to test for interaction and for main ef...

journal_title：Statistics in medicine

authors： Dabrowska DM,Doksum KA,Feduska NJ,Husing R,Neville P

更新日期：1992-08-01 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：:Appropriate quantification of added usefulness offered by new markers included in risk prediction algorithms is a problem of active research and debate. Standard methods, including statistical significance and c statistic are useful but not sufficient. Net reclassification improvement (NRI) offers a simple intuitive w...

journal_title：Statistics in medicine

authors： Pencina MJ,D'Agostino RB Sr,Steyerberg EW

更新日期：2011-01-15 00:00:00

abstract：:The use of both sequential designs and adaptive treatment allocation are effective in reducing the number of patients receiving an inferior treatment in a clinical trial. In large samples, when the asymptotic normality of test statistics can be utilized, a standard sequential design can be combined with adaptive alloc...

journal_title：Statistics in medicine

authors： Stallard N,Rosenberger WF

更新日期：2002-02-28 00:00:00

abstract：:A statistical definition of surrogate endpoints as well as validation criteria was first presented by Prentice. Freedman et al. supplemented these criteria with the so-called proportion explained. Buyse and Molenberghs pointed to inadequacies of these criteria and suggested a new definition of surrogacy based on (i) t...

journal_title：Statistics in medicine

authors： Molenberghs G,Geys H,Buyse M

更新日期：2001-10-30 00:00:00

abstract：:We consider Cox proportional hazards regression when the covariate vector includes error-prone discrete covariates along with error-free covariates, which may be discrete or continuous. The misclassification in the discrete error-prone covariates is allowed to be of any specified form. Building on the work of Nakamura...

journal_title：Statistics in medicine

authors： Zucker DM,Spiegelman D

更新日期：2008-05-20 00:00:00

abstract：:Competing compartment models of different complexities have been used for the quantitative analysis of dynamic contrast-enhanced magnetic resonance imaging data. We present a spatial elastic net approach that allows to estimate the number of compartments for each voxel such that the model complexity is not fixed a pri...

journal_title：Statistics in medicine

authors： Sommer JC,Gertheiss J,Schmid VJ

更新日期：2014-03-15 00:00:00

abstract：:The introduction of potent antiretroviral therapies for treatment of HIV infection typically results in a dramatic reduction in plasma HIV RNA concentration, often to levels undetectable by current measurement practices. However, although a high proportion of patients achieve 'undetectability', many then experience a ...

journal_title：Statistics in medicine

authors： McKinnon EJ,James IR,John M,Mallal SA

更新日期：2003-02-15 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：:We propose the use of Gaussian mixed models to analyse statistically 24 hour ambulatory blood pressure data from clinical trials. We develop specific models and apply them to data from a clinical study that compares two angiotensin-converting enzyme inhibitors. We investigate and discuss computing issues related to th...

journal_title：Statistics in medicine

authors： Selwyn MR,Difranco DM

更新日期：1993-09-30 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：:We compared eight nominal 95 per cent confidence intervals for the ratio of two Poisson parameters, both assumed small, on their true coverage (the probability that the interval includes the ratio of Poisson parameters) and median width. The commonly used log-linear interval, justified by asymptotic considerations, pr...

journal_title：Statistics in medicine

authors： Barker L,Cadwell BL

更新日期：2008-09-10 00:00:00

abstract：:R2 has been criticized as a measure of model performance when predicting a dichotomous outcome, both because its value is often low and because it is sensitive to the prevalence of the event of interest. The C statistic is more widely used to measure model performance in a 0/1 setting. We use a simple parametric famil...

journal_title：Statistics in medicine

authors： Ash A,Shwartz M

更新日期：1999-02-28 00:00:00

abstract：:Mediation analysis is a standard approach to understanding how and why an intervention works in social and medical sciences. However, the presence of missing data, especially missing not at random data, poses a great challenge for the applicability of this approach in practice. Current methods for handling such missin...

journal_title：Statistics in medicine

authors： Li W,Zhou XH

更新日期：2017-11-10 00:00:00

abstract：:The power prior has been widely used in many applications covering a large number of disciplines. The power prior is intended to be an informative prior constructed from historical data. It has been used in clinical trials, genetics, health care, psychology, environmental health, engineering, economics, and business. ...

journal_title：Statistics in medicine

authors： Ibrahim JG,Chen MH,Gwon Y,Chen F

更新日期：2015-12-10 00:00:00

abstract：:Biostatistical concepts and methods apply to various problems arising in actual U.S. legal cases. These involve: measures of association, assessing the potential effect of omitted variables and the Peters-Belson approach to regression. In particular, we present the inapplicability of Fisher's exact test in the case wh...

journal_title：Statistics in medicine

authors： Gastwirth JL,Greenhouse SW

更新日期：1995-08-15 00:00:00