Paired versus two-sample design for a clinical trial of treatments with dichotomous outcome: power considerations.

abstract：:Using genetic recombination data to make inferences about chiasmata on the tetrad during meiosis is a classic problem dating back to Weinstein's paper in 1936 (Genetics 21, 155-199). In the last few years, Weinstein's methods have been revived and applied to new problems, but a number of important statistical issues r...

journal_title：Biometrics

authors： Yu K,Feingold E

更新日期：2002-06-01 00:00:00

abstract：:Estimating the number of clusters in a data set is a crucial step in cluster analysis. In this article, motivated by the gap method (Tibshirani, Walther, and Hastie, 2001, Journal of the Royal Statistical Society B63, 411-423), we propose the weighted gap and the difference of difference-weighted (DD-weighted) gap met...

journal_title：Biometrics

authors： Yan M,Ye K

更新日期：2007-12-01 00:00:00

abstract：:Long-term follow-up is common in many medical investigations where the interest lies in predicting patients' risks for a future adverse outcome using repeatedly measured predictors over time. A key quantity is the likelihood of developing an adverse outcome among individuals who survived up to time s given their covar...

journal_title：Biometrics

authors： Maziarz M,Heagerty P,Cai T,Zheng Y

更新日期：2017-03-01 00:00:00

abstract：:Three properties of interest in bioavailability studies using compartmental models are the area under the concentration curve, the maximum concentration, and the time to maximum concentration. Methods are described for finding designs that minimize the variance of the estimates of these quantities in such a model. The...

journal_title：Biometrics

authors： Atkinson AC,Chaloner K,Herzberg AM,Juritz J

更新日期：1993-06-01 00:00:00

abstract：:A procedure is given for estimating the ratio of the means of two populations using the data from two independent random samples when the observations are normally distributed with population variances that are related to the population means. ...

journal_title：Biometrics

authors： Cox CP

更新日期：1985-03-01 00:00:00

abstract：:The clinical trial design in which the endpoint is measured both at baseline and at the end of the study is used in a variety of situations. For two-group designs, test such as the t test or analysis of covariance are commonly used to evaluate treatment efficacy. Often such pretest-posttest trials restrict participati...

journal_title：Biometrics

authors： Follmann DA

更新日期：1991-06-01 00:00:00

abstract：:Suppose that a heterogeneous group of individuals is followed over time and that each individual can be in state 0 or state 1 at each time point. The sequence of states is assumed to follow a binary Markov chain. In this paper we model the transition probabilities for the 0 to 0 and 1 to 0 transitions by two logistic ...

journal_title：Biometrics

authors： Muenz LR,Rubinstein LV

更新日期：1985-03-01 00:00:00

abstract：:The characteristics of deleterious genes have been of great interest in both theory and practice in genetics. Because of the complex genetic mechanism of these deleterious genes, most current studies try to estimate the overall magnitude of mortality effects on a population, which is characterized classically by the n...

journal_title：Biometrics

authors： Lee JK,Lascoux M,Newton MA,Nordheim EV

更新日期：1999-06-01 00:00:00

abstract：:We consider Bayesian methodology for comparing two or more unlabeled point sets. Application of the technique to a set of steroid molecules illustrates its potential utility involving the comparison of molecules in chemoinformatics and bioinformatics. We initially match a pair of molecules, where one molecule is regar...

journal_title：Biometrics

authors： Dryden IL,Hirst JD,Melville JL

更新日期：2007-03-01 00:00:00

abstract：SUMMARY:We develop sample size formulas for studies aiming to test mean differences between a treatment and control group when all-or-none nonadherence (noncompliance) and selection bias are expected. Recent work by Fay, Halloran, and Follmann (2007, Biometrics 63, 465-474) addressed the increased variances within grou...

journal_title：Biometrics

authors： Shardell MD,El-Kamary SS

更新日期：2009-06-01 00:00:00

abstract：:In this article, we propose approximate sample size formulas for establishing equivalence or noninferiority of two treatments in match-pairs design. Using the ratio of two proportions as the equivalence measure, we derive sample size formulas based on a score statistic for two types of analyses: hypothesis testing and...

journal_title：Biometrics

authors： Tang ML,Tang NS,Chan IS,Chan BP

更新日期：2002-12-01 00:00:00

abstract：:Increasingly, genetic studies of tumors of the same histologic diagnosis are elucidating subtypes that are distinct with respect to clinical endpoints such as response to treatment and survival. This raises concerns about the efficiency of using the simple log-rank test for analysis of treatment effect on survival in ...

journal_title：Biometrics

authors： Li Y,Betensky RA,Louis DN,Cairncross JG

更新日期：2002-03-01 00:00:00

abstract：:Conventional statistical determinations of sample size in phase II studies typically lead to sample sizes of the order of 25 (Schoenfeld, 1980, International Journal of Radiation Oncology, Biology and Physics 6, 371-374). When the development of new treatments is proceeding rapidly relative to the recruitment of suita...

journal_title：Biometrics

authors： Whitehead J

更新日期：1985-06-01 00:00:00

abstract：:An important objective in biomedical and environmental risk assessment is estimation of minimum exposure levels that induce a pre-specified adverse response in a target population. The exposure points in such settings are typically referred to as benchmark doses (BMDs). Parametric Bayesian estimation for finding BMDs ...

journal_title：Biometrics

authors： Fang Q,Piegorsch WW,Simmons SJ,Li X,Chen C,Wang Y

更新日期：2015-12-01 00:00:00

abstract：:We consider the problem of testing for a difference in the probability of success from matched binary pairs. Starting with three standard inexact tests, the nuisance parameter is first estimated and then the residual dependence is eliminated by maximization, producing what I call an E+M P-value. The E+M P-value based ...

journal_title：Biometrics

authors： Lloyd CJ

更新日期：2008-09-01 00:00:00

abstract：:Multilist capture-recapture methods are commonly used to estimate the size of elusive populations. In many situations, lists are stratified by distinguishing features, such as age or sex. Stratification has often been used to reduce biases caused by heterogeneity in the probability of list membership among members of ...

journal_title：Biometrics

authors： Sutherland JM,Schwarz CJ,Rivest LP

更新日期：2007-09-01 00:00:00

abstract：:Power and sample-size formulas for testing the homogeneity of relative risks using the score method are presented. The homogeneity score test (Gart, 1985, Biometrika 72, 673-677) is formally equivalent to the Pearson chi-square test, although they look different. Results of this paper may be useful in assessing the va...

journal_title：Biometrics

authors： Nam JM

更新日期：1999-03-01 00:00:00

abstract：:This paper considers methods for estimating the relationship between a binary response Y and the genetic effects responsible for a second binary trait Z. The responses Y are observed only for target individuals, and the responses Z are observed only for the relatives of these targets. The analysis consists of two part...

journal_title：Biometrics

authors： Houwing-Duistermaat JJ,van Houwelingen HC,de Winter JP

更新日期：2000-09-01 00:00:00

abstract：:Understanding HIV incidence, the rate at which new infections occur in populations, is critical for tracking and surveillance of the epidemic. In this article, we derive methods for determining sample sizes for cross-sectional surveys to estimate incidence with sufficient precision. We further show how to specify samp...

journal_title：Biometrics

authors： Konikoff J,Brookmeyer R

更新日期：2015-12-01 00:00:00

abstract：:The classical concordance correlation coefficient (CCC) to measure agreement among a set of observers assumes data to be distributed as normal and a linear relationship between the mean and the subject and observer effects. Here, the CCC is generalized to afford any distribution from the exponential family by means of...

journal_title：Biometrics

authors： Carrasco JL

更新日期：2010-09-01 00:00:00

abstract：:We describe an exact, unconditional, non-randomized procedure for producing confidence intervals for the grand mean in a normal-normal random effects meta-analysis. The procedure targets meta-analyses based on too few primary studies, ≤ 7 , say, to ...

journal_title：Biometrics

authors： Michael H,Thornton S,Xie M,Tian L

更新日期：2019-06-01 00:00:00

abstract：:Often with data from immunoassays, the concentration-response relationship is nonlinear and intra-assay response variance is heterogeneous. Estimation of the standard curve is usually based on a nonlinear heteroscedastic regression model for concentration-response, where variance is modeled as a function of mean respo...

journal_title：Biometrics

authors： Belanger BA,Davidian M,Giltinan DM

更新日期：1996-03-01 00:00:00

abstract：:We describe group sequential tests for a bivariate response. The tests are defined in terms of the two response components jointly, rather than through a single summary statistic. Such methods are appropriate when the two responses concern different aspects of a treatment; for example, one might wish to show that a ne...

journal_title：Biometrics

authors： Jennison C,Turnbull BW

更新日期：1993-09-01 00:00:00

abstract：:The concentration of carbon monoxide in the blood of a cigarette smoker varies in response to the frequency and dose of CO delivered by the cigarettes he smokes and by the rate at which CO washes out of his blood. Moments of first passage times or exit times above a nominal threshold can be calculated using a stochast...

journal_title：Biometrics

authors： Marcus AH,Czajkowski S Jr

更新日期：1979-09-01 00:00:00

abstract：:Complex issues arise when investigating the association between longitudinal immunologic measures and time to an event, such as time to relapse, in cancer vaccine trials. Unlike many clinical trials, we may encounter patients who are cured and no longer susceptible to the time-to-event endpoint. If there are cured pat...

journal_title：Biometrics

authors： Brown ER,Ibrahim JG

更新日期：2003-09-01 00:00:00

abstract：:We propose a new estimation method for multivariate failure time data using the quadratic inference function (QIF) approach. The proposed method efficiently incorporates within-cluster correlations. Therefore, it is more efficient than those that ignore within-cluster correlation. Furthermore, the proposed method is e...

journal_title：Biometrics

authors： Xue L,Wang L,Qu A

更新日期：2010-06-01 00:00:00

abstract：:Many techniques of functional data analysis require choosing a measure of distance between functions, with the most common choice being L2 distance. In this article we show that using a weighted L2 distance, with a judiciously chosen weight function, can improve the performance of various statistical methods for funct...

journal_title：Biometrics

authors： Chen H,Reiss PT,Tarpey T

更新日期：2014-09-01 00:00:00

abstract：:This paper proposes a two-stage phase I-II clinical trial design to optimize dose-schedule regimes of an experimental agent within ordered disease subgroups in terms of the toxicity-efficacy trade-off. The design is motivated by settings where prior biological information indicates it is certain that efficacy will imp...

journal_title：Biometrics

authors： Lin R,Thall PF,Yuan Y

更新日期：2020-03-01 00:00:00

abstract：:Computer simulation has been used to compare four methods for calculating confidence intervals for simple rate ratios estimated from cohort studies. The method proposed by Cornfield (1956. In Proceedings of the Third Berkeley Symposium on Mathematical Statistics and Probability. Vol. IV, 135-148) for interval estimati...

journal_title：Biometrics

authors： Howe GR

更新日期：1983-06-01 00:00:00

abstract：:There has been great interest in developing nonlinear structural equation models and associated statistical inference procedures, including estimation and model selection methods. In this paper a general semiparametric structural equation model (SSEM) is developed in which the structural equation is composed of nonpar...

journal_title：Biometrics

authors： Guo R,Zhu H,Chow SM,Ibrahim JG

更新日期：2012-06-01 00:00:00