共查询到10条相似文献,搜索用时 5 毫秒
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Sequential analyses in clinical trials have ethical and economic advantages over fixed sample size methods. The sequential probability ratio test (SPRT) is a hypothesis testing procedure which evaluates data as it is collected. The original SPRT was developed by Wald for one-parameter families of distributions and later extended by Bartlett to handle the case of nuisance parameters. However, Bartlett's SPRT requires independent and identically distributed observations. In this paper we show that Bartlett's SPRT can be applied to generalized linear model (GLM) contexts. Then we propose an SPRT analysis methodology for a Poisson generalized linear mixed model (GLMM) that is suitable for our application to the design of a multicenter randomized clinical trial that compares two preventive treatments for surgical site infections. We validate the methodology with a simulation study that includes a comparison to Neyman–Pearson and Bayesian fixed sample size test designs and the Wald SPRT. 相似文献
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Leila Janani Mohammad Ali Mansournia Kazem Mohammad Mahmood Mahmoodi Kamran Mehrabani 《Journal of Statistical Computation and Simulation》2017,87(4):640-651
Relative risks (RRs) are often considered as preferred measures of association in randomized controlled trials especially when the binary outcome of interest is common. To directly estimate RRs, log-binomial regression has been recommended. Although log-binomial regression is a special case of generalized linear models, it does not respect the natural parameter constraints, and maximum likelihood estimation is often subject to numerical instability that leads to convergence problems. Alternative methods for solving log-binomial regression convergence problems have been proposed. A Bayesian approach also was introduced, but the comparison between this method and frequentist methods has not been fully explored. We compared five frequentist and one Bayesian methods for estimating RRs under a variety of scenario. Based on our simulation study, there is not a method that can perform well based on different statistical properties, but COPY 1000 and modified log-Poisson regression can be considered in practice. 相似文献
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Kelley M. Kidwell Nicholas J. Seewald Qui Tran Connie Kasari Daniel Almirall 《Journal of applied statistics》2018,45(9):1628-1651
In behavioral, educational and medical practice, interventions are often personalized over time using strategies that are based on individual behaviors and characteristics and changes in symptoms, severity, or adherence that are a result of one's treatment. Such strategies that more closely mimic real practice, are known as dynamic treatment regimens (DTRs). A sequential multiple assignment randomized trial (SMART) is a multi-stage trial design that can be used to construct effective DTRs. This article reviews a simple to use ‘weighted and replicated’ estimation technique for comparing DTRs embedded in a SMART design using logistic regression for a binary, end-of-study outcome variable. Based on a Wald test that compares two embedded DTRs of interest from the ‘weighted and replicated’ regression model, a sample size calculation is presented with a corresponding user-friendly applet to aid in the process of designing a SMART. The analytic models and sample size calculations are presented for three of the more commonly used two-stage SMART designs. Simulations for the sample size calculation show the empirical power reaches expected levels. A data analysis example with corresponding code is presented in the appendix using data from a SMART developing an effective DTR in autism. 相似文献
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Influenza vaccine efficacy trials: a simulation approach to understand failures from the past
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The success of a seasonal influenza vaccine efficacy trial depends not only upon the design but also upon the annual epidemic characteristics. In this context, simulation methods are an essential tool in evaluating the performances of study designs under various circumstances. However, traditional methods for simulating time‐to‐event data are not suitable for the simulation of influenza vaccine efficacy trials because of the seasonality and heterogeneity of influenza epidemics. Instead, we propose a mathematical model parameterized with historical surveillance data, heterogeneous frailty among the subjects, survey‐based heterogeneous number of daily contact, and a mixed vaccine protection mechanism. We illustrate our methodology by generating multiple‐trial data similar to a large phase III trial that failed to show additional relative vaccine efficacy of an experimental adjuvanted vaccine compared with the reference vaccine. We show that small departures from the designing assumptions, such as a smaller range of strain protection for the experimental vaccine or the chosen endpoint, could lead to smaller probabilities of success in showing significant relative vaccine efficacy. Copyright © 2015 John Wiley & Sons, Ltd. 相似文献
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It is challenging to estimate the statistical power when a complicated testing strategy is used to adjust for the type-I error for multiple comparisons in a clinical trial. In this paper, we use the Bonferroni Inequality to estimate the lower bound of the statistical power assuming that test statistics are approximately normally distributed and the correlation structure among test statistics is unknown or only partially known. The method was applied to the design of a clinical study for sample size and statistical power estimation. 相似文献
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David R. Bickel 《Revue canadienne de statistique》2011,39(4):610-631
The normalized maximum likelihood (NML) is a recent penalized likelihood that has properties that justify defining the amount of discrimination information (DI) in the data supporting an alternative hypothesis over a null hypothesis as the logarithm of an NML ratio, namely, the alternative hypothesis NML divided by the null hypothesis NML. The resulting DI, like the Bayes factor but unlike the P‐value, measures the strength of evidence for an alternative hypothesis over a null hypothesis such that the probability of misleading evidence vanishes asymptotically under weak regularity conditions and such that evidence can support a simple null hypothesis. Instead of requiring a prior distribution, the DI satisfies a worst‐case minimax prediction criterion. Replacing a (possibly pseudo‐) likelihood function with its weighted counterpart extends the scope of the DI to models for which the unweighted NML is undefined. The likelihood weights leverage side information, either in data associated with comparisons other than the comparison at hand or in the parameter value of a simple null hypothesis. Two case studies, one involving multiple populations and the other involving multiple biological features, indicate that the DI is robust to the type of side information used when that information is assigned the weight of a single observation. Such robustness suggests that very little adjustment for multiple comparisons is warranted if the sample size is at least moderate. The Canadian Journal of Statistics 39: 610–631; 2011. © 2011 Statistical Society of Canada 相似文献
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《Journal of Statistical Computation and Simulation》2012,82(4):385-398
In this paper, we derive several recurrence relations satisfied by the single and product moments of order statistics from a generalized half logistic distribution. These generalize the corresponding results for the half logistic distribution established by Balakrishnan (1985). The relations established in this paper will enable one to compute the single and product moments of all order statistics for all sample sizes in a simple recursive manner; this may be done for any choice of the shape parameter k. These moments can then be used to determine the best linear unbiased estimators of location and scale parameters from complete as well as Type-I1 censored samples. 相似文献
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N. Balakrishnan 《Journal of Statistical Computation and Simulation》2013,83(9):1704-1721
In this paper, we establish several recurrence relations for the single and product moments of progressively Type-II right-censored order statistics from a generalized half-logistic distribution. The use of these relations in a systematic recursive manner enables the computation of all the means, variances, and covariances of progressively Type-II right-censored order statistics from the generalized half-logistic distribution for all sample sizes n, effective sample sizes m, and all progressive censoring schemes (R 1, …, R m ). The results established here generalize the corresponding results for the usual order statistics due to Balakrishnan and Sandhu [Recurrence relations for single and product moments of order statistics from a generalized half-logistic distribution with applications to inference, J. Stat. Comput. Simul. 52 (1995), pp. 385–398.]. The moments so determined are then utilized to derive the best linear unbiased estimators of the scale and location–scale parameters of the generalized half-logistic distribution. The best linear unbiased predictors of censored failure times are discussed briefly. Finally, a numerical example is presented to illustrate the inferential method developed here. 相似文献
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Analyzing multiple endpoints in a confirmatory randomized clinical trial—an approach that addresses stratification,missing values,baseline imbalance and multiplicity for strictly ordinal outcomes
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Confirmatory randomized clinical trials with a stratified design may have ordinal response outcomes, ie, either ordered categories or continuous determinations that are not compatible with an interval scale. Also, multiple endpoints are often collected when 1 single endpoint does not represent the overall efficacy of the treatment. In addition, random baseline imbalances and missing values can add another layer of difficulty in the analysis plan. Therefore, the development of an approach that provides a consolidated strategy to all issues collectively is essential. For a real case example that is from a clinical trial comparing a test treatment and a control for the pain management for patients with osteoarthritis, which has all aforementioned issues, multivariate Mann‐Whitney estimators with stratification adjustment are applicable to the strictly ordinal responses with stratified design. Randomization based nonparametric analysis of covariance is applied to account for the possible baseline imbalances. Several approaches that handle missing values are provided. A global test followed by a closed testing procedure controls the family wise error rate in the strong sense for the analysis of multiple endpoints. Four outcomes indicating joint pain, stiffness, and functional status were analyzed collectively and also individually through the procedures. Treatment efficacy was observed in the combined endpoint as well as in the individual endpoints. The proposed approach is effective in addressing the aforementioned problems simultaneously and straightforward to implement. 相似文献