Using pilot data to size a two-arm randomized trial to find a nearly optimal personalized treatment strategy.

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：:In studies with survival or time-to-event outcomes, a competing risk is an event whose occurrence precludes the occurrence of the primary event of interest. Specialized statistical methods must be used to analyze survival data in the presence of competing risks. We conducted a review of randomized controlled trials wi...

journal_title：Statistics in medicine

authors： Austin PC,Fine JP

更新日期：2017-04-15 00:00:00

abstract：:In genetic association studies, it is typically thought that genetic variants and environmental variables jointly will explain more of the inheritance of a phenotype than either of these two components separately. Traditional methods to identify gene-environment interactions typically consider only one measured enviro...

journal_title：Statistics in medicine

authors： Pashova H,LeBlanc M,Kooperberg C

更新日期：2013-01-30 00:00:00

abstract：:For clinical trials with multiple treatment arms or endpoints a variety of sequentially rejective, weighted Bonferroni-type tests have been proposed, such as gatekeeping procedures, fixed sequence tests, and fallback procedures. They allow to map the difference in importance as well as the relationship between the var...

journal_title：Statistics in medicine

authors： Bretz F,Maurer W,Brannath W,Posch M

更新日期：2009-02-15 00:00:00

abstract：:An observed confidence distribution is proposed as a measure of strength of evidence for practically equivalent efficacies of two treatments. The concept is independent of prior opinions about relevant sizes of a difference in efficacy. It also avoids retrospective power calculations for trials with missed recruitment...

journal_title：Statistics in medicine

authors： Mau J

更新日期：1988-12-01 00:00:00

abstract：:Rao proposed and compared several approaches for predicting future observations in a growth curve model. The assessment of associated prediction efficiency for different prediction methods were evaluated by Cross-Validation Assessment Error (CVAE). He used three data sets, each with a limited number of subjects (13-27...

journal_title：Statistics in medicine

authors： Tian JJ,Shukla R,Buncher CR

更新日期：1994-11-15 00:00:00

abstract：:In medical and health studies, heterogeneities in clustered count data have been traditionally modeled by positive random effects in Poisson mixed models; however, excessive zeros often occur in clustered medical and health count data. In this paper, we consider a three-level random effects zero-inflated Poisson model...

journal_title：Statistics in medicine

authors： Ma R,Hasan MT,Sneddon G

更新日期：2009-08-15 00:00:00

abstract：:A survey is conducted at w of K selection units or lists, e.g. health care institutions or weeks in a year, to estimate N, the total number of individuals with particular characteristics. Our estimator utilizes two items determined for each survey participant: the number, u, among the w lists in S and the number, j, a...

journal_title：Statistics in medicine

authors： Laska EM,Meisner M,Wanderling J

更新日期：2009-07-30 00:00:00

abstract：:Longitudinal designs are important in medical research and in many other disciplines. Complete longitudinal studies, in which each subject is evaluated at each measurement occasion, are often very expensive and motivate a search for more efficient designs. Recently developed statistical methods foster the use of inten...

journal_title：Statistics in medicine

authors： Helms RW

更新日期：1992-10-01 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：:In drug-drug interaction (DDI) research, a two drug interaction is usually predicted by individual drug pharmacokinetics (PK). Although subject-specific drug concentration data from clinical PK studies on inhibitor/inducer or substrate's PK are not usually published, sample mean plasma drug concentrations and their st...

journal_title：Statistics in medicine

authors： Li L,Yu M,Chin R,Lucksiri A,Flockhart DA,Hall SD

更新日期：2007-09-10 00:00:00

abstract：:For bivariate meta-analysis of diagnostic studies, likelihood approaches are very popular. However, they often run into numerical problems with possible non-convergence. In addition, the construction of confidence intervals is controversial. Bayesian methods based on Markov chain Monte Carlo (MCMC) sampling could be u...

journal_title：Statistics in medicine

authors： Paul M,Riebler A,Bachmann LM,Rue H,Held L

更新日期：2010-05-30 00:00:00

abstract：:A stochastic approximation EM algorithm (SAEM) is described for exploratory factor analysis of dichotomous or ordinal variables. The factor structure is obtained from sufficient statistics that are updated during iterations with the Robbins-Monro procedure. Two large-scale simulations are reported that compare accurac...

journal_title：Statistics in medicine

authors： Camilli G,Geis E

更新日期：2019-09-20 00:00:00

abstract：:Combinatorial drugs have been widely applied in disease treatment, especially chemotherapy for cancer, due to its improved efficacy and reduced toxicity compared with individual drugs. The study of combinatorial drugs requires efficient experimental designs and proper follow-up statistical modeling techniques. Linear ...

journal_title：Statistics in medicine

authors： Xiao Q,Wang L,Xu H

更新日期：2019-01-30 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 Cardiac Arrythmia Suppression Trial was stopped much earlier than planned. Statistical considerations played a very important role in the decision. Flexible group sequential testing was developed for the trial by implementing a Lan and DeMets procedure with use of the permutation test. We compute P-values from the...

journal_title：Statistics in medicine

authors： Pawitan Y,Hallstrom A

更新日期：1990-09-01 00:00:00

abstract：:Biological drug products are therapeutic moieties manufactured by a living system or organisms. These are important life-saving drug products for patients with unmet medical needs. Because of expensive cost, only a few patients have access to life-saving biological products. Most of the early biological products will ...

journal_title：Statistics in medicine

authors： Lin JR,Chow SC,Chang CH,Lin YC,Liu JP

更新日期：2013-02-10 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：:In case-control studies, the Cochran-Armitage (CA) trend test is powerful for detection of an association between a risk allele and a marker. To apply this test, a score should be assigned to the genotypes based on the genetic model. When the underlying genetic model is unknown, the trend test statistic is a function ...

journal_title：Statistics in medicine

authors： Zheng G

更新日期：2003-08-30 00:00:00

abstract：:In the analysis of composite endpoints in a clinical trial, time to first event analysis techniques such as the logrank test and Cox proportional hazard test do not take into account the multiplicity, importance, and the severity of events in the composite endpoint. Several generalized pairwise comparison analysis met...

journal_title：Statistics in medicine

authors： Verbeeck J,Spitzer E,de Vries T,van Es GA,Anderson WN,Van Mieghem NM,Leon MB,Molenberghs G,Tijssen J

更新日期：2019-12-30 00:00:00

abstract：:Down's syndrome screening is currently carried out using a combination of biochemical markers measured in maternal serum samples; these include MSAFP, Total hCG, uE3 and Free beta-hCG. Recently a number of papers have compared the effectiveness of different combinations of these markers. Some recommend MSAFP, Total hC...

journal_title：Statistics in medicine

authors： Dunstan FD,Gray JC,Nix AB,Reynolds T

更新日期：1997-07-15 00:00:00

abstract：:The analysis of multivariate time-to-event (TTE) data can become complicated due to the presence of clustering, leading to dependence between multiple event times. For a long time, (conditional) frailty models and (marginal) copula models have been used to analyze clustered TTE data. In this article, we propose a gene...

journal_title：Statistics in medicine

authors： Tran TMP,Abrams S,Braekers R

更新日期：2020-05-30 00:00:00

abstract：:Group sequential hypothesis testing is now widely used to analyze prospective data. If Monte Carlo simulation is used to construct the signaling threshold, the challenge is how to manage the type I error probability for each one of the multiple tests without losing control on the overall significance level. This paper...

journal_title：Statistics in medicine

authors： Silva IR

更新日期：2016-04-30 00:00:00

abstract：:Summarizing the information of many studies using a meta-analysis becomes more and more important, also in the field of diagnostic studies. The special challenge in meta-analysis of diagnostic accuracy studies is that in general sensitivity and specificity are co-primary endpoints. Across the studies both endpoints ar...

journal_title：Statistics in medicine

authors： Zapf A,Hoyer A,Kramer K,Kuss O

更新日期：2015-12-20 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：:The analysis of patient-reported outcomes or other psychological traits can be realized using the Rasch measurement model. When the objective of a study is to compare groups of individuals, it is important, before the study, to define a sample size such that the group comparison test will attain a given power. The Ras...

journal_title：Statistics in medicine

authors： Hardouin JB,Blanchin M,Feddag ML,Le Néel T,Perrot B,Sébille V

更新日期：2015-07-20 00:00:00

abstract：:Population dynamic models, commonly used tools in the study of epidemics and other complex population processes, are implicit non-linear mathematical equations. Inference based on such models can be difficult due to the problems associated with high dimensional parameters that may be non-identified and complex likelih...

journal_title：Statistics in medicine

authors： Brookhart MA,Hubbard AE,van der Laan MJ,Colford JM Jr,Eisenberg JN

更新日期：2002-12-15 00:00:00

abstract：:Pearson's chi-squared, the likelihood-ratio, and Fisher-Freeman-Halton's test statistics are often used to test the association of unordered r x c tables. Asymptotical, exact conditional, or exact conditional with mid-p adjustment methods are commonly used to compute the p-value. We have compared test power and signif...

journal_title：Statistics in medicine

authors： Lydersen S,Pradhan V,Senchaudhuri P,Laake P

更新日期：2007-10-15 00:00:00

abstract：:In recent years, quality control charts have been increasingly applied in the healthcare environment, for example, to monitor surgical performance. Risk-adjusted cumulative (CUSUM) charts that utilize risk scores like the Parsonnet score to estimate the probability of death of a patient from an operation turn out to b...

journal_title：Statistics in medicine

authors： Knoth S,Wittenberg P,Gan FF

更新日期：2019-05-30 00:00:00

abstract：:In a phase I clinical trial in cancer patients, the drug involved had one known main adverse effect, which also occurs spontaneously in cancer patients with a fairly high frequency. Experiments in rats have shown marked effects of the drug on tumour growth in high doses, but also dose-dependent toxicity. Consequently,...

journal_title：Statistics in medicine

authors： Møller S

更新日期：1995-05-15 00:00:00