Bayesian predictive approach to interim monitoring in clinical trials.

abstract：:Many reactions in enzymology are governed by the Michaelis-Menten equation. Characterising these reactions requires the estimation of the parameters K(M) and V(max) which determine the Michaelis-Menten equation and this is done by observing rates of reactions at a set of substrate concentrations. The choice of substra...

journal_title：Statistics in medicine

authors： Matthews JN,Allcock GC

更新日期：2004-02-15 00:00:00

abstract：:Instrumental variable estimates of causal effects can be biased when using many instruments that are only weakly associated with the exposure. We describe several techniques to reduce this bias and estimate corrected standard errors. We present our findings using a simulation study and an empirical application. For th...

journal_title：Statistics in medicine

authors： Davies NM,von Hinke Kessler Scholder S,Farbmacher H,Burgess S,Windmeijer F,Smith GD

更新日期：2015-02-10 00:00:00

abstract：:Tree-based methods have become popular for analyzing complex data structures where the primary goal is risk stratification of patients. Ensemble techniques improve the accuracy in prediction and address the instability in a single tree by growing an ensemble of trees and aggregating. However, in the process, individua...

journal_title：Statistics in medicine

authors： Banerjee M,Ding Y,Noone AM

更新日期：2012-07-10 00:00:00

abstract：:Machine learning techniques such as classification and regression trees (CART) have been suggested as promising alternatives to logistic regression for the estimation of propensity scores. The authors examined the performance of various CART-based propensity score models using simulated data. Hypothetical studies of v...

journal_title：Statistics in medicine

authors： Lee BK,Lessler J,Stuart EA

更新日期：2010-02-10 00:00:00

abstract：:The Response Evaluation Criteria in Solid Tumors are used as standard guidelines for the clinical evaluation of cancer treatments. The assessment is based on the anatomical tumor burden: change in size of target lesions and evolution of nontarget lesions (NTL). Despite unquestionable advantages of this standard tool, ...

journal_title：Statistics in medicine

authors： Król A,Tournigand C,Michiels S,Rondeau V

更新日期：2018-06-15 00:00:00

abstract：:Clinical trials studying treatments for rare diseases are challenging to design and conduct due to the limited number of patients eligible for the trial. One design used to address this challenge is the small n, sequential, multiple assignment, randomized trial (snSMART). We propose a new snSMART design that investiga...

journal_title：Statistics in medicine

authors： Fang F,Hochstedler KA,Tamura RN,Braun TM,Kidwell KM

更新日期：2021-02-20 00:00:00

abstract：:Individuals may vary in their responses to treatment, and identification of subgroups differentially affected by a treatment is an important issue in medical research. The risk of misleading subgroup analyses has become well known, and some exploratory analyses can be helpful in clarifying how covariates potentially i...

journal_title：Statistics in medicine

authors： Kim HJ,Lu B,Nehus EJ,Kim MO

更新日期：2019-02-10 00:00:00

abstract：:Ordinal data appear in a wide variety of scientific fields. These data are often analyzed using ordinal logistic regression models that assume proportional odds. When this assumption is not met, it may be possible to capture the lack of proportionality using a constrained structural relationship between the odds and t...

journal_title：Statistics in medicine

authors： Capuano AW,Dawson JD

更新日期：2013-06-15 00:00:00

abstract：:Hypothesis testing, in which the null hypothesis specifies no difference between treatment groups, is an important tool in the assessment of new medical interventions. For randomized clinical trials, permutation tests that reflect the actual randomization are design-based analyses for such hypotheses. This means that ...

journal_title：Statistics in medicine

authors： Berger VW

更新日期：2000-05-30 00:00:00

abstract：:Surrogate endpoint validation has been well established by the meta-analytical correlation-based approach as outlined in the seminal work of Buyse et al. (Biostatistics, 2000). Surrogacy can be assumed if strong associations on individual and study levels can be demonstrated. Alternatively, if an effect on a true endp...

journal_title：Statistics in medicine

authors： Schürmann C,Sieben W

更新日期：2016-03-30 00:00:00

abstract：:Interim analyses are conducted to allow for early termination of the trial, for ethical as well as economical reasons. Here we consider interim analyses in repeated measurements studies where the measurements are binary. Two methods for analysing this kind of data are compared according to their operating characterist...

journal_title：Statistics in medicine

authors： Spiessens B,Lesaffre E,Verbeke G

更新日期：2003-02-28 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：:Although there are many models which are used to calculate the health benefits (and thus the cost-effectiveness) of vaccination programmes, they can be divided into two groups: those which assume a constant force of infection, that is a constant per-susceptible rate of infection; and those which assume that the force ...

journal_title：Statistics in medicine

authors： Edmunds WJ,Medley GF,Nokes DJ

更新日期：1999-12-15 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：: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：:Exploring the possible reasons for heterogeneity between studies is an important aspect of conducting a meta-analysis. This paper compares a number of methods which can be used to investigate whether a particular covariate, with a value defined for each study in the meta-analysis, explains any heterogeneity. The main ...

journal_title：Statistics in medicine

authors： Thompson SG,Sharp SJ

更新日期：1999-10-30 00:00:00

abstract：:Having a surrogate for a definitive endpoint in a clinical trial can sometimes be useful when it is impractical, invasive or very time consuming to obtain the definitive endpoint. This paper discusses methods for assessing whether the surrogate-endpoint results of a trial can be used in place of definitive-endpoint re...

journal_title：Statistics in medicine

authors： Korn EL,Albert PS,McShane LM

更新日期：2005-01-30 00:00:00

abstract：:Sexually transmitted infections (STIs) with Chlamydia trachomatis, Neisseria gonorrhoeae, and Trichomonas vaginalis are among the most common infectious diseases in the United States, disproportionately affecting young women. Because a significant portion of the infections present no symptoms, infection control relies...

journal_title：Statistics in medicine

authors： Li Z,Liu H,Tu W

更新日期：2015-09-10 00:00:00

abstract：:In failure time studies involving a chronic disease such as cancer, several competing causes of mortality may be operating. Commonly, the conventional statistical technique of Kaplan-Meier, which is only meaningfully interpreted by assuming independence of failure types and the censoring mechanism, is employed in clin...

journal_title：Statistics in medicine

authors： Tai BC,Machin D,White I,Gebski V,EOI (The European Osteosarcoma Intergroup).

更新日期：2001-03-15 00:00:00

abstract：:In this paper we examine the efficiency of a generalization of the traditional normal linear (LDA) or quadratic (QDA) discriminant analysis. This procedure (the generalized discriminant analysis, GDA) replaces each normal density used in the traditional classification rule by a Fourier series density estimator which '...

journal_title：Statistics in medicine

authors： Odom-Maryon T,Langholz B,Niland J,Azen S

更新日期：1991-03-01 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：:The back-calculation method has been used to estimate the number of HIV infections from AIDS incidence data in a particular population. We present an extension of back calculation that provides estimates of the numbers of HIV infectives in different stages of infection. We model the staging process with a time-depende...

journal_title：Statistics in medicine

authors： Longini IM Jr,Byers RH,Hessol NA,Tan WY

更新日期：1992-04-01 00:00:00

abstract：:A method is presented which allows us to adapt the sample size as well as the number and time points of interim analyses to the treatment difference observed at an interim look during the course of a clinical trial with censored survival time as the endpoint. The method allows the inclusion of data inspections during ...

journal_title：Statistics in medicine

authors： Schäfer H,Müller HH

更新日期：2001-12-30 00:00:00

abstract：:The relative concentration index (RCI) and the absolute concentration index (ACI) have been widely used for monitoring health disparities with ranked health determinants. The RCI has been extended to allow value judgments about inequality aversion by Pereira in 1998 and by Wagstaff in 2002. Previous studies of the ext...

journal_title：Statistics in medicine

authors： Yu M,Liu B,Li Y,Zou ZJ,Breen N

更新日期：2019-01-15 00:00:00

abstract：:A targeted poll was undertaken to compare and contrast models of data monitoring of randomized clinical trials sponsored by the National Institutes of Health. In an attempt to represent the institutes which conduct clinical trials, twelve individuals were selected and asked to respond to a questionnaire specifically p...

journal_title：Statistics in medicine

authors： Geller NL,Stylianou M

更新日期：1993-03-01 00:00:00

abstract：:In noninferiority studies, a limit of indifference is used to express a tolerance in results such that the clinician would regard such results as being acceptable or 'not worse'. We applied this concept to a measure of accuracy, the Receiver Operating Characteristic (ROC) curve, for a sequence of tests. We expressed a...

journal_title：Statistics in medicine

authors： Ahmed AE,Schubert CM,McClish DK

更新日期：2013-07-20 00:00:00

abstract：:There are many settings in which the distribution of error in a mismeasured covariate varies with the value of another covariate. Take, for example, the case of HIV phylogenetic cluster size, large values of which are an indication of rapid HIV transmission. Researchers wish to find behavioral correlates of HIV phylog...

journal_title：Statistics in medicine

authors： Parveen N,Moodie E,Brenner B

更新日期：2017-07-30 00:00:00

abstract：:Prevalence data provided by cancer registries are generally biased, since the patients that were diagnosed before the starting of the registry's activity cannot be included in the statistics. The relevance of this incompleteness bias is estimated in this paper. Incidence and relative survival are modelled as parametri...

journal_title：Statistics in medicine

authors： Capocaccia R,De Angelis R

更新日期：1997-02-28 00:00:00

abstract：:Although population-based molecular association studies are becoming increasingly popular, methodology for the meta-analysis of these studies has been neglected, particularly with regard to two issues: testing Hardy-Weinberg equilibrium (HWE), and pooling results in a manner that reflects a biological model of gene ef...

journal_title：Statistics in medicine

authors： Thakkinstian A,McElduff P,D'Este C,Duffy D,Attia J

更新日期：2005-05-15 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