Biomarker discovery using targeted maximum-likelihood estimation: application to the treatment of antiretroviral-resistant HIV infection.

abstract：:The goal of phase I cancer trials is to determine the highest dose of a treatment regimen with an acceptable toxicity rate. Traditional designs for phase I trials, such as the Continual Reassessment Method (CRM) and the 3 + 3 design, require each patient or a cohort of patients to be fully evaluated for the dose-limit...

journal_title：Statistics in medicine

authors： Polley MY

更新日期：2011-07-30 00:00:00

abstract：:In many biomedical and epidemiological studies, data are often clustered due to longitudinal follow up or repeated sampling. While in some clustered data the cluster size is pre-determined, in others it may be correlated with the outcome of subunits, resulting in informative cluster size. When the cluster size is info...

journal_title：Statistics in medicine

authors： Chen Z,Zhang B,Albert PS

更新日期：2011-07-10 00:00:00

abstract：:The continual reassessment method (CRM) is an adaptive design for Phase I trials whose operating characteristics, including appropriate sample size, probability of correctly identifying the maximum tolerated dose, and the expected proportion of participants assigned to each dose, can only be determined via simulation....

journal_title：Statistics in medicine

authors： Braun TM

更新日期：2020-09-16 00:00:00

abstract：:Orthogonal polynomial scores (OPS) is a simple, biologically meaningful approach to characterize longitudinal data in phase I and II clinical pharmacology trials. It describes average, linear, quadratic and higher order polynomial characteristics of each subject's response over time with use of composite scores comput...

journal_title：Statistics in medicine

authors： Bradstreet TE

更新日期：1993-04-15 00:00:00

abstract：:In randomised trials, continuous endpoints are often measured with some degree of error. This study explores the impact of ignoring measurement error and proposes methods to improve statistical inference in the presence of measurement error. Three main types of measurement error in continuous endpoints are considered:...

journal_title：Statistics in medicine

authors： Nab L,Groenwold RHH,Welsing PMJ,van Smeden M

更新日期：2019-11-30 00:00:00

abstract：:The weighted average treatment effect is a causal measure for the comparison of interventions in a specific target population, which may be different from the population where data are sampled from. For instance, when the goal is to introduce a new treatment to a target population, the question is what efficacy (or ef...

journal_title：Statistics in medicine

authors： Tao Y,Fu H

更新日期：2019-02-10 00:00:00

abstract：:This paper presents a general approach for simultaneously assessing, from serial data, diagnostic consistency, interrater reliability and incidence of a strictly progressive disease. Observed data are viewed as incomplete: diagnostic errors are not distinguished from true diagnoses. We introduce a broad class of model...

journal_title：Statistics in medicine

authors： Espeland MA,Murphy WC,Leverett DH

更新日期：1988-03-01 00:00:00

abstract：:In the comparison of two or more treatment groups to a control group, consider a study with non-decreasing repeated measurements of the same characteristic taken over a common set of time points for each subject. Based on the vector of possibly incomplete responses from each subject, this paper considers asymptoticall...

journal_title：Statistics in medicine

authors： Davis CS

更新日期：1996-12-15 00:00:00

abstract：:In biomedical research such as the development of vaccines for infectious diseases or cancer, study outcomes measured by an assay or device are often collected from multiple sources or laboratories. Measurement error that may vary between laboratories needs to be adjusted for when combining samples across data sources...

journal_title：Statistics in medicine

authors： Huang Y,Huang Y,Moodie Z,Li S,Self S

更新日期：2012-12-10 00:00:00

abstract：:Drop-out often occurs in clinical trials with multiple visits and drop-out is often informative in the sense that the population of patients who dropped out is different from the population of patients who completed the study. To handle data with informative drop-out, an intention-to-treat analysis, which evaluates tr...

journal_title：Statistics in medicine

authors： Shao J,Zhong B

更新日期：2003-08-15 00:00:00

abstract：:The relative importance of prognostic factors in regression can be measured either by standardized regression coefficients or by percentages of explained variation in a dependent variable. One advantage of using explained variation is the direct comparability of qualitative prognostic factors with others, or of groups...

journal_title：Statistics in medicine

authors： Schemper M

更新日期：1993-12-30 00:00:00

abstract：:Two important qualities of controlled clinical trials are that they reduce dependence on historical standards for evaluating therapy and separate the effect of treatment from the confounding influence of time. Whatever the theory of the clinical trial, however, time has not easily been banished from the analysis of me...

journal_title：Statistics in medicine

authors： Senn SJ

更新日期：1989-11-01 00:00:00

abstract：:A three-component, competing-risk mortality model, developed for animal survival data, fits human life table data for all ages over a range of mean life spans from 16 to 74 years. The competing risks are a novel exponentially-decreasing hazard, dominant during immaturity; a constant hazard, dominant during adulthood; ...

journal_title：Statistics in medicine

authors： Siler W

更新日期：1983-07-01 00:00:00

abstract：:Adaptive designs have been proposed for clinical trials in which the nuisance parameters or alternative of interest are unknown or likely to be misspecified before the trial. Although most previous works on adaptive designs and mid-course sample size re-estimation have focused on two-stage or group-sequential designs ...

journal_title：Statistics in medicine

authors： Bartroff J,Lai TL

更新日期：2008-05-10 00:00:00

abstract：:I reflect on the statistical methods of the Christakis-Fowler studies on network-based contagion of traits by checking the sensitivity of these kinds of results to various alternate specifications and generative mechanisms. Despite the honest efforts of all involved, I remain pessimistic about establishing whether bin...

journal_title：Statistics in medicine

authors： Thomas AC

更新日期：2013-02-20 00:00:00

abstract：:Although prostate cancer and benign prostatic hyperplasia are major health problems in U.S. men, little is known about the early stages of the natural history of prostate disease. A molecular biomarker called prostate specific antigen (PSA), together with a unique longitudinal bank of frozen serum, now allows a histor...

journal_title：Statistics in medicine

authors： Pearson JD,Morrell CH,Landis PK,Carter HB,Brant LJ

更新日期：1994-03-15 00:00:00

abstract：:To construct a confidence interval for the mean of a log-normal distribution in small samples, we propose likelihood-based approaches - the signed log-likelihood ratio and modified signed log-likelihood ratio methods. Extensive Monte Carlo simulation results show the advantages of the modified signed log-likelihood ra...

journal_title：Statistics in medicine

authors： Wu J,Wong AC,Jiang G

更新日期：2003-06-15 00:00:00

abstract：:Methods for dealing with tied event times in the Cox proportional hazards model are well developed. Also, the partial likelihood provides a natural way to handle covariates that change over time. However, ties between event times and the times that discrete time-varying covariates change have not been systematically s...

journal_title：Statistics in medicine

authors： Xin X,Horrocks J,Darlington GA

更新日期：2013-06-30 00:00:00

abstract：:Many epidemiologic investigations are designed to study the effects of multiple exposures. Most of these studies are analysed either by fitting a risk-regression model with all exposures forced in the model, or by using a preliminary-testing algorithm, such as stepwise regression, to produce a smaller model. Research ...

journal_title：Statistics in medicine

authors： Greenland S

更新日期：1993-04-30 00:00:00

abstract：:When estimating causal effects, unmeasured confounding and model misspecification are both potential sources of bias. We propose a method to simultaneously address both issues in the form of a semi-parametric sensitivity analysis. In particular, our approach incorporates Bayesian Additive Regression Trees into a two-p...

journal_title：Statistics in medicine

authors： Dorie V,Harada M,Carnegie NB,Hill J

更新日期：2016-09-10 00:00:00

abstract：:In this paper, we investigate the impact of time-invariant covariates when fitting transition mixed models. This is carried out by emphasizing on the role of baseline responses on the estimation process. Transition models are allowed for two cases of exogenous and endogenous baseline responses. We illustrate these con...

journal_title：Statistics in medicine

authors： Rikhtehgaran R,Kazemi I,Verbeke G

更新日期：2014-11-30 00:00:00

abstract：:Longitudinal biomarker data are often collected in studies, providing important information regarding the probability of an outcome of interest occurring at a future time. With many new and evolving technologies for biomarker discovery, the number of biomarker measurements available for analysis of disease progression...

journal_title：Statistics in medicine

authors： Zhao L,Murray S,Mariani LH,Ju W

更新日期：2020-11-20 00:00:00

abstract：:The semi-Markov assumption emphasizes the importance of time spent in a state. In order to compute this type of multistate model, most transition times are always considered to be exactly identified or right censored. However, in the longitudinal analysis of chronic diseases, investigators are often confronted with in...

journal_title：Statistics in medicine

authors： Foucher Y,Giral M,Soulillou JP,Daures JP

更新日期：2007-12-30 00:00:00

abstract：:Patient reported outcome and observer evaluative studies in clinical trials and post-hoc analyses often use instruments that measure responses on ordinal-rating or Likert scales. We propose a flexible distributional approach by modeling the change scores from the baseline to the end of the study using independent beta...

journal_title：Statistics in medicine

authors： Zou KH,Carlsson MO,Quinn SA

更新日期：2010-10-30 00:00:00

abstract：:Epidemiologic and clinical studies routinely collect longitudinal measures of multiple outcomes, including biomarker measures, cognitive functions, and clinical symptoms. These longitudinal outcomes can be used to establish the temporal order of relevant biological processes and their association with the onset of cli...

journal_title：Statistics in medicine

authors： Yang L,Gao S

更新日期：2013-03-15 00:00:00

abstract：:The objective of this report is to provide a basis to inform decisions about priorities for developing statistical research initiatives in the field of public health surveillance for emerging threats. Rapid information system advances have created a vast opportunity of secondary data sources for information to enhance...

journal_title：Statistics in medicine

authors： Rolka H,Burkom H,Cooper GF,Kulldorff M,Madigan D,Wong WK

更新日期：2007-04-15 00:00:00

abstract：:We present graphical and numerical methods for assessing the adequacy of the logistic regression model for stratified case-control data. The proposed methods are derived from the cumulative sum of residuals over the covariate or linear predictor. Under the assumed model, the cumulative residual process converges weakl...

journal_title：Statistics in medicine

authors： Arbogast PG,Lin DY

更新日期：2005-01-30 00:00:00

abstract：:Outcomes research often requires estimating the impact of a binary treatment on a binary outcome in a non-randomized setting, such as the effect of taking a drug on mortality. The data often come from self-selected samples, leading to a spurious correlation between the treatment and outcome when standard binary depend...

journal_title：Statistics in medicine

authors： Bhattacharya J,Goldman D,McCaffrey D

更新日期：2006-02-15 00:00:00

abstract：:In a typical bioequivalence trial, summary measures of the plasma concentration versus time profile are used to compare two formulations of a drug product. Commonly used measures include area under the curve (AUC), maximum plasma concentration (C(max)) and time to maximum concentration (T(max)). Equivalence of these s...

journal_title：Statistics in medicine

authors： Mauger DT,Chinchilli VM

更新日期：2000-10-30 00:00:00

abstract：:Data monitoring committees (DMCs) have become an increasingly common component of randomized clinical trials in recent years. As experience has accumulated, and more individuals and organizations have become involved in such activities, a variety of approaches to the operation of such committees has inevitably arisen....

journal_title：Statistics in medicine

authors： Ellenberg SS

更新日期：2001-09-15 00:00:00