Statistical challenges in assessing potential efficacy of complex interventions in pilot or feasibility studies.

abstract：:The analysis of fecundity data is challenging and requires consideration of both highly timed and interrelated biologic processes in the context of essential behaviors such as sexual intercourse during the fertile window. Understanding human fecundity is further complicated by presence of a sterile population, i.e. co...

journal_title：Statistical methods in medical research

authors： McLain AC,Sundaram R,Buck Louis GM

更新日期：2016-02-01 00:00:00

abstract：:The probability model for periodic screening was extended to provide statistical inference for sensitivity depending on sojourn time, in which the sensitivity was modeled as a function of time spent in the preclinical state and the sojourn time. The likelihood function with the proposed sensitivity model was then eval...

journal_title：Statistical methods in medical research

authors： Kim S,Wu D

更新日期：2016-04-01 00:00:00

abstract：:Accelerated failure time model is a popular model to analyze censored time-to-event data. Analysis of this model without assuming any parametric distribution for the model error is challenging, and the model complexity is enhanced in the presence of large number of covariates. We developed a nonparametric Bayesian met...

journal_title：Statistical methods in medical research

authors： Zhang Z,Sinha S,Maiti T,Shipp E

更新日期：2018-04-01 00:00:00

abstract：:We propose a hierarchical Bayesian methodology to model spatially or spatio-temporal clustered survival data with possibility of cure. A flexible continuous transformation class of survival curves indexed by a single parameter is used. This transformation model is a larger class of models containing two special cases ...

journal_title：Statistical methods in medical research

authors： Hurtado Rúa SM,Dey DK

更新日期：2016-02-01 00:00:00

abstract：:In this article, we present an overview and tutorial of statistical methods for meta-analysis of diagnostic tests under two scenarios: (1) when the reference test can be considered a gold standard and (2) when the reference test cannot be considered a gold standard. In the first scenario, we first review the conventio...

journal_title：Statistical methods in medical research

authors： Ma X,Nie L,Cole SR,Chu H

更新日期：2016-08-01 00:00:00

abstract：:The accuracy of a diagnostic test, which is often quantified by a pair of measures such as sensitivity and specificity, is critical for medical decision making. Separate studies of an investigational diagnostic test can be combined through meta-analysis; however, such an analysis can be threatened by publication bias....

journal_title：Statistical methods in medical research

authors： Piao J,Liu Y,Chen Y,Ning J

更新日期：2019-10-01 00:00:00

abstract：:Bayes or empirical Bayes methods to improve inferential accuracy for a population mean has been widely adopted in medical research. As the joint prior distribution of both the mean and variance parameters can be difficult to specify or estimate, most of these methods have relied on certain level of simplifications of ...

journal_title：Statistical methods in medical research

authors： Shen C

更新日期：2019-06-01 00:00:00

abstract：:Competing risks data often exist within a center in multi-center randomized clinical trials where the treatment effects or baseline risks may vary among centers. In this paper, we propose a subdistribution hazard regression model with multivariate frailty to investigate heterogeneity in treatment effects among centers...

journal_title：Statistical methods in medical research

authors： Ha ID,Christian NJ,Jeong JH,Park J,Lee Y

更新日期：2016-12-01 00:00:00

abstract：:We propose a flexible and computationally efficient penalized estimation method for a semi-parametric linear transformation model with current status data. To facilitate model fitting, the unknown monotone function is approximated by monotone B-splines, and a computationally efficient hybrid algorithm involving the Fi...

journal_title：Statistical methods in medical research

authors： Lu M,Liu Y,Li CS

更新日期：2020-01-01 00:00:00

abstract：:Multilevel models were originally developed to allow linear regression or ANOVA models to be applied to observations that are not mutually independent. This lack of independence commonly arises due to clustering of the units of observations into 'higher level units' such as patients in hospitals. In linear mixed model...

journal_title：Statistical methods in medical research

authors： Rabe-Hesketh S,Yang S,Pickles A

更新日期：2001-12-01 00:00:00

abstract：:Comparison of sequences that have descended from a common ancestor based on an explicit stochastic model of substitutions, insertions and deletions has risen to prominence in the last decade. Making statements about the positions of insertions-deletions (abbr. indels) is central in sequence and genome analysis and is ...

journal_title：Statistical methods in medical research

authors： Miklós I,Novák A,Satija R,Lyngsø R,Hein J

更新日期：2009-10-01 00:00:00

abstract：:Joint models for recurrent and terminal events have not been yet developed for clustered data. The goals of our study are to develop a statistical framework for modelling clustered recurrent and terminal events and to perform dynamic predictions of the terminal event in family studies. We propose a joint nested frailt...

journal_title：Statistical methods in medical research

authors： Choi YH,Jacqmin-Gadda H,Król A,Parfrey P,Briollais L,Rondeau V

更新日期：2020-05-01 00:00:00

abstract：:When comparing two different kinds of group therapy or two individual treatments where patients within each arm are nested within care providers, clustering of observations may occur in both arms. The arms may differ in terms of (a) the intraclass correlation, (b) the outcome variance, (c) the cluster size, and (d) th...

journal_title：Statistical methods in medical research

authors： Candel MJ,van Breukelen GJ

更新日期：2015-10-01 00:00:00

abstract：:We review recent work on the application of pseudo-observations in survival and event history analysis. This includes regression models for parameters like the survival function in a single point, the restricted mean survival time and transition or state occupation probabilities in multi-state models, e.g. the competi...

journal_title：Statistical methods in medical research

authors： Andersen PK,Perme MP

更新日期：2010-02-01 00:00:00

abstract：:Researchers and clinicians often need to know whether a new method of measurement is equivalent to an established one that is already in use. For this problem, the estimation of limits of agreement advocated by Bland and Altman is a widely used solution. However, this approach ignores two vital issues in method compar...

journal_title：Statistical methods in medical research

authors： Alanen E

更新日期：2012-08-01 00:00:00

abstract：:This article outlines the statistical developments that have taken place in the use of the EM algorithm in emission and transmission tomography during the past decade or so. We discuss the statistical aspects of the modelling of the projection data for both the emission and transmission cases and define the relevant p...

journal_title：Statistical methods in medical research

authors： Kay J

更新日期：1997-03-01 00:00:00

abstract：:Rapid Early Action for Coronary Treatment (REACT) was a multisite trial testing a community intervention to reduce the delay between onset of symptoms of acute myocardial infarction (MI) and patients' arrival at a hospital emergency department. The study employed a group-randomized trial design, with ten communities r...

journal_title：Statistical methods in medical research

authors： Murray DM,Feldman HA,McGovern PG

更新日期：2000-04-01 00:00:00

abstract：:Variable selection in semiparametric mixed models for longitudinal data remains a challenge, especially in the presence of multiple correlated outcomes. In this paper, we propose a model selection procedure that simultaneously selects fixed and random effects using a maximum penalized likelihood method with the adapti...

journal_title：Statistical methods in medical research

authors： Li Z,Liu H,Tu W

更新日期：2018-10-01 00:00:00

abstract：:This paper presents and compares several methods of measuring continuous baseline covariate imbalance in clinical trial data. Simulations illustrate that though the t-test is an inappropriate method of assessing continuous baseline covariate imbalance, the test statistic itself is a robust measure in capturing imbalan...

journal_title：Statistical methods in medical research

authors： Ciolino JD,Martin RH,Zhao W,Hill MD,Jauch EC,Palesch YY

更新日期：2015-04-01 00:00:00

abstract：:In the clinical development of some new infectious disease drugs, early clinical pharmacology trials may predict with high confidence that the efficacious doses are well below the range of the safety margin. In this case, a dose-ranging study may be unnecessary after a proof-of-concept (PoC) study testing the highest ...

journal_title：Statistical methods in medical research

authors： Fan XF,Gallo P,Su G,Menton R,Segal F

更新日期：2019-12-01 00:00:00

abstract：:Statistical methods for spatial health data to identify the significant covariates associated with the health outcomes are of critical importance. Most studies have developed variable selection approaches in which the covariates included appear within the spatial domain and their effects are fixed across space. Howeve...

journal_title：Statistical methods in medical research

authors： Choi J,Lawson AB

更新日期：2018-01-01 00:00:00

abstract：:Regression models are frequently used to model the functional relationship between an interesting outcome parameter and one or more potentially relevant explanatory variables. Objectives can be to set up as a prognostic model, for example, or an estimation model for a certain parameter of interest. Determining half-li...

journal_title：Statistical methods in medical research

authors： Mayer B,Keller F,Syrovets T,Wittau M

更新日期：2016-10-01 00:00:00

abstract：:Most statistical developments in the joint modelling area have focused on the shared random-effect models that include characteristics of the longitudinal marker as predictors in the model for the time-to-event. A less well-known approach is the joint latent class model which consists in assuming that a latent class s...

journal_title：Statistical methods in medical research

authors： Proust-Lima C,Séne M,Taylor JM,Jacqmin-Gadda H

更新日期：2014-02-01 00:00:00

abstract：:A dynamic treatment regime is a set of decision rules for how to treat a patient at multiple time points. At each time point, a treatment decision is made depending on the patient's medical history up to that point. We consider the infinite-horizon setting in which the number of decision points is very large. Specific...

journal_title：Statistical methods in medical research

authors： Goldberg Y,Pollak M,Mitelpunkt A,Orlovsky M,Weiss-Meilik A,Gorfine M

更新日期：2017-08-01 00:00:00

abstract：:The estimation of population parameters using complex survey data requires careful statistical modelling to account for the design features. This is further complicated by unit and item nonresponse for which a number of methods have been developed in order to reduce estimation bias. In this paper, we address some issu...

journal_title：Statistical methods in medical research

authors： Geraci M

更新日期：2016-08-01 00:00:00

abstract：:We propose a novel likelihood method for analyzing time-to-event data when multiple events and multiple missing data intervals are possible prior to the first observed event for a given subject. This research is motivated by data obtained from a heart monitor used to track the recovery process of subjects experiencing...

journal_title：Statistical methods in medical research

authors： Green CL,Brownie C,Boos DD,Lu JC,Krucoff MW

更新日期：2016-04-01 00:00:00

abstract：:The three-class Youden index serves both as a measure of medical test accuracy and a criterion to choose the optimal pair of cutoff values for classifying subjects into three ordinal disease categories (e.g. no disease, mild disease, advanced disease). We present a Bayesian nonparametric approach for estimating the th...

journal_title：Statistical methods in medical research

authors： Carvalho VI,Branscum AJ

更新日期：2018-03-01 00:00:00

abstract：:Couples with diseases associated with the sexual chromosomes, as well as families in countries where the desire for a male is extreme, are interested in influencing the sex of the baby. We propose an original composite likelihood approach to analyse the relation between sex of the newborn and timing of the intercourse...

journal_title：Statistical methods in medical research

authors： Tiberi S,Scarpa B,Sartori N

更新日期：2018-11-01 00:00:00

abstract：:Censored data make survival analysis more complicated because exact event times are not observed. Statistical methodology developed to account for censored observations assumes that patients' withdrawal from a study is independent of the event of interest. However, in practice, some covariates might be associated to b...

journal_title：Statistical methods in medical research

authors： Willems S,Schat A,van Noorden MS,Fiocco M

更新日期：2018-02-01 00:00:00

abstract：:Purpose The prevalence estimates of binary variables in sample surveys are often subject to two systematic errors: measurement error and nonresponse bias. A multiple-bias analysis is essential to adjust for both biases. Methods In this paper, we linked the latent class log-linear and proxy pattern-mixture models to ad...

journal_title：Statistical methods in medical research

authors： Khalagi K,Mansournia MA,Motevalian SA,Nourijelyani K,Rahimi-Movaghar A,Bakhtiyari M

更新日期：2018-10-01 00:00:00