A proof-of-concept-to-confirmatory multiple adaptation design in the development of an anti-viral treatment.

abstract：:Pattern-mixture model (PMM)-based controlled imputations have become a popular tool to assess the sensitivity of primary analysis inference to different post-dropout assumptions or to estimate treatment effectiveness. The methodology is well established for continuous responses but less well established for binary res...

journal_title：Statistical methods in medical research

authors： Lu K

更新日期：2020-12-01 00:00:00

abstract：:For semi-continuous data which are a mixture of true zeros and continuously distributed positive values, the use of two-part mixed models provides a convenient modelling framework. However, deriving population-averaged (marginal) effects from such models is not always straightforward. Su et al. presented a model that ...

journal_title：Statistical methods in medical research

authors： Tom BD,Su L,Farewell VT

更新日期：2016-10-01 00:00:00

abstract：:Cluster-level dynamic treatment regimens can be used to guide sequential treatment decision-making at the cluster level in order to improve outcomes at the individual or patient-level. In a cluster-level dynamic treatment regimen, the treatment is potentially adapted and re-adapted over time based on changes in the cl...

journal_title：Statistical methods in medical research

authors： NeCamp T,Kilbourne A,Almirall D

更新日期：2017-08-01 00:00:00

abstract：BACKGROUND:Dyspepsia diagnoses and treatment decisions are made in situations in which multiple factors must be taken into account. Evolving from neuro-biological insights, artificial neural networks (ANNs) can employ multiple factors in resolving medical prediction, classification, pattern recognition, and pattern com...

journal_title：Statistical methods in medical research

authors： García-Altés A,Santín D,Barenys M

更新日期：2007-08-01 00:00:00

abstract：:The statistical analysis of genome-wide association studies (GWASs) with multiple diseases and shared controls (SCs) is discussed. The usual method for analyzing data from these studies is to compare each individual disease with either the SCs or the pooled controls which include other diseases. We observed that apply...

journal_title：Statistical methods in medical research

authors： Chen Z,Huang H,Ng HK

更新日期：2016-04-01 00:00:00

abstract：:The semiparametric Cox regression model is often fitted in the modeling of survival data. One of its main advantages is the ease of interpretation, as long as the hazards rates for two individuals do not vary over time. In practice the proportionality assumption of the hazards may not be true in some situations. In ad...

journal_title：Statistical methods in medical research

authors： Calsavara VF,Milani EA,Bertolli E,Tomazella V

更新日期：2020-08-01 00:00:00

abstract：:The random effects model in meta-analysis is a standard statistical tool often used to analyze the effect sizes of the quantity of interest if there is heterogeneity between studies. In the special case considered here, meta-analytic data contain only the sample means in two treatment arms and the sample sizes, but no...

journal_title：Statistical methods in medical research

authors： Sangnawakij P,Böhning D,Niwitpong SA,Adams S,Stanton M,Holling H

更新日期：2019-01-01 00:00:00

abstract：:Several epidemiological parameters have been introduced for quantifying the population impact of a certain exposure on morbidity on a population level, termed 'attributable risk' (AR). Of these definitions, the AR as suggested by Levin in 1953 or some algebraic transformations of it are most commonly used. A structure...

journal_title：Statistical methods in medical research

authors： Uter W,Pfahlberg A

更新日期：2001-06-01 00:00:00

abstract：:In biomedical research, various longitudinal markers measuring different quantities are often collected over time. For example, repeated measures of psychometric scores are very informative about the degradation process toward dementia. These trajectories are generally nonlinear with an acceleration of the decline a f...

journal_title：Statistical methods in medical research

authors： Segalas C,Helmer C,Jacqmin-Gadda H

更新日期：2020-09-01 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：:In recent years, there has been a prominent discussion in the literature about the potential for overestimation of the treatment effect when a clinical trial stops at an interim analysis due to the experimental treatment showing a benefit over the control. However, there has been much less attention paid to the conver...

journal_title：Statistical methods in medical research

authors： Marschner IC,Schou IM

更新日期：2019-10-01 00:00:00

abstract：:Nonlinear mixed-effects modeling is one of the most popular tools for analyzing repeated measurement data, particularly for applications in the biomedical fields. Multiple integration and nonlinear optimization are the two major challenges for likelihood-based methods in nonlinear mixed-effects modeling. To solve thes...

journal_title：Statistical methods in medical research

authors： Yuan M,Zhu Z,Yang Y,Zhao M,Sasser K,Hamadeh H,Pinheiro J,Xu XS

更新日期：2020-08-24 00:00:00

abstract：:Clinical trials are expensive and time-consuming and so should also be used to study how treatments work, allowing for the evaluation of theoretical treatment models and refinement and improvement of treatments. These treatment processes can be studied using mediation analysis. Randomised treatment makes some of the a...

journal_title：Statistical methods in medical research

authors： Goldsmith KA,Chalder T,White PD,Sharpe M,Pickles A

更新日期：2018-06-01 00:00:00

abstract：:Surveys are key means of obtaining policy-relevant information not available from routine sources. Bias arising from non-participation is typically handled by applying weights derived from limited socio-demographic characteristics. This approach neither captures nor adjusts for differences in health and related behavi...

journal_title：Statistical methods in medical research

authors： Gray L,Gorman E,White IR,Katikireddi SV,McCartney G,Rutherford L,Leyland AH

更新日期：2020-04-01 00:00:00

abstract：:Guarding against false positive selections is important in many applications. We discuss methods based on subsampling and sample splitting for controlling the expected number of false positives and assigning p-values. They are generic and especially useful for high-dimensional settings. We review encouraging results f...

journal_title：Statistical methods in medical research

authors： Bühlmann P,Rütimann P,Kalisch M

更新日期：2013-10-01 00:00:00

abstract：:We often seek to estimate the impact of an exposure naturally occurring or randomly assigned at the cluster-level. For example, the literature on neighborhood determinants of health continues to grow. Likewise, community randomized trials are applied to learn about real-world implementation, sustainability, and popula...

journal_title：Statistical methods in medical research

authors： Balzer LB,Zheng W,van der Laan MJ,Petersen ML

更新日期：2019-06-01 00:00:00

abstract：:In multi-regional trials, the underlying overall and region-specific accrual rates often do not hold constant over time and different regions could have different start-up times, which combined with initial jump in accrual within each region often leads to a discontinuous overall accrual rate, and these issues associa...

journal_title：Statistical methods in medical research

authors： Deng Y,Zhang X,Long Q

更新日期：2017-04-01 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：: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

abstract：:This paper reviews models for incomplete continuous and categorical longitudinal data. In terms of Rubin's classification of missing value processes we are specifically concerned with the problem of nonrandom missingness. A distinction is drawn between the classes of selection and pattern-mixture models and, using sev...

journal_title：Statistical methods in medical research

authors： Kenward MG,Molenberghs G

更新日期：1999-03-01 00:00:00

abstract：:Spatial resolution plays an important role in functional magnetic resonance imaging studies as the signal-to-noise ratio increases linearly with voxel volume. In scientific studies, where functional magnetic resonance imaging is widely used, the standard spatial resolution typically used is relatively low which ensure...

journal_title：Statistical methods in medical research

authors： Liu Z,Bartsch AJ,Berrocal VJ,Johnson TD

更新日期：2019-04-01 00:00:00

abstract：:Within paediatric populations, there may be distinct age groups characterised by different exposure-response relationships. Several regulatory guidance documents have suggested general age groupings. However, it is not clear whether these categorisations will be suitable for all new medicines and in all disease areas....

journal_title：Statistical methods in medical research

authors： Wadsworth I,Hampson LV,Bornkamp B,Jaki T

更新日期：2020-09-01 00:00:00

abstract：:Publication bias frequently appears in meta-analyses when the included studies' results (e.g., p-values) influence the studies' publication processes. Some unfavorable studies may be suppressed from publication, so the meta-analytic results may be biased toward an artificially favorable direction. Many statistical tes...

journal_title：Statistical methods in medical research

authors： Lin L

更新日期：2020-10-01 00:00:00

abstract：:One purpose of a longitudinal study is to gain a better understanding of how an outcome of interest changes among a given population over time. In what follows, a trajectory will be taken to mean the series of measurements of the outcome variable for an individual. Group-based trajectory modelling methods seek to iden...

journal_title：Statistical methods in medical research

authors： Davies CE,Glonek GF,Giles LC

更新日期：2017-08-01 00:00:00

abstract：:We propose a semiparametric multi-state frailty model to analyze clustered event-history data subject to interval censoring. The proposed model is motivated by an attempt to study the life course of dental caries at the tooth level, taking into account the multiplicity of caries states and the intra-oral clustering of...

journal_title：Statistical methods in medical research

authors： Pak D,Li C,Todem D

更新日期：2019-09-01 00:00:00

abstract：:Agreement between two methods of clinical measurement can be quantified using the differences between observations made using the two methods on the same subjects. The 95% limits of agreement, estimated by mean difference +/- 1.96 standard deviation of the differences, provide an interval within which 95% of differenc...

journal_title：Statistical methods in medical research

authors： Bland JM,Altman DG

更新日期：1999-06-01 00:00:00

abstract：:Many non-experimental studies use propensity-score methods to estimate causal effects by balancing treatment and control groups on a set of observed baseline covariates. Full matching on the propensity score has emerged as a particularly effective and flexible method for utilizing all available data, and creating well...

journal_title：Statistical methods in medical research

authors： Austin PC,Stuart EA

更新日期：2017-12-01 00:00:00

abstract：:This study proposes semiparametric models for analysis of hierarchical count data containing excess zeros and overdispersion simultaneously. The methods discussed in this paper handle nonlinear covariate effects through flexible semiparametric multilevel regression techniques. This is performed by providing a comprehe...

journal_title：Statistical methods in medical research

authors： Mahmoodi M,Moghimbeigi A,Mohammad K,Faradmal J

更新日期：2018-04-01 00:00:00

abstract：:In the past two decades, it has become increasingly clear that genetic factors contribute to the aetiology of many common diseases including cancers, coronary disease, allergy and psychiatric disorders. While one goal of genetic epidemiological studies is to locate susceptibility genes for these complex diseases, it i...

journal_title：Statistical methods in medical research

authors： Liang KY,Beaty TH

更新日期：2000-12-01 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