Estimation of data adaptive minimal clinically important difference with a nonconvex optimization procedure.

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：: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 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：:Propensity score methods are common for estimating a binary treatment effect when treatment assignment is not randomized. When exposure is measured on an ordinal scale (i.e. low-medium-high), however, propensity score inference requires extensions which have received limited attention. Estimands of possible interest w...

journal_title：Statistical methods in medical research

authors： Lopez MJ,Gutman R

更新日期：2017-04-01 00:00:00

abstract：:The threat of pandemics has made influenza surveillance systems a priority in epidemiology services around the world. The emergence of A-H1N1 influenza has required accurate surveillance systems in order to undertake specific actions only when and where they are necessary. In that sense, the main goal of this article ...

journal_title：Statistical methods in medical research

authors： Martinez-Beneito MA,Botella-Rocamora P,Zurriaga O

更新日期：2011-04-01 00:00:00

abstract：:Identification of cancer patient subgroups using high throughput genomic data is of critical importance to clinicians and scientists because it can offer opportunities for more personalized treatment and overlapping treatments of cancers. In spite of tremendous efforts, this problem still remains challenging because o...

journal_title：Statistical methods in medical research

authors： Wei W,Sun Z,da Silveira WA,Yu Z,Lawson A,Hardiman G,Kelemen LE,Chung D

更新日期：2019-07-01 00:00:00

abstract：:We consider two exact unconditional procedures to test the difference between two multinomials with ordered categorical data. Exact unconditional procedures are compared to other approaches based on the Wilcoxon mid-rank test and the proportional odds model. We use a real example from an arthritis pain study to illust...

journal_title：Statistical methods in medical research

authors： Shan G,Ma C

更新日期：2016-02-01 00:00:00

abstract：BACKGROUND:Recent literature on the comparison of machine learning methods has raised questions about the neutrality, unbiasedness and utility of many comparative studies. Reporting of results on favourable datasets and sampling error in the estimated performance measures based on single samples are thought to be the m...

journal_title：Statistical methods in medical research

authors： Khondoker M,Dobson R,Skirrow C,Simmons A,Stahl D

更新日期：2016-10-01 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：:This paper presents a new model-based generalized functional clustering method for discrete longitudinal data, such as multivariate binomial and Poisson distributed data. For this purpose, we propose a multivariate functional principal component analysis (MFPCA)-based clustering procedure for a latent multivariate Gau...

journal_title：Statistical methods in medical research

authors： Lim Y,Cheung YK,Oh HS

更新日期：2020-11-01 00:00:00

abstract：:The positivity assumption, or the experimental treatment assignment (ETA) assumption, is important for identifiability in causal inference. Even if the positivity assumption holds, practical violations of this assumption may jeopardize the finite sample performance of the causal estimator. One of the consequences of p...

journal_title：Statistical methods in medical research

authors： Ju C,Schwab J,van der Laan MJ

更新日期：2019-06-01 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：: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：:Optimal therapeutic decisions can be made according to disease prognosis, where the residual lifetime is extensively used because of its straightforward interpretation and formula. To predict the residual lifetime in a dynamic manner, a longitudinal biomarker that is repeatedly measured during the post-baseline follow...

journal_title：Statistical methods in medical research

authors： Lin X,Li R,Yan F,Lu T,Huang X

更新日期：2019-04-01 00:00:00

abstract：:Many statistical studies report p-values for inferential purposes. In several scenarios, the stochastic aspect of p-values is neglected, which may contribute to drawing wrong conclusions in real data experiments. The stochastic nature of p-values makes their use to examine the performance of given testing procedures o...

journal_title：Statistical methods in medical research

authors： Vexler A,Yu J,Zhao Y,Hutson AD,Gurevich G

更新日期：2018-12-01 00:00:00

abstract：:This paper reviews recent developments by the Washington/Brown groups for the study of anatomical shape in the emerging new discipline of computational anatomy. Parametric representations of anatomical variation for computational anatomy are reviewed, restricted to the assumption of small deformations. The generation ...

journal_title：Statistical methods in medical research

authors： Miller M,Banerjee A,Christensen G,Joshi S,Khaneja N,Grenander U,Matejic L

更新日期：1997-09-01 00:00:00

abstract：:This paper illustrates the use of multidimensional scaling methods (MDS) to examine space-time patterns in epidemic data. The paper begins by outlining the principles of MDS. The model is then formally specified and illustrated by application to two data sets. The first is partly a tutorial example. It uses monthly re...

journal_title：Statistical methods in medical research

authors： Cliff AD,Haggett P,Smallman-Raynor MR,Stroup DF,Williamson GD

更新日期：1995-06-01 00:00:00

abstract：:Sample size calculations are needed to design and assess the feasibility of case-control studies. Although such calculations are readily available for simple case-control designs and univariate analyses, there is limited theory and software for multivariate unconditional logistic analysis of case-control data. Here we...

journal_title：Statistical methods in medical research

authors： Gail MH,Haneuse S

更新日期：2019-03-01 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：:Two tests are proposed for checking the linearity of nonparametric function in partially linear models. The first one is based on a Crámer-von Mises statistic. This test can detect the local alternative converging to the null at the parametric rate 1/square root n. A bootstrap resample technique is provided to calcula...

journal_title：Statistical methods in medical research

authors： Liang H

更新日期：2006-06-01 00:00:00

abstract：:Longitudinal zero-inflated count data are encountered frequently in substance-use research when assessing the effects of covariates and risk factors on outcomes. Often, both the time to a terminal event such as death or dropout and repeated measure count responses are collected for each subject. In this setting, the l...

journal_title：Statistical methods in medical research

authors： Zhu H,DeSantis SM,Luo S

更新日期：2018-04-01 00:00:00

abstract：:Clinical trials investigating the efficacy of two or more doses of an experimental treatment compared to a single reference arm are not uncommon. In such situations, if each dose is compared to the reference arm using an un-adjusted significance level, consideration of the Type I familywise error is likely to be requi...

journal_title：Statistical methods in medical research

authors： Fernandes N,Stone A

更新日期：2011-12-01 00:00:00

abstract：:In many applications of zero-inflated models, score tests are often used to evaluate whether the population heterogeneity as implied by these models is consistent with the data. The most frequently cited justification for using score tests is that they only require estimation under the null hypothesis. Because this es...

journal_title：Statistical methods in medical research

authors： Hsu WW,Todem D,Mawella NR,Kim K,Rosenkranz RR

更新日期：2020-12-01 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：:In reliability theory, diagnostic accuracy, and clinical trials, the quantity P ( X > Y ) + ...

journal_title：Statistical methods in medical research

authors： Verbeeck J,Deltuvaite-Thomas V,Berckmoes B,Burzykowski T,Aerts M,Thas O,Buyse M,Molenberghs G

更新日期：2020-12-01 00:00:00

abstract：:Cancer mortality risk estimates are essential for planning resource allocation and designing and evaluating cancer prevention and management strategies. However, mortality figures generally become available after a few years, making necessary to develop reliable procedures to provide current and near future mortality ...

journal_title：Statistical methods in medical research

authors： Ugarte MD,Goicoa T,Etxeberria J,Militino AF

更新日期：2012-10-01 00:00:00

abstract：:Covariate-adaptive designs are widely used to balance covariates and maintain randomization in clinical trials. Adaptive designs for discrete covariates and their asymptotic properties have been well studied in the literature. However, important continuous covariates are often involved in clinical studies. Simply disc...

journal_title：Statistical methods in medical research

authors： Li X,Zhou J,Hu F

更新日期：2019-06-01 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：: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：: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