abstract：:We examine the use of randomization-based inference for analyzing multiarmed randomized clinical trials, including the application of conditional randomization tests to multiple comparisons. The view is taken that the linkage of the statistical test to the experimental design (randomization procedure) should be recogn...

journal_title：Statistics in medicine

authors： Wang Y,Rosenberger WF,Uschner D

更新日期：2020-02-20 00:00:00

abstract：:Despite our best efforts, missing outcomes are common in randomized controlled clinical trials. The National Research Council's Committee on National Statistics panel report titled The Prevention and Treatment of Missing Data in Clinical Trials noted that further research is required to assess the impact of missing da...

journal_title：Statistics in medicine

authors： Cook T,Zea R

更新日期：2020-01-30 00:00:00

abstract：:Quantifying socioeconomic disparities and understanding the roots of inequalities are growing topics in cancer research. However, socioeconomic differences are challenging to investigate mainly due to the lack of accurate data at individual-level, while aggregate indicators are only partially informative. We implement...

journal_title：Statistics in medicine

authors： Mezzetti M,Palli D,Dominici F

更新日期：2020-01-15 00:00:00

abstract：:Few methods are currently available for group sequential analysis of recurrent events data subject to a terminal event in the clinical trial setting. This research helps fill this gap by developing a completely nonparametric group sequential monitoring procedure for use with the two-sample Tayob and Murray statistic. ...

journal_title：Statistics in medicine

authors： Xia M,Murray S,Tayob N

更新日期：2019-12-30 00:00:00

abstract：:In the analysis of composite endpoints in a clinical trial, time to first event analysis techniques such as the logrank test and Cox proportional hazard test do not take into account the multiplicity, importance, and the severity of events in the composite endpoint. Several generalized pairwise comparison analysis met...

journal_title：Statistics in medicine

authors： Verbeeck J,Spitzer E,de Vries T,van Es GA,Anderson WN,Van Mieghem NM,Leon MB,Molenberghs G,Tijssen J

更新日期：2019-12-30 00:00:00

abstract：:Many neuroscientists are interested in how connectomes (graphical representations of functional connectivity between areas of the brain) change in relation to covariates. In statistics, changes like this are analyzed using regression, where the outcomes or dependent variables are regressed onto the covariates. However...

journal_title：Statistics in medicine

authors： Yang D,Deych E,Shands B,Campbell MC,Perlmutter JS,Petersen S,Schlaggar BL,Shannon W

更新日期：2019-12-20 00:00:00

abstract：:Statistical inference based on correlated count measurements are frequently performed in biomedical studies. Most of existing sample size calculation methods for count outcomes are developed under the Poisson model. Deviation from the Poisson assumption (equality of mean and variance) has been widely documented in pra...

journal_title：Statistics in medicine

authors： Li D,Zhang S,Cao J

更新日期：2019-12-10 00:00:00

abstract：:Cox proportional hazard regression model is a popular tool to analyze the relationship between a censored lifetime variable with other relevant factors. The semiparametric Cox model is widely used to study different types of data arising from applied disciplines such as medical science, biology, and reliability studie...

journal_title：Statistics in medicine

authors： Ghosh A,Basu A

更新日期：2019-11-30 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：:Differences between arm-based (AB) and contrast-based (CB) models for network meta-analysis (NMA) are controversial. We compare the CB model of Lu and Ades (2006), the AB model of Hong et al(2016), and two intermediate models, using hypothetical data and a selected real data set. Differences between models arise prima...

journal_title：Statistics in medicine

authors： White IR,Turner RM,Karahalios A,Salanti G

更新日期：2019-11-30 00:00:00

abstract：:Subjects in randomized controlled trials do not always comply to the treatment condition they have been assigned to. This may cause the estimated effect of the intervention to be biased and also affect efficiency, coverage of confidence intervals, and statistical power. In cluster randomized trials non-compliance may ...

journal_title：Statistics in medicine

authors： Moerbeek M,Schie SV

更新日期：2019-11-20 00:00:00

abstract：:We suggest measures to quantify the degrees of necessity and of sufficiency of prognostic factors for dichotomous and for survival outcomes. A cause, represented by certain values of prognostic factors, is considered necessary for an event if, without the cause, the event cannot develop. It is considered sufficient fo...

journal_title：Statistics in medicine

authors： Gleiss A,Schemper M

更新日期：2019-10-15 00:00:00

abstract：:We consider the problem of identifying subgroups of participants in a clinical trial that have enhanced treatment effect. Recursive partitioning methods that recursively partition the covariate space based on some measure of between groups treatment effect difference are popular for such subgroup identification. The m...

journal_title：Statistics in medicine

authors： Steingrimsson JA,Yang J

更新日期：2019-09-20 00:00:00

abstract：:A stochastic approximation EM algorithm (SAEM) is described for exploratory factor analysis of dichotomous or ordinal variables. The factor structure is obtained from sufficient statistics that are updated during iterations with the Robbins-Monro procedure. Two large-scale simulations are reported that compare accurac...

journal_title：Statistics in medicine

authors： Camilli G,Geis E

更新日期：2019-09-20 00:00:00

abstract：:Assessing the calibration of methods for estimating the probability of the occurrence of a binary outcome is an important aspect of validating the performance of risk-prediction algorithms. Calibration commonly refers to the agreement between predicted and observed probabilities of the outcome. Graphical methods are a...

journal_title：Statistics in medicine

authors： Austin PC,Steyerberg EW

更新日期：2019-09-20 00:00:00

abstract：:In observational studies with censored data, exposure-outcome associations are commonly measured with adjusted hazard ratios from multivariable Cox proportional hazards models. The difference in restricted mean survival times (RMSTs) up to a pre-specified time point is an alternative measure that offers a clinically m...

journal_title：Statistics in medicine

authors： Conner SC,Sullivan LM,Benjamin EJ,LaValley MP,Galea S,Trinquart L

更新日期：2019-09-10 00:00:00

abstract：:In many countries, the monitoring of child growth does not occur in a regular manner, and instead, we may have to rely on sporadic observations that are subject to substantial measurement error. In these countries, it can be difficult to identify patterns of poor growth, and faltering children may miss out on essentia...

journal_title：Statistics in medicine

authors： Anderson C,Xiao L,Checkley W

更新日期：2019-08-30 00:00:00

abstract：:Girardeau, Ravaud and Donner in 2008 presented a formula for sample size calculations for cluster randomised crossover trials, when the intracluster correlation coefficient, interperiod correlation coefficient and mean cluster size are specified in advance. However, in many randomised trials, the number of clusters is...

journal_title：Statistics in medicine

authors： Kelly TL,Pratt N

更新日期：2019-08-15 00:00:00

abstract：:We consider statistical procedures for hypothesis testing of real valued functionals of matched pairs with missing values. In order to improve the accuracy of existing methods, we propose a novel multiplication combination procedure. Dividing the observed data into dependent (completely observed) pairs and independent...

journal_title：Statistics in medicine

authors： Amro L,Konietschke F,Pauly M

更新日期：2019-07-30 00:00:00

abstract：:In psychiatry, clinicians use criteria sets from the Diagnostic and Statistical Manual of Mental Disorders to diagnose mental disorders. Most criteria sets have several symptom domains, and in order to be diagnosed, an individual must meet the minimum number of symptoms required by each domain. Some efforts are now fo...

journal_title：Statistics in medicine

authors： Mauro C,Shear MK,Wang Y

更新日期：2019-07-20 00:00:00

abstract：:In recent years, quality control charts have been increasingly applied in the healthcare environment, for example, to monitor surgical performance. Risk-adjusted cumulative (CUSUM) charts that utilize risk scores like the Parsonnet score to estimate the probability of death of a patient from an operation turn out to b...

journal_title：Statistics in medicine

authors： Knoth S,Wittenberg P,Gan FF

更新日期：2019-05-30 00:00:00

abstract：:Correlation is inherent in longitudinal studies due to the repeated measurements on subjects, as well as due to time-dependent covariates in the study. In the National Longitudinal Study of Adolescent to Adult Health (Add Health), data were repeatedly collected on children in grades 7-12 across four waves. Thus, obser...

journal_title：Statistics in medicine

authors： Irimata KM,Broatch J,Wilson JR

更新日期：2019-05-30 00:00:00

abstract：:In this paper, we develop methods to combine multiple biomarker trajectories into a composite diagnostic marker using functional data analysis (FDA) to achieve better diagnostic accuracy in monitoring disease recurrence in the setting of a prospective cohort study. In such studies, the disease status is usually verifi...

journal_title：Statistics in medicine

authors： Li H,Gatsonis C

更新日期：2019-05-20 00:00:00

abstract：:Binary classification rules based on covariates typically depend on simple loss functions such as zero-one misclassification. Some cases may require more complex loss functions. For example, individual-level monitoring of HIV-infected individuals on antiretroviral therapy requires periodic assessment of treatment fail...

journal_title：Statistics in medicine

authors： Xu Y,Liu T,Daniels MJ,Kantor R,Mwangi A,Hogan JW

更新日期：2019-05-20 00:00:00

abstract：:Instrumental variable is an essential tool for addressing unmeasured confounding in observational studies. Two-stage predictor substitution (2SPS) estimator and two-stage residual inclusion (2SRI) are two commonly used approaches in applying instrumental variables. Recently, 2SPS was studied under the additive hazards...

journal_title：Statistics in medicine

authors： Ying A,Xu R,Murphy J

更新日期：2019-05-10 00:00:00

abstract：:The lifetime risk of a clinical condition is the probability of onset of the condition during one's lifespan. Recent advances in Alzheimer's disease (AD) research have identified screening tests for biomarkers that can identify persons who are in the earliest stages of the AD process but who do not yet have any clinic...

journal_title：Statistics in medicine

authors： Brookmeyer R,Abdalla N

更新日期：2019-04-30 00:00:00

abstract：:The "some invalid, some valid instrumental variable estimator" (sisVIVE) is a lasso-based method for instrumental variables (IVs) regression of outcome on an exposure. In principle, sisVIVE is robust to some of the IVs in the analysis being invalid, in the sense of being related to the outcome variable through pathway...

journal_title：Statistics in medicine

authors： Bao Y,Clarke PS,Smart M,Kumari M

更新日期：2019-04-30 00:00:00

abstract：:Statistical methods for analyzing recurrent events have attracted significant attention. The majority of existing works consider situations in which subjects are observed over time periods and events of interest that occurred during the course of follow-up are recorded. In some applications, a subject may leave the st...

journal_title：Statistics in medicine

authors： Su PF,Zhong J,Ou HT

更新日期：2019-04-15 00:00:00

abstract：:One Sentence Summary: A Bayesian repeated measures model based on quantitative muscle strength data from a prospective Natural History Study was developed to determine disease progression and design clinical trials for GNE myopathy, a rare and slowly progressive muscle disease. GNE myopathy is a rare muscle disease ch...

journal_title：Statistics in medicine

authors： Quintana M,Shrader J,Slota C,Joe G,McKew JC,Fitzgerald M,Gahl WA,Berry S,Carrillo N

更新日期：2019-04-15 00:00:00

abstract：:We extend the method proposed in a recent work by the Authors for trial-level general surrogate evaluation to allow combinations of biomarkers and provide a procedure for finding the "best" combination of biomarkers based on the absolute prediction error summary of surrogate quality. We use a nonparametric Bayesian mo...

journal_title：Statistics in medicine

authors： Gabriel EE,Sachs MC,Daniels MJ,Halloran ME

更新日期：2019-03-30 00:00:00

abstract：:When designing a study to develop a new prediction model with binary or time-to-event outcomes, researchers should ensure their sample size is adequate in terms of the number of participants (n) and outcome events (E) relative to the number of predictor parameters (p) considered for inclusion. We propose that the mini...

journal_title：Statistics in medicine

authors： Riley RD,Snell KI,Ensor J,Burke DL,Harrell FE Jr,Moons KG,Collins GS

更新日期：2019-03-30 00:00:00

abstract：:In the medical literature, hundreds of prediction models are being developed to predict health outcomes in individuals. For continuous outcomes, typically a linear regression model is developed to predict an individual's outcome value conditional on values of multiple predictors (covariates). To improve model developm...

journal_title：Statistics in medicine

authors： Riley RD,Snell KIE,Ensor J,Burke DL,Harrell FE Jr,Moons KGM,Collins GS

更新日期：2019-03-30 00:00:00

abstract：:Identification of the latency period for the effect of a time-varying exposure is key when assessing many environmental, nutritional, and behavioral risk factors. A pre-specified exposure metric involving an unknown latency parameter is often used in the statistical model for the exposure-disease relationship. Likelih...

journal_title：Statistics in medicine

authors： Peskoe SB,Spiegelman D,Wang M

更新日期：2019-03-30 00:00:00

abstract：:In biomedical studies and clinical trials, repeated measures are often subject to some upper and/or lower limits of detection. Hence, the responses are either left or right censored. A complication arises when more than one series of responses is repeatedly collected on each subject at irregular intervals over a perio...

journal_title：Statistics in medicine

authors： Lachos VH,A Matos L,Castro LM,Chen MH

更新日期：2019-03-15 00:00:00

abstract：:The use of the last observation carried forward (LOCF) method for imputing missing outcome data in randomized clinical trials has been much criticized and its shortcomings are well understood. However, only recently have published studies widely started using more appropriate imputation methods. Consequently, meta-ana...

journal_title：Statistics in medicine

authors： Mavridis D,Salanti G,Furukawa TA,Cipriani A,Chaimani A,White IR

更新日期：2019-02-28 00:00:00

abstract：:In this paper, we consider fitting a flexible and interpretable additive regression model in a data-rich setting. We wish to avoid pre-specifying the functional form of the conditional association between each covariate and the response, while still retaining interpretability of the fitted functions. A number of recen...

journal_title：Statistics in medicine

authors： Petersen A,Witten D

更新日期：2019-02-20 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：: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：: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：: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