abstract：:Optimal prevention and treatment strategies for a disease of multiple causes, such as pneumonia, must be informed by the population distribution of causes among cases, or cause-specific case fractions (CSCFs). CSCFs may further depend on additional explanatory variables. Existing methodological literature in disease e...

journal_title：Statistics in medicine

authors： Wu Z,Chen I

更新日期：2021-02-20 00:00:00

abstract：:The correct identification of change-points during ongoing outbreak investigations of infectious diseases is a matter of paramount importance in epidemiology, with major implications for the management of health care resources, public health and, as the COVID-19 pandemic has shown, social live. Onsets, peaks, and infl...

journal_title：Statistics in medicine

authors： Veres-Ferrer EJ,Pavía JM

更新日期：2021-02-20 00:00:00

abstract：:Clinical trials studying treatments for rare diseases are challenging to design and conduct due to the limited number of patients eligible for the trial. One design used to address this challenge is the small n, sequential, multiple assignment, randomized trial (snSMART). We propose a new snSMART design that investiga...

journal_title：Statistics in medicine

authors： Fang F,Hochstedler KA,Tamura RN,Braun TM,Kidwell KM

更新日期：2021-02-20 00:00:00

abstract：:The microbiome plays an important role in human health by mediating the path from environmental exposures to health outcomes. The relative abundances of the high-dimensional microbiome data have an unit-sum restriction, rendering standard statistical methods in the Euclidean space invalid. To address this problem, we ...

journal_title：Statistics in medicine

authors： Zhang H,Chen J,Feng Y,Wang C,Li H,Liu L

更新日期：2021-02-20 00:00:00

abstract：:In modern observational studies using electronic health records or other routinely collected data, both the outcome and covariates of interest can be error-prone and their errors often correlated. A cost-effective solution is the two-phase design, under which the error-prone outcome and covariates are observed for all...

journal_title：Statistics in medicine

authors： Tao R,Lotspeich SC,Amorim G,Shaw PA,Shepherd BE

更新日期：2021-02-10 00:00:00

abstract：:For massive survival data, we propose a subsampling algorithm to efficiently approximate the estimates of regression parameters in the additive hazards model. We establish consistency and asymptotic normality of the subsample-based estimator given the full data. The optimal subsampling probabilities are obtained via m...

journal_title：Statistics in medicine

authors： Zuo L,Zhang H,Wang H,Liu L

更新日期：2021-01-30 00:00:00

abstract：:Substantial advances in Bayesian methods for causal inference have been made in recent years. We provide an introduction to Bayesian inference for causal effects for practicing statisticians who have some familiarity with Bayesian models and would like an overview of what it can add to causal estimation in practical s...

journal_title：Statistics in medicine

authors： Oganisian A,Roy JA

更新日期：2021-01-30 00:00:00

abstract：:In this commentary, we revisit Sir Austin Bradford Hill's seminal Alfred Watson Memorial Lecture in 1962 through the eyes of two practicing biostatisticians of the current era. We summarize some eternal takeaway messages from Hill's lecture regarding observations and experiments translated through the modern lexicon o...

journal_title：Statistics in medicine

authors： Dempsey W,Mukherjee B

更新日期：2021-01-15 00:00:00

abstract：:In time-to-event analysis, the traditional summary measures have been based on the hazard function, survival function, quantile event time, restricted mean event time, and residual lifetime. Under competing risks, furthermore, typical summary measures have been the cause-specific hazard function and cumulative inciden...

journal_title：Statistics in medicine

authors： Jia Y,Jeong JH

更新日期：2021-01-06 00:00:00

abstract：:In different fields of applications including, but not limited to, behavioral, environmental, medical sciences, and econometrics, the use of panel data regression models has become increasingly popular as a general framework for making meaningful statistical inferences. However, when the ordinary least squares (OLS) m...

journal_title：Statistics in medicine

authors： Hamiye Beyaztas B,Bandyopadhyay S

更新日期：2020-12-20 00:00:00

abstract：:Residuals in normal regression are used to assess a model's goodness-of-fit (GOF) and discover directions for improving the model. However, there is a lack of residuals with a characterized reference distribution for censored regression. In this article, we propose to diagnose censored regression with normalized rando...

journal_title：Statistics in medicine

authors： Li L,Wu T,Feng C

更新日期：2020-12-13 00:00:00

abstract：:The stability-controlled quasi-experiment (SCQE) is an approach to study the effects of nonrandomized, newly adopted treatments. While covariate adjustment techniques rely on a "no unobserved confounding" assumption, SCQE imposes an assumption on the change in the average nontreatment outcome between successive cohort...

journal_title：Statistics in medicine

authors： Hazlett C,Maokola W,Wulf DA

更新日期：2020-12-10 00:00:00

abstract：:This article introduces a global hypothesis test intended for studies with multiple endpoints. Our test makes use of a priori predictions about the direction of the result of each endpoint and we weight these predictions using the sample correlation matrix. The global alternative hypothesis concerns a parameter, ...

journal_title：Statistics in medicine

authors： Montgomery RN,Mahnken JD

更新日期：2020-12-10 00:00:00

abstract：:We propose a joint modeling approach to investigating the observed and latent risk factors of mixed types of outcomes. The proposed model comprises three parts. The first part is an exploratory factor analysis model that summarizes latent factors through multiple observed variables. The second part is a proportional h...

journal_title：Statistics in medicine

authors： Pan D,Wei Y,Song X

更新日期：2020-12-09 00:00:00

abstract：:In medical research, a two-phase study is often used for the estimation of the area under the receiver operating characteristic curve (AUC) of a diagnostic test. However, such a design introduces verification bias. One of the methods to correct verification bias is inverse probability weighting (IPW). Since the probab...

journal_title：Statistics in medicine

authors： Wu Y

更新日期：2020-11-30 00:00:00

abstract：:There are inconsistencies between the formulas for the variance of standardized mean difference (SMD) in the Cochrane Handbook for Systematic Reviews and the variance reported in other sources. Instead of the variance appropriate for the SMD of a crossover experiment, the Cochrane Handbook uses the variance appropriat...

journal_title：Statistics in medicine

authors： Kitchenham B,Madeyski L

更新日期：2020-11-30 00:00:00

abstract：:The article is motivated by a nephrology study in Taiwan, which enrolled hemodialysis patients who suffered from vascular access thrombosis. After treatment, some patients were cured of thrombosis, while some may experience recurrence of either type (acute or nonacute) of vascular access thrombosis. Our major interest...

journal_title：Statistics in medicine

authors： Chen CM,Shen PS,Lin CC,Wu CC

更新日期：2020-11-30 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：:Although all clinical trials are designed and monitored using more than one endpoint, methods are needed to assure that decision criteria are chosen to reflect the clinically relevant tradeoffs that assure the trial's scientific integrity. This article presents a framework for the design and monitoring clinical trials...

journal_title：Statistics in medicine

authors： Hu J,Blatchford PJ,Goldenberg NA,Kittelson JM

更新日期：2020-11-20 00:00:00

abstract：:The relationship between association and surrogacy has been the focus of much debate in the surrogate marker literature. Recently, the individual causal association (ICA) has been introduced as a metric of surrogacy in the causal inference framework, when both the surrogate and the true endpoint are normally distribut...

journal_title：Statistics in medicine

authors： Meyvisch P,Alonso A,Van der Elst W,Molenberghs G

更新日期：2020-11-20 00:00:00

abstract：:Mesothelioma is a rare cancer caused by exposure to asbestos. Belgium has a known long history of asbestos production, resulting in one of the highest mesothelioma mortality rates worldwide. While the production of asbestos has stopped completely, the long latency period of mesothelioma, which can fluctuate between 20...

journal_title：Statistics in medicine

authors： Petrof O,Neyens T,Nuyts V,Nackaerts K,Nemery B,Faes C

更新日期：2020-11-20 00:00:00

abstract：:In genetic association studies, mixed effects models have been widely used in detecting the pleiotropy effects which occur when one gene affects multiple phenotype traits. In particular, bivariate mixed effects models are useful for describing the association of a gene with a continuous trait and a binary trait. Howev...

journal_title：Statistics in medicine

authors： Zhang Q,Yi GY

更新日期：2020-11-20 00:00:00

abstract：:In multivariate network meta-analysis (NMA), the piecemeal nature of the evidence base means that there may be treatment-outcome combinations for which no data is available. Most existing multivariate evidence synthesis models are either unable to estimate the missing treatment-outcome combinations, or can only do so ...

journal_title：Statistics in medicine

authors： Waddingham E,Matthews PM,Ashby D

更新日期：2020-10-30 00:00:00

abstract：:The EQ-5D, a widely used multiattribute utility instrument, is commonly used in health economic evaluations where the goal is to decide on which treatments to reimburse. Like other instruments, value sets of the EQ-5D are constructed using valuation studies typically valuing a subset of the health states and using pre...

journal_title：Statistics in medicine

authors： Shams S,Pullenayegum E

更新日期：2020-10-15 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：:Assessing and comparing the performance of correlated predictive scores are of current interest in precision medicine. Given the limitations of available theoretical approaches for assessing and comparing the predictive accuracy, numerical methods are highly desired which, however, have not been systematically develop...

journal_title：Statistics in medicine

authors： Zhang Y,Shao Y

更新日期：2020-09-10 00:00:00

abstract：:Conventional seamless phase 2/3 design with fixed sample size determination (SSD) has gained its popularity in oncology drug development due to attractive features such as significantly shortening the development timeline, minimizing sample size, as well as early decision making. However, this design is not immune to ...

journal_title：Statistics in medicine

authors： Teng Z,Tian Y,Liu Y,Liu G

更新日期：2020-08-15 00:00:00

abstract：:It is often of interest to use observational data to estimate the causal effect of a target exposure or treatment on an outcome. When estimating the treatment effect, it is essential to appropriately adjust for selection bias due to observed confounders using, for example, propensity score weighting. Selection bias du...

journal_title：Statistics in medicine

authors： Parast L,Griffin BA

更新日期：2020-08-15 00:00:00

abstract：:In lifetime data, like cancer studies, there may be long term survivors, which lead to heavy censoring at the end of the follow-up period. Since a standard survival model is not appropriate to handle these data, a cure model is needed. In the literature, covariate hypothesis tests for cure models are limited to parame...

journal_title：Statistics in medicine

authors： López-Cheda A,Jácome MA,Van Keilegom I,Cao R

更新日期：2020-07-30 00:00:00

abstract：:The concept of broad sense agreement (BSA) has recently been proposed for studying the relationship between a continuous measurement and an ordinal measurement. They developed a nonparametric procedure for estimating the BSA index, which is only applicable to completely observed data. In this work, we consider the pro...

journal_title：Statistics in medicine

authors： Dai T,Guo Y,Peng L,Manatunga A

更新日期：2020-06-30 00:00:00

abstract：:The analysis of multivariate time-to-event (TTE) data can become complicated due to the presence of clustering, leading to dependence between multiple event times. For a long time, (conditional) frailty models and (marginal) copula models have been used to analyze clustered TTE data. In this article, we propose a gene...

journal_title：Statistics in medicine

authors： Tran TMP,Abrams S,Braekers R

更新日期：2020-05-30 00:00:00

abstract：:Mediation analyses can help us to understand the biological mechanism in which an exposure or treatment affects an outcome. Single mediator analyses have been used in various applications, but may not be appropriate for analyzing intricate mechanisms involving multiple mediators that affect each other. Thus, in this a...

journal_title：Statistics in medicine

authors： Lai EY,Shih S,Huang YT,Wang S

更新日期：2020-05-15 00:00:00

abstract：:Detecting safety signals in clinical trial safety data is known to be challenging due to high dimensionality, rare occurrence, weak signal, and complex dependence. We propose a new hierarchical testing approach for analyzing safety data from a typical randomized clinical trial. This approach accounts for the hierarchi...

journal_title：Statistics in medicine

authors： Tan X,Chen BE,Sun J,Patel T,Ibrahim JG

更新日期：2020-05-15 00:00:00

abstract：:Diseases can be interconnected. In the recent years, there has been a surge of multidisease studies. Among them, HDN (human disease network) analysis takes a system perspective, examines the interconnections among diseases along with their individual properties, and has demonstrated great potential. Most of the existi...

journal_title：Statistics in medicine

authors： Ma C,Li Y,Shia B,Ma S

更新日期：2020-04-30 00:00:00

abstract：:The mixed effect models for repeated measures (MMRM) analysis is sometimes used as a primary analysis in longitudinal randomized clinical trials. The SE for the treatment effect in the MMRM analysis is usually estimated by assuming the orthogonality of the fixed effect and variance-covariance parameters, which is the ...

journal_title：Statistics in medicine

authors： Maruo K,Ishii R,Yamaguchi Y,Doi M,Gosho M

更新日期：2020-04-30 00:00:00

abstract：:This paper introduces a new spatial scan statistic designed to adjust cluster detection for longitudinal confounding factors indexed in space. The functional-model-adjusted statistic was developed using generalized functional linear models in which longitudinal confounding factors were considered to be functional cova...

journal_title：Statistics in medicine

authors： Ahmed MS,Genin M

更新日期：2020-04-15 00:00:00

abstract：:Cancer immunotherapy trials have two special features: a delayed treatment effect and a cure rate. Both features violate the proportional hazard model assumption and ignoring either one of the two features in an immunotherapy trial design will result in substantial loss of statistical power. To properly design immunot...

journal_title：Statistics in medicine

authors： Wei J,Wu J

更新日期：2020-03-15 00:00:00

abstract：:While genome-wide association studies (GWASs) have been widely used to uncover associations between diseases and genetic variants, standard SNP-level GWASs often lack the power to identify SNPs that individually have a moderate effect size but jointly contribute to the disease. To overcome this problem, pathway-based ...

journal_title：Statistics in medicine

authors： Yang Y,Basu S,Zhang L

更新日期：2020-03-15 00:00:00

abstract：:Composite endpoints are frequently used in clinical trials, but simple approaches, such as the time to first event, do not reflect any ordering among the endpoints. However, some endpoints, such as mortality, are worse than others. A variety of procedures have been proposed to reflect the severity of the individual en...

journal_title：Statistics in medicine

authors： Follmann D,Fay MP,Hamasaki T,Evans S

更新日期：2020-02-28 00:00:00

abstract：:Many complex diseases are known to be affected by the interactions between genetic variants and environmental exposures beyond the main genetic and environmental effects. Study of gene-environment (G×E) interactions is important for elucidating the disease etiology. Existing Bayesian methods for G×E interaction studie...

journal_title：Statistics in medicine

authors： Ren J,Zhou F,Li X,Chen Q,Zhang H,Ma S,Jiang Y,Wu C

更新日期：2020-02-28 00:00:00