Cluster without fluster: The effect of correlated outcomes on inference in randomized clinical trials.

abstract：:Clinical trials, especially the randomized clinical trial, have been and will remain the gold standard for the evaluation of new interventions, including pharmaceuticals, biologics, medical devices, procedures, or behavioral modifications. Despite more than five decades of experience, there are still challenges in the...

journal_title：Statistics in medicine

authors： DeMets DL

更新日期：2012-11-10 00:00:00

abstract：:Prevention studies, as distinguished from studies investigating treatments for established disease, present some distinct challenges. Perhaps the most extensive experience with preventive agents is in the area of infectious diseases; vaccines have been extremely effective in preventing many such diseases. Vaccines hav...

journal_title：Statistics in medicine

authors： Ellenberg SS

更新日期：2004-01-30 00:00:00

abstract：:In England and Wales, a large-scale multiple statistical surveillance system for infectious disease outbreaks has been in operation for nearly two decades. This system uses a robust quasi-Poisson regression algorithm to identify abberrances in weekly counts of isolates reported to the Health Protection Agency. In this...

journal_title：Statistics in medicine

authors： Noufaily A,Enki DG,Farrington P,Garthwaite P,Andrews N,Charlett A

更新日期：2013-03-30 00:00:00

abstract：:Between-community variance or community-by-time variance is one of the key factors driving the cost of conducting group randomized trials, which are often very expensive. We investigated empirically whether between-community variance could be reduced by controlling individual- and/or community-level covariates and ide...

journal_title：Statistics in medicine

authors： Feng Z,Diehr P,Yasui Y,Evans B,Beresford S,Koepsell TD

更新日期：1999-03-15 00:00:00

abstract：:Conditional power (CP) is the probability that the final study result will be statistically significant, given the data observed thus far and a specific assumption about the pattern of the data to be observed in the remainder of the study, such as assuming the original design effect, or the effect estimated from the c...

journal_title：Statistics in medicine

authors： Lachin JM

更新日期：2005-09-30 00:00:00

abstract：:Population heterogeneity is frequently observed among patients' treatment responses in clinical trials because of various factors such as clinical background, environmental, and genetic factors. Different subpopulations defined by those baseline factors can lead to differences in the benefit or safety profile of a the...

journal_title：Statistics in medicine

authors： Chiu YD,Koenig F,Posch M,Jaki T

更新日期：2018-12-20 00:00:00

abstract：:Cancer patients, chronic kidney disease patients, and subjects infected with HIV are routinely monitored over time using biomarkers that represent key health status indicators. Furthermore, biomarkers are frequently used to guide initiation of new treatments or to inform changes in intervention strategies. Since key m...

journal_title：Statistics in medicine

authors： Saha-Chaudhuri P,Heagerty PJ

更新日期：2018-08-15 00:00:00

abstract：:Public health interventions are often designed to target communities defined either geographically (e.g. cities, counties) or socially (e.g. schools or workplaces). The group randomized trial (GRT) is regarded as the gold standard for evaluating these interventions. However, community leaders may object to randomizati...

journal_title：Statistics in medicine

authors： Pennell ML,Hade EM,Murray DM,Rhoda DA

更新日期：2011-07-10 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：:A frequent objective in medical research is the investigation of differences in patient survival between several experimental treatments and one standard treatment. In order to assess these differences statistically, we have to apply adjustments for multiple comparisons to prevent an increased number of false-positive...

journal_title：Statistics in medicine

authors： Herberich E,Hothorn T

更新日期：2012-01-13 00:00:00

abstract：:The back-calculation method has been used to estimate the number of HIV infections from AIDS incidence data in a particular population. We present an extension of back calculation that provides estimates of the numbers of HIV infectives in different stages of infection. We model the staging process with a time-depende...

journal_title：Statistics in medicine

authors： Longini IM Jr,Byers RH,Hessol NA,Tan WY

更新日期：1992-04-01 00:00:00

abstract：:In medical and health studies, heterogeneities in clustered count data have been traditionally modeled by positive random effects in Poisson mixed models; however, excessive zeros often occur in clustered medical and health count data. In this paper, we consider a three-level random effects zero-inflated Poisson model...

journal_title：Statistics in medicine

authors： Ma R,Hasan MT,Sneddon G

更新日期：2009-08-15 00:00:00

abstract：:Standard approaches to analysis of randomized controlled trials (RCTs) using Markov models make it difficult to generalize treatment effects to new patient groups and synthesize evidence across trials. This paper demonstrates how pair-wise and mixed treatment comparison meta-analysis can be applied to event history da...

journal_title：Statistics in medicine

authors： Price MJ,Welton NJ,Ades AE

更新日期：2011-01-30 00:00:00

abstract：:Correlation is always a concern in the analysis of clustered data. One area of interest is to develop a general correlation modelling approach for high dimensional data with unbalanced hierarchical and heterogeneous data structures, e.g. multilevel data. Commonly used correlation structures might have limitation for s...

journal_title：Statistics in medicine

authors： Chao EC

更新日期：2006-07-30 00:00:00

abstract：:Juvenile dermatomyositis (JDM) is a rare autoimmune disease that may lead to serious complications, even to death. We develop a 2-state Markov regression model in a Bayesian framework to characterise disease progression in JDM over time and gain a better understanding of the factors influencing disease risk. The trans...

journal_title：Statistics in medicine

authors： De Iorio M,Gallot N,Valcarcel B,Wedderburn L

更新日期：2018-05-10 00:00:00

abstract：:This paper presents tables analogous to T-tables for use in the 2 x 2 multinomial trial, where the continuity corrected Z-statistic is used to make exact unconditional inference. This is the first solution of a discrete exact unconditional inference problem involving a multivariate nuisance parameter for which no anci...

journal_title：Statistics in medicine

authors： Shuster JJ

更新日期：1992-05-01 00:00:00

abstract：:This paper considers the problem of selecting a set of regressors when the response variable is distributed according to a specified parametric model and observations are censored. Under a Bayesian perspective, the most widely used tools are Bayes factors (BFs), which are undefined when improper priors are used. In or...

journal_title：Statistics in medicine

authors： Cabras S,Castellanos ME,Perra S

更新日期：2014-11-20 00:00:00

abstract：:We examine the properties of several tests for goodness-of-fit for multinomial logistic regression. One test is based on a strategy of sorting the observations according to the complement of the estimated probability for the reference outcome category and then grouping the subjects into g equal-sized groups. A g x c c...

journal_title：Statistics in medicine

authors： Fagerland MW,Hosmer DW,Bofin AM

更新日期：2008-09-20 00:00:00

abstract：:Small but important therapeutic effects of new treatments can be most efficiently detected through the study of large randomized prospective series of patients. Such large scale clinical trials are nowadays commonplace. The alternative is years of polemic and debate surrounding several trials each too small to detect ...

journal_title：Statistics in medicine

authors： McPherson K

更新日期：1982-01-01 00:00:00

abstract：:Arming the immune system against cancer has emerged as a powerful tool in oncology during recent years. Instead of poisoning a tumor or destroying it with radiation, therapeutic cancer vaccine, a type of cancer immunotherapy, unleashes the immune system to combat cancer. This indirect mechanism-of-action of vaccines p...

journal_title：Statistics in medicine

authors： Xu Z,Zhen B,Park Y,Zhu B

更新日期：2017-02-20 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：: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：:When the event time of interest depends on the censoring time, conventional two-sample test methods, such as the log-rank and Wilcoxon tests, can produce an invalid test result. We extend our previous work on estimation using auxiliary variables to adjust for dependent censoring via multiple imputation, to the compari...

journal_title：Statistics in medicine

authors： Hsu CH,Taylor JM

更新日期：2009-02-01 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 field study sought to measure the effects of dental delivery and school-based, dental health education on use of dental health care by children in grades K-6. We attempted to control for two potential confounding factors by an approximate randomization of children into treatment groups with stratification on grad...

journal_title：Statistics in medicine

authors： Oler J,Bentley JM

更新日期：1983-01-01 00:00:00

abstract：:Clinical studies that involve the recording of two or more distinct and well-defined events on each subject give rise to multiple event data. Treatment comparisons are usually reported in univariate analyses of time to first event or number of events observed. However, this approach may not uncover the 'full story' of...

journal_title：Statistics in medicine

authors： Barai U,Teoh N

更新日期：1997-04-30 00:00:00

abstract：:This paper assesses the coverage probability of commonly used confidence intervals for the standardized mortality ratio (SMR) when death certificates are missing. It also proposes alternative confidence interval approaches with coverage probabilities close to .95. In epidemiology, the SMR is an important measure of ri...

journal_title：Statistics in medicine

authors： Timkova J,Kotik L,Tomasek L

更新日期：2017-11-30 00:00:00

abstract：:Health authorities are often alerted to suspected cancer clusters near the vicinity of potential point sources by members of the public. A surveillance system, where administrative regions around the potential point sources are regularly monitored for high disease rates, would allow for responses which are easier to o...

journal_title：Statistics in medicine

authors： Le ND,Petkau AJ,Rosychuk R

更新日期：1996-04-15 00:00:00

abstract：:The use of the continual reassessment method (CRM) and other model-based approaches to design Phase I clinical trials has increased owing to the ability of the CRM to identify the maximum tolerated dose better than the 3 + 3 method. However, the CRM can be sensitive to the variance selected for the prior distribution ...

journal_title：Statistics in medicine

authors： Zhang J,Braun TM,Taylor JM

更新日期：2013-06-15 00:00:00

abstract：:It is unclear to what extent the incremental predictive performance of a novel biomarker is impacted by the method used to control for standard predictors. We investigated whether adding a biomarker to a model with a published risk score overestimates its incremental performance as compared to adding it to a multivari...

journal_title：Statistics in medicine

authors： Xanthakis V,Sullivan LM,Vasan RS,Benjamin EJ,Massaro JM,D'Agostino RB Sr,Pencina MJ

更新日期：2014-07-10 00:00:00