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International Journal of Biostatistics

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https://www.readbyqxmd.com/read/28453440/median-analysis-of-repeated-measures-associated-with-recurrent-events-in-presence-of-terminal-event
#1
Rajeshwari Sundaram, Ling Ma, Subhashis Ghoshal
Recurrent events are often encountered in medical follow up studies. In addition, such recurrences have other quantities associated with them that are of considerable interest, for instance medical costs of the repeated hospitalizations and tumor size in cancer recurrences. These processes can be viewed as point processes, i.e. processes with arbitrary positive jump at each recurrence. An analysis of the mean function for such point processes have been proposed in the literature. However, such point processes are often skewed, leading to median as a more appropriate measure than the mean...
April 28, 2017: International Journal of Biostatistics
https://www.readbyqxmd.com/read/28441139/empirical-likelihood-in-nonignorable-covariate-missing-data-problems
#2
Yanmei Xie, Biao Zhang
Missing covariate data occurs often in regression analysis, which frequently arises in the health and social sciences as well as in survey sampling. We study methods for the analysis of a nonignorable covariate-missing data problem in an assumed conditional mean function when some covariates are completely observed but other covariates are missing for some subjects. We adopt the semiparametric perspective of Bartlett et al. (Improving upon the efficiency of complete case analysis when covariates are MNAR. Biostatistics 2014;15:719-30) on regression analyses with nonignorable missing covariates, in which they have introduced the use of two working models, the working probability model of missingness and the working conditional score model...
April 20, 2017: International Journal of Biostatistics
https://www.readbyqxmd.com/read/28361783/a-theorem-at-the-core-of-colliding-bias
#3
Doron J Shahar, Eyal Shahar
Conditioning on a shared outcome of two variables can alter the association between these variables, possibly adding a bias component when estimating effects. In particular, if two causes are marginally independent, they might be dependent in strata of their common effect. Explanations of the phenomenon, however, do not explicitly state when dependence will be created and have been largely informal. We prove that two, marginally independent, causes will be dependent in a particular stratum of their shared outcome if and only if they modify each other's effects, on a probability ratio scale, on that value of the outcome variable...
March 31, 2017: International Journal of Biostatistics
https://www.readbyqxmd.com/read/28343164/a-quantitative-concordance-measure-for-comparing-and-combining-treatment-selection-markers
#4
Zhiwei Zhang, Shujie Ma, Lei Nie, Guoxing Soon
Motivated by an HIV example, we consider how to compare and combine treatment selection markers, which are essential to the notion of precision medicine. The current literature on precision medicine is focused on evaluating and optimizing treatment regimes, which can be obtained by dichotomizing treatment selection markers. In practice, treatment decisions are based not only on efficacy but also on safety, cost and individual preference, making it difficult to choose a single cutoff value for all patients in all settings...
March 25, 2017: International Journal of Biostatistics
https://www.readbyqxmd.com/read/28328527/on-the-conditional-power-in-survival-time-analysis-considering-cure-fractions
#5
Andreas Kuehnapfel, Fabian Schwarzenberger, Markus Scholz
Conditional power of survival endpoints at interim analyses can support decisions on continuing a trial or stopping it for futility. When a cure fraction becomes apparent, conditional power cannot be calculated accurately using simple survival models, e.g. the exponential model. Non-mixture models consider such cure fractions. In this paper, we derive conditional power functions for non-mixture models, namely the non-mixture exponential, the non-mixture Weibull, and the non-mixture Gamma models. Formulae were implemented in the R package CP...
March 17, 2017: International Journal of Biostatistics
https://www.readbyqxmd.com/read/28207413/combinatorial-mixtures-of-multiparameter-distributions-an-application-to-bivariate-data
#6
Valeria Edefonti, Giovanni Parmigiani
Abstract: We introduce combinatorial mixtures - a flexible class of models for inference on mixture distributions whose components have multidimensional parameters. The key idea is to allow each element of the component-specific parameter vectors to be shared by a subset of other components. This approach allows for mixtures that range from very flexible to very parsimonious and unifies inference on component-specific parameters with inference on the number of components. We develop Bayesian inference and computational approaches for this class of distributions, and illustrate them in an application...
February 16, 2017: International Journal of Biostatistics
https://www.readbyqxmd.com/read/28195544/on-stratified-adjusted-tests-by-binomial-trials
#7
Asanao Shimokawa, Etsuo Miyaoka
To estimate or test the treatment effect in randomized clinical trials, it is important to adjust for the potential influence of covariates that are likely to affect the association between the treatment or control group and the response. If these covariates are known at the start of the trial, random assignment of the treatment within each stratum would be considered. On the other hand, if these covariates are not clear at the start of the trial, or if it is difficult to allocate the treatment within each stratum, completely randomized assignment of the treatment would be performed...
February 14, 2017: International Journal of Biostatistics
https://www.readbyqxmd.com/read/28160542/testing-equality-of-treatments-under-an-incomplete-block-crossover-design-with-ordinal-responses
#8
Kung-Jong Lui
The generalized odds ratio (GOR) for paired sample is considered to measure the relative treatment effect on patient responses in ordinal data. Under a three-treatment two-period incomplete block crossover design, both asymptotic and exact procedures are developed for testing equality between treatments with ordinal responses. Monte Carlo simulation is employed to evaluate and compare the finite-sample performance of these test procedures. A discussion on advantages and disadvantages of the proposed test procedures based on the GOR versus those based on Wald's tests under the normal random effects proportional odds model is provided...
February 3, 2017: International Journal of Biostatistics
https://www.readbyqxmd.com/read/28157692/bayesian-variable-selection-methods-for-matched-case-control-studies
#9
Josephine Asafu-Adjei, G Tadesse Mahlet, Brent Coull, Raji Balasubramanian, Michael Lev, Lee Schwamm, Rebecca Betensky
Matched case-control designs are currently used in many biomedical applications. To ensure high efficiency and statistical power in identifying features that best discriminate cases from controls, it is important to account for the use of matched designs. However, in the setting of high dimensional data, few variable selection methods account for matching. Bayesian approaches to variable selection have several advantages, including the fact that such approaches visit a wider range of model subsets. In this paper, we propose a variable selection method to account for case-control matching in a Bayesian context and apply it using simulation studies, a matched brain imaging study conducted at Massachusetts General Hospital, and a matched cardiovascular biomarker study conducted by the High Risk Plaque Initiative...
January 31, 2017: International Journal of Biostatistics
https://www.readbyqxmd.com/read/27930367/multiple-comparisons-using-composite-likelihood-in-clustered-data
#10
Mahdis Azadbakhsh, Xin Gao, Hanna Jankowski
We study the problem of multiple hypothesis testing for correlated clustered data. As the existing multiple comparison procedures based on maximum likelihood estimation could be computationally intensive, we propose to construct multiple comparison procedures based on composite likelihood method. The new test statistics account for the correlation structure within the clusters and are computationally convenient to compute. Simulation studies show that the composite likelihood based procedures maintain good control of the familywise type I error rate in the presence of intra-cluster correlation, whereas ignoring the correlation leads to erratic performance...
November 1, 2016: International Journal of Biostatistics
https://www.readbyqxmd.com/read/27889706/using-relative-statistics-and-approximate-disease-prevalence-to-compare-screening-tests
#11
Frank Samuelson, Craig Abbey
Schatzkin et al. and other authors demonstrated that the ratios of some conditional statistics such as the true positive fraction are equal to the ratios of unconditional statistics, such as disease detection rates, and therefore we can calculate these ratios between two screening tests on the same population even if negative test patients are not followed with a reference procedure and the true and false negative rates are unknown. We demonstrate that this same property applies to an expected utility metric...
November 1, 2016: International Journal of Biostatistics
https://www.readbyqxmd.com/read/27889705/effect-estimation-in-point-exposure-studies-with-binary-outcomes-and-high-dimensional-covariate-data-a-comparison-of-targeted-maximum-likelihood-estimation-and-inverse-probability-of-treatment-weighting
#12
Menglan Pang, Tibor Schuster, Kristian B Filion, Mireille E Schnitzer, Maria Eberg, Robert W Platt
Inverse probability of treatment weighting (IPW) and targeted maximum likelihood estimation (TMLE) are relatively new methods proposed for estimating marginal causal effects. TMLE is doubly robust, yielding consistent estimators even under misspecification of either the treatment or the outcome model. While IPW methods are known to be sensitive to near violations of the practical positivity assumption (e. g., in the case of data sparsity), the consequences of this violation in the TMLE framework for binary outcomes have been less widely investigated...
November 1, 2016: International Journal of Biostatistics
https://www.readbyqxmd.com/read/27838682/sample-size-for-assessing-agreement-between-two-methods-of-measurement-by-bland-altman-method
#13
Meng-Jie Lu, Wei-Hua Zhong, Yu-Xiu Liu, Hua-Zhang Miao, Yong-Chang Li, Mu-Huo Ji
The Bland-Altman method has been widely used for assessing agreement between two methods of measurement. However, it remains unsolved about sample size estimation. We propose a new method of sample size estimation for Bland-Altman agreement assessment. According to the Bland-Altman method, the conclusion on agreement is made based on the width of the confidence interval for LOAs (limits of agreement) in comparison to predefined clinical agreement limit. Under the theory of statistical inference, the formulae of sample size estimation are derived, which depended on the pre-determined level of α, β, the mean and the standard deviation of differences between two measurements, and the predefined limits...
November 1, 2016: International Journal of Biostatistics
https://www.readbyqxmd.com/read/27831920/joint-model-for-mortality-and-hospitalization
#14
Yuqi Chen, Wensheng Guo, Peter Kotanko, Len Usvyat, Yuedong Wang
Modeling hospitalization is complicated because the follow-up time can be censored due to death. In this paper, we propose a shared frailty joint model for survival time and hospitalization. A random effect semi-parametric proportional hazard model is assumed for the survival time and conditional on the follow-up time, hospital admissions or total length of stay is modeled by a generalized linear model with a nonparametric offset function of the follow-up time. We assume that the hospitalization and the survival time are correlated through a latent subject-specific random frailty...
November 1, 2016: International Journal of Biostatistics
https://www.readbyqxmd.com/read/27497870/semiparametric-regression-estimation-for-recurrent-event-data-with-errors-in-covariates-under-informative-censoring
#15
Hsiang Yu, Yu-Jen Cheng, Ching-Yun Wang
Recurrent event data arise frequently in many longitudinal follow-up studies. Hence, evaluating covariate effects on the rates of occurrence of such events is commonly of interest. Examples include repeated hospitalizations, recurrent infections of HIV, and tumor recurrences. In this article, we consider semiparametric regression methods for the occurrence rate function of recurrent events when the covariates may be measured with errors. In contrast to the existing works, in our case the conventional assumption of independent censoring is violated since the recurrent event process is interrupted by some correlated events, which is called informative drop-out...
November 1, 2016: International Journal of Biostatistics
https://www.readbyqxmd.com/read/27232635/multi-locus-test-and-correction-for-confounding-effects-in-genome-wide-association-studies
#16
Donglai Chen, Chuanhai Liu, Jun Xie
Genome-wide association studies (GWAS) examine a large number of genetic variants, e. g., single nucleotide polymorphisms (SNP), and associate them with a disease of interest. Traditional statistical methods for GWASs can produce spurious associations, due to limited information from individual SNPs and confounding effects. This paper develops two statistical methods to enhance data analysis of GWASs. The first is a multiple-SNP association test, which is a weighted chi-square test derived for big contingency tables...
November 1, 2016: International Journal of Biostatistics
https://www.readbyqxmd.com/read/27092657/mendelian-randomization-using-public-data-from-genetic-consortia
#17
John R Thompson, Cosetta Minelli, Fabiola Del Greco M
Mendelian randomization (MR) is a technique that seeks to establish causation between an exposure and an outcome using observational data. It is an instrumental variable analysis in which genetic variants are used as the instruments. Many consortia have meta-analysed genome-wide associations between variants and specific traits and made their results publicly available. Using such data, it is possible to derive genetic risk scores for one trait and to deduce the association of that same risk score with a second trait...
November 1, 2016: International Journal of Biostatistics
https://www.readbyqxmd.com/read/26882561/tree-based-method-for-aggregate-survival-data-modeling
#18
Asanao Shimokawa, Yoshitaka Narita, Soichiro Shibui, Etsuo Miyaoka
In many scenarios, a patient in medical research is treated as a statistical unit. However, in some scenarios, we are interested in treating aggregate data as a statistical unit. In such situations, each set of aggregated data is considered to be a concept in a symbolic representation, and each concept has a hyperrectangle or multiple points in the variable space. To construct a tree-structured model from these aggregate survival data, we propose a new approach, where a datum can be included in several terminal nodes in a tree...
November 1, 2016: International Journal of Biostatistics
https://www.readbyqxmd.com/read/26812804/testing-equality-in-ordinal-data-with-repeated-measurements-a-model-free-approach
#19
Kung-Jong Lui
In randomized clinical trials, we often encounter ordinal categorical responses with repeated measurements. We propose a model-free approach with using the generalized odds ratio (GOR) to measure the relative treatment effect. We develop procedures for testing equality of treatment effects and derive interval estimators for the GOR. We further develop a simple procedure for testing the treatment-by-period interaction. To illustrate the use of test procedures and interval estimators developed here, we consider two real-life data sets, one studying the gender effect on pain scores on an ordinal scale after hip joint resurfacing surgeries, and the other investigating the effect of an active hypnotic drug in insomnia patients on ordinal categories of time to falling asleep...
November 1, 2016: International Journal of Biostatistics
https://www.readbyqxmd.com/read/26656800/adaptive-design-for-staggered-start-clinical-trial
#20
Ao Yuan, Qizhai Li, Ming Xiong, Ming T Tan
In phase II and/or III clinical trial study, there are several competing treatments, the goal is to assess the performances of the treatments at the end of the study, the trial design aims to minimize risks to the patients in the trial, according to some given allocation optimality criterion. Recently, a new type of clinical trial, the staggered-start trial has been proposed in some studies, in which different treatments enter the same trial at different times. Some basic questions for this trial are whether optimality can still be kept? under what conditions? and if so how to allocate the the coming patients to treatments to achieve such optimality? Here we propose and study a class of adaptive designs of staggered-start clinical trials, in which for given optimality criterion object, we show that as long as the initial sizes at the beginning of the successive trials are not too large relative to the total sample size, the proposed design can still achieve optimality criterion asymptotically for the allocation proportions as the ordinary trials; if these initial sample sizes have about the same magnitude as the total sample size, full optimality cannot be achieved...
November 1, 2016: International Journal of Biostatistics
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