Read by QxMD icon Read

Statistical Methods in Medical Research

Feiyang Niu, Jianhui Zhou, Thu H Le, Jennie Z Ma
Motivated by a genetic investigation on the progressive decline in renal function in a clinical trial study of kidney disease, we develop a practical test for evaluating the group difference in trajectories under a semi-parametric modeling framework. For the temporal patterns or trajectories of longitudinal data, B-splines are used to approximate the function non-parametrically. Such approximation asymptotically converts the problem of testing trajectory difference into the significance test of regression coefficients that can be simply estimated by generalized estimating equations...
June 2017: Statistical Methods in Medical Research
Xiaonan Xue, Mimi Y Kim, Tao Wang, Mark H Kuniholm, Howard D Strickler
Longitudinal studies of rare events such as cervical high-grade lesions or colorectal polyps that can recur often involve correlated binary data. Risk factor for these events cannot be reliably examined using conventional statistical methods. For example, logistic regression models that incorporate generalized estimating equations often fail to converge or provide inaccurate results when analyzing data of this type. Although exact methods have been reported, they are complex and computationally difficult. The current paper proposes a mathematically straightforward and easy-to-use two-step approach involving (i) an additive model to measure associations between a rare or uncommon correlated binary event and potential risk factors and (ii) a permutation test to estimate the statistical significance of these associations...
June 2017: Statistical Methods in Medical Research
Francesca Ieva, Christopher H Jackson, Linda D Sharples
In chronic diseases like heart failure (HF), the disease course and associated clinical event histories for the patient population vary widely. To improve understanding of the prognosis of patients and enable health care providers to assess and manage resources, we wish to jointly model disease progression, mortality and their relation with patient characteristics. We show how episodes of hospitalisation for disease-related events, obtained from administrative data, can be used as a surrogate for disease status...
June 2017: Statistical Methods in Medical Research
R Gutman, D B Rubin
The estimation of causal effects in nonrandomized studies should comprise two distinct phases: design, with no outcome data available; and analysis of the outcome data according to a specified protocol. Here, we review and compare point and interval estimates of common statistical procedures for estimating causal effects (i.e. matching, subclassification, weighting, and model-based adjustment) with a scalar continuous covariate and a scalar continuous outcome. We show, using an extensive simulation, that some highly advocated methods have poor operating characteristics...
June 2017: Statistical Methods in Medical Research
Amy S Nowacki, Wenle Zhao, Yuko Y Palesch
Response-adaptive randomization (RAR) offers clinical investigators benefit by modifying the treatment allocation probabilities to optimize the ethical, operational, or statistical performance of the trial. Delayed primary outcomes and their effect on RAR have been studied in the literature; however, the incorporation of surrogate outcomes has not been fully addressed. We explore the benefits and limitations of surrogate outcome utilization in RAR in the context of acute stroke clinical trials. We propose a novel surrogate-primary (S-P) replacement algorithm where a patient's surrogate outcome is used in the RAR algorithm only until their primary outcome becomes available to replace it...
June 2017: Statistical Methods in Medical Research
Jaya M Satagopan, Sara H Olson, Robert C Elston
This paper is concerned with the estimation of the logarithm of disease odds (log odds) when evaluating two risk factors, whether or not interactions are present. Statisticians define interaction as a departure from an additive model on a certain scale of measurement of the outcome. Certain interactions, known as removable interactions, may be eliminated by fitting an additive model under an invertible transformation of the outcome. This can potentially provide more precise estimates of log odds than fitting a model with interaction terms...
April 2017: Statistical Methods in Medical Research
Marcelo Azevedo Costa, Thiago de Souza Rodrigues, André Gabriel Fc da Costa, René Natowicz, Antônio Pádua Braga
This work proposes a sequential methodology for selecting variables in classification problems in which the number of predictors is much larger than the sample size. The methodology includes a Monte Carlo permutation procedure that conditionally tests the null hypothesis of no association among the outcomes and the available predictors. In order to improve computing aspects, we propose a new parametric distribution, the Truncated and Zero Inflated Gumbel Distribution. The final application is to find compact classification models with improved performance for genomic data...
April 2017: Statistical Methods in Medical Research
Wei Liu, Bo Zhang, Hui Zhang, Zhiwei Zhang
There is growing interest in assessing immune biomarkers, which are quick to measure and potentially predictive of long-term efficacy, as surrogate endpoints in randomized, placebo-controlled vaccine trials. This can be done under a principal stratification approach, with principal strata defined using a subject's potential immune responses to vaccine and placebo (the latter may be assumed to be zero). In this context, principal surrogacy refers to the extent to which vaccine efficacy varies across principal strata...
April 2017: Statistical Methods in Medical Research
Achmad Efendi, Reza Drikvandi, Geert Verbeke, Geert Molenberghs
In this paper, we develop a simple diagnostic test for the random-effects distribution in mixed models. The test is based on the gradient function, a graphical tool proposed by Verbeke and Molenberghs to check the impact of assumptions about the random-effects distribution in mixed models on inferences. Inference is conducted through the bootstrap. The proposed test is easy to implement and applicable in a general class of mixed models. The operating characteristics of the test are evaluated in a simulation study, and the method is further illustrated using two real data analyses...
April 2017: Statistical Methods in Medical Research
Guo-Liang Tian, Man-Lai Tang, Qin Wu, Yin Liu
Although the item count technique is useful in surveys with sensitive questions, privacy of those respondents who possess the sensitive characteristic of interest may not be well protected due to a defect in its original design. In this article, we propose two new survey designs (namely the Poisson item count technique and negative binomial item count technique) which replace several independent Bernoulli random variables required by the original item count technique with a single Poisson or negative binomial random variable, respectively...
April 2017: Statistical Methods in Medical Research
Arvid Sjölander, Stijn Vansteelandt
The attributable fraction is a commonly used measure that quantifies the public health impact of an exposure on an outcome. It was originally defined for binary outcomes, but an extension has recently been proposed for right-censored survival time outcomes; the so-called attributable fraction function. A maximum likelihood estimator of the attributable fraction function has been developed, which requires a model for the outcome. In this paper, we derive a doubly robust estimator of the attributable fraction function...
April 2017: Statistical Methods in Medical Research
Yong Chen, Yulun Liu, Jing Ning, Lei Nie, Hongjian Zhu, Haitao Chu
Diagnostic systematic review is a vital step in the evaluation of diagnostic technologies. In many applications, it involves pooling pairs of sensitivity and specificity of a dichotomized diagnostic test from multiple studies. We propose a composite likelihood (CL) method for bivariate meta-analysis in diagnostic systematic reviews. This method provides an alternative way to make inference on diagnostic measures such as sensitivity, specificity, likelihood ratios, and diagnostic odds ratio. Its main advantages over the standard likelihood method are the avoidance of the nonconvergence problem, which is nontrivial when the number of studies is relatively small, the computational simplicity, and some robustness to model misspecifications...
April 2017: Statistical Methods in Medical Research
Tanzy Mt Love, Sally W Thurston, Philip W Davidson
The Seychelles Child Development Study is a research project with the objective of examining associations between prenatal exposure to low doses of methylmercury from maternal fish consumption and children's developmental outcomes. Whether methylmercury has neurotoxic effects at low doses remains unclear and recommendations for pregnant women and children to reduce fish intake may prevent a substantial number of people from receiving sufficient nutrients that are abundant in fish. The primary findings of the Seychelles Child Development Study are inconsistent with adverse associations between methylmercury from fish consumption and neurodevelopmental outcomes...
April 2017: Statistical Methods in Medical Research
Dipankar Bandyopadhyay, Diana M Galvis, Victor H Lachos
Often in biomedical research, we deal with continuous (clustered) proportion responses ranging between zero and one quantifying the disease status of the cluster units. Interestingly, the study population might also consist of relatively disease-free as well as highly diseased subjects, contributing to proportion values in the interval [0, 1]. Regression on a variety of parametric densities with support lying in (0, 1), such as beta regression, can assess important covariate effects. However, they are deemed inappropriate due to the presence of zeros and/or ones...
April 2017: Statistical Methods in Medical Research
Zhong Guan, Jing Qin
The receiver operating characteristic curve is commonly used for assessing diagnostic test accuracy and for discriminatory ability of a medical diagnostic test in distinguishing between diseases and non-diseased individuals. With the advance of technology, many genetic variables and biomarker variables are easily collected. The most challenging problem is how to combine clinical, genetic, and biomarker variables together to predict disease status. If one is interested in predicting t-year survival, however, the status of "case" (death) and "control" (survival) at the given t-year is unknown for those individuals who were censored before t-year...
April 2017: Statistical Methods in Medical Research
Michael J Lopez, Roee Gutman
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 with an ordinal exposure are the average treatment effects between each pair of exposure levels. Using these estimands, it is possible to determine an optimal exposure level. Traditional methods, including dichotomization of the exposure or a series of binary propensity score comparisons across exposure pairs, are generally inadequate for identification of optimal levels...
April 2017: Statistical Methods in Medical Research
Spencer Lourens, Ying Zhang, Jeffrey D Long, Jane S Paulsen
Executive dysfunction is a deficiency in skills of planning and problem solving that characterizes many neuropsychiatric disorders. The Towers Task is a commonly used measure of planning and problem solving for assessing executive function. Towers Task data are usually zero-inflated and right-censored, and ignoring these features can result in biased inference for the disease characterization of executive dysfunction. In this manuscript, a mixed-effects model for longitudinal censored semicontinuous data is developed for analyzing longitudinal Towers Task data from the PREDICT-HD study...
April 2017: Statistical Methods in Medical Research
Nicholas R Latimer, K R Abrams, P C Lambert, M J Crowther, A J Wailoo, J P Morden, R L Akehurst, M J Campbell
Estimates of the overall survival benefit of new cancer treatments are often confounded by treatment switching in randomised controlled trials (RCTs) - whereby patients randomised to the control group are permitted to switch onto the experimental treatment upon disease progression. In health technology assessment, estimates of the unconfounded overall survival benefit associated with the new treatment are needed. Several switching adjustment methods have been advocated in the literature, some of which have been used in health technology assessment...
April 2017: Statistical Methods in Medical Research
Ilaria Ardoino, Monica Lanzoni, Giuseppe Marano, Patrizia Boracchi, Elisabetta Sagrini, Alice Gianstefani, Fabio Piscaglia, Elia M Biganzoli
The interpretation of regression models results can often benefit from the generation of nomograms, 'user friendly' graphical devices especially useful for assisting the decision-making processes. However, in the case of multinomial regression models, whenever categorical responses with more than two classes are involved, nomograms cannot be drawn in the conventional way. Such a difficulty in managing and interpreting the outcome could often result in a limitation of the use of multinomial regression in decision-making support...
April 2017: Statistical Methods in Medical Research
Fetch more papers »
Fetching more papers... Fetching...
Read by QxMD. Sign in or create an account to discover new knowledge that matter to you.
Remove bar
Read by QxMD icon Read

Search Tips

Use Boolean operators: AND/OR

diabetic AND foot
diabetes OR diabetic

Exclude a word using the 'minus' sign

Virchow -triad

Use Parentheses

water AND (cup OR glass)

Add an asterisk (*) at end of a word to include word stems

Neuro* will search for Neurology, Neuroscientist, Neurological, and so on

Use quotes to search for an exact phrase

"primary prevention of cancer"
(heart or cardiac or cardio*) AND arrest -"American Heart Association"