Evaluation of Approaches to Analyzing Continuous Correlated Eye Data When Sample Size Is Small. 2018

Jing Huang, and Jiayan Huang, and Yong Chen, and Gui-Shuang Ying
a Division of Biostatistics , Center for Clinical Epidemiology and Biostatistics.

To evaluate the performance of commonly used statistical methods for analyzing continuous correlated eye data when sample size is small. We simulated correlated continuous data from two designs: (1) two eyes of a subject in two comparison groups; (2) two eyes of a subject in the same comparison group, under various sample size (5-50), inter-eye correlation (0-0.75) and effect size (0-0.8). Simulated data were analyzed using paired t-test, two sample t-test, Wald test and score test using the generalized estimating equations (GEE) and F-test using linear mixed effects model (LMM). We compared type I error rates and statistical powers, and demonstrated analysis approaches through analyzing two real datasets. In design 1, paired t-test and LMM perform better than GEE, with nominal type 1 error rate and higher statistical power. In design 2, no test performs uniformly well: two sample t-test (average of two eyes or a random eye) achieves better control of type I error but yields lower statistical power. In both designs, the GEE Wald test inflates type I error rate and GEE score test has lower power. When sample size is small, some commonly used statistical methods do not perform well. Paired t-test and LMM perform best when two eyes of a subject are in two different comparison groups, and t-test using the average of two eyes performs best when the two eyes are in the same comparison group. When selecting the appropriate analysis approach the study design should be considered.

UI MeSH Term Description Entries
D003198 Computer Simulation Computer-based representation of physical systems and phenomena such as chemical processes. Computational Modeling,Computational Modelling,Computer Models,In silico Modeling,In silico Models,In silico Simulation,Models, Computer,Computerized Models,Computer Model,Computer Simulations,Computerized Model,In silico Model,Model, Computer,Model, Computerized,Model, In silico,Modeling, Computational,Modeling, In silico,Modelling, Computational,Simulation, Computer,Simulation, In silico,Simulations, Computer
D003627 Data Interpretation, Statistical Application of statistical procedures to analyze specific observed or assumed facts from a particular study. Data Analysis, Statistical,Data Interpretations, Statistical,Interpretation, Statistical Data,Statistical Data Analysis,Statistical Data Interpretation,Analyses, Statistical Data,Analysis, Statistical Data,Data Analyses, Statistical,Interpretations, Statistical Data,Statistical Data Analyses,Statistical Data Interpretations
D005128 Eye Diseases Diseases affecting the eye. Eye Disorders,Eye Disease,Eye Disorder
D006801 Humans Members of the species Homo sapiens. Homo sapiens,Man (Taxonomy),Human,Man, Modern,Modern Man
D015233 Models, Statistical Statistical formulations or analyses which, when applied to data and found to fit the data, are then used to verify the assumptions and parameters used in the analysis. Examples of statistical models are the linear model, binomial model, polynomial model, two-parameter model, etc. Probabilistic Models,Statistical Models,Two-Parameter Models,Model, Statistical,Models, Binomial,Models, Polynomial,Statistical Model,Binomial Model,Binomial Models,Model, Binomial,Model, Polynomial,Model, Probabilistic,Model, Two-Parameter,Models, Probabilistic,Models, Two-Parameter,Polynomial Model,Polynomial Models,Probabilistic Model,Two Parameter Models,Two-Parameter Model
D018401 Sample Size The number of units (persons, animals, patients, specified circumstances, etc.) in a population to be studied. The sample size should be big enough to have a high likelihood of detecting a true difference between two groups. (From Wassertheil-Smoller, Biostatistics and Epidemiology, 1990, p95) Sample Sizes,Size, Sample,Sizes, Sample
D019468 Disease Management A broad approach to appropriate coordination of the entire disease treatment process that often involves shifting away from more expensive inpatient and acute care to areas such as preventive medicine, patient counseling and education, and outpatient care. This concept includes implications of appropriate versus inappropriate therapy on the overall cost and clinical outcome of a particular disease. (From Hosp Pharm 1995 Jul;30(7):596) Disease Managements,Management, Disease,Managements, Disease

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