Confidence intervals for assessing equivalence of two treatments with combined unilateral and bilateral data.

Responses from the paired organs are generally highly correlated in bilateral studies, statistical procedures ignoring the correlation could lead to incorrect results. Note the intraclass correlation in the study of combined unilateral and bilateral outcomes; 11 confidence intervals (CIs) including 7 asymptotic CIs and 4 Bootstrap-resampling CIs for assessing the equivalence of 2 treatments are derived under Rosner's correlated binary data model. Performance is evaluated with respect to the empirical coverage probability (ECP), the empirical coverage width (ECW) and the ratio of the mesial non-coverage probability to the non-coverage probability (RMNCP) via simulation studies. Simulation results show that (i) all CIs except for the Wald CI and the bias-corrected Bootstrap percentile CI generally produce satisfactory ECPs and hence are recommended; (ii) all CIs except for the bias-corrected Bootstrap percentile CI provide preferred RMNCPs and are more symmetrical; (iii) as the measurement of the dependence increases, the ECWs of all CIs except for the score CI and the profile likelihood CI show increasing patterns that look like linear, while there is no obvious pattern on the ECPs of all CIs except for the profile likelihood CI. A data set from an otolaryngologic study is used to illustrate the proposed methods.

Confidence intervals of the difference between areas under two ROC curves in matched-pair experiments.

In a matched-pair study, when outcomes of two diagnostic tests are ordinal/continuous, the difference between two correlated areas under ROC curves (AUCs) is usually used to compare the overall discriminatory ability of two diagnostic tests. This article considers confidence interval (CI) construction problems of difference between two correlated AUCs in a matched-pair experiment, and proposes 13 hybrid CIs based on variance estimates recovery with the maximum likelihood estimation, Delong's statistic, Wilson score statistic (WS) and WS with continuity correction, the modified Wald statistic (MW) and MW with continuity correction and Agresti-Coull statistic, and three Bootstrap-resampling-based CIs. For comparison, we present traditional parametric and nonparametric CIs. Simulation studies are conducted to assess the performance of the proposed CIs in terms of empirical coverage probabilities, empirical interval widths, and ratios of the mesial noncoverage probabilities to the noncoverage probabilities. Two examples from clinical studies are illustrated by the proposed methodologies. Empirical results evidence that the hybrid Agresti-Coull CI with the empirical estimation (EAC) behaved most satisfactorily because its coverage probability was quite close to the prespecified confidence level with short interval width. Hence, we recommend the usage of the EAC CI in applications.

Simultaneous confidence interval for assessing non-inferiority with assay sensitivity in a three-arm trial with binary endpoints.

A three-arm trial including an experimental treatment, an active reference treatment and a placebo is often used to assess the non-inferiority (NI) with assay sensitivity of an experimental treatment. Various hypothesis-test-based approaches via a fraction or pre-specified margin have been proposed to assess the NI with assay sensitivity in a three-arm trial. There is little work done on confidence interval in a three-arm trial. This paper develops a hybrid approach to construct simultaneous confidence interval for assessing NI and assay sensitivity in a three-arm trial. For comparison, we present normal-approximation-based and bootstrap-resampling-based simultaneous confidence intervals. Simulation studies evidence that the hybrid approach with the Wilson score statistic performs better than other approaches in terms of empirical coverage probability and mesial-non-coverage probability. An example is used to illustrate the proposed approaches.

Construction of confidence intervals for the risk differences in stratified design with correlated bilateral data.

A stratified study is often designed for adjusting a confounding effect or effect of different centers/groups in two treatments or diagnostic tests, and the risk difference is one of the most frequently used indices in comparing efficiency between two treatments or diagnostic tests. This article presented five simultaneous confidence intervals (CIs) for risk differences in stratified bilateral designs accounting for the intraclass correlation and developed seven CIs for the common risk difference under the homogeneity assumption. The performance of the CIs is evaluated with respect to the empirical coverage probabilities, empirical coverage widths and ratios of mesial noncoverage probability and the noncoverage probability under various scenarios. Empirical results show that Wald simultaneous CI, Haldane simultaneous CI, Score simultaneous CI based on Bonferroni method and simultaneous CI based on bootstrap-resampling method perform satisfactorily and hence be recommended for applications, the CI based on the weighted-least-square (WLS) estimator, the CIs based on Mantel-Haenszel estimator, the CI based on Cochran statistic and the CI based on Score statistic for the common risk difference behave well even under small sample sizes. A real data example is used to demonstrate the proposed methodologies.

Bootstrap resampling approach to disaggregate analysis of road crashes in Hong Kong.

Road safety affects health and development worldwide; thus, it is essential to examine the factors that influence crashes and injuries. As the relationships between crashes, crash severity, and possible risk factors can vary depending on the type of collision, we attempt to develop separate prediction models for different crash types (i.e., single- versus multi-vehicle crashes and slight injury versus killed and serious injury crashes). Taking advantage of the availability of crash and traffic data disaggregated by time and space, it is possible to identify the factors that may contribute to crash risks in Hong Kong, including traffic flow, road design, and weather conditions. To remove the effects of excess zeros on prediction performance in a highly disaggregated crash prediction model, a bootstrap resampling method is applied. The results indicate that more accurate and reliable parameter estimates, with reduced standard errors, can be obtained with the use of a bootstrap resampling method. Results revealed that factors including rainfall, geometric design, traffic control, and temporal variations all determined the crash risk and crash severity. This helps to shed light on the development of remedial engineering and traffic management and control measures.

Quantification of proteome changes in bovine muscle from two-dimensional electrophoresis data.

Proteome changes in the longissimus thoracis bovine muscle in response to pre-slaughter stress were assessed on the basis of two-dimensional electrophoresis (2-DE) data. In this study, the bootstrap resampling statistical technique and a new measure of relative change of the volume of 2-DE protein spots are shown to be more efficient than commonly used statistics to reliably quantify changes in protein abundance in stress response. The data are supplied in this article and are related to "Tackling proteome changes in the longissimus thoracis bovine muscle in response to pre-slaughter stress" by Franco et al. [1].

The analysis of threshold effect using Empower Stats software / 中华流行病学杂志

In many studies about biomedical research factors influence on the outcome variable,it has no influence or has a positive effect within a certain range.Exceeding a certain threshold value,the size of the effect and/or orientation will change,which called threshold effect.Whether there are threshold effects in the analysis of factors (x) on the outcome variable (y),it can be observed through a smooth curve fitting to see whether there is a piecewise linear relationship.And then using segmented regression model,LRT test and Bootstrap resampling method to analyze the threshold effect.Empower Stats software developed by American X & Y Solutions Inc has a threshold effect analysis module.You can input the threshold value at a given threshold segmentation simulated data.You may not input the threshold,but determined the optimal threshold analog data by the software automatically,and calculated the threshold confidence intervals.