Statistical Analysis
tumor
MAY, TUMOR
To estimate effect size, 2 readers (K.P.R. and M.J.O.) retrospectively calculated mpRECIST in 5 patients with low-grade mucinous AA. The mean change in tumor size over a 6-month time period in patients receiving treatments was a 1.6% increase; in those without treatment, the increase was 9%. Based on these preliminary data, a 7.4% (95% CI, 3.0%-11.7%) effect size was observed. The SD of residuals was 3.5% for the random effects introduced by the 2 readers. Considering both the variation introduced by different readers and variation of the treatment effects, the combined SD of differences was 4.1%. Based on these preliminary data, we deemed a difference of at least 5% in mpRECIST-determined tumor size change to be clinically meaningful. Assuming a crossover analysis of variance square root of mean square error of 4.0% and a 1-sided α = .05, it was estimated that 24 patients would provide 80% power to detect a 5% difference; enrollment of up to 30 patients was planned to have complete 6- and 12-month tumor measures for 24 patients.Crossover analyses were performed according to Senn methods.A safety monitoring rule was in place to stop the trial early if a Fisher exact test ever detected a difference between the treatment and observation period in this composite measure that was ever significant at the α = .05 level. OS was estimated in each group and graphed by Kaplan-Meier methods. Comparison between the treatment first and observation first groups was performed with a log-rank test. Kaplan-Meier curves were implemented in Stata statistical software version 16 (StataCorp). All other analyses were performed in SAS statistical software version 9.4 (SAS Institute). Data were analyzed from November 2021 to May 2022.