Statistical analysis
This phase 2 study aimed to discriminate a promising ORR of 40% from a ORR of 10% using a type I error of 0.05 and power of 80%. With this assumption, 13 evaluable patients were needed.Patients who received at least one dose of either study drug were included for safety analysis and efficacy analysis. Descriptive statistics and the Clopper-Pearson method were used to summarize the proportion of patients with response and to calculate the 95% confidence intervals (CI), respectively. All time-to-event endpoints (PFS, DoR, and OS) were analyzed by the Kaplan–Meier method with a 95% CI. The 95% CI for the median times was estimated using the Brookmeyer-Crowley method. All statistical analyses were conducted using SAS 9.4 (SAS Institute Inc, Cary, NC, USA).