Statistics and reproducibility
toxicity, tumor
TUMOR
The clinical trial was a non-randomized phase I trial. For the dose escalation component, a traditional 3 + 3 design was used. By study design, 3–6 patients were expected to enroll. An additional 15-patient expansion cohort was planned. With 6 patients treated at the MTD in the dose escalation phase and an additional 15-patient expansion cohort, a minimum of 21 patients were expected to be available for assessing safety and estimating efficacy at the MTD. The trial closed early following the accrual of 5 patients to the expansion cohort due to activation of the phase 3 SWOG/NRG S1914, which enrolled this population. A total of 20 patients were enrolled. Descriptive statistics (including frequency, proportion, median, and range) were used to summarize patient demographics, tumor characteristics, toxicity, and response status. A waterfall plot was generated to describe the relative changes in tumor size using R version 4.0.4. OS, PFS, and FFP were analyzed by the Kaplan–Meier methodThe sample size for correlative studies was based on the presence of sufficient material to perform the studies. No statistical method was used to predetermine the sample size for correlative studies. No data were excluded from the analyses. This trial did not include randomization, and investigators were not blinded during experiments and outcome assessment. Statistical analysis of IHC/IC data was performed using descriptive statistics. Comparisons across two groups (e.g., responders vs. non-responders or non-progressor vs. progressor) were performed using a two-sided 