Analysis plan
Tumors
TUMORS, SECONDARY, ADVERSE EVENTS
The primary analytic objective is to measure the overall response rate according to Response Evaluation Criteria in Solid Tumors 1.1. We also aim to determine the percentage of patients with either a complete or partial response as well as the corresponding Clopper–Pearson exact 95% CI around this measure. These results will provide useful information concerning the potential efficacy of this treatment for planning future studies.There are six secondary outcomes of interest for this pilot study: 1) response rate for the target lesion(s), 2) response rate for the nontarget lesion(s), 3) duration-of-response for the target lesion(s), 4) PFS, 5) OS and 6) tolerability assessed using Patient-Reported Outcome–CTCAE. The analytical approach for each measure is as follows: 1) for the response rate measures, we will estimate the percentage of responders (complete or partial response) and corresponding Clopper–Pearson exact 95% CI; 2) for the duration-of-response among responders, we will estimate the mean and median response duration, with the corresponding 95% CI for the mean and interquartile range for the median; 3) for time-to-event measures (PFS and OS), we will estimate Kaplan–Meier survival curves and median time-to-event times as well as percent PFS and OS at 6 months and 1 year post-treatment; 4) for tolerability, we will estimate the percentage of patients with different adverse events assessed using the Patient-Reported Outcome–CTCAE and corresponding Clopper–Pearson exact 95% CI.Additional exploratory subgroup analyses are limited by the small sample size but will be considered if adequate sample size is achieved in each subgroup. A main consideration for exploratory analysis will be to compare response rates and other oncological outcomes (duration-of-response, PFS, OS) between patients with HPV-associated oropharyngeal squamous-cell carcinoma and those with non-HPV-associated oropharyngeal cancer/nonoropharyngeal HNSCC.