Statistics and trial design
toxicity, tumour
ADVERSE EVENTS, PROGRESSION, TUMOUR
The trial was designed as a multicenter, non-randomized, open-label, phase II study. A Simon two-stage design was employed, with the null hypothesis that CBR, Patient demographics, clinical features and response details were described using summary statistics, such as medians, ranges, frequencies and proportions. Progression free survival and OS analyses were conducted using the Kaplan-Meier method by cohort.Medians and confidence intervals were reported to assess PFS and OS. Treatment related toxicity was evaluated using frequencies and proportions of adverse events based on severities and attributions. The clinical benefit rate and 95% CI for each cohort were estimated to evaluate the efficacy of treatment. Association between clinical benefit and biomarkers was evaluated using Chi-squared test or Fisher exact test. Association between biomarkers and survival outcomes was evaluated using Cox proportional hazards models. Individual patient’s changes in tumour response over time were displayed using spider plots.