Statistical analysis
tumor
TUMOR, DISEASE CHARACTERISTIC
The sample size of this trial was calculated according to an exact single-stage design. A total ORR ≤ 14% (the ORR was 13.5% in our previous observational study on GEMOX for advanced ICC, unpublished data) was considered not clinically meaningful; an overall response ≥40% was considered promising. Thirty patients were needed to achieve a power of 90% with a significant level of 5%. All efficacy and safety endpoints were analyzed in all subjects who obtained at least one dose of toripalimab.The ORRs and 95% confidence intervals (CIs) were determined by the Clopper-Pearson method. We calculated the number of subjects with a best tumor response and the DCR. We calculated the maximum percentage change in the sum of the diameters of the target lesions for each subject (from baseline) and pictured these parameters as a waterfall plot. We used the Kaplan‒Meier method to summarize OS, PFS, and DoR. Medians and 95% CIs (calculated with the Brookmeyer-Crowley method) are presented. Further, 95% CIs for OS and PFS rates were determined based on the Greenwood formula. Post hoc ORR subgroup analyses stratified by selected demographic and disease characteristics were conducted accordingly. Exploratory subgroup analysis stratified by intensity of tumor PD-L1 and DDR-related gene mutation was performed.All data were analyzed by the aid of SPSS 19.0 software (IBM, Armonk, NY, USA) and R software version 4.1.0 (The R Foundation, Vienna, Austria). This trial is registered with Clinicaltrials.gov, number NCT03951597.