Phase 2 study of first-line toripalimab in combination with gemcitabine and 5-fluorouracil for unresectable or metastatic biliary tract carcinoma

A phase 2 study at Jiangmen Central Hospital (China) evaluated toripalimab (anti-PD-1) in combination with gemcitabine and 5-fluorouracil chemotherapy as first-line treatment for unresectable or metastatic biliary tract carcinoma (U/M BTC). The primary endpoints were objective response rate and progession-free survival. The study included 30 patients (23 response-evaluable), with a median follow-up duration of 16.0 months. Partial response was observed in three patients (ORR 13%), with response durations ranging from 6.3 to 21.8 months, and stable disease was observed in 16 patients (disease control rate 82.6%). The median progression-free survival and median overall survival were 5.3 months (95% CI 3.6-7.0) and 11.7 months (6.1-17.3), respectively. Subgroup analyses revealed no significant association between PD-L1 status or tumor mutational burden with PFS. All patients experienced adverse events, and eight patients (26.7%) experienced grade 3 or 4 AEs. No grade 5 AEs were observed. The authors conclude that toripalimab in combination with gemcitabine and 5-fluorouracil showed promising efficacy and was well-tolerated as first-line therapy in U/M BTC.