Camrelizumab plus oxaliplatin‐based chemotherapy as first‐line therapy for advanced biliary tract cancer: A multicenter, phase 2 trial

Abstract Biliary tract cancer (BTC) is a highly malignant tumor with limited treatment options and poor prognosis. Our study aimed to evaluate camrelizumab plus oxaliplatin‐based chemotherapy as first‐line therapy for advanced BTC. In this multicenter, open‐label, phase 2 trial conducted in China ( ClinicalTrials.gov , NCT03092895), untreated patients with advanced BTC were given camrelizumab (3 mg/kg iv drip injection, every 2 weeks) plus typical FOLFOX4 (Cam‐FOLFOX4 group; infusional 5‐fluorouracil, leucovorin and oxaliplatin) or GEMOX (Cam‐GEMOX group; infusional gemcitabine and oxaliplatin). The primary endpoint was objective response rate (ORR). Ninety‐two patients were enrolled: 29 received Cam‐FOLFOX4 and 63 received Cam‐GEMOX. The confirmed ORR and disease control rate were 16.3% (95% confidence interval [CI] = 9.4‐25.5) and 75.0% (95% CI = 64.9‐83.4), respectively. Median duration of response was 8.7 months (95% CI = 5.1‐not reached). Median progression‐free survival and overall survival were 5.3 months (95% CI = 3.7‐5.7) and 12.4 months (95% CI = 8.9‐16.1), respectively. Grade ≥3 treatment‐related adverse events (TRAEs) occurred in 82.8% of patients receiving Cam‐FOLFOX4 and in 68.3% receiving Cam‐GEMOX, with no unexpected effects observed. Six (6.5%) patients discontinued treatment due to TRAE. Camrelizumab plus FOLFOX4 or GEMOX as first‐line treatment was effective and tolerable for Chinese patients with advanced BTC, warranting phase 3 trials.