Multicenter phase 2 trial of first-line pembrolizumab plus lenvatinib and reduced-dose GEMOX for advanced biliary tract cancer

A multicenter phase 2 trial in China evaluated the combination of pembrolizumab plus lenvatinib with reduced dose gemcitabine and oxaliplatin (GEMOX) as first-line therapy for unresectable or metastatic biliary tract cancer (BTC). Sixty patients received six to eight cycles of this combination, followed by maintenance with pembrolizumab and lenvatinib. The primary endpoint was objective response rate. At a median follow-up of 16.0 months, the complete response rate was 5.0%, the partial response rate was 50.0% (ORR 55.0%) and the disease control rate was 93.3%; the median progression-free survival was 12.5 months (95% CI 7.93-16.3) and the median overall survival was 19.5 months (11.97-NE). Elevated baseline CA19-9 (> 37 U/mL) and CEA levels (> 5 ng/mL) were independently associated with poorer OS and PFS, respectively. The toxicity was manageable.The authors conclude thatpembrolizumab plus lenvatinib with reduced-dose GEMOX had promising activity and a favorable safety profile as first-line therapy for advanced BTC.