Real-World Evidence Confirms Clinical Efficacy of Durvalumab or Pembrolizumab Plus Gemcitabine and Cisplatin in Advanced Biliary Tract Cancer

Durvalumab or pembrolizumab combined with gemcitabine and cisplatin (GemCis) has evolved to become a standard first-line therapy option for patients with unresectable or metastatic biliary tract cancer (BTC). Because real-world populations often differ from those enrolled in prospective clinical trials, this retrospective analysis presented at ASCO GI 2026 was conducted to evaluate the outcomes in patients treated with first-line durvalumab or pembrolizumab plus GemCis in real-world clinical practice.

The retrospective analysis included 255 patients with histologically or cytologically confirmed BTC, spanning intrahepatic and extrahepatic cholangiocarcinoma (CCA) as well as gallbladder cancer. Between March 2023 and May 2025, 210 patients received first-line durvalumab plus GemCis, and 45 patients received pembrolizumab plus GemCis. Overall survival (OS) and progression-free survival (PFS) were the primary endpoints, while objective response rate (ORR) and disease control rate (DCR) served as secondary endpoints.

The median age of all patients was 66 years, and 58.8% were male. Most patients (77%) had recurrent or metastatic disease, whereas 23% had locally advanced disease. Primary tumor locations included intrahepatic CCA (40.4%), extrahepatic CCA (42.0%), and gallbladder cancer (16.5%). Across the overall population, the median OS was 14.4 months (95% CI, 12.6-16.5), and the median PFS was 6.9 months (95% CI, 6.0-8.0). The ORR was 15.3%, and the DCR was 72.5%.

Multivariable analysis identified several factors associated with poorer OS outcomes. These included elevated CA 19-9 levels (hazard ratio [HR], 1.87; 95% CI, 1.11-3.15; P=.018), gallbladder cancer compared with extrahepatic CCA (HR, 1.99; 95% CI, 1.10-3.59; P=.023), and recurrent or metastatic disease compared with locally advanced disease (HR, 2.50; 95% CI, 1.31-4.77; P=.005)

These findings support the clinical utility of combining durvalumab or pembrolizumab with GemCis as first-line therapy for advanced BTC and reinforce their effectiveness in real-world practice settings.

Source

1. Kim S, Jeong H, Cheon J, et al. Real-world outcomes of durvalumab or pembrolizumab plus gemcitabine and cisplatin in advanced biliary tract cancer. Presented at: ASCO Gastrointestinal Cancers Symposium. January 7-9, 2026; San Francisco, CA. Abstract 546.