Real-world effectiveness and prognostic factors of durvalumab plus chemotherapy in a multicentric cohort with advanced biliary tract cancer.

Background: Biliary tract cancer (BTC) is an aggressive biliary tract cancer, arising from the bile ducts and gallbladder, with a poor prognosis. The TOPAZ-1 trial of durvalumab plus first-line chemotherapy (gemcitabine plus cisplatin) showed improved survival vs chemotherapy alone. This real-world study aimed to confirm the effectiveness of this regimen.

Methods: This retrospective, multicenter study included patients with advanced BTC treated with first-line durvalumab plus platinum chemotherapy at the Linkou, Taoyuan, and Tucheng branches of Chang Gung Memorial Hospital as well as at Taipei Veterans General Hospital between August 2021 and June 2023.

Results: Among the 45 patients with advanced biliary tree cancer treated with durvalumab plus cisplatin and gemcitabine as first-line treatment, the objective response rate was 31.1% (14 partial responses). An additional 40% (18 patients) had stable disease. The median progression-free survival was 5.6 months (95%CI, 4.4-6.9) and median overall survival was 15.8 months (95%CI, 7.9-23.8). Responders had significantly longer survival than non-responders (15.8 vs 3.3 months). Although higher durvalumab doses (1000-1500 mg) appeared to have improved efficacy compared to lower doses (<1000 mg), the difference was not statistically significant. On multivariate analysis, poor ECOG performance status (≥2) and a high neutrophil-lymphocyte ratio were independent prognostic factors for shorter overall survival.

Conclusion: This real-world study demonstrated the comparable efficacy of durvalumab plus chemotherapy to the TOPAZ-1 trial for patients with advanced BTC and identified prognostic factors. There was a trend toward improved efficacy with higher durvalumab dosing (1000-1500 mg) vs lower dosing, though further research is needed to confirm this relationship.