Durvalumab in Advanced Biliary Tract Cancer: Real‐World Data From a Large Cohort of Patients Across Multiple International Centers

Background: We recently published the first real-world multicenter and multi-institutional study of cisplatin, gemcitabine, and durvalumab in patients with advanced biliary tract cancer (BTC). Here we present an expanded patient cohort with an increased sample size and longer median follow-up. Methods: The study population included patients with advanced BTC, who received cisplatin/gemcitabine plus durvalumab at 55 centers across 12 countries in Europe, the United States, and Asia. The primary endpoints of the study were progression-free survival (PFS) and overall survival (OS). Secondary endpoints were overall response rate (ORR) and safety. Results: Overall, 1358 patients were enrolled. Median PFS was 7.6 months (95% CI: 7.2-8.1), and median OS was 15.6 months (95% CI: 14.8-16.5). ORR was 35.6%, and DCR was 82.7%. Any grade AEs occurred in 1213 patients (89.3%). Grade 3-4 AEs occurred in 597 patients (43.2%). The rate of immune-related AEs (irAE) was 20.3%. Grade 3-4 irAE occurred in 3.0% of patients. At the multivariate analysis for OS, normal albumin level (HR 0.68, 95% CI 0.57-0.81, p < 0.0001), CEA levels within normal ranges (HR 0.68, 95% CI 0.57-0.82, p < 0.0001), NLR < 3 (HR 0.62, 95% CI 0.52-0.74, p < 0.0001), ECOG PS 0 (HR 0.51, 95% CI 0.42-0.61, p < 0.0001), and prior surgery (HR 0.80, 95% CI 0.65-0.99, p = 0.036) were positive prognostic factors. Conclusion: The updated findings, derived from an expanded cohort, further support the adoption of durvalumab in combination with gemcitabine and cisplatin in routine clinical practice, reinforcing the efficacy and safety outcomes demonstrated in the phase III TOPAZ-1 trial.