Survival Outcomes of Durvalumab in Combination with Cisplatin and Gemcitabine in Advanced Biliary Tract Cancer: Real-World Results from a Single Italian Institution.

INTRODUCTION

The TOPAZ-1 phase III trial showed a survival benefit with durvalumab plus gemcitabine and cisplatin in patients with advanced biliary tract cancer (BTC). To understand this combination's real-world efficacy and tolerability, we conducted a retrospective analysis of its first-line treatment outcomes.

METHODS

We included patients with unresectable, locally advanced, or metastatic BTC treated with cisplatin, gemcitabine, plus durvalumab. The primary endpoint was overall survival (OS).

RESULTS

Thirty-three patients were enrolled. Median OS was NR and median progression free survival (PFS) was 7.6 months, after a median follow-up of 13.5 months. The investigator-assessed overall response rate was 34.5%, with stable disease in 53.0% of patients. High baseline CEA levels were associated with poor survival. Any grade adverse events (AEs) occurred in 97% of patients. Immune-related AEs (irAEs) occurred in 16% (grade >2: 6%). Presence of TP53 mutation was related to a worse OS; conversely the presence of ARID1A genomic alteration was related to a better PFS. A tendence toward a better OS was found for BRCAness patients which did not reach the statistical significance. On the other hand, BRCAness patients showed significantly higher PFS compared to no BRCAness patients.

CONCLUSION

This real-world analysis largely confirmed the TOPAZ-1 findings, supporting gemcitabine, cisplatin, and durvalumab as a first-line standard of care for patients with advanced BTC.