KEY TAKEAWAYS
- The TOPAZ-1 phase 3 trial aimed to assess the efficacy and safety of the durvalumab + gemcitabine + cisplatin as a first-line treatment for BTC in a real-world setting .
- The primary endpoint was OS.
- Real-world analysis supported gemcitabine, cisplatin, and durvalumab as standard first-line treatment for advanced BTC.
This phase 3 trial demonstrated a survival benefit with anti-PD-L1 durvalumab when combined with gemcitabine and cisplatin (durvalumab + gemcitabine + cisplatin) for patients (pts) with advanced biliary tract cancer (BTC).
Andrea Casadei Gardini and the team designed this trial to further understand efficacy and tolerability of this combination, They conducted a worldwide multicenter retrospective analysis to evaluate its efficacy and safety as a new first-line standard treatment in a real-world setting in pts with advanced BTC.
The study included pts with unresectable, locally advanced, or metastatic BTC treated at 38 sites across 10 countries (Europe, United States, and Asia) with durvalumab + gemcitabine + cisplatin. The primary endpoint of the study was to evaluate overall survival (OS).
About 618 pts were enrolled in the study. As per the data cutoff on January 20, 2024, the median follow-up duration was 8.5 months (95% CI: 7.9-9.4). During this period, 52.9% pts discontinued to take the treatment due to disease progression, and 30.4% had died.
The median OS and progression-free survival (PFS) was 15.1 months (95% CI 13.4-29.1) and 8.1 months (95% CI 7.5-8.7) respectively. The overall response rate (ORR) was 31.7%, which comprised complete responses (CR) in 2.5% and partial responses (PR) in 29.6%. Stable disease (SD) was observed in 46.7% pts, concluding a disease control rate (DCR) of 78.8%.
Adverse events (AEs) of any grade occurred in 92.8% of pts, with grade 3-4 AEs were in reported in 46.0% of cases. The most frequent AEs included fatigue (55.1%), anemia (48.7%), neutropenia (46.0%), and thrombocytopenia (38.5%). Immune-mediated AEs (imAEs) occurred in 19.7% pts, with grade 3-4 imAEs in 2.5% of cases.
The analysis concluded that the real-world analysis largely validated the findings from the TOPAZ-1 trial, supporting durvalumab + gemcitabine + cisplatin as a standard first-line therapy for advanced BTC. This underscored the efficacy of the said treament and its potential as a clinical standard in pts with advanced BTC.
The trial was sponsored by AstraZeneca.
Source: https://cslide.ctimeetingtech.com/esmogi24hybrid/attendee/confcal/show/session/3
Clinical Trial: https://clinicaltrials.gov/study/NCT03875235
Gardini A C, Fornaro L, Lonardi S, et al. (2024). “Durvalumab plus gemcitabine and cisplatin in advanced biliary tract cancer: A large real-life worldwide population.” Presented at ESMO-GI 2024, (Abstract 293P)
