Real-World Analysis of Gemcitabine Plus Cisplatin, Durvalumab as a First-Line Treatment in BTC

A real-world analysis of the phase 3 TOPAZ-1 study conducted by Margherita Rimini, MD, and colleagues published in the European Journal of Cancer found that gemcitabine plus cisplatin and durvalumab shows potential as a first-line treatment for advanced biliary tract cancer (BTC).

The retrospective analysis reviewed the data of 666 patients with unresectable locally advanced or metastatic BTC who received treatment at 39 sites across 11 countries. The study’s primary end point was overall survival (OS).

Dr. Rimini and colleagues reported that the median OS rate was 15.1 months, the median progression-free survival was 8.2 months, the investigator-assessed overall response rate was 32.7%, and the stable disease rate was 45.2%.

High baseline carcinoembryonic antigen levels, Eastern Cooperative Oncology Group performance status greater than 0, metastatic disease, and a neutrophil-to-lymphocyte ratio greater than 3 were associated with lower survival odds.

Twenty percent of patients reported immune-related adverse events (irAEs), and 92.9% reported any grade AEs. While no deaths were related to immunotherapy, 3 (0.5%) were treatment related. Of the irAEs, the most frequently reported were rash (8.2% all grades; 0.3% grade >2), itching (10.3% all grades; 0.2% grade >2), and hypothyroidism (5.1% all grades; 0.3% grade >2).

Researchers noted that of the genes recommended for testing by the European Society for Medical Oncology, no outcomes differences were discovered. Following a comparative analysis, which included a historical group of patients who received chemotherapy alone compared with those who received gemcitabine plus cisplatin and durvalumab, Dr. Rimini and colleagues found that the combination therapy group demonstrated an increased survival benefit.

“This first global 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,” the investigators wrote.