The median progression-free survival of patients with advanced biliary tract cancer, including cholangiocarcinoma (CCA) treated with durvalumab plus gemcitabine and cisplatin was 8.9 months, according to an early exploratory analysis of real-world data published in the journal Liver International. The median overall survival of the patients was 12.9  months, their overall response rate was 34.5%, and the disease-control rate of the combination therapy was 87.6%.

The study authors said that these results mostly confirm the results obtained in a phase 3, randomized, controlled trial called TOPAZ-1, which led to approval of cisplatin plus gemcitabine and durvalumab in the United States and Europe for the first-line standard of care of patients with previously untreated unresectable or metastatic biliary tract cancer, in terms of progression-free and overall survival as well as safety, “supporting the use of this combination in clinical practice.”

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The present study aimed to investigate the safety and efficacy of this new standard treatment in a real-world setting.

The team led by Andrea Casadei-Gardini, MD, PhD, from the Vita-Salute San Raffaele University in Milan, Italy, analyzed 145 patients with unresectable, locally advanced or metastatic biliary tract cancer treated with durvalumab plus gemcitabine and cisplatin between February 2022 and November 2022. The median follow-up was 8.5 months. 

The primary endpoint was progression-free survival.  Secondary endpoints were overall survival, overall response rate, and safety.

Almost all patients (94.5%) had an adverse event following treatment, with more than a third (35.2%) experiencing a grade 3 or 4 adverse event. 

The rate of immune-mediated adverse events was 22.7%, and the rate of grade 3 or 4 immune-mediated adverse events was 2.1%.

Statistical analysis, nonviral etiology, Eastern Cooperative Oncology Group performance status greater than 0, and a neutrophil to lymphocyte ratio of 3 or more correlated with shorter progression-free survival.


Rimini M, Fornaro L, Lonardi S, et al. Durvalumab plus gemcitabine and cisplatin in advanced biliary tract cancer: an early exploratory analysis of real-world data. Liver Int. Published online July 14, 2023. doi:10.1111/liv.15641