Combination therapy sets new survival standard for advanced biliary tract cancer

In a follow-up analysis to the pivotal TOPAZ-1 study, which established the combination therapy of durvalumab (an immunotherapy drug) plus gemcitabine and cisplatin (GemCis, chemotherapy drugs) as the first-line treatment for people with advanced biliary tract cancer (aBTC), researchers have shown that after three years more than twice as many study participants treated with durvalumab plus GemCis had survived compared to those treated with a placebo plus GemCis. The new results in the Journal of Hepatology, published by Elsevier, establish a new survival benchmark for people living with aBTC and reinforce durvalumab plus GemCis as a standard of care for first-line treatment for aBTC.

Biliary tract cancers (BTCs) are a group of cancers that includes intrahepatic and extrahepatic cholangiocarcinoma (bile duct cancer inside or outside the liver) and gallbladder cancer. Up to 85% of individuals with BTCs are diagnosed with advanced disease, at which point prognosis is poor, curative surgery is not feasible, and the five-year survival rate ranges from approximately 3-13%. Even when potentially curative surgery is possible, 57% of patients with extrahepatic cholangiocarcinoma and 61-98% of patients with intrahepatic cholangiocarcinoma experience recurrence or death within five years, underscoring the need for more effective treatments for BTCs.

This new data cut-off represents the longest follow-up reported in aBTC studies to date; this updated analysis of overall survival and safety in the TOPAZ-1 study was conducted approximately three years after the last participant began the study. The results showed that after three years, more than twice as many participants treated with durvalumab plus GemCis were alive compared to those treated with placebo plus GemCis. These results can provide reassurance about long-term survival benefits of this treatment regimen to patients and caregivers.

Lead investigator Do-Youn Oh, MD, PhD, Division of Medical Oncology, Department of Internal Medicine, Seoul National University Hospital, and Cancer Research Institute, Seoul National University College of Medicine, Seoul, Republic of Korea, explains, "Following on from the previous analysis, we assessed the length of time that participants survived after starting treatment as well as any serious side effects they experienced. We also examined the characteristics of participants still alive at least 30 months after starting treatment to see if any patterns were apparent in this group of 'extended long-term survivors' (eLTS). Finally, we assessed the subsequent anticancer therapy received by participants after finishing the study treatment and any impact on how long participants survived."

Dr. Oh notes, "The TOPAZ-1 study readouts had already changed the treatment landscape for aBTC. We are very pleased to report that the benefits of the durvalumab plus GemCis regimen persist after more than three years, and that the long-term survival benefits were observed without the need to continue chemotherapy over an extended period of time, as participants received a maximum of eight cycles of chemotherapy. In addition, no specific subgroup was driving or excluded from the eLTS population, suggesting that all patients with aBTC should be treated with durvalumab plus GemCis since participants appeared to have benefited from the treatment regardless of baseline characteristics. We hope the results will help physicians, patients, and caregivers make informed decisions for the treatment of aBTC."

Source:
Journal reference:

Oh, D.-Y., et al. (2025). Durvalumab plus chemotherapy in advanced biliary tract cancer: 3-year overall survival update from the phase III TOPAZ-1 study. Journal of Hepatology. doi.org/10.1016/j.jhep.2025.05.003.

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