Breakthrough in Long-Term Survival for Advanced Biliary Tract Cancer Using Combination Therapy

In a significant advancement for the treatment of advanced biliary tract cancer (aBTC), recent follow-up results from the pivotal TOPAZ-1 clinical trial have established a new survival benchmark. The study focused on the efficacy of a combination therapy involving durvalumab, an immunotherapy agent, combined with chemotherapy drugs gemcitabine and cisplatin (GemCis). This regimen was already recognized as the first-line treatment for aBTC, a challenging cancer type with generally poor prognosis.

The latest analysis, conducted approximately three years after patient enrollment, revealed that over twice as many individuals treated with durvalumab plus GemCis survived compared to those who received a placebo plus GemCis. This long-term follow-up underscores the durability of the treatment benefits and provides hope for improved management of aBTC.

Biliary tract cancers, which include intrahepatic and extrahepatic cholangiocarcinoma as well as gallbladder cancer, are typically diagnosed at an advanced stage in up to 85% of cases. These advanced cases have limited treatment options and a five-year survival rate between 3% and 13%. Even patients who undergo potentially curative surgery face high recurrence rates, emphasizing the urgent need for more effective therapies.

The updated findings, published in the Journal of Hepatology, reinforce durvalumab plus GemCis as a standard of care for first-line treatment. Notably, the long-term survival benefits were achieved without extended chemotherapy, as patients received a maximum of eight cycles.

Lead investigator Dr. Do-Youn Oh from Seoul National University highlights that these results demonstrate the potential for durable remission in aBTC patients and suggest that treatment benefits apply broadly across different patient subgroups. The study assessed not only overall survival but also patterns in long-term survivors, with no specific subgroup excluded from benefiting.

This breakthrough provides essential insights for clinicians, patients, and caregivers, guiding future treatment planning and decision-making for advanced biliary tract cancer.

Source: https://medicalxpress.com/news/2025-07-survival-benchmark-patients-advanced-biliary.html