Longitudinal Study Sets New Survival Benchmark for Patients with 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.”

Notes for editors

The article is “Durvalumab plus chemotherapy in advanced biliary tract cancer: 3-year overall survival update from the phase III TOPAZ-1 study," by Do-Youn Oh, Aiwu Ruth He, Shukui Qin, Li-Tzong Chen, Takuji Okusaka, Jin Won Kim, Thatthan Suksombooncharoen, Myung Ah Lee, Masayuki Kitano, Howard A. Burris, Mohamed Bouattour, Suebpong Tanasanvimon, Renata Zaucha, Antonio Avallone, Juan Cundom, Aleksandra Kuzko, Julie Wang, Ioannis Xynos, Arndt Vogel, and Juan W. Valle (https://doi.org/10.1016/j.jhep.2025.05.003). It appears online in advance of the Journal of Hepatology, volume 83, issue 5 (November 2025) published by Elsevier.

The article is openly available at https://www.journal-of-hepatology.eu/article/S0168-8278(25)02201-9/fulltext.

Full text of this article is also available to credentialed journalists upon request; contact Freya Weise at +33 (1) 71 16 55 00 or [email protected]. Journalists wishing to interview the authors should contact Do-Youn Oh, MD, PhD, at [email protected].

Clinical trial registration: ClinicalTrials.gov Identifier: NCT03875235; https://clinicaltrials.gov/study/NCT03875235.

This study was sponsored by AstraZeneca. The sponsor of the study had a role in study design, data analysis, data interpretation, and writing of the report. The sites entered the data; the funders had clinical research associates and monitors to liaise with the study.

About the Journal of Hepatology

The Journal of Hepatology, the premier journal devoted to liver diseases, is the official journal of the European Association for the Study of the Liver (EASL). It publishes original papers, reviews, case reports, and letters to the Editor concerned with clinical and basic research in the field of hepatology. The journal has a 2024 Impact Factor of 33 (Source: Journal Citation Reports™ from Clarivate, 2025). www.journal-of-hepatology.eu

About EASL

In the fifty plus years since EASL was founded, it has grown from a small organization that played host to 70 participants at its first meeting, to becoming the leading international liver association. EASL attracts the foremost hepatology experts as members and has an impressive track record in promoting research in liver disease, supporting wider education, and promoting changes in European liver policy. www.easl.eu

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