In a follow-up analysis of the pivotal TOPAZ-1 study, which established the combination of the PD-L1 inhibitor durvalumab plus gemcitabine and cisplatin as the first-line treatment for advanced biliary tract cancer (BTC), researchers have shown that after 3 years, more than twice as many study participants treated with durvalumab plus the chemotherapy doublet had survived compared to those treated with placebo plus chemotherapy. These results, published by Oh et al in the Journal of Hepatology, establish a new survival benchmark for people living with advanced BTC and reinforce durvalumab plus gemcitabine and cisplatin as standard of care for the first-line treatment of the disease.
BTCs are a group of cancers that includes intrahepatic and extrahepatic cholangiocarcinoma 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 5-year survival rate ranges from approximately 3% to 13%. Even when potentially curative surgery is possible, 57% of patients with extrahepatic cholangiocarcinoma and 61% to 98% of patients with intrahepatic cholangiocarcinoma experience recurrence or death within 5 years, underscoring the need for more effective treatments for BTCs.
This new data cut-off represents the longest follow-up reported in advanced BTC studies to date; this updated analysis of overall survival and safety in the TOPAZ-1 study was conducted approximately 3 years after the last participant began the study. The results showed that after 3 years, more than twice as many participants treated with durvalumab plus gemcitabine and cisplatin were alive compared to those treated with placebo plus gemcitabine and cisplatin. These results can provide reassurance about long-term survival benefits of this treatment regimen to patients and practitioners, say the authors.
Lead investigator Do-Youn Oh, MD, PhD, of the Division of Medical Oncology, Department of Internal Medicine, Seoul National University Hospital, and Cancer Research Institute, Seoul National University College of Medicine, 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. 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 noted, “The TOPAZ-1 study readouts had already changed the treatment landscape for advanced BTC. We are very pleased to report that the benefits of the durvalumab plus gemcitabine and cisplatin regimen persist after more than 3 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 extended long-term survivor population, suggesting that all patients with advanced BTC should be treated with durvalumab plus gemcitabine and cisplatin 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 advanced BTC.”
Disclosure: For full disclosures of the study authors, visit journal-of-hepatology.eu.
