Not one but a group of tumors that affect certain parts of the gastrointestinal system. Biliary tract cancer is a rare and aggressive pathology: its symptoms are not evident and therefore the majority of the 210 thousand new diagnoses that are registered worldwide every year occur when the disease is already in an advanced stage, with few therapeutic options. “Biliary tract cancer is a rare but constantly growing pathology, with around 5,400 new cases estimated every year in Italy – explains Carmine Pinto, Director of Medical Oncology of the Comprehensive Cancer Centre, AUSL-IRCCS of Reggio Emilia -. There are no screening tests or routine diagnostic tests capable of identifying the disease in its early stages, when surgical removal is still possible. Difficulties related to the lack of specific symptoms too often lead to diagnoses at an advanced stage.” A tumor in which the addition of immunotherapy to chemo, however, shows that it can provide significant benefits, as evidenced by the data presented during the Cholangiocarcinoma Foundation Conference 2024 in Salt Lake City, Utah.
Biliary tract tumors, immunotherapy put to the test in clinical practice by Tiziana Moriconi 19 December 2023
The tumor
The cells of the biliary tract may be affected – and therefore we speak of cholangiocarcinoma – of the gallbladder or of the ampulla of Vater, the site where the bile and pancreatic ducts connect to the small intestine. Only about 30% of patients are candidates for surgery. For patients with advanced disease, who cannot be operated on or with metastases, it is now possible to combine the immunotherapy durvalumab with chemotherapy with cisplatin and gemcitabine, which is the regimen used for over a decade. “The updated data from the TOPAZ-1 study, with a longer follow-up, show a 26% reduction in the risk of death at 3 years for patients with advanced biliary tract cancer treated with durvalumab and chemotherapy compared to chemotherapy alone chemotherapy, with double the number of patients alive”, underlines Pinto. “A particularly significant progress in a setting of neoplastic pathology in which the prognosis is historically unfavorable. These results reinforce the long-term benefit of the combination including immunotherapy as the first-line standard of care for patients with advanced disease. After more than a decade without real progress, even for patients with biliary tract tumors we have an improvement in terms of chances of survival, a very important result never achieved before.”
Cholangiocarcinoma Day, patients ask for access to genomic tests by Sara Carmignani 15 February 2024
Long-term results
The data presented in the USA represent the most extensive survival follow-up ever reported in a global randomized Phase III study in this setting. Median overall survival was 12.9 months with durvalumab plus chemotherapy versus 11.3 months with chemotherapy alone. More than twice as many patients with the durvalumab-based regimen were alive at three years compared with chemotherapy alone (14.6% vs. 6.9%). “The improvement brought by durvalumab in three-year survival data in patients with advanced biliary tract cancer is good news – states Paolo Leonardi, President of the Association of Italian Cholangiocarcinoma Patients (APIC) – and a result that increases hope in the search for treatments for patients with these rare and difficult-to-treat tumors. The path that leads to the diagnosis is too often late, making the lives of the people who receive it even more dramatic. Two things seem fundamental to us: that knowledge of the disease spreads, so that it can be suspected sooner, and that those who have a diagnosis of cholangiocarcinoma are referred early to reference centres, where complete management by a dedicated multidisciplinary team, able to best define the diagnostic and therapeutic path”.
Liver tumors, green light for immunotherapy for the two most frequent forms by Dario Rubino 12 March 2024