Biliary tract tumor

Biliary tract tumors account for about 1.1% of new cancer cases. This category includes several pathological entities, such as gallbladder neoplasms and cholangiocarcinomas (CCA), both intrahepatic and extrahepatic. This distinction is crucial for the diagnostic pathway, biological, genetic, and molecular features, choice of therapy, and the possibility of inclusion in experimental protocols. 

The main risk factors include gallbladder stones, primary sclerosing cholangitis, Caroli disease, hepatic cirrhosis (of viral or post-alcoholic origin), some parasitic infections, hereditary diseases (such as hemochromatosis, tyrosinemia, glycogen storage diseases, Wilson's disease), and exposure to substances like radon, asbestos, nitrosamines, and dioxin. These conditions share the ability to induce a state of chronic inflammation of the biliary tract. 

DIAGNOSTIC PATHWAY AND DEFINITION OF TREATMENT STRATEGY 

FIRST VISIT: 

Patients can access our facility in various ways: they may be referred by their general practitioner, external entities, internal clinics or endoscopy services, or even by complex surgical structures. Under the National Health Service, the patient must be physically present for the consultation. During the first visit, the patient’s general clinical conditions and comorbidities are analyzed, and existing diagnostic documentation is reviewed. Sometimes, it is necessary to request and review the tumor tissue used for diagnosis. 

Both patients with recent diagnoses and those already receiving treatment elsewhere who need a specialist consultation are admitted to the first visit. 

DIAGNOSTIC PATHWAY: 

Diagnosis and staging proceed in tandem with assessment of resectability. 

In this context, the multidisciplinary approach is of fundamental importance, and our Institute has a very active multidisciplinary group on these pathologies. 

It starts with an evaluation of the patient’s general condition and imaging and laboratory tests (such as chest/abdominal CT scan and blood work). CT scans assess tumor extension, relationships with adjacent organs and vessels, the presence of metastases, and possible complications (e.g., pulmonary thromboembolism). 

When needed, endoscopic retrograde cholangiopancreatography (ERCP) may be performed, allowing targeted visualization of the biliary tract, sampling of tumor tissue, and, in cases of obstructive jaundice, a therapeutic-palliative approach (placement of a biliary stent). In selected cases, percutaneous transhepatic cholangiography (PTC) is also an option, performed by inserting a catheter into the bile ducts through the abdominal wall under radiological guidance. 

TREATMENT 

Each patient receives a personalized treatment plan according to the most advanced scientific standards. Thanks to clinical research, we are able to offer innovative therapeutic solutions, including targeted treatments based on the tumor’s genetic features, immunotherapies, and approaches based on new mechanisms of action or strategies. Patients are always considered for inclusion in clinical trials when available. 

Patients undergoing systemic treatment also receive continuous monitoring of their clinical condition, with the help of Pain Therapy, Supportive Care, and Clinical Nutrition services to ensure effective control of symptoms related to the disease as well as side effects from treatments. 

Finally, the therapeutic pathway is multidisciplinary and collegial, involving ongoing reassessment of possible loco-regional and/or palliative therapies to be integrated with systemic treatment. 

TREATMENT OF LOCALIZED DISEASE 

RESECTABLE DISEASE: Up to 30% of biliary tract tumor patients are diagnosed when the disease is still radically operable. In these cases, radical surgery is the therapeutic approach recommended by guidelines. The type of surgery depends on the tumor’s site, size, and the need to achieve disease-free resection margins. 

UNRESECTABLE DISEASE: In cases of locally advanced, unresectable disease, systemic treatment is indicated using protocols similar to those for advanced or metastatic disease. If there is a significant reduction in disease after treatment, the feasibility of surgical options may be reassessed. 

It is worth noting that our Institute has innovative research protocols that, where possible, integrate medical and loco-regional therapies, up to (in very select cases) liver transplantation. 

TREATMENT OF METASTATIC DISEASE 

For patients with metastatic disease, the start of first-line treatment is considered, aiming to prolong survival and manage tumor-associated symptoms. 

Systemic treatment may consist of one or more chemotherapy drugs (generally a combination of gemcitabine, cisplatin, and immunotherapy), chosen based on a comprehensive clinical assessment. 

The duration of chemotherapy and any modifications of treatment regimens are evaluated according to the patient’s tolerance and the treatment’s efficacy, as well as the patient’s general condition. 

In recent years, clinical research has demonstrated the effectiveness of various targeted drugs after progression on first-line therapy. Our Institute is a reference center for its extensive analysis of the tumor’s molecular characteristics at the time of diagnosis of advanced disease. This allows us to promptly identify therapeutic options available for patients showing progression after first-line treatment. 

For patients whose disease does not have molecular alterations for which drug therapy is available, standard treatment consists of chemotherapy, usually with fluoropyrimidine-based regimens. 

Whenever possible, we always prioritize the opportunity to enroll in an experimental trial. 

It should be emphasized that biliary tract tumors are highly aggressive and may lead to a rapid deterioration in clinical condition. The possibility of systemic treatment, especially after progression during standard therapies, must be assessed on a case-by-case basis, carefully considering the risks, benefits, and tolerability of the available options for each patient.