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Cholangiocarcinoma, a rare tumor of the bile duct

Cholangiocarcinoma, a cancer of the bile ducts, is a rare and aggressive tumor. In most cases there is no identified causes, however it can often be linked to other risk factors such as inflammatory diseases, cirrhosis, or infections. Surgery is the only potentially curative solution, but it is only possible for a limited number of patients and the risk of relapse remains high.

Chemotherapy and immunotherapy are the standard therapy for patients with cholangiocarcinoma who are not eligible for surgery or with a disease that has progressed after surgery. Developments in immunotherapy and new targeted therapies bring greater hope and better quality of life for patients.

Intrahepatic or extrahepatic primary bile duct cancer

Cholangiocarcinoma is a primary cancer, rather than a metastatic cancer, that has spread from a primary tumor to another organ. It develops in the bile ducts that carry the digestive fluid bile from the gall bladder to the small intestine. There are three main subtypes of cholangiocarcinoma1 that depend on the location of the tumor in the biliary system:

  • Intrahepatic cholangiocarcinoma, which occurs inside the liver in the small or medium bile ducts.
  • Hilar or perihilar extrahepatic cholangiocarcinoma, which occurs where the right and left hepatic ducts join and leave the liver up to the junction with the gall bladder.
  • Distal cholangiocarcinoma, also known as extrahepatic cholangiocarcinoma, which occurs outside the liver after the hepatic bile ducts have joined to form the common bile duct.

There is considerable regional variation, but the latest data suggest that localization of the cholangiocarcinoma is intrahepatic more or less half the time and extrahepatic the other half.2

A rare and aggressive form of cancer

Cholangiocarcinoma is a rare and aggressive tumor. It affects 1–3 in 100,000 people in Europe, with approximately 10,000 new cases each year.3 The five-year survival rate is 9%, but 0% if metastasized.4

Cholangiocarcinoma is considered a rare cancer. In Western Europe, North America and Australasia, estimates of occurrence vary between 0.3 and 3.5 cases per 100,000 every year. To give an example, this prevalence would amount to around 8,000 cases in the United States and 2,000 in France.5

Cholangiocarcinoma is more common in regions with a higher incidence of infections caused by a liver parasite. Thailand (90 cases per 100,000 population) has the highest incidence in the world,6 followed by China and Korea.

The actual number of cases is probably underestimated, since this form of cancer is difficult to diagnose. It is sometimes wrongly diagnosed as another type of cancer.7

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Illustrated infographic presenting the cholangiocarcinoma

Often linked to medical history

Several factors increase the risk of developing a bile duct cancer. Frequently, they are related with a history of previous disease or inflammation of the bile ducts.8 Other risk factors also include age (patients are on average 70 years old)9, obesity, diabetes, and exposure to certain types of chemicals.

Diagnosis: imaging and biopsy

Diagnostic tests depend on the signs and symptoms, age, general health, as well as the results of previous tests. Bile duct cancer is often diagnosed late because many patients display no signs or symptoms. The first signs of the disease are often non-specific and appear when the tumor is large and compresses the large bile ducts, which can cause jaundice (yellowing of the skin) in extrahepatic cholangiocarcinoma. However, the intrahepatic form may be found by chance through X-rays or MRI taken for other reasons, or show up as an anomaly in blood tests, or because bile secretion is reduced or stops completely. Unfortunately, there are no reliable biological markers specific to these bile duct cancers.

Imaging alone (ultrasound, scan or MRI) can detect the tumor, locate it and determine whether it has spread to other organs. It can also help determine if the tumor can be surgically removed (whether it is resectable or unresectable).10, 11 Next, a biopsy (extracting a fragment of tissue) and its analysis (histology) can provide more information on the tumor.

Surgery dominates treatment options

Surgical resection is the only potential option for curing cholangiocarcinoma. If surgery is not possible, palliative chemotherapy or radiation therapy may be offered. A full liver transplant may also be considered. It is also recommended to treat the jaundice at the same time as the cancer.

New therapies are being developed for this aggressive disease with limited treatment options currently available. Over the past few years, a number of targeted therapies have been approved. These therapies target the genes, proteins or surrounding tissue that help the cancer to survive and grow. They block cancer cells from proliferating without damaging healthy cells.

Immunotherapy aims to boost the body’s natural defenses to fight the cancer and also brings new hope for cholangiocarcinoma patients.

Photo d'un patient atteint de cholangiocarcinome et son aidant

AND SERVIER?

To address the ever-increasing incidence of cancer and the growing need for therapeutic solutions, we have made oncology a priority focus for development. We allocate over half of our R&D budget in oncology, with the ambition to become an innovative player focused on developing medicines targeting hard-to-treat cancers with high unmet medical needs. We wish to initiate therapeutic progress where generally speaking needs are not yet met, known as hard-to-treat cancers, such as digestive cancers (stomach cancer, pancreatic cancer, and cholangiocarcinoma, a rare and aggressive form of bile duct cancer), glioma, or brain tumors, hematologic cancers (acute myeloid leukemia, acute lymphoblastic leukemia) and pediatric cancers.

Servier leads its R&D programs around two approaches:

  • Immuno-oncology, to activate the immune system against cancer cells
  • Targeted therapies, to specifically target one of the biological mechanisms of cancer cells

WHAT YOU NEED TO KNOW

  1. Cholangiocarcinoma is a rare and aggressive bile duct cancer. It is often diagnosed late as the initial signs are non-specific.
  2. It is linked to a history of bile duct disease or inflammation, or to parasitic infections. Obesity and alcohol abuse are risk factors.
  3. Surgical resection is the only potential option for curing cholangiocarcinoma. Targeted therapies and immunotherapy represent new hope for patients.