Hepatocellular carcinoma and intrahepatic cholangiocarcinoma are two distinct types of liver cancer. Hepatocellular carcinoma arises from liver cells, and cholangiocarcinoma arises from the cells lining the bile ducts. Hepatocellular carcinoma almost exclusively happens in individuals with underlying liver damage most commonly due to hepatitis (B or C) or metabolic syndrome (diabetes and obesity).
An estimated 42,230 Americans are expected to be diagnosed with these liver cancers in 2021. While they’re not among the 10 most common cancers, they are among the top 10 causes of cancer mortality, which is evidence to their aggressiveness.
Cancer health disparities for people with liver cancer
The racial and ethnic disparities in the incidence of and access to treatment and outcomes are striking with these cancers. Due to their poor outcomes, timely evaluation and a comprehensive workup are essential to achieving the best outcomes. For example, a patient with underlying hepatitis (B or C) is a candidate for screenings for hepatocellular carcinoma.
With early detection, the patient would be able to undergo surgery for cholangiocarcinoma or a liver transplant for hepatocellular carcinoma for their best chance of a cure.
Unfortunately, the majority of these patients are diagnosed at a late stage, which eliminates surgery as a treatment option. However, new and evolving therapies for late-stage patients are changing their prognosis and likelihood of survival.
New targeted therapies for hepatocellular carcinoma and intrahepatic cholangiocarcinoma
New targeted therapies are infiltrating the rapidly evolving treatment landscape for these cancers. Hepatocellular carcinoma, a disease with few therapeutic options prior to 2008, now has more than 10 agents—with either FDA approval or a high level of evidence for activity.
Therefore, in-depth knowledge about these treatments and their expected side effects is needed to appropriately select and transition between treatments. Given that the majority of patients with hepatocellular carcinoma have underlying liver damage, they may not be candidates for treatment or may be more prone to side effects.
Intrahepatic cholangiocarcinoma is a disease with poor prognosis. Historically, chemotherapy was the only therapeutic option for these patients—but over the past two years, new targeted agents have found their way to treatment options for these cancers. For example, the two targeted drugs pemigatinib and infigratinib have been approved by the FDA for patients with fibroblast growth factor receptor (FGFR) alterations.
Other targets with FDA-approved or National Comprehensive Cancer Network® (NCCN)–recommended therapies include IDH mutation and BRAF mutation. An estimated 15% of patients with intrahepatic cholangiocarcinoma have tumor alterations that qualify them as candidates for targeted therapy. Timely molecular testing provides the opportunity to treat these patients appropriately while allowing innovative drug development to become a reality for patient care.
This post was authored by Dr. Afsaneh Barzi
Last updated January 3, 2022
