Overview
Adult hepatocellular carcinoma (HCC), also known as hepatoma, is the most common primary malignant tumor of the liver occurring in adults. It arises from hepatocytes, the main functional cells of the liver, and predominantly develops in the setting of chronic liver disease and cirrhosis. Major risk factors include chronic hepatitis B virus (HBV) and hepatitis C virus (HCV) infection, alcohol-related liver disease, non-alcoholic steatohepatitis (NASH), aflatoxin exposure, and metabolic conditions such as hereditary hemochromatosis and alpha-1 antitrypsin deficiency. The disease primarily affects the hepatobiliary system but can metastasize to the lungs, bones, lymph nodes, and adrenal glands. Key clinical features include abdominal pain or discomfort (particularly in the right upper quadrant), hepatomegaly, weight loss, fatigue, jaundice, ascites, and deterioration of liver function. Many patients are asymptomatic in early stages, and the tumor is often detected through surveillance programs in high-risk populations using ultrasound and serum alpha-fetoprotein (AFP) measurement. Paraneoplastic manifestations such as hypercalcemia, erythrocytosis, and hypoglycemia may occasionally occur. The treatment landscape for adult HCC is complex and depends on tumor stage, liver function (often assessed by the Child-Pugh score), and the patient's overall performance status. Curative options include surgical resection, liver transplantation, and local ablation therapies (radiofrequency or microwave ablation) for early-stage disease. Transarterial chemoembolization (TACE) is used for intermediate-stage disease. For advanced-stage HCC, systemic therapies have expanded significantly and now include tyrosine kinase inhibitors (such as sorafenib and lenvatinib), immune checkpoint inhibitors (such as atezolizumab combined with bevacizumab), and other targeted agents. Despite advances, prognosis remains poor for patients diagnosed at advanced stages, underscoring the importance of early detection and prevention strategies.
Also known as:
Multifactorial
Caused by a mix of several genes and environmental factors
Adult
Begins in adulthood (age 18 or older)
FDA & Trial Timeline
10 eventsSeattle Children's Hospital — PHASE1
Hong Wu — NA
University of Wisconsin, Madison — PHASE1, PHASE2
Riboscience, LLC. — PHASE2
AstraZeneca — PHASE1
Asan Medical Center — PHASE2
Bristol-Myers Squibb — PHASE1, PHASE2
Beijing Biotech — PHASE1, PHASE2
Second Affiliated Hospital, School of Medicine, Zhejiang University — PHASE2
UNICANCER — PHASE3
Data sourced from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.
Treatments
6 availableNexavar
NEXAVAR is indicated for the treatment of patients with unresectable hepatocellular carcinoma (HCC)
IMFINZI
In combination with tremelimumab-actl for the treatment of adult patients with unresectable hepatocellular carcinoma (uHCC)
Imjudo
In combination with durvalumab for the treatment of adult patients with unresectable hepatocellular carcinoma (uHCC)
Tecentriq and Avastin
TECENTRIQ, in combination with bevacizumab, for the treatment of patients with unresectable or metastatic hepatocellular carcinoma who have not received prior systemic therapy.
LENVIMA
LENVIMA is indicated for the first-line treatment of patients with unresectable hepatocellular carcinoma (HCC)
Stivarga
Treatment of patients with hepatocellular carcinoma (HCC or liver cancer) who have been previously treated with sorafenib.
Treatment Centers
8 centersBaylor College of Medicine Rare Disease Center ↗
Baylor College of Medicine
📍 Houston, TX
🏥 NORDStanford Medicine Rare Disease Center ↗
Stanford Medicine
📍 Stanford, CA
🔬 UDNNIH Clinical Center Undiagnosed Diseases Program ↗
National Institutes of Health
📍 Bethesda, MD
🔬 UDNUCLA UDN Clinical Site ↗
UCLA Health
📍 Los Angeles, CA
🔬 UDNBaylor College of Medicine UDN Clinical Site ↗
Baylor College of Medicine
📍 Houston, TX
🔬 UDNHarvard/MGH UDN Clinical Site ↗
Massachusetts General Hospital
📍 Boston, MA
🏥 NORDMayo Clinic Center for Individualized Medicine ↗
Mayo Clinic
📍 Rochester, MN
👤 Mayo Clinic Center for Individualized Medicine
🏥 NORDUCLA Rare Disease Day Program ↗
UCLA Health
📍 Los Angeles, CA
Financial Resources
3 resourcesTravel Grants
No travel grants are currently matched to Adult hepatocellular carcinoma.
Community
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1 articlesCaregiver Resources
NORD Caregiver Resources
Support, advocacy, and financial assistance for caregivers of rare disease patients.
Mental Health Support
Rare disease caregiving can be isolating. Connect with counseling and peer support.
Family & Caregiver Grants
Financial assistance programs specifically for caregivers of rare disease patients.
Social Security Disability
Learn how rare disease patients may qualify for SSDI/SSI benefits.
Common questions about Adult hepatocellular carcinoma
What is Adult hepatocellular carcinoma?
Adult hepatocellular carcinoma (HCC), also known as hepatoma, is the most common primary malignant tumor of the liver occurring in adults. It arises from hepatocytes, the main functional cells of the liver, and predominantly develops in the setting of chronic liver disease and cirrhosis. Major risk factors include chronic hepatitis B virus (HBV) and hepatitis C virus (HCV) infection, alcohol-related liver disease, non-alcoholic steatohepatitis (NASH), aflatoxin exposure, and metabolic conditions such as hereditary hemochromatosis and alpha-1 antitrypsin deficiency. The disease primarily affect
How is Adult hepatocellular carcinoma inherited?
Adult hepatocellular carcinoma follows a multifactorial inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.
At what age does Adult hepatocellular carcinoma typically begin?
Typical onset of Adult hepatocellular carcinoma is adult. Age of onset can vary across affected individuals.
Are there clinical trials for Adult hepatocellular carcinoma?
Yes — 20 recruiting clinical trials are currently listed for Adult hepatocellular carcinoma on UniteRare. See the clinical trials section on this page for phase, sponsor, and site details sourced from ClinicalTrials.gov.
Which specialists treat Adult hepatocellular carcinoma?
25 specialists and care centers treating Adult hepatocellular carcinoma are listed on UniteRare, sourced from ClinicalTrials.gov principal investigators, published research, and the NPPES NPI registry.
What treatment and support options exist for Adult hepatocellular carcinoma?
5 patient support programs are currently tracked on UniteRare for Adult hepatocellular carcinoma. See the treatments and support programs sections for copay assistance, eligibility, and contact details.