Adult hepatocellular carcinoma

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ORPHA:210159OMIM:114550C22.0
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6FDA treatments145Active trials346Specialists8Treatment centers3Financial resources

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UniteRare data is sourced from FDA.gov, ClinicalTrials.gov, Orphanet, OMIM, and NORD.
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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:

Inheritance

Multifactorial

Caused by a mix of several genes and environmental factors

Age of Onset

Adult

Begins in adulthood (age 18 or older)

Orphanet ↗OMIM ↗NORD ↗

FDA & Trial Timeline

10 events
Apr 2026Cytokine Armored GPC3 Specific Chimeric Antigen Receptor Expressing T-cells in Adults With Solid Tumors

Seattle Children's Hospital — PHASE1

TrialNOT YET RECRUITING
Apr 2026Safety and Efficacy of LVD + C-TACE + Tis/Len for Unresectable Right-Liver HCC

Hong Wu — NA

TrialNOT YET RECRUITING
Apr 2026Hepzato Kit and Opdualag for Metastatic Melanoma and Liver Metastasis

University of Wisconsin, Madison — PHASE1, PHASE2

TrialRECRUITING
Apr 2026Evaluation of RBS2418 in Combination With Tremelimumab Plus Durvalumab in Participants With Advanced Unresectable Hepatocellular Carcinoma

Riboscience, LLC. — PHASE2

TrialRECRUITING
Mar 2026A Phase I Dose Escalation and Dose Expansion Study to Investigate the Pharmacokinetics and Safety of Subcutaneous Durvalumab

AstraZeneca — PHASE1

TrialNOT YET RECRUITING
Mar 2026A Prospective Study to Evaluate the Safety and Efficacy of the Combination Therapy of Irpagratinib, Atezolizumab, and Bevacizumab in Patients With Hepatocellular Carcinoma

Asan Medical Center — PHASE2

TrialRECRUITING
Mar 2026A Study to Evaluate the Safety and Tolerability of Pumitamig Alone or In Combination With Ipilimumab in Participants With First-Line Advanced or Unresectable Hepatocellular Carcinoma (HCC) (ROSETTA HCC-206)

Bristol-Myers Squibb — PHASE1, PHASE2

TrialRECRUITING
Mar 2026Dual-Target GPC3/B7-H3 CAR-NK Cells for Advanced HCC

Beijing Biotech — PHASE1, PHASE2

TrialRECRUITING
Mar 2026A Prospective, Randomized Study of TACE Combined With Sintilimab, Bevacizumab, and Ipilimumab N01 Treating Advanced HCC

Second Affiliated Hospital, School of Medicine, Zhejiang University — PHASE2

TrialNOT YET RECRUITING
Mar 2026Liver-directed Chemotherapy After Surgery of Liver Metastases of Colorectal Cancer in Patients With High Risk of Recurrence of Their Disease

UNICANCER — PHASE3

TrialNOT YET RECRUITING

Data sourced from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.

Treatments

6 available

Nexavar

sorafenib· Bayer HealthCare Pharmaceuticals, Inc.

NEXAVAR is indicated for the treatment of patients with unresectable hepatocellular carcinoma (HCC)

IMFINZI

durvalumab· AstraZeneca Pharmaceuticals LPOrphan Drug

In combination with tremelimumab-actl for the treatment of adult patients with unresectable hepatocellular carcinoma (uHCC)

Imjudo

tremelimumab-actl· AstraZeneca Pharmaceuticals LPOrphan Drug

In combination with durvalumab for the treatment of adult patients with unresectable hepatocellular carcinoma (uHCC)

Tecentriq and Avastin

atezolizumab + bevacizumab· Genentech, Inc.Orphan Drug

TECENTRIQ, in combination with bevacizumab, for the treatment of patients with unresectable or metastatic hepatocellular carcinoma who have not received prior systemic therapy.

LENVIMA

lenvatinib· Eisai Inc.Orphan Drug

LENVIMA is indicated for the first-line treatment of patients with unresectable hepatocellular carcinoma (HCC)

Stivarga

regorafenib· Bayer HealthCare Pharmaceuticals, Inc.■ Boxed WarningOrphan Drug

Treatment of patients with hepatocellular carcinoma (HCC or liver cancer) who have been previously treated with sorafenib.

Clinical Trials

20 recruitingView all trials with filters →
Other20 trials
Dissection of the Molecular and Immunological Interaction in Patients With HCC of Viral and Non-viral Etiologies
Active
PI: Tsang-Wu Liu (Taiwan Cooperative Oncology Group, NHRI) · Sites: Tainan; Taipei +2 more · Age: 2099 yrs
AI-Driven Quantitative Decision and Surgical Planning System for Liver Cancer
Enrolling by Invitation
PI: Shuo Jin, PhD (Beijing Tsinghua Chang Gung Hospital) · Sites: Beijing, Beijing Municipality · Age: 1899 yrs
The Detection of Small Early Liver Cancer With Natural History Follow up
Actively Recruiting
PI: Ellie Barnes (University of Oxford) · Sites: Oxford, Oxfordshire · Age: 18100 yrs
PET TDM FDG-Choline as a Decision-making Tool for Routine Care on the Liver Transplant List for HCC
Actively Recruiting
PI: Guillaume Lassailly, MD (University Hospital, Lille) · Sites: Lille · Age: 1899 yrs
Chronic Hepatitis B Virus Infection in Zambia
Active
PI: Michael J Vinikoor, MD (University of Alabama at Birmingham) · Sites: Lusaka · Age: 1899 yrs
Value of Negative 18FDG PET/CT in Milan Out, Up-to-seven in, HCC Transplant Candidates
Actively Recruiting
· Sites: Antwerp; Brussels +4 more · Age: 1880 yrs
Molecular Basis of Pediatric Liver Cancer
Actively Recruiting
· Sites: Pittsburgh, Pennsylvania · Age: 099 yrs
Triple Therapy for Intermediate-advanced HCC With BDTT (TALENP002)
Actively Recruiting
· Sites: Fuzhou, Fujian; Fuzhou, Fujian +4 more · Age: 1875 yrs
The Efficacy and Safety of EOB-MRI Guided Microwave Ablation for Early HCC: A Multicenter, Prospective, Observational Study
Active
· Sites: Jinan · Age: 1899 yrs
Liver Function Investigation With SPECT
Active
· Sites: St Leonards, New South Wales · Age: 18100 yrs
On-treatment Surveillance of Tumor Evolution and Response to Systemic Treatment in Bile Duct and Liver Cancer
Actively Recruiting
PI: Karin Jirström, PhD, MD, professor (Region Skåne) · Sites: Kristianstad; Lund +1 more · Age: 1899 yrs
The Overall Survival of Patients With Unresectable HCC in Real-life
Actively Recruiting
PI: Masao Omata, PhD, MD (Tokyo University) · Sites: Beijing; Hong Kong +1 more · Age: 1899 yrs
Long-Term Survival With Lenvatinib-Based Therapy in Unresectable Liver Cancer
Active
· Sites: Tianjin · Age: 1899 yrs
Precision Medicine for Liver Tumours With Quantitative Magnetic Resonance Imaging and Whole Genome Sequencing
Active
PI: Rajarshi Banerjee (Perspectum Ltd) · Sites: Basingstoke, Hampshire · Age: 1899 yrs
Development of 3D Multi-Parametric Ultrasound (MPUS) as a Decision Support Tool for Patients
Actively Recruiting
PI: Ahmed El Kaffas, PhD (University of California, San Diego) · Sites: Palo Alto, California; San Diego, California +1 more · Age: 1899 yrs
An Observational Study to Learn More About How Well a Treatment Works When Given After Treatment With Atezolizumab and Bevacizumab or Another Similar Combination of Drugs in Adults With Liver Cancer That Cannot be Treated With Surgery
Actively Recruiting
· Sites: Baltimore, Maryland; Ann Arbor, Michigan +18 more · Age: 1899 yrs
Characterizing Disease Biology, Treatment and Toxicity in Older Adults With Hepatocellular Carcinoma
Actively Recruiting
PI: Sukeshi Patel Arora, MD (Mays Cancer Center) · Sites: San Antonio, Texas · Age: 1899 yrs
A Multicenter Prospective Study on the Performance of Spectral CT for Evaluating Treatment Response After TACE in Hepatocellular Carcinoma
Actively Recruiting
· Sites: Nanjing · Age: 1875 yrs
Immunophenotype of PBMC to Early Predict Response to Immunotherapy in Hepatocellular Carcinoma
Actively Recruiting
PI: Laura Gramantieri (IRCCS Azienda Ospedaliero-Universitaria di Bologna) · Sites: Bologna, Laura Gramantieri · Age: 1899 yrs
Liquid Biopsy for Hepatocellular Carcinoma Molecular Characterization After Y90
Actively Recruiting
PI: Sirish Kishore, MD (VA Palo Alto Health Care System) · Sites: Palo Alto, California · Age: 1899 yrs

Specialists

Showing 25 of 346View all specialists →
KM
Kannan Thanikachalam, MD
BUFFALO, NY
Specialist
PI on 1 active trial
SM
Susan Perez, MD
Specialist
PI on 1 active trial
JF
Jia Fan
Specialist
PI on 6 active trials3 Adult hepatocellular carcinoma publications
JZ
Jian Zhou
Specialist
PI on 6 active trials3 Adult hepatocellular carcinoma publications
CP
Chao Liu, PHD
Specialist
PI on 9 active trials
MM
Mark Yarchoan, MD
BALTIMORE, MD
Specialist
PI on 2 active trials
HK
Howard Katzenstein
JACKSONVILLE, FL
Specialist
PI on 2 active trials4 Adult hepatocellular carcinoma publications
SP
Stanislaw R. Burzynski, MD, PhD
HOUSTON, TX
Specialist
PI on 29 active trials
ZP
Zhenfeng Zhang, MD, PhD
Specialist
PI on 4 active trials
SP
Shuo Jin, PhD
AUSTIN, TX
Specialist
PI on 5 active trials
AM
Andrew N. de la Torre, MD
AUSTIN, TX
Specialist
PI on 2 active trials1 Adult hepatocellular carcinoma publication
TP
Tim Meyer, MD, BSc, MRCP, PhD
Specialist
PI on 2 active trials
UM
Udo Rudloff, M.D.
BETHESDA, MD
Specialist
PI on 4 active trials
JM
Jonathan Strosberg, MD
TAMPA, FL
Specialist
PI on 2 active trials

Treatment Centers

8 centers
🏥 NORD

Baylor College of Medicine Rare Disease Center

Baylor College of Medicine

📍 Houston, TX

🏥 NORD

Stanford Medicine Rare Disease Center

Stanford Medicine

📍 Stanford, CA

🔬 UDN

NIH Clinical Center Undiagnosed Diseases Program

National Institutes of Health

📍 Bethesda, MD

🔬 UDN

UCLA UDN Clinical Site

UCLA Health

📍 Los Angeles, CA

🔬 UDN

Baylor College of Medicine UDN Clinical Site

Baylor College of Medicine

📍 Houston, TX

🔬 UDN

Harvard/MGH UDN Clinical Site

Massachusetts General Hospital

📍 Boston, MA

🏥 NORD

Mayo Clinic Center for Individualized Medicine

Mayo Clinic

📍 Rochester, MN

👤 Mayo Clinic Center for Individualized Medicine

🏥 NORD

UCLA Rare Disease Day Program

UCLA Health

📍 Los Angeles, CA

Financial Resources

3 resources
IMFINZI(durvalumab)AstraZeneca Pharmaceuticals LP
Imjudo(tremelimumab-actl)AstraZeneca Pharmaceuticals LP
Tecentriq and Avastin(atezolizumab + bevacizumab)Genentech, Inc.

Travel Grants

No travel grants are currently matched to Adult hepatocellular carcinoma.

Search all travel grants →NORD Financial Assistance ↗

Community

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Latest news about Adult hepatocellular carcinoma

1 articles
Clinical trialCLINICALTRIALSApr 24, 2026
New Clinical Trial: Attention and Interpretation Modification (AIM) for Fear of Cancer Recurrence: An Intervention Development Study (NCT01517945)
Researchers at Memorial Sloan Kettering Cancer Center are testing a smartphone app designed to help breast cancer and rare cancer survivors deal with the fear t
See all news about Adult hepatocellular carcinoma

Caregiver 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.