Overview
Pediatric hepatocellular carcinoma (also called childhood liver cancer or pediatric HCC) is a rare but serious cancer that starts in the main cells of the liver, called hepatocytes. It is the most common type of primary liver cancer in children and teenagers, though it is still much rarer in children than in adults. The liver is a vital organ that filters the blood, helps digest food, and removes toxins from the body. When cancer develops in the liver, it can interfere with all of these important functions. Children with pediatric HCC often have an underlying liver condition that has been present since birth or early childhood, such as hepatitis B infection, certain metabolic liver diseases, or cirrhosis (scarring of the liver). Common symptoms include a swollen or painful belly, unexplained weight loss, nausea, yellowing of the skin or eyes (jaundice), and extreme tiredness. Some children have no symptoms early on, which can make the cancer harder to catch. Treatment usually involves a combination of surgery to remove the tumor, chemotherapy drugs, and sometimes a liver transplant. The outlook depends heavily on how early the cancer is found and whether it can be fully removed. When caught early and treated aggressively, some children can be cured. Research into new treatments, including targeted therapies and immunotherapy, is ongoing and offering new hope for families.
Also known as:
Key symptoms:
Swollen or bloated bellyA lump or mass that can be felt in the upper right side of the abdomenBelly pain or discomfortUnexplained weight lossLoss of appetiteNausea and vomitingExtreme tiredness or fatigueYellowing of the skin or whites of the eyes (jaundice)Pale or clay-colored stoolsDark urineFever without a clear causeSlowed growth or failure to thrive in younger children
Clinical phenotype terms (9)— hover any for plain English
Variable
Can be inherited in different ways depending on the underlying gene
Childhood
Begins in childhood, roughly ages 1 to 12
FDA & Trial Timeline
10 eventsWest China Hospital
Medical University of Warsaw — NA
Baylor College of Medicine — PHASE1
Zhongda Hospital
National Taiwan University Hospital — NA
Beijing Hospital — NA
Sotio Biotech Inc. — PHASE1, PHASE2
University of Colorado, Denver
Baylor College of Medicine — PHASE1
University Medical Center Groningen — PHASE2
Data sourced from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.
Treatments
3 availableTecentriq 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
Lipiodol
Selective hepatic intra-arterial use for imaging tumors in adults with known hepatocellular carcinoma (HCC)
Nexavar
Treatment of unresectable hepatocellular carcinoma
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
2 resourcesYervoy
Bristol-Myers Squibb Company
Yervoy Patient Support (BMS Access Support)
Travel Grants
No travel grants are currently matched to Pediatric hepatocellular carcinoma.
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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.
Questions for your doctor
Bring these to your next appointment
- Q1.What stage is my child's cancer, and what does that mean for treatment options?,Is surgery or a liver transplant possible for my child, and what are the chances of a cure?,Are there clinical trials available that my child might qualify for?,Does my child have an underlying genetic or liver condition that caused this cancer, and should other family members be tested?,What are the short-term and long-term side effects of the chemotherapy drugs being recommended?,How will we monitor whether the treatment is working, and what happens if it stops working?,What support services — such as nutrition counseling, mental health support, or financial assistance — are available to our family?
Common questions about Pediatric hepatocellular carcinoma
What is Pediatric hepatocellular carcinoma?
Pediatric hepatocellular carcinoma (also called childhood liver cancer or pediatric HCC) is a rare but serious cancer that starts in the main cells of the liver, called hepatocytes. It is the most common type of primary liver cancer in children and teenagers, though it is still much rarer in children than in adults. The liver is a vital organ that filters the blood, helps digest food, and removes toxins from the body. When cancer develops in the liver, it can interfere with all of these important functions. Children with pediatric HCC often have an underlying liver condition that has been pre
At what age does Pediatric hepatocellular carcinoma typically begin?
Typical onset of Pediatric hepatocellular carcinoma is childhood. Age of onset can vary across affected individuals.
Are there clinical trials for Pediatric hepatocellular carcinoma?
Yes — 12 recruiting clinical trials are currently listed for Pediatric 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 Pediatric hepatocellular carcinoma?
25 specialists and care centers treating Pediatric 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 Pediatric hepatocellular carcinoma?
2 patient support programs are currently tracked on UniteRare for Pediatric hepatocellular carcinoma. See the treatments and support programs sections for copay assistance, eligibility, and contact details.