Fibrolamellar hepatocellular carcinoma

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7Active trials31Specialists8Treatment centers

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

Fibrolamellar hepatocellular carcinoma (often called fibrolamellar carcinoma or FLC) is a rare type of liver cancer that mainly affects young people, typically adolescents and young adults. Unlike the more common form of liver cancer (hepatocellular carcinoma or HCC), fibrolamellar carcinoma usually develops in people who have healthy livers with no history of liver disease, cirrhosis, or hepatitis infection. This makes it quite different from typical liver cancer. The tumor gets its name from the way it looks under a microscope — the cancer cells are surrounded by bands (lamellae) of fibrous tissue. The disease often grows slowly at first and may not cause symptoms until the tumor becomes large. Common symptoms include a mass or pain in the upper right part of the abdomen, unintended weight loss, fatigue, and sometimes jaundice (yellowing of the skin and eyes). Because symptoms can be vague, the cancer is sometimes found at an advanced stage. The main treatment for fibrolamellar carcinoma is surgery to remove the tumor, which offers the best chance for long-term survival. When the cancer is caught early and can be completely removed, outcomes are more favorable. Unfortunately, standard chemotherapy drugs used for typical liver cancer have shown limited effectiveness against fibrolamellar carcinoma. Liver transplantation may be considered in select cases. Research is ongoing to find targeted therapies that work specifically against this cancer, and clinical trials are actively exploring new treatment options.

Also known as:

Key symptoms:

Abdominal pain, especially in the upper right areaA lump or mass that can be felt in the abdomenUnintended weight lossLoss of appetiteFatigue and feeling tiredJaundice (yellowing of the skin and eyes)Nausea or vomitingFeeling full quickly after eatingEnlarged liverShoulder pain (referred pain from the liver)Swollen abdomen from fluid buildup (ascites)Fever of unknown cause

Inheritance

Sporadic

Usually appears on its own, not inherited from a parent

Age of Onset

Juvenile

Begins in the teen years

Orphanet ↗NORD ↗

FDA & Trial Timeline

8 events
May 2026A DNAJB1-PRKACA Fusion Kinase Peptide Vaccine Combined With Glutamine Antagonist DRP-104, Nivolumab, and Ipilimumab in Patients With Advanced Stage Fibrolamellar Carcinoma (FLC)

Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins — PHASE1

TrialNOT YET RECRUITING
Jul 2025Peptide Vaccine for Fibrolamellar Hepatocellular Carcinoma Patients and Other Tumor Entities Carrying the Driver Fusion DNAJB1-PRKACA

University Hospital Tuebingen — PHASE1

TrialRECRUITING
Jun 2025Testing the Addition of an Anti-cancer Drug, DT2216, to the Usual Chemotherapy Treatment for Relapsed or Refractory Solid Tumors and Fibrolamellar Carcinoma

Children's Oncology Group — PHASE1, PHASE2

TrialRECRUITING
Feb 2024DRP-104 (Glutamine Antagonist) in Combination With Durvalumab in Patients With Advanced Stage Fibrolamellar Carcinoma (FLC)

Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins — PHASE1, PHASE2

TrialRECRUITING
Sep 2023FusionVAC22_01: Fusion Transcript-based Peptide Vaccine Combined with Immune Checkpoint Inhibition

University Hospital Tuebingen — PHASE1

TrialRECRUITING
Nov 2022Safety and Efficacy of Cyclophosphamide, Sorafenib, Bevacizumab, and Atezolizumab in Pediatric Solid Tumor Patients

St. Jude Children's Research Hospital — PHASE1, PHASE2

TrialRECRUITING
Jan 2021Nivolumab, Fluorouracil, and Interferon Alpha 2B for the Treatment of Unresectable Fibrolamellar Cancer

M.D. Anderson Cancer Center — PHASE1, PHASE2

TrialRECRUITING
Nov 2020Checkpoint Inhibition In Pediatric Hepatocellular Carcinoma

Allison O'Neill, MD — PHASE2

TrialRECRUITING

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

Treatments

No FDA-approved treatments are currently listed for Fibrolamellar hepatocellular carcinoma.

7 clinical trialsare actively recruiting — trials can provide access to cutting-edge therapies.

View clinical trials →

Clinical Trials

7 recruitingView all trials with filters →
Phase 21 trial
Checkpoint Inhibition In Pediatric Hepatocellular Carcinoma
Phase 2
Actively Recruiting
PI: Allison O'Neill, MD (Dana-Farber Cancer Institute) · Sites: San Francisco, California; Boston, Massachusetts +3 more · Age: 030 yrs
Phase 12 trials
Peptide Vaccine for Fibrolamellar Hepatocellular Carcinoma Patients and Other Tumor Entities Carrying the Driver Fusion DNAJB1-PRKACA
Phase 1
Actively Recruiting
· Sites: Tübingen; Tübingen · Age: 1299 yrs
FusionVAC22_01: Fusion Transcript-based Peptide Vaccine Combined with Immune Checkpoint Inhibition
Phase 1
Actively Recruiting
PI: Salih, Prof. Dr. (CCU Translational Immunology) · Sites: Tübingen, Baden-Würtemberg · Age: 1899 yrs

Specialists

Showing 25 of 31View all specialists →
GL
Gadi Lalazar
NEW YORK, NY
Specialist
2 Fibrolamellar hepatocellular carcinoma publications
SS
Sanford M Simon
Specialist
4 Fibrolamellar hepatocellular carcinoma publications
PC
Philip Coffino
SAN FRANCISCO, CA
Specialist
3 Fibrolamellar hepatocellular carcinoma publications
DR
David Requena
Specialist
2 Fibrolamellar hepatocellular carcinoma publications
MS
Mahsa Shirani
Specialist
2 Fibrolamellar hepatocellular carcinoma publications
MT
Michael S Torbenson
ROCHESTER, MN
Specialist
2 Fibrolamellar hepatocellular carcinoma publications
ZB
Zachary J Brown
Specialist
2 Fibrolamellar hepatocellular carcinoma publications
TP
Timothy M Pawlik
COLUMBUS, OH
Specialist
2 Fibrolamellar hepatocellular carcinoma publications
BS
Bassem Shebl
Specialist
2 Fibrolamellar hepatocellular carcinoma publications
TF
Tova M Finkelstein
Specialist
2 Fibrolamellar hepatocellular carcinoma publications
DN
Denise Ng
SAN FRANCISCO, CA
Specialist
2 Fibrolamellar hepatocellular carcinoma publications
YJ
Ype P de Jong
Specialist
2 Fibrolamellar hepatocellular carcinoma publications
CR
Charles M Rice
Specialist
2 Fibrolamellar hepatocellular carcinoma publications
YS
Yasuyuki Shigematsu
Specialist
1 Fibrolamellar hepatocellular carcinoma publication
KT
Kazuhito Tanaka
Specialist
1 Fibrolamellar hepatocellular carcinoma publication
MM
Mark Yarchoan, MD
BALTIMORE, MD
Specialist
PI on 2 active trials
MM
Marina Baretti, MD
BALTIMORE, MD
Specialist
PI on 2 active trials
GT
Gregory M Tiao
PALO ALTO, CA
Specialist
PI on 1 active trial7 Fibrolamellar hepatocellular carcinoma publications
MO
Michael V Ortiz
Specialist
PI on 2 active trials18 Fibrolamellar hepatocellular carcinoma publications
CD
Camila MV Moniz, Doctor
SAN ANTONIO, TX
Specialist
PI on 1 active trial
AM
Allison O'Neill, MD
Specialist
PI on 3 active trials1 Fibrolamellar hepatocellular carcinoma publication

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

Travel Grants

No travel grants are currently matched to Fibrolamellar hepatocellular carcinoma.

Search all travel grants →NORD Financial Assistance ↗

Community

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Latest news about Fibrolamellar hepatocellular carcinoma

Disease timeline:

New recruiting trial: Peptide Vaccine for Fibrolamellar Hepatocellular Carcinoma Patients and Other Tumor Entities Carrying the Driver Fusion DNAJB1-PRKACA

A new clinical trial is recruiting patients for Fibrolamellar hepatocellular carcinoma

New recruiting trial: Safety and Efficacy of Cyclophosphamide, Sorafenib, Bevacizumab, and Atezolizumab in Pediatric Solid Tumor Patients

A new clinical trial is recruiting patients for Fibrolamellar hepatocellular carcinoma

New recruiting trial: Nivolumab, Fluorouracil, and Interferon Alpha 2B for the Treatment of Unresectable Fibrolamellar Cancer

A new clinical trial is recruiting patients for Fibrolamellar hepatocellular carcinoma

New recruiting trial: FusionVAC22_01: Fusion Transcript-based Peptide Vaccine Combined with Immune Checkpoint Inhibition

A new clinical trial is recruiting patients for Fibrolamellar hepatocellular carcinoma

New recruiting trial: DRP-104 (Glutamine Antagonist) in Combination With Durvalumab in Patients With Advanced Stage Fibrolamellar Carcinoma (FLC)

A new clinical trial is recruiting patients for Fibrolamellar hepatocellular carcinoma

New recruiting trial: Checkpoint Inhibition In Pediatric Hepatocellular Carcinoma

A new clinical trial is recruiting patients for Fibrolamellar 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.

Questions for your doctor

Bring these to your next appointment

  • Q1.Can my tumor be completely removed with surgery, and what are the risks of the operation?,Has the cancer spread beyond the liver, and how does that affect my treatment options?,Was the DNAJB1-PRKACA fusion gene found in my tumor, and what does that mean for my treatment?,Are there any clinical trials available for fibrolamellar carcinoma that I might be eligible for?,What is the plan for monitoring me after treatment to catch any recurrence early?,Should I be seen at a specialized cancer center with experience treating fibrolamellar carcinoma?,What support services are available to help me cope with the emotional and practical challenges of this diagnosis?

Common questions about Fibrolamellar hepatocellular carcinoma

What is Fibrolamellar hepatocellular carcinoma?

Fibrolamellar hepatocellular carcinoma (often called fibrolamellar carcinoma or FLC) is a rare type of liver cancer that mainly affects young people, typically adolescents and young adults. Unlike the more common form of liver cancer (hepatocellular carcinoma or HCC), fibrolamellar carcinoma usually develops in people who have healthy livers with no history of liver disease, cirrhosis, or hepatitis infection. This makes it quite different from typical liver cancer. The tumor gets its name from the way it looks under a microscope — the cancer cells are surrounded by bands (lamellae) of fibrous

How is Fibrolamellar hepatocellular carcinoma inherited?

Fibrolamellar hepatocellular carcinoma follows a sporadic inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

At what age does Fibrolamellar hepatocellular carcinoma typically begin?

Typical onset of Fibrolamellar hepatocellular carcinoma is juvenile. Age of onset can vary across affected individuals.

Are there clinical trials for Fibrolamellar hepatocellular carcinoma?

Yes — 7 recruiting clinical trials are currently listed for Fibrolamellar 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 Fibrolamellar hepatocellular carcinoma?

25 specialists and care centers treating Fibrolamellar hepatocellular carcinoma are listed on UniteRare, sourced from ClinicalTrials.gov principal investigators, published research, and the NPPES NPI registry.