Hepatocellular adenoma

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ORPHA:54272D13.4
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1Active trials18Specialists8Treatment centers

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

Hepatocellular adenoma (HCA), also known as hepatic adenoma or liver cell adenoma, is a rare benign tumor of the liver arising from hepatocytes. It predominantly affects women of reproductive age and is strongly associated with the use of oral contraceptive pills (OCPs) and other estrogen-containing hormonal therapies. Other risk factors include anabolic steroid use, glycogen storage diseases (particularly types I and III), obesity, and metabolic syndrome. The tumor primarily affects the liver and can range from a single lesion to multiple adenomas; when ten or more lesions are present, the condition is termed hepatic adenomatosis. Hepatocellular adenomas are classified into several molecular subtypes based on their genetic and pathological features: HNF1A-inactivated (H-HCA), inflammatory (I-HCA), beta-catenin-activated (b-HCA), and unclassified. This subtyping has important clinical implications, as beta-catenin-activated adenomas carry a higher risk of malignant transformation to hepatocellular carcinoma, while HNF1A-inactivated adenomas are generally considered lower risk. Many patients are asymptomatic, with the adenoma discovered incidentally on imaging. However, key symptoms can include right upper quadrant abdominal pain, a palpable abdominal mass, and in some cases, life-threatening intra-abdominal hemorrhage due to spontaneous rupture of the tumor. Management of hepatocellular adenoma depends on size, subtype, symptoms, and risk of complications. The first-line approach includes discontinuation of oral contraceptives or other hormonal agents, which may lead to tumor regression. Surgical resection is generally recommended for adenomas larger than 5 cm due to the increased risk of hemorrhage and malignant transformation, and for all beta-catenin-activated subtypes regardless of size. For smaller, stable lesions, surveillance with periodic imaging (MRI) is often employed. In cases of hepatic adenomatosis or recurrent disease, liver transplantation may rarely be considered. Embolization may be used in acute hemorrhagic settings. Regular follow-up is essential to monitor for growth or malignant transformation.

Inheritance

Sporadic

Usually appears on its own, not inherited from a parent

Age of Onset

Adult

Begins in adulthood (age 18 or older)

Orphanet ↗NORD ↗

FDA & Trial Timeline

1 event
Oct 2024A Phase 2 Open-label Single-arm Trial of JAK1 Inhibitor for the Treatment of Large Inflammatory Hepatocellular Adenomas

Assistance Publique - Hôpitaux de Paris — PHASE2

TrialRECRUITING

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

Treatments

No FDA-approved treatments are currently listed for Hepatocellular adenoma.

1 clinical trialare actively recruiting — trials can provide access to cutting-edge therapies.

View clinical trials →

Clinical Trials

1 recruitingView all trials with filters →
Phase 21 trial
A Phase 2 Open-label Single-arm Trial of JAK1 Inhibitor for the Treatment of Large Inflammatory Hepatocellular Adenomas
Phase 2
Actively Recruiting
PI: Jean-Charles NAULT, PUPH (Assistance Publique des Hôpitaux de Paris (APHP)) · Sites: Bobigny · Age: 1865 yrs

Specialists

18 foundView all specialists →
JP
Jean-Charles NAULT, PUPH
Specialist
PI on 1 active trial
CD
Camila MV Moniz, Doctor
SAN ANTONIO, TX
Specialist
PI on 1 active trial
SM
Shivaani Kummar, MD
PORTLAND, OR
Specialist
PI on 5 active trials
HP
Henry Pitot
ROCHESTER, MN
Specialist
PI on 2 active trials2 Hepatocellular adenoma publications
AM
Antonius Miller
Specialist
PI on 2 active trials79 Hepatocellular adenoma publications
EM
Esther Mena Gonzalez, M.D.
Specialist
PI on 1 active trial
TK
Tal Hetzroni Kedem
Specialist
PI on 1 active trial
JM
John Janik, MD
AUGUSTA, GA
Specialist
PI on 2 active trials
DF
D Mirza, MBBS, MS, FRCS
Specialist
PI on 1 active trial

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 Hepatocellular adenoma.

Search all travel grants →NORD Financial Assistance ↗

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Latest news about Hepatocellular adenoma

Disease timeline:

New recruiting trial: A Phase 2 Open-label Single-arm Trial of JAK1 Inhibitor for the Treatment of Large Inflammatory Hepatocellular Adenomas

A new clinical trial is recruiting patients for Hepatocellular adenoma

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 Hepatocellular adenoma

What is Hepatocellular adenoma?

Hepatocellular adenoma (HCA), also known as hepatic adenoma or liver cell adenoma, is a rare benign tumor of the liver arising from hepatocytes. It predominantly affects women of reproductive age and is strongly associated with the use of oral contraceptive pills (OCPs) and other estrogen-containing hormonal therapies. Other risk factors include anabolic steroid use, glycogen storage diseases (particularly types I and III), obesity, and metabolic syndrome. The tumor primarily affects the liver and can range from a single lesion to multiple adenomas; when ten or more lesions are present, the co

How is Hepatocellular adenoma inherited?

Hepatocellular adenoma follows a sporadic inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

At what age does Hepatocellular adenoma typically begin?

Typical onset of Hepatocellular adenoma is adult. Age of onset can vary across affected individuals.

Are there clinical trials for Hepatocellular adenoma?

Yes — 1 recruiting clinical trial is currently listed for Hepatocellular adenoma on UniteRare. See the clinical trials section on this page for phase, sponsor, and site details sourced from ClinicalTrials.gov.

Which specialists treat Hepatocellular adenoma?

18 specialists and care centers treating Hepatocellular adenoma are listed on UniteRare, sourced from ClinicalTrials.gov principal investigators, published research, and the NPPES NPI registry.