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hepatoblastoma (HBL) - Clinical Profile, Genes & Research | Kisho | Kisho

Rare Disease

hepatoblastoma

MONDO:0018666

Also known as:HBLhepatoblastoma, malignantpaediatric embryonal hepatoma

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Overview

Hepatoblastoma (HB) is a malignant hepatic tumor and is the most common pediatric liver cancer. It is characterized by anorexia, weight loss and an enlarged abdomen. HB is more common in patients with familial adenomatous polyposis (FAP), and can occur in patients with other pre-existing liver conditions. About 5% of HB cases are associated with genetic factors, especially overgrowth syndromes, such as Beckwith-Wiedemann syndrome (BWS) or hemihypertrophy.

TreatmentsNo approved treatments · 2 in pipeline

Clinical Trials31 registered · 12 recruiting

Diagnosis

Biomarker and diagnostic research for hepatoblastoma has been reported in the published literature.

Treatment

Compiled from FDA-approved drug records, orphan drug designations, and FDA Adverse Event Reporting System (FAERS) safety data. GeneReviews management guidance is included where available.

No approved treatments are currently available for hepatoblastoma. An additional 2 compounds hold orphan drug designation.

Orphan Drug Designations

While no drugs are FDA-approved specifically for hepatoblastoma, some of the following designated compounds may be used off-label in clinical practice. Treatment decisions should be made in consultation with a specialist familiar with this condition.

The following drugs have received orphan drug designation from the FDA for hepatoblastoma. Orphan designation reflects regulatory interest and does not indicate approval for treatment.

Brand Name	Generic Name	Sponsor

Research Landscape

Sourced from ClinicalTrials.gov and PubMed. Publication types are classified using NLM-curated article metadata; research type breakdowns reflect the nature of published work for this disease.

31 clinical trials registered, 12 recruiting. Interventions under study include drug therapy, other interventions, procedural interventions, and biologic therapy. Pipeline includes 4 PHASE3, 11 PHASE2, 11 PHASE1. Research is sponsored by a mix of industry and academic institutions.

NCT ID	Title	Phase	Sponsor	Status
[NCT03959800](https://clinicaltrials.gov/study/NCT03959800)

Data assembled from 5 of 12 sources · Last updated Sep 18, 2026, 11:26 AM UTC

MONDOHPOClinicalTrials.govFDAPubMedHGNCClinVarClinGenGeneReviewsOrphanetOMIMRUSP

Medical References

External Resources

Orphanet

European rare disease database

Orphanet:449

GARD

Genetic and Rare Diseases Info Center

GARD:2657

Questions for Your Care Team

→Are any of the 31 active clinical trials a good fit for my situation?
→What is the current development status of N-hydroxy-N-(methylacylfulvene)urea, and when might it become available?
→Are there specialists or centers of excellence for this condition in my area?
→Is there a patient registry I should consider joining?

← Back to All Diseases

Research Type	Count	% of Total
Laboratory research	27	36%
Research summaries	24	32%
Disease patterns and progression	8	11%
Testing and diagnosis research	6	8%
New treatment approaches	5	7%
Patient case studies	2	3%
Clinical study results	2	3%

hepatoblastoma

Overview

Diagnosis

Treatment

Orphan Drug Designations

Research Landscape

Medical References

External Resources

Orphanet

GARD

Subtypes

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Questions for Your Care Team

Questions for Your Care Team

Clinical Trials

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