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autosomal dominant polycystic liver disease - Clinical Profile, Genes & Research | Kisho | Kisho

Rare Disease

autosomal dominant polycystic liver disease

MONDO:0000447

Also known as:AD polycystic liver diseaseADPLDPCLD

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Overview

An autosomal dominant inherited condition characterized by many cysts of various sizes scattered throughout the liver.

OnsetADULT

TreatmentsNo approved treatments · 2 in pipeline

Clinical Trials1 registered · 1 recruiting

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 autosomal dominant polycystic liver disease. An additional 2 compounds hold orphan drug designation.

Orphan Drug Designations

While no drugs are FDA-approved specifically for autosomal dominant polycystic liver disease, 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 autosomal dominant polycystic liver disease. Orphan designation reflects regulatory interest and does not indicate approval for treatment.

Brand Name	Generic Name	Sponsor

Prognosis

Estimated prevalence: 1-9 in 100,000 (Uncommon).

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.

1 clinical trial registered, 1 recruiting. Interventions under study include other interventions. Research is primarily sponsored by academic and government institutions.

25 publications have been identified in PubMed for autosomal dominant polycystic liver disease. Research spans Case Report / Case Series (24%), Review / Meta-Analysis (20%), and Basic Science / Preclinical (20%).

Research Type	Count	% of Total
Patient case studies	6	24%

Data assembled from 5 of 12 sources · Last updated Oct 3, 2026, 8:13 PM UTC

MONDOHPOClinicalTrials.govFDAPubMedHGNCClinVarClinGenGeneReviewsOrphanetOMIMRUSP

Medical References

External Resources

Orphanet

European rare disease database

Orphanet:2924

GARD

Genetic and Rare Diseases Info Center

GARD:9457

Questions for Your Care Team

→Are any of the 1 active clinical trial a good fit for my situation?
→What is the current development status of octreotide, 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

Kisho

autosomal dominant polycystic liver disease

Overview

Treatment

Orphan Drug Designations

Prognosis

Research Landscape

Medical References

External Resources

Orphanet

GARD

Questions for Your Care Team

Questions for Your Care Team

Clinical Trials

Recent Publications

Latest News & Developments

The population frequency of predicted pathogenic variants in the genes associated with Autosomal Dominant Polycystic Liver Disease (ADPLD) and kidney cysts.

autosomal dominant polycystic liver disease

Overview

Treatment

Orphan Drug Designations

Prognosis

Research Landscape

Medical References

External Resources

Orphanet

GARD

Subtypes

Related Diseases

Questions for Your Care Team

Questions for Your Care Team

Clinical Trials

Recent Publications

Latest News & Developments

The population frequency of predicted pathogenic variants in the genes associated with Autosomal Dominant Polycystic Liver Disease (ADPLD) and kidney cysts.