Renal-hepatic-pancreatic dysplasia

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ORPHA:294415OMIM:208540Q45.8
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Overview

Renal-hepatic-pancreatic dysplasia (RHPD) is an extremely rare genetic condition in which the kidneys, liver, and pancreas do not develop properly. The word 'dysplasia' means abnormal growth or development of tissue. In this disease, these three vital organs form cysts (fluid-filled sacs) and have disorganized tissue structure, which prevents them from working correctly. The kidneys may be enlarged with many cysts and cannot filter blood properly. The liver often shows abnormal bile duct development and fibrosis (scarring), and the pancreas may also have cysts and structural problems. This condition is typically detected before birth or shortly after birth. Affected babies often have very underdeveloped kidneys, which can lead to reduced amniotic fluid during pregnancy (called oligohydramnios). Many affected infants are stillborn or die shortly after birth due to severe kidney failure and lung underdevelopment. Some cases also involve other birth defects, such as abnormalities of the bones or other organs. Because this condition is so severe and rare, treatment options are extremely limited. Care is primarily supportive, focusing on managing symptoms and providing comfort. There is currently no cure for renal-hepatic-pancreatic dysplasia. Genetic counseling is strongly recommended for affected families to understand the risk of recurrence in future pregnancies.

Also known as:

Key symptoms:

Enlarged kidneys with many cystsLiver fibrosis or scarringAbnormal bile ducts in the liverCysts in the pancreasLow amniotic fluid during pregnancyUnderdeveloped lungsKidney failure at or before birthAbnormal facial features related to low amniotic fluidFailure to thriveSkeletal abnormalities in some casesEnlarged abdomen due to large kidneysAbsent or very low urine output

Clinical phenotype terms (34)— hover any for plain English
Pancreatic dysplasiaHP:0005232Neonatal cholestatic liver diseaseHP:0006566Abnormal liver parenchyma morphologyHP:0030146Elevated gamma-glutamyltransferase levelHP:0030948Abnormal pancreatic duct morphologyHP:0030992Elevated circulating aspartate aminotransferase concentrationHP:0031956Elevated circulating alanine aminotransferase concentrationHP:0031964Pancreatic fibrosisHP:0100732
Inheritance

Autosomal recessive

Passed on when both parents carry the same gene change; often skips generations

Age of Onset

Neonatal

Begins at or shortly after birth (first 4 weeks)

Orphanet ↗OMIM ↗NORD ↗

Treatments

No FDA-approved treatments are currently listed for Renal-hepatic-pancreatic dysplasia.

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No actively recruiting trials found for Renal-hepatic-pancreatic dysplasia at this time.

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Search ClinicalTrials.gov ↗Join the Renal-hepatic-pancreatic dysplasia community →

No specialists are currently listed for Renal-hepatic-pancreatic dysplasia.

View NORD Rare Disease Centers ↗Undiagnosed Disease Network ↗

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 Renal-hepatic-pancreatic dysplasia.

Search all travel grants →NORD Financial Assistance ↗

Community

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Caregiver Resources

NORD Caregiver Resources

Support, advocacy, and financial assistance for caregivers of rare disease patients.

Mental Health Support

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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 specific genetic mutation was found, and what does it mean for our family?,What is the chance this could happen again in a future pregnancy?,Is prenatal genetic testing available for future pregnancies?,Can you refer us to a genetic counselor who specializes in this type of condition?,What palliative care and support services are available for our family?,Are there any research studies or registries we can participate in?,Should other family members be tested to see if they are carriers?

Common questions about Renal-hepatic-pancreatic dysplasia

What is Renal-hepatic-pancreatic dysplasia?

Renal-hepatic-pancreatic dysplasia (RHPD) is an extremely rare genetic condition in which the kidneys, liver, and pancreas do not develop properly. The word 'dysplasia' means abnormal growth or development of tissue. In this disease, these three vital organs form cysts (fluid-filled sacs) and have disorganized tissue structure, which prevents them from working correctly. The kidneys may be enlarged with many cysts and cannot filter blood properly. The liver often shows abnormal bile duct development and fibrosis (scarring), and the pancreas may also have cysts and structural problems. This co

How is Renal-hepatic-pancreatic dysplasia inherited?

Renal-hepatic-pancreatic dysplasia follows a autosomal recessive inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

At what age does Renal-hepatic-pancreatic dysplasia typically begin?

Typical onset of Renal-hepatic-pancreatic dysplasia is neonatal. Age of onset can vary across affected individuals.