Biliary tract malformation-renal failure syndrome

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Overview

Biliary tract malformation-renal failure syndrome, also known as Lücke-Beardmore syndrome, is an extremely rare condition that affects both the bile ducts (the tubes that carry bile from the liver to the intestine) and the kidneys. In this syndrome, babies are born with abnormally formed or blocked bile ducts, which prevents bile from draining properly from the liver. This leads to a buildup of bile that can damage the liver over time, causing jaundice (yellowing of the skin and eyes), poor growth, and liver problems. At the same time, the kidneys do not develop or function normally, leading to kidney failure that can range from mild to severe. This condition typically presents in the newborn period or early infancy. Affected babies may show signs of jaundice shortly after birth, along with pale-colored stools and dark urine due to the bile duct problems. Kidney issues may be detected through blood tests, urine tests, or imaging studies. Some children may also have other developmental abnormalities. Treatment is mainly supportive and depends on the severity of both the liver and kidney problems. Surgical procedures such as the Kasai operation may be attempted to restore bile flow. Kidney failure may require dialysis or eventually kidney transplantation. In severe cases, liver transplantation may also be considered. Because this syndrome is so rare, management is highly individualized and requires a team of specialists working together.

Also known as:

Key symptoms:

Yellowing of the skin and eyes (jaundice)Pale or clay-colored stoolsDark urineEnlarged liverPoor weight gain and growthKidney failureSwelling due to fluid retentionFeeding difficulties in infancyItchy skin from bile buildupAbnormal kidney structureFatigue and low energyHigh blood pressure from kidney problems

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 ↗NORD ↗

Treatments

No FDA-approved treatments are currently listed for Biliary tract malformation-renal failure syndrome.

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No actively recruiting trials found for Biliary tract malformation-renal failure syndrome at this time.

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No specialists are currently listed for Biliary tract malformation-renal failure syndrome.

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 Biliary tract malformation-renal failure syndrome.

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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.How severe is my child's liver and kidney involvement, and what does that mean for their future?,Will my child need surgery, and if so, what are the risks and expected outcomes?,Is organ transplantation likely to be needed, and when should we start planning for it?,What dietary changes should we make to support my child's liver and kidney health?,Should our family undergo genetic testing, and what would the results mean for future pregnancies?,What signs or symptoms should prompt us to go to the emergency room?,Are there any clinical trials or research studies that my child might be eligible for?

Common questions about Biliary tract malformation-renal failure syndrome

What is Biliary tract malformation-renal failure syndrome?

Biliary tract malformation-renal failure syndrome, also known as Lücke-Beardmore syndrome, is an extremely rare condition that affects both the bile ducts (the tubes that carry bile from the liver to the intestine) and the kidneys. In this syndrome, babies are born with abnormally formed or blocked bile ducts, which prevents bile from draining properly from the liver. This leads to a buildup of bile that can damage the liver over time, causing jaundice (yellowing of the skin and eyes), poor growth, and liver problems. At the same time, the kidneys do not develop or function normally, leading t

How is Biliary tract malformation-renal failure syndrome inherited?

Biliary tract malformation-renal failure syndrome follows a autosomal recessive inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

At what age does Biliary tract malformation-renal failure syndrome typically begin?

Typical onset of Biliary tract malformation-renal failure syndrome is neonatal. Age of onset can vary across affected individuals.