Disorder of bilirubin metabolism and excretion

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ORPHA:309816
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Overview

Disorder of bilirubin metabolism and excretion is a group of rare conditions in which the body has trouble processing or removing bilirubin, a yellow substance produced when red blood cells break down. Normally, the liver converts bilirubin into a form that can be excreted through bile into the intestines. When this process does not work properly, bilirubin builds up in the blood, leading to jaundice — a yellowing of the skin and the whites of the eyes. This group includes several well-known conditions such as Gilbert syndrome, Crigler-Najjar syndrome (types I and II), Dubin-Johnson syndrome, and Rotor syndrome. Each of these has a different underlying cause and severity. Symptoms can range from very mild (barely noticeable jaundice that comes and goes) to severe (dangerously high bilirubin levels in newborns that can cause brain damage if untreated). In milder forms like Gilbert syndrome, many people live completely normal lives and may not even know they have the condition. In more severe forms like Crigler-Najjar syndrome type I, intensive treatment including phototherapy (special light treatment) and sometimes liver transplantation may be needed. Diagnosis typically involves blood tests measuring bilirubin levels, liver function tests, and genetic testing. Treatment depends on the specific type and severity, ranging from no treatment needed to lifelong management strategies.

Key symptoms:

Yellowing of the skin (jaundice)Yellowing of the whites of the eyesDark-colored urinePale or clay-colored stoolsFatigue or tirednessAbdominal discomfortItching of the skinPoor feeding in newbornsIrritability in infantsNauseaUnexplained weight loss in severe casesEnlarged liver in some types

Inheritance

Variable

Can be inherited in different ways depending on the underlying gene

Age of Onset

Variable

Can begin at different ages, from infancy through adulthood

Orphanet ↗NORD ↗

Treatments

No FDA-approved treatments are currently listed for Disorder of bilirubin metabolism and excretion.

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No actively recruiting trials found for Disorder of bilirubin metabolism and excretion at this time.

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No specialists are currently listed for Disorder of bilirubin metabolism and excretion.

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 Disorder of bilirubin metabolism and excretion.

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Community

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Latest news about Disorder of bilirubin metabolism and excretion

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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.Which specific type of bilirubin disorder does my child or I have, and how severe is it?,What genetic test results confirm the diagnosis, and should other family members be tested?,What bilirubin level is considered dangerous for my specific condition, and how often should it be monitored?,Are there medications, foods, or activities I should avoid to prevent jaundice flare-ups?,Will liver transplantation be needed, and if so, when is the best time to consider it?,Are there any clinical trials or new treatments, such as gene therapy, that I might be eligible for?,What emergency signs should I watch for, and what should I do if bilirubin levels spike suddenly?

Common questions about Disorder of bilirubin metabolism and excretion

What is Disorder of bilirubin metabolism and excretion?

Disorder of bilirubin metabolism and excretion is a group of rare conditions in which the body has trouble processing or removing bilirubin, a yellow substance produced when red blood cells break down. Normally, the liver converts bilirubin into a form that can be excreted through bile into the intestines. When this process does not work properly, bilirubin builds up in the blood, leading to jaundice — a yellowing of the skin and the whites of the eyes. This group includes several well-known conditions such as Gilbert syndrome, Crigler-Najjar syndrome (types I and II), Dubin-Johnson syndrome,