Treatment for Alagille Syndrome
How do doctors treat Alagille syndrome?
Doctors may refer people with Alagille syndrome to a hepatologist, a doctor who specializes in liver diseases, to treat liver symptoms and complications. Doctors may also refer patients to specialists who focus on other parts of the body, such as the heart, blood vessels, or kidneys.
Doctors may also recommend changes in diet and nutrition.
Doctors may prescribe ursodiol (Actigall, Urso) to improve the flow of bile from the liver to the small intestine. This medicine may help relieve severe itchy skin and reduce fatty deposits that appear as yellow bumps on the skin.
To help treat itchy skin, doctors may prescribe medicines such as cholestyramine, rifampin, naltrexone, or antihistamines. Doctors may also recommend using skin moisturizers, keeping baths and showers short, and trimming fingernails to prevent skin damage from scratching.
In some cases, liver symptoms may be severe. For example, itchy skin may disturb sleep and affect everyday activities. The size and number of fatty deposits may change appearance or affect vision, eating, or movement. If these severe symptoms don’t improve with medicine, doctors may recommend operations such as
- partial external biliary diversion (PEBD). In PEBD, surgeons remove part of the small intestine and use it to connect the gallbladder to an opening in the abdomen called a stoma. Bile leaves the body through the stoma and is collected in a pouch.
- liver transplant. A doctor may recommend a liver transplant if severe itchy skin and fatty deposits don’t respond to other treatments.
If liver problems lead to enlargement of the spleen, doctors may recommend avoiding contact sports to prevent spleen injury.
Other signs and health problems
Doctors who specialize in the heart, blood vessels, or kidneys may prescribe medicines or recommend surgeries to treat health problems and prevent complications.
Some signs of Alagille syndrome typically don’t cause health problems or require treatment. Examples include bones in the spine shaped like butterflies, called butterfly vertebrae, and white or gray-white rings on the corneas, called posterior embryotoxon.
How do doctors treat the complications of Alagille syndrome?
If cirrhosis leads to liver failure, a liver transplant may be needed. People with Alagille syndrome who have other severe health problems, such as serious heart defects, may not be good candidates for a liver transplant. In some cases, doctors may be able to correct other health problems before a liver transplant.
Doctors may treat serious heart defects and blood vessel problems with surgery.
Can I prevent Alagille syndrome?
Experts have not yet found a way to prevent Alagille syndrome. People with Alagille syndrome should see their doctors regularly to help manage symptoms, health problems, and complications. Doctors may be able to prevent some complications or prevent complications from getting worse.
This content is provided as a service of the National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), part of the National Institutes of Health. The NIDDK translates and disseminates research findings to increase knowledge and understanding about health and disease among patients, health professionals, and the public. Content produced by the NIDDK is carefully reviewed by NIDDK scientists and other experts.