Primary Sclerosing Cholangitis, or PSC, is a rare liver disease
that causes the bile ducts inside and outside the liver to become
inflamed, scarred and blocked. As this process continues, liver
cells die, and scarring (cirrhosis) develops. Eventually, the liver
begins to fail, and a liver transplant is needed. People with PSC
are also at high risk for developing cancer of the bile ducts (cholangiocarcinoma).
While waiting for a liver transplant, children can suffer from severe
abdominal pain, jaundice, weight loss, growth failure and a profound
lack of energy. Approximately three out of every 100,000 individuals
are diagnosed with PSC.
PSCs cause is not known, but many researchers think it might
be an autoimmune disease. When the immune system is working correctly,
it protects the body from infections caused by bacteria and viruses.
In the case of an autoimmune disease, the body does not recognize
certain cells and body parts as part of itself. The body then goes
to war against itself, damaging the body part it thinks is foreign.
In many cases, PSC occurs along with a type of inflammatory bowel
disease in which the colon becomes inflamed and develops ulcers
(colitis), but it may also be associated with cystic fibrosis and
disorders of the immune system.
PSC progresses slowly, so a person can have the disease for years
before symptoms appear. Symptoms are caused by either the bile not
being drained properly through the bile ducts or the liver not functioning
due to damage.
The main symptoms are:
1. Itching (may occur when toxins in bile get into the
2. Fatigue (feeling tired all the time)
3. Jaundice (yellowing of the eyes or skin; the color
change is due to bile that gets in the blood and eventually in the
skin and eyes)
4. Fever, chills and soreness in the upper part of the
abdomen (cholangitis caused by infected bile ducts)
As the disease progresses, chronic fatigue, loss of appetite, weight
loss and continued jaundice may occur. In the advanced stages, swelling
can occur in the abdomen and feet. Liver failure may take many years
Since people with primary sclerosing cholangitis may not have
noticeable symptoms for many years, the disease often is suspected
due to blood tests that show a high level of liver enzymes (which
indicate abnormal liver function). The disease also might be suspected
due to a history of inflammatory bowel disease.
PSC can be diagnosed by injecting dye into the bile ducts and taking
an x-ray (ERCP). The test can determine the cause and site of the
blockage. After the patient is sedated, a lighted and flexible endoscope
(instrument used to visually examine the inside of certain body
parts) is inserted through the mouth, stomach and into the small
A thin tube is placed through the scope and into the pancreatic
and bile ducts, and dye is injected to show the bile ducts on the
x-ray. If the x-ray shows the bile ducts are narrowed, the doctor
may diagnose the problem as primary sclerosing cholangitis.
Sometimes a special test called a MRCP is performed to visualize
the bile ducts. It does not require endoscopy or radiation, but
this test may not always be reliable, especially in children.
As the disease progresses, a liver biopsy is usually needed to determine
how much liver damage has occurred.
Currently there is no cure for primary sclerosing cholangitis. Treatment
is directed at managing symptoms and opening narrowed bile ducts.
Symptoms of this disease can sometimes be managed by:
1. Antibiotics to treat bile ducts that have become
2. Diets low in salt and medication to treat swelling
of the abdomen and feet caused by fluid retention
3. Vitamin supplements since people with primary sclerosing
cholangitis often do not have enough vitamins A, D, E and K
4. Medications to control itching caused by too much
bile in the bloodstream and to improve bile flow. Ursodeoxycholic
acid (Actigall) may result in an improvement in liver tests.
Endoscopic or surgical procedures may be used to open major blockages
in bile ducts. A catheter is a thin, flexible tube used to drain
bile from the ducts and relieve the obstruction.
A stent (artificial device in the form of a hollow tube) may be
placed in the bile ducts after they have been opened in order to
keep the ducts open. Some patients may undergo surgery in order
to explore the bile ducts and open major blockages.
Liver transplantation may be an option if the liver begins to fail.
Liver transplantation is very effective in the treatment of patients
with advanced liver disease caused by primary sclerosing cholangitis.
If a liver transplant is determined to be the best treatment option,
the doctor and patient care team will focus on preventing complications
and will treat symptoms while waiting for the donor liver.
PSC is a disease that tends to become more severe over time. The
Morgan PSC Foundation seeks to improve long-term outcomes for patients
around the country.
Medication does not have a major impact on slowing the progression
of primary sclerosing cholangitis, but it has an important role
in treating complications. For instance, medicine can help stop
itching, but it does not help a patient survive longer.
Children with primary sclerosing cholangitis account for about
2 percent of all liver transplants done in children. Liver
transplantation is successful in 90 percent of these children who
go on to have a good quality of life after recovery.