PSC Q&A

What is primary sclerosing cholangitis?
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.

What causes PSC?

PSC’s 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. 
What are the signs and symptoms?
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 bloodstream)
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 to develop.
How is primary sclerosing cholangitis diagnosed?
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 intestine.
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.
How is primary sclerosing cholangitis treated?
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 infected
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.
What is the long-term prognosis?
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.



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