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Basic PSC Facts

PSC can affect people from any race, gender, or age.

Primary Sclerosing Cholangitis (PSC) is a rare liver disease that damages the bile ducts inside and outside the liver. With PSC, bile ducts become inflamed, and the inflammation leads to scarring and narrowing of the affected ducts. Eventually, blockages may occur. As the scarring blocks more and more ducts, bile becomes trapped in the liver. This damages the liver and can result in fibrosis and cirrhosis of the liver and liver failure. Patients may eventually require a liver transplant.

Your Liver and Why It's Important

The liver is the largest internal organ in the body and has more than 500 vital functions to help keep your body healthy, including:

  • Making bile to help break down fats in the small intestine for digesting food, and for removing waste products or toxic substances
  • Producing proteins for blood plasma
  • Removing bilirubin and cholesterol, and metabolizing drugs
  • Storing carbohydrates, vitamins, and minerals

BASIC FACTS of PSC

Primary Sclerosing Cholangitis (PSC) does not discriminate based on age, race, or gender, but affects more males than females.

  • Many PSC patients are asymptomatic at the time of diagnosis.
  • It is estimated that there are more than 30,000 PSC patients in the United States.
  • One in every 10,000 people worldwide is diagnosed with PSC.
  • PSC often is found in people with inflammatory bowel disease (IBD), most often ulcerative colitis (UC) and sometimes Crohn’s disease.
  • More than 75 percent of PSC patients have UC. PSC also is associated with other autoimmune diseases.

RECEIVING DIAGNOSIS

PSC is a rare disease. It can take time to receive a PSC diagnosis, but, thanks to an increase in knowledge about this rare disease, that is changing. As you likely know, in the beginning, PSC symptoms can be minor or completely absent. Oftentimes, a blood test indicating a liver enzyme issue is the first sign that something is wrong.

Liver chemistry tests include:

•       alkaline phosphatase (ALP)

•       alanine aminotransferase (ALT)

•       aspartate aminotransferase (AST)

•       gamma-glutamyltranspeptidase (GGT)

Elevated ALP and GGT usually indicate an injury to the bile ducts and are almost always elevated in PSC. The ALT and AST may also be elevated in PSC. The diagnosis of PSC requires an image of the bile ducts called a cholangiogram. This is usually done by a special MRI called an MRCP (magnetic cholangiopancreatography). Sometimes, a procedure called ERCP (endoscopic retrograde cholangiopancreatography) is performed instead. Rarely, a liver biopsy is also needed for diagnosis. Additional blood tests may be performed to exclude other diseases, which can look like, or mimic, PSC.

CAUSE

Although no one knows the causes of PSC, research is underway to better understand the disease, develop more effective treatments, and, eventually, find a cure.

  • PSC may have genetic, autoimmune, and environmental origins
  • PSC progression is different for each person
  • PSC isn’t contagious, and is not caused by alcohol consumption
  • PSC cannot be transmitted through kissing, sexual activity, touching, or blood transfusions
  • Although there may be a genetic predisposition to PSC, most children of PSCers are healthy and unaffected

COMMON SYMPTOMS OF PSC

Although many PSC patients report few, if any, symptoms, some experience the following:

  • Pruritus or intense itching: Particularly on soles of feet or palms of hands, though it can occur anywhere, including in eyes and mouth.
  • Fatigue: Feeling run down, unable to get enough sleep, flu-like exhaustion that does not go away with sleep.
  • Depression: Feelings of despair or hopelessness.
  • Pain: Felt in the right side or in the middle of the abdomen towards the rib cage. This is often termed right upper-quadrant (RUQ) pain. The pain may extend to the shoulder blade area, may be of any intensity, and may last for an indefinite period of time.
  • Jaundice: Yellowing of eyes and skin caused by excess bilirubin that the liver cannot process. May be accompanied by dark urine.
  • Chills and Fever: These may be signs of a bacterial infection in the bile ducts, a condition called cholangitis. A cholangitis attack requires immediate medical attention and antibiotics.

Signs that PSC has progressed to cirrhosis, evidence that liver transplantation may be indicated, include:

  • Ascites: Buildup of fluids in the abdomen
  • Encephalopathy: Personality changes, confusion, and sleep disturbances caused by a buildup of toxins, such as ammonia, in the blood
  • Varices: Swollen veins prone to bleeding, usually in the esophagus. When bleeding occurs, it can cause vomiting of blood or passage of black, tarry stool. Medical attention should be sought immediately when this happens.
  • Splenomegaly: An enlarged spleen
  • Abnormal Blood Tests:  Several blood tests, including bilirubin, creatinine, and International Normalized Ratio (INR), may become abnormal and may indicate whether a liver transplant is needed 

Although the average time from diagnosis to liver transplantation is 15-20 years, some patients need transplants sooner, and some never need a transplant.

TREATMENTS

At this time, there are no FDA-approved medications for the treatment and cure of PSC, but there are medications and procedures that help relieve some PSC symptoms.

  • An inserted stent can widen a blocked bile duct.
  • Some medications help with the itching that commonly occurs in PSC patients. (See more about medications at the end of this section.)
  • At this time, the only definitive treatment is a liver transplant. Some PSCers live long and productive lives with the disease and never need a transplant. Other PSCers end up needing a transplant, and they return to health. In some cases, the PSC returns post-transplant. This is called recurrent PSC. There are some PSCers who have undergone multiple liver transplants.

Clinical Trials:

There are an increasing number of ongoing PSC clinical trials. PSC Partners encourages everyone to research and discuss all potential medications and treatments, including participation in clinical trials, with their healthcare professionals.

To learn more about PSC clinical trials, visit PSCPartnersRegistry.org.

PSC Partners supports research to discover causes, treatments, and a cure for PSC. Click here to learn more about current and past research studies.

COMPLICATIONS

Complications of PSC can include:

  • Bacterial Cholangitis
  • Biliary Stone Disease
  • Dominant Stricture (especially in the common bile duct)
  • Cholangiocarcinoma (cancer of the bile ducts)
  • Colon cancer, for those who also have IBD (ulcerative colitis or Crohn's disease)
  • night sweats, nausea 
  • vitamin deficiency of fat soluble vitamins, A, D, E, or K
  • stool changes 
  • weight loss

In the later stages of PSC, symptoms can include: 

  • clotting problems 
  • nail clubbing 
  • jaundice
  • variceal bleeding (spontaneous bleeding in the veins around the esophagus)
  • ascites (fluid in the belly) 
  • hepatic encephalopathy (HE or mental fog), and other problems.

To better understand PSC and related diseases, visit the Medical Terms page on this website.

COMMON PSC MEDICATIONS

Click here for the PSC Partners Statement About the Use of Medications Not Approved by the FDA for PSC

Cholestyramine (Questran)

Cholestyramine is used to treat high cholesterol and also has been proven effective in combating pruritus (itching) due to bile blockages. Cholestyramine is not absorbed in the bloodstream; it remains inside the gut until excretion. Cholestyramine and urso should not be taken at the same time. The general recommendation is to stagger doses of the two medications at least four hours apart.

Rifampin (Rifampicin)

Rifampin is an antibiotic mostly used for the treatment of tuberculosis. However, because it activates receptors in the gut and liver, it has also been shown to help relieve pruritus (itching). Rifampin may cause changes in other medications you are taking and alter the way the medicines react in your system. Make sure your doctor is aware of all other medications you are taking, along with Rifampin, in order to avoid any possible conflicts.

Ursodeoxycholic acid (Ursodiol, Urso Forte, UDCA, URSO, Actigall, Ursofalk)

Urso is a popular medication used for patients diagnosed with PSC. In addition, some anti-inflammatory properties have been noticed. Urso helps to fight toxic bile acids that build up in the liver cells. In the United States, Urso is available under different trade names such as Actigall (typically 300 mg. capsules) and URSO (typically 250 mg tablets). Urso Forte is available in a 500 mg tablet and, along with Urso 250, is the only FDA approved treatment for patients with PBC (Primary Biliary Cholangitis), a disease similar to PSC. Another variation of Urso is available in Australia under the name Ursofalk (250 mg capsules). Ursofalk is also available in a liquid formulation for pediatric patients.

Ursodiol is typically prescribed in typical dosages of 15-30 mg/kg a day. The benefit of high-dose ursodiol is questionable, and a recent study suggests that high doses may actually be detrimental. For this reason, most hepatologists prescribing ursodiol today will uses doses in the range of 15 to 20 mg/kg a day. Another unproven, but suggested benefit of ursodiol may be reduction in risk of colon cancer and cholangiocarcinoma.

Vancomycin

Oral Vancomycin is an antibiotic used for gram-positive bacteria. Some doctors have been prescribing oral vancomycin for PSC. Other than small case studies and small clinical trials, there is not enough evidence about the efficacy and safety of Vancomycin in treating PSC. The Mayo Clinic is currently conducting a clinical trial with oral vancomycin.

For information on Vancomycin and PSC, check out:

NON-PRESCRIPTION REMEDIES

PSC Partners Seeking a Cure believes that medical professionals should be the primary source of information for treating your PSC. Check with your doctor before making changes to your health routine or taking any alternative or herbal products.

  • Natural Products: It is important to remember that a product labeled as “natural” does not necessarily mean it is safe for the PSC patient. Supplements and herbs, despite being “natural,” can be toxic to the liver. The production and distribution of these supplements are not regulated as carefully as the production of prescription medications. In the U.S., the Food and Drug Administration does not test or regulate these products. “Natural” products can be sold with little testing and with no proof of efficacy. Sometimes the herb or supplement itself can cause liver damage. In other cases, impurities or toxins introduced during the preparation of the product may be toxic to the liver.

QUALITY OF LIFE

Thousands of people with PSC live active and full lives. There are proactive steps you can take to prepare for your future, receive the support you need, and ensure you stay up to date on the progress toward treatments and a cure:

Get Educated: We know it can be overwhelming, but education is power, and we're here to help. Check out these resources to get your started:

Join Registry: Join the PSC Partners Patient Registry. The registry stores information about those affected by PSC. This information helps researchers and clinicians unlock the mystery of PSC as they search for treatments and a cure.

OTHER RESOURCES

Complete your profile and join PSC Partners Seeking a Cure in advancing PSC research towards a cure.