Primary Biliary Cholangitis PBC Approved Treatment Guidelines

What Is PBC?

Primary biliary cholangitis (PBC), previously called primary biliary cirrhosis, is an autoimmune disease that damages bile ducts in the liver.

The only approved treatments for PBC are ursodeoxycholic acid (ursodiol, UDCA, Actigall, URSO 250, URSO Forte) and obeticholic acid (available only through specialty pharmacies in the U.S. and Canada). Fibrates may sometimes be used along with ursodeoxycholic acid. Other treatments may be given to treat complications. The first-line of treatment involves ursodeoxycholic acid, which is generally well-tolerated by people with PBC, and is used to delay the progression to end-stage liver disease, and to improve survival.

Some people can't tolerate ursodeoxycholic acid so obeticholic acid may be used. It helps reduce alkaline phosphatase, gamma-glutamyl transpeptidase, and aminotransferase levels, but it has not been shown to improve survival or disease-related symptoms.

For some people, the drugs methotrexate (Trexall, Rasuvo, Otrexup) and colchicine (Colcrys, Mitigare) may be given. There is currently no evidence these drugs provide any benefit.

Complications of primary biliary cholangitis may require treatment.

Itching: For mild symptoms, moisturizers may help. Moderate to severe itching is treated with medications such as cholestyramine (Questran, Questran Light, and Cholestyramine Light). Severe itching may require liver transplantation.

Metabolic bone disease: Lifestyle measures to reduce bone loss include adequate intake of calcium and vitamin D, exercise, quitting smoking, counseling on fall prevention, and avoiding heavy alcohol use. Bisphosphonates such as alendronate (Fosamax, Binosto, Fosamax Plus D) are the preferred treatment in patients at high risk of fractures.

Malabsorption: Treatment includes restricting dietary fats. May also include supplementation with medium-chain triglycerides (MCTs) if more calories are needed. If pancreatic insufficiency is suspected, pancreatic enzyme replacement may be effective.

Vitamin deficiencies: 1,000 to 1,500 mg calcium and 1,000 IU vitamin D daily through the diet and, if needed, supplements, are recommended. Other dietary supplements such as vitamins A, E, and K may be needed.

Hypothyroidism: Treated with thyroid hormone replacement in a dose that keeps TSH levels in the normal range.

High cholesterol and fatty growths under the skin: Plasmapheresis (a blood purification procedure) to decrease cholesterol levels in the blood may be performed.

Anemia: Oral iron replacement therapy is recommended.

Dry eyes, mouth, and vagina: For dry eyes, use artificial tears initially and pilocarpine (Salagen, Isopto Carpine) or cevimeline (Evoxac) in fpeople who do not respond to artificial tears. Cyclosporine ophthalmic emulsion (Restasis) may also be used. For dry mouth and difficulty swallowing, saliva substitutes may be tried. Pilocarpine or cevimeline can be used in patients who do not respond to saliva substitutes. Moisturizers may be used for vaginal dryness.