Medical Care

The goals of treatment are to slow the progression rate of the disease and to alleviate the symptoms (eg, pruritus, osteoporosis, sicca syndrome). Liver transplantation appears to be the only life-saving procedure.

Pharmacotherapy

Ursodeoxycholic acid (UDCA) is the major medication used to slow the progression of the disease. Patients with early disease have clinical, biochemical, and histologic improvement. Reports suggest that UDCA delays the need for transplantation and delays death. The efficacy of this medication in late stages (ie, cirrhosis) is questionable. Patients who achieve biochemical response to UDCA after 1 year of treatment reportedly have a similar survival rate to the matched control population, and this observation might be used to identify the population of nonresponders who will require alternative or additional treatments. The use of UDCA after transplantation with evidence of recurrence of the disease has been associated with biochemical response, although its role in delaying histologic progression needs further investigation.^{[10, 11]}

In May 2016, the FDA approved obeticholic acid to be used in combination with UDCA for primary biliary cholangitis (PBC) in adult patients with an inadequate response to UDCA, or as a single therapy in adults unable to tolerate UDCA.^[12]Approval was based on the international phase 3 POISE trial of patients (n = 216) with PBC. The trial included patients who had not shown a reduction in alkaline phosphatase (ALP) levels with UDCA at optimal dosage for at least 1 year or as monotherapy in those who were intolerant to UDCA. The primary endpoint (ie, ALP <1.67 × ULN) was met in 47% of the obeticholic acid 10 mg group and 46% in the titration group (ie, 5-10 mg) compared to 10% in the placebo plus UDCA group (p<0.0001).<ref>13</ref>

Immunosuppressive agents inhibit immune reactions that mediate the progression of the disease.

Methotrexate: Results of various trials suggest improvement in biochemical and histologic findings after treatment.

Corticosteroids may alleviate symptoms and improve biochemical and histologic findings. Corticosteroid-induced osteoporosis is of great concern. The dangers of using bisphosphonates requires further study.^[14]

Cyclosporine has some therapeutic potential.

Colchicine has been used with limited effect.

Antipruritic treatment

Pruritus is often refractory to medical therapy and significantly impacts the patient's quality of life. Antihistamines are the first-line agents to relieve pruritus in early stages and are the first line medication for patients with mild-to-moderate pruritus. Use caution in patients with cirrhosis and signs of encephalopathy because antihistamines can further depress brain function.

Cholestyramine and colestipol are effective in sequestering bile salts in the enteric lumen. A 1- to 4-day delay is expected before the itching remits.

Rifampin can also be used, but the precise mechanism of action is unclear (may involve inhibition of bile acid uptake into hepatocytes and facilitation of excretion of dihydroxy and monohydroxy bile acids and toxic bile acids). Rifampin is used in patients whose pruritus is not responding to cholestyramine.

Some evidence suggests that dronabinol (Marinol) can be used to good effect.

Plasmapheresis has also been implemented for patients with severe pruritus intractable to medical treatment. Results have been good.

Activity

Increased activity is recommended, especially in postmenopausal women, to prevent osteoporosis.

Surgical Care

As the disease progresses to cirrhosis, an elevated bilirubin level, a prolonged prothrombin time, and a decreased albumin level can be found. The increased bilirubin level is an ominous sign of disease progression, and liver transplantation must be considered. Liver transplantation appears to be the only life-saving procedure.

Medication

American Liver Foundation. Primary biliary cholangitis (PBC, primary biliary cirrhosis). March 29, 2016. Available at http://www.liverfoundation.org/abouttheliver/info/pbc/. Accessed: June 3, 2016.
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