Primary Biliary Cirrhosis Treatment (PBC)

  • Medical Author:

    John M. Vierling M.D. is Professor of Medicine and Surgery at the Baylor College of Medicine in Houston, Texas, where he also serves as Director of Baylor Liver Health and Chief of Hepatology. In addition, he is the Director of Advanced Liver Therapies, a center devoted to clinical research in hepatobiliary diseases at St. Luke's Episcopal Hospital. Dr. Vierling is board certified in internal medicine and gastroenterology and a Fellow of the American College of Physicians.

  • Medical Editor: Bhupinder S. Anand, MBBS, MD, DPHIL (OXON)
    Bhupinder S. Anand, MBBS, MD, DPHIL (OXON)

    Bhupinder S. Anand, MBBS, MD, DPHIL (OXON)

    Dr. Anand received MBBS degree from Medical College Amritsar, University of Punjab. He completed his Internal Medicine residency at the Postgraduate Institute of medical Education and Research, Chandigarh, India. He was trained in the field of Gastroenterology and obtained the DPhil degree. Dr. Anand is board-certified in Internal Medicine and Gastroenterology.

woman with abdominal pain

Cyclosporine (Sandimmune, Neoral, Gengraf)

Cyclosporine is a powerful immunosuppressive drug, cyclosporine (Sandimmune, Neoral, Gengraf) is used primarily to prevent rejection of transplanted organs. The drug prevents production of an important signal required for lymphocytes to divide (reproduce) and generate inflammation. A large study of 349 PBC patients, comparing cyclosporine with an inactive drug, showed some benefit from the cyclosporine. The frequency of the side effects of high blood pressure and decreased kidney function, however, make this drug unacceptable for long-term use.

Methotrexate (Rheumatrex, Trexall)

Methotrexate both suppresses the immune system and prevents cells from dividing. This drug has been used successfully in severe rheumatoid arthritis and an immunologic skin disease called psoriasis. Initial limited trials in patients with PBC did not show a benefit, and serious side effects included ulcerations of the mouth, hair loss, and pneumonia. Additionally, preliminary reports of randomized, controlled trials of methotrexate therapy of PBC in Europe noted a higher than expected rate of a form of pneumonia that scars the lungs. Moreover, a recently published randomized, controlled trial of low dose methotrexate in PBC showed serious toxicity over a six-year period. Currently, a large trial in the United States comparing UDCA alone to a combination of UDCA and methotrexate is underway. At present, it is premature to recommend the use of methotrexate to treat PBC outside of clinical trials.

Obeticolic acid (Ocaliva)

Ocaliva was approved by the FDA in May 2016 for the treatment of PBC in combination with ursodeoxycholic acid (UDCA) in adults with inadequate response or are intolerant to UDCA, or as a single therapy in adults unable to tolerate UDCA. In such patients, nearly 50% showed an improvement in the liver tests. The most common adverse effect is pruritus, observed in over 50% patients. The most common side effects include fatigue, abdominal pain and discomfort, joint pain, pain in the middle part of the throat, dizziness, constipation, and itching.

Medically Reviewed by a Doctor on 6/7/2016

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