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- PBC facts
- What treatments are used in patients with PBC?
- Ursodeoxycholic acid (UDCA)
- Colchicine (Colcrys)
- Immunosuppressive medications
- Budesonide (Entocort)
- Azathioprine (Imuran)
- Cyclosporine (Sandimmune, Neoral, Gengraf)
- Methotrexate (Rheumatrex, Trexall)
- Obeticolic acid (Ocaliva)
- Cholestyramine (Questran) for itching
- Rifampin for itching
- Opiod antagonists for itching
- Charcoal hemoperfusion for itching
- Osteoporosis medications
- Treatment of elevated serum cholesterol and xanthomas
- Treatment of malabsorption of fat and fat-soluble vitamins (A, D, E and K)
- Treatment of edema and ascites
- Treatment of bleeding from varices
- Treatment of hepatic encephalopathy
- Treatment of enlarged spleen
- Treatment of Sicca syndrome
- Treatment of Raynaud's phenomenon
- Treatment of scleroderma
- Treatment of gallstones
- Which specialties of doctors treat PBC?
- What is the role of liver transplantation in PBC?
- What is the future for PBC?
Corticosteroids, for example, prednisone, prednisolone, and budesonide (Entocort) inhibit the initiation of immune responses, including those initial responses required for perpetuation of autoimmunity reactions. A randomized (treatment assigned by chance) controlled trial was carried out comparing a placebo with a low dose of prednisolone over a 3-year period. This study showed that prednisolone improved liver function and did not significantly increase the rate of bone thinning or demineralization. (Osteoporosis is a potential side effect of steroids). Another randomized trial compared UDCA and placebo with UDCA and prednisolone in patients with early stages of PBC. Although improvement in liver function was similar for both groups, only the combination of UDCA and prednisolone resulted in markedly improved liver biopsies.
It is noteworthy that the principal benefits of corticosteroids were seen in patients with early stages of the disease on liver biopsy. Still, these treatments did not result in a full remission or cure. Moreover, neither the size nor duration of these trials was sufficient to determine an effect on survival without liver transplantation. Accordingly, more data are needed to confirm the benefit and safety in PBC of steroids alone or in combination with UDCA. Nevertheless, these studies disproved an earlier notion that corticosteroids would cause rapid progression of the bone disease osteoporosis in patients with PBC.
Budesonide is a steroid that is more rapidly processed (metabolized) in the liver and, therefore, presumably would be less injurious to bone than other steroids. This drug was studied in selected patients with PBC who had had suboptimal (less than favorable) responses to UDCA. Unfortunately, budesonide was ineffective in this group. In fact it significantly worsened osteoporosis and did not prevent progression of the PBC. In contrast, a randomized trial comparing UDCA and placebo with a combination of budesonide and UDCA showed the combination to be more effective, while bone thinning (loss of mineral density) was comparable in the two groups. But here again, more data are needed to confirm the benefit and safety of this combination.
Imuran prevents the production of new lymphocytes (white blood cells that take part in immune responses) by blocking cell division (reproduction) of the lymphocytes. The consequence of this action is to reduce the number of new inflammatory cells entering the sites of inflammation. A large study comparing the effect of azathioprine with an inactive drug (placebo) in 248 patients with PBC, however, showed no benefit. Consequently, this drug is not currently recommended for use in PBC patients outside of research protocols.