Primary biliary cirrhosis (PBC) is a presumed
autoimmune disease of the liver, which predominantly affects middle age women. Most patients are diagnosed when asymptomatic. The disease is characterised by chronic, granulomatous
inflammation of the small bile ducts, which leads to progressive ductopenia,
cholestasis,
fibrosis,
cirrhosis and eventual
liver failure. All PBC patients with abnormal liver biochemistry should be considered for
therapy.
Ursodeoxycholic acid (
URSO) treatment reduces intracellular hydrophobic
bile acid levels and thereby may have a cytoprotective effect on cell membranes.
URSO may also act as an immunomodulating agent. Multicenter randomised controlled trials proved that the treatment is associated with a marked improvement in serum
biochemical markers of
cholestasis, i.e.
bilirubin, ALP, GGT, including fall in serum
cholesterol levels. Treatment does not seem to benefit the symptoms of
fatigue,
pruritus, and
osteoporosis. UDCA has been shown when given in a dose of 15 mg/kg daily for up to 4 years to prolong the time to
liver transplantation or death. Immunosuppressive therapy: based on the immunological abnormalities, several immunosuppressive drugs have been tested. Neither
azathioprine nor
cyclosporine was found in large enough trials to show beneficial effect on survival.
D-penicillamine, cholchicin, methotrexát,
prednisolone were found without significant long-term benefit. Combination
therapy with
URSO and budenoside appears to add some benefit to
URSO monotherapy, but further studies are needed.
Liver transplantation. The most crucial question is the timing. Serum
bilirubin, Mayo risk score and some other factors such as uncontrollable
pruritus and severe
osteoporosis influence the decision. Recurrence of PBC in allograft is rare, the progress is slow, and is no reason for not recommending
transplantation. Symptomatic treatment of
pruritus,
sicca syndrome and preventive treatment of
osteoporosis, neuropathy and fat soluble
vitamin deficiency is also important.