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Ursodiol for the long-term treatment of primary biliary cirrhosis. The UDCA-PBC Study Group
R E Poupon1, R Poupon, B Balkau
1INSERM Unité 21, Villejuif, France.
Insights
Long-term ursodiol therapy significantly slows primary biliary cirrhosis progression and reduces liver transplant needs. This treatment offers major improvements for patients with this liver disease.
Area of Science:
- Hepatology
- Gastroenterology
- Clinical Pharmacology
Background:
- Primary biliary cirrhosis (PBC) is a chronic liver disease.
- Ursodiol (ursodeoxycholic acid) shows promise in improving PBC patient outcomes.
- The long-term efficacy of ursodiol in PBC management remains uncertain.
Purpose of the Study:
- To evaluate the long-term efficacy of ursodiol in slowing the progression of primary biliary cirrhosis.
- To assess the impact of ursodiol on the need for liver transplantation in PBC patients.
Main Methods:
- A randomized controlled trial involving 145 patients with biopsy-proven PBC.
- Patients were assigned to receive either ursodiol (13-15 mg/kg/day) or a placebo.
- Follow-up included an initial two-year randomized phase and a subsequent two-year open-label ursodiol treatment phase.
Main Results:
- Ursodiol treatment significantly reduced disease progression compared to placebo (P < 0.002).
- The need for liver transplantation or referral was significantly lower in the ursodiol group (P = 0.003).
- Ursodiol therapy also significantly reduced the combined endpoint of liver transplantation or death (P = 0.005).
Conclusions:
- Long-term ursodiol therapy is effective in slowing the progression of primary biliary cirrhosis.
- Ursodiol treatment significantly decreases the likelihood of requiring a liver transplant.
- Disease progression and transplant need were predicted by baseline bilirubin levels and cirrhosis signs.
Background:
Ursodiol (ursodeoxycholic acid) therapy leads to major improvements in patients with primary biliary cirrhosis. The benefit of long-term treatment is uncertain.
Methods:
We randomly assigned 145 patients with biopsy-proved primary biliary cirrhosis to receive ursodiol (13 to 15 mg per kilogram of body weight per day) (72 patients) or placebo (73 patients). After two years of follow-up, because of the benefit from ursodiol, all patients completing the study received ursodiol in an open trial and were monitored for two more years. The end points in the assessment of efficacy were as follows: progression of disease, as defined by the presence of hyperbilirubinemia, variceal bleeding, ascites, or encephalopathy; liver transplantation or a referral for that procedure; and liver transplantation (or a referral) or death.
Results:
Disease progressed significantly less frequently in the ursodiol group than in the placebo group (P < 0.002; relative risk, 0.28; 95 percent confidence interval, 0.12 to 0.63). The probability of liver transplantation or a referral for that procedure and the probability of transplantation or death were significantly lower in the group assigned to ursodiol than in the group assigned to placebo (for transplantation alone, P = 0.003; relative risk, 0.21; 95 percent confidence interval, 0.07 to 0.66; for transplantation or death, P = 0.005; relative risk, 0.32; 95 percent confidence interval, 0.14 to 0.74). High bilirubin levels and, to a lesser extent, signs of cirrhosis at entry into the trial were predictive of disease progression, liver transplantation or a referral, and transplantation or death.
Conclusions:
Long-term ursodiol therapy slows the progression of primary biliary cirrhosis and reduces the need for liver transplantation.