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Results of long-term ursodiol treatment for patients with primary biliary cirrhosis
Roberta Jorgensen1, Paul Angulo, E Rolland Dickson
1Division of Gastroenterology and Hepatology, Mayo Clinic, Rochester, Minnesota 55905, USA.
Insights
Ursodeoxycholic acid (UDCA) treatment for primary biliary cirrhosis shows long-term benefits, especially in early-stage disease. While many patients achieve normal liver tests, further therapeutic options are needed for those with incomplete responses.
Area of Science:
- Hepatology
- Clinical Pharmacology
- Gastroenterology
Background:
- Primary biliary cirrhosis (PBC) treatment with ursodeoxycholic acid (UDCA) is linked to improved liver function, stable disease histology, reduced esophageal varices, and increased transplant-free survival.
- Limited data exists on the very long-term outcomes for PBC patients undergoing UDCA therapy.
Purpose of the Study:
- To evaluate the long-term outcomes of patients with primary biliary cirrhosis participating in a randomized controlled trial of UDCA.
- To assess the sustained efficacy and safety of UDCA over an extended follow-up period of up to 12 years.
Main Methods:
- A randomized, controlled trial involving 180 patients with PBC, comparing UDCA (n=89) to placebo (n=91) from April 1988 to March 1992.
- Following the randomized phase, all patients received active UDCA treatment and were followed for an additional 8 years, totaling up to 12 years of observation.
Main Results:
- Of the initial 180 patients, 28 from the UDCA group and 42 from the placebo group died or underwent transplantation.
- Patients who died or were transplanted had significantly more advanced histological disease at the study's entry (p < 0.001).
- Among the 110 patients remaining, 76 were regularly followed, 25 returned questionnaires, and 9 were lost to follow-up. Twenty-two of the 76 regularly followed patients achieved normal liver tests (ALP, bilirubin, AST), with lower baseline ALP and AST levels (p < 0.05).
Conclusions:
- Ursodeoxycholic acid (UDCA) demonstrates the most significant benefit when initiated in the early stages of primary biliary cirrhosis.
- While UDCA monotherapy leads to biochemical normalization in a considerable proportion of patients, alternative therapeutic strategies remain necessary for individuals with suboptimal biochemical responses.
Objective:
When ursodeoxycholic acid (UDCA) is used for the treatment of primary biliary cirrhosis, it has been associated with biochemical improvement, histological stability, reduced risk of esophageal varices, and increased survival free of transplantation. There is limited information available about the long-term outcome of these patients with primary biliary cirrhosis on UDCA treatment. To address this, we reviewed the long-term results from patients enrolled in our original randomized study with up to 12 yr of follow-up.
Methods:
From April 1988 to March 1992, a total of 180 patients were enrolled into a randomized, controlled trial evaluating UDCA (n = 89) versus placebo (n = 91). When the randomized portion of the study concluded in May 1992, patients were switched to active medication and followed for up to an additional 8 yr.
Results:
Twenty-eight patients originally assigned to UDCA and 42 patients originally assigned to placebo have died or undergone transplantation. The patients who died or were transplanted were more histologically advanced at entry (p < 0.001). Seventy-six of the remaining 110 patients return for regular follow-up; mailed questionnaires were returned by an additional 25 patients, and nine patients have been lost to follow-up. Twenty-two of the 76 patients we follow have normal liver tests (ALP, bilirubin, and AST). Patients with normal liver tests had significantly lower levels of ALP and AST at baseline (p < 0.05), but did not differ in histological stage or total bilirubin from those with persistently abnormal tests.
Conclusions:
UDCA appears to be of most benefit when instituted in early stage disease. Although a substantial percentage of patients will achieve biochemical normalization on UDCA alone, there is a continued need for therapeutic options for others who have less complete biochemical responses.
