Summary of Research Presented at American Association Study of Liver Disease Conference
By Keith Lindor, MD & Roberta Jorgensen, MS, RN
Mayo Clinic Rochester
The results from a study evaluating ursodeoxycholic acid (UDCA) in combination with methotrexate were recently reported (Combes, Emerson & Flye, 2003).
The study was started in 1994 and enrolled a total of 265 patients. Patients were randomized to either UDCA alone or UDCA in combination with methotrexate. The two groups were looked at in terms of treatment failures which were defined as death, transplantation, variceal bleeding, doubling of bilirubin, development of ascites, lowering of albumin or progression by 2 stages or to cirrhosis. The average duration of treatment was approximately 7 years.
The results of the study showed no difference in the two groups in terms of the numbers of treatment failures or the time to treatment failure. However the initial histologic stage did have a bearing on outcome as there were more treatment failures in the group of patients who were in histologic stage 3 or 4 on entry into the study than in the group who were in histologic 1 and 2. This study shows that there appears to be no benefit from the addition of methotrexate to UDCA, but confirms the importance of starting UDCA when in early histologic stage.
A recent study from France supports the benefit of UDCA in improving long-term survival in PBC patients, and the favorable outcome of liver transplantation in PBC (Corpechot, Carrat, Bahr, Poupon & Poupon, 2003). A total of 375 patients were followed for up to 22 years. They found that the 20-year survival of patients with PBC treated with UDCA and in cases of liver failure with liver transplant was similar to the survival rate of a control population (a group matched according to age and gender but without liver disease). The survival of patients without liver transplant was less than that of the control population.
UDCA treatment and liver transplant when needed improve long-term outcomes in primary biliary cirrhosis. UDCA significantly improves long-term survival particularly when it is started in early histologic stage of the disease.
Combes, B., Emerson, S., Flye, N. (2003). The primary biliary cirrhosis (PBC) ursodiol (UDCA) plus methotrexate (MTX) or its placebo study (PUMPS) - a multicenter randomized trial. Hepatology, 38 (4), suppl 1, 210A.
Corpechot, C., Carrat, F., Bahr, A., Poupon, R., & Poupon, R. (2003). The impact of ursodeoxycholic acid (UDCA) therapy with or without liver transplantation (OLT) on long-term survival in primary biliary cirrhosis. Hepatology, 38 (4), suppl 1, 519A.