Abstract

BACKGROUND In some patients with primary biliary cirrhosis, ursodeoxycholic acid causes full biochemical normalisation of laboratory data; in others, indexes improve but do not become normal.

AIMS To characterise complete and incomplete responders.

METHODS Seventy patients with primary biliary cirrhosis were treated with ursodeoxycholic acid 10–15 mg/kg/day and followed up for 6–13 years.

RESULTS In 23 patients (33%) with mainly stage I or II disease, cholestasis indexes and aminotransferases normalised within 1–5 years, except for antimitochondrial antibodies. Histological findings improved. Indexes were not normalised in 47 patients (67%) although the improvement of their biochemical functions parallelled the trend in the first group. In these incomplete responders histological findings improved to a lesser extent. The only difference between the two groups before treatment was higher levels of alkaline phosphatase and γ glutamyl transpeptidase in the incomplete responders. At onset of treatment the discriminant value separating responders from incomplete responders was 660 U/l for alkaline phosphatase and 131 U/l for γ glutamyl transpeptidase. One year later it was 239 and 27 U/l (overall predictive value for responders 92%, for incomplete responders 81%). There were no differences between the two groups concerning immune status, antimitochondrial antibody subtypes, liver histology, or any other data. HLA-B39, DRB1*08, DQB1*04 dominated in both groups.

CONCLUSIONS In patients with mainly early stages of primary biliary cirrhosis, higher values of alkaline phosphatase and γ glutamyl transpeptidase are the only biochemical indexes which allow discrimination between patients who will completely or incompletely respond to ursodeoxycholic acid treatment.