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. 2000 Jan;46(1):121–126. doi: 10.1136/gut.46.1.121

Characterisation of patients with primary biliary cirrhosis responding to long term ursodeoxycholic acid treatment

M Leuschner 1, C Dietrich 1, T You 1, C Seidl 1, J Raedle 1, G Herrmann 1, H Ackermann 1, U Leuschner 1
PMCID: PMC1727784  PMID: 10601067

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.


Keywords: primary biliary cirrhosis; prognostic indexes; full response to ursodeoxycholic acid; incomplete responders; anti-p53 autoantibodies; HLA typing

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Figure 1  .

Figure 1  

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Alkaline phosphatase concentrations during ursodeoxycholic acid (UDCA) treatment. ***p<0.001, ****p<0.0001 (since incomplete responders were treated for a longer period of time, the six year value was compared with the five year value of full responders).

Figure 2  .

Figure 2  

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Glutamate dehydrogenase (GLDH) concentrations during ursodeoxycholic acid (UDCA) treatment. *p<0.05 (since incomplete responders were treated for a longer period of time, the six year value was compared with the five year value of full responders).

Figure 3  .

Figure 3  

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Alanine aminotransferase (ALT) concentrations during ursodeoxycholic acid (UDCA) treatment. *p<0.05, **p<0.01 (since incomplete responders were treated for a longer period of time, the six year value was compared with the five year value of full responders).

Figure 4  .

Figure 4  

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IgM levels during ursodeoxycholic acid (UDCA) treatment. *p<0.05 (since incomplete responders were treated for a longer period of time, the six year value was compared with the five year value of full responders).

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