Long-term prospective study of the effect of ursodeoxycholic acid on cystic fibrosis-related liver disease.
OBJECTIVE
To evaluate the efficacy of UDCA in arresting the progression of the early multifocal hepatic lesion to overt CF-related NBC.


BACKGROUND
Focal biliary cirrhosis is an early hepatic pathologic change related to the ion transport defect in cystic fibrosis. The factors involved in the progression of focal to nodular biliary cirrhosis are not clear. Ursodeoxycholic--a hydrophilic, nontoxic, choleretic, and hepatoprotective exogenous bile acid--has been reported to be effective in the management of cholestatic liver disease.


METHODS
For 10 years at 6-month intervals, 70 individuals with cystic fibrosis (38 men and 32 women; age range, 2--29 years) were examined using hepatosplenomegaly, liver function tests, and ultrasound liver scan. Patients demonstrating evidence of liver involvement at the onset or during the study received ursodeoxycholic acid 20 mg/kg body weight.


RESULTS
After the administration of ursodeoxycholic acid, the progression of nodular biliary cirrhosis ultrasound changes was arrested, hepatic function was preserved, and no variceal bleeding was observed. No case of focal biliary cirrhosis progressed to nodular biliary cirrhosis. Furthermore, the multifocal, multilobular changes suggestive of focal biliary cirrhosis on ultrasound scan were reversed to normal.


CONCLUSIONS
Ursodeoxycholic acid is effective in improving cholestasis and hepatic dysfunction in nodular biliary cirrhosis and, also, in reversing the early sonography findings suggestive of focal biliary cirrhosis. It is speculated that ursodeoxycholic acid may prove to affect the natural history of cystic fibrosis-related liver disease.
Fourth Department of Pediatrics, Aristotle University of Thessaloniki, Greece. sarvanit@med.auth.gr
S
S
Nousia-Arvanitakis
M
M
Fotoulaki
H
H
Economou
M
M
Xefteri
A
A
Galli-Tsinopoulou
D000293:Adolescent; D000328:Adult; D002648:Child; D002675:Child, Preschool; D002756:Cholagogues and Choleretics; D003550:Cystic Fibrosis / Q000150:complications*; D005260:Female; D006801:Humans; D008107:Liver Diseases / Q000000981:diagnostic imaging / Q000188:drug therapy* / Q000209:etiology*; D008111:Liver Function Tests; D008137:Longitudinal Studies; D008297:Male; D011446:Prospective Studies; D016896:Treatment Outcome; D014463:Ultrasonography; D014580:Ursodeoxycholic Acid / Q000627:therapeutic use*
D002756:Cholagogues and Choleretics; D014580:Ursodeoxycholic Acid