Long-term prospective study of the effect of ursodeoxycholic acid on cystic fibrosis-related liver disease

Goals: 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 r elated to the ion transport defect in cystic fibrosis. The factors involved in the progression of focal to nodular biliary cirrhosis are not clear. Ur sodeoxycholic--a hydrophilic, nontoxic, choleretic, and hepatoprotective ex ogenous bile acid-has been reported to be effective in the management of ch olestatic liver disease. Study: For 10 years at 6-month intervals, 70 individuals with cystic fibros is (38 men and 32 women; age range, 2-29 years) were examined using hepatos plenomegaly, liver function tests, and ultrasound liver scan. Patients demo nstrating evidence of liver involvement at the onset or during the study re ceived 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 funct ion 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. Conclusion: Ursodeoxycholic acid is effective in improving cholestasis and hepatic dysfunction in nodular biliary cirrhosis and, also, in reversing th e early sonography findings suggestive of focal biliary cirrhosis. It is sp eculated that ursodeoxycholic acid may prove to affect the natural history of cystic fibrosis-related liver disease.