URSODEOXYCHOLIC ACID FOR LIVER-DISEASE ASSOCIATED WITH CYSTIC-FIBROSIS - A DOUBLE-MIND MULTICENTER TRIAL

Liver disease is increasingly recognized as a major cause of morbidity in cystic fibrosis (CF). Preliminary data suggest that ursodeoxycholi c acid (UDCA) may be beneficial for treatment of this manifestation, W e performed a double-blind, multicenter trial in these patients to est ablish efficacy and safety of UDCA in terms of the improvement of clin ical and nutritional indicators besides standard liver function tests, We also intended to establish whether taurine supplementation has a b eneficial effect in patients receiving UDCA From June to December 1990 , we enrolled in 12 centers 55 CF patients with liver disease (39 male subjects; median age, 13.8 years). They were randomly assigned to rec eive for 1 year one of-the following treatments: UDCA (15 mg/kg body w eight daily) plus taurine (30 mg/kg body weight. daily), UDCA plus pla cebo, placebo plus taurine, or double placebo, Clinical and laboratory evaluations were performed every 3 months, After 1 year, deterioratio n of overall clinical conditions, as indicated by the Shwachman-Kulezy cki score (SKS), occurred in patients who received placebo but not in those who received UDCA (P = .025), Patients treated with UDCA also sh owed an improvement in gamma-glutamyl transpeptidase(GGT) (P = .004) a nd 5'-nucleotidase (P = .006) levels. Treatment with taurine was follo wed by a significant increase in serum prealbumin levels (P = .053), a trend toward a reduction in fat malabsorption, and no effect on the b iochemical profile, No severe side effects occurred with any treatment , Thus, we concluded that UDCA administration improves clinical and bi ochemical parameters in CF patients with liver disease. Taurine supple mentation may be indicated in patients with severe pancreatic insuffic iency and poor nutritional status.