Background: In adults with chronic cholestatic liver disorders, controlled studies have shown a reduction of clinical, biochemical and possibly histological parameters during long-term medication with ursodeoxycholic acid (UDCA). It is not yet clear, however, whether similar effects can be achieved in children. Therefore, we retrospectively evaluated the use of UDCA in typical liver diseases of childhood.
Method: 20 children were treated for at least 6 months (age at start of therapy 5-87, median 24 months; diagnosis: biliary atresia n = 10, Alagille's syndrome n = 4, intrahepatic biliary hypoplasia n = 3, Byler disease n = 3). Pruritus, liver cell injury, cholestasis, synthetic liver function and weight and height for age before medication with UDCA (7-26, mean 13 mg/kg BW/d) was compared to values after 3, 6, 12, 18 and 24 months of therapy, with special attention towards possible adverse effects.
Results: No adverse effects of UDCA necessitating modification of therapy were encountered. During the first year of medication, weight for age improved in 15 patients, but pruritus in only four. During UDCA treatment, GIDH and gamma GT decreased significantly. GOT and GPT declined in the majority of patients. No significant changes of bilirubin and parameters of liver synthesis were seen.
Conclusion: Long-term medication with UDCA appears to be safe in children. Thus, controlled studies of UDCA medication in children are justified, and are urgently needed to further investigate the prognostic significance of the positive effects of UDCA identified in this retrospective analysis.