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Ursodeoxycholic acid therapy in children with cholestatic liver disease
1Department of Pediatrics, Hacettepe University Faculty of Medicine, Ankara.
Insights
Ursodeoxycholic acid treatment effectively improved cholestatic liver disease in children. The study found significant clinical and biochemical improvements with no adverse effects, suggesting it is a safe and effective pediatric therapy.
Area of Science:
- Pediatric Gastroenterology
- Hepatology
- Pharmacology
Background:
- Ursodeoxycholic acid (UDCA) benefits are known in adults, but pediatric data is limited.
- Cholestatic liver diseases in children require effective treatment options.
Purpose of the Study:
- To evaluate the efficacy and safety of ursodeoxycholic acid (UDCA) in pediatric patients with intrahepatic cholestasis.
Main Methods:
- Twenty-four children with various intrahepatic cholestases received UDCA (15-20 mg/kg/day) for 12 months.
- Clinical and biochemical markers of cholestasis were monitored regularly.
- Liver biopsies were analyzed at baseline and after 12 months for 17 patients.
Main Results:
- All patients experienced pruritus amelioration, with 16.7% achieving complete itch disappearance.
- Significant reductions were observed in ALT, AST, ALP, total bilirubin, and GGT levels.
- Liver biopsies showed improved cholestasis, but no significant change in fibrosis. No adverse effects were reported.
Conclusions:
- Ursodeoxycholic acid demonstrates safety and efficacy in treating pediatric intrahepatic cholestasis.
- UDCA offers a promising therapeutic option for children suffering from cholestatic liver conditions.
Abstract:
The beneficial effect of ursodeoxycholic add have been documented in adults but experience with this agent is limited in the pediatric population. The objective of this study was to evaluate ursodeoxycholic acid treatment in children with cholestatic liver disease. Twenty-four patients with intrahepatic cholestasis (neonatal hepatitis 7, Byler disease 7, idiopathic intrahepatic cholestasis 10) whose ages ranged from 1.5 months to 15 years were treated with ursodeoxycholic acid (15-20 mg/kg/day) for 12 months. Liver biopsy was performed initially on all patients and on 17 at the end of the twelve months. The outcome was evaluated by monitoring clinical and biochemical markers of cholestasis, including alanine aminotransferase, aspartate aminotransferase, alkaline phosphatase, gamma-glutamyl transpeptidase, cholesterol, total serum tasting bile acids and total and conjugated bilirubin at entry and every three months of treatment. Pruritus was ameliorated in all patients; there was complete disappearance of itching in 16.7 percent. There were significant decreases in mean serum levels of alanine aminotransferase, aspartate aminotransferase, alkaline phosphatase, total bilirubin and gamma-glutamyl transpeptidase. Liver biopsy specimens showed a significant improvement in the cholestasis but not in fibrosis. No adverse effects of therapy were noted. The improvements in the clinical and biochemical parameters and tolerability of the drug suggest that ursodeoxycholic acid is a safe and effective treatment in children with intrahepatic cholestasis.