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Ursodeoxycholic acid for treatment of cholestasis in children on long-term total parenteral nutrition: a pilot study
M I Spagnuolo1, R Iorio, A Vegnente
1Department of Pediatrics, University of Naples Federico II, Italy.
Insights
Ursodeoxycholic acid (UDCA) effectively treats cholestatic liver disease (CLD) in children receiving total parenteral nutrition (TPN). Treatment led to normalization of liver markers and recovery in most patients.
Area of Science:
- Pediatric Gastroenterology
- Hepatology
- Clinical Nutrition
Background:
- Cholestatic liver disease (CLD) is a serious complication of long-term total parenteral nutrition (TPN).
- CLD can necessitate TPN withdrawal, impacting patient management.
- Identifying effective treatments for TPN-associated CLD is crucial.
Purpose of the Study:
- To evaluate the efficacy of ursodeoxycholic acid (UDCA) in treating TPN-associated CLD in pediatric patients.
- To assess the impact of UDCA on clinical and biochemical markers of cholestasis.
Main Methods:
- A pilot study involving seven children with TPN-associated CLD treated with UDCA.
- Monitoring of liver function tests including GGT, ALP, conjugated bilirubin, and ALT.
- Observation of treatment response during UDCA therapy and upon temporary discontinuation.
Main Results:
- UDCA treatment led to the resolution of CLD signs and normalization of liver markers within 4-8 weeks in all children.
- Temporary UDCA discontinuation while on TPN caused a transient increase in liver enzymes, which normalized upon reintroduction.
- No relapse of cholestasis was observed after UDCA withdrawal upon achieving full oral feeding.
Conclusions:
- Ursodeoxycholic acid (UDCA) demonstrates significant effectiveness in treating TPN-related cholestasis in children.
- UDCA offers a viable therapeutic option for managing this complication.
- The study highlights the importance of sustained UDCA treatment during TPN.
Background & Aims:
Cholestatic liver disease (CLD) is a frequent and sometimes fatal complication of total parenteral nutrition (TPN) that may require withdrawal of TPN. The aim of this pilot study was to evaluate ursodeoxycholic acid (UDCA) as treatment of TPN-associated CLD.
Methods:
Seven children (4 boys and 3 girls) undergoing long-term TPN because of intractable diarrhea syndrome developed cholestasis and were treated with UDCA. Treatment efficacy was evaluated by monitoring clinical and biochemical markers of CLD, including gamma-glutamyl transpeptidase (GGT), alkaline phosphatase (ALP), conjugated bilirubin, and alanine aminotransferase (ALT).
Results:
In all children, UDCA was associated with the disappearance of signs of CLD and with normalization of biochemical markers of cholestasis within 4-8 weeks. A rebound increase of GGT, ALP, and ALT serum levels was observed in 3 children in whom UDCA was temporarily discontinued while they were still undergoing TPN. However, after reinstitution of UDCA, markers of cholestasis normalized in all cases. UDCA was withdrawn on reinstitution of full oral feeding; there was no relapse of cholestasis. Six children fully recovered. One child died because of the lack of vascular access.
Conclusions:
UDCA appears to be an effective treatment for TPN-related cholestasis in children.
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