Ursodeoxycholic acid for treatment of cholestasis in children on long-term total parenteral nutrition: a pilot study.
OBJECTIVE
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 UDCA was withdrawn on reinstitution of full oral feeding; there was no relapse of cholestasis.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.
Spagnuolo
M I
MI
Department of Pediatrics, University of Naples Federico II, Italy.
Iorio
R
R
Vegnente
A
A
Guarino
A
A
D000410:Alanine Transaminase / Q000097:blood; D000469:Alkaline Phosphatase / Q000097:blood; D001663:Bilirubin / Q000097:blood; D002779:Cholestasis / Q000097:blood / Q000188:drug therapy* / Q000209:etiology; D005765:Gastrointestinal Agents / Q000627:therapeutic use*; D006801:Humans; D007223:Infant; D008297:Male; D010289:Parenteral Nutrition, Total / Q000009:adverse effects*; D010865:Pilot Projects; D013997:Time Factors; D016896:Treatment Outcome; D014580:Ursodeoxycholic Acid / Q000627:therapeutic use*; D005723:gamma-Glutamyltransferase / Q000097:blood
D005765:Gastrointestinal Agents; D014580:Ursodeoxycholic Acid; D005723:gamma-Glutamyltransferase; D000410:Alanine Transaminase; D000469:Alkaline Phosphatase; D001663:Bilirubin