Use of Ursodiol in Infants and Children

Marcia L. Buck, Pharm.D., FCCP

Pediatr Pharm. 2009;15(2) 

In This Article

Adverse Effects

Ursodiol is generally well tolerated. In clinical trials of adults, the most frequently reported adverse effects have been nausea (in 14-17% of patients), dyspepsia (16%), diarrhea (25-27%), constipation (9-26%), headache (18-25%), dizziness (16%), and back or abdominal pain (11-43%).[1,3] In children, the most frequently reported adverse effect has been diarrhea.[12]

Although ursodiol administration has not been associated with the development of hepatic injury during clinical use, there is a potential risk for heptatoxicity from accumulation of the lithocholic acid metabolite. Lithocholic acid has been found to produce cholestatic injury and liver failure in animal models. This could theoretically occur in humans who lack the ability to form sulfate conjugates. It is recommended that all patients have serum transaminases measured prior to starting therapy and periodically thereafter, guided by the current recommendations for monitoring their underlying hepatic or biliary disease.[1,2,3]

A single report of an ursodiol overdose in an infant resulted in no significant adverse effects.[16] The patient was a 39 day old infant, born at 32 weeks, who was being treated with ursodiol for PNAC and hyperbilirubinemia. She was discharged on 20 mg ursodiol (2 mL of a 10 mg/mL compounded suspension) every 8 hours, to give a dose of 25 mg/kg/day. The preparation that the patient received at discharge was a 60 mg/mL suspension. Although the instructions on the label were to give 0.33 mL (20 mg) of this more concentrated suspension, the parents continued to administer 2 mL doses. This resulted in the patient receiving 80 mg every 8 hours (148 mg/kg/day). The error was discovered six days later and the family was sent to an emergency department. The patient appeared to be in no distress, with no history of vomiting or diarrhea. Her serum transaminases were within the normal range (AST 33 U/L and ALT 22 U/L) and serum bilirubin was 1.6 mg/dL. She was discharged without further intervention. While this case report demonstrates the relative safety of ursodiol, it also highlights the need for caution when using extemporaneous (compounded) formulations.


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