Pediatric Biliary Atresia Medication

Updated: Feb 02, 2021
  • Author: Steven M Schwarz, MD, FAAP, FACN, AGAF; Chief Editor: Carmen Cuffari, MD  more...
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Medication Summary

In patients with chronic cholestatic conditions and bile duct patency, ursodeoxycholic acid (ie, ursodiol, UDCA) has also been shown to enhance bile flow. [23] For infants following portoenterostomy, UDCA may improve outcomes, and the drug was previously thought to be associated with minimal toxicity. However, one recent study evaluating UDCA in adults with primary sclerosing cholangitis found that long-term, high-dose treatment was associated with a higher rate of severe adverse events, including progression to cirrhosis. While the published experience with UDCA in biliary atresia has not shown similar deleterious outcomes, patients receiving long-term therapy with this agent should be carefully monitored. [24]

In order to prevent cholangitis postoperatively, prophylaxis with trimethoprim-sulfamethoxazole has been used on a long-term basis. Unfortunately, conclusive data supporting the use of this agent, or the other drugs described above, in the management of biliary atresia are not available.

In years past, high-dose methylprednisolone was used in the immediate postoperative period. Results from the START (Steroids in Biliary Atresia Randomized Trial) study was not superior to surgery alone. The trial concluded that the proportion of infants with biliary atresia who underwent hepatoportoenterostomy, high-dose corticosteroids following surgery did not result in statistically significant treatment differences in bile drainage at 6 months, although a small clinical benefit could not be excluded. Also, corticosteroid therapy was associated with earlier onset of serious adverse events in children with biliary atresia. [21]


Bile acids

Class Summary

These agents enhance bile salt-dependent biliary flow.

Ursodiol (Actigall, Urso)

Shown to promote bile flow in cholestatic conditions associated with a patent extrahepatic biliary system. Following portoenterostomy in infants with biliary atresia, the drug may be useful in enhancing biliary drainage.



Class Summary

Long-term antibiotic prophylaxis may reduce the incidence of cholangitis following portoenterostomy.

Trimethoprim-Sulfamethoxazole (Bactrim, Septra)

Cholangitis is a common complication, both acutely and long term, following the Kasai procedure. When used prophylactically, may reduce the incidence of cholangitis, though conclusive supportive information is not available.