Pediatric Caroli Disease Treatment & Management

Updated: Oct 20, 2017
  • Author: Mutaz I Sultan, MD, MBChB; Chief Editor: Carmen Cuffari, MD  more...
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Approach Considerations

The management is for complications of portal hypertension and cholangitis because these are the main presenting features in children.


Medical Care

Ursodeoxycholic acid can decrease the frequency of Caroli disease complications due to cholelithiasis. Broad-spectrum antibiotic coverage, including anaerobic coverage, is indicated in cases of cholangitis. Patients with cholestasis should receive fat-soluble vitamin supplementation. Because patients with Caroli syndrome or Caroli disease are at an increased risk for cholangiocarcinoma, initial radiographic (ie, ultrasonography, MRI) and serologic (ie, CA19-9, CEA) screening should be performed.


Surgical Care

Surgical treatment may be necessary for recurrent or refractory cholangitis. Obstructing stones can be removed and bile flow can be maintained by means of a hepaticojejunostomy or external drainage. In cases of localized stasis, lobectomy can be curative and can also reduce the risk of cholangiocarcinoma. Liver transplantation may be indicated in severe cases of refractory or chronic cholangitis, liver failure, or malignant transformation. [7]

Liver transplantation represents an uncommon indication for Caroli disease. Excellent results have been reported, as shown by multicentric European and American registry reports. [8]

Isolated or combined kidney and liver transplantations have become available for children with polycystic liver and kidney disease. Immunosuppression after isolated renal transplantation may lead to an increased number of episodes of cholangitis and worsening liver condition. Therefore, a combined liver-kidney transplantation may be required in children with established end-stage renal failure and advanced chronic liver disease. [9]

 Patients who have developed esophageal varices should receive prophylaxis with a nonselective beta-blocker and variceal endoscopic therapy. The shunting procedure can provide relief from portal hypertension because liver function may be well preserved.