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.

Medication

Nakanuma Y, Harada K, Sato Y, Ikeda H. Recent progress in the etiopathogenesis of pediatric biliary disease, particularly Caroli's disease with congenital hepatic fibrosis and biliary atresia. Histol Histopathol. 2010 Feb. 25 (2):223-35. [Medline].
Wang ZX, Li YG, Wang RL, et al. Clinical classification of Caroli's disease: an analysis of 30 patients. HPB (Oxford). 2015 Mar. 17 (3):278-83. [Medline].
Srinath A, Shneider BL. Congenital Hepatic Fibrosis and Autosomal Recessive Polycystic Kidney Disease. J Pediatr Gastroenterol Nutr. 2012 May. 54(5):580-7. [Medline].
Sato Y, Ren XS, Nakanuma Y. Caroli’s Disease: Current Knowledge of Its Biliary Pathogenesis Obtained from an Orthologous Rat Model. Int J Hepat. July 2012. doi:10.1155/2012/107945. [Medline].
Leelawat K, Sakchinabut S, Narong S, Wannaprasert J. Detection of serum MMP-7 and MMP-9 in cholangiocarcinoma patients: evaluation of diagnostic accuracy. BMC Gastroenterol. 2009 Apr 30. 9:30. [Medline].
Rawat D, Kelly DA, Milford DV, et al. Phenotypic Variation and Long-Term Outcome in Children With Congenital Hepatic Fibrosis. J Pediatr Gastroenterol Nutr. August 2013. 57 (2):161-6. [Medline].
[Guideline] Murray KF, Carithers RL Jr. AASLD practice guidelines: Evaluation of the patient for liver transplantation. Hepatology. 2005 Jun. 41(6):1407-32. [Medline].
Lai Q, Lerut J. Proposal for an algorithm for liver transplantation in Caroli's disease and syndrome: putting an uncommon effort into a common task. Clin Transplant. 2016 Jan. 30 (1):3-9. [Medline].
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Wang ZX, Yan LN, Li B, Zeng Y, Wen TF, Wang WT. Orthotopic liver transplantation for patients with Caroli's disease. Hepatobiliary Pancreat Dis Int. 2008 Feb. 7(1):97-100. [Medline].
Alvarez F, Bernard O, Brunelle F, et al. Congenital hepatic fibrosis in children. J Pediatr. 1981 Sep. 99(3):370-5. [Medline].
Bockhorn M, Malago M, Lang H, et al. The role of surgery in Caroli's disease. J Am Coll Surg. 2006 Jun. 202(6):928-32. [Medline].
D'Agata ID, Jonas MM, Perez-Atayde AR, Guay-Woodford LM. Combined cystic disease of the liver and kidney. Semin Liver Dis. 1994 Aug. 14(3):215-28. [Medline].
Guy F, Cognet F, Dranssart M, et al. Caroli's disease: magnetic resonance imaging features. Eur Radiol. 2002 Nov. 12(11):2730-6. [Medline].
Habib S, Shakil O, Couto OF, et al. Caroli's disease and orthotopic liver transplantation. Liver Transpl. 2006 Mar. 12(3):416-21. [Medline].
Hunter FM, Akdamar K, Sparks RD, et al. Congenital dilation of the intrahepatic bile ducts. Am J Med. 1966 Feb. 40(2):188-94. [Medline].
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Kassahun WT, Kahn T, Wittekind C, et al. Caroli's disease: liver resection and liver transplantation. Experience in 33 patients. Surgery. 2005 Nov. 138(5):888-98. [Medline].
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Mall JC, Ghahremani GG, Boyer JL. Caroli's disease associated with congenital hepatic fibrosis and renal tubular ectasia. A case report. Gastroenterology. 1974 May. 66(5):1029-35. [Medline].
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Media Gallery

Hepatic ultrasonogram of a neonate with Caroli disease. Multiple dilated intrahepatic bile ducts are present. Courtesy of Richard Bellah, MD, The Children's Hospital of Philadelphia.

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Author

Mutaz I Sultan, MBChB, MD Assistant Professor of Pediatrics, Al-Quds University Medical College; Pediatric Gastroenterologist and Hepatologist, Division of Pediatrics, Makassed Hospital, Palestine

Mutaz I Sultan, MBChB, MD is a member of the following medical societies: American Gastroenterological Association, North American Society for Pediatric Gastroenterology, Hepatology and Nutrition

Disclosure: Nothing to disclose.

Specialty Editor Board

Mary L Windle, PharmD Adjunct Associate Professor, University of Nebraska Medical Center College of Pharmacy; Editor-in-Chief, Medscape Drug Reference

Disclosure: Nothing to disclose.

Chief Editor

Carmen Cuffari, MD Associate Professor, Department of Pediatrics, Division of Gastroenterology/Nutrition, Johns Hopkins University School of Medicine

Carmen Cuffari, MD is a member of the following medical societies: American College of Gastroenterology, American Gastroenterological Association, North American Society for Pediatric Gastroenterology, Hepatology and Nutrition, Royal College of Physicians and Surgeons of Canada

Disclosure: Received honoraria from Prometheus Laboratories for speaking and teaching; Received honoraria from Abbott Nutritionals for speaking and teaching. for: Abbott Nutritional, Abbvie, speakers' bureau.

Additional Contributors

Robert Baldassano, MD Director, Center for Pediatric Inflammatory Bowel Disease, Children's Hospital of Philadelphia; Professor, Department of Pediatrics, Division of Gastroenterology and Nutrition, University of Pennsylvania School of Medicine

Robert Baldassano, MD is a member of the following medical societies: Alpha Omega Alpha, American Academy of Pediatrics, American Gastroenterological Association, North American Society for Pediatric Gastroenterology, Hepatology and Nutrition

Disclosure: Received consulting fee from Abbott, Inc for consulting.

David A Piccoli, MD Chief of Pediatric Gastroenterology, Hepatology and Nutrition, The Children's Hospital of Philadelphia; Professor, University of Pennsylvania School of Medicine

David A Piccoli, MD is a member of the following medical societies: American Association for the Study of Liver Diseases, American Gastroenterological Association, North American Society for Pediatric Gastroenterology, Hepatology and Nutrition

Disclosure: Nothing to disclose.

Joshua R Friedman, MD, PhD Director and Head of Disease Biology, Janssen Research and Development

Joshua R Friedman, MD, PhD is a member of the following medical societies: American Academy of Pediatrics, American Association for the Study of Liver Diseases, North American Society for Pediatric Gastroenterology, Hepatology and Nutrition

Disclosure: Received salary from Johnson & Johnson for employment.

Melissa Kennedy, MD Attending Physician, Division of Gastroenterology, Hepatology, and Nutrition, Children's Hospital of Philadelphia

Melissa Kennedy, MD is a member of the following medical societies: American Academy of Pediatrics, North American Society for Pediatric Gastroenterology, Hepatology and Nutrition

Disclosure: Nothing to disclose.

Sections Pediatric Caroli Disease
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Pediatric Caroli Disease Treatment & Management

Approach Considerations

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