Pediatric Caroli Disease Follow-up

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

Further Outpatient Care

Liver function and transaminase levels can be monitored on an outpatient basis.

Ultrasonography can be used to monitor stones.

No guidelines have been established regarding monitoring for the development of cholangiocarcinoma. Annual evaluation by MRI and measurement of CA19-9 and CEA is one approach, although the benefit of such screening has not been tested. CEA is relatively nonspecific and can be elevated in other bile duct pathologies such as cholangitis and hepatolithiasis. Although CA19-9 is more specific, it can also be elevated in benign bile duct disease.

Some promising studies have investigated the use of new tumor markers such as matrix metalloproteinase-7 (MMP-7) to differentiate between benign bile duct disease and cholangiocarcinoma. Further investigation is required before these markers can be recommended.

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Further Inpatient Care

Indications for hospitalization in patients with Caroli disease are as follows:

  • Suspected cholangitis or sepsis

  • Obstructing cholelithiasis requiring invasive intervention

  • Complications of end-stage liver disease

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Prognosis

Prognosis depends on the specific manifestations.

Hepatic manifestations

In the few patients who have intrahepatic ductal ectasia without associated congenital hepatic fibrosis (ie, Caroli disease), the frequency and severity of episodes of cholangitis, which may result in sepsis or death, largely determine the prognosis. Progressive liver failure may also develop, and liver transplantation may be required.

Patients with both ductal ectasia and congenital hepatic fibrosis (ie, Caroli syndrome) are subject to the risks and consequences of recurrent cholangitis, as described above. They are also at risk for the complications of cirrhosis and portal hypertension.

The risk of cholangiocarcinoma in individuals with Caroli disease or Caroli syndrome is estimated to be 100-fold higher than the general population. Cholangiocarcinoma following resection of Caroli disease or syndrome confined to a single lobe has not been reported.

Liver transplantation in patients with Caroli disease or Caroli syndrome is indicated in patients with refractory disease or with complications of hepatic fibrosis and is associated with excellent outcomes and long-term survival. Patients undergoing combined liver and kidney transplantation rather than liver transplantation alone generally have better outcomes. [10, 11]

Renal manifestations

The degree of polycystic kidney disease associated with Caroli syndrome varies. Patients who present with renal disease as neonates or infants are more likely to have severe kidney disease with enlarged cystic kidneys and progressive renal failure than others. Other patients may have normal-appearing kidneys or minimal cystic changes with only mild deficits in renal function.

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