(2-B) 31. A first-degree family
member may be considered for living donation in Alagille syndrome, but donor evaluation must include careful assessment to rule out bile duct hypoplasia that may include liver biopsy and/or cholangiography (2-B); if the potential donor and recipient share the same mutant Jagged 1 or Notch 2 allele the donor should be carefully evaluated for bile duct hypoplasia and vascular anomalies, but LRLT is not advisable in most circumstances. (2-B) Biliary atresia (BA) is universally fatal if untreated and is the single most common cause of liver disease leading to LT in children.[123, 124] Diagnosis of BA and performance of a hepatoportoenterostomy (HPE; Kasai Procedure) by 8 to 10 weeks of age is optimal for transplant-free survival beyond early childhood. selleck products Infants with BA with vitamin K nonresponsive coagulopathy, hypoalbuminemia, histologically advanced cirrhosis, ascites, portal hypertension, and poor nutritional status prior to HPE have poor outcomes. Following HPE, up to 70% of BA patients may have prolonged transplant-free survival if the total serum bilirubin falls below 2 mg/dL within 3 months following the HPE.[7, 124, 126] Children with biliary atresia splenic malformation (BASM) may have less favorable rates of transplant-free survival as reported in some studies,[7, 125, 127-131]
but not others.[132, 133] Post-HPE complications include ongoing cholestasis,
cholangitis, portal hypertension with or without variceal hemorrhage, poor weight gain, fat soluble vitamin Palbociclib deficiencies, hepatopulmonary syndrome, porto-pulmonary hypertension, and rarely hepatocellular carcinoma. Post-HPE regimens to promote bile flow (i.e., ursodeoxycholic acid) in BA patients are not standardized.[124, 126, 134-136] Prophylactic antibiotic regimens with either trimethoprim/sufamethaxazole or neomycin reduce recurrent rates of cholangitis and improve survival.[137, 138] High-dose corticosteroid therapy initiated within 72 hours of HPE was not shown to improve bile drainage at 6 months, nor did it enhance transplant-free Galactosylceramidase survival up to 2 years of age. Aggressive nutritional support to ensure adequate growth and prevention of fat soluble vitamin deficiency can improve neurodevelopmental and transplant outcome.[27, 103, 140, 141] Management of portal hypertension remains poorly studied in children and use of beta-blocker therapy for primary prophylaxis of variceal hemorrhage is controversial in childhood. Variceal hemorrhage may be the sentinel event that prompts LT evaluation. Anecdotal cases of hepatocellular carcinoma (HCC) in BA patients have been reported, including patients less than 1 year of age, but the risk of HCC in BA is low.