Treatment of pediatric cholestatic liver disease

Cholestatic liver disease affects a small percentage of children, but therapy results in millions of healthcare dollars being spent each year. Close monitoring of nutritional status, pruritus, and complications from portal hypertension should improve the patient’s quality of life and survival without liver transplantation. Other comorbid conditions, such as cardiac or renal disease, must also be integrated into the care plan and will affect the overall prognosis of the patient. Portal hypertension leads to ascites, variceal hemorrhage, and infection, which can result in significant mortality if not promptly recognized and treated. Surgical shunts are being used less because the expertise to perform endoscopic sclerotherapy and endoscopic band ligation is available at most medical centers. However, many cholestatic diseases, including biliary atresia, progressive familial intrahepatic cholestasis (PFIC) I, II, and III, and Alagille syndrome, may still require liver transplantation (Table 1). The cost of this procedure can exceed several hundred thousand dollars per patient, without including the annual costs of immunosuppressant therapy and medical monitoring. Meticulous medical management of nutrition and the sequelae of portal hypertension may prolong survival and reduce the potential morbidity and mortality of liver transplantation.