Though liver transplantation remains a technically challenging operative procedure and patients are complex in their perioperative management, the use of whole organ, reduced size, split liver, and even living donors has resulted in an overall improved patient survival approaching 90% for infants and children alike. These techniques have also reduced waiting list mortality.
The indications for liver transplantation have become somewhat more liberal and have improved outcomes for children with hepatoblastoma, a pediatric disease with a here-to-fore dismal prognosis.
Improved immunosuppression includes multiple drugs and in some centers even steroid-free immunosuppression. These protocols have limited the adverse impact of allograft rejection while minimizing the complications of immunosuppression including infection and PTLD. Chronic rejection awaits a better solution.
Over the past 30 years, orthotopic liver transplantation (OLT) in the pediatric population has evolved from a heroic last ditch intervention to become the current standard of care. The pioneering development of surgical procedures such as reduced-size, living donor (LD), and split liver transplantation along with the evolution of immunosuppressive management has improved 1-year survival rates to more than 90%. Because short-term outcomes have improved so dramatically, attention is now focused on the long-term, optimizing quality of life and cognitive development.
Indications for Transplantation
Table 99-1 reviews primary diagnoses leading to pediatric transplantation. These disease entities define the bimodal age distribution of pediatric transplant recipients. Infants and children in the first few years of life represent patients with biliary atresia unresponsive to portoenterostomy, unresectable hepatoblastoma and, occasionally, rapidly progressive hepatic failure secondary to metabolic abnormalities such as neonatal tyrosinemia or neonatal hepatic vascular tumors. Older children and adolescents present with metabolic disturbances, fulminant liver failure, and cirrhosis from underlying chronic liver disease.
Indications for Liver Transplantation at CCHMC from 2000 to 2010
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Table 99-1 Indications for Liver Transplantation at CCHMC from 2000 to 2010
|Primary Diagnosis ||Number of Patients ||% of Total |
|Neonatal cholestasis |
|Biliary atresia ||84 ||31.3 |
|Alagille syndrome ||10 ||3.7 |
|Idiopathic ||1 ||0.4 |
|Primary sclerosing cholangitis ||9 ||3.4 |
|TPN-induced ||5 ||1.9 |
|Metabolic Disease |
|Alpha1 anti-trypsin deficiency ||16 ||6.0 |
|Tyrosinemia ||3 ||1.1 |
|Glycogen storage disease—IV ||3 ||1.1 |
|Hyperoxaluria ||1 ||0.4 |
|Wilson disease ||2 ||0.8 |
|Cystic fibrosis ||2 ||0.8 |
|Urea cycle disorders ||11 ||4.1 |
|Acute Liver Failure |
|Untyped ||30 ||11.2 |
|Drug-induced ||2 ||0.8 |
|Other ||2 ||0.8 |
|Neonatal ||2 ||0.8 |
|Autoimmune ||11 ||4.1 |
|Hepatitis C ||1 ||0.4 |
|Hepatoblastoma ||20 ||7.5 |
|Hepatocellular carcinoma ||3 ||1.1 |
|Vascular ||3 ||1.1 |
|Other ||4 ||1.5 |
|Primary nonfunction ||3 ||1.1 |
|Hepatic artery thrombosis ||8 ||3.0 |
|Chronic rejection ||9 ||3.4 |
|Biliary tract complications ||2 ||0.8 |
|Other ||21 ||7.8 |
|Total ||268 || |
Children with extrahepatic biliary atresia constitute at least 50% of the pediatric liver transplant population. Successful biliary drainage via Kasai portoenterostomy is the most important factor governing preservation of liver function ...