Comparison of Outcome between Pediatric Liver Transplantation for Metabolic Diseases and Biliary Atresia.

Abstract

773 Children with metabolic diseases (MD) have a defined enzyme deficiency in an often normal liver. MD are, after biliary atresia (BA) the second largest indication for liver transplantation (LT) in children. Cure of the disease and prevention of secondary damage to other organs or malignant degeneration in the liver are the main goals for treatment. Most MD patients have a normal liver function and no signs of portal hypertension. Furthermore, pretransplant abdominal surgery in MD patients is uncommon. The outcome of LT in patients with MD is considered to be superior to that for other indications. The aim of this study was to compare the outcome of LT in terms of patient survival, graft survival, and morbidity in MD and BA patients. From November 1982 to June 1997 24 children were transplanted for MD and 52 for BA. The mean (SD) Child-Pugh score in MD patients was 8.3 (3.2) and 9.8 (2.1) in BA patients (p<0.05). All but two BA patients (96%) had previous abdominal surgery compared to only five (24%) of the MD patients (p<0.001). Median follow-up in the MD patients was 3.5 year (range 1-9.3) and 4.2 year (range 0.3-14.1) in BA patients. One and five year patient survival in the MD patients was 96% and 84% compared to 84% and 70% for BA patients (p=ns). One and five year graft survival in the MD patients was 75% and 58% compared to 75% and 64% in BA patients (p=ns). During follow up three patients (13%) in the MD group died compared to 15 (29%) in the BA group (p=ns). The percentage of patients who experienced postoperative bleeding, gastrointestinal, biliary, vascular, infectious, and neurological complications or rejections was not different between both groups. Furthermore, there was no difference observed in ICU stay, days on the ventilator, complication rate, and intervention rate. Seven (29%) children of the MD group were retransplanted compared to 11 (21%) of the BA group (p=ns). Conclusion: Despite their better pretransplant condition the outcome after LT for MD is not different from that after LT for BA.