Combined en bloc liver pancreas transplantation for children with CF

Cystic fibrosis (CF) is an inherited genetic defect in epithelial chloride transport that results a multisystem disease affecting the sweat glands and the pulmonary and digestive systems. Although pulmonary disease remains the primary cause of morbidity and mortality in these patients, up to 8% may develop focal biliary cirrhosis and portal hypertension. Liver transplantation is an accepted therapy for these patients. About 85% of CF patients develop pancreatic exocrine insufficiency and up to 34% will develop diabetes requiring insulin. We describe a series of 9 patients who underwent liver transplantation (6 transplantation of liver only [LO], and 3 combined en bloc liver-pancreas [LP] transplantation) for CF-related liver disease at our institution,. All 9 patients had pretransplant pancreatic exocrine insufficiency requiring enzyme supplementation. Of the 9, 5 patients (55%) had preoperative insulin dependent diabetes, including all 3 patients who underwent liver pancreas transplantation. Liver-pancreas transplants were performed en bloc. One-year patient survival for both LO and LP patients was 100%. Five-year patient survival was 83% for the LO patients and 100% for the LP patients. All LP patients are off insulin and do not require pancreatic enzyme supplementation. All patients receiving LO continue to need exogenous pancreatic enzyme supplementation. In the 6 patients who received LO, 2 were insulin dependent prior to surgery, and they remain insulin dependent after transplant. Of the 4 patients who received LO who were not insulin dependent pretransplant, 3 have now developed insulin-dependent diabetes. Patients receiving LO were more likely to be diabetic and require exogenous pancreatic enzymes after transplant than those receiving LP (83% vs. 0%, P = 0.04, and 100% vs. 0%, P = 0.01, respectively). In conclusion, patients with CIF and end-stage liver disease requiring liver transplantation may benefit from combined liver-pancreas transplantation.