ORTHOTOPIC LIVER TRANSPLANTATION FOR ALAGILLE SYNDROME

Abstract

P705 Aims: Alagille syndrome is a dominantly inherited multisystem disorder involving the liver, heart, eyes, face, and skeleton, caused by mutations in Jagged 1 with a prevalence of approximately one in 70,000 live births. Orthotopic Liver transplantation (OLTx) is considered the mainstay of treatment of the syndrome’s associated end-stage liver disease. Methods: From October 1997 through January 2004, 242 pediatric OLTx were performed in 215 patients (pts) at “Ospedali Riuniti di Bergamo”. Alagille Syndrome was the indication in 18 pts with a median age af 2,74 years (range 0,7-16,7) who represent the subject for these report. We reviewed the data of these pts for technical features of the OLTx, incidence and type of complications, the medical conditions related to the syndrome, the need for retransplantation and the patient and graft survival. Results: There were 10 females and 8 males, weighting a median of 11.5 kg (range 6-27 kg). During the preoperative work-up 8 pts (44,4%) underwent diagnostic cardiac cathetherization, which led to 3 angioplasties of the pulmonary artery and 2 surgical procedures (closure of interventricular defect and correction of aortic coarctation). 12 Pts (66.7%) received a LLS graft, 6 pts (33.3%) received a whole graft. Median follow up was 874 days (range 5-2034). Major surgical complications requiring a reintervention occured in 8 patients (44.4%) (3 laparotomy deiscence, 2 hemoperitoneum, 2 occlusions, 1 deiscence of bowel anastomosis). No hepatic artery thrombosis was observed. Infections with positive blood cultures were diagnosed in 8 patients (44,4 %). Four patients had biliary complications (22,2%), 3 (stenosis) were successfully treated by percutaneous techniques (PTC) and one (fistula) by retransplantation (with LLS graft) after failed PTC. Four pts (22,2%) had acute rejection treated with steroids. Two pts (11.1%) died, one in the early post-operative period for intraabdominal bleeding and one suffered a fatal PTLD 3 years after tranplantation. All the other pts are alive with an actuarial pts survival of 94% at one year and 81% at 5 years. The actuarial graft survival is 89% at 1 year and 76% at 5 years. Conclusions: Patient with Alagille syndrome, despite the associated anomalies of the cardiovascular system, can be treated successfully by a combined approach between cardiologist, radiologist, cardio-thoracic and liver transplant surgeons. With careful planning and operative managment the results are comparable to those obtained with other more common cholestatic diseases.