title: Outcomes of liver transplantation for patients with Alagille syndrome: the studies of pediatric liver transplantation experience.
abstract: Alagille syndrome (ALGS) is a multisystem disorder that manifests as childhood cholestasis. Reports of liver transplantation (LT) for patients with ALGS have come largely from single centers, which have reported survival rates of 57% to 79%. The aim of this study was to determine LT outcomes for patients with ALGS. We performed a retrospective analysis of the Studies of Pediatric Liver Transplantation database, which contains information about 3153 pediatric LT recipients. Data were available for 91 patients with ALGS and for 236 age-matched patients with biliary atresia (BA). The frequency of complex cardiac anomalies was lower in the LT group with ALGS versus published ALGS series (5% versus 13%). The pretransplant glomerular filtration rate (GFR) was <90 mL/minute/1.73 m(2) in 18% of the LT patients with ALGS and in 5% of the LT patients with BA (P < 0.001). The height deficit at listing was worse for the ALGS patients (66%) versus the BA patients (22%). The 1-year patient survival rates were 87% for the ALGS patients and 96% for the BA patients (P = 0.002). The deaths in the ALGS group mostly occurred within the first 30 days. No pretransplant factors associated with death were identified in the ALGS group. A survival analysis revealed that biliary (P = 0.02), vascular (P < 0.001), central nervous system (CNS; P < 0.001), and renal complications (P < 0.001) after LT were associated with death in the ALGS group. Renal insufficiency in the ALGS patients worsened after LT, and at 1 year, GFR was <90 mL/minute/1.73 m(2) in 22% of the LT patients with ALGS but in only 8% of the patients with BA (P = 0.0014). More LT pediatric patients with ALGS either were currently receiving special education (50% versus 30% for BA patients, P = 0.02) or had received special education in the past (60% versus 36%, P = 0.01). Vascular, CNS, and renal complications were increased in the ALGS patients after LT, and this reflected multisystem involvement. Although the 1-year survival rate was modestly lower for the ALGS patients versus the BA patients, the clustering of deaths within the first 30 days is notable and warrants increased vigilance and further investigation.
authors.affiliation: Division of Gastroenterology, Hepatology, and Nutrition, Hospital for Sick Children, Toronto, Canada. binita.kamath@sickkids.ca
authors.forename: Binita M
authors.initials: BM
authors.lastname: Kamath
authors.forename: Wanrong
authors.initials: W
authors.lastname: Yin
authors.forename: Heather
authors.initials: H
authors.lastname: Miller
authors.forename: Ravinder
authors.initials: R
authors.lastname: Anand
authors.forename: Elizabeth B
authors.initials: EB
authors.lastname: Rand
authors.forename: Estella
authors.initials: E
authors.lastname: Alonso
authors.forename: John
authors.initials: J
authors.lastname: Bucuvalas
mesh_terms: D000293:Adolescent; D016738:Alagille Syndrome / Q000601:surgery / Q000628:therapy*; D001656:Biliary Atresia / Q000628:therapy; D002648:Child; D002675:Child, Preschool; D015331:Cohort Studies; D005260:Female; D005919:Glomerular Filtration Rate; D006085:Graft Survival; D006801:Humans; D007223:Infant; D016031:Liver Transplantation / Q000379:methods*; D008297:Male; D051437:Renal Insufficiency / Q000601:surgery / Q000628:therapy; D012189:Retrospective Studies; D016896:Treatment Outcome