title: Long-term outcome in 42 pediatric liver transplant patients with alpha 1-antitrypsin deficiency: a single-center experience.
abstract: BACKGROUND
  We examined the long-term outcome of transplantation for alpha 1-antitrypsin deficiency (A1ATD).
  
  
  METHODS
  Data were reviewed on 42 transplants in 35 children with A1ATD over 42 yr and compared with 129 transplants in 116 children with biliary atresia (BA).
  
  
  RESULTS
  Over 50% of patients were followed up for >10 yr. A1ATD were older than BA at transplantation, median age, 6.0 vs. 1.0 yr (p < 0.0001), and transplanted earlier in the course of liver failure (total bilirubin, 2.7 mg/dL [1.4-6.9] vs. 9.7 mg/dL [2.9-15.4], p = 0.005). Patient survival was greater in A1ATD than BA: one-yr post-transplant, 82.7% vs. 67.9%; five yr, 76.5% vs. 60.2%; and 10 yr, 76.5% vs. 55.9% (p = 0.03). Death-censored graft survival was similar: one-yr post-transplant, 68.4% vs. 66.2%; five yr, 68.4% vs. 55.8%; and 10 yr, 68.4% vs. 52.5% (p = 0.2). Deaths were from infection, hemorrhage, and graft failure <6 months post-transplant. Patient survival improved at five yr from 33.3% pre-cyclosporine (CSA) (1969-1984) (n = 6) to 76.5% in the CSA era (1985-1994) (n = 17) and 100% with tacrolimus (1995-2006) (n = 12) (p = 0.007).
  
  
  CONCLUSIONS
  The age at transplantation and the degree of liver dysfunction were related to the differences in graft and patient survival between A1AT and BA.
authors.affiliation: Division of Transplantation, Department of Surgery, University of Minnesota, Minneapolis, MN, USA. hughesmg@musc.edu
authors.forename: Michael G
authors.initials: MG
authors.lastname: Hughes
authors.forename: Khalid M
authors.initials: KM
authors.lastname: Khan
authors.forename: Angelika C
authors.initials: AC
authors.lastname: Gruessner
authors.forename: Harvey
authors.initials: H
authors.lastname: Sharp
authors.forename: Mark
authors.initials: M
authors.lastname: Hill
authors.forename: Tun
authors.initials: T
authors.lastname: Jie
authors.forename: Raja
authors.initials: R
authors.lastname: Kandaswamy
authors.forename: Abhinav
authors.initials: A
authors.lastname: Humar
authors.forename: William D
authors.initials: WD
authors.lastname: Payne
authors.forename: Rainer W G
authors.initials: RW
authors.lastname: Gruessner
mesh_terms: D001656:Biliary Atresia / Q000401:mortality*; D002648:Child; D002675:Child, Preschool; D005260:Female; D005500:Follow-Up Studies; D006084:Graft Rejection / Q000188:drug therapy / Q000401:mortality*; D006085:Graft Survival / Q000502:physiology*; D006801:Humans; D007166:Immunosuppressive Agents / Q000627:therapeutic use*; D007223:Infant; D007231:Infant, Newborn; D016030:Kidney Transplantation / Q000401:mortality; D016031:Liver Transplantation / Q000401:mortality*; D008297:Male; D011379:Prognosis; D011446:Prospective Studies; D012189:Retrospective Studies; D015996:Survival Rate; D019896:alpha 1-Antitrypsin Deficiency / Q000401:mortality*
chemical_list: D007166:Immunosuppressive Agents