title: Liver transplantation in children with cystic fibrosis: experience in our centre and preliminary results with a combined en bloc liver-pancreas graft.
abstract: OBJECTIVE
  Cystic fibrosis (CF) is a multisystemic disease, with some patients developing end-stage liver disease (ESLD), requiring liver transplantation (LT). These children usually present with severe mutations of the CFTR gene. Almost 100% of patients with severe mutations develop exocrine pancreatic insufficiency, leading later to endocrine insufficiency. Immunosuppression accelerates the development of insulin-dependent diabetes (IDD) in transplanted children with CF. Our aims were: (1) to analyze our experience with CF-related ESLD children who received LT, and the relationship to the development of IDD; (2) to report our preliminary results with en bloc liver-pancreas transplantation (CLPT).
  
  
  METHODS
  9 children (6M/3F) with CF and ESLD underwent LT between 1993 and 2010; median age and weight were 12.3 years (range: 5.4-17.0) and 36.7 kg (range: 14.2-58.5), respectively. 4 patients received a whole graft, 4 had reduced grafts (1 split) and 1 underwent CLPT. Immunosuppression followed the protocols at the time of transplantation.
  
  
  RESULTS
  Liver function was restored in all patients and none of them needed re-transplantation. Median follow-up was 105 months (range: 4-206). 1 child died of respiratory failure at 23 months after transplantation while awaiting pulmonary transplantation. Survival (Kaplan-Meier) at 105 months was 87.5%. 4 children already had IDD before transplantation and 3 developed diabetes immediately after transplantation. 2 had not developed IDD at the end of the study: the youngest at the time of LT (5.4 years, follow-up 7.1 years) and the girl who had had CLPT and who recovered normal exocrine and endocrine pancreatic function after transplantation.
  
  
  CONCLUSIONS
  LT is a realistic option to treat CF-related ESLD children. IDD is common in these patients. En bloc liver-pancreas transplantation is an appealing option, since it simultaneously restores exocrine function and prevents IDD. This procedure has clear technical advantages over simultaneous isolated liver and pancreas transplantation.
authors.affiliation: Hospital Universitario La Paz, Pediatric Surgery, Madrid, Spain. mriamf@hotmail.com
authors.forename: M
authors.initials: M
authors.lastname: Miguel
authors.forename: A M
authors.initials: AM
authors.lastname: Andres
authors.forename: M
authors.initials: M
authors.lastname: Lopez-Santamaria
authors.forename: S
authors.initials: S
authors.lastname: Barrena
authors.forename: L
authors.initials: L
authors.lastname: Hierro
authors.forename: F
authors.initials: F
authors.lastname: Hernandez
authors.forename: M
authors.initials: M
authors.lastname: Ramírez
authors.forename: E
authors.initials: E
authors.lastname: Frauca
authors.forename: J L
authors.initials: JL
authors.lastname: Encinas
authors.forename: S
authors.initials: S
authors.lastname: Lopez-Fernandez
authors.forename: P
authors.initials: P
authors.lastname: Jara
authors.forename: J A
authors.initials: JA
authors.lastname: Tovar
mesh_terms: D000293:Adolescent; D002648:Child; D002675:Child, Preschool; D003550:Cystic Fibrosis / Q000503:physiopathology / Q000601:surgery*; D005260:Female; D006801:Humans; D008111:Liver Function Tests; D016031:Liver Transplantation / Q000379:methods* / Q000401:mortality; D008297:Male; D016035:Pancreas Transplantation / Q000379:methods* / Q000401:mortality; D016019:Survival Analysis; D016896:Treatment Outcome