title: Treatment of ornithine transcarbamylase deficiency in girls by auxiliary liver transplantation: conceptual changes in a living-donor program.
abstract: OBJECTIVE
  Ornithine transcarbamylase (OTC) deficiency is an X-chromosome-linked genetic disorder resulting in hyperammonemia hepatic dysfunction, coma, and serious neurological sequelae. This report describes an experience in treating this condition with living-related liver transplantation.
  
  
  METHODS
  Three children with OTC were treated with a living-related liver transplantation. Potential donors were evaluated with the allopurinol loading test to exclude heterozygotes. FK506 and low-dose steroids were used for immunosuppression. Auxiliary partial orthotopic transplantation (APOLT) was used in two of the cases.
  
  
  RESULTS
  All three children survived and are doing well without protein restriction.
  
  
  CONCLUSIONS
  These observations suggest that in OTC removal of the native liver is not necessary because (1) a partial liver segment with normal enzyme activity corrects the hyperammonemia, (2) in case of graft failure the native liver is an available backup, and (3) the native liver has the ability to recover function. As long as living-related donors serve as the main source of donor organs, APOLT is a safer approach to liver transplantation in OTC.
authors.affiliation: Department of Transplant Surgery, Kyoto University Hospital, Japan.
authors.forename: M
authors.initials: M
authors.lastname: Kasahara
authors.forename: T
authors.initials: T
authors.lastname: Kiuchi
authors.forename: K
authors.initials: K
authors.lastname: Uryuhara
authors.forename: Y
authors.initials: Y
authors.lastname: Ogura
authors.forename: K
authors.initials: K
authors.lastname: Takakura
authors.forename: H
authors.initials: H
authors.lastname: Egawa
authors.forename: K
authors.initials: K
authors.lastname: Asonuma
authors.forename: S
authors.initials: S
authors.lastname: Uemoto
authors.forename: Y
authors.initials: Y
authors.lastname: Inomata
authors.forename: K
authors.initials: K
authors.lastname: Tanaka
mesh_terms: D000592:Amino Acid Metabolism, Inborn Errors / Q000503:physiopathology / Q000601:surgery*; D000641:Ammonia / Q000097:blood*; D002675:Child, Preschool; D005260:Female; D006801:Humans; D008099:Liver / Q000000981:diagnostic imaging / Q000473:pathology; D008111:Liver Function Tests; D016031:Liver Transplantation; D019520:Living Donors; D014057:Tomography, X-Ray Computed; D016896:Treatment Outcome
chemical_list: D000641:Ammonia