title: Long-term survival after liver transplantation in patients with familial amyloid polyneuropathy.
abstract: OBJECTIVE
  Familial amyloid polyneuropathy (FAP), which is a fatal disorder inherited in an autosomal dominant fashion, is characterized by systemic accumulation of polymerized transthyretin (TTR) in the peripheral nerves and systemic organs. Liver transplantation has become an accepted treatment of this disorder because it stops the major production of amyloidogenic TTR. However, improved survival of transplant patients compared with that of nontransplant patients has not been sufficiently demonstrated. This study investigated whether transplantation improved the long-term outcome of patients by comparing the survival of patients who had transplantations with that of patients who had not had transplantations.
  
  
  METHODS
  Eighty consecutive patients with FAP Val30Met who visited Kumamoto University Hospital between January 1990 and December 2010 were studied. The transplant group consisted of 37 patients who had a partial hepatic graft via living donor transplantation in Japan or who underwent liver transplantation in Sweden, Australia, or the United States. The nontransplant group consisted of 43 patients with FAP. Survival was evaluated by using Kaplan-Meier analysis, and the difference in survival was examined via the log-rank test.
  
  
  RESULTS
  The transplant group had prolonged survival (p < 0.001) compared with the nontransplant group. The estimated probability of survival at 10 years was 56.1% for the nontransplant group vs 100% for the transplant group.
  
  
  CONCLUSIONS
  Liver transplantation should be considered as an effective treatment in clinical management of patients with FAP Val30Met.
  
  
  METHODS
  This study provides Class III evidence that liver transplantation prolongs survival in patients with FAP Val30Met.
authors.affiliation: Departments of Neurology, Graduate School of Medical Sciences, Kumamoto University, Kumamoto, Japan. taro-yamashita@fc.kuh.kumamoto-u.ac.jp
authors.forename: T
authors.initials: T
authors.lastname: Yamashita
authors.forename: Y
authors.initials: Y
authors.lastname: Ando
authors.forename: S
authors.initials: S
authors.lastname: Okamoto
authors.forename: Y
authors.initials: Y
authors.lastname: Misumi
authors.forename: T
authors.initials: T
authors.lastname: Hirahara
authors.forename: M
authors.initials: M
authors.lastname: Ueda
authors.forename: K
authors.initials: K
authors.lastname: Obayashi
authors.forename: M
authors.initials: M
authors.lastname: Nakamura
authors.forename: H
authors.initials: H
authors.lastname: Jono
authors.forename: M
authors.initials: M
authors.lastname: Shono
authors.forename: K
authors.initials: K
authors.lastname: Asonuma
authors.forename: Y
authors.initials: Y
authors.lastname: Inomata
authors.forename: M
authors.initials: M
authors.lastname: Uchino
mesh_terms: D000328:Adult; D000368:Aged; D028227:Amyloid Neuropathies, Familial / Q000401:mortality* / Q000601:surgery*; D005260:Female; D006801:Humans; D053208:Kaplan-Meier Estimate; D016031:Liver Transplantation / Q000401:mortality*; D008297:Male; D008875:Middle Aged; D015996:Survival Rate; D017741:Survivors; D016896:Treatment Outcome