title: Manifestations of transthyretin-related familial amyloidotic polyneuropathy: long-term follow-up of Japanese patients after liver transplantation.
abstract: OBJECTIVE
  To observe which symptoms of transthyretin-related familial amyloidotic polyneuropathy (FAP) progressed in the long term after liver transplantation (LT), focusing on cardiac, kidney, and ocular symptoms.
  
  
  METHODS
  We reviewed the medical records of 34 Japanese patients with FAP, who underwent LT between 1994 and 2006. The mean follow-up period (± SD) after LT was 9.6 ± 3.4 years. Of the 34 patients, 30 had FAP amyloidogenic transthyretin (ATTR) Val30Met, 1 had FAP ATTR Ser50Ile, and 3 had FAP ATTR Tyr114Cys.
  
  
  RESULTS
  The 10-year survival rates from the onset of FAP and from the time of LT were 100% and 91.4%, respectively. Progression of ocular amyloidosis was seen in 17 (50%) patients, 13 of whom had de novo amyloid deposits in the vitreous body; progression of cardiac amyloidosis was seen in 10 (29%) patients, 4 of whom had newly granular sparkling echo on echocardiography, and 9 of whom had newly implanted pacemakers or implantable cardioverter-defibrillators. Although the mean serum creatinine levels did not increase significantly after LT in any of the patients, the estimated glomerular filtration rate had decreased significantly by 7 years after LT.
  
  
  CONCLUSIONS
  Although LT is life-saving for patients with FAP, we observed progression of the ocular and cardiac symptoms of FAP in a significant number of these patients over the long term after LT.
authors.affiliation: Department of Transplantation and Pediatric Surgery, Kumamoto University, 1-1-1 Honjo, Kumamoto, 860-8556, Japan.
authors.forename: Yuki
authors.initials: Y
authors.lastname: Ohya
authors.forename: Sadahisa
authors.initials: S
authors.lastname: Okamoto
authors.forename: Masayoshi
authors.initials: M
authors.lastname: Tasaki
authors.forename: Mitsuharu
authors.initials: M
authors.lastname: Ueda
authors.forename: Hirofumi
authors.initials: H
authors.lastname: Jono
authors.forename: Konen
authors.initials: K
authors.lastname: Obayashi
authors.forename: Kazuhisa
authors.initials: K
authors.lastname: Takeda
authors.forename: Hideaki
authors.initials: H
authors.lastname: Okajima
authors.forename: Katsuhiro
authors.initials: K
authors.lastname: Asonuma
authors.forename: Ryuhei
authors.initials: R
authors.lastname: Hara
authors.forename: Hidenobu
authors.initials: H
authors.lastname: Tanihara
authors.forename: Yukio
authors.initials: Y
authors.lastname: Ando
authors.forename: Yukihiro
authors.initials: Y
authors.lastname: Inomata
mesh_terms: D000328:Adult; D028227:Amyloid Neuropathies, Familial / Q000401:mortality / Q000473:pathology / Q000601:surgery*; D018450:Disease Progression; D005123:Eye / Q000473:pathology; D005500:Follow-Up Studies; D006801:Humans; D007564:Japan; D007668:Kidney / Q000378:metabolism / Q000473:pathology; D007677:Kidney Function Tests; D016031:Liver Transplantation; D008297:Male; D008875:Middle Aged; D009206:Myocardium / Q000473:pathology; D012189:Retrospective Studies; D015996:Survival Rate; D016896:Treatment Outcome