title: Liver transplantation for hereditary tyrosinemia type I: analysis of the UNOS database.
abstract: Patients with HT-1 can develop progressive liver disease and have a high incidence of HCC. LT is indicated in patients with fulminant liver failure, HCC or decompensated chronic liver disease refractory to NTBC. To determine the need for LT and outcomes after LT in children with HT-1. Children with HT-1 who had LT between 10/1987 and 5/2008 were identified from the UNOS database. Of 11,467 children in the UNOS database, 125 (1.1%) required LT secondary to HT-1. Mean age at LT was two and half yr (s.d. ± 3.6 yr). Mean age at LT during the first 10 yr of the study (1.82, s.d. ± 2.86 yr) was significantly lower than in the last decade (3.70, s.d. ± 4.42 yr), p = 0.01. Nearly half of the patients (58, 46.4%) were transplanted between 1988 and 1992. Overall, one- and five-yr patient survival was 90.4% and 90.4%, respectively. LT is a valuable option for children with HT-1 with fulminant liver failure or when medical treatment fails. The rate of LT for children with HT-1 has decreased and age at transplant increased over the last decade most probably reflecting the effect of early diagnosis and treatment with NTBC.
authors.affiliation: Department of Pediatrics, Mount Sinai School of Medicine, New York, NY, USA. Ronen.arnon@mountsinai.org
authors.forename: Ronen
authors.initials: R
authors.lastname: Arnon
authors.forename: Rachel
authors.initials: R
authors.lastname: Annunziato
authors.forename: Tamir
authors.initials: T
authors.lastname: Miloh
authors.forename: Melissa
authors.initials: M
authors.lastname: Wasserstein
authors.forename: Hiroshi
authors.initials: H
authors.lastname: Sogawa
authors.forename: Monique
authors.initials: M
authors.lastname: Wilson
authors.forename: Frederick
authors.initials: F
authors.lastname: Suchy
authors.forename: Nanda
authors.initials: N
authors.lastname: Kerkar
mesh_terms: D000293:Adolescent; D016009:Chi-Square Distribution; D002648:Child; D016208:Databases, Factual; D005260:Female; D005500:Follow-Up Studies; D006084:Graft Rejection; D006085:Graft Survival; D006801:Humans; D017093:Liver Failure / Q000209:etiology* / Q000503:physiopathology / Q000601:surgery*; D008111:Liver Function Tests; D016031:Liver Transplantation / Q000009:adverse effects / Q000379:methods* / Q000706:statistics & numerical data; D008297:Male; D011183:Postoperative Complications / Q000453:epidemiology / Q000503:physiopathology; D012189:Retrospective Studies; D018570:Risk Assessment; D012720:Severity of Illness Index; D016896:Treatment Outcome; D020176:Tyrosinemias / Q000150:complications* / Q000175:diagnosis