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Liver transplantation for hereditary tyrosinemia type I: analysis of the UNOS database

Ronen Arnon1, Rachel Annunziato, Tamir Miloh

  • 1Department of Pediatrics, Mount Sinai School of Medicine, New York, NY, USA. Ronen.arnon@mountsinai.org

Pediatric Transplantation
|April 21, 2011
PubMed

Insights

Liver transplantation (LT) is a vital treatment for children with tyrosinemia type 1 (HT-1) experiencing liver failure. The study shows LT is effective, with survival rates of 90.4%, and its use has declined due to early diagnosis and treatment.

Area of Science:

  • Hepatology
  • Pediatric Gastroenterology
  • Transplant Surgery

Background:

  • Hereditary tyrosinemia type 1 (HT-1) can lead to progressive liver disease and hepatocellular carcinoma (HCC).
  • Liver transplantation (LT) is a critical intervention for HT-1 patients with fulminant liver failure, HCC, or medically refractory decompensated chronic liver disease.

Purpose of the Study:

  • To evaluate the indications for and outcomes of LT in children diagnosed with HT-1.
  • To analyze trends in LT for HT-1 over a 20-year period.

Main Methods:

  • Retrospective analysis of the UNOS database for pediatric patients with HT-1 who underwent LT between October 1987 and May 2008.
  • Statistical comparison of transplant characteristics and survival rates.

Main Results:

  • 125 (1.1%) of 11,467 pediatric patients in the UNOS database had HT-1 requiring LT.
  • The mean age at LT increased significantly from the first decade (1.82 years) to the second decade (3.70 years) of the study (p=0.01).
  • Overall one- and five-year patient survival rates post-LT were 90.4%.

Conclusions:

  • LT remains a valuable option for children with HT-1 facing severe liver complications.
  • The decreasing rate and increasing age at LT suggest improved early diagnosis and treatment with nitisinone (NTBC) have reduced the need for transplantation.

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