Hepatocyte transplantation followed by auxiliary liver transplantation--a novel treatment for ornithine transcarbamylase deficiency

Am J Transplant. 2008 Feb;8(2):452-7. doi: 10.1111/j.1600-6143.2007.02058.x.

Abstract

We report the first successful use of hepatocyte transplantation as a bridge to subsequent auxiliary partial orthotopic liver transplantation (APOLT) in a child antenatally diagnosed with severe ornithine transcarbamylase (OTC) deficiency. A total of 1.74 x 10(9) fresh and cryopreserved hepatocytes were administered intraportally into the liver over a period of 6 months. Immunosuppression was with tacrolimus and prednisolone. A sustained decrease in ammonia levels and a gradual increase in serum urea were observed except during episodes of sepsis in the first 6 months of life. The patient was able to tolerate a normal protein intake and presented a normal growth and neurological development. APOLT was successfully performed at 7 months of age. We conclude that hepatocyte transplantation can be used in conjunction with APOLT as an effective treatment for severe OTC-deficient patients, improving neurodevelopmental outcomes.

Publication types

  • Case Reports
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adolescent
  • Female
  • Hepatocytes / transplantation*
  • Humans
  • Infant, Newborn
  • Liver Transplantation*
  • Male
  • Ornithine Carbamoyltransferase Deficiency Disease / surgery*
  • Ornithine Carbamoyltransferase Deficiency Disease / therapy*
  • Pregnancy
  • Prenatal Diagnosis
  • Treatment Outcome