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

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.

• Authors: J Puppi, N Tan, R R Mitry, R D Hughes, S Lehec, G Mieli-Vergani, J Karani, M P Champion, N Heaton, R Mohamed, A Dhawan
• Journal: American journal of transplantation : official journal of the American Society of Transplantation and the American Society of Transplant Surgeons (Am J Transplant) Vol. 8 Issue 2 Pg. 452-7 (Feb 2008) ISSN: 1600-6143 [Electronic] United States
• PMID: 18211511 (Publication Type: Case Reports, Journal Article, Research Support, Non-U.S. Gov't)

Topics

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