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

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

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    98 Citations (Scopus)

    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
    Original languageEnglish
    Pages (from-to)452 - 457
    Number of pages6
    JournalAmerican Journal of Transplantation
    Volume8
    Issue number2
    DOIs
    Publication statusPublished - Feb 2008

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