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Hepatocyte Transplantation Followed by Auxiliary Liver Transplantation—a Novel Treatment for Ornithine Transcarbamylase Deficiency
Author(s) -
Puppi J.,
Tan N.,
Mitry R. R.,
Hughes R. D.,
Lehec S.,
MieliVergani G.,
Karani J.,
Champion M. P.,
Heaton N.,
Mohamed R.,
Dhawan A.
Publication year - 2008
Publication title -
american journal of transplantation
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 2.89
H-Index - 188
eISSN - 1600-6143
pISSN - 1600-6135
DOI - 10.1111/j.1600-6143.2007.02058.x
Subject(s) - ornithine transcarbamylase deficiency , medicine , transplantation , ornithine transcarbamylase , liver transplantation , ornithine carbamoyltransferase , hepatocyte , orotic acid , urea cycle , ornithine , biochemistry , biology , in vitro , amino acid , arginine
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 × 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.