Hepatocyte Transplantation Followed by Auxiliary Liver Transplantation—a Novel Treatment for Ornithine Transcarbamylase Deficiency

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 × 109 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 year

  2008

• Venue

  American Journal of Transplantation

• Publication date

  2008-02-01

• Fields of study

  Medicine

• Identifiers
  DOI 10.1111/j.1600-6143.2007.02058.xPMID 18211511
• External record

  Open on Semantic Scholar

• Source metadata

  Semantic Scholar, PubMed

• No claims are published for this paper.

• No concepts are published for this paper.

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