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

Abstract

† These authors contributed equally to this work.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.

Encephalopathy by hyperammonemia in teenagers

“…En la serie más extensa hallada la supervivencia fue del 91%, la hiperamoniemia y la dieta restrictiva fueron erradicadas en estos pacientes 14 . Actualmente también se valora la posibilidad de realizar transplante de hepatocitos como puente a un transplante ortotópico parcial hepático 15 .…”

Advancing Transplantation

“…Thus, cell maturity may prove to be critical for diseases requiring functional differentiated cells. In some instances of end-stage liver disease, and for inborn errors of liver metabolism, an entire liver may not be required and auxiliary partial liver transplantation has been suggested as a therapeutic option, 243 although a graft representing at least 35% of a liver would be necessary.…”

Adult Stem Cell Therapy in Chronic Liver Diseases

“…[kg]) has been demonstrated in human clinical trials [7], it has been reported that replacement of at least 10% of the hepatocellular mass is needed to compensate for missing or inactive proteins in genetic inborn errors of metabolism [8].…”