title: 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.affiliation: Institute of Liver Studies, King's College Hospital, London, UK.
authors.forename: J
authors.initials: J
authors.lastname: Puppi
authors.forename: N
authors.initials: N
authors.lastname: Tan
authors.forename: R R
authors.initials: RR
authors.lastname: Mitry
authors.forename: R D
authors.initials: RD
authors.lastname: Hughes
authors.forename: S
authors.initials: S
authors.lastname: Lehec
authors.forename: G
authors.initials: G
authors.lastname: Mieli-Vergani
authors.forename: J
authors.initials: J
authors.lastname: Karani
authors.forename: M P
authors.initials: MP
authors.lastname: Champion
authors.forename: N
authors.initials: N
authors.lastname: Heaton
authors.forename: R
authors.initials: R
authors.lastname: Mohamed
authors.forename: A
authors.initials: A
authors.lastname: Dhawan
mesh_terms: D000293:Adolescent; D005260:Female; D022781:Hepatocytes / Q000637:transplantation*; D006801:Humans; D007231:Infant, Newborn; D016031:Liver Transplantation; D008297:Male; D020163:Ornithine Carbamoyltransferase Deficiency Disease / Q000601:surgery* / Q000628:therapy*; D011247:Pregnancy; D011296:Prenatal Diagnosis; D016896:Treatment Outcome