Hepatocyte transplantation in a 4-year-old girl with peroxisomal biogenesis disease: technique, safety, and metabolic follow-up.
Transplantation
; 76(4): 735-8, 2003 Aug 27.
Article
en En
| MEDLINE
| ID: mdl-12973120
ABSTRACT
Hepatocyte transplantation is an investigational alternative to orthotopic liver transplantation to treat liver based inborn errors of metabolism. We report successful hepatocyte transplantation in a 4-year-old girl with infantile Refsum disease. Hepatocytes were isolated from the left liver segment of two male donors using a classic two-step perfusion method. Fresh cells were transplanted first and then cryopreserved cells, for a total of 2 billion cells. Total bile acids and abnormal dihydroxycoprostanoïc acid markedly decreased in the patient's serum, indicating resolution of cholestasis and re-population of liver cells. Pipecholic acid decreased by 40% and c26c22 fatty acid ratio by 36% after 18 months. Donor chromosomes sequences were detected on biopsy posttransplant, indicating engraftment. Hepatocyte transplantation is a safe and promising technique in the treatment of rare inborn errors of metabolism. Future improvements of cell viability and prevention of apoptosis may increase engraftment and subsequent re-population.
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Colección:
01-internacional
Base de datos:
MEDLINE
Asunto principal:
Trastorno Peroxisomal
/
Hepatocitos
Tipo de estudio:
Observational_studies
/
Prognostic_studies
Límite:
Child, preschool
/
Female
/
Humans
Idioma:
En
Revista:
Transplantation
Año:
2003
Tipo del documento:
Article
País de afiliación:
Bélgica