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 c26:c22 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.
journal_name
Transplantationjournal_title
Transplantationauthors
Sokal EM,Smets F,Bourgois A,Van Maldergem L,Buts JP,Reding R,Bernard Otte J,Evrard V,Latinne D,Vincent MF,Moser A,Soriano HEdoi
10.1097/01.TP.0000077420.81365.53subject
Has Abstractpub_date
2003-08-27 00:00:00pages
735-8issue
4eissn
0041-1337issn
1534-6080journal_volume
76pub_type
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