[Allogenic liver transplantation: a form of "gene therapy" in metabolic diseases. Munich results and a review].
BACKGROUND
Liver transplantation is the method of choice for metabolic diseases and end-stage liver failure.


METHODS
At the Klinikum Grosshadern we have performed liver transplantation for inborn errors of metabolism in 24 patients (5.3% of all transplantations, 16 adults, age 39 +/- 13 years; 8 children, age 9 +/- 3 years); 19 patients received a transplant for end-stage liver disease, and in 5 cases because of fulminant hepatic failure.


RESULTS
Twenty-four patients received 27 transplants. In 3 cases, a split-liver transplantation was performed; one patient received a combined lung-liver graft. The 5-year survival rate for children is 100% and for adults 68%.


CONCLUSIONS
Liver transplantation for inborn errors of metabolism not only replaces the diseased organ, but also leads to complete reversal of the metabolic defect.
Stangl
M J
MJ
Chirurgische Klinik und Poliklinik, Ludwig-Maximilians-Universität, Klinikum Grosshadern, München. Manfred.Stangl@gch.med.uni-muenchen.de
Beuers
U
U
Schauer
R
R
Lang
T
T
Gerbes
A
A
Briegel
J
J
Da Silva
L
L
Schildberg
F W
FW
Rau
H G
HG
D000328:Adult; D002648:Child; D003414:Crigler-Najjar Syndrome / Q000175:diagnosis / Q000235:genetics / Q000601:surgery; D015316:Genetic Therapy; D006008:Glycogen Storage Disease / Q000175:diagnosis / Q000601:surgery; D006432:Hemochromatosis / Q000175:diagnosis / Q000235:genetics / Q000601:surgery; D006467:Hemophilia A / Q000175:diagnosis / Q000601:surgery; D006527:Hepatolenticular Degeneration / Q000175:diagnosis / Q000235:genetics / Q000601:surgery; D006801:Humans; D006938:Hyperlipoproteinemia Type II / Q000175:diagnosis / Q000601:surgery; D006959:Hyperoxaluria / Q000175:diagnosis / Q000601:surgery; D007231:Infant, Newborn; D008107:Liver Diseases / Q000175:diagnosis / Q000235:genetics / Q000601:surgery*; D016031:Liver Transplantation / Q000379:methods / Q000401:mortality; D008661:Metabolism, Inborn Errors / Q000175:diagnosis / Q000235:genetics / Q000601:surgery*; D008875:Middle Aged; D013997:Time Factors; D014184:Transplantation, Homologous; D020176:Tyrosinemias / Q000175:diagnosis / Q000601:surgery; D019896:alpha 1-Antitrypsin Deficiency / Q000175:diagnosis / Q000235:genetics / Q000601:surgery