Hepatocyte transplantation as a treatment for glycogen storage disease type 1a.
Treatment of many inherited disorders of hepatic metabolism is still challenging. Hepatocyte transplantation was done in a 47-year-old woman who had glycogen storage disease type 1a and severe fasting hypoglycaemia. 2 billion viable hepatocytes were infused via an indwelling portal-vein catheter, followed by a triple immunosuppression regimen with mycophenolate mofetil, tacrolimus, and steroids. 9 months after transplantation, on only tacrolimus, she eats a normal diet and can fast for 7 h without experiencing hypoglycaemia. Our results show that hepatocyte transplantation might be an alternative to liver transplantation in glycogen storage disease type 1a.
Muraca
Maurizio
M
Gerunda
Giorgio
G
Neri
Daniele
D
Vilei
Maria-Teresa
MT
Granato
Anna
A
Feltracco
Paolo
P
Meroni
Muzio
M
Giron
Gianpiero
G
Burlina
Alberto B
AB
D002408:Catheters, Indwelling; D017690:Cell Transplantation; D005260:Female; D005500:Follow-Up Studies; D005953:Glycogen Storage Disease Type I / Q000175:diagnosis / Q000601:surgery*; D006801:Humans; D016031:Liver Transplantation; D008875:Middle Aged; D011169:Portal Vein; D016896:Treatment Outcome