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.
HEPATOCYTE TRANSPLANTATION AS A TREATMENT FOR GLYCOGEN STORAGE DISEASE TYPE 1a / Muraca, M.; Gerunda, G.; Neri, D.; Vilei, Mt; Granato, A.; Feltracco, P.; Meroni, M.; Giron, G.; Burlina, Ab. - In: THE LANCET. - ISSN 0140-6736. - STAMPA. - 359:9303(2002), pp. 317-318. [10.1016/S0140-6736(02)07529-3]
HEPATOCYTE TRANSPLANTATION AS A TREATMENT FOR GLYCOGEN STORAGE DISEASE TYPE 1a
Gerunda, G.;
2002
Abstract
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.
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