Hepatocyte transplantation as a treatment for glycogen storage disease type 1a.

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.

• Authors: Maurizio Muraca, Giorgio Gerunda, Daniele Neri, Maria-Teresa Vilei, Anna Granato, Paolo Feltracco, Muzio Meroni, Gianpiero Giron, Alberto B Burlina
• Journal: Lancet (London, England) (Lancet) Vol. 359 Issue 9303 Pg. 317-8 (Jan 26 2002) ISSN: 0140-6736 [Print] England
• PMID: 11830200 (Publication Type: Case Reports, Letter, Research Support, Non-U.S. Gov't)

Topics

• Catheters, Indwelling
• Cell Transplantation
• Female
• Follow-Up Studies
• Glycogen Storage Disease Type I (diagnosis, surgery)
• Humans
• Liver Transplantation
• Middle Aged
• Portal Vein
• Treatment Outcome