Enzyme replacement therapy for Fabry's disease
Fischbacher C
Record ID 32004000135
English
Authors' objectives:
This study aims to assess the effectiveness of enzyme replacement therapy for Fabry's disease.
Authors' recommendations:
We found two RCTs of enzyme replacement therapy (ERT) for Fabry's disease. One RCT reported similar improvements in pain and quality of life with ERT and with placebo, while the other RCT reported larger improvements in pain and quality of life with ERT than with placebo. Both studies reported substantial improvement in histological appearance of affected tissues and reduction in the concentration of Gb3 with ERT compared with placebo.
The evidence to date suggests that, based on important proxy outcomes, enzyme replacement therapy (ERT) is probably beneficial in men with the classic form of Fabry's disease. However, this conclusion would be strengthened by consistent, long-term evidence of clinical benefit.
Authors' methods:
Review
Details
Project Status:
Completed
URL for project:
http://www.wihrd.soton.ac.uk
Year Published:
2003
English language abstract:
An English language summary is available
Publication Type:
Not Assigned
Country:
England
MeSH Terms
- Fabry Disease
- Lysosomal Storage Diseases, Nervous System
- alpha-Galactosidase
- Isoenzymes
Contact
Organisation Name:
Wessex Institute for Health Research and Development
Contact Address:
Pauline King. Wessex Institute for Health Research and Development, Boldrewood Medical School, Bassett Crescent East, Highfield, Southampton. SO16 7PX Tel. +44 1703 595661 Fax +44 1703 595662
Copyright:
Bazian Ltd, Wessex Institute for Health Research and Development
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