Replacement enzyme therapy in Fabry's disease
Pichon Riviere A, Augustovski F, Cernadas C, Ferrante D, Regueiro A, Garcia Marti S
Record ID 32005000652
Spanish
Authors' objectives:
The aim of this study was to assess the use of replacement enzyme therapy in Fabry's disease
Authors' results and conclusions:
Four randomized clinical trials were carried out where the replacement enzyme therapy was analyzed in patients with Fabry's disease. In all the cases, they are well-designed studies that showed promising findings. However, they were studies in few patients and with short follow-up periods (less than 1 year) where only intermediate findings were analyzed, e.g. if the therapy was able to remove the gb3 deposits in the endothelium and if it could control pain. These trials did not analyze long-term hard results as the ability to reduce mortality or to prevent major complications as kidney failure or severe cardiovascular or brain events.
Authors' recommendations:
Though these results are promising and it is the only specific therapy available, still more studies are needed to clear up the long-term therapy benefits, especially as regards mortality reduction and severe heart, kidney and brain events and the occurrence of side effects when continuing treatment beyond the periods studied.
Authors' methods:
Overview
Details
Project Status:
Completed
URL for project:
http://www.iecs.org.ar/
Year Published:
2004
English language abstract:
An English language summary is available
Publication Type:
Not Assigned
Country:
Argentina
MeSH Terms
- Renal Replacement Therapy
- alpha-Galactosidase
- Fabry Disease
- Isoenzymes
- Kidney Diseases
- Kidney Failure, Chronic
- Recombinant Proteins
Contact
Organisation Name:
Institute for Clinical Effectiveness and Health Policy
Contact Address:
Dr. Emilio Ravignani 2024, Buenos Aires - Argentina, C1414 CABA
Contact Name:
info@iecs.org.ar
Contact Email:
info@iecs.org.ar
Copyright:
Institute for Clinical Effectiveness and Health Policy (IECS)
This is a bibliographic record of a published health technology assessment from a member of INAHTA or other HTA producer. No evaluation of the quality of this assessment has been made for the HTA database.