Enzyme replacement therapy in Gaucher Disease
Hernández-Vásquez A, Ciapponi A, Pichon-Riviere A, Augustovski F, García Martí S, Alcaraz A, Bardach A, López A, Rey-Ares L
Record ID 32016000902
Spanish
Authors' recommendations:
Low-quality evidence suggests that enzyme replacement therapy in Gaucher disease shows positive results in improving hematologic and organ parameters, specially at short-term. No good-quality evidence showing benefits in mortality or quality of life has been found.
In spite of its high cost and the little evidence available, the clinical practice guidelines consulted recommend enzyme replacement therapy in Gaucher disease Type 1 and 3, for patients who meet predetermined criteria, but not for Type 2 because of it poor prognosis and lack of evidence of the effectiveness of treatments. Additionally, US and Latin-American health sponsors include them in their coverage when the patients meet predetermined criteria.
Details
Project Status:
Completed
Year Published:
2016
URL for published report:
www.iecs.org.ar/home-ets/
English language abstract:
An English language summary is available
Publication Type:
Not Assigned
Country:
Argentina
MeSH Terms
- Humans
- Enzyme Replacement Therapy
- Gaucher Disease
- Glucosylceramidase
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.