[Enzymatic replacement treatment for type I gaucher's disease patients]

Comite d'Avaluacio de Medicaments d'Utilitzacio Hospitalaria (CAMUH)
Record ID 32010000713
Authors' objectives:

To review the scientific evidence available on the efficacy, safety and efficiency of imiglucerase and alpha velaglucerase compared with any treatment alterative, including placebo, in adult patients with confirmed type I GD diagnosis.

Authors' recommendations: The available results confirm the efficiency and safety of imiglucerase and velaglucerase as ERT in type I GD patients. Even so, the clinical significance of the main variable used, increase in haemoglobin, is not clear. Likewise, other variables such as quality of life or bone disease would have been more appropriate as the main variable of the studies.
Project Status: Completed
Year Published: 2011
English language abstract: An English language summary is available
Publication Type: Not Assigned
Country: Spain
MeSH Terms
  • Lysosomal Storage Diseases
Organisation Name: Agencia de Qualitat i Avaluacio Sanitries de Catalunya
Contact Address: Antoni Parada, CAHTA, Roc Boronat, 81-95 (2nd floor), 08005 Barcelona, Spain, Tel. +34 935 513 928, Fax: +34 935 517 510
Contact Name: direccio@aatrm.catsalut.net / aparada@aatrm.catsalut.net
Contact Email: direccio@aatrm.catsalut.net / aparada@aatrm.catsalut.net
Copyright: Catalan Agency for Health Information, Assessment and Quality (CAHIAQ)
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.