[Enzyme replacement therapy in mucopolysaccharidoses I, II and VI; 2011]

Sunyer B, Paladio N, Almazan C
Record ID 32011001422
Authors' objectives:

To assess the scientific evidence available on the efficacy, safety and efficiency of treatment with laronidase, idursulfase and galsulfase, compared with any alternative treatment in patients with MPS I, II and VI, respectively.

Authors' recommendations: ERT is proposed as an alternative treatment for MPS I, II and VI. Laronidase, idursulfase and galsulfase are ERT drugs –with orphan designation– approved by the EMA to treat MPS I, II and VI, respectively. ERT, in adult patients with light-moderate MPS phenotypes and in the short term, improve the distance walked and breathing capacity, they reduce organomegaly and are well-tolerated. However, given the limited apparent benefit of this improvement, as well as the lack of evidence about the impact on quality of life and on the neurological disease evolution in patients with MPS I and II, as well as the high cost-opportunity of these treatments in a context of limited resources, their indication conditions should be defined with the consensus of professionals and experts. In parallel, studies must be promoted to permit the assessment of the impact of ERT on the quality of life and the progression of the disease. In addition, monitoring the treatment with ERT in existing registers should be favoured to be able to assess the effects of these treatments in the long term, and in clinical practice conditions.
Project Status: Completed
Year Published: 2011
English language abstract: An English language summary is available
Publication Type: Not Assigned
Country: Spain
MeSH Terms
  • Mucopolysaccharidoses
  • Mucopolysaccharidosis I
  • Mucopolysaccharidosis III
  • Mucopolysaccharidosis VI
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.