Enzyme replacement therapy for people with Fabry's disease - horizon scanning review

Record ID 32002000851
Authors' objectives:

To summarise the current research evidence on enzyme replacement therapy for people with Fabry's disease.

Authors' recommendations: - Clinical impact: There is a small known patient group of up to 200 symptomatic men that would be able to benefit from enzyme replacement therapy for Fabrys disease. The role of the therapy for asymptomatic and screen detected cases is yet to be determined. - Service impact: People with symptomatic diagnosed Fabrys disease are seen by specialists at 4 major centres in the UK. The introduction of specific therapy will affect the service provided, but may in the long term reduce the need for renal support and incidence of major cardiac events. - Patient issues: Although small, the patient group is knowledgeable about these new therapies and the role it may play in the long-term management of their disease. - Financial and overall NHS impact: The overall NHS impact could be large. The existence of a treatment could also lead to the identification of more people with the disease but currently asymptomatic, increasing the demand for enzyme replacement therapy and the cost impact.
Authors' methods: Overview
Project Status: Completed
Year Published: 2001
URL for published report: http://www.hsric.nihr.ac.uk/search
English language abstract: An English language summary is available
Publication Type: Not Assigned
Country: England, United Kingdom
MeSH Terms
  • Fabry Disease
Organisation Name: NIHR Horizon Scanning Centre
Contact Address: The NIHR Horizon Scanning Centre, Department of Public Health, Epidemiology, and Biostatistics, School of Health and Population Sciences, University of Birmingham, 90 Vincent Drive, Edgbaston, Birmingham, B15 2SP. United Kingdom. Tel: +44 121 414 7831, Fax: +44 121 2269
Contact Name: c.packer@bham.ac.uk
Contact Email: c.packer@bham.ac.uk
Copyright: National Horizon Scanning Centre
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.