Prevalence and prognosis of paroxysmal nocturnal haemoglobinurea and the clinical and cost-effectiveness of eculizumab
Connock M, Wang D, Fry-Smith A, Moore D
Record ID 32008100046
English
Authors' objectives:
To review the evidence on the prevalence, natural history and prognosis of paroxysmal nocturnal haemoglobinuria (PNH) and to systematically review the clinical effectiveness and cost- effectiveness of eculizumab (Soliris®) treatment.
Authors' recommendations:
· The prevalence of PNH in the UK lies within the limit that defines an ultra-orphan disease.· Thrombosis is a major cause of death. From 3 European studies an average thrombosis rate corresponded to 4.22 per 100 patient years.· Studies of cohorts of European PNH patients indicate the median survival after diagnosis ranges from ~10 to ~27 years. More recently studied cohorts have bettersurvival.· For haemolytic patients with a history of transfusions recruited to trials eculizumab was highly effective at reducing haemolysis and transfusion requirement. It improved quality of life, reduced anaemia and diminished the rate of thrombosis about 7 fold.· Preliminary analysis suggests that the ICER for eculizumab versus SC likely lies between £0.5M and £1.4M per life year gained for patients like those recruited to clinical trials and between £2.8M and £3.2M per life year gained for all diagnosed patients.
Authors' methods:
Systematic Review
Details
Project Status:
Completed
URL for project:
http://www.birmingham.ac.uk/Documents/college-mds/haps/projects/WMHTAC/REPreports/2008/PNH.pdf
Year Published:
2008
URL for published report:
http://www.birmingham.ac.uk/Documents/college-mds/haps/projects/WMHTAC/REPreports/2008/PNH_revisions_to_previous_version.pdf
URL for additional information:
http://www.birmingham.ac.uk/research/activity/mds/projects/HaPS/PHEB/WMHTAC/REP/reports-list.aspx
English language abstract:
An English language summary is available
Publication Type:
Not Assigned
Country:
England
MeSH Terms
- Costs and Cost Analysis
- Antibodies, Monoclonal
- Hemoglobinuria, Paroxysmal
Contact
Organisation Name:
West Midlands Health Technology Assessment Collaboration
Contact Address:
Elaena Donald-Lopez, West Midlands Health Technology Assessment Collaboration, Department of Public Health and Epidemiology, University of Birmingham, Edgbaston, Birmingham B15 2TT Tel: +44 121 414 7450; Fax: +44 121 414 7878
Contact Name:
louise.a.taylor@bham.ac.uk
Contact Email:
louise.a.taylor@bham.ac.uk
Copyright:
West Midlands Health Technology Assessment Collaboration (WMHTAC)
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.