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
  • Complement Inactivating Agents
  • Hemoglobinuria, Paroxysmal
  • Antibodies, Monoclonal, Humanized
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: <p>University of Birmingham</p>
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