Authors' objectives:

This review assessed the evidence about the clinical effectiveness and cost effectiveness of pegvisomant for the treatment of acromegaly in patients whose IGF- 1 levels fail to normalise in response to other treatments.

Authors' results and conclusions: Clinical effectiveness reviewEighteen publications were included. These described one RCT (pegvisomant vs. placebo) and 17 non-randomised or subgroup studies mostly of before and after design. Very little evidence extended beyond one year follow-up.Main findings:Pegvisomant rapidly normalised IGF-1 in the majority of patients and substantially reduced IGF-1 in all compliant patients. Reduced IGF-1 was accompanied on average by a doubling in GH levels. Tumour size, at least in the short term, was apparently unaffected by pegvisomant treatment but intermittent monitoring by MRI is widely recommended in view of raised GH levels and the short-term nature of evidence. The drug had a generally safe adverse event profile but in a few patients treatment induced raised liver enzymes that required treatment withdrawal; in a few patients withdrawal needed to be permanent. Treatment was associated with improvement in some of the signs and symptoms of the disease. Limited evidence about disease-risk markers from small, non-randomised, short-term studies indicated that pegvisomant treatment may reduce risk of cardiovascular disease, of diabetes and of maladjusted bone turnover.

Authors' recommendations: Pegvisomant treatment for acromegaly is highly effective for improving patients’ IGF- 1 level. Evidence is lacking about the long term effects of treatment in respect of improved signs and symptoms of disease, quality of life, patient compliance and safety. An economic evaluation using a simple decision tree model indicated that pegvisomant was very unlikely to represent good value for money according to currently applied standards. The prevalence of acromegaly (~58 / 106) falls just outside the definition for an ultraorphan disease (< 20 / 106) but within the orphan criterion; as such pegvisomant might be considered by some wholly or partially exempt from usually applied valuefor- money criteria and subject to other criteria as yet ill defined or incompletely applied by national or local reimbursement bodies.

Authors' methods: Systematic review

Project Status: Completed

URL for project: http://www.birmingham.ac.uk/Documents/college-mds/haps/projects/WMHTAC/REPreports/2007/Pegviso.pdf

Year Published: 2007

URL for published report: 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

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)