Skip to main content
Article thumbnail
Location of Repository

Clinical effectiveness and cost-effectiveness of pegvisomant for the treatment of acromegaly: a systematic review and economic evaluation

By Dr. David J. Moore, Y. Adi, M. Connock and Sue Bayliss

Abstract

Background: Acromegaly, an orphan disease usually caused by a benign pituitary tumour, is characterised by hyper-secretion of growth hormone (GH) and insulin-like growth factor I (IGF-1). It is associated with reduced life expectancy, cardiovascular problems, a variety of insidiously progressing detrimental symptoms and metabolic malfunction. Treatments include surgery, radiotherapy and pharmacotherapy. Pegvisomant (PEG) is a genetically engineered GH analogue licensed as a third or fourth line option when other treatments have failed to normalise IGF-1 levels. \ud Methods: Evidence about effectiveness and cost-effectiveness of PEG was systematically reviewed. Data were extracted from published studies and used for a narrative synthesis of evidence. A decision analytical economic model was identified and modified to assess the cost-effectiveness of PEG. \ud Results: One RCT and 17 non-randomised studies were reviewed for effectiveness. PEG substantially reduced and rapidly normalised IGF-1 levels in the majority of patients, approximately doubled GH levels, and improved some of the signs and symptoms of the disease. Tumour size was unaffected at least in the short term. PEG had a generally safe adverse event profile but a few patients were withdrawn from treatment because of raised liver enzymes. An economic model was identified and adapted to estimate the lower limit for the cost-effectiveness of PEG treatment versus standard care. Over a 20 year time horizon the incremental cost-effectiveness ratio was £81,000/QALY and £212,000/LYG. To reduce this to £30K/QALY would require a reduction in drug cost by about one third. \ud Conclusion: PEG is highly effective for improving patients' IGF-1 level. Signs and symptoms of disease improve but evidence is lacking about long term effects on improved signs and symptoms of disease, quality of life, patient compliance and safety. Economic evaluation indicated that if current standards (UK) for determining cost-effectiveness of therapies were to be applied to PEG it would be considered not to represent good value for money

Topics: RC, RM
Publisher: BioMed Central Ltd.
Year: 2009
OAI identifier: oai:wrap.warwick.ac.uk:2679

Suggested articles

Citations

  1. (2006). AcroQol Study Group: Validity and clinical applicability of the acromegaly quality of life questionnaire, AcroQoL: a 6-month prospective study. doi
  2. (2006). AN: Reductions of circulating matrix metalloproteinase 2 and vascular endothelial growth factor levels after treatment with pegvisomant in subjects with acromegaly. doi
  3. (2007). Buchfelder M: Treatment of acromegaly with the GH receptor antagonist pegvisomant in clinical practice: safety and efficacy evaluation from the German Pegvisomant Observational Study. doi
  4. (2006). Cambridgeshire joint prescribing group decision document: Pegvisomant (Somavert, Pfizer) for treatment of acromegaly.
  5. (2002). Cardiovascular risk factors in acromegaly before and after normalization of serum IGF-I levels with the GH antagonist pegvisomant. The Journal of clinical endocrinology and metabolism doi
  6. (2006). Chang TC: Associations of remission status and lanreotide treatment with quality of life in patients with treated acromegaly. doi
  7. (2005). Cotreatment of acromegaly with a somatostatin analog and a growth hormone receptor antagonist. doi
  8. (2005). ER: Assessment of quality of life in patients with uncontrolled vs. controlled acromegaly using the Acromegaly Quality of Life Questionnaire (AcroQoL). Clin Endocrinol (Oxf) doi
  9. (2001). for Reviews and Dissemination University of York: Undertaking systematic reviews of research on effectiveness: CRDs guidance for those carrying out or commisioning reviews. 4. 2nd edition.
  10. (2007). Formulary: doi
  11. (2004). G: Systemic complications of acromegaly: epidemiology, pathogenesis, and management. Endocr Rev doi
  12. (2004). Ghigo E, Minuto F: Cost-of-illness study in acromegalic patients in Italy. doi
  13. (2004). Growth hormone and pituitary radiotherapy, but not serum insulin-like growth factor-I concentrations, predict excess mortality in patients with acromegaly. doi
  14. (2005). H: A nationwide survey of mortality in acromegaly. doi
  15. (2006). I: Elevated transaminases during medical treatment of acromegaly: a review of the German pegvisomant surveillance experience and a report of a patient with histologically proven chronic mild active hepatitis. doi
  16. (2006). KauppinenMakelin R: Quality of life in treated patients with acromegaly. doi
  17. (2002). Klibanski A: Effects of a growth hormone receptor antagonist on bone markers in acromegaly. Clinical endocrinology doi
  18. (2008). Melmed S: Lipodystrophy in patients with acromegaly receiving pegvisomant. doi
  19. (2006). Melmed S: Medical progress: Acromegaly. [Review] [102 refs].
  20. (2005). ML: Glucose homeostasis and safety in patients with acromegaly converted from longacting octreotide to pegvisomant. The Journal of clinical endocrinology and metabolism doi
  21. (2001). ML: Treatment of acromegaly with the growth hormone-receptor antagonist pegvisomant. The New England journal of medicine 2000, 342:1171-1177. 8 . L e l y A J v a n d e r , H ut so n doi
  22. NHS reference costs 2005-06, Mental Health Services: Inpatient Data. MHIPA2 adult: acute care 2007. Ref Type: Data File
  23. (2007). Partnership: Therapeutic Development Assessment Pegvisomant (Somavert®).
  24. (1998). PE: Mortality and cancer incidence in acromegaly: a retrospective cohort study. doi
  25. (2004). PJ: High levels of 150-kDa insulin-like growth factor binding protein three ternary complex in patients with acromegaly and the effect of pegvisomantinduced serum IGF-I normalization. doi
  26. (2005). PU: Alternate-day administration of pegvisomant maintains normal serum insulin-like growth factor-I levels in patients with acromegaly. doi
  27. (1999). Rajasoorya C: Epidemiology of acromegaly. Pituitary doi
  28. (1993). RN: An audit of outcome of treatment in acromegaly.
  29. (2004). Roelfsema F: Decreased quality of life in patients with acromegaly despite long-term cure of growth hormone excess. doi
  30. (2004). Somavert: Summary of Product Characteristics.
  31. (2001). Song F: The effectiveness of somatostatin analogues in the treatment of acromegaly.
  32. (2005). ten Have SMTH: Combined therapy with somatostatin analogues and weekly pegvisomant in active acromegaly[erratum appears in Lancet. doi
  33. (2007). Trainer PJ: Gender, body weight, disease activity, and previous radiotherapy influence the response to pegvisomant. doi
  34. (2007). Trainer PJ: Pegvisomant interference in GH assays results in underestimation of GH levels. doi
  35. (2003). Trainer PJ: Pegvisomant-induced serum insulin-like growth factor-I normalization in patients with acromegaly returns elevated markers of bone turnover to normal. The Journal of clinical endocrinology and metabolism doi
  36. (2005). Trainer PJ: Quality of life (QOL) in patients with acromegaly is severely impaired: use of a novel measure of QOL: acromegaly quality of life questionnaire. doi
  37. (2007). Treatment with growth hormone receptor antagonist in acromegaly: effect on cardiac structure and performance. doi
  38. (2002). Yates AP: Serum lipoprotein changes following IGF-I normalization using a growth hormone receptor antagonist in acromegaly. Clinical Endocrinology doi
  39. (2003). Yates AP: The effect of pegvisomant-induced serum IGF-I normalization on serum leptin levels in patients with acromegaly. Clinical Endocrinology doi

To submit an update or takedown request for this paper, please submit an Update/Correction/Removal Request.