Cost-utility analysis of deferasirox compared to standard therapy with desferrioxamine for patients requiring iron chelation therapy in the United Kingdom

References

• Zurlo MG, De Stefano P, Borgna-Pignatti C, . Survival and causes of death in thalassaemia major. Lancet 1989;2: 27–30
   PubMed Web of Science ®Google Scholar
• Olivieri NF, Brittenham GM. Iron-chelating therapy and the treatment of thalassemia. Blood 1997;89: 739–61
   PubMed Web of Science ®Google Scholar
• Kushner JP, Porter JP, Olivieri NF. Secondary iron overload. Hematology Am Soc Hematol Educ Program 2001:47–61
   PubMedGoogle Scholar
• National Horizon Scanning Centre. New and Emerging Technology Briefing: Deferasirox (ICL670) for iron overload. New and Emerging Technology Briefing. The University Of Birmingham. January 2005. http://www.pcpoh.bham.ac.uk/publichealth/horizon/pdf_files/2005reports/deferasirox.pdf [Last accessed 2 April 2008]
   Google Scholar
• Rose C, Gattermann N, Glimm E, . Deferasirox (Exjade®, ICL670), the novel, once-daily oral iron chelator, is well tolerated and effective in treating transfusional iron overload in patients with a range of rare anaemias. 11th Congress of the European Haematology Association, The Netherlands: Amsterdam, 15–18 June 2006 [poster 0022]
   Google Scholar
• Yang LP, Keam SJ, Keating GM. Deferasirox: a review of its use in the management of transfusional chronic iron overload. Drugs 2007;67: 2211–30
   PubMed Web of Science ®Google Scholar
• World Health Organisation. Thalassaemia and other haemoglobinopathies. http://www.who.int/gb/ebwha/pdf_files/EB118/B118_5-en.pdf [Last accessed 2 April 2008]
   Google Scholar
• Malcovati L, Porta MG, Pascutto C, . Prognostic factors and life expectancy in myelodysplastic syndromes classified according to WHO criteria: a basis for clinical decision making. J Clin Oncol 2005;23: 7594–603
   PubMed Web of Science ®Google Scholar
• Borgna-Pignatti C, Cohen A. Evaluation of a new method of administration of the iron chelating agent deferoxamine. J Pediatr 1997;130: 86–8
   PubMed Web of Science ®Google Scholar
• Gabutti V, Piga A. Results of long-term iron-chelating therapy. Acta Haematol 1996;95: 26–36
   PubMed Web of Science ®Google Scholar
• Brittenham GM, Griffith PM, Nienhuis AW, . Efficacy of deferoxamine in preventing complications of iron overload in patients with thalassemia major. N Engl J Med 1994;331: 567–73
   PubMed Web of Science ®Google Scholar
• Novartis Pharmaceuticals UK Ltd. Deferasirox Summary of Product Characteristics. Available from http://emc.medicines.org.uk [Last accessed 2 April 2008]
   Google Scholar
• Cappellini MD, Cohen A, Piga A, . A phase 3 study of deferasirox (ICL670), a once-daily oral iron chelator, in patients with beta-thalassemia. Blood 2006;107: 3455–62
   PubMed Web of Science ®Google Scholar
• Piga A, Galanello R, Forni GL, . Randomized phase II trial of deferasirox (Exjade, ICL670), a once-daily, orally-administered iron chelator, in comparison to deferoxamine in thalassemia patients with transfusional iron overload. Haematologica 2006;91: 873–80
   PubMed Web of Science ®Google Scholar
• Porter J, Vichinsky E, Rose C. A phase II study with ICL670 (Exjade®), a once-daily oral iron chelator, in patients with various transfusion-dependent anaemias and iron overload. 46th American Society of Haematology meeting, San Diego, USA, 4–7 December 2004 [abstract 3193]
   Google Scholar
• Vichinsky E, Fischer R, Pakbaz Z, . Satisfaction and convenience of chelation therapy in patients with sickle cell disease (SCD): Comparison between deferasirox (Exjade®, ICL670) and deferoxamine (DFO). 47th American Society of Haematology meeting, Atlanta, USA, 10–13 December 2005 [abstract 2334]
   Google Scholar
• European Medicines Agency (EMEA). Exjade European Public Assessment Report (EPAR). http://www.emea.europa.eu/humandocs/Humans/EPAR/exjade/exjade.htm [Last accessed 2 April 2008]
   Google Scholar
• Novartis Pharmaceuticals UK Ltd. Desferal Summary of Product Characteristics. Available from http://emc.medicines.org.uk [Last accessed 2 April 2008]
   Google Scholar
• Abetz L, Baladi JF, Jones P, Rofail D. The impact of iron overload and its treatment on quality of life: results from a literature review. Health Qual Life Outcomes 2006;4: 73
   PubMed Web of Science ®Google Scholar
• Rofail D, Abetz L, Viala M, . Health-related quality of life, satisfaction, and self-esteem in iron overload. UK PRO results, 2006 [unpublished data]
   Google Scholar
• Vichinsky E, Onyekwere O, Porter J, . A randomised comparison of deferasirox versus deferoxamine for the treatment of transfusional iron overload in sickle cell disease. Br J Haematol 2007;136: 501–8
   PubMed Web of Science ®Google Scholar
• Cappellini MD, Bejaoui M, Agaoglu L, . Patient satisfaction with deferasirox (Exjade®, ICL670) an oral form of chelation therapy versus desferrioxamine an infused chelation therapy. 47th American Society of Haematology meeting, Atlanta, USA, 10–13 December 2005 [abstract 2704]
   Google Scholar
• National Institute of Health and Clinical Excellence (NICE). Guide to the Methods of Technology Appraisal. 2004 April. Available from: http://www.nice.org.uk/aboutnice/howwework/devnicetech/technologyappraisalprocessguides/guide_to_the_methods_of_technology_appraisal_reference_n0515.jsp [Last accessed 2 April 2008]
   Google Scholar
• Porter J, Galanello R, Saglio G, . Relative response of patients with myelodysplastic syndromes and other transfusion-dependent anaemias to deferasirox (ICL670): a 1-yr prospective study. Eur J Haematol 2008;80: 168–76
   PubMed Web of Science ®Google Scholar
• British National Formulary Issue 54, London: British Medical Association, September 2007.
   Google Scholar
• Desrosiers MP, Payne K, Proskorovsky I, . Estimated total annual costs of infused iron chelation therapy in the United Kingdom. 11th Congress of the European Haematology Association, Amsterdam, The Netherlands, 15–18 June 2006 [poster 0810]
   Google Scholar
• Mansel R. Cost-utility analysis of anastrozole versus tamoxifen as adjuvant therapy in 245 postmenopausal women with early breast cancer: a UK national health service perspective. 29th European Society for Medical Oncology, Vienna, Austria, 29 Oct–2 Nov 2004
   Google Scholar
• Curtis L, Netten A. Unit costs of health and social care. The University of Kent, Personal Social Services Research Unit, 2005
   Google Scholar
• Biolab Medical Unit. http://www.biolab.co.uk/singles.html [Last accessed 2 April 2008]
   Google Scholar
• Tolley K, Oyee J, Jewitt K, Ossa D. UK community derived utilities using time trade off for oral versus subcutaneous iron chelation therapy for the treatment of chronic iron overload. Presented at the 10th Annual European International Society of Pharmacoeconomics and Outcomes Research (ISPOR), Dublin, Ireland, 20–23 Oct 2007
   Google Scholar
• Ipsos MORI. website. Available from http://www.mori.com [Last accessed 5 Dec 2006]
   Google Scholar
• Osborne RH, De Abreu LR, Dalton A, . Quality of life related to oral versus subcutaneous iron chelation: a time trade-off study. Value Health 2007;10: 451–6
   PubMed Web of Science ®Google Scholar
• Anderson JP, Moser RJ. Parasite screening and treatment among Indochinese refugees. Cost-benefit/utility and the General Health Policy Model. JAMA 1985;253: 2229–35
   PubMed Web of Science ®Google Scholar
• Barry HC, Ebell MH, Hickner J. Evaluation of suspected urinary tract infection in ambulatory women: a cost-utility analysis of office-based strategies. J Fam Pract 1997;44: 49–60
   PubMed Web of Science ®Google Scholar
• Ebell MH, Warbasse L, Brenner C. Evaluation of the dyspeptic patient: a cost-utility study. J Fam Pract 1997;44: 545–55
   PubMed Web of Science ®Google Scholar
• Rosner B, Glynn RJ, Lee ML. The Wilcoxon signed rank test for paired comparisons of clustered data. Biometrics 2006;62: 185–92
   PubMed Web of Science ®Google Scholar
• Delea TE, Sofrygin O, Thomas SK, . Cost effectiveness of once-daily oral chelation therapy with deferasirox versus infusional deferoxamine in transfusion-dependent thalassaemia patients: US healthcare system perspective. Pharmacoeconomics 2007;25: 329–42
   PubMed Web of Science ®Google Scholar
• Rose C, Brechignac S, Vassilief D, . Positive impact of iron chelation therapy (CT) on survival in regularly transfused MDS patientsA prospective analysis by the GFM. 49th American Society of Haematology meeting, Atlanta, Georgia, USA, 8–11 December 2007 [abstract 249]
   Google Scholar
• Borgna-Pignatti C, De Stefano P, Zonta L, . Growth and sexual maturation in thalassemia major. J Pediatr 1985;106: 150–5
   PubMed Web of Science ®Google Scholar
• Arboretti R, Tognoni G, Alberti D. Pharmacosurveillance and quality of care of thalassaemic patients. A large scale epidemiological survey. Eur J Clin Pharmacol 2001;56: 915–22
   PubMed Web of Science ®Google Scholar
• Fischer R, Longo F, Nielsen P, . Monitoring long-term efficacy of iron chelation therapy by deferiprone and desferrioxamine in patients with beta-thalassaemia major: application of SQUID biomagnetic liver susceptometry. Br J Haematol 2003;121: 938–48
   PubMed Web of Science ®Google Scholar
• Cappellini MD, Bejaoui M, Agaoglu L, . Prospective evaluation of patient-reported outcomes during treatment with deferasirox or deferoxamine for iron overload in patients with beta-thalassemia. Clin Ther 2007;29: 909–17
   PubMed Web of Science ®Google Scholar
• Delea TE, Hagiwara M, Thomas SK, . Outcomes, utilization, and costs among thalassemia and sickle cell disease patients receiving deferoxamine therapy in the United States. Am J Hematol 2008;83: 263–70
   PubMed Web of Science ®Google Scholar
• Fung EB, Harmatz P, Milet M, . Morbidity and mortality in chronically transfused subjects with thalassemia and sickle cell disease: a report from the multi-center study of iron overload. Am J Hematol 2007;82: 255–65
   PubMed Web of Science ®Google Scholar
• Vichinsky E, Coates T, Thompson A, . Long-term efficacy and safety of deferasirox (Exjade, ICL670), a once-daily oral iron chelator, in patients with sickle cell disease (SCD). 49th American Society of Haematology meeting. Atlanta Georgia, USA, 8–11 December 2007 [abstract 3395]
   Google Scholar
• Drummond M, Sculpher M, Torrence G. Methods for the economic evaluation of health care programmes, 3rd edn. Oxford: Oxford University Press, 2005
   Google Scholar
• Kuo HT, Tsai MY, Peng CT, Wu KH. Pilot study on the “quality of life” as reflected by psychosocial adjustment of children with thalassemia major undergoing iron-chelating treatment in western Taiwan. Hemoglobin 2006;30: 291–9
   PubMed Web of Science ®Google Scholar
• Ismail A, Campbell MJ, Ibrahim HM, Jones GL. Health related quality of life in Malaysian children with thalassaemia. Health Qual Life Outcomes 2006;4: 39
   PubMed Web of Science ®Google Scholar