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Cost-Utility Analysis of Primary Prophylaxis versus Treatment On-Demand for Individuals with Severe Haemophilia

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Abstract

Objective: To assess the cost effectiveness of primary prophylaxis with clotting factor instead of treatment following a bleed (on-demand) for individuals with severe haemophilia.

Design: Different data sources on the clinical effects and costs of treatments were combined using a Markov model.

Setting: English treatment centres.

Perspective: UK societal.

Participants: Hypothetical cohorts of 100 individuals with severe haemophilia A or B or severe von Willebrands disease.

Interventions: Primary prophylaxis treatment on-demand with clotting factor.

Outcome measures: Costs, quality-adjusted life-years (QALYs) and incremental cost per QALY in UK pounds (£, 1999/2000 values).

Results: The baseline results showed that treating individuals with severe haemophilia A/severe von Willebrands disease or severe haemophilia B with primary prophylaxis instead of treatment on-demand cost an additional £46 500 and £8600 per QALY gained, respectively. However, the results were extremely sensitive to a number of factors including the clotting factor unit cost, the time between prophylactic doses and the discount rate.

Conclusions: Despite the high costs of treatment, primary prophylaxis was cost effective compared with treatment on-demand in some scenarios. Primary prophylaxis is more likely to be cost effective for individuals with severe haemophilia B compared with individuals with severe haemophilia A/severe von Willebrands disease. Further research is required to assess the relationship between methods of clotting factor infusion and health-related quality-of-life.

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References

  1. Nilsson IM. Hemophilia. Stockholm: Pharmacia Plasma Products, 1994

    Google Scholar 

  2. Pettersson H, Ahlberg A, Nilsson IM. A radiologic classification of hemophilic arthropathy. Clin Orthop 1980; 149: 153–9

    PubMed  Google Scholar 

  3. Pettersson H, Nilsson IM, Hedner U, et al. Radiological evaluation of prophylaxis in severe haemophilia. Acta Paediatr Scand 1981; 70 (4): 565–70

    Article  PubMed  CAS  Google Scholar 

  4. Bohn RL, Avorn J, Glynn RJ, et al. Prophylactic use of factor VIII: an economic evaluation. Thromb Haemost 1998; 79 (5): 932–7

    PubMed  CAS  Google Scholar 

  5. Miners AH, Sabin CA, Tolley KH, et al. Assessing the effectiveness and cost-effectiveness of prophylaxis against bleeding in patients with severe haemophilia and severe von Willebrand’s disease. J Intern Med 1998; 244 (6): 515–22

    Article  PubMed  CAS  Google Scholar 

  6. Smith PS, Teutsch SM, Shaffer PA, et al. Episodic versus prophylactic infusions for hemophilia A: a cost-effectiveness analysis. J Pediatr 1996; 129 (3): 424–31

    Article  PubMed  CAS  Google Scholar 

  7. Szucs TD, Öffner A, Schramm W. Socioeconomic impact of haemophilia care: results of a pilot study. Haemophilia 1996; 2: 211–7

    Article  Google Scholar 

  8. Beck JR, Pauker SG. The Markov process in medical prognosis. Med Decis Making 1983; 3 (4): 419–58

    Article  PubMed  CAS  Google Scholar 

  9. Drummond MF, O’Brien B, Stoddart GL, et al. Methods for the economic evaluation of health care programmes. 2nd ed. Oxford: Oxford University Press, 1997

    Google Scholar 

  10. Williams A. Economics of coronary artery bypass grafting. BMJ 1985; 291 (6491): 326–9

    Article  PubMed  CAS  Google Scholar 

  11. Triemstra AHM, Smit C, Van der Ploeg HM, et al. Mortality in patients with hemophilia. Ann Intern Med 1995; 123: 823–7

    PubMed  CAS  Google Scholar 

  12. Office for National Statistics. Age specific death rates by gender, 1996. London: HMSO, 1998

    Google Scholar 

  13. Seagroatt V, Tan HS, Goldacre M, et al. Elective total hip replacement: incidence, emergency readmission rate, and postoperative mortality. BMJ 1991; 303 (6815): 1431–5

    Article  PubMed  CAS  Google Scholar 

  14. Miners AH, Sabin CA, Tolley KH, et al. Primary prophylaxis for individuals with severe haemophilia: how many hospital visits could treatment prevent? J Intern Med 2000; 247: 493–9

    Article  PubMed  CAS  Google Scholar 

  15. Miners AH, Sabin CA, Tolley KH, et al. Assessing health-related quality-of-life in individuals with haemophilia. Haemophilia 1999; 5: 378–85

    Article  PubMed  CAS  Google Scholar 

  16. Kind P, Dolan P, Gudex C, et al. Variations in population health status: results from a United Kingdom national questionnaire survey. BMJ 1998; 316 (7133): 736–41

    Article  PubMed  CAS  Google Scholar 

  17. Laupacis A, Bourne R, Rorabeck C, et al. The effect of elective total hip replacement on health-related quality of life. J Bone Joint Surg Am 1993; 75 (11): 1619–26

    PubMed  CAS  Google Scholar 

  18. Allain JP. Dose requirement for replacement therapy in hemophilia A. Thromb Haemost 1979; 42 (3): 825–31

    PubMed  CAS  Google Scholar 

  19. Lofqvist T, Nilsson IM, Berntorp E, et al. Haemophilia prophylaxis in young patients: a long-term follow-up. J Intern Med 1997; 241 (5): 395–400

    Article  PubMed  CAS  Google Scholar 

  20. Nilsson IM, Berntorp E, Lofqvist T, et al. Twenty-five years’ experience of prophylactic treatment in severe haemophilia A and B. J Intern Med 1992; 232 (1): 25–32

    Article  PubMed  CAS  Google Scholar 

  21. UK Haemophilia Centre Directors Organization Executive Committee. Guidelines on therapeutic products to treat haemophilia and other hereditary coagulation disorders. Haemophilia 1997; 3: 63–77

    Article  Google Scholar 

  22. Carlsson M, Bjorkman S, Berntorp E. Multidose pharmacokinetics of factor IX: implications for dosing in prophylaxis. Haemophilia 1998; 4: 83–8

    Article  PubMed  CAS  Google Scholar 

  23. Rickard KA. Guidelines for therapy and optimal dosages of coagulation factors for treatment of bleeding and surgery in haemophilia. Haemophilia 1995; 1 Suppl. 1: 8–13

    Article  Google Scholar 

  24. Fitzpatrick R, Shortall E, Sculpher M, et al. Primary total hip replacement surgery: a systematic review of outcomes and modelling of cost-effectiveness associated with different prostheses. Health Technology Assessment Report 1998; 2 (20)

    Google Scholar 

  25. Office for National Statistics. New earnings survey. London: HMSO, 1998

    Google Scholar 

  26. White A, Nicolass G, Foster K, et al. Health survey for England 1991. London: HMSO, 1993

    Google Scholar 

  27. Stevens A, Colin-Jones D, Gabbay J. ’Quick and clean’: authoritative health technology assessment for local health care contracting. Health Trends 1995; 27 (2): 37–42

    PubMed  CAS  Google Scholar 

  28. Szucs TD, Offner A, Kroner B, et al. Resource utilisation in haemophiliacs treated in Europe: results from the European study on socioeconomic aspects of haemophilia care. The European Socioeconomic Study Group. Haemophilia 1998; 4 (4): 498–501

    Article  PubMed  CAS  Google Scholar 

  29. Aledort LM. Hemophilia: yesterday, today, and tomorrow. Mount Sinai J Med 1996; 63 (3–4): 225–35

    CAS  Google Scholar 

  30. Aledort LM, Bohn RL. Prophylaxis and continuous infusion for hemophilia: can we afford it? Blood Coagul Fibrinolysis 1996; 7 Suppl. 1: S35–7

    PubMed  Google Scholar 

  31. Bátorová A, Martinowitz U, Makai F, et al. Adjusted dose continuous infusion of F VIII is superior to intermittent bolus therapy for major surgery in hemophilia A [abstract]. Haemophilia 1998; 4: 182

    Google Scholar 

  32. Bátorová A, Makai F, Kopác C, et al. Continuous infusion of recombinant factor VIII (KogenateR) for surgery in hemophilia A [abstract]. Haemophilia 1998; 4: 36

    Google Scholar 

  33. Bona RD, Weinstein RA, Weisman SJ, et al. The use of continuous infusion of factor concentrates in the treatment of hemophilia. Am J Hematol 1989; 32 (1): 8–13

    Article  PubMed  CAS  Google Scholar 

  34. Campbell PJ, Rickard KA. Continuous and intermittent infusion of coagulation factor concentrates in patients undergoing surgery: a single centre Australian experience. Aust N Z J Med 1998; 28 (4): 440–5

    Article  PubMed  CAS  Google Scholar 

  35. Dasani H, Jones JAH, Loran C, et al. Effective haemostasis with mononine factor IX continuous infusion for two major surgeries and one large thigh haematoma in three patients with haemophilia B [abstract]. Haemophilia 1998; 4: 184

    Google Scholar 

  36. Doughty HA, Coles J, Parmar K, et al. The successful removal of a bleeding intracranial tumour in a severe haemophiliac using an adjusted dose continuous infusion of monoclonal factor VIII. Blood Coagul Fibrinolysis 1995; 6 (1): 31–4

    Article  PubMed  CAS  Google Scholar 

  37. Hathaway WE, Christian MJ, Clarke SL, et al. Comparison of continuous and intermittent factor VIII concentrate therapy in hemophilia A. Am J Hematol 1984; 17: 85–8

    Article  PubMed  CAS  Google Scholar 

  38. Hay CR, Doughty HI, Savidge GF. Continuous infusion of factor VIII for surgery and major bleeding. Blood Coagul Fibrinolysis 1996; 7 Suppl. 1: S15–9

    PubMed  CAS  Google Scholar 

  39. Ljung RCR. Prophylactic treatment in Sweden: overtreatment or optimal model? Haemophilia 1998; 4 (4): 409–12

    Article  PubMed  CAS  Google Scholar 

  40. Löfqvist T, Nilsson IM, Petersson C. Orthopaedic surgery in hemophilia: 20 years’ experience in Sweden. Clin Orthop 1996; 332: 232–41

    Article  PubMed  Google Scholar 

  41. Manco-Johnson MJ. Family issues in continuous infusion therapy with factor VIII. Blood Coagul Fibrinolysis 1996; 7 Suppl. 1: S21–5

    PubMed  CAS  Google Scholar 

  42. Martinowitz U, Schulman S, Gitel S, et al. Adjusted dose continuous infusion of factor VIII in patients with haemophilia A. Br J Haematol 1992; 82 (4): 729–34

    Article  PubMed  CAS  Google Scholar 

  43. Martinowitz UP, Schulman S. Continuous infusion of factor concentrates: review of use in hemophilia A and demonstration of safety and efficacy in hemophilia B. Acta Haematol 1995; 94 Suppl. 1: 35–42

    Article  PubMed  Google Scholar 

  44. McMillan CW, Webster WP, Roberts HR, et al. Continuous intravenous infusion of factor VIII in classic haemophilia. Br J Haematol 1970; 18 (6): 659–67

    Article  PubMed  CAS  Google Scholar 

  45. Morfini M, Messori A, Longo G. Factor VIII pharmacokinetics: intermittent infusion versus continuous infusion. Blood Coagul Fibrinolysis 1996; 7 Suppl. 1: S11–4

    PubMed  CAS  Google Scholar 

  46. Richardson NGB, Miller AL, O’shaughnessy DF. Successful treatment of acute subdural haemorrhage with continuous intravenous infusion of factor VIII on a 17 year old with haemophilia A [letter]. Haemophilia 1996; 2 (173): 176

    Google Scholar 

  47. Rochat C, McFadyen ML, Schwyzer R, et al. Continuous infusion of intermediate-purity factor VIII in haemophilia A patients undergoing elective surgery. Haemophilia 1999; 5: 181–6

    Article  PubMed  CAS  Google Scholar 

  48. Schulman S, Gitel S, Zivelin A, et al. The feasibility of using concentrates containing factor IX for continuous infusion. Haemophilia 1995; 1: 103–10

    Article  Google Scholar 

  49. Schulman S, Martinowitz U. Continuous infusion instead of bolus injections of factor concentrate? Haemophilia 1996; 2: 189–91

    Article  Google Scholar 

  50. Schulman S, Smith O, Wallensten R, et al. Continuous infusion of factor IX for surgery with a chemically treated and virus treated filtered concentrate (Nanotiv) [abstract]. Haemophilia 1998; 4: 182

    Google Scholar 

  51. Schulman S, Wallensten R, White B, et al. Efficacy of a high purity, chemically treated and nanofiltered factor IX concentrate for continuous infusion in haemophilia patients undergoing surgery. Haemophilia 1999; 5: 96–100

    Article  PubMed  CAS  Google Scholar 

  52. Tengborn L, Berntorp E. Continuous infusion of factor IX concentrate to induce immune tolerance in two patients with haemophilia B. Haemophilia 1998; 4 (1): 56–9

    Article  PubMed  CAS  Google Scholar 

  53. Varon D, Schulman S, Beshari D, et al. Home therapy with continuous infusion of factor VIII after minor surgery or serious haemorrhage. Haemophilia 1996; 2: 207–10

    Article  Google Scholar 

  54. Varon D, Martinowitz U. Continuous infusion therapy in haemophilia. Haemophilia 1998; 4 (4): 431–5

    Article  PubMed  CAS  Google Scholar 

  55. Collins PW, Khair KS, Liesner R, et al. Complications experienced with central venous catheters in children with congenital bleeding disorders. Br J Haematol 1997; 99 (1): 206–8

    Article  PubMed  CAS  Google Scholar 

  56. Liesner RJ, Vora AJ, Hann IM, et al. Use of central venous catheters in children with severe congenital coagulopathy. Br J Haematol 1995; 91 (1): 203–7

    Article  PubMed  CAS  Google Scholar 

  57. Miller K, Buchanan GR, Zappa S, et al. Implantable venous access devices in children with hemophilia: a report of low infection rates. J Pediatr 1998; 132 (6): 934–8

    Article  PubMed  CAS  Google Scholar 

  58. Ragni MV, Hord JD, Blatt J. Central venous catheter infection in haemophiliacs undergoing prophylaxis or immune tolerance with clotting factor concentrate. Haemophilia 1997; 3: 90–5

    Article  Google Scholar 

  59. Santagostino E, Gringeri A, Muca-Perja M, et al. A prospective clinical trial of implantable central venous access in children with haemophilia. Br J Haematol 1998; 102 (5): 1224–8

    Article  PubMed  CAS  Google Scholar 

  60. van den Berg HM, Fischer K, Roosendaal G, et al. The use of the Port-A-Cath in children with haemophilia: a review. Haemophilia 1998; 4 (4): 418–20

    Article  Google Scholar 

  61. Vidler V, Richards M, Vora A. Central venous catheter-associated thrombosis in severe haemophilia. Br J Haematol 1999; 104: 461–4

    Article  PubMed  CAS  Google Scholar 

  62. Warrier I, Baird-Cox K, Lusher JM. Use of central venous catheters in children with haemophilia: one haemophilia treatment centre experience. Haemophilia 1997; 3: 194–8

    Article  Google Scholar 

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Acknowledgements

The authors would like to thank Martin Buxton for comments on an earlier draft of the manuscript.

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Authors and Affiliations

  1. Health Economics Research Group, Brunel University, Uxbridge, UK

    Alexander H. Miners

  2. Department of Primary Care and Population Sciences, Royal Free Hospital and University College Medical School, London, UK

    Caroline A. Sabin

  3. GlaxoSmithKline, Stockley Park, Uxbridge, UK

    Keith H. Tolley

  4. Royal Free Hampstead NHS Trust, Katharine Dormandy Haemophilia Centre, London, UK

    Christine A. Lee

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  1. Alexander H. Miners
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  2. Caroline A. Sabin
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Correspondence to Alexander H. Miners.

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Miners, A.H., Sabin, C.A., Tolley, K.H. et al. Cost-Utility Analysis of Primary Prophylaxis versus Treatment On-Demand for Individuals with Severe Haemophilia. Pharmacoeconomics 20, 759–774 (2002). https://doi.org/10.2165/00019053-200220110-00005

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