Seminars in Hematology
Volume 45 , Pages S31-S34 , April 2008

Investing in Hemophilia Care: Benefits and Costs for Patients and Society

  • Luciana Scalone

      Affiliations

    • Corresponding Author InformationAddress correspondence to Luciana Scalone, PharmD, DSc, Center of Pharmacoeconomics, Department of Medical Pharmacology, University of Milan, Via Vanvitelli, 32, I-20129, Milan, Italy.

References 

  1. Triemstra M, Rosendaal FR, Smit C, Van der Ploeg HM, Briët E. Mortality in patients with hemophilia (Changes in a Dutch population from 1986 to 1992 and 1973 to 1986). Ann Intern Med. 1995;123:823–827
  2. Leissinger C, Wulff K, Abdou A. Inhibitor prevalence and association with morbidity in severe hemophilia A patients. Blood. 2001;98:535;(abstr)
  3. Soucie JM, Cianfrini C, Janco RL, Kulkarni R, Hambleton J, Evatt B, et al. Joint range-of-motion limitations among young males with hemophilia: Prevalence and risk factors. Blood. 2004;103:2467–2473
  4. Morfini M, Haya S, Tagariello G, Pollmann H, Quintana M, Siegmund B, et al. European study on orthopaedic status of haemophilia patients with inhibitors. Haemophilia. 2007;13:606–612
  5. Darby SC, Keeling DM, Spooner RJ, Wan Kan S, Giangrande PL, Collins PW, et al. The incidence of factor VIII and factor IX inhibitors in the hemophilia population of the UK and their effect on subsequent mortality, 1977-99. J Thromb Haemost. 2004;2:1047–1054
  6. Quintana-Molina M, Martínez-Bahamonde F, González-García E, Romero-Garrido J, Villar-Camacho A, Jiménez-Yuste V, et al. Surgery in haemophilic patients with inhibitor: 20 years of experience. Haemophilia. 2004;10(suppl 2):30–40
  7. Tjønnfjord GE, Brinch L, Gedde-Dahl T, Brosstad FR. Activated prothrombin complex concentrate (FEIBA) treatment during surgery in patients with inhibitors to FVIII/IX. Haemophilia. 2004;10:174–178
  8. Dimichele D. Inhibitors: Resolving diagnostic and therapeutic dilemmas. Haemophilia. 2002;8:280–287
  9. O'Connell N, McMahon C, Smith J, Khair K, Hann I, Liesner R, et al. Recombinant factor VIIa in the management of surgery and acute bleeding episodes in children with haemophilia and high responding inhibitors. Br J Haematol. 2002;116:632–635
  10. Santagostino E, Gringeri A, Mannucci PM. State of care for hemophilia in pediatric patients. Paediatr Drugs. 2002;4:149–157
  11. Ingerslev J. Efficacy and safety of recombinant factor VIIa in the prophylaxis of bleeding in various surgical procedures in hemophilic patients with factor VIII and factor IX inhibitors. Semin Thromb Hemost. 2000;26:425–432
  12. Leissinger CA. Use of prothrombin complex concentrates and activated prothrombin complex concentrates as prophylactic therapy in haemophilia patients with inhibitors. Haemophilia. 1999;5(suppl 3):25–32
  13. Shapiro A, Gilchrist GS, Hoots WK, Cooper HA, Gastineau DA. Prospective, randomised trial of two doses of rFVIIa (NovoSeven) in haemophlia patients with inhibitors undergoing surgery. Thromb Haemost. 1998;80:773–778
  14. Lusher J, Ingerslev J, Roberts H, Hedner U. Clinical experience with recombinant factor VIIa. Blood Coagul Fibrinolysis. 1998;9:119–128
  15. Negrier C, Goudemand J, Sultan Y, Bertrand M, Rothschild C, Lauroua P. Multicenter retrospective study on the utilization of FEIBA in France in patients with factor VIII and factor IX inhibitors (French FEIBA Study Group. Factor Eight Inhibitor Bypassing Activity). Thromb Haemost. 1997;77:1113–1119
  16. Mariani G, Ghirardini A, Bellocco R. Immune tolerance in hemophilia—Principal results from the international registry (Report of the factor VIII and IX Subcommittee). Thromb Haemost. 1994;72:155–158
  17. Hedner U, Glazer S, Pingel K, Alberts KA, Blombäck M, Schulman S, et al. Successful use of rFVIIa in a patient with severe haemophilia A subjected to synovectomy. Lancet. 1988;2:1193
  18. Hutchinson RJ, Penner JA, Hensinger RN. Anti-inhibitor coagulant complex (Autoplex) in hemophilia inhibitor patients undergoing synovectomy. Pediatrics. 1983;71:631–633
  19. Sjamsoedin LJ, Heijnen L, Mauser-Bunschoten EP, van Geijlswijk JL, van Houwelingen H, van Asten P, et al. The effect of activated prothrombin complex concentrate (FEIBA) on joint and muscle bleeding in patients with hemophilia A and antibodies to factor VIII (A double-blind clinical trial). N Engl J Med. 1981;305:717–721
  20. Gringeri A, Mantovani LG, Scalone L, Mannucci PM. Cost of care and quality of life for patients with hemophilia complicated by inhibitors: The COCIS study group. Blood. 2003;102:2358–2363
  21. Scalone L, Mantovani LG, Mannucci PM, Gringeri A. The COCIS Study Investigators: Quality of life is associated to the orthopaedic status in haemophilic patients with inhibitors. Haemophilia. 2006;12:154–162
  22. Scalone L, Gringeri A, Mannucci PM, Von Mackensen S, Mantovani LG. Costs and quality of life in hemophilia: Comparison between patients with and patients without inhibitors. Value Health. 2005;8:A5;(abstr)
  23. Morfini M, Auerswald G, Kobelt RA, Rivolta GF, Rodriguez-Martorell J, Scaraggi FA, et al. Prophylactic treatment of haemophilia patients with inhibitors: Clinical experience with recombinant factor VIIa in European Haemophilia Centres. Haemophilia. 2007;13:502–507
  24. Valentino LA. FEIBA prophylaxis for patients with haemophilia and inhibitors. Haemophilia. 2006;12(suppl 5):26–31
  25. Stephens JM, Joshi AV, Sumner M, Botteman MF. Health economic review of recombinant activated factor VII for treatment of bleeding episodes in hemophilia patients with inhibitors. Expert Opin Pharmacother. 2007;8:1127–1136
  26. Solimeno LP, Perfetto OS, Pasta G, Santagostino E. Total joint replacement in patients with inhibitors. Haemophilia. 2006;12(suppl 3):113–116
  27. Allen G, Aledort L. Therapeutic decision-making in inhibitor patients. Am J Hematol. 2006;81:71–72
  28. Astermark J, Morado M, Rocino A, van den Berg HM, von Depka M, Gringeri A, et al. Current European practice in immune tolerance induction therapy in patients with haemophilia and inhibitors. Haemophilia. 2006;12:363–371
  29. Monahan PE, Aledort LM. Hemophilia Inhibitor Study Group: Factors affecting choice of hemostatic agent for the hemophilia patient with an inhibitor antibody. Am J Hematol. 2004;77:346–350
  30. Astermark J, Rocino A, Von Depka M, Van Den Berg HM, Gringeri A, Mantovani LG, et al. Current use of by-passing agents in Europe in the management of acute bleeds in patients with haemophilia and inhibitors. Haemophilia. 2007;13:38–45
  31. Konkle BA, Ebbesen LS, Erhardtsen E, Bianco RP, Lissitchkov T, Rusen L, et al. Randomized, prospective clinical trial of recombinant factor VIIa for secondary prophylaxis in hemophilia patients with inhibitors. J Thromb Haemost. 2007;5:1904–1913
  32. Leissinger CA. Prophylaxis in haemophilia patients with inhibitors. Haemophilia. 2006;12(suppl 6):67–73
  33. Samuelson PA, Nordhaus WD. Economics. Columbus, OH: McGraw-Hill; 2004;
  34. Mantovani LG, Monzini MS, Mannucci PM, Scalone L, Villa M, Gringeri A, et al. Differences between patients', physicians' and pharmacists' preferences for treatment products in haemophilia: A discrete choice experiment. Haemophilia. 2005;11:589–597
  35. Borghetti F, Gringeri A, Scalone L, von Mackensen S, Mantovani LG. Preferences towards coagulation factor concentrates used to treat hemophilic patients with inhibitors: The patients', physicians' and pharmacists' perspective. Haematologica. 2007;92:225;(abstr)

 STATEMENT OF CONFLICT OF INTEREST: The author declares no conflict of interest.

PII: S0037-1963(08)00059-0

doi: 10.1053/j.seminhematol.2008.03.019

Seminars in Hematology
Volume 45 , Pages S31-S34 , April 2008