Prophylaxis for adults with haemophilia: One size does not fit all

Blood Transfusion

Volumn 10, Issue 2, 2012, Pages 169-173

  Fischer, Kathelijn ^a,b  

^a UNIVERSITY MEDICAL CENTER UTRECHT   (Netherlands)

^b UNIVERSITY MEDICAL CENTER UTRECHT   (Netherlands)

Author keywords

[No Author keywords available]

Indexed keywords

BLOOD CLOTTING FACTOR 8; RECOMBINANT BLOOD CLOTTING FACTOR 8; RECOMBINANT BLOOD CLOTTING FACTOR 9;

AREA UNDER THE CURVE; ARTHROPATHY; DENMARK; EUROPE; GENETIC HETEROGENEITY; HEMOPHILIA; HUMAN; NETHERLANDS; PHENOTYPE; PROPHYLAXIS; PROTEIN FUNCTION; REVIEW; SWEDEN;

COAGULANTS; FACTOR IX; FACTOR VIII; HEMOPHILIA A; HUMANS;

EID: 84860531391     PISSN: 17232007     EISSN: None     Source Type: Journal    
DOI: 10.2450/2012.0174-11     Document Type: Review

References (33)

1. Ahlberg A. Haemophilia in Sweden VII. Incidence, treatment and prophylaxis of arthropathy and other musculo-skeletal manifestations of haemophilia A and B. Acta Orthop Scand 1965; 77 (Suppl.): 5-99.
2. den Uijl I, Fischer K, Van der Bom JG, et al. Analysis of low frequency bleeding data: the association of joint bleeds according to baseline FVIII activity levels. Haemophilia 2011; 17: 41-4.
3. Nilsson IM, Blomback M, Ahlberg A. Our experience in Sweden with prophylaxis on haemophilia. Bibl Haematol 1970; 34: 111-24.
4. Löfqvist T, Nilsson IM, Berntorp E, Pettersson H. Haemophilia prophylaxis in young patients- a long-term follow-up. J Intern Med 1997; 241: 395-400. (Pubitemid 27244664)
5. Van den Berg HM, Fischer K, Mauser-Bunschoten EP, et al. Long term outcome of individualised prophylactic treatment of children with severe haemophilia. Br J Haematol 2001; 107: 561-5. (Pubitemid 32222823)
6. Manco-Johnson MJ, Abshire TC, Shapiro AD, et al. Prophylaxis versus episodic treatment to prevent joint disease in boys with severe hemophilia. N Engl J Med 2007; 357: 535-44. (Pubitemid 47236278)
7. Gringeri A, Lundin B, von Mackensen S, et al. A randomized clinical trial of prophylaxis in children with hemophilia A (the ESPRIT Study). J Thromb Haemost 2011; 9: 700-10.
8. Astermark J, Petrini P, Tengborn L, et al. Primary prophylaxis in severe haemophilia should be started at an early age but can be individualized. Br J Haematol 1999; 105: 1109-13. (Pubitemid 29309124)
9. Fischer K, Van der Bom JG, Mauser-Bunschoten EP, et al. Effects of postponing prophylactic treatment on long-term outcome in patients with severe haemophilia. Blood 2002; 99: 2337-41. (Pubitemid 34525418)
10. Aledort LM, Haschmeyer RH, Pettersson H. A longitudinal study of orthopaedic outcomes for severe factor-VIII-deficient haemophiliacs. The Orthopaedic Outcome Study Group. J Intern Med 1994; 236: 391-9. (Pubitemid 24323199)
11. Schramm W, Royal S, Kroner B, et al. Clinical outcomes and resource utilization associated with haemophilia care in Europe. Haemophilia 2002; 8: 33-43. (Pubitemid 34257917)
12. Collins P, Faradji A, Morfini M, et al. Efficacy and safety of secondary prophylactic vs. on-demand sucrose-formulated recombinant factor VIII treatment in adults with severe hemophilia A: results from a 13-month crossover study. J Thromb Haemost 2010; 8: 83-9.
13. Valentino LA. Secondary prophylaxis therapy: what are the benefits, limitations and unknowns? Haemophilia 2004; 10: 147-57. (Pubitemid 38312641)
14. Steen-Carlsson K, Hojgard S, Lindgren A, et al. Costs of on-demand and prophylactic treatment for severe haemophilia in Norway and Sweden. Haemophilia 2004; 10: 515-26. (Pubitemid 39317762)
15. Collins PW, Blanchette VS, Fischer K, et al. Break-through bleeding in relation to predicted factor VIII levels in patients receiving prophylactic treatment for severe hemophilia A. J Thromb Haemost 2009; 7: 413-20.
16. Walsh CE, Valentino LA. Factor VIII prophylaxis for adult patients with severe haemophilia A: results of a US survey of attitudes and practices. Haemophilia 2009; 15: 1014-21.
17. de Moerloose P, Urbancik W, Van den Berg HM, Richards M. A survey of adherence to haemophilia therapy in six European countries: results and recommendations. Haemophilia 2008; 14: 931-8.
18. Richards M, Altisent C, Batorova A, et al. Should prophylaxis be used in adolescent and adult patients with severe haemophilia? An European survey of practice and outcome data. Haemophilia 2007; 13: 473-9. (Pubitemid 47337985)
19. Fischer K, Van der Bom JG, Prejs R, et al. Discontinuation of prophylactic therapy in severe haemophilia: incidence and effects on outcome. Haemophilia 2001; 7: 544-50. (Pubitemid 34026747)
20. van Dijk K, Fischer K, Van der Bom JG, et al. Can longterm prophylaxis for severe haemophilia be stopped in adulthood? Results from Denmark and the Netherlands. Br J Haematol 2005; 130: 107-12. (Pubitemid 40979141)
21. Fischer K, Collins P, Bjorkman S, et al. Trends in bleeding patterns during prophylaxis for severe haemophilia: observations from a series of prospective clinical trials. Haemophilia 2011; 17: 433-8.
22. Van den Berg HM, De Groot PH, Fischer K. Phenotypic heterogeneity in severe hemophilia. J Thromb Haemost 2007; 5 (Suppl. 1): 151-6. (Pubitemid 46958828)
23. Plug I, Van der Bom JG, Peters M, et al. Thirty years of hemophilia treatment in the Netherlands, 1972-2001. Blood 2004; 104: 3494-500. (Pubitemid 39564418)
24. den Uijl Ingrid EM. Variation in FVIII/FIX activity in haemophilia:classification and clinical implications. 22-3-2011. University of Utrecht, Utrecht, the Netherlands. Dissertation.
25. Escuriola-Ettingshausen C, Halimeh S, Kurnik K, et al. Symptomatic onset of severe haemophilia A in childhood is dependent on the presence of prothrombotic risk factors. Thromb Haemost 2001; 85: 218-20. (Pubitemid 32140563)
26. Kurnik K, Kreuz W, Horneff S, et al. Effects of the factor V G1691A mutation and the factor II G20210A variant on the clinical expression of severe hemophilia A in children-results of a multicenter studys. Haematologica 2007; 92: 982-5. (Pubitemid 350144247)
27. van Dijk K, Van der Bom JG, Fischer K, et al. Phenotype of severe hemophilia A and plasma levels of risk factors for thrombosis. J Thromb Haemost 2007; 5: 1062-4. (Pubitemid 46691738)
28. Grunewald M, Siegemund A, Grunewald A, et al. Paradoxical hyperfibrinolysis is associated with a more intensely haemorrhagic phenotype in severe congenital haemophilia. Haemophilia 2002; 8: 768-75. (Pubitemid 35446629)
29. Santagostino E, Mancuso ME, Tripodi A, et al. Severe hemophilia with mild bleeding phenotype: molecular characterization and global coagulation profile. J Thromb Haemost 2010; 8: 737-43.
30. Wartiovaara-Kautto U, Joutsi-Korhonen L, Ilveskero S, et al. Platelets significantly modify procoagulant activities in haemophilia A. Haemophilia 2011; 17: 743-51.
31. Van Dijk K, Fischer K, Van der Bom JG, et al. Variability in clinical phenotype of severe haemophilia: the role of the first joint bleed. Haemophilia 2005; 11: 438-43. (Pubitemid 41283110)
32. Schulman S, Eelde A, Holmstrom M, et al. Validation of a composite score for clinical severity of hemophilia. J Thromb Haemost 2008; 6: 1113-21. (Pubitemid 351859120)
33. Fischer K, Pouw ME, Lewandowski D, et al. A modeling approach to evaluate long-term outcome of prophylactic and on demand treatment strategies for severe hemophilia A. Haematologica 2011; 96: 738-43.