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Personalised treatment

haemophilia A and B. Acta Orthop Scand Suppl 1965;Suppl-132.

15. van den Berg HM et al. Long-term outcome of individualized prophylactic treatment of children with severe haemophilia. Br J Haematol 2001;112(3):561–5.

16. Fischer K et al. The effects of postponing prophylactic treatment on long-term outcome in patients with severe hemophilia. Blood 2002; 1;99(7):2337–41.

17. Fischer K et al. Prophylactic treatment for severe haemophilia: comparison of an intermediate- dose to a high-dose regimen. Haemophilia 2002;8(6):753–60.

18. Feldman BM et al. Tailored prophylaxis in severe hemophilia A: interim results from the first 5 years of the Canadian Hemophilia Primary Prophylaxis Study. J Thromb Haemost 2006;4(6):1228–36.

19. Carlsson M et al. Pharmacokinetic dosing in prophylactic treatment of hemophilia A. Eur J Haematol 1993;51(4):247–52.

20. Collins PW 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(3):413–20.

factor consumption compared to short-acting products.

The use of long-acting product at the same dosing, that is, with the same number of injections as with current short-acting concentrates, could contribute to target higher troughs needed in some patients to reduce the frequency of breakthrough or subclinical bleeding episodes


It is now clear that a multi-dimensional approach should be adopted in tailoring and monitoring prophylactic regimens in patients with haemophilia, taking into account the bleeding symptoms, laboratory data (through levels, PK), assessment of joint status, quality of life, and not last, costs of treatment. The availability of long-acting concentrates will offer new opportunities to better tailor prophylactic treatment. l

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2. Ramgren O. Haemophilia in Sweden. III. Symptomatology, with special reference to differences between haemophilia A and B. Acta Med Scand 1962;171:237-42.

3. Nilsson IM et al. Twenty-five years’ experience of prophylactic treatment in severe haemophilia A and B. J Intern Med 1992;232(1):25–32.

4. Nilsson IM. Experience with prophylaxis in Sweden. Semin Hematol 1993;30(3 Suppl 2):16–9.

5. Gringeri A et al. A randomized clinical trial of prophylaxis in children with hemophilia A (the ESPRIT Study). J Thromb Haemost 2011;9(4):700–10.

6. Astermark J et al. Primary prophylaxis in severe haemophilia should be started at an early age but can be individualized. Br J Haematol 1999;105(4):1109–13.

7. Fischer K et al. The effects of postponing prophylactic treatment on long-term outcome in patients with severe hemophilia. Blood 2002;1;99(7):2337–41.

8. Manco-Johnson MJ et al. Prophylaxis versus episodic treatment to prevent joint disease in boys with severe hemophilia. N Engl J Med 2007;9;357(6):535–44.

9. Roosendaal G et al. Articular cartilage is more susceptible to blood induced damage at young than at old age. J Rheumatol 2000;27(7):1740–4.

10. Richards M et al. A United Kingdom Haemophilia Centre Doctors’ Organization guideline approved by the British Committee for Standards in Haematology: guideline on the use of prophylactic factor VIII concentrate in children and adults with severe haemophilia A. Br J Haematol 2010;149(4):498–507.

11. Collins P 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 Jan;8(1):83-9.

12. Tagliaferri A et al. Secondary prophylaxis in adolescent and adult haemophiliacs. Blood Transfus 2008;6 Suppl 2:s17–s20.

13. Tagliaferri A et al. Effects of secondary prophylaxis started in adolescent and adult haemophiliacs. Haemophilia 2008;14(5):945–51.

14. Ahlberg A. Haemophilia in Sweden. VII. Incidence, treatment and prophylaxis of arthropathy and other musculo-skeletal manifestations of

21. Bjorkman S et al. Pharmacokinetics and dose requirements of factor VIII over the age range 3–74 years: a population analysis based on 50 patients with long-term prophylactic treatment for haemophilia A. Eur J Clin Pharmacol 2009;65(10):989–98.

22. Collins PW et al. Implications of coagulation factor VIII and IX pharmacokinetics in the prophylactic treatment of haemophilia. Haemophilia 2011;17(1):2–10.

23. Collins PW et al. Factor VIII requirement to maintain a target plasma level in the prophylactic treatment of severe hemophilia A: influences of variance in pharmacokinetics and treatment regimens. J Thromb Haemost 2010;8(2):269-75.

24. Bjorkman S et al. Population pharmacokinetics of recombinant factor VIII: the relationships of pharmacokinetics to age and body weight. Blood 2012;12;119(2):612–8.

25. Bjorkman S et al. Comparative pharmacokinetics of plasma- and albumin-free recombinant factor VIII in children and adults: the influence of blood sampling schedule on observed age-related differences and implications for dose tailoring. J Thromb Haemost 2010;8(4):730–6.

26. Sheiner LB et al. Forecasting individual pharmacokinetics. Clin Pharmacol Ther 1979;26(3):294–305.

27. Bjorkman S. Limited blood sampling for pharmacokinetic dose tailoring of FVIII in the prophylactic treatment of haemophilia A. Haemophilia 2010;1;16(4):597–605.

28. Jayandharan GR et al. The phenotypic heterogeneity of severe hemophilia. Semin Thromb Hemost 2008;34(1):128–41.

29. Dargaud Y et al. Evaluation of thrombin generating capacity in plasma from patients with haemophilia A and B. Thromb Haemost 2005;93(3):475–80.


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