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Personalised treatment


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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.


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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


Conclusions


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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