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Factor VIII products


Treatment Start once weekly


Port implant if needed Shorten interval


Patient starts to infuse himself Tailor dose if bleed occurs


Consider lowering annual dose


per kg body weight if bleed pattern permits


Modulate dose according to intervening comorbidities


Death Figure 1: Life-long prophylaxis in haemophilia A


Summary and concluding remarks An algorithm for using lifelong prophylaxis is depicted in Figure 1. The algorithm is mainly, and schematically, showing the prophylactic program used at the Malmö Center and modulated according to economic demands. Prophylaxis starts before joint disease has started. Infusions are supported by healthcare professionals, with the teaching of guardians. If venous access is difficult, a port is implanted, for example, Port-A-Cath. Dose intervals are shortened, with the goal of achieving treatment every second day within the first years of life. Haemophilia nurse visits at home, at day care and at school (for example) are very important. The patient starts to manage infusions, and takes over from the guardians, somewhere between 7 and 12 years of age. During childhood and adolescence, the patient is encouraged to live a normal life, with participation in school athletics. Prophylactic infusions are tailored to individual needs, for example, prior to intense sport activities. When reaching adulthood, treatment can sometimes be modulated as tendency to bleed may become less. When comorbidities occur, the treatment schedule, if needed, should be tailored differently if bleeding risk increases (for example, malignant disease) or if anticoagulant therapy is needed (for example, myocardial infarction, arterial fibrillation). Any licenced FVIII concentrate can be used and recommended doses are in the range 25–40 IU/kg body weight given every


second day. Due to the shorter half-life of FVIII in children, and due to the higher risk of bleeding compared to adults, the higher dose range is often used during childhood. As mentioned above, several other protocols have been used and published and some authors advocate not to giving infusions in connection with danger signals such as vaccination and infections. As studies with formal high evidence are merely impossible to do in haemophilia prophylaxis, we have to rely on cohort studies and comparisons between countries. Even if intense schedules seem to give best long-term medical outcome, they are extremely costly, and economical considerations and concerns sometimes have to guide the treatment. Despite these uncertainties, it can be concluded that modern haemophilia prophylaxis has dramatically improved the lives of patients. l


References 1. Berntorp E et al. Consensus perspectives on prophylactic therapy for haemophilia: summary statement. Haemophilia : the official journal of the World Federation of Hemophilia. 2003;9 Suppl1:1–4.


2. Roosendaal G, Lafeber FP. Blood-induced joint damage in hemophilia. Seminars in thrombosis and hemostasis. 2003 Feb;29(1):37–42.


3. Manco-Johnson MJ et al. Prophylaxis versus episodic treatment to prevent joint disease in boys with severe hemophilia. New Engl J Med2007;357(6):535–44.


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


Individual


Birth


Age (years) 1 1–2


7–12 1–18 ≥18


5. van Creveld S. Prophylaxis in haemophilia. Lancet 1971;1(7696):450.


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


7. Berntorp E, de Moerloose P, Ljung RC. The role of prophylaxis in bleeding disorders. Haemophilia : the official journal of the World Federation of Hemophilia 16; Suppl 5:189–93.


8. 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.


9. Khawaji M, Astermark J, Berntorp E. Lifelong prophylaxis in a large cohort of adult patients with severe haemophilia: a beneficial effect on orthopaedic outcome and quality of life. Eur J Haematol 2012 Jan 5.


10. Lindvall K, Von Mackensen S, Berntorp E. Quality of life in adult patients with haemophilia – a single centre experience from Sweden. Haemophilia : the official journal of the World Federation of Hemophilia. 2012 Mar 8.


11. Fischer K et al. Prophylactic treatment for severe haemophilia: comparison of an intermediate- dose to a high-dose regimen. Haemophilia: the official journal of the World Federation of Hemophilia 2002;8(6):753–60.


12. 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.


13. Schimpf K, Fischer B, Rothmann P. [A controlled study of treating haemophilia A on an out-patient basis (author’s transl)]. Dtsch Med Wochenschr. 1976;101(5):141–8.


14. Carlsson M, Berntorp E, Bjorkman S, Lindvall K. Pharmacokinetic dosing in prophylactic treatment of hemophilia A. Eur J Haematol 1993;51(4):247–52.


15. Lindvall K et al. Daily dosing prophylaxis for haemophilia: a randomized crossover pilot study evaluating feasibility and efficacy. Haemophilia: the official journal of the World Federation of Hemophilia 2012;18(6):855–9.


16. Khawaji M, Astermark J, Von Mackensen S, Akesson K, Berntorp E. Bone density and health-related quality of life in adult patients with severe haemophilia. Haemophilia: the official journal of the World Federation of Hemophili. 2011;17(2):304–11.


17. Auerswald G, Bidlingmaier C, Kurnik K. Early prophylaxis/FVIII tolerization regimen that avoids immunological danger signals is still effective in minimizing FVIII inhibitor developments in previously untreated patients--long-term follow-up and continuing experience. Haemophilia: the official journal of the World Federation of Hemophilia 2012;18(1):e18–20.


18. Gouw SC et al. Intensity of factor VIII treatment and inhibitor development in children with severe hemophilia A: the RODIN study. Blood 2013;121(20):4046–55.


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