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MASAC Recommendation Concerning Prophylaxis

Date: 
February 28, 2016
ID: 
241
Revisions: 
170; 179
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PDF icon 241Prophylaxis.pdf119.57 KB
  • In view of the demonstrated benefits of prophylaxis (regular administration of clotting factor concentrate to prevent bleeding) begun at a young age in persons with hemophilia A or B, MASAC recommends that prophylaxis be considered optimal therapy for individuals with severe hemophilia A or B (factor VIII or factor IX <1%). (1-4) Prophylactic therapy should be instituted early (prior to the onset of frequent bleeding), with the aim of keeping the trough FVIII or FIX level above 1% between doses. Optimal dosing and frequency should be determined for each individual by appropriate laboratory monitoring. (5)It is also recommended that individuals on prophylaxis have regular follow-up visits to evaluate joint status, to document any complications such as inhibitors, and to record any bleeding episodes that occur during prophylaxis.

     

    There are no clear cut guidelines as to when to stop prophylaxis. Joint bleeds with subsequent joint destruction are a lifelong problem for these individuals. (6) Therefore, they may continue to benefit from prophylaxis throughout their life.

     

    As always, a careful analysis of health risks and benefits must be performed by consumers and their healthcare providers. After a thorough discussion with their medical team, persons with hemophilia and their families should decide if prophylaxis is appropriate for them or their child. This decision should be evaluated periodically, particularly in light of emerging data and changes in bleeding pattern and clotting factor usage.

     

    As is the case with all recommendations, MASAC will periodically reexamine this recommendation as new data emerge.

     

    References:

    1.   Petrini P, Lindvall N, Egberg N, Blombäck M: Prophylaxis with factor concentrates in preventing hemophilic arthropathy. Am J Pediat Hematol Oncol 1991; 12: 280-287.

    2.   Brackman HH, Eickhoff HJ, Oldenburg J et al.: Long-term therapy and on-demand treatment of children and adolescents with severe hemophilia A: 12 years of experience.Haemost 1992;22:251-8.

    3.   Nilsson IM, Berntorp E, Löfqvist T, Petterson H.: Twenty-five years experience of prophylactic treatment in severe haemophilia A and B. J Intern Med 1992; 232: 25-32.

    4.   Manco-Johnson M, et al. Prophylaxis versus episodic treatment to prevent joint disease in boys with severe hemophilia. N Engl J Med 2007; 357: 535-544.

    5.   Carlsson M, Berntrop E, Bjorkman S, Lindvall K: Pharmacokinetic dosing in prophylactic treatment of hemophilia A. Eur J Haematol 1993; 31: 247-252.

    6.   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: 391-399.