MASAC Document #179
MASAC Recommendation Concerning Prophylaxis
(Regular Administration of Clotting Factor Concentrate to Prevent Bleeding)
The following recommendation was approved by the Medical and Scientific Advisory Council (MASAC) on November 3, 2007, and adopted by the NHF Board of Directors on November 4, 2007.
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In view of the demonstrated benefits of prophylaxis (regular administration of clotting factor 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. This can usually be accomplished by giving 25-50 FVIII units/kg three times per week or every other day (5), or 40-100 FIX units/kg two to three times weekly. It is also recommended that individuals on prophylaxis have regular follow-up visits to evaluate joint status, to document any complications, 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 health care 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 and clotting factor usage.
As is the case with all recommendations, MASAC will periodically reexamine this recommendation as new data emerge.
1. 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: 251-258.
2. 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.
3. 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.
4. Carlsson M, Berntrop E, Bjorkman S, Lindvall K: Pharmacokinetic dosing in prophylactic treatment of hemophilia A. Eur J Haematol 1993; 31: 247-252.
5. 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.
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.
This material is provided for your general information only. NHF does not give medical advice or engage in the practice of medicine. NHF under no circumstances recommends particular treatment for specific individuals and in all cases recommends that you consult your physician or local treatment center before pursuing any course of treatment.
Copyright 2007 National Hemophilia Foundation. To facilitate the dissemination of these medical recommendations, reproduction of any material in this publication in whole or in part will be permitted provided: 1) a specific reference to the MASAC recommendation number and title is included and 2) the reproduction is not intended for use in connection with the marketing, sale or promotion of any product or service. NHF reserves the right to make the final determination of compliance with this policy. For questions or to obtain a copy of the most recent recommendations, please contact the NHF Director of Communications at 1-800-42-HANDI or visit the NHF website at www.hemophilia.org.