Role of effective prophylaxis

 
Ineffective prophylaxis may not provide sufficient levels of rFVIII to assure protection from bleeds and completely prevent joint damage1

 

  • The historical aim of standard half-life rFVIII prophylaxis was to achieve trough levels of 1%, which may not be enough to completely prevent joint damage in all patients2
  • Lack of compliance to prophylaxis could lead to sub-optimal protection, resulting in increased bleeding, including joint bleeds3,4
  • Joint arthropathy, during prophylaxis with sub-optimal protection, may start sub-clinically and progress slowly, making early diagnosis and timely treatment difficult5
Data

Joint arthropathy in primary prophylaxis develops subtly over many years, the ankle joints being the first and most severely affected5

  • The key to successful long-term outcomes, and for preventing long-term joint damage, is effective prophylaxis, that prevents bleeding into the joints,5 and quick and effective treatment of joint bleeds when they occur6

Effective adherent prophylaxis supports long-term joint health, which may help PwH to live a liberated life5,7

rFVIII, recombinant Factor VIII.

References

1. Nijdam A, Foppen W, van der Schouw YT, Mauser-Bunschoten EP, Schutgens RE, Fischer K. Long-term effects of joint bleeding before starting prophylaxis in severe haemophilia. Haemophilia 2016 Nov;22(6):852–8.

2. Poon M-C, Lee A. Individualized prophylaxis for optimizing hemophilia care: can we apply this to both developed and developing nations? Thromb J. 2016 Oct 4;14(Suppl 1):32.

3. Den Uijl I, Biesma D, Grobbee D, Fischer K. Turning severe into moderate haemophilia by prophylaxis: are we reaching our goal? Blood Transfus. 2013 Jul;11(3):364–9.

4. Schrijvers LH, Beijvelt-van der Zande M, Peters M, Lock J, Cnossen MH, Schuurmans MJ, et al. Adherence to prophylaxis and bleeding outcome in haemophilia: a multicentre study. Br J Haematol. 2016;174:454–60.

5. Oldenburg J. Optimal treatment strategies for hemophilia: achievements and limitations of current prophylactic regimens. Blood. 2015 Mar 26;125(13):2038–44.

6. Carcao M, Hilliard P, Escobar MA, Solimeno L, Mahlangu J, Santagostino E. Optimising musculoskeletal care for patients with haemophilia. Eur J Haematol. 2015 Dec;95(Suppl 81):11-21.

7. Oldenburg J, Pasi J, Pabinger I, Nolan B, Kulkarni R, Blanchette V, et al. Improvements in joint health during long-term use of recombinant factor VIII Fc fusion protein prophylaxis in subjects with haemophilia A. Poster presented at the 12th Annual Congress of the European Association for Haemophilia and Allied Disorders, 6–8 February 2019, Prague, Czech Republic. P158.

Role of effective prophylaxis

 
Ineffective prophylaxis may not provide sufficient levels of rFVIII to assure protection from bleeds and completely prevent joint damage1

 

  • The historical aim of standard half-life rFVIII prophylaxis was to achieve trough levels of 1%, which may not be enough to completely prevent joint damage in all patients2
  • Lack of compliance to prophylaxis could lead to sub-optimal protection, resulting in increased bleeding, including joint bleeds3,4
  • Joint arthropathy, during prophylaxis with sub-optimal protection, may start sub-clinically and progress slowly, making early diagnosis and timely treatment difficult5
Data

Joint arthropathy in primary prophylaxis develops subtly over many years, the ankle joints being the first and most severely affected5

  • The key to successful long-term outcomes, and for preventing long-term joint damage, is effective prophylaxis, that prevents bleeding into the joints,5 and quick and effective treatment of joint bleeds when they occur6

Effective adherent prophylaxis supports long-term joint health, which may help PwH to live a liberated life5,7

rFVIII, recombinant Factor VIII.

References

1. Nijdam A, Foppen W, van der Schouw YT, Mauser-Bunschoten EP, Schutgens RE, Fischer K. Long-term effects of joint bleeding before starting prophylaxis in severe haemophilia. Haemophilia 2016 Nov;22(6):852–8.

2. Poon M-C, Lee A. Individualized prophylaxis for optimizing hemophilia care: can we apply this to both developed and developing nations? Thromb J. 2016 Oct 4;14(Suppl 1):32.

3. Den Uijl I, Biesma D, Grobbee D, Fischer K. Turning severe into moderate haemophilia by prophylaxis: are we reaching our goal? Blood Transfus. 2013 Jul;11(3):364–9.

4. Schrijvers LH, Beijvelt-van der Zande M, Peters M, Lock J, Cnossen MH, Schuurmans MJ, et al. Adherence to prophylaxis and bleeding outcome in haemophilia: a multicentre study. Br J Haematol. 2016;174:454–60.

5. Oldenburg J. Optimal treatment strategies for hemophilia: achievements and limitations of current prophylactic regimens. Blood. 2015 Mar 26;125(13):2038–44.

6. Carcao M, Hilliard P, Escobar MA, Solimeno L, Mahlangu J, Santagostino E. Optimising musculoskeletal care for patients with haemophilia. Eur J Haematol. 2015 Dec;95(Suppl 81):11-21.

7. Oldenburg J, Pasi J, Pabinger I, Nolan B, Kulkarni R, Blanchette V, et al. Improvements in joint health during long-term use of recombinant factor VIII Fc fusion protein prophylaxis in subjects with haemophilia A. Poster presented at the 12th Annual Congress of the European Association for Haemophilia and Allied Disorders, 6–8 February 2019, Prague, Czech Republic. P158.