The efficacy of prophylaxis therapy in the treatment of children with severe forms of haemophilia A has been universally acknowledged for some time now, to the extent that it has come to be the most commonly adopted strategy for the treatment of these young patients
However, the new evidence concerning the considerable benefits that prophylactic factor VIII replacement therapy has on the quality of life of patients with haemophilia A has also led to the development of a number of treatment protocols addressing those patients who were previously almost exclusively destined for on-demand therapy, namely, teenagers, adults and, in general, moderate and mild cases.
The various prophylaxis therapy protocols can now be classified in three categories, based primarily on the time at which the patient starts therapy.
More specifically, we talk about:
- primary prophylaxis, when treatment is commenced after the onset of the first haemarthrosis, before the appearance of documented joint disease, and in any case before the age of 3 years;
- secondary prophylaxis, when treatment is commenced after the onset of at least two haemarthroses, but before the appearance of documented joint disease;
- tertiary prophylaxis, when treatment is commenced after the onset of documented joint disease.
Although primary and secondary prophylaxis are by far the most effective in preventing chronic joint disease, tertiary prophylaxis has also shown great benefits, and all three approaches are now considered in the most recent guidelines for the treatment of haemophilia.
- Srivastava A. et al, Guidelines for the management of hemophilia. Haemophilia (2013), 19, e1–e47
- Taglaferri A. et al, Benefits of prophylaxis versus on-demand treatment in adolescents and adults with severe haemophilia A: the POTTER study, Thromb Haemost. (2015) Jul;114(1):35-45.
- Tagliaferri A. et al, Effects of secondary prophylaxis started in adolescents and adult haemophiliacs. Haemophilia. (2008) Sep;14(5):945-51