ABSTRACT
Since the late 1950s, prophylactic therapy has been used in patients with severe hemophilia
in order to convert their phenotype into the phenotype of moderate hemophilia. In
the early years, prophylaxis was mostly started after the development of arthropathy.
This secondary prophylaxis decreased the number of bleeds but did not halt the development
of arthropathy. With the start of primary prophylaxis, however, it was shown that
both joint bleeds and arthropathy could be prevented. Although its benefits have been
proved, prophylaxis has been used as standard treatment for children in only few countries.
There are now favorable results with prophylaxis on bleeding and arthropathy with
more than 25 years of follow-up. In general, the strategies used for prophylaxis can
be divided into those that are targeted at preinfusion clotting factor levels of >
1% and those that are guided by clinical bleeding patterns of individual patients.
In both types of strategies, treatment should be started early, before the start of
joint damage. However, clotting factor consumption in the first group of strategies
is expected to be higher, whereas bleeding frequencies are expected to be slightly
lower. The differences in long-term outcome between both strategies may only become
apparent after more than two decades. In addition, some patients may be able to stop
taking prophylaxis in adulthood while maintaining a low bleeding frequency. In conclusion,
although the optimal prophylactic regimen is still under debate, it is well established
that prophylaxis reduces or even prevents joint bleeds and hemophilic arthropathy.
Therefore, prophylaxis should be offered to all children with severe hemophilia, at
least until adulthood.
KEYWORD
Prophylaxis - hemophilic arthropathy - bleeding frequency - dose - start - duration
