Introduction
The goal of prophylactic treatment of hemophilia is to convert the severe form of
the disorder into a milder form by administration of factors VIII or IX. The rationale
behind this is that chronic arthropathy, the hallmark of hemophilia after repeated
bleedings, is less frequent and less severe in moderate hemophilia (i.e., factor VIII
or factor IX concentrations of 1% to 4% of normal) than in severe hemophilia (i.e.,
factor VIII/factor IX concentrations lower than 1% of normal).1 Keep in mind, however, that prophylactic treatment also provides protection from
all other forms of hemorrhage that may occur spontaneously or as a result of trivial
trauma in the untreated hemophilic child. Today, prophylactic treatment is available
to only a few hemophilia patients in the world, although it is recommended by the
World Health Organization (WHO) and the World Federation of Haemophilia (WFH): “Since
the main goal is to prevent joint bleeding and its sequelae, prophylaxis should be
considered optimal management for persons with severe hemophilia A or B (i.e., with
basal factor VIII and/or factor IX levels <1% of normal). Treatment should be started
at the age of 1-2 years and be continued indefinitely. Where prophylaxis is not feasible
or appropriate, on-demand therapy should be given.”2 In the United States, the Medical and Scientific Advisory Council of the National
Hemophilia Foundation has recommended that “prophylaxis should be considered the optimal
therapy for children with severe hemophilia A or B.”3 The aim of this chapter is to discuss prophylactic infusion regimens in the management
of hemophilia.
