Adverse events in the prophylaxis of haemophilia

Whilst prophylaxis undoubtedly offers many advantages, the potential for adverse effects must also be borne in mind. Modern plasma-derived products have an extremely good safety record with regard to transmission of pathogens, although continuous vigilance is required as new pathogens continue to emerge, eg new variant Creutzfeldt-Jakob disease. There is no evidence that prophylactic treatment is associated with an increased incidence of inhibitors, and it is now recognized that genetic factors are the most significant in conferring susceptibility. Although subtle immunological abnormalities have also been observed in patients with haemophilia, there is no evidence that these are of any clinical significance. There has been a growing trend to use indwelling venous catheters for prophylaxis. The risk of infection has been appreciated for some time, although it has only recently been possible to quantify this with more precision. The risk of catheter-associated thrombosis is now recognized to be higher than hitherto appreciated. Whilst sporting activities are to be encouraged, there is a potential for significant trauma in children with prophylaxis, as plasma coagulation factor levels remain far below normal with the usual regimens.