Congenital bleeding disorders : rational treatment options

There are rational, effective choices available for the treatment of common inherited bleeding disorders, according to assessment of safety, efficacy and cost. All currently available products for patients with haemophilia A (factor VIII deficiency) are comparable in terms of efficacy and viral safety. However, high purity products are recommended for those with coexisting human immunodeficiency virus (HIV) infection. Many patients with mild haemophilia A and most with von Willebrand's disease can be treated with desmopressin, which can be given as an intranasal spray in some countries. For the treatment of patients with factor XI deficiency, fresh frozen plasma remains the standard care, although solvent-detergent-treated fresh frozen plasma and factor XI concentrate are currently being investigated as alternatives. In the treatment of haemophilia B (factor IX deficiency), purified factor IX concentrates are particularly useful in clinical settings where large amounts of concentrate are to be used (e.g. surgical prophylaxis). Their usefulness in other contexts needs clarification. Treatment of inhibitors that may develop in response to administered coagulation factors is still limited to the use of prothrombin complex concentrates and porcine factor VIII. Active clinical trials are currently assessing the efficacy and safety of recombinant factor VIIa, Xa and tissue factor in this indication.