Factor replacement therapy in haemophilia--are there models for developing countries?

Successful models for factor replacement in severe haemophilia involves prophylactic or on-demand administration of large quantities (1500-9000 IU kg-1 year-1) of very high purity factor concentrates starting early in life. The prohibitive cost of these protocols make them completely impractical in developing countries where the quantity of factor used for replacement therapy is much lower and varies considerably (25-500 IU kg-1 year-1). At this level of treatment, as some joint damage is inevitable, the aim of therapy shifts to preventing disability and preserving reasonable joint function rather than perfect architecture. There are no carefully recorded data on long-term outcome of musculo-skeletal function on these doses. Appropriate studies are needed to document such data. With regard to products for factor replacement, economic compulsions lead to the use a variety of factor concentrates and blood-bank products for replacement therapy. Most patients in the developing world do not have access to adequate replacement therapy. Among the rest, some get limited quantities of plasma-derived concentrates while others use cryoprecipitate, fresh frozen plasma or even whole blood. Since the superiority of virus-inactivated purified factor concentrates in achieving the aims of replacement therapy is well established, the aim should be to provide this for all people with haemophilia. This can be achieved by production of such concentrates locally or by importing them. Different models are possible depending on the circumstances in each country.