Abstract
Haemophilia is an x-chromosomal linked, hereditary lifelong disease which causes damages of bones and joints as well as early death.
Per definition there are three different forms, the severe form (less than 1% of coagulation factor activity) demands regular substitution therapy with the missing coagulation factor.
Haemophilia therapy developed since the 1960ies rapidly and today safe clotting factor concentrates are available, plasma – derived as well as recombinant.
Nevertheless some 80% of the patients affected do not receive treatment at all and only some 5 to 10% receive the so called “state of the art” treatment of their bleeding disorder.
Non- viral- inactivated- clotting factor concentrates caused an epidemic of HIV/Aids within this population in the 1970ies and 1980ies, with deaths of some 50% (in some Haemophilia Treatment Centers even higher) of continuous treated patients.
This fatal experience promoted research not only in gene- and biotechnology, but, despite of the early promises of the involved scientists and industries, a satisfying solution for Persons with Haemophilia is still missing.
Today plasma derived clotting factor concentrates have to undergo various viral- inactivation- procedures, donor screening and pool- as well as final product testing is essential, strict legislation on blood safety issues became standard in most countries.
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