Affiliations: MD, Lecturer in Public Health, Institute for Social
Medicine, Medical University of Vienna, Chairman of the European Haemophilia
Consortium | MD, Department of Urology, Medical University of
Vienna, Vienna General Hospital, Institute for Social Medicine, Center for
Public Health, medical University of Vienna, Rooseveltplatz 2, A-1090 Vienna,
Austria. E-mail: hubert.hartl@meduni-wien.ac.at
Note: [] Corresponding address: Dr. Hubert K. Hartl, Directorate-General
for Public Health, Federal Ministry for Health and Women, Radetzkystr. 2,
A-1030 Vienna Austria. Tel.: +43 1 71100 4137; Fax: +43 1 713 4404 1789;
E-mail: hubert.hartl@bmgf.gv.at
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.
