Development of treatment
It has taken many decades of careful scientific research to arrive where we are today in terms of the treatment options available for haemophilia. In the UK, most people with haemophilia are treated safely and effectively with recombinant (genetically engineered) Factor VIII or Factor IX. These are manufactured using clotting factor genes, which are cleverly inserted into ‘host’ cells that can produce large quantities of concentrated clotting factors. Early (sometimes called first-generation) recombinant clotting factor concentrates had a human protein (albumin) added to the final products to help stabilise them, but this was removed in the late 1990s when the ‘second-generation’ recombinant clotting factors were developed.
Third-generation recombinant clotting factors, which are manufactured without the addition of any animal or human proteins within the culture media, became available in 2003. Finally, the method of purification has been updated so that there are no animal or human proteins at all in the manufacturing process. The first of these was a recombinant Factor IX which was approved for use in the EU in 1997.
Key milestones in the development of haemophilia treatment are outlined below.
||First use of a blood transfusion to control haemophilia reported in The Lancet
||First plasma transfusion to treat haemophilia
||Haemophilia identified as a clotting factor deficiency
||First factor VIII concentrate becomes commercially available
||First successful treatment of patients with Factor VIII inhibitors using Factor VIIa
||Factor VIII gene is identified and cloned
||All Factor VIII and IX products now subjected to at least one virus inactivation or reduction method for safe elimination of HIV, hepatitis C and B viruses
||Serological test developed to screen for the hepatitis C virus
||‘First-generation’ recombinant Factor VIII concentrate approved
||First recombinant Factor IX concentrate approved (no animal or human protein in manufacture)
||‘Second-generation’ recombinant Factor VIII concentrate approved
||‘Third-generation’ recombinant Factor VIII concentrate approved
||First recombinant Factor VIII concentrate approved with no animal or human protein used in the manufacture (US market)