Treating haemophilia

Only 30 years ago, most people with haemophilia would have been expected to die as a result of their condition – often through bleeding complications arising during childhood surgery. Today, thankfully, haemophilia is not considered to be a life-threatening condition, and most people can expect to live long and active lives. This positive metamorphosis has come about only since the discovery that the missing clotting factors associated with haemophilia can be replaced, quite simply, using products produced from normal human blood.

Early treatments for haemophilia involved the infusion of whole blood or plasma (the clear fluid part of the blood) to control bleeding episodes, but, unfortunately, clotting factor levels were not high enough to stop serious bleeding. During the 1960s and 1970s, freeze-dried concentrates made from human plasma and containing high levels of Factors VIII and IX became available, which could be kept at home and used when needed. These ‘clotting factor concentrates’ revolutionised haemophilia care allowing patients to travel, have jobs, and live full and independent lives. Tragically, these early products were also responsible for the transmission of blood-borne viruses such as hepatitis B and C and HIV – and large numbers of the haemophilia community were infected.

In the 1990s, modern treatment using safer factor concentrates again improved the outlook for individuals with haemophilia. These treatments contained clotting factor concentrates that underwent stringent viral inactivation processing, which almost entirely eliminated the threat of viral transmission. Today’s most modern treatments – ‘recombinant’ (i.e. genetically engineered) clotting factors VIII and IX – became available in the UK towards the end of the 1990s, and most newly diagnosed patients can expect to receive one of these.