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Haemophilia, von Willebrand disease and other coagulation defects
How is it treated?
Treatment is based at hospitals with a haemophilia centre and there are currently twenty-five Comprehensive Care Centres across the UK, so designated because of the wide range of services they provide. The principle of management is the replacement of the deficient factor by intravenous injection. For those severely affected, regular injection to prevent bleeding (prophylaxis) is well established, especially in children and as a result joint disease has proved to be largely preventable. In the UK, the concentrated clotting factors used for the treatment are now almost exclusively produced artificially (recombinant factors), but concentrates derived from human blood plasma are still often used to treat VWD.
Plasma derived factor concentrates were associated with the transmission of hepatitis and then human immunodeficiency virus (HIV) until 1986 when improved donor selection and viral inactivation techniques were introduced. Over one thousand two hundred people with haemophilia were infected with HIV and the majority have died, although modern treatment has been very effective for those who survive. More than four thousand contracted hepatitis (mostly due to hepatitis C virus) and although some have recovered completely, either spontaneously or through increasingly effective treatment, the majority have evidence of chronic infection and some have developed cirrhosis, liver failure or, more rarely, liver cancer. Plasma derived factor concentrates are now very safe but it is expected that recombinant factor will eventually replace the plasma derived products used for haemophilia care in the developed world.
About six per cent of patients with haemophilia A in the UK have inhibitors (antibodies) to factor VIII and this makes their treatment both difficult and very expensive. The most severely deficient patients are at greatest risk. Inhibitors are rare in haemophilia B, VWD and other inherited coagulation disorders.
For mildly affected patients with haemophilia A and VWD it is often possible to use a hormone called desmopressin (DDAVP) instead of factor VIII concentrate but a recombinant von Willebrand factor is not yet available.
What are the symptoms?
| Inheritance patterns and prenatal diagnosis 
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