Medicines for Haemophilia

What it is

Haemophilia is an inherited condition in which the blood does not clot properly because one of the proteins needed for clotting (a clotting factor) is missing or reduced. The two main types are haemophilia A and B, depending on which factor is affected. It mostly affects males, while females can be carriers. The main problem is prolonged or excessive bleeding — not usually from small cuts, but internal bleeding, especially into joints and muscles, which can cause pain, swelling and, over time, joint damage. Severity varies from mild to severe. It is diagnosed with blood clotting tests and often known from family history.

How it is treated

Haemophilia is managed by specialist haemophilia centres, and modern treatment allows most people to lead full, active lives. The main treatment is replacing the missing clotting factor, either to treat bleeds or, in more severe cases, regularly to prevent them (prophylaxis). Newer treatments can reduce bleeding in other ways, and gene therapy is emerging. Alongside this, prompt treatment of bleeds, protecting the joints, careful management around surgery and dental work, and avoiding medicines that increase bleeding risk are important. People are taught to recognise and respond to bleeds early.