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What types of haemophilia treatment are available in the UK?

What types of haemophilia treatment are available in the UK?

There are three main groups of treatments available for treating haemophilia. The first is standard factor replacement therapy. This will either be with factor VIII (8) or factor IX (9) depending on whether you have haemophilia A or haemophilia B.22,24

Factor VIII (8) and IX (9) used to be taken from donated blood. But these days, recombinant factor is usually used instead. This works just as well as factor purified from blood, but it’s manufactured, so there is no infection risk.

Some people have factor only when they have a bleed. This is called on-demand treatment. Most people in the UK with severe haemophilia have preventative treatment with factor, called prophylaxis (pronounced prof-ill-ax-iss).25 This helps to prevent bleeds. How often you need to treat varies, but in general, people with haemophilia A need prophylaxis 3 times a week or every other day and those with haemophilia B twice a week.26,18

A newer type of factor replacement is called extended half-life (EHL) factor. This can last longer in the blood stream than standard factor, so many people on prophylaxis need treatment less often. With haemophilia A, you may only need treatment twice a week instead of 3 times a week.27

For haemophilia B treatment may be weekly or even once a fortnight.28 Treatment for bleeds can be quicker too, with control after a single injection of EHL factor.27,28

The third type of treatment currently available is a bypassing agent called emicizumab (pronounced em-ih-siz-oo-mab). This is a monoclonal antibody – a single type of immune system protein that can be manufactured in large quantities and used as a treatment. Emicizumab is an injection you have just under the skin (subcutaneously).29 You have it once a week to prevent bleeds instead of having replacement factor.29 It only works for haemophilia A and is currently only available for people who have inhibitors in the UK.29 This means that factor VIII no longer helps them because their immune system works against it. Emicizumab works by attaching to the clotting factors IX (9) and X (10) and bringing them together. This activates blood clotting even when you have no factor VIII (8).29

There is more information in our FAQ on the difference between treatments for haemophilia A and B.

Although all of these options are all available to patients, their use is strictly monitored and robust guidance is in place to ensure that the products are suitable for the patient before they are prescribed.