Most cases of haemophilia are severe and need preventative treatment. This involves regular injections of clotting factor medicine.
If your child has haemophilia, you'll be trained to give them the injections when they're young.
They'll be taught how to inject themselves when they're older, to help avoid regular hospital appointments.
In some cases, injections may be given into a device called an implantable port, which can be surgically placed under the skin.
This port is connected to a blood vessel near the heart, so you do not need to try to find a vein for every injection.
People who have preventative treatment will need regular follow-up appointments with their care team so their progress can be monitored.
Preventative treatment is usually continued for life. It may be possible for someone to change to on-demand treatment, but they may be advised to switch back to preventative treatment if they have any episodes of significant bleeding.
There are different types of haemophilia. This page covers the most common types: haemophilia A and haemophilia B. They have similar symptoms but need different treatments because different clotting factors are affected.
Preventative treatment for haemophilia A involves regular injections of a medicine called octocog alfa (Advate). Read about octocog alfa (Advate) on the European Medicines Agency's website.
This medicine is an engineered version of clotting factor VIII (8), the clotting factor people with haemophilia A do not have enough of. Injections every 48 hours are often recommended.
Side effects of octocog alfa are uncommon but can include:
- an itchy skin rash
- redness and soreness at the site of the injection
Preventative treatment for people with haemophilia B involves regular injections of a medicine called nonacog alfa (BeneFix). Read about nonacog alfa (BeneFix) on the European Medicines Agency's website.
This is an engineered version of clotting factor IX (9), which people with haemophilia B do not have enough of. Injections twice a week are often recommended.
Side effects of nonacog alfa are uncommon but can include:
- altered taste
- feeling sick (nausea)
- discomfort and swelling at the injection site
In mild or moderate cases, treatment for haemophilia may only be necessary as an immediate response to bleeding.
People with haemophilia A can be treated on-demand with injections of octocog alfa or a medicine called desmopressin.
Desmopressin is a synthetic hormone. It works by stimulating the production of clotting factor VIII (8) and is usually given by injection.
Possible side effects of desmopressin include:
- stomach pain
- feeling sick (nausea)
On-demand treatment for haemophilia B usually involves injections of nonacog alfa.
Complications can include:
- developing inhibitors in your immune system
- joint problems
Some people who take blood clotting factor medicine develop antibodies in their immune system, called inhibitors, which make the medicine less effective.
People having treatment for haemophilia should be regularly tested for inhibitors.
Inhibitors can be treated with immune tolerance induction (ITI). This involves daily injections of clotting factors so the immune system should begin to recognise them and stop producing inhibitors.
ITI is usually offered to people with severe haemophilia A. People with haemophilia B may be offered ITI, but it's less effective and there's a risk of severe allergic reaction.
People with mild or moderate haemophilia A who develop inhibitors may be offered either bypass therapy or immunosuppressants.
Bypass therapy uses a medicine called a bypass agent to stop bleeding when it happens.
Immunosuppressants are medicines that suppress your immune system.
Joint bleeds can damage the soft spongy tissue in your joints (cartilage), and the thin layer of tissue lining the inside of the joint (synovium). The more damaged a joint is, the more vulnerable it is to bleeding.
Joint damage is more common in older adults with severe haemophilia. It's hoped that modern treatments mean children growing up with haemophilia today will not have joint damage.