What treatment do most people with haemophilia have?
Most people with haemophilia A or B have treatment to replace the missing clotting factor in their blood. These days this is made entirely in the lab, not from human blood and is called ‘recombinant’ factor. You have the treatment as injections into a vein (intravenous injections).
Most people with severe and many people with moderate haemophilia have clotting factor injections on a regular basis to help stop them from having any bleeding.25 This is called prophylaxis (pronounced prof-ill-ax-iss) or preventative treatment. How often you need treatment is worked out individually for you, from your blood test results.25 But if you have haemophilia A, you usually have injections every other day, or 3 times a week.25 If you have haemophilia B, you usually have them about twice a week.25 (The missing clotting factor in haemophilia B lasts longer in the blood stream, so you can have it a bit less often.)
Newer types of clotting factor have been developed that last longer so you can have injections less often. These are called extended half-life factor replacement. If you have haemophilia A, you may be able to have factor injections twice a week, instead of every other day.27 If you have haemophilia B, you may be able to have injections weekly or even fortnightly.28,30
Some people who could be on prophylaxis don’t like to have regular injections, so they choose to only have clotting factor injections if they have a bleed. This is entirely a personal choice, but it does mean that you have a greater risk of developing bad damage to your joints from repeated bleeds over many years. Internationally accepted guidelines for treating severe haemophilia recommend prophylaxis for this reason.25
People on prophylaxis can still have serious bleeds, particularly if they knock or injure themselves. If this happens, you will need extra factor injections. You also need to have extra factor injections if you’re going to have an operation or dental treatment that could cause bleeding. Generally, you need fewer of the extended half-life factor injections to control a bleed than the regular recombinant factor.27,28,30
There are sometimes other treatments for haemophilia, as well as factor replacement. You may have tablets such as tranexamic acid (Cyklokapron, pronounced sy-kloh-kap-ron) to stop clots breaking down, particularly if you have bleeding from your gums or elsewhere in your mouth.22,24 If you have mild haemophilia A, you may have an injection or a nasal spray of a drug called desmopressin (pronounced dez-moh-press-in) that can help blood to clot.22,24 A bypassing agent treatment, Emicizumab, is now available on the NHS.29 This is a copy of a single immune system protein (a monoclonal antibody) that can be manufactured in large amounts for use as a treatment. This is used for people with haemophilia A who have inhibitors that are resistant to factor VIII (8).29 Having inhibitors is serious because it means that factor injections don’t work. The bypassing agent replaces factor VIII in the body’s clotting system by binding to factor IX (9) and factor X (10).29 In this way, it enables the blood to clot. You have this treatment as an injection under the skin (subcutaneously) once a week.29 In future, this treatment may become available for people with haemophilia A who don’t have inhibitors.31