Will treatment improve my quality of life?
Thanks to research and the development of new treatments, the outlook for haemophilia is the best it’s ever been. Replacement clotting factor is now made entirely in the lab or, in the case of plasma derived product, with rigorous safety procedures, so blood-borne infections are no longer a risk. People with severe haemophilia are now advised to have preventative treatment rather than waiting until they have a bleed, minimising the risk of joint damage.25 This is called prophylaxis (pronounced prof-ill-ax-iss).
If you have severe haemophilia, you are at risk of major bleeding into one of your joints or muscles.40 As well as being very painful, repeated bleeding causes damage to the body. Your knees, elbows or ankles are particularly likely to be affected.40 Joints can become inflamed and permanently distorted. Bleeds into muscles can cause pressure on nerves, permanently damaging them if not treated. Having regular preventative injections of clotting factor reduces all these risks.8 With preventative treatment, many people with haemophilia have only 1 or 2 potentially serious bleeds a year at most. With the newest generation of longer-lasting (or ‘extended half-life’) clotting factors, bleed rates are showing signs of falling even lower.27,28
Research is also addressing long-term problems of haemophilia treatment, such as inhibitor development, which stops clotting factor from working.26 For some people who didn’t respond to treatment for inhibitors, that was extremely serious, meaning they had great difficulty controlling bleeds.26,29 Now if you are in this situation, there is a treatment for people with haemophilia A called emicizumab (pronounced em-ih-siz-oo-mab) that you have weekly, as a small injection under the skin.29 You are likely to have your schedule of preventative treatment worked out specifically for you.25 This means you can time your treatment so that your clotting factor levels are at their highest when it best suit you – when you are playing sports for instance.34 In turn, keeping strong and healthy through sports can help you manage your haemophilia by strengthening your bones, muscles and joints.13
We’re not trying to understate the potential difficulties. If you have haemophilia, you need to accept that life is a little more complicated. You can help yourself by sticking to your treatment schedule as closely as possible and generally looking after your health. There are practical issues around having factor with you all the time and finding somewhere to give yourself treatment when you’re away from home. Haemophilia will always be difficult for children to come to terms with – being different from their friends, as well as learning to cope with needing regular treatment injections. But the focus these days is very much on enabling you to do what you want to do, rather than telling you what you can’t do.