Could there be more

FAQs - The Fundamentals of Haemophilia Treatment

Most people with haemophilia need treatment at some point. How regularly depends on whether they have mild, moderate or severe haemophilia. Your child will have a blood test to find this out.

Haemophilia means your child’s blood has a lower than normal level of one of the body’s essential clotting factors. It they have less than 1% of normal, they have severe haemophilia. If they have more than 5% of the normal level, their haemophilia is mild.8  People with between 1% and 5% of the normal clotting factor level have moderate haemophilia.8

People with severe haemophilia and some people with moderate haemophilia need regular injections of the clotting factor their body is lacking They usually have these injections into a vein (intravenously) about 2 or 3 times a week.18 Young children may also be offered a central venous access device (CVAD) such as a Port-A-Cath to make infusions easier.19 This treatment is called ‘prophylaxis’ (pronounced prof-ill-ax-iss) and the aim is to prevent bleeding. They will also need extra treatment before an operation or having a tooth out.

People with mild or moderate haemophilia may also need preventative treatment before surgery.22 They may be able to have tablets or a nasal spray to stop bleeding, rather than injections of clotting factor.21,22,23

Even with regular preventative treatment, people with severe haemophilia can have bleeding episodes, usually after a knock or accident but sometimes for no obvious reason. People with mild and moderate haemophilia may also have bleeds after an accident or injury. If anyone with haemophilia has a bleed into a muscle or joint, they will need injections of clotting factor until the bleeding is under control. Bruises and minor cuts will often get better on their own but signs of a more serious bleed can include:9

  • Stiffness of a joint
  • Tingling or pins and needles
  • Heat
  • Swelling
  • Pain

If in doubt, seek medical help sooner rather than later.

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 This means that factor VIII no longer helps them because their immune system works against it. Emicizumab only works in haemophilia A and 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.

The main difference is that they need injections to replace different clotting factors. In haemophilia A, factor VIII (8) is missing. In haemophilia B, it is factor IX (9) that is missing.

These two clotting factors last for different lengths of time in the blood. So people with severe haemophilia A generally need preventative injections 3 times a week or every other day and those with severe haemophilia B, twice a week.18 This is called prophylaxis (pronounced prof-ill-ax-iss).

There are new treatments for haemophilia A and B that are available. These are known as ‘extended half-life’ factor replacement. They stay in the blood stream for longer, so you don’t have to give yourself the prophylaxis injections so often. For haemophilia A, you may be able to have factor injections twice a week, instead of every other day.27 For haemophilia B, you may be able to have factor injections once a week or once a fortnight instead of twice a week.28,30

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

Most people with severe and moderate haemophilia self-treat. As babies and children of course, their parents are taught how to give the injections. But when they’re old enough, haemophilia doctors and nurses encourage children to learn to do it themselves.32

Knowing how to give your own injections gives you a lot more freedom and independence.32 You don’t have to keep going to hospital. You can go out and about more easily without having to worry about being within reach of the person who usually does your injections. Some people time their injections so that they have one just before taking part in sport, for example.

Doing your own injections, or, for children, your parents doing it, also means that you can give yourself treatment straight away should you have a bleed. That way, you are less likely to have long term problems and can recover more quickly.32

Treatment for haemophilia works by replacing the clotting factor that you would normally have in your blood.

Blood clotting is very complicated. There is what is called a ‘clotting cascade’33 - when proteins in the blood are activated to stop bleeding. There are 13 of these proteins or clotting factors.

For a clot to form, the first clotting factor acts on the second, which acts on the next and so on – the ‘cascade’. A clot won’t form until all the steps in the cascade have completed.

In haemophilia, one of the proteins is missing or defficient. That’s why the blood doesn’t clot and you have bleeding. The gap in the clotting cascade is filled with factor replacement. If you have haemophilia A, you will be prescribed factor VIII (8). If you have haemophilia B, you will be prescribed factor IX (9).

The clotting factors get used up all the time, which is why you need to have treatment injections so often. You need to have your factor as an injection as it needs to be delivered directly into your blood.

Clotting Cascade

Most people with severe and many people with moderate haemophilia A choose to have regular treatment to help prevent bleeds. This is called prophylaxis (pronounced prof-ill-ax-iss). Most people with moderate haemophilia A and all people with mild haemophilia A will treat on-demand, that is following a bleed or for surgery.

Factor VIII (the missing clotting factor in haemophilia A) doesn’t last long in the blood stream. So in general, people with haemophilia A on prophylaxis need treatment about 3 times a week, or every other day.25

These days, treatment schedules are worked out individually for you.34 You have a series of blood tests carried out. These look at how your body manages and uses up factor and are called pharmacokinetics.34 They help your haematologist (a specialist doctor in blood disorders, pronounced heem-a-toll-o-jist) to advise on how much factor VIII (8) you need each time you treat yourself and how often you need to do this. They will also take into account your age, weight, history of bleeds and how active you are.34,35

There are now newer treatments available. For haemophilia A, there is now a form of factor VIII (8) that lasts longer in the blood stream.27 This treatment doesn’t suit everyone. You have pharmacokinetic tests carried out on your blood using the new treatment to find out.27 If it is suitable for you, the tests will tell your haematologist how often you need prophylaxis injections. For different people, this can vary from once every 3 days to once every 5 days.27 If you’ve previously needed injections every other day, that means at least a third fewer injections. Over a year, that’s more than 50 injections you don’t need to have.

Although doctors recommend strongly that everyone with severe haemophilia has prophylaxis, some people with haemophilia choose not to. They prefer to treat only if they have a bleed. In that case, how often you have treatment will be impossible to predict. The newer longer lasting factor VIII (8) does tend to get bleeding under control with fewer injections. For some people, a single injection may be enough.27

Just about everyone with severe haemophilia A gives themselves their factor injections once they are old enough. Most people become expert at this and can do it pretty quickly. The time it takes varies depending on how much factor you need, but it should only take around 15 to 20 minutes. People do generally get very used to doing it because they have to do it so often.

Everyone with haemophilia is advised to have some factor and their infusion kit with them at all times, although people with mild haemophilia should only use this in consultation with their haemophilia centre. You never know what’s going to happen and unfortunately even a minor accident can cause bleeding that needs immediate treatment.

Most people with severe haemophilia B choose to have regular treatment to help prevent bleeds. This is called prophylaxis (pronounced prof-ill-ax-iss). Most people with moderate haemophilia B and all people with mild haemophilia B will treat on-demand, that is following a bleed or for surgery.

Factor IX (9) - the missing clotting factor in haemophilia B – lasts a little longer in the blood stream than the factor VIII (8) used to treat haemophilia A. So in general, people with severe haemophilia B need treatment about twice a week.25

These days, treatment schedules are worked out individually for you.34 You have a series of blood tests carried out. These look at how your body manages and uses up factor and are called pharmacokinetics.34 They help your haematologist (a specialist doctor in blood disorders, pronounced heem-a-toll-o-jist) to advise on how much factor IX (9) you need each time you treat yourself and how often you need to do this. They will also take into account your age, weight, history of bleeds and how active you are.35

There are now newer treatments available. For haemophilia B, there is now a form of factor IX (9) that lasts longer in the blood stream.28 This treatment doesn’t suit everyone. You have pharmacokinetic tests carried out on your blood using the new treatment to find out.28 If it is suitable for you, the tests will tell your haematologist how often you need prophylaxis injections. For different people, this can vary from once a week to once a fortnight.28 If you’ve previously needed injections twice a week, that means half as many or even a quarter of the number of injections you used to have.

Although doctors recommend strongly that everyone with severe haemophilia has prophylaxis, some people with haemophilia choose not to. They prefer to treat only if they have a bleed. In that case, how often you have treatment will be impossible to predict. The newer longer lasting factor IX does tend to get bleeding under control with fewer injections. In many situations, a single injection may be enough.28,30

Just about everyone with severe haemophilia B gives themselves their factor injections once they are old enough. Most people become expert at this and can do it pretty quickly. The time it takes varies depending on how much factor you need, but it should only take around 15 to 20 minutes. People do generally get very used to doing it because they have to do it so often.

Everyone with haemophilia is advised to have some factor and their infusion kit with them at all times, although people with mild haemophilia should only use this in consultation with their haemophilia centre. You never know what’s going to happen and unfortunately even a minor accident can cause bleeding that needs immediate treatment.

Because clotting factors are manufactured forms of natural body substances, there are very few side effects compared to drugs you may take for other medical conditions. Clotting factors used to be only made from human blood and then there was a risk of picking up an infection from the donors’ blood, such as hepatitis or HIV. In the 80s, donor screening as well as techniques for treated plasma-derived clotting factors such as dry heat, solvent-detergent treatment, and pasteurisation vastly reduced this risk. Starting in the late 90s, most patients in the UK now use clotting factors made in the lab, without the need for human blood plasma.

The most common problem is pain at the injection site when having the treatment.

There are a couple of ways of helping children to cope. One is putting anaesthetic cream on the injection site about half an hour before giving the treatment.36 This numbs the area so they are less likely to find the needle going in painful, but they can still find having the injection upsetting. Another is to put in a central venous access device often called a ‘port’. This is a device that sits just under the skin that you inject into.32 The needle still has to go through the skin, but it’s less painful. The port connects to a vein inside the body so the treatment can still get straight into your blood. There can be problems with venous access devices. They can become infected or blocked and then have to be taken out.32

Some people with haemophilia develop a strong dislike of injecting and prefer to only treat if they have a bleed rather than have preventative factor (prophylaxis – pronounced prof-ill-ax-iss). This means they will have fewer injections. But it also means they are more at risk of painful bleeds into joints and muscles.25 Over years, joints can become seriously damaged by the bleeds, causing pain and disability.25, 36

Having repeated injections over a lifetime can cause problems with veins. People with haemophilia are advised to vary their injection sites. Even so, though some veins become unusable most people with haemophilia become very good at finding usable veins.

The most serious side effect of treatment with clotting factors is the development of inhibitors.37  These are antibodies to the clotting factor and mean your immune system is reacting against it.38 About 3 in every 10 people with haemophilia A develop inhibitors at some point.37 It is much rarer in haemophilia B, with about 3 in every 100 people developing them.37

Developing inhibitors is serious because it means that controlling bleeding is much more difficult.38 People with inhibitors may have bleeding controlled with a treatment called activated prothrombin complex concentrate or another clotting factor called VIIa (7a).26,29

There is treatment to get rid of inhibitors. It involves having larger and more frequent infusions of clotting factor to get your immune system to learn to tolerate it again.39 This is called Immune Tolerance Induction. This is effective in about 2 out of every 3 people who develop inhibitors.29 

There is a newer treatment available called emicizumab (pronounced em-ih-siz-oo-mab).29 This is an injection under the skin that you have once a week. It works by bypassing factor VIII (8) in blood clotting and so controls bleeding.29

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.

Thanks to the introduction of preventative treatment, improvements in safety of plasma products and the development of clotting factor made in the lab, people with haemophilia are growing up healthier and with less lasting damage from bleeds than they used to.

That’s not to say that there aren’t some demands placed on people with haemophilia that others’ don’t have. You will always need to have treatment, whether that’s regular preventative treatment (prophylaxis, pronounced prof-ill-ax-iss) or treatment for bleeds.25

Everyone with haemophilia should have their care co-ordinated through a haemophilia centre.41 You may have all your treatment managed through a comprehensive care centre (CCC), but some people live too far away to go regularly and will have their day to day treatment through a haemophilia centre (HTC) at a nearer hospital. The treatment your HTC gives you should always be overseen by the haemophilia comprehensive care centre because that is the centre of expertise. They will have specialist nurses, physios and other health professionals, not only specialist doctors.42 People who have their haemophilia managed through a comprehensive care centre do better overall than those who don’t.42

Every now and then, you will have to travel to your comprehensive care centre. A couple of times a year, you will need to see the haemophilia specialist physiotherapist. They need to check your joints for damage from any bleeds and can give you an exercise programme to strengthen your muscles and joints and make bleeds less likely.26

As a person with haemophilia you should have contact with your centre at least once a year. If you have severe haemophilia this will be at least twice a year and for very young children, it will be 3-4 times a year.43

In between check-ups, you give your treatment yourself at home. Or, for children, usually their parents give it. Being able to give your own treatment at home does make life easier. But making sure you have the absolute minimum of long term damage from bleeds means that you should always have factor and infusion kits with you.

Giving themselves treatment becomes second nature to most people and they are able to inject themselves quickly and relatively easily, keeping to good hygiene standards so that they don’t pick up an infection. People also learn to spot symptoms of bleeds so that they can treat as early as possible and minimise damage. Very early signs can be difficult to spot – people talk about tingling or bubbling, stiffness and warmth.

You should have been given a Bleeding Disorder Information Card by your haemophilia comprehensive care centre. This has your diagnosis and the number of your haemophilia comprehensive care centre. You should have this on you all the time – just in case. Many people also wear a medical alert bracelet to tell people you have haemophilia.

However well you manage it, haemophilia will affect your daily life to some extent. You’ll always have to put a little bit of extra planning or thought into what you do and where you go than other people need to. But with preventative treatment (prophylaxis) there is less of an impact than there used to be.

There are some implications for your general health if you have haemophilia. Because of the risk of bleeding, it’s important to take very good care of your teeth to prevent decay and gum disease.44 You need to see the dentist and hygienist regularly.44

There are some over-the-counter medicines you need to avoid, including aspirin and anti-inflammatories such as ibuprofen.45 If in any doubt at all (including about herbal medicines) it’s best to check with your haemophilia centre.45 You can have vaccinations as injections under the skin (subcutaneously), but shouldn’t have injections into a muscle because of the risk of bleeding.46,47

One area that’s changed radically for people with haemophilia over the years is advice about keeping fit and taking part in sports. Being active is now encouraged and the emphasis is on what you can do, rather than what you can’t.13 There are very few sports that you can’t take part in. Individual planning for prophylaxis means that your treatment schedule can be planned around your activity so that your factor levels are at their highest when you are most active.34

Travel also needs a bit of planning ahead. You’ll need to take enough factor with you and that also means having a letter from your treatment centre explaining why you need it. Finding out where the nearest haemophilia centre is to where you’re going is also a good idea.

Aside from needing to arrange storage and access to somewhere to self-treat, life at school, college or work should be pretty much the same as for anyone else. Anti-discrimination legislation means that employers have to make reasonable adjustments to accommodate you.48 There are still some jobs you may not be able to do, such as joining the armed forces, the police or the fire service or being an airline pilot.42 But those aside, you can pretty much choose anything that suits you. There will always be the issue of how open you want to be about your condition and, particularly for children, feeling a little different to everyone else. Remember that who you tell and how much will always be up to you.

Guidelines for the Management of Hemophilia (General Care and Management of Hemophilia: 1.1 What is haemophilia?). World Federation of Hemophilia. http://www1.wfh.org/publications/files/pdf-1494.pdf. Last updated April 2013. Accessed 22nd August 2018

2 National Organization for Rare Disorders. Hemophilia A. https://rarediseases.org/rare-diseases/hemophilia-a/. Accessed 22nd August 2018

3 National Organization for Rare Disorders. Hemophilia B. https://rarediseases.org/rare-diseases/hemophilia-b/. Accessed 22nd August 2018

4 UK National Haemophilia Database. UK Bleeding Disorder Statistics for April 2016 to March 2017. http://www.ukhcdo.org/wp-content/uploads/2018/02/Bleeding-Disorder-Statistics-for-April-2016-to-March-2017-for-UKHCDO.pdf. Accessed 5th November 2018

5 World Federation of Hemophilia. How do you get haemophilia? https://www.wfh.org/en/page.aspx?pid=644. Accessed 22nd August 2018

6 World Federation of Hemophilia. Carriers and women with haemophilia. https://www.wfh.org/en/abd/carriers/carriers-and-women-with-hemophilia-en. Accessed 22nd August 2018

7 World Federation of Hemophilia. Inheritance of haemophilia. https://www.wfh.org/en/abd/carriers/carriers-inheritance-of-hemophilia-en. Accessed 5th November 2018

8 Haemophilia. BMJ Best Practice. https://bestpractice.bmj.com/topics/en-gb/468/aetiology. Last updated April 2017. Accessed 22nd August 2018

9 Haemophilia. BMJ Best Practice. https://bestpractice.bmj.com/topics/en-gb/468/diagnosis-approach. Last updated April 2017. Accessed 22nd August 2018

10 World Federation of Hemophilia. Carriers and women with haemophilia. https://www.wfh.org/en/abd/carriers/carriers-and-women-with-hemophilia-en. Accessed 22nd August 2018

11 NHS. Causes Haemophlia. https://www.nhs.uk/conditions/haemophilia/causes/. Accessed 22nd August 2018

12 NHS. Symptoms Haemophilia. https://www.nhs.uk/conditions/haemophilia/symptoms/. Accessed 22nd August 2018

13 Guidelines for the Management of Hemophilia (General Care and Management of Hemophilia: 1.4 Fitness and physical activity). World Federation of Hemophilia. http://www1.wfh.org/publications/files/pdf-1494.pdf. Last updated April 2013. Accessed 22nd August 2018

14 The Haemophilia Society. What to expect from your treatment centre. http://haemophilia.org.uk/bleeding-disorders/treatment-centres/expectations-treatment-centre/. Accessed 22nd August 2018

15 Haemnet. Haemophilia Nursing. https://www.haemnet.com/external/care/haemophilia-nursing. Accessed 22nd August 2018

16 Haemnet. Haemophilia Physiotherapy. https://www.haemnet.com/external/care/haemphilia-physiotherapy. Accessed 22nd August 2018

17 NHS. Treatment. https://www.nhs.uk/conditions/haemophilia/treatment/ Last updated April 2017. Accessed 22nd August 2018

18 Haemophilia. BMJ Best Practice. https://bestpractice.bmj.com/topics/en-gb/468/prevention. Last updated April 2017. Accessed 22nd August 2018

19 Valentino LA1, Ewenstein B, Navickis RJ, Wilkes MM. Haemophilia. Central venous access devices in haemophilia. 2004 Mar;10(2):134-46. https://www.ncbi.nlm.nih.gov/pubmed/14962202 . Accessed November 2018

20 Haemophilia. BMJ Best Practice. https://bestpractice.bmj.com/topics/en-gb/468/history-exam. Last updated April 2017. Accessed 22nd August 2018

21 Treatment of hemophilia (What other treatments are helpful to treat bleeding in hemophiliacs?). Canadian Hemophilia Society. https://www.hemophilia.ca/treatment-of-hemophilia/. Accessed 22nd August 2018

22  Hemophilia A (Overview: management). Medscape. https://emedicine.medscape.com/article/779322-overview. Last updated December 2017 Accessed 22nd August 2018

23 Desmopressin (Mild to moderate haemophilia and von Willebrand's disease). BNF. https://bnf.nice.org.uk/drug/desmopressin.html. Accessed 22nd August 2018

24 Hemophilia B (Overview: management). Medscape. https://emedicine.medscape.com/article/779434-overview. Last updated June 2017. Accessed 22nd August 2018

25 Guidelines for the Management of Hemophilia (General Care and Management of Hemophilia: 1.6 Prophylactic factor replacement therapy). World Federation of Hemophilia. http://www1.wfh.org/publications/files/pdf-1494.pdf. Last updated April 2013. Accessed 22nd August 2018

26 Haemophilia (Management: Approach). BMJ Best Practice. https://bestpractice.bmj.com/topics/en-gb/468/management-approach. Last updated April 2017. Accessed 22nd August 2018

27 Mahlangu J, Powell JS, Ragni MV et al. Phase 3 study of recombinant factor VIII Fc fusion protein in severe hemophilia A. Blood, 2014, vol 123, issue 3, pp317-25. http://www.bloodjournal.org/content/bloodjournal/early/2013/11/13/blood-2013-10-529974.full.pdf

28 Santagostino E, Martinowitx U, Lissitchkov T et al. Long-acting recombinant coagulation factor IX albumin fusion protein (rIX-FP) in hemophilia B: results of a phase 3 trial. BLOOD, April 2016, 127 (14), pp1761-9. In.

http://www.bloodjournal.org/content/bloodjournal/early/2016/01/11/blood-2015-09-669234.full.pdf. Accessed 22nd August 2018

29 Clinical Commissioning Policy: Emicizumab as prophylaxis in people with congenital haemophilia A with factor VIII inhibitors (all ages) NHS England. https://www.england.nhs.uk/wp-content/uploads/2018/07/1717-emicizumab.pdf. Published July 2018. Accessed 22nd August 2018

30 Powell JS, Pasi KJ, Ragni MV et al. Phase 3 Study of Recombinant Factor IX Fc Fusion Protein in Hemophilia B

NEJM, 2013, 369 (24), pp2313-23. In. https://www.nejm.org/doi/full/10.1056/nejmoa1305074. Accessed 22nd August 2018

31 Shima M, Hanabusa H, Taki M, et al. Long-term safety and efficacy of emicizumab in a phase 1/2 study in patients with hemophilia A with or without inhibitors (Discussion). Blood Advances. 2017 1:1891-1899; http://www.bloodadvances.org/content/1/22/1891?sso-checked=true#sec-12. Accessed 22nd August 2018

32 Guidelines for the Management of Hemophilia (General Care and Management of Hemophilia: 1.7 Home therapy). World Federation of Hemophilia. http://www1.wfh.org/publications/files/pdf-1494.pdf. Last updated April 2013. Accessed 22nd August 2018

33 Oxford Handbook of Clinical Pharmacy (3 ed.)  (Therapy-related issues: cardiovascular system: Understanding anticoagulation: Coagulation factors). Oxford Medicine Online. http://oxfordmedicine.com/view/10.1093/med/9780198735823.001.0001/med-9780198735823-chapter-16?rskey=6N7NwM&result=5. Published online April 2017. Accessed 22nd August 2018

34 Current Issues in Prophylaxis. World Federation of Hemophilia. https://www.wfh.org/en/abd/prophylaxis/current-issues-in-prophylaxis. Last updated March 2016. Accessed 22nd August 2018

35 Updates to the Guidelines for the Management of Hemophilia. World Hemophilia Federation. http://www1.wfh.org/publications/files/pdf-1512.pdf. Published April 2013. Accessed 22nd August 2018

36 Guidelines for the Management of Hemophilia (General Care and Management of Hemophilia: 1.9 Pain management). World Federation of Hemophilia. http://www1.wfh.org/publications/files/pdf-1494.pdf. Last updated April 2013. Accessed 22nd August 2018

37 Haemophilia (Complications). BMJ Best Practice. https://bestpractice.bmj.com/topics/en-gb/468/complications. Last updated April 2017. Accessed 22nd August 2018

38 What are inhibitors? World Hemophilia Federation. https://www.wfh.org/en/page.aspx?pid=651. Accessed 22nd August 2018

39 How does immune tolerance induction work? World Hemophilia Federation. https://www.wfh.org/en/page.aspx?pid=647. Last updated December 2014. Accessed 22nd August 2018

40 Guidelines for the Management of Hemophilia (General Care and Management of Hemophilia: Table 1-3: Approximate frequency of bleeding at different sites). World Federation of Hemophilia. http://www1.wfh.org/publications/files/pdf-1494.pdf. Last updated April 2013. Accessed 22nd August 2018

41 Hemophilia A (Overview: Background). Medscape. https://emedicine.medscape.com/article/779322-overview#a2. Last updated December 2017. Accessed 22nd August 2018

42 Hemophilia A Treatment and Management (Approach considerations). Medscape. https://emedicine.medscape.com/article/779322-treatment?src=refgatesrc1. Last updated December 2017. In

43 NHS England, 2013/14 NHS STANDARD CONTRACT FOR HAEMOPHILIA (ALL AGES) https://www.england.nhs.uk/wp-content/uploads/2013/06/b05-haemophilia.pdf. Last accessed December 2018

44 Guidelines for the Management of Hemophilia (1.11 Dental care and management). World Federation of Hemophilia. http://www1.wfh.org/publications/files/pdf-1472.pdf. Last updated April 2013. Accessed 22nd August 2018

45 Drugs that can cause bleeding. World Hemophilia Federatio. https://www.wfh.org/en/page.aspx?pid=639. Last updated February 2010. Accessed 22nd August 2018

46 Guidelines for the Management of Hemophilia (2.4 Vaccinations). World Federation of Hemophilia. http://www1.wfh.org/publications/files/pdf-1472.pdf. Last updated April 2013. Accessed 22nd August 2018

47 Understanding Haemophilia (Medical and dental treatment: Medicines). The Haemophilia Society. http://haemophilia.org.uk/wp-content/uploads/2017/04/Understanding_haemophilia_WEB.pdf. Published February 2017. Accessed 22nd August 2018

48 Careers with a bleeding disorder and the world of work. The Haemophilia Society. http://haemophilia.org.uk/wp-content/uploads/2017/02/careers_info.pdf.pdf. Last updated November 2014. Accessed 22nd August 2018

49 Specialised Services Circular (SSC) 1652 Sept 2016, on advice of NHS England

50 Elocta SmPC. Electronic Medicines Compendium. https://www.medicines.org.uk/emc/product/5100/smpc. Last updated December 2017. Accessed 22nd August 2018

51 Idelvion SmPC. Electronic Medicines Compendium. https://www.medicines.org.uk/emc/product/2146/smpc. Last updated January 2018. Accessed 22nd August 2018

52 Alprolix SmPC. Electronic Medicines Compendium. https://www.medicines.org.uk/emc/product/7277/smpc. Last updated July 2018. Accessed 22nd August 2018

53 Nathwani AC, Davidoff AM, Tuddenham EGD/ Gene Therapy for Hemophilia. Hematology/Oncology Clinics of North America, October 2017, vol 31, issue 5, pp853-68. https://www.sciencedirect.com/science/article/pii/S0889858817301041?via%3Dihub. Accessed 22nd August 2018

54 HOPE-B: Trial of AMT-061 in Severe or Moderately Severe Hemophilia B Patients. Clinical Trials.Gov

https://clinicaltrials.gov/ct2/show/NCT03569891?term=hemophilia+B&rank=15. Last updated July 2018. Accessed 22nd August 2018

55 Long-term Safety and Efficacy Study of SPK-9001 in Individuals with Hemophilia B. Clinical Trials.Gov. https://clinicaltrials.gov/ct2/show/NCT03307980?term=SPK9001&rank=1. Last updated July 2018. Accessed 22nd August 2018

56 Single-Arm Study to Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia Patients (BMN270-301). Clinical Trials.Gov. https://clinicaltrials.gov/ct2/show/NCT03370913. Last updated July 2018. Accessed 22nd August 2018

57 Pasi KJ, Georgiev P, Mant T et al. A subcutaneously administered investigational RNAi therapeutic (Fitusiran, ALN-AT3) targeting antithrombin for treatment of hemophilia:  interim weekly and monthly dosing results in patients with hemophilia A or B. Presentation to American Hematology Society annual meeting, 2015. http://www.alnylam.com/web/assets/ASH-2015_fitusiran_010715.pdf.  Accessed 22nd August 2018

58 A Study of Fitusiran (ALN-AT3SC) in Severe Hemophilia A and B Patients Without Inhibitors. Clinical Trials.gov

https://clinicaltrials.gov/ct2/show/NCT03417245. Last updated June 2018. Accessed 22nd August 2018
59 Korte W. The Potential Close Future of Hemophilia Treatment - Gene Therapy, TFPI Inhibition, Antithrombin Silencing, and Mimicking Factor VIII with an Engineered Antibody. Transfusion Medicine and Hemotherapy, 2018, 45, 2.
https://www.karger.com/Article/FullText/488152. Accessed 22nd August 2018
60 A Trial Evaluating the Efficacy and Safety of Prophylactic Administration of Concizumab in Haemophilia A and B Patients with Inhibitors (explorer™4).
https://clinicaltrials.gov/ct2/show/NCT03196284?term=concizumab&cond=Hemophilia&rank=3 Last updated October 2018. Accessed 22nd August 2018
61 A Trial Evaluating Efficacy and Safety of Prophylactic Administration of Concizumab in Patients with Severe Haemophilia A Without Inhibitors (explorer™5).
https://clinicaltrials.gov/ct2/show/NCT03196297?term=concizumab&cond=Hemophilia&rank=1 Last updated September 2018. Accessed 22nd August 2018
62 Multiple Escalating Dose Study of BAY1093884 in Adults With Haemophilia A or B With or Without Inhibitors
https://clinicaltrials.gov/ct2/show/NCT03597022?term=anti-TFPI&cond=Hemophilia&rank=1  Last updated October 2018. Accessed 5th November 2018

 

Please note that the information provided in the above FAQs is accurate as of December 2018.

The patient advocacy campaign called, Could there be more?, is sponsored by pharmaceutical company, Sobi, who is working in collaboration with The Haemophilia Society and the haemophilia community to launch new haemophilia awareness resources for patients.

Logos

FAQs - The Fundamentals of Haemophilia Treatment

Most people with haemophilia need treatment at some point. How regularly depends on whether they have mild, moderate or severe haemophilia. Your child will have a blood test to find this out.

Haemophilia means your child’s blood has a lower than normal level of one of the body’s essential clotting factors. It they have less than 1% of normal, they have severe haemophilia. If they have more than 5% of the normal level, their haemophilia is mild.8  People with between 1% and 5% of the normal clotting factor level have moderate haemophilia.8

People with severe haemophilia and some people with moderate haemophilia need regular injections of the clotting factor their body is lacking They usually have these injections into a vein (intravenously) about 2 or 3 times a week.18 Young children may also be offered a central venous access device (CVAD) such as a Port-A-Cath to make infusions easier.19 This treatment is called ‘prophylaxis’ (pronounced prof-ill-ax-iss) and the aim is to prevent bleeding. They will also need extra treatment before an operation or having a tooth out.

People with mild or moderate haemophilia may also need preventative treatment before surgery.22 They may be able to have tablets or a nasal spray to stop bleeding, rather than injections of clotting factor.21,22,23

Even with regular preventative treatment, people with severe haemophilia can have bleeding episodes, usually after a knock or accident but sometimes for no obvious reason. People with mild and moderate haemophilia may also have bleeds after an accident or injury. If anyone with haemophilia has a bleed into a muscle or joint, they will need injections of clotting factor until the bleeding is under control. Bruises and minor cuts will often get better on their own but signs of a more serious bleed can include:9

  • Stiffness of a joint
  • Tingling or pins and needles
  • Heat
  • Swelling
  • Pain

If in doubt, seek medical help sooner rather than later.

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 This means that factor VIII no longer helps them because their immune system works against it. Emicizumab only works in haemophilia A and 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.

The main difference is that they need injections to replace different clotting factors. In haemophilia A, factor VIII (8) is missing. In haemophilia B, it is factor IX (9) that is missing.

These two clotting factors last for different lengths of time in the blood. So people with severe haemophilia A generally need preventative injections 3 times a week or every other day and those with severe haemophilia B, twice a week.18 This is called prophylaxis (pronounced prof-ill-ax-iss).

There are new treatments for haemophilia A and B that are available. These are known as ‘extended half-life’ factor replacement. They stay in the blood stream for longer, so you don’t have to give yourself the prophylaxis injections so often. For haemophilia A, you may be able to have factor injections twice a week, instead of every other day.27 For haemophilia B, you may be able to have factor injections once a week or once a fortnight instead of twice a week.28,30

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

Most people with severe and moderate haemophilia self-treat. As babies and children of course, their parents are taught how to give the injections. But when they’re old enough, haemophilia doctors and nurses encourage children to learn to do it themselves.32

Knowing how to give your own injections gives you a lot more freedom and independence.32 You don’t have to keep going to hospital. You can go out and about more easily without having to worry about being within reach of the person who usually does your injections. Some people time their injections so that they have one just before taking part in sport, for example.

Doing your own injections, or, for children, your parents doing it, also means that you can give yourself treatment straight away should you have a bleed. That way, you are less likely to have long term problems and can recover more quickly.32

Treatment for haemophilia works by replacing the clotting factor that you would normally have in your blood.

Blood clotting is very complicated. There is what is called a ‘clotting cascade’33 - when proteins in the blood are activated to stop bleeding. There are 13 of these proteins or clotting factors.

For a clot to form, the first clotting factor acts on the second, which acts on the next and so on – the ‘cascade’. A clot won’t form until all the steps in the cascade have completed.

In haemophilia, one of the proteins is missing or defficient. That’s why the blood doesn’t clot and you have bleeding. The gap in the clotting cascade is filled with factor replacement. If you have haemophilia A, you will be prescribed factor VIII (8). If you have haemophilia B, you will be prescribed factor IX (9).

The clotting factors get used up all the time, which is why you need to have treatment injections so often. You need to have your factor as an injection as it needs to be delivered directly into your blood.

Clotting Cascade

Most people with severe and many people with moderate haemophilia A choose to have regular treatment to help prevent bleeds. This is called prophylaxis (pronounced prof-ill-ax-iss). Most people with moderate haemophilia A and all people with mild haemophilia A will treat on-demand, that is following a bleed or for surgery.

Factor VIII (the missing clotting factor in haemophilia A) doesn’t last long in the blood stream. So in general, people with haemophilia A on prophylaxis need treatment about 3 times a week, or every other day.25

These days, treatment schedules are worked out individually for you.34 You have a series of blood tests carried out. These look at how your body manages and uses up factor and are called pharmacokinetics.34 They help your haematologist (a specialist doctor in blood disorders, pronounced heem-a-toll-o-jist) to advise on how much factor VIII (8) you need each time you treat yourself and how often you need to do this. They will also take into account your age, weight, history of bleeds and how active you are.34,35

There are now newer treatments available. For haemophilia A, there is now a form of factor VIII (8) that lasts longer in the blood stream.27 This treatment doesn’t suit everyone. You have pharmacokinetic tests carried out on your blood using the new treatment to find out.27 If it is suitable for you, the tests will tell your haematologist how often you need prophylaxis injections. For different people, this can vary from once every 3 days to once every 5 days.27 If you’ve previously needed injections every other day, that means at least a third fewer injections. Over a year, that’s more than 50 injections you don’t need to have.

Although doctors recommend strongly that everyone with severe haemophilia has prophylaxis, some people with haemophilia choose not to. They prefer to treat only if they have a bleed. In that case, how often you have treatment will be impossible to predict. The newer longer lasting factor VIII (8) does tend to get bleeding under control with fewer injections. For some people, a single injection may be enough.27

Just about everyone with severe haemophilia A gives themselves their factor injections once they are old enough. Most people become expert at this and can do it pretty quickly. The time it takes varies depending on how much factor you need, but it should only take around 15 to 20 minutes. People do generally get very used to doing it because they have to do it so often.

Everyone with haemophilia is advised to have some factor and their infusion kit with them at all times, although people with mild haemophilia should only use this in consultation with their haemophilia centre. You never know what’s going to happen and unfortunately even a minor accident can cause bleeding that needs immediate treatment.

Most people with severe haemophilia B choose to have regular treatment to help prevent bleeds. This is called prophylaxis (pronounced prof-ill-ax-iss). Most people with moderate haemophilia B and all people with mild haemophilia B will treat on-demand, that is following a bleed or for surgery.

Factor IX (9) - the missing clotting factor in haemophilia B – lasts a little longer in the blood stream than the factor VIII (8) used to treat haemophilia A. So in general, people with severe haemophilia B need treatment about twice a week.25

These days, treatment schedules are worked out individually for you.34 You have a series of blood tests carried out. These look at how your body manages and uses up factor and are called pharmacokinetics.34 They help your haematologist (a specialist doctor in blood disorders, pronounced heem-a-toll-o-jist) to advise on how much factor IX (9) you need each time you treat yourself and how often you need to do this. They will also take into account your age, weight, history of bleeds and how active you are.35

There are now newer treatments available. For haemophilia B, there is now a form of factor IX (9) that lasts longer in the blood stream.28 This treatment doesn’t suit everyone. You have pharmacokinetic tests carried out on your blood using the new treatment to find out.28 If it is suitable for you, the tests will tell your haematologist how often you need prophylaxis injections. For different people, this can vary from once a week to once a fortnight.28 If you’ve previously needed injections twice a week, that means half as many or even a quarter of the number of injections you used to have.

Although doctors recommend strongly that everyone with severe haemophilia has prophylaxis, some people with haemophilia choose not to. They prefer to treat only if they have a bleed. In that case, how often you have treatment will be impossible to predict. The newer longer lasting factor IX does tend to get bleeding under control with fewer injections. In many situations, a single injection may be enough.28,30

Just about everyone with severe haemophilia B gives themselves their factor injections once they are old enough. Most people become expert at this and can do it pretty quickly. The time it takes varies depending on how much factor you need, but it should only take around 15 to 20 minutes. People do generally get very used to doing it because they have to do it so often.

Everyone with haemophilia is advised to have some factor and their infusion kit with them at all times, although people with mild haemophilia should only use this in consultation with their haemophilia centre. You never know what’s going to happen and unfortunately even a minor accident can cause bleeding that needs immediate treatment.

Because clotting factors are manufactured forms of natural body substances, there are very few side effects compared to drugs you may take for other medical conditions. Clotting factors used to be only made from human blood and then there was a risk of picking up an infection from the donors’ blood, such as hepatitis or HIV. In the 80s, donor screening as well as techniques for treated plasma-derived clotting factors such as dry heat, solvent-detergent treatment, and pasteurisation vastly reduced this risk. Starting in the late 90s, most patients in the UK now use clotting factors made in the lab, without the need for human blood plasma.

The most common problem is pain at the injection site when having the treatment.

There are a couple of ways of helping children to cope. One is putting anaesthetic cream on the injection site about half an hour before giving the treatment.36 This numbs the area so they are less likely to find the needle going in painful, but they can still find having the injection upsetting. Another is to put in a central venous access device often called a ‘port’. This is a device that sits just under the skin that you inject into.32 The needle still has to go through the skin, but it’s less painful. The port connects to a vein inside the body so the treatment can still get straight into your blood. There can be problems with venous access devices. They can become infected or blocked and then have to be taken out.32

Some people with haemophilia develop a strong dislike of injecting and prefer to only treat if they have a bleed rather than have preventative factor (prophylaxis – pronounced prof-ill-ax-iss). This means they will have fewer injections. But it also means they are more at risk of painful bleeds into joints and muscles.25 Over years, joints can become seriously damaged by the bleeds, causing pain and disability.25, 36

Having repeated injections over a lifetime can cause problems with veins. People with haemophilia are advised to vary their injection sites. Even so, though some veins become unusable most people with haemophilia become very good at finding usable veins.

The most serious side effect of treatment with clotting factors is the development of inhibitors.37  These are antibodies to the clotting factor and mean your immune system is reacting against it.38 About 3 in every 10 people with haemophilia A develop inhibitors at some point.37 It is much rarer in haemophilia B, with about 3 in every 100 people developing them.37

Developing inhibitors is serious because it means that controlling bleeding is much more difficult.38 People with inhibitors may have bleeding controlled with a treatment called activated prothrombin complex concentrate or another clotting factor called VIIa (7a).26,29

There is treatment to get rid of inhibitors. It involves having larger and more frequent infusions of clotting factor to get your immune system to learn to tolerate it again.39 This is called Immune Tolerance Induction. This is effective in about 2 out of every 3 people who develop inhibitors.29 

There is a newer treatment available called emicizumab (pronounced em-ih-siz-oo-mab).29 This is an injection under the skin that you have once a week. It works by bypassing factor VIII (8) in blood clotting and so controls bleeding.29

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.

Thanks to the introduction of preventative treatment, improvements in safety of plasma products and the development of clotting factor made in the lab, people with haemophilia are growing up healthier and with less lasting damage from bleeds than they used to.

That’s not to say that there aren’t some demands placed on people with haemophilia that others’ don’t have. You will always need to have treatment, whether that’s regular preventative treatment (prophylaxis, pronounced prof-ill-ax-iss) or treatment for bleeds.25

Everyone with haemophilia should have their care co-ordinated through a haemophilia centre.41 You may have all your treatment managed through a comprehensive care centre (CCC), but some people live too far away to go regularly and will have their day to day treatment through a haemophilia centre (HTC) at a nearer hospital. The treatment your HTC gives you should always be overseen by the haemophilia comprehensive care centre because that is the centre of expertise. They will have specialist nurses, physios and other health professionals, not only specialist doctors.42 People who have their haemophilia managed through a comprehensive care centre do better overall than those who don’t.42

Every now and then, you will have to travel to your comprehensive care centre. A couple of times a year, you will need to see the haemophilia specialist physiotherapist. They need to check your joints for damage from any bleeds and can give you an exercise programme to strengthen your muscles and joints and make bleeds less likely.26

As a person with haemophilia you should have contact with your centre at least once a year. If you have severe haemophilia this will be at least twice a year and for very young children, it will be 3-4 times a year.43

In between check-ups, you give your treatment yourself at home. Or, for children, usually their parents give it. Being able to give your own treatment at home does make life easier. But making sure you have the absolute minimum of long term damage from bleeds means that you should always have factor and infusion kits with you.

Giving themselves treatment becomes second nature to most people and they are able to inject themselves quickly and relatively easily, keeping to good hygiene standards so that they don’t pick up an infection. People also learn to spot symptoms of bleeds so that they can treat as early as possible and minimise damage. Very early signs can be difficult to spot – people talk about tingling or bubbling, stiffness and warmth.

You should have been given a Bleeding Disorder Information Card by your haemophilia comprehensive care centre. This has your diagnosis and the number of your haemophilia comprehensive care centre. You should have this on you all the time – just in case. Many people also wear a medical alert bracelet to tell people you have haemophilia.

However well you manage it, haemophilia will affect your daily life to some extent. You’ll always have to put a little bit of extra planning or thought into what you do and where you go than other people need to. But with preventative treatment (prophylaxis) there is less of an impact than there used to be.

There are some implications for your general health if you have haemophilia. Because of the risk of bleeding, it’s important to take very good care of your teeth to prevent decay and gum disease.44 You need to see the dentist and hygienist regularly.44

There are some over-the-counter medicines you need to avoid, including aspirin and anti-inflammatories such as ibuprofen.45 If in any doubt at all (including about herbal medicines) it’s best to check with your haemophilia centre.45 You can have vaccinations as injections under the skin (subcutaneously), but shouldn’t have injections into a muscle because of the risk of bleeding.46,47

One area that’s changed radically for people with haemophilia over the years is advice about keeping fit and taking part in sports. Being active is now encouraged and the emphasis is on what you can do, rather than what you can’t.13 There are very few sports that you can’t take part in. Individual planning for prophylaxis means that your treatment schedule can be planned around your activity so that your factor levels are at their highest when you are most active.34

Travel also needs a bit of planning ahead. You’ll need to take enough factor with you and that also means having a letter from your treatment centre explaining why you need it. Finding out where the nearest haemophilia centre is to where you’re going is also a good idea.

Aside from needing to arrange storage and access to somewhere to self-treat, life at school, college or work should be pretty much the same as for anyone else. Anti-discrimination legislation means that employers have to make reasonable adjustments to accommodate you.48 There are still some jobs you may not be able to do, such as joining the armed forces, the police or the fire service or being an airline pilot.42 But those aside, you can pretty much choose anything that suits you. There will always be the issue of how open you want to be about your condition and, particularly for children, feeling a little different to everyone else. Remember that who you tell and how much will always be up to you.

Guidelines for the Management of Hemophilia (General Care and Management of Hemophilia: 1.1 What is haemophilia?). World Federation of Hemophilia. http://www1.wfh.org/publications/files/pdf-1494.pdf. Last updated April 2013. Accessed 22nd August 2018

2 National Organization for Rare Disorders. Hemophilia A. https://rarediseases.org/rare-diseases/hemophilia-a/. Accessed 22nd August 2018

3 National Organization for Rare Disorders. Hemophilia B. https://rarediseases.org/rare-diseases/hemophilia-b/. Accessed 22nd August 2018

4 UK National Haemophilia Database. UK Bleeding Disorder Statistics for April 2016 to March 2017. http://www.ukhcdo.org/wp-content/uploads/2018/02/Bleeding-Disorder-Statistics-for-April-2016-to-March-2017-for-UKHCDO.pdf. Accessed 5th November 2018

5 World Federation of Hemophilia. How do you get haemophilia? https://www.wfh.org/en/page.aspx?pid=644. Accessed 22nd August 2018

6 World Federation of Hemophilia. Carriers and women with haemophilia. https://www.wfh.org/en/abd/carriers/carriers-and-women-with-hemophilia-en. Accessed 22nd August 2018

7 World Federation of Hemophilia. Inheritance of haemophilia. https://www.wfh.org/en/abd/carriers/carriers-inheritance-of-hemophilia-en. Accessed 5th November 2018

8 Haemophilia. BMJ Best Practice. https://bestpractice.bmj.com/topics/en-gb/468/aetiology. Last updated April 2017. Accessed 22nd August 2018

9 Haemophilia. BMJ Best Practice. https://bestpractice.bmj.com/topics/en-gb/468/diagnosis-approach. Last updated April 2017. Accessed 22nd August 2018

10 World Federation of Hemophilia. Carriers and women with haemophilia. https://www.wfh.org/en/abd/carriers/carriers-and-women-with-hemophilia-en. Accessed 22nd August 2018

11 NHS. Causes Haemophlia. https://www.nhs.uk/conditions/haemophilia/causes/. Accessed 22nd August 2018

12 NHS. Symptoms Haemophilia. https://www.nhs.uk/conditions/haemophilia/symptoms/. Accessed 22nd August 2018

13 Guidelines for the Management of Hemophilia (General Care and Management of Hemophilia: 1.4 Fitness and physical activity). World Federation of Hemophilia. http://www1.wfh.org/publications/files/pdf-1494.pdf. Last updated April 2013. Accessed 22nd August 2018

14 The Haemophilia Society. What to expect from your treatment centre. http://haemophilia.org.uk/bleeding-disorders/treatment-centres/expectations-treatment-centre/. Accessed 22nd August 2018

15 Haemnet. Haemophilia Nursing. https://www.haemnet.com/external/care/haemophilia-nursing. Accessed 22nd August 2018

16 Haemnet. Haemophilia Physiotherapy. https://www.haemnet.com/external/care/haemphilia-physiotherapy. Accessed 22nd August 2018

17 NHS. Treatment. https://www.nhs.uk/conditions/haemophilia/treatment/ Last updated April 2017. Accessed 22nd August 2018

18 Haemophilia. BMJ Best Practice. https://bestpractice.bmj.com/topics/en-gb/468/prevention. Last updated April 2017. Accessed 22nd August 2018

19 Valentino LA1, Ewenstein B, Navickis RJ, Wilkes MM. Haemophilia. Central venous access devices in haemophilia. 2004 Mar;10(2):134-46. https://www.ncbi.nlm.nih.gov/pubmed/14962202 . Accessed November 2018

20 Haemophilia. BMJ Best Practice. https://bestpractice.bmj.com/topics/en-gb/468/history-exam. Last updated April 2017. Accessed 22nd August 2018

21 Treatment of hemophilia (What other treatments are helpful to treat bleeding in hemophiliacs?). Canadian Hemophilia Society. https://www.hemophilia.ca/treatment-of-hemophilia/. Accessed 22nd August 2018

22  Hemophilia A (Overview: management). Medscape. https://emedicine.medscape.com/article/779322-overview. Last updated December 2017 Accessed 22nd August 2018

23 Desmopressin (Mild to moderate haemophilia and von Willebrand's disease). BNF. https://bnf.nice.org.uk/drug/desmopressin.html. Accessed 22nd August 2018

24 Hemophilia B (Overview: management). Medscape. https://emedicine.medscape.com/article/779434-overview. Last updated June 2017. Accessed 22nd August 2018

25 Guidelines for the Management of Hemophilia (General Care and Management of Hemophilia: 1.6 Prophylactic factor replacement therapy). World Federation of Hemophilia. http://www1.wfh.org/publications/files/pdf-1494.pdf. Last updated April 2013. Accessed 22nd August 2018

26 Haemophilia (Management: Approach). BMJ Best Practice. https://bestpractice.bmj.com/topics/en-gb/468/management-approach. Last updated April 2017. Accessed 22nd August 2018

27 Mahlangu J, Powell JS, Ragni MV et al. Phase 3 study of recombinant factor VIII Fc fusion protein in severe hemophilia A. Blood, 2014, vol 123, issue 3, pp317-25. http://www.bloodjournal.org/content/bloodjournal/early/2013/11/13/blood-2013-10-529974.full.pdf

28 Santagostino E, Martinowitx U, Lissitchkov T et al. Long-acting recombinant coagulation factor IX albumin fusion protein (rIX-FP) in hemophilia B: results of a phase 3 trial. BLOOD, April 2016, 127 (14), pp1761-9. In.

http://www.bloodjournal.org/content/bloodjournal/early/2016/01/11/blood-2015-09-669234.full.pdf. Accessed 22nd August 2018

29 Clinical Commissioning Policy: Emicizumab as prophylaxis in people with congenital haemophilia A with factor VIII inhibitors (all ages) NHS England. https://www.england.nhs.uk/wp-content/uploads/2018/07/1717-emicizumab.pdf. Published July 2018. Accessed 22nd August 2018

30 Powell JS, Pasi KJ, Ragni MV et al. Phase 3 Study of Recombinant Factor IX Fc Fusion Protein in Hemophilia B

NEJM, 2013, 369 (24), pp2313-23. In. https://www.nejm.org/doi/full/10.1056/nejmoa1305074. Accessed 22nd August 2018

31 Shima M, Hanabusa H, Taki M, et al. Long-term safety and efficacy of emicizumab in a phase 1/2 study in patients with hemophilia A with or without inhibitors (Discussion). Blood Advances. 2017 1:1891-1899; http://www.bloodadvances.org/content/1/22/1891?sso-checked=true#sec-12. Accessed 22nd August 2018

32 Guidelines for the Management of Hemophilia (General Care and Management of Hemophilia: 1.7 Home therapy). World Federation of Hemophilia. http://www1.wfh.org/publications/files/pdf-1494.pdf. Last updated April 2013. Accessed 22nd August 2018

33 Oxford Handbook of Clinical Pharmacy (3 ed.)  (Therapy-related issues: cardiovascular system: Understanding anticoagulation: Coagulation factors). Oxford Medicine Online. http://oxfordmedicine.com/view/10.1093/med/9780198735823.001.0001/med-9780198735823-chapter-16?rskey=6N7NwM&result=5. Published online April 2017. Accessed 22nd August 2018

34 Current Issues in Prophylaxis. World Federation of Hemophilia. https://www.wfh.org/en/abd/prophylaxis/current-issues-in-prophylaxis. Last updated March 2016. Accessed 22nd August 2018

35 Updates to the Guidelines for the Management of Hemophilia. World Hemophilia Federation. http://www1.wfh.org/publications/files/pdf-1512.pdf. Published April 2013. Accessed 22nd August 2018

36 Guidelines for the Management of Hemophilia (General Care and Management of Hemophilia: 1.9 Pain management). World Federation of Hemophilia. http://www1.wfh.org/publications/files/pdf-1494.pdf. Last updated April 2013. Accessed 22nd August 2018

37 Haemophilia (Complications). BMJ Best Practice. https://bestpractice.bmj.com/topics/en-gb/468/complications. Last updated April 2017. Accessed 22nd August 2018

38 What are inhibitors? World Hemophilia Federation. https://www.wfh.org/en/page.aspx?pid=651. Accessed 22nd August 2018

39 How does immune tolerance induction work? World Hemophilia Federation. https://www.wfh.org/en/page.aspx?pid=647. Last updated December 2014. Accessed 22nd August 2018

40 Guidelines for the Management of Hemophilia (General Care and Management of Hemophilia: Table 1-3: Approximate frequency of bleeding at different sites). World Federation of Hemophilia. http://www1.wfh.org/publications/files/pdf-1494.pdf. Last updated April 2013. Accessed 22nd August 2018

41 Hemophilia A (Overview: Background). Medscape. https://emedicine.medscape.com/article/779322-overview#a2. Last updated December 2017. Accessed 22nd August 2018

42 Hemophilia A Treatment and Management (Approach considerations). Medscape. https://emedicine.medscape.com/article/779322-treatment?src=refgatesrc1. Last updated December 2017. In

43 NHS England, 2013/14 NHS STANDARD CONTRACT FOR HAEMOPHILIA (ALL AGES) https://www.england.nhs.uk/wp-content/uploads/2013/06/b05-haemophilia.pdf. Last accessed December 2018

44 Guidelines for the Management of Hemophilia (1.11 Dental care and management). World Federation of Hemophilia. http://www1.wfh.org/publications/files/pdf-1472.pdf. Last updated April 2013. Accessed 22nd August 2018

45 Drugs that can cause bleeding. World Hemophilia Federatio. https://www.wfh.org/en/page.aspx?pid=639. Last updated February 2010. Accessed 22nd August 2018

46 Guidelines for the Management of Hemophilia (2.4 Vaccinations). World Federation of Hemophilia. http://www1.wfh.org/publications/files/pdf-1472.pdf. Last updated April 2013. Accessed 22nd August 2018

47 Understanding Haemophilia (Medical and dental treatment: Medicines). The Haemophilia Society. http://haemophilia.org.uk/wp-content/uploads/2017/04/Understanding_haemophilia_WEB.pdf. Published February 2017. Accessed 22nd August 2018

48 Careers with a bleeding disorder and the world of work. The Haemophilia Society. http://haemophilia.org.uk/wp-content/uploads/2017/02/careers_info.pdf.pdf. Last updated November 2014. Accessed 22nd August 2018

49 Specialised Services Circular (SSC) 1652 Sept 2016, on advice of NHS England

50 Elocta SmPC. Electronic Medicines Compendium. https://www.medicines.org.uk/emc/product/5100/smpc. Last updated December 2017. Accessed 22nd August 2018

51 Idelvion SmPC. Electronic Medicines Compendium. https://www.medicines.org.uk/emc/product/2146/smpc. Last updated January 2018. Accessed 22nd August 2018

52 Alprolix SmPC. Electronic Medicines Compendium. https://www.medicines.org.uk/emc/product/7277/smpc. Last updated July 2018. Accessed 22nd August 2018

53 Nathwani AC, Davidoff AM, Tuddenham EGD/ Gene Therapy for Hemophilia. Hematology/Oncology Clinics of North America, October 2017, vol 31, issue 5, pp853-68. https://www.sciencedirect.com/science/article/pii/S0889858817301041?via%3Dihub. Accessed 22nd August 2018

54 HOPE-B: Trial of AMT-061 in Severe or Moderately Severe Hemophilia B Patients. Clinical Trials.Gov

https://clinicaltrials.gov/ct2/show/NCT03569891?term=hemophilia+B&rank=15. Last updated July 2018. Accessed 22nd August 2018

55 Long-term Safety and Efficacy Study of SPK-9001 in Individuals with Hemophilia B. Clinical Trials.Gov. https://clinicaltrials.gov/ct2/show/NCT03307980?term=SPK9001&rank=1. Last updated July 2018. Accessed 22nd August 2018

56 Single-Arm Study to Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia Patients (BMN270-301). Clinical Trials.Gov. https://clinicaltrials.gov/ct2/show/NCT03370913. Last updated July 2018. Accessed 22nd August 2018

57 Pasi KJ, Georgiev P, Mant T et al. A subcutaneously administered investigational RNAi therapeutic (Fitusiran, ALN-AT3) targeting antithrombin for treatment of hemophilia:  interim weekly and monthly dosing results in patients with hemophilia A or B. Presentation to American Hematology Society annual meeting, 2015. http://www.alnylam.com/web/assets/ASH-2015_fitusiran_010715.pdf.  Accessed 22nd August 2018

58 A Study of Fitusiran (ALN-AT3SC) in Severe Hemophilia A and B Patients Without Inhibitors. Clinical Trials.gov

https://clinicaltrials.gov/ct2/show/NCT03417245. Last updated June 2018. Accessed 22nd August 2018
59 Korte W. The Potential Close Future of Hemophilia Treatment - Gene Therapy, TFPI Inhibition, Antithrombin Silencing, and Mimicking Factor VIII with an Engineered Antibody. Transfusion Medicine and Hemotherapy, 2018, 45, 2.
https://www.karger.com/Article/FullText/488152. Accessed 22nd August 2018
60 A Trial Evaluating the Efficacy and Safety of Prophylactic Administration of Concizumab in Haemophilia A and B Patients with Inhibitors (explorer™4).
https://clinicaltrials.gov/ct2/show/NCT03196284?term=concizumab&cond=Hemophilia&rank=3 Last updated October 2018. Accessed 22nd August 2018
61 A Trial Evaluating Efficacy and Safety of Prophylactic Administration of Concizumab in Patients with Severe Haemophilia A Without Inhibitors (explorer™5).
https://clinicaltrials.gov/ct2/show/NCT03196297?term=concizumab&cond=Hemophilia&rank=1 Last updated September 2018. Accessed 22nd August 2018
62 Multiple Escalating Dose Study of BAY1093884 in Adults With Haemophilia A or B With or Without Inhibitors
https://clinicaltrials.gov/ct2/show/NCT03597022?term=anti-TFPI&cond=Hemophilia&rank=1  Last updated October 2018. Accessed 5th November 2018

 

Please note that the information provided in the above FAQs is accurate as of December 2018.

The patient advocacy campaign called, Could there be more?, is sponsored by pharmaceutical company, Sobi, who is working in collaboration with The Haemophilia Society and the haemophilia community to launch new haemophilia awareness resources for patients.

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