Different types of treatments are available for managing haemophilia and may vary depending on how severe it is. Prompt, effective treatment and prevention of bleeding significantly reduces the risk of complications and disruptions to school, work and family life.
Clotting factor concentrates (CFCs)
Bleeding can be controlled or prevented by replacing the missing clotting factor in the blood through an infusion of clotting factor concentrate. The level of factor VIII or factor IX is increased temporarily, so infusions need to be repeated. For some minor bleeding episodes, one infusion may be enough to stop bleeding. For more serious bleeding or where the first infusion has been delayed, treatment may be needed once or twice a day until the bleeding stops.
Recombinant factor VIII and factor IX concentrates are made using genetic technology and aren’t made from blood.
Treatment can be given in two ways
|On-demand||Treatment is given when bleeding occurs, such as after an injury.||– very young children |
– some adults with severe haemophilia
– people with mild or moderate haemophilia
|Prophylaxis||Treatment is given regularly to prevent bleeding before it starts.||– children with severe haemophilia |
– most adults with severe haemophilia
– some people with moderate haemophilia who have
frequent bleeding problems
Treatment will be needed by everyone with haemophilia if they are bleeding. It will also be necessary to prevent bleeding if they need an operation or have an injury where there is a risk of bleeding.
Clotting factor concentrate is given intravenously (into a vein) into the bloodstream through a needle. Children can have anaesthetic cream rubbed onto the skin before an injection to reduce any pain. The puncture caused by the needle is small and does not result in excessive bleeding.
Standard half-life (SHL) treatment
Standard half-life factor concentrates stay in the blood for a relatively short time and require repeat injections for prophylaxis – every 2-3 days for haemophilia A and twice a week for haemophilia B.
Extended half-life (EHL) treatment
These are factor VIII or FIX products that can stay in the bloodstream for longer. If this treatment is suitable for your child, they may be able to have injections to prevent bleeds (prophylaxis) less often or may stay on the same dose/schedule and have higher trough levels. They are likely to need fewer injections to control any bleeds they have. Half-lives are shorter in children and young people than adults.
Emicizumab is a subcutaneous (under the skin) treatment used to prevent or reduce bleeding in people with severe haemophilia A that is given weekly, two weekly or once a month. It is an antibody which mimics the action of factor VIII. Emicizumab can be prescribed for very young babies. However, the clotting system doesn’t fully mature until 6 months of age, so you will need to discuss with your haemophilia team what the best option for you and your child is. Emicizumab is not a treatment for bleeds, serious injury or major surgery; factor VIII would need to be given to treat bleeds.
For more information please visit our website https://haemophilia.org.uk/resources/publications/factsheets/
Bleeding must be treated as soon as possible. Prompt treatment helps relieve pain quickly, shortens recovery time and reduces the chance of permanent damage.
Treatment with regular infusions of clotting factor aims to prevent the long-term damage caused by bleeding into joints and muscles. Prophylaxis works by stopping the bleeding that occurs without any obvious injury, often called spontaneous bleeding. It also reduces the risk of bleeding from minor injuries. Research has shown that prophylaxis gives children the best chance to reach adulthood without damage to their joints. How often the infusions need to be given is decided for the individual but is typically every other day for haemophilia A and 2-3 times a week for haemophilia B because factor IX lasts a bit longer in the blood.
Most people with severe haemophilia and some with moderate haemophilia learn to treat themselves/their children with clotting factor at home. Home treatment has many advantages:
- makes regular treatment possible
- treatment can be given as soon as possible after bleeding starts
- there is less disruption to home, school and working life
- greater independence and control for the parent/person with haemophilia
- reduced need for hospital attendances
- early treatment reduces recovery time and risk of permanent damage
At first parents are taught by a specialist haemophilia nurse how to give treatment to their child. In time children will learn how to self-treat – most can do this by the time they reach secondary school.
A butterfly needle is inserted into a vein in the hand or arm, the clotting factor is infused and the needle is removed. Alternatively, in very small children or those whose veins are quite difficult to access, an implantable port-a-cath (see below) can be inserted to make having the injections easier. This is a small device inserted under the skin (under general anaesthetic in the operating theatre). Part of the port has a small container called a reservoir and this is placed under the skin. A special needle can be put into the port through the skin so the factor can be injected.
DDAVP® is a synthetic drug that can be suitable for some people with milder forms of haemophilia A. It releases factor VIII stored in the lining of blood vessels, increasing the amount of factor VIII circulating in the blood. This increase can be enough to control minor bleeding episodes and to prevent bleeding from minor operations including dentistry. It is given as a subcutaneous injection (under the skin like a vaccination) or as a nasal spray. It can’t work for severe haemophilia as there are no stores of factor VIII.
Tranexamic acid is a medicine that helps to hold a clot in place once it has formed. It comes as a liquid or tablet and can also be used in a mouthwash. It can be particularly helpful for bleeding in the mouth, nosebleeds or heavy periods. It’s often used at the same time as clotting factor or DDAVP® but can be used on its own.