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Treatment for haemophilia today is very effective. The type of treatment will depend on the severity of the haemophilia. The aim is to increase the levels of the missing clotting factor to stop bleeding.

For some patients (usually those with more severe disease), the missing clotting factor is injected into the bloodstream using a needle. Bleeding stops when enough clotting factor reaches the spot that is bleeding.

Bleeding should be treated as quickly as possible. Quick treatment will help reduce pain and damage to the joints, muscles, and organs. If bleeding is treated quickly, less blood product is needed to stop the bleeding.

With an adequate quantity of treatment products and proper care, people with haemophilia can live perfectly healthy lives.

Types of treatment

Factor concentrates

Factor concentrates are the treatment of choice for severe haemophilia. They can be made from human blood (called plasma-derived products) or manufactured using genetically engineered cells that carry a human factor gene (called recombinant products). Factor concentrates are made in sophisticated manufacturing facilities. All commercially prepared factor concentrates are treated to remove or inactivate blood-borne viruses.

Factor concentrates may also be used in mild and moderate haemophilia for procedures, surgery or a severe injury or bleed.

DDAVP

People with mild haemophilia A may sometimes use desmopressin (also called DDAVP) to treat minor bleeding. DDAVP is a synthetic hormone that stimulates the release of factor VIII from a persons own cells.

Tranexamic acid

This helps to prevent the breakdown of clots and is often used at the same time as treatment with factor VIII or IX, for example if dental treatment is needed.

In mild haemophilia this may be the only treatment that is required for a small procedure or bleed.

It is important that patients discuss their treatment options with their own haemophilia centre.

Treatment regimens

There are several different treatment regimens that people with haemophilia may follow. This will depend on the type of haemophilia a patient has as well as the severity of the haemophilia. The choice of treatment regimen will be decided between the patient (or their parents/carers) and the healthcare team looking after them.

Treatment options:

‘Prophylactic’ – to prevent the bleed starting in the first place

This is when treatment is taken regularly, usually on alternate days if you have haemophilia A, or twice a week if you have haemophilia B. The level of prophylaxis varies between individuals depending on many factors including age, severity of haemophilia and lifestyle.

Prophylaxis helps the blood to clot, minimises the likelihood of long term joint damage and can help people to have a more normally active lifestyle. Many people build their treatment regimen around sporting activities. Look at the sports and exercise section of our website for more information.

Having prophylaxis does mean regular injections of factor, which does require some time commitment from the person with haemophilia or their parents or carers. Nurse specialists at the haemophilia centre will teach parents, carers and patients how to self inject to allow independence.

In young children, due to the small size of their veins, a portacath may be inserted to allow regular treatment to be given.

‘on-demand’ – treatment once a bleed has started

This regimen is more common in patients with mild or moderate haemophilia but is also used by some with severe haemophilia. Treatment is given at the time a bleed happens.

It is important to treat promptly as a delay in treatment may lead to damage, for example in joints.

Select content reproduced with permission from www.wfh.org

More information about treatment of Haemophilia can be found on the NHS Website and on the World Federation of Hemophilia website here and here.