What is the treatment for haemophilia?
Treatment and care for people with haemophilia involves a team of specialist health professionals to cover the range of their care needs. This is known as ‘comprehensive care’.
Comprehensive care is coordinated by a Haemophilia Treatment Centre.
Haemophilia Treatment Centres
There is at least one specialist Haemophilia Treatment Centre (HTC) in every state and territory in Australia, located in a major public hospital.
HTCs have a team of health professionals available, including doctors, nurses, physiotherapists, and social workers, counsellors or psychologists with expertise in the treatment and care of people with bleeding disorders. They also have access to specialist laboratory and diagnostic testing and can give referrals to genetic counselling and testing services.
HTCs have a comprehensive care approach and the team can work with other services that might be needed, such as paediatricians (children’s health doctors), gynaecologists (women’s health specialists), obstetricians (for pregnancy and childbirth), GPs (general practitioners) and other relevant health care services.
Each person affected by haemophilia will have their own treatment plan, which is developed with them individually and reviewed regularly/as needed with their haematologist (blood diseases specialist doctor) or haemophilia nurse practitioner. It may include a plan for regular treatment and for injuries or surgery.
There are a variety of treatment products used to treat haemophilia. It is important that people with haemophilia discuss these treatment options with their Haemophilia Treatment Centre.
New and emerging treatments for haemophilia
Researchers are always looking for new ways to treat haemophilia.
New treatments are now in development and becoming available.
Read the latest on new haemophilia therapies in National Haemophilia
Replacement factor therapy
Clotting factor concentrates replace the missing clotting factor in the blood and are called replacement factor therapy.
The treatment is infused (injected) into a vein at home by people trained to treat themselves. When it is difficult to use a vein, the treatment is sometimes infused through a port, a small device implanted under the skin to connect to a big central vein closer to the heart. Parents or carers can be trained for both methods of treatment. The treatment can also be infused at the Haemophilia Treatment Centre, in the emergency department or in the community by an educated general practitioner (GP) or community nurse.
Replacement factor therapy can be given:
- As prophylaxis – given regularly to prevent bleeds or reduce bleeding from an injury. How often it is given is tailored to the individual and can be given at different intervals, ranging from daily to fortnightly.
- On demand – before surgery, childbirth or dental treatment, or after an injury or accident, or once a bleed has started.
Recombinant factor is the most widely used type of concentrate. This is made by genetic engineering and contains little or no material from human blood or animals. There are several brands available manufactured by different pharmaceutical companies.
Standard vs extended half-life
When clotting factor concentrates are used for replacement factor therapy, they do not stay in the body but are gradually used up. The amount of time it takes for the body to use up half of the clotting factor is called the half-life. The half-life will vary from person to person.
The Haemophilia Treatment Centre will discuss this with the person or parents of a child and may test the person’s blood after an infusion to see how long their personal response to the factor is. Understanding the half-life is important for planning when to have infusions, for example, before high risk activities such as competition sport.
Some people will be prescribed recombinant factor which is an extended half-life concentrate (EHL). The effect of an EHL lasts for longer in the body than a standard half-life concentrate and as a result they do not need to be infused as often.
Plasma factor concentrates
Plasma factor concentrates are also used by some people. These are made from the plasma (pale yellow fluid part) in human blood.
Non-factor therapies work differently to replacement factor therapy. These treatments do not replace factor VIII or factor IX but work to support the body’s capacity to clot in other ways.
These treatments are used ONLY as prophylaxis to prevent bleeding episodes and not to treat acute bleeding episodes, for example, after an injury. They are injected under the skin (sub-cutaneousl injection) at regular intervals. How often these treatments are given will vary depending on the type of treatment. The Haemophilia Treatment Centre will train parents or the person with haemophilia so that they can inject their treatment independently.
Types of non-factor therapies
FVIII mimetic: even though it is not the same as factor VIII, this is a molecule that copies the function of factor VIII to hep the body form a clot. This can be used to treat people with haemophilia A both with and without inhibitors.
Other non-factor therapies: other treatments also work differently from factor replacement therapy and use a variety of different approaches to help the body form clots.
Non-factor therapies are still a relatively new treatment option for people with haemophilia and some treatments are still in the clinical trial stage. There is ongoing research and development in this area, which may lead to other new treatments for haemophilia A and haemophlia B in the future. It is very important that people with haemophilia discuss these treatment options with their Haemophilia Treatment Centre to determine which one would best suit them.
Gene therapy for haemophilia aims to provide the body with a functioning version of the factor VIII or factor IX gene so that the body can produce enough clotting factor for the blood to clot normally.
Gene therapy is still a relatively new treatment for haemophilia and there is a lot of research in this area and new information becoming available. Initial results are promising, with a decrease in the number of bleeding episodes and the need for people to have prophylaxis treatment.
There are a lot of important considerations before undertaking gene therapy, for example, will you be suitable to have gene therapy, how long will the effect of treatment last, side effects, etc. All of these need to be discussed with your Haemophilia Treatment Centre.
Gene therapy has been registered for use in some countries.
In Australia there are currently a number of advanced experimental clinical trials for gene therapy. Some Haemophilia Treatment Centres are participating in the trials. People who are interested can discuss this with their Haemophilia Treatment Centre as this is constantly changing.
This is a synthetic hormone that releases the body’s stored factor VIII into the bloodstream to help blood clot. It is used for treating some people with mild haemophilia A and some women with bleeding disorder symptoms. DDAVP can be given as a slow infusion into a vein, but may also be given as an injection subcutaneously (into the fatty tissue under the skin), or in special circumstances as a nasal spray. Individuals can discuss these alternatives with their Haemophilia Treatment Centre to see if it is an option for them.
This slows blood clots from breaking down after they have been formed. It can help to treat mouth or nosebleeds, gut bleeding or bleeding after dental work. Most commonly it is taken as tablets, syrup or in a mouthwash.
Hormone treatment can help women who have heavy menstrual bleeding. The hormones can increase factor VIII levels. This can include oral contraceptives (birth control pills) or the Mirena ® or Kyleena® IUD/intrauterine device.
After treatment with a clotting factor product, some people with haemophilia may develop antibodies – known as ‘inhibitors’ – which may mean treatment with factor replacement therapy no longer works for them or is less effective.
There are a number of ways to treat inhibitors, including bypassing agents and non-factor therapies. Management will vary depending on the person’s individual circumstances and the Haemophilia Treatment Centre will discuss the treatment options with them.