Haemophilia treatment

There are a variety of treatment products used to treat haemophilia.

Clotting factor concentrates

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.

Plasma-derived factor concentrates

are also used by some people.

These are made from the plasma (pale yellow fluid part) in human blood.

Clotting factor concentrate can be given:

  • “On demand” – before surgery, childbirth or dental treatment, or after an injury or accident, or once a bleed has started.
  • As “prophylaxis” – taken regularly, two to three times a week, to prevent bleeds or reduce bleeding from injury.

Factor concentrates are infused (injected) into a vein at home by parents trained to treat their child, or by people themselves when they are confident enough to do it, or at the Haemophilia Centre.

Desmopressin (DDAVP)

This is a synthetic hormone which 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 injection 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.

Tranexamic acid

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

such as oral contraceptives (birth control pills), can help women who have heavy menstrual bleeding. The hormones can increase factor VIII levels.

Inhibitors

After treatment with a clotting factor product, some people with haemophilia may develop antibodies – known as ‘inhibitors’ - which make treatment less effective. There are a number of ways to treat inhibitors and many people are successful in overcoming them while others have ongoing problems.

How often do people need treatment?

Current treatment for people with moderate or severe haemophilia aims to prevent bleeding, pain and joint damage. In Australia the usual treatment for most children and young people with severe haemophilia is prophylaxis, where factor concentrate is injected twice to three times a week to keep factor levels high enough to prevent ‘spontaneous’ bleeds. Many children and young people have benefited from prophylaxis, which reduces the risk of developing the arthritis and joint problems often experienced by those older people who grew up without adequate treatment due to limited product. Treatment also involves exercise guided by a haemophilia physiotherapist. This helps to prevent bleeds and protect joints as well as maintaining a healthy body and mind.

Even when treating with factor replacement regularly, bleeding into joints and muscles may still occur. To recover fully from a bleed, people with haemophilia are advised to apply the RICE principles (Rest, Ice, Compression, Elevation) when they have a bleed and to work with their Haemophilia Centre team, including their haemophilia physiotherapist, to rehabilitate the joint or muscle back to full function.

Most people with haemophilia will need treatment for injuries apart from minor cuts and scratches or in preparation for surgical and some dental procedures.People with mild haemophilia will not need prophylaxis and will only need treatment for a bleed when it occurs, usually after trauma or injury, or when they are preparing for surgery or some dental work.

The local Haemophilia Centre team will help with learning how to recognize a bleed and deal with it promptly, and how to prevent and rehabilitate injuries and bleeds.

Treatment product safety

Haemophilia treatment product safety is a high priority for the Australian Red Cross Blood Service, government agencies such as the National Blood Authority and the Australian government regulator, Therapeutic Goods Administration, manufacturers, and the bleeding disorders community. Manufacture of blood products, such as plasma factor concentrates, is carefully regulated and monitored to make sure that blood products are now as safe as possible from infections that can be transmitted by blood, uch as Human Immunodeficiency Virus (HIV), hepatitis B and C and variant Creutzfeldt-Jakob Disease (vCJD):

  • In Australia blood donors are screened and blood donations are tested for HIV, hepatitis B and C, human T-cell lymphotropic virus (HTLV) and syphilis
  • When they are manufactured, factor concentrates made from human plasma are treated with several processes to remove or inactivate HIV and viral hepatitis and, as far as possible, exclude other known infectious agents that are passed on by blood
  • Most people are now treated with recombinant clotting factor, which is genetically engineered and contains little or no human or animal material. There have been no reports that viruses have been transmitted by recombinant products.

In Australia during the mid-1980s some people with haemophilia acquired HIV from contaminated clotting factor concentrates made from human plasma. During the early 1990s many people with bleeding disorders also found that they had been exposed to hepatitis C through the clotting factor concentrates they used for their treatment.

The risk of new infections from using human blood products is now thought to be extremely low. However it cannot be entirely excluded, particularly if the risk came from a new or unknown type of blood-borne virus or other micro-organisms causing disease. Because of this, people using these products and patient advocacy organizations such as HFA continue to take a strong and watchful interest in product safety.

What about those already affected by HIV or hepatitis C?

  • Ongoing support is needed to help with the social, emotional and health impacts
  • People with hepatitis C are advised to keep a regular check on their hepatitis C and their liver health
  • People with HIV need care from a doctor and health care team specializing in HIV as well as from the Haemophilia Centre team
  • HFA continues to provide advocacy, education and support.

Tips for people with haemophilia or girls/women who carry the gene:

  • Stay in regular contact with your local Haemophilia Centre team and make sure you keep up with anything new
  • A regular review may be recommended by your Haemophilia Centre, depending on the severity of your bleeding disorder and your individual needs
  • Report all joint or muscle bleeding to your Haemophilia Centre team so that each episode can be properly assessed and rehabilitated
  • Let your dentist or your doctor know you have a bleeding disorder so that any procedures such as surgery or dental procedures that may result in bleeds can be managed in liaison with your Haemophilia Centre team
  • If you use treatment product, know which one you use and your usual dose
  • Always carry your Haemophilia Centre treatment wallet card and show it to doctors, nurses, dentists, ambulance officers and other health professionals who provide your care. Consider wearing a medical alert bracelet.
Last Reviewed: 1 July 2013
Reproduced with kind permission from Haemophilia Foundation Australia.

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References

1. Haemophilia Foundation Australia. Haemophilia. Last updated July 2013. http://www.haemophilia.org.au/documents/item/2040 (accessed Sept 2013).
Haemophilia Foundation Australia (HFA)

Haemophilia Foundation Australia (HFA)

Haemophilia Foundation Australia (HFA) represents the Australian bleeding disorders community. We are committed to improving treatment and care through representation and advocacy, education and the promotion of research.