Summary
- Clotting factor replacement therapy temporarily increases clotting factor levels, using the specific clotting factor that people with haemophilia are missing.
- The most common type is recombinant factor concentrate, made in a laboratory.
- Treatment is given as an infusion into a vein, which takes a few minutes and can be done at home.
- Treatment can be given on demand when needed – for example, after an injury – or as a preventative treatment (prophylaxis).
Standard half-life factor therapy
Extended half-life factor therapy
What is clotting factor replacement therapy?
Clotting factor replacement therapy is used to treat some people with haemophilia.
Haemophilia occurs when a person doesn’t have enough of a specific clotting factor in their blood to control bleeding properly. A clotting factor is a protein in blood that controls bleeding. When a person has an injury that causes bleeding, more than 20 proteins are involved in the chain reaction to make a clot that stops the bleeding, including multiple clotting factors.
- People with haemophilia A don’t have enough clotting factor VIII (8).
- People with haemophilia B don’t have enough clotting factor IX (9).
Clotting factor replacement therapy uses clotting factor concentrates to increase the level of clotting factor in a person’s blood temporarily, replacing the ‘missing’ factor.
Types of clotting factor concentrates
Most clotting factor replacement therapy uses recombinant factor. This is made in a laboratory and has very little, if any, material from humans or animals.
Plasma-derived clotting factor concentrate may be used in some circumstances. This is made from human blood plasma (the clear yellow fluid in blood).
When clotting factor concentrate is given
There are two main ways the treatment is given:
- prophylaxis – regular treatment to prevent bleeding episodes (‘bleeds’) or to reduce bleeding from injuries. The treatment schedule is personalised and can range from daily to every 2 weeks.
- on demand – treatment before surgery, childbirth or dental procedures, after an injury, or when a bleed starts.
On demand treatment is often used:
- for people with mild or moderate haemophilia who don’t have bleeds as often
- as a bleed treatment for people having non-factor therapies. Non-factor therapies don’t replace factor VIII or factor IX. Instead, they support the body to clot in other ways.
Children and adults of any gender can be given clotting factor concentrates.
How clotting factor concentrate is given
Treatment with clotting factor concentrate is given through a slow injection into a vein. This is called an infusion. If it’s hard to use a vein, the treatment might be infused through a small surgically implanted device (called a port) that connects to a large vein near the heart.
If you have haemophilia, you can be trained to do the infusion yourself, as can parents or carers. The infusion can also be given at a Haemophilia Treatment Centre (HTC), in a hospital emergency department or by a trained GP or community nurse.
If you have regular treatment, you can keep a stock of treatment at home to infuse according to your treatment plan. Clotting factor concentrates come in an easy-to-use pack of powder and liquid to mix. Packs are usually stored in the fridge but can often be stored at room temperature for 3-6 months, depending on the product.
The half-life of concentrates
Clotting factor concentrates don’t stay in the body for long – they’re gradually used up. The time it takes for the body to use up half of the clotting factor is called the half-life. This timeframe varies from person to person. The factor is generally cleared away more quickly in children than adults.
You will have peaks and troughs – times when there is a high amount of clotting factor in the blood (straight after your dose) and times when it’s lower (when the factor has been used up). The goal of prophylaxis is to maintain levels high enough to prevent bleeds.
Your HTC will work with you to understand your (or your child’s) personal half-life through pharmacokinetic (PK) testing. Treatment centre staff may check your blood after treatment to see how long the factor works for you. This helps decide when to schedule infusions – for example, before high-risk activities like competitive sports.
The three clotting factor replacement therapy types are:
- standard half-life (SHL)
- extended half-life (EHL)
- ultra-long half-life (UHL).
People in Australia usually receive EHL therapies. These last longer in the body than SHL therapies so you don’t need to have them as often, generally once or twice a week. Using EHL therapies can reduce the number of bleeds to 1-2 bleeds per year, although it can vary for individuals.
There have been new developments in UHLs that last longer in the body than EHLs, so that you only need to have them about once a week. UHLs for haemophilia A have been in advanced clinical trials in Australia.
Personalised treatment plans
Treatment for haemophilia is usually prescribed by a haematologist (a doctor who specialises in blood diseases) or a haemophilia nurse practitioner at an HTC.
Haemophilia treatment plans are personalised. These plans are developed specifically for you and your circumstances and can be updated whenever needed. Your treatment plan may include a plan for regular treatment and a plan for injuries or surgery.
Australian governments fund a range of clotting factor and other blood products used to treat haemophilia through the National Blood Authority. This means the approved products are provided at no cost to patients who need them.
Treatment safety
All medical treatments come with some risks.
Common side effects
The most common side effects for clotting factor replacement therapy are bruising and redness at the spot where you had the injection and allergic reactions.
Inhibitors
One of the biggest risks of clotting factor replacement therapy is the possibility of developing inhibitors. This is when the body’s immune system identifies the clotting factor concentrate as foreign and mistakenly produces antibodies (inhibitors) that block the factor concentrate’s ability to work or cause it to work less effectively.
If people who haven’t had treatment before have clotting factor replacement therapy, inhibitors develop in about 20–30% of those with haemophilia A and 3–5% of people with haemophilia B. Inhibitors usually show up within the f irst 75 days of treatment. Switching between concentrates with different half-lives is generally safe and doesn’t cause an increased risk of inhibitors.
There are several ways to treat inhibitors, and management depends on your individual circumstances. If you develop inhibitors, your HTC will discuss treatment options with you.
Risk of infection
- Recombinant factor: recombinant products are not made from human blood so are not affected by blood-borne viruses like HIV and hepatitis B or C.
- Plasma-derived factor: when plasma-derived clotting factor replacement therapy is manufactured, it has multiple steps to inactivate blood-borne viruses like HIV and hepatitis B or C, so the risk of infection is extremely low.
New and emerging treatments
Researchers are always looking for new ways to treat haemophilia and improve current treatments. There is ongoing research into UHLs.
Keep in touch with your HTC to find out what’s new, discuss your treatment options and determine which one would be best for you. You can also stay up to date with new and emerging treatments through Haemophilia Foundation news, websites and education sessions, including the Australian Bleeding Disorders Conference.
Living well with haemophilia treatment
Managing your haemophilia treatment well can help you enjoy a full life. Your HTC is there to give you advice and support. They can refer you to other services if you need them. Although everyone’s experience can be different, you may find it valuable to connect with other people with haemophilia to share common experiences and tips. Your HTC and your local haemophilia foundation can help you with this.
Travel
Many people with haemophilia travel both within Australia and overseas. This can involve a little extra planning and discussion with your HTC. Check the locations and available times of HTCs in the places where you’re travelling before you set off, in case you run into trouble.
You may need to take treatment product with you. This may involve carrying it with you in a cooler bag and storing it in a fridge on your trip. If you’ll be away for a long time or going overseas, start planning with your HTC early.
Your HTC can also help with documents for customs and security. You may also wish to carry your ABDR (Australian Bleeding Disorders Registry) patient card with your diagnosis and treatment plan in your wallet or on your phone, set up your medical emergency info on your phone or to wear a medical alert bracelet or necklace.
Be informed and aware
Understanding your haemophilia and knowing how to manage it makes a big difference.
- Be aware of how your body responds to treatment and plan around it.
- Build a good relationship with your haemophilia team and don’t be afraid to ask questions.
- Keep up with the latest treatments and services.
More information
For more information, speak to your HTC.
Visit the Haemophilia Foundation Australia website for:
- the Gene and Emerging Therapies Hub
- contact details for Haemophilia Treatment Centres (HTCs), which are located in every state and territory.
References
Srivastava A, Santagostino E, Dougall A, et al. WFH guidelines for the management of hemophilia, 3rd edition. Haemophilia. 2020: 26(Suppl 6): 1-158. Accessed 1 August 2022. Available from https://doi.org/10.1111/hae.14046.
Reviewers:
HFA Gene and Emerging Therapies consumer review Group (individuals not named for privacy reasons).
Australian Haemophilia Centre Directors’ Organisation: Dr Sally Campbell, Dr Stephanie P’ng. ABDR Data Managers: Marina Goruppi, Venessa Mavrinac-Tiddy.
ANZ Haemophilia Psychosocial Group: Tanya Grueneweld, Jane Portnoy, Emma Wells.
Australian Haemophilia Nurses’ Group: Megan Walsh.
The video resources on this page are published by World Federation of Hemophilia (WFH) and are linked here with permission.
© 2023 World Federation of Hemophilia https://sdm.wfh.org/
This fact sheet has been funded by unrestricted education grants from CSL Behring and Sanofi. HFA does not promote any particular pharmaceutical product.