Hemophilia: Should I have regularly scheduled or on-demand clotting factor replacement?

Get the facts
Compare your options
What matters most to you?
Where are you leaning now?
What else do you need to make your decision?

1. Get the Facts

Your options

Have regularly scheduled blood clotting treatment.
Have clotting factor replacement on demand.

Key points to remember

You will likely have fewer bleeds and a lower risk of problems linked to bleeding if you use regularly scheduled treatment. You may be able to do more activities without fear of bleeding.
You won't need treatment as often if you use on-demand replacement, so it costs less. You can wait to give yourself shots until you think you need them or just before you do an activity that might cause a bleed.
Regularly scheduled therapy costs more than on-demand treatment. Clotting factors that are made in a lab cost the most, but these also offer more protection against infections. Not all health insurers cover the cost of this preventive treatment.
When you have a bleed, on-demand treatment may not always be practical or possible. Serious bleeding, such as after a head injury, can be fatal if it is not treated right away.

FAQs

What is hemophilia?

Hemophilia is a rare genetic bleeding disorder that almost always occurs only in men. People who have hemophilia have clotting factors that don't work correctly. Blood-clotting factors are needed to help stop bleeding after a cut or injury and to prevent bleeding that happens for no clear reason. A person with hemophilia often needs treatment to prevent blood loss and stop internal bleeding.

There are two major types of hemophilia, although the symptoms are the same.

Hemophilia A is caused by a lack of active clotting factor VIII (8). About 1 out of every 5,000 male babies is born with hemophilia A.¹
Hemophilia B (Christmas disease) is caused by a lack of active clotting factor IX (9). It is less common, and occurs in 1 out of every 30,000 male babies.¹

Hemophilia is usually labeled by how severe it is. There are three levels of hemophilia, but they can overlap. The severity of the disease is defined by how much clotting factor is produced and in what situations bleeding most often occurs.

Mild hemophilia: Clotting factor level is 5% of normal or greater. Mild hemophilia might not be recognized unless there is a lot of bleeding after a major injury or surgery.
Moderate hemophilia: Clotting factor level is 1% to 5% of normal. Bleeding usually follows a fall, sprain, or strain.
Severe hemophilia: Clotting factor level is less than 1% of normal. Bleeding often happens one or more times a week for no clear reason.

The percentage of clotting factors stays about the same throughout a person's life. All family members who have hemophilia usually will have similar forms.

In very rare cases, a person develops a form of hemophilia, called acquired hemophilia, that is not inherited. If you have acquired hemophilia, your clotting factors don't work right because your body makes antibodies that attack them.

What is clotting factor replacement for hemophilia?

Clotting factor replacement is a way to replace missing amounts of clotting factor in your blood. When you have hemophilia, you can inject doses of clotting factor concentrates to help your blood to clot. Clotting factors come from donated blood or are produced in a lab as a powder form that you mix with sterile water and inject into your veins.

How much clotting factor you need depends on how bad your hemophilia is, where the bleeding is, and how much bleeding you have already had.

Clotting factor replacement can be done in one of two ways:

On a regularly scheduled basis, to prevent bleeding problems, or
On demand, to prevent or control a bleeding problem that has happened or is about to happen

Clotting factor replacement doesn't cure hemophilia or fix damage that has already happened, such as swelling from repeated problems with bleeding in the joints.

How safe is clotting factor replacement?

In the United States, blood products are carefully tested. If there is a chance that donated blood has been infected with a virus, such as HIV, hepatitis B, or hepatitis C, it is not used.

The risk of getting a virus from donated clotting factor is very low. But people who receive many units of donated blood factor concentrate over their lives have a slightly increased risk of getting one of these viruses. Plus, hepatitis Aand parvovirus (the virus that causes fifth disease) are harder to detect than these other viruses. They can sometimes be spread by donated blood. If you use clotting factor that is made in a lab, you have almost no risk of getting an infection.²

What are the main types of clotting factor replacement?

There are three main types of clotting factor replacement:

Fresh frozen plasma is the liquid part of blood (plasma) taken from a donor and frozen for later use.
- Frozen plasma has all types of clotting factors, so it can be used to treat many different bleeding problems. It costs less than other kinds of clotting factor.
- A lot of plasma is needed to provide enough clotting factor to prevent bleeds. It must be frozen. It's usually used in a hospital and is not easily kept at home.
Cryoprecipitate is a blood product that has all clotting factors, so it can also be used to treat many kinds of bleeding problems.
- It is concentrated, so you need less of it than you would of frozen plasma to stop a bleed. But you need more of it than you do of other types of factor concentrates, including the kind made in a lab, to raise clotting factors to a safe level.
- It's not likely to spread a virus, because it comes from only one donor. It costs less than other types of concentrated clotting factor.
- It must be frozen, so it is usually used in a hospital and is not easily kept at home.
Factor concentratesare used to prevent or treat bleeds that happen outside a hospital.
- If you give yourself (infuse) factor concentrate on a regular basis, you can prevent some bleeds from happening. And if you infuse soon after a bleed begins, you can stop the bleed before it gets bad.
- You can carry factor concentrates with you. They are easily stored and can be infused at home.
- Donated factor concentrate comes from blood from many donors and has a high level of clotting factor. Donors are carefully screened. Donated blood is purified to kill most viruses.
- Clotting factors that are made in a lab do not come from donated blood. Clotting factors for hemophilia A carry a slight risk of containing a virus. But the clotting factors produced for hemophilia B do not have this risk. Clotting factors made in a lab cost much more than factors made from donated blood.

What are the benefits of regularly scheduled treatment?

You likely will have fewer bleeds if you get clotting factor on a regular schedule. This is especially important if you have severe hemophilia. You will also have a lower risk of problems linked to bleeding, such as long-term damage from repeated bleeding into your muscles or joints.

You may be able to do more activities without fear of causing a bleed. A study done in Europe found that people who had regularly scheduled treatment had better general health and fewer bleeding problems.³

What are the benefits of on-demand treatment?

On-demand treatment is not needed as often as scheduled treatment, so it costs less. Health insurers are more likely to pay some of the cost.

You won't have to give yourself injections of clotting factor as often as you would with scheduled treatment. You would do it only as needed.

If you think that you are having a bleeding problem, on-demand treatment can quickly control bleeding. You will likely get to know your body and be able to tell when a bleed has started, even before you have many symptoms.

You can give yourself a shot before you begin activities where there is a high risk of bleeding.

What are the risks of scheduled treatment?

Treatment is costly, and health insurers don't always cover the cost of this preventive treatment. If you use clotting factor that is made in a lab, it may cost even more, and supplies may not always be available.

If you use donated clotting factors, you increase your risk of getting an infection.

Usually injections must be scheduled 3 times a week. This can get in the way of your daily life. Some people may have a hard time giving themselves shots that often.

You may increase your risk of developing an inhibitor.

What are the risks of on-demand treatment?

When bleeding is suspected, you may not always be able to act quickly.

If your hemophilia is severe and you often have bleeds, you may not be able to prevent some bleeding problems. You might have long-term damage and start to have other health problems.

If you have a sudden accident, it may be hard to treat a bleed. Serious bleeding, such as after a head injury, can be fatal if it is not treated right away.

Some children with severe hemophilia who have a lot of bleeds miss more school than other children. That may mean that they get lower scores on achievement tests than children who have fewer bleeds.⁴

2. Compare Options

	Have regularly scheduled replacement treatment	Have on-demand treatment
What is usually involved?	You give yourself scheduled clotting factor shots, usually 3 times a week.	You give yourself clotting factor shots as needed, in response to a bleeding event or before an activity that may cause bleeding.
What are the benefits?	You will likely have fewer bleeds. Scheduled treatment prevents joint damage from repeated bleeding. You may be able to do more activities without fear of causing a bleeding injury.	You don't need to give yourself shots as often. You save on the cost of clotting factor, because you do not need it as often. Health insurers are more likely to cover some of the cost of treatment.
What are the risks and side effects?	Treatment is expensive. Health insurers may not cover the cost. Regularly scheduled injections may interrupt your daily life. You have a slight risk of getting an infection if you use donated clotting factor.	You may not be able to prevent some bleeds. When bleeding occurs, quick treatment may not be practical or possible. Severe bleeding, such as a head injury, can be fatal if it's not treated right away.

Personal stories

Are you interested in what others decided to do? Many people have faced this decision. These personal stories may help you decide.

If you need more information, see the topic Hemophilia.

Personal stories about having clotting factor replacement treatment

These stories are based on information gathered from health professionals and consumers. They may be helpful as you make important health decisions.

"I am a pretty active guy. I love to go on bike rides, play with my dog, and hike. I don't want to have to give up the things I enjoy because of bleeding risk. I decided to try regularly scheduled therapy, even though it is sort of a hassle. Being able to maintain my lifestyle is worth the inconvenience of scheduling the infusions and uncertainty about whether supplies are available. "

— Alex, age 19

"I've learned to adapt my lifestyle so bleeding episodes are more predictable. Plus, now that I'm a little older, I've slowed down a little. I miss some of my usual activities, but don't want to have to deal with regularly scheduled therapy. I also don't want to figure out the insurance and all that goes with that. I have enough on my mind than to go through all the complications that go with regularly scheduled therapy. I'm going with on-demand therapy. "

— Gene, age 51

"I tend to worry a lot, and with hemophilia—well, you never know when a bleeding episode is going to happen. I have a pretty moderate routine. I walk a little bit and swim every now and again. But I just never know what will trigger bleeding. I decided to try regularly scheduled therapy so I would be able to relax more. My wife and I talked a lot about it and decided the expense was manageable and that we would lead happier lives knowing that we were doing all we could to control my bleeding. "

— James, age 33

"I hate shots! And getting clotting factor replacement therapy is like one, long, drawn-out shot. Having regularly scheduled therapy three times a week is just too much for me. I would rather be extra careful and take precautions to prevent bleeding episodes as best as I can rather than have the frequent infusions. Plus, it is cheaper. For me, the decision to have available on-demand therapy was easy. "

— Sam, age 37

3. Your Feelings

Your personal feelings are just as important as the medical facts. Think about what matters most to you in this decision, and show how you feel about the following statements.

Reasons to have regularly scheduled treatment

Reasons to have on-demand treatment

I don't mind scheduling my shots around my other activities.

I don't want to have to schedule my shots.

More important

Equally important

More important

I want to be protected from a bleeding problem at all times.

I am comfortable getting treatment only when I need it.

More important

Equally important

More important

Even if my health insurer doesn't cover the cost, regular treatment is worth it to me.

I don't want to pay for regular treatment.

More important

Equally important

More important

I don't mind giving myself shots 3 times a week.

I don't want to give myself any more shots than I have to.

More important

Equally important

More important

My other important reasons:

More important

Equally important

More important

4. Your Decision

Now that you've thought about the facts and your feelings, you may have a general idea of where you stand on this decision. Show which way you are leaning right now.

Scheduled treatment

On-demand treatment

Leaning toward

Undecided

Leaning toward

5. Quiz Yourself

Check the facts.

1. Which treatment may let you do more activities without fear of bleeding?

Scheduled treatment
On-demand treatment
I'm not sure

You're right. With scheduled treatment, you may be able to do more activities without fear of bleeding.

2. Which treatment allows you to decide when you need a shot?

Scheduled treatment
On-demand treatment
I'm not sure

You're right. With on-demand treatment, you can wait to give yourself a shot until you think you it or just before an activity that could cause a bleed.

3. Which treatment may lead to fewer bleeds and a lower risk of problems linked to bleeding?

Scheduled treatment
On-demand treatment
I'm not sure

You're right. You will likely have fewer bleeds and a lower risk of problems linked to bleeding if you use scheduled treatment.

Decide what's next.

1. Do you understand the options available to you?

2. Are you clear about which benefits and side effects matter most to you?

3. Do you have enough support and advice from others to make a choice?

Certainty.

1. How sure do you feel right now about your decision?

Not sure at all

Somewhat sure

Very sure

2. Check what you need to do before you make this decision.

Nothing. I'm ready to take action.
I want to discuss the options with others.
I want to learn more about my options.

3. Use the following space to list questions, concerns, and next steps.

References

Citations

Hillman RS, et al. (2005). Hemophilia and other intrinsic pathway defects. In RS Hillman, et al. eds., Hematology in Clinical Practice, 4th ed., chap. 32, pp. 368–379. New York: McGraw-Hill.
Roberts HR, et al. (2006). Hemophilia A and hemophilia B. In MA Lichtman et al., eds., Williams Hematology, 7th ed., pp. 1867–1886. New York: McGraw-Hill.
Fischer K, et al. (2002). Prophylactic versus on-demand treatment strategies for severe haemophilia: A comparison of costs and long-term outcome. Haemophilia, 8(6): 745–752.
Shapiro AD, et al. (2001). Defining the impact of hemophilia: The Academic Achievement in Children with Hemophilia study. Pediatrics, 108(6): 1–6.