Should I have regularly scheduled or on-demand clotting factor replacement therapy for hemophilia?
Introduction
This information will help you understand your choices, whether you share in the decision-making process or rely on your doctor's recommendation.
Key points in making your decision
Consider the following if you have been diagnosed with a moderate to severe form of hemophilia A or hemophilia B and are trying to decide how best to use clotting replacement therapy.
- You likely will have fewer bleeding episodes and a lower risk of serious complications if you use regularly scheduled preventive (prophylactic) therapy. You may be able to participate more freely in activities that might otherwise cause a bleeding injury.
- You will not need treatment as often if you use on-demand clotting replacement therapy. You will be spared some of the difficulties with injections by waiting until you think you need therapy or until just before you do an activity that might cause a bleeding episode.
- Regularly scheduled therapy will require a steady supply of clotting factor. There are also clotting factors that are made in a lab and offer the greatest protection against viral infection.
- When bleeding is suspected, on-demand therapy may not always be practical or feasible. Serious bleeding, such as after a head injury, poses a great danger of bleeding that may be fatal if it is not treated immediately.
A 2002 European study
that compared outcomes from on-demand and regularly scheduled treatment reported fewer joint bleeding episodes and better general health in people who received regularly scheduled clotting factor replacement.
Medical Information
What is hemophilia?
Hemophilia is a rare genetic bleeding disorder that almost always occurs in males. A person has hemophilia when he or she inherits problems with certain blood clotting factors, making them unable to work properly. Blood-clotting factors are needed to help stop bleeding after a cut or injury and to prevent spontaneous bleeding.
The hemophilia gene can contain many different errors, leading to different degrees of abnormality in the amount of clotting factor produced. A person with hemophilia often needs treatment to prevent severe 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 deficiency of active clotting factor VIII (8). Approximately 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, occurring in 1 out of every 30,000 male babies.1
Hemophilia is usually classified by how severe it is. There are three levels of hemophilia, although 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 VIII or clotting factor IX level is 5% of normal or greater. Mild hemophilia might not be recognized unless there is excessive bleeding after a major injury or surgery.
- Moderate hemophilia: Clotting factor VIII or clotting factor IX level is 1% to 5% of normal. Bleeding usually follows a fall, sprain, or strain.
- Severe hemophilia: Clotting factor VIII or clotting factor IX level is less than 1% of normal. Bleeding often happens one or more times a week for no apparent reason (spontaneously).
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 properly because your body makes antibodies that attack them.
What is clotting factor replacement therapy for hemophilia?
Clotting factor concentrates can help control bleeding in people with hemophilia by helping blood to clot properly. They do not cure hemophilia or repair damage that may have already occurred, such as inflammation from repeated episodes of bleeding in the joints. The severity of hemophilia, the location of the bleeding, and how much bleeding has already occurred often determines how much clotting factor concentrate is given.
In Canada and the United States, blood products are carefully screened. Blood that is suspected of being contaminated with a virus, such as the human immunodeficiency virus (HIV), hepatitis B, or hepatitis C, is not used. The risk of contracting a virus from donated factor concentrate is very low. But people who receive many units of donated factor concentrate over their lives have a slightly increased risk of being infected with one of these viruses. In addition, hepatitis A virus and parvovirus (the virus that causes fifth disease) are harder to detect and can sometimes be transmitted through donated factor concentrates.
Clotting factor concentrates are not always effective, because the body can develop antibodies called inhibitors, which destroy the newly introduced factor VIII or IX.
What are the main types of clotting factor replacement?
Clotting factor replacement therapy is a way to introduce missing or inadequate amounts of clotting factor VIII or IX components into your blood. Replacing factor VIII will help blood to clot in people who have hemophilia A. Replacing factor IX will help blood to clot in people who have hemophilia B. Clotting factor replacement therapy can be used two ways: on a regularly scheduled basis, to prevent bleeding episodes; or on demand, to prevent or control a bleeding episode that has occurred or is likely to occur.
Clotting factor replacement therapy comes in three main forms: fresh frozen plasma, cryoprecipitate, or factor concentrates.
- Fresh frozen plasma is the liquid part of blood (plasma) taken from a donor and frozen for later use.
- Fresh frozen plasma contains all types of clotting factors, so it can be used to treat a variety of bleeding problems.
- A large amount of plasma is required to provide enough clotting factor to prevent bleeding. It must be frozen, so it is usually used in a hospital and is not easily kept at home.
- Cryoprecipitate is a blood product that contains all clotting factors, so it can be used to treat many varieties of bleeding disorders.
- A bleeding episode requires less cryoprecipitate than fresh frozen plasma because cryoprecipitate has been concentrated. Cryoprecipitate is not likely to transmit a disease because it is from only one donor.
- Although cryoprecipitate is concentrated, a relatively large amount is still required (compared to factor concentrates and recombinant products) to increase clotting factors to a safe level. Cryoprecipitate must be frozen, so it is usually used in a hospital and is not easily kept at home.
- Factor concentrates are used to prevent or treat bleeding episodes that occur outside a hospital.
- A factor concentrate that is infused on a regular basis can prevent some bleeding episodes from occurring. If you have a factor concentrate infusion soon after a bleeding episode begins, it can prevent bleeding from becoming severe.
- Clotting factor concentrates are portable, are easily stored, and can be infused at home.
- Donated factor concentrate is derived from screened blood from many donors and has a high concentration of factor VIII or IX. Donors are carefully screened, and donated blood goes through purifying processes that kill most viruses.
- Recombinant clotting factors are produced in a lab (using recombinant DNA technology) and are not derived from donated blood. Clotting factors for hemophilia A contain some human or animal proteins, so there is still some risk that they may contain a virus. But the clotting factors produced for hemophilia B do not contain any human or animal products.
What are the benefits of regularly scheduled therapy?
You likely will have fewer bleeding episodes, which may be especially important if you have severe hemophilia. You also will have a lower risk of serious complications and long-term damage from repeated bleeding episodes into your muscles or joints.
You may have an improved quality of life. You could more freely participate in activities and exercises that might otherwise cause a bleeding injury.
What are the benefits of on-demand therapy?
On-demand therapy is not needed as often as regularly scheduled treatment.
You will not have to give yourself injections as often.
If a bleeding episode is suspected, prompt on-demand infusions can quickly and effectively control bleeding. You will likely get familiar with your body and be able to tell when a bleeding episode has started, even before you have many symptoms.
Infusions can be given before activities where there is a high risk of bleeding.
What are the risks of regularly scheduled therapy?
If you use donated clotting factors, you increase your risk of contracting a viral infection.
Usually the infusions must be scheduled 3 times a week, which can disrupt your daily activities. In addition, some people may have a difficult time with the frequent injections.
If you use recombinant clotting factors that are produced in a lab, supplies may not always be available. Shortages are more likely to occur with recombinant clotting factors than with plasma-derived products.
You may increase your risk of developing an inhibitor.
What are the risks of on-demand therapy?
When bleeding is suspected, a quick response may not always be practical or feasible.
If you have severe hemophilia with frequent bleeding episodes, you may not prevent some episodes. As a result, you may develop complications and long-term damage. Also, some children with severe hemophilia who have frequent bleeding episodes miss more school than average, which may cause them to score lower on achievement tests than children who experience fewer bleeding episodes.2
If you have an unexpected accident, it may be difficult to treat a bleeding episode.
Serious bleeding, such as after a head injury, poses a great danger. Head injuries can cause bleeding that may be fatal if they are not treated immediately.
If you need more information, see the topic Hemophilia.
Your Information
Your choices are:
- Have regularly scheduled blood clotting therapy.
- Have a clotting factor replacement available on demand.
The decision about whether to use regularly scheduled therapy or on-demand therapy takes into account your personal feelings and the medical facts.
Deciding about therapy for hemophilia Reasons to have regularly scheduled therapy Reasons to have on-demand therapy
- You may feel safer knowing that you are consistently helping to prevent unexpected bleeding episodes.
- You may feel more able to participate freely in activities you enjoy.
- You will be helping to prevent serious bleeding that might follow an accident in which you are not able to react quickly. A head injury, for example, can cause bleeding that may be fatal if it is not treated immediately.
- You may be able to prevent complications, such as bleeding into joints, that develop from inadequate clotting factor replacement.
Are there other reasons you might want to have regularly scheduled therapy?
- You may feel secure knowing that you are ready to help prevent a bleeding episode when you participate in a high-risk activity or to help control bleeding when you suspect that it has started.
- With on-demand therapy, you get the replacement only when you feel that you need it.
- You will not have to give yourself injections as often.
Are there other reasons you might want to have on-demand therapy?
These personal stories may help you make your decision.
Wise Health Decision
Use this worksheet to help you make your decision. After completing it, you should have a better idea of how you feel about regularly scheduled and on-demand therapies. Discuss the worksheet with your doctor.
Circle the answer that best applies to you.
I prefer a routine of having clotting factor replacement and knowing that my supply is in good shape in case anything happens. Yes No Unsure I don't handle injections very well and don't want to have infusions any more often than I have to. Yes No Unsure I don't want to risk the possibility of developing complications from repeated bleeding episodes. Yes No Unsure I don't want to be tied down to a rigid schedule of infusions. Yes No Unsure I feel more secure when I am doing physical activities if I am helping to prevent bleeding episodes that might be caused by an injury. Yes No Unsure I don't like to live my life being overly cautious; with on-demand therapy, the clotting factor replacement will be there if and when I need it. Yes No Unsure I want to limit the number of times I am infused because I am worried about the possibility of hepatitis or another virus passing into my blood through clotting factor replacement. Yes No Unsure Use the following space to list any other important concerns you have about this decision.
What is your overall impression?
Your answers in the above worksheet are meant to give you a general idea of where you stand on this decision. You may have one overriding reason to have on-demand therapy or to have regularly scheduled therapy.
Check the box below that represents your overall impression about your decision.
Leaning toward regularly scheduled therapy
Leaning toward on-demand therapy
Return to the topic Hemophilia.
Other Places To Get Help
Organizations
National Hemophilia Foundation (NHF) 116 West 32nd Street, 11th Floor New York, NY 10001 Phone: (212) 328-3700 Fax: (212) 328-3777 E-mail: HANDI@hemophilia.org Web Address: www.hemophilia.org The National Hemophilia Foundation (NHF) is dedicated to the cures of inherited bleeding disorders and the prevention and treatment of their complications through education, advocacy, and research. The NHF has chapters throughout the country and a communications network that brings health professionals and the public the latest news about bleeding disorders. NHF's Web site provides information on the nature, symptoms, and treatments of many disorders.
World Federation of Hemophilia 1425 René Lévesque Boulevard West Suite 1010 Montréal, QC H3G 1T7 Canada Phone: (514) 875-7944 Fax: (514) 875-8916 E-mail: wfh@wfh.org Web Address: www.wfh.org The World Federation of Hemophilia works to introduce, improve, and maintain care for people with hemophilia and related bleeding disorders around the world. The WFH provides various health care development programs and publications. The Web site provides general information on the disease, as well as research updates. Links to other organizations and further research resources are also listed.
| Author: | Robin Parks, MS Ralph Poore | Last Updated: October 12, 2007 |
| Medical Review: | Anne C. Poinier, MD - Internal Medicine Brian Leber, MDCM, FRCPC - Hematology | |
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