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Current Treatments

Treatment Options for Bleeding Disorders

There are many different types of therapies for bleeding disorders, and new ones are in development. Each person may respond to a treatment in their own way, so it is important to work closely with your hematologist to find a treatment that works for you.

Factor replacement therapies: Often referred to as “factor,” these products use a molecule that is either similar to natural factor found in humans (recombinant) or use an actual human molecule (plasma derived.) These treatments increase the amount of factor in the body to levels that lead to better clotting, and therefore less bleeding. The therapy is taken intravenously via an injection into a vein. This process is also called "infusion." There are two types of factor replacement therapies: standard half-life (SHL) and extended half-life (EHL)

  • Standard half-life therapies: Standard half-life therapies are used to treat hemophilia A and B, some types of von Willebrand disease, and some rare factor disorders. Dosing can be anywhere from three times a week to every day, depending on the person.
  • Extended half-life (EHL) therapies: EHL contains a molecule that has been modified in some way to delay the breaking down of factor in the body. This results in higher levels of factor in the body lasting for longer, resulting in less frequent infusions. How long the factor is effective in the body depends on the person. Extended half-life therapies are mostly used to treat hemophilia A and B.
  • Bypassing agents are used to treat bleeds in people with hemophilia with inhibitors. These treatments contain other factors that can stimulate the formation of a clot and stop bleeding.

For more information on replacement clotting factor treatments, see information below.

Non-factor replacement therapies: These products help prevent bleeding or assist in better clotting using other methods in the body besides factor replacement therapy. Non-factor replacement therapies include:

  • Emicizumab (Hemlibra) is a therapy used to treat hemophilia A, to prevent bleeding episodes in people both with and without inhibitors. It is known as a factor VIII(8) mimetic because it mimics, or imitates, the way factor VIII(8) works. It brings together factor IX(9) and factor X (10), which allows the blood to clot. Unlike factor replacement therapy, in which the missing factor is injected directly into a person’s vein (called an infusion), emicizumab is given by an injection under the skin, called a subcutaneous injection. Emicizumab was approved by the FDA to treat people with hemophilia A with inhibitors in 2017 and for people with hemophilia A without inhibitors in 2018.

Read the MASAC recommendation on Emicizumab

  • Desmopressin (DDAVP) is the synthetic version of vasopressin, a natural antidiuretic hormone that helps stop bleeding. In patients with mild hemophilia, it can be used for joint and muscle bleeds, for nose and mouth bleeds, and before and after surgery. It comes in an injectable form and a nasal spray. The manufacturer of DDAVP nasal spray issued a recall of all US products and does not expect to begin resupplying until 2022. DDAVP is used to treat von Willebrand disease and mild hemophilia A.
  • Aminocaproic acid (Amicar) and tranexamic acid prevents the breakdown of blood clots. It is often recommended before dental procedures, and to treat nose and mouth bleeds. It is taken orally, as a tablet or liquid. MASAC recommends that a dose of clotting factor be taken first to form a clot, then aminocaproic acid, to preserve the clot and keep it from being broken down prematurely. This can be used to manage bleeding in people with hemophilia A, B and VWD.

See all FDA approved products for the treatment of bleeding disorders


Factor Replacement Therapy

Replacement clotting factor treatments are infused. This means they are injected from a needle placed into the vein (called intravenously). This process takes time; it isn’t like getting a quick shot. Patients who are first diagnosed with hemophilia and need immediate treatment will initially be treated at the HTC, the doctor’s office, or the emergency department. Later, patients (or caregivers) will learn to infuse the factor at home (called home therapy).

Various devices (called ports, catheters, or PICC lines) may be used to make it easier to administer clotting factor products into the bloodstream. Some of these may be surgically inserted under the skin in the chest area. Your doctor and you will decide which type of device is right for you, or your child.

Clotting factor (called factor) is a dried powder form of the missing clotting factor; it is mixed with water to become a liquid again before it is administered. Some clotting factor products, called plasma-derived factor, are made from donated human blood plasma. Others, called recombinant clotting factor, are genetically engineered in a laboratory and do not use human blood proteins.

The National Hemophilia Foundation's (NHF) Medical and Scientific Advisory Council (MASAC) encourages the use of recombinant factor products for young children. Because recombinant products do not contain human blood, they cannot transmit harmful viruses such as hepatitis. That said, the clotting factors that contain human blood are safer today because of better screening of blood donors and better methods to destroy any blood-borne viruses that could be present in the donated blood. Discuss with your doctor or the HTC staff the benefits and challenges of each treatment plan so that you can determine which type of clotting factor product is right for your specific needs. The dosage may vary depending on the severity of the bleeding disorder, weight, and type of bleed.


Clotting Factor Amounts

The amount of clotting factor (called factor) needed to treat a particular type of bleeding episode will vary:

  • According to the location of the bleeding site
  • If it is a site of repeated bleeding (for example, if it is at a target joint)
  • If the bleeding could cause damage to the brain or other vital organs

Sometimes another dose is needed several hours after the first dose is given to stop the bleeding. The amount of factor depends on several other things as well, including:

  • Weight
  • The number of units in the bottle (called a vial), which can vary depending on the type of product. As weight increases, dosages will tend to get larger. Your treatment team will help you learn how much factor you need and when to treat a potential bleeding episode


Clotting Factor Half-Life

When clotting factor (called factor) is given, the level of clotting factor in the blood rises; but this rise lasts only for a certain number of hours (called half-life). Half-life is the amount of time it takes the body to “use up” half of the clotting factor level that is circulating in the body. If some time has passed since giving your child treatment and he or she still has pain, stiffness, or other signs of bleeding, another factor treatment may be needed. Sometimes another treatment is recommended to be sure that a stable clot has formed and the bleeding does not start again once the level of clotting factor drops after treatment.

Your HTC can provide exact instructions on when to repeat treatment.

Having factor available is important because you never know when a bleed might occur and prompt treatment is critical; you should always have clotting factor (called factor) with you. This includes when you’re home, in the car, on vacation, and even when you go to the emergency room.

The Centers for Disease Control and Prevention’s (CDC) rule of thumb is to always have at home at least enough clotting factor to give 2 high-dose treatments—that's enough to treat a major traumatic event, such as a head bleed.

Talk to your HTC about the factor and supplies you need, their storage and disposal, and how to order supplies.


Making Your Clotting Factor Work for You: Understanding Half-Life in Your Life