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.
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.
- 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) 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.
Gene therapy is a way of treating a genetic disease or disorder by providing people with working copies of the gene to correct the disease or disorder. There are different approaches to gene therapy, including gene transfer and gene editing.
Currently, gene therapies for Hemophilia A and Hemophilia B work differently in the body and have different results. It is important that you work with your Hemophilia Treatment Center to learn more about gene therapy, to determine if you are eligible, to make certain you understand the risks and benefits, and to ensure you have the information you need to make the best decision for you.
Hemophilia B gene therapy has been approved by the FDA for the treatment of adults with hemophilia B who currently use factor IX (FIX) prophylaxis therapy, or have current or historical life-threatening hemorrhage, or have repeated, serious spontaneous bleeding episodes.
Treatment Options for Hemophilia
The main medication to treat hemophilia A is concentrated FVIII product, called clotting factor or simply factor. There are two types of clotting factor: plasma-derived and recombinant. Plasma-derived factor is made from human plasma. Recombinant factor products are developed in a lab through the use of DNA technology. While plasma-derived FVIII products are still available, approximately 75% of the hemophilia community takes a recombinant FVIII product.
These factor therapies are injected into a vein (called “infusion”) in the arm or hand, or through a port in the chest. The NHF's Medical and Scientific Advisory Council (MASAC) encourages the use of recombinant clotting factor products over plasma-derived because they are safer from blood-borne viruses and diseases.
To maintain enough clotting factor in the bloodstream to prevent bleeds, patients with severe hemophilia are typically prescribed a regular treatment regimen, called prophylaxis – or prophy for short. This means a person will infuse their medication on a regular schedule – for example every day or every other day, depending on how long the factor lasts in the body. MASAC recommends prophylaxis as optimal therapy for all people with severe hemophilia A.
New treatments that use other ways of preventing bleeds are also available. These treatments are known as non-factor replacement therapies. One available therapy is emicizumab, a laboratory-engineered protein that works by performing a key function in the clotting cascade that is normally carried out by the FVIII protein. It can be prescribed for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adults and children of all ages, newborn and older, with hemophilia A with and without factor VIII inhibitors. Emicizumab is not infused, but injected under the skin (subcutaneously.)
It is important to discuss all treatment options with your doctor or the staff at your HTC.
DDAVP (desmopressin acetate) 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.
Aminocaproic 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.
The Future of Hemophilia A Treatment
There are many new treatments for hemophilia A being developed, from gene therapy to new non-factor replacement therapies. Visit the Future Therapies section for updated information on the pipeline of new therapies, as well as extensive information on the development of gene therapy as a treatment for hemophilia.
Treatment Options for von Willebrand Disease
The most common treatment for VWD is DDAVP (desmopressin acetate), which stimulates the release of VWF from cells. This also increases the level of FVIII (factor 8). DDAVP comes in two forms:
Injectable DDVAP: This is injected into a vein or just under the skin, and is used to treat milder forms of VWD, usually type 1.
Nasal spray: This high-dose nasal spray is used to treat milder forms of VWD, usually type 1.
NOTE: Stimate, the DDAVP nasal spray, was subject to a recall in September 2020. Ferring Pharmecuticals, the company that makes Stimate, does not anticipate resupplying Stimate until 2022. Read more about this recall here.
It is important to know that DDAVP is an antidiuretic, which means it causes the body to retain water. Because of this, it’s important to restrict fluids so you don’t develop a condition known as hyponatremia, reduced sodium in the bloodstream.
More severe forms of VWD are treated with factor replacement therapy, which are either plasma-derived (made from human blood products) or recombinant (synthetic). These clotting factor products are rich in VWF and factor VIII (factor 8). Clotting factor products are injected into a vein in the arm to replace the missing factor in the blood. They may also be used to treat mild VWD in people who do not respond to DDVAP.
Hormone therapy, such as birth control pills, or oral contraceptives, can be taken to reduce heavy menstrual bleeding. The hormones in birth control pills can increase the levels of VWF and factor VIII (factor 8) in the blood.
Aminocaproic acid and tranexamic acid prevent the breakdown of blood clots. These drugs are often recommended before dental procedures, to treat nose and mouth bleeds, and for heavy menstrual bleeding. Antifibrinolytics are taken by mouth, 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 prematurely broken down.