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There are various treatment options for the genetic disease
Factor replacement therapies
Standard Half-life therapy, extended half-life therapy, and bypass agents

Factor replacement treatments are when the individual is injected through a vein treatment that contains molecules that are similar to the human body's production of blood clotting.

Non-factors therapy is when the individual is injected with a medication that helps prevent bleeding and assist with clotting.

Different treatments are used to treat Hemophilia A, B, and C
Replacement therapy is used for A and B

Gene therapy is also an option for patients. Its main purpose is to provide people with the correct gene to combat the disease. However, because every individual's body is different it produces a different outcome.

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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.

Read MASAC's 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) 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.

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.

Watch our video on how clotting factor works.

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.
See all FDA-approved treatments for hemophilia A.
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.
Explore a complete list of FDA-approved therapies to treat VWD.
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.

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Current Treatments | National Hemophilia Foundation

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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.

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Caused by abnormal F11 gene creating low factor XI levels or none at all. Compared to A and B, Hemophilia C has less severe symptoms. People with Hemophilia C may experience prolonged bleeding after surgery, but not spontaneous bleeding, as well as no bleeding in joints.

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The life expectancy is about 10 years less than the average human life span. Chilren who are diagnosed and receive treatment early in life  have a normal life expectancy.

The individual will require medical attention for the rest of their life and should avoid activities that can cause internal bleeding or scratches that will require blood to clot. Also the normal over the counter drugs such as Ibuprofen, naproxen and aspirin prevent blood clotting.

Being that maintaing a healthy life style to sustain maximum overall health, the individual should consult with their doctor when it comes to physical training. Managing stress is also recommended and good dental hygiene.

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Hemophilia is a bleeding disorder that slows blood clotting, leading to:

- excessive bleeding from cuts and injuries, or surgery and dental work
- blood in urine or stool
- sudden nosebleeds
- pain, swelling, or tightness in joints

For severe hemophilia, bleeding in the brain can occur if bumped on the head, which can cause:

- seizures
- paralysis

Serious cases also include bleeding in internal organs, joints, or muscles.

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Besides genes mutating, individuals usually inherit the disorder since the genes responsible for hemophilia are located on the X chromosome. Depending on which parent has the defective gene on the X chromosome, it can be passed down to either only their daughters or to sons and/or daughters

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The National Hemophilia Foundation

It offers resource, education information on all blood related disorders.

Training is offered for individuals who want to be leaders in their community to be able to build a strong community for those who are also affected by such disorder.

Providing outreach for individuals who either are undiagnosed or are unable to find help.

Financial assistance is also key for anyone who is suffereing from such diseases and is offered through this support group.

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There is no cure for Hemophilia due to the protein needed for clotting. The most common Hemophilia is type A is missing clotting factor 8. The solution is to engineer mesenchymal stem cells, which is a adult stem cell that can produce high levels of factor, allowing the cells to act as a carrier for the gene.

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- Hemophilia A: Affects 12 in 100,000 people in the US
- Hemophilia B: Affects 4 in 100,000 men in the US, hard to estimate # of women affected since it's hard to differentiate between hemophilia and heavy period or postpartum bleeding
- Hemophilia C: Affects 1 in 100,000 men or women

- Prevalence highest in Midwestern & Northeastern states
- 4 in 10 males have severe form of the disorder
- Average age of people affected is 23.5 years
- Affects white and Hispanic people more than black or Asian

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- “Current Treatments.” National Hemophilia Foundation, https://www.hemophilia.org/bleeding-disorders-a-z/treatment/current-treatments.
- “Researching a Treatment for Hemophilia.” Wake Forest University School of Medicine, https://school.wakehealth.edu/research/institutes-and-centers/wake-forest-institute-for-regenerative-medicine/research/cell-and-gene-therapy-research/researching-a-treatment-for-hemophilia#:~:text=People%20with%20hemophilia%20A%2C%20the%20most%20common%20type%2C,that%20they%20produce%20high%20levels%20of%20factor%20VIII.
- “Researching a Treatment for Hemophilia.” Wake Forest University School of Medicine, https://school.wakehealth.edu/research/institutes-and-centers/wake-forest-institute-for-regenerative-medicine/research/cell-and-gene-therapy-research/researching-a-treatment-for-hemophilia#:~:text=People%20with%20hemophilia%20A%2C%20the%20most%20common%20type%2C,that%20they%20produce%20high%20levels%20of%20factor%20VIII.
- “What Is Hemophilia?” Cleveland Clinic, https://my.clevelandclinic.org/health/diseases/14083-hemophilia#:~:text=What%20is%20the%20outlook%20for%20people%20with%20hemophilia%3F,especially%20for%20those%20who%20are%20considered%20high%20responders.
- "Hemophilia." Mayo Clinic, https://www.mayoclinic.org/diseases-conditions/hemophilia/symptoms-causes/syc-20373327
- "Diagnosing Hemophilia in Children." NYU Lagone Health, https://nyulangone.org/conditions/hemophilia-in-children/diagnosis#:~:text=Prenatal%20Genetic%20Tests,close%20relative%20with%20the%20condition.
- "Hemophilia." Medline Plus, https://medlineplus.gov/genetics/condition/hemophilia/
- "Coagulation Tests." healthline, https://www.healthline.com/health/coagulation-tests
- "What is Hemophilia?" Centers for Disease Control and Prevention, https://www.cdc.gov/ncbddd/hemophilia/facts.html

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Before birth, babies can be tested using:

- Amniocentesis: removing cells from (amniotic) fluid that surrounds the baby in the womb

-Chorionic Villus Sampling (CVS): taking tissue samples from the placenta

Clotting factor tests can also be done, and can determine the severity of the disorder. These tests measure your blood's ability to clot and how long it takes

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Caused by abnormal F9 gene, creating low factor IX levels or none at all. Depending on factor IX levels, symptoms range from mild (6 - 49%) to severe (<1%). Hemophilia B is still serious but may be less serious than Hemophilia A. Compared to Hemophilia A, people with Hemophilia B have less bleeding in joints, fewer spontaneous bleeding, and are less likely to develop issues with antibodies.

- formerly known as Christmas disease

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Caused by abnormal F8 gene creating either low factor VIII levels or none at all. Depending on how much factor VIII a person has, symptoms range from mild (5% - 40%) to severe (<1%).

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Photo of normal blood clotting process; Hemophilia would result in blood clot incomplete or delayed

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Mutations in gene located on X chromosome that provides instructions for making clotting/coagulation factor proteins responsible for forming blood clots.

We have 13 different clotting factors/proteins in our bodies responsible for forming blood clots by working together. This means that multiple types of hemophilia can occur: