How Is Hemophilia Treated?

Hemophilia is a bleeding disorder that prevents the blood from clotting properly. This can cause excessive bleeding, especially in the muscles, joints, and tissues. There is currently no cure for hemophilia, but several treatment options can help manage the symptoms.¹

Treatment options may vary depending on the severity of hemophilia, your age, and other factors.¹

Treatment goals

Hemostasis is the body’s process of stopping excessive bleeding. Coagulation is the process of blood clotting. A balance of both bleeding and clotting is what is called hemostatic balance.²

For those without hemophilia, bleeding and clotting are balanced. For those with hemophilia, the scale tips toward bleeding. The goal of hemophilia treatment is to bring clotting back into the balance.²

Clotting factor products

Clotting factor products are used to treat disorders that impair the body's ability to form blood clots. There are 2 types of clotting factor products used in hemophilia:³

• Plasma-derived factor concentrates
• Recombinant factor concentrates

These are also known as factor replacement therapies.³

Plasma-derived factor concentrates

Medicine made from donated human blood plasma is known as plasma-derived factor concentrates. These products contain natural clotting factors. There are different types of plasma-derived clotting factors, including:^3,4

• Factor VIII (8) – Treats hemophilia A
• Factor IX (9) – Treats hemophilia B
• Von Willebrand factor (VWF) – This protein helps the platelets in the blood stick together. Low levels of VWF can lead to problems with blood clotting and cause von Willebrand disease (VWD). VWD is similar to hemophilia but is more common and is often less severe.
• Activated prothrombin complex concentrate (FEIBA)

Recombinant factor concentrates

Like plasma-derived factor concentrates, recombinant factor concentrates replace the missing or reduced number of clotting factors in the blood. Unlike plasma-derived factors, these products are lab-made.³

Scientists figured out how to make clotting factor proteins from animal cells. The human gene that makes the desired factor is put into animal cells grown in a laboratory. These cells then make lots of the clotting factor. Recombinant factor concentrates do not contain viruses or unwanted proteins because the product does not come from human blood.³

Non-factor replacement therapies

Factor replacement products raise factor levels in the blood. Non-factor replacement products increase the likelihood that the blood will clot if a blood vessel is injured.²

Harmful antibodies can be made inside the body of a person with hemophilia who is taking factor replacements. These antibodies are known as inhibitors. Inhibitors attack the factor replacement, making it very hard to treat bleeding. In hemophilia A, this immune response causes factor 8 inhibitors.²

Because non-factor replacement therapies do not use factors, people with inhibitors can use this type of therapy to manage bleeds.²

Hemlibra^® (emicizumab)

Hemlibra^® (emicizumab) is the first-in-class treatment that acts as a bridge so other factors in the blood can work and treat bleeding in people with hemophilia A. This drug binds to both factor IX (9) and factor X (10), creating a natural function of the missing or defective factor 8 in hemophilia A.^2,5

Hemlibra is given as a weekly, every 2 weeks, or monthly injection instead of daily or multiple times a week like factor replacements. However, non-factor replacements cannot be given for active bleeds. This type of therapy is for prevention (prophylaxis).^2,5

DDAVP^® (desmopressin acetate)

DDAVP^® (desmopressin acetate) is a lab-made hormone that promotes blood clotting through increasing levels of VWF and factor 8. DDAVP is usually given to people with VWD during procedures or surgeries to reduce the amount of bleeding and bruising. It may also be given to those with mild hemophilia A.^3,6

DDAVP is not given to those with hemophilia B because factor 9 is the problem for people with this condition. It is also not given to children under age 2 and is used with caution in pregnant women. Drinking too much water while taking DDAVP can lead to serious electrolyte imbalance and even seizures.^3,6

Stimate^® (desmopressin) is a nasal spray used in hemophilia A. In 2020, the makers of Stimate recalled this drug because of problems with drug concentrations. This product was an important first-line therapy for nearly 30 years, allowing people with hemophilia an alternative to intravenous (IV) treatment. Hemophilia organizations are working to find safe alternatives to this drug.^3,7

Drugs that prevent blood clots from breaking down

Other treatments for hemophilia include drugs that prevent the body from breaking down blood clots. These drugs are known as antifibrinolytics or fibrinolytic inhibitors. Drugs in this class used to treat hemophilia include:^3,8-10

• Amicar^® (epsilon aminocaproic acid)
• Cyklokapron^®, Lysteda^® (tranexamic acid)

Other plasma products

Cryoprecipitate is a blood product made from donor blood. Cryoprecipitate can only be made from fresh frozen plasma (FFP). FFP is plasma separated from whole blood and frozen within 8 hours of donation.¹¹

Cryoprecipitate has mostly factor I (fibrinogen) and factor 8, but it also has some other clotting factors in it. However, cryoprecipitate does not undergo viral inactivation like other clotting products. Because of this, cryoprecipitate should only be used in life- and limb-threatening emergencies when factor 8 replacement is not available.^6,11

Other treatments

Most people with hemophilia do not have a vitamin K deficiency. However, vitamin K is required for making multiple clotting factors including II (2), VII (7), IX (9), and X (10). Eating foods high in vitamin K, like green, leafy veggies, may help clotting. However, talk to your doctor or nutritionist before making dietary changes.^12,13

For those with inhibitors, steroids or immunosuppressants might be needed. These drugs reduce inflammation and might help prevent additional damage from harmful antibodies.¹⁴

Women with hemophilia may need hormone therapies to help during menstruation. For example, birth control drugs might help decrease excessive bleeding during menstruation.¹⁵

Gene therapy

Gene therapy is a new way to fix certain health problems in our bodies. Scientists use tiny parts of our genes to help our bodies work better. They add these genes into our cells to replace the ones that are not working properly. It's like giving our bodies a little instruction manual to fix the issue.¹⁶

Gene therapy most often uses viral vectors. A viral vector is a tiny, harmless part of a virus that is used to deliver important instructions to our cells. It acts like a special delivery vehicle, carrying specific genes that can help fix certain health problems. The viral vector is safe because it does not cause any illness itself. It only helps deliver the helpful genes to where they need to go in our bodies.¹⁶

In June 2023, the US Food and Drug Administration (FDA) approved a gene therapy called Roctavian™ (valoctocogene roxaparvovec-rvox). This gene therapy is approved for adults with severe hemophilia A with specific types of antibodies in their blood.¹⁷

Hemgenix^® (etranacogene dezaparvovec), was approved in November 2022 to help adults with Hemophilia B who:¹⁸

• Need factor IX (9) therapy often
• Have had life-threatening bleeding, or
• Have serious, frequent, unexpected bleeding episodes