Without treatment, people with hemophilia and other bleeding disorders are at risk for serious complications, including prolonged bleeding after injuries or surgery, life-threatening brain bleeds, and progressive damage to organs and joints. Treatment for bleeding disorders can be extremely expensive and difficult to access. Globally, highly effective treatment for hemophilia A and B is primarily available to just 15 percent of the population — mostly in high-income countries, according to research in The Lancet Haematology.

In the United States, many people with bleeding disorders are treated at hemophilia treatment centers (HTCs). The Centers for Disease Control and Prevention (CDC) funds the network of approximately 141 HTCs across the U.S. and its territories. Each HTC is staffed with hematologists (specialists in blood-related diseases), orthopedists (joint specialists), laboratory technicians, and nurses with expertise in bleeding disorders.

Bleeding disorders occur when the body lacks or produces faulty clotting factors — proteins needed to form blood clots and stop bleeding. Replacing missing clotting factors is a key part of treatment for many people with hemophilia. Other treatments can help prevent bleeds and support the blood coagulation (clotting) process.

In this article, we’ll explore available treatment options for hemophilia.

1. Factor Replacement Therapies

Factor replacement therapies are given through an intravenous (IV) infusion. They work by replacing the missing clotting factor in the blood. There are two types: standard half-life therapies and extended half-life therapies.

• Standard half-life therapies are typically infused every one to three days.
• Extended half-life therapies are designed to stay in the bloodstream longer, so they can be given less often, sometimes just once or twice a week.

How often someone receives factor therapy depends on the severity of their bleeding disorder and their treatment plan. Factor therapy may be used in three main ways:

• Prophylaxis treatment — Regular infusions to help prevent bleeding before it starts
• On-demand treatment — Infusions given as soon as a bleed starts or after an injury
• Perioperative treatment — Infusions given before, during, or after a surgery or dental procedure to help prevent heavy bleeding

Some factor replacement therapies are approved by the U.S. Food and Drug Administration (FDA) for all three uses, while others are only approved for one or two.

Recombinant Products

Recombinant products are lab-made clotting factors. About 75 percent of people with hemophilia use them to help their blood clot.

Brands of recombinant factor VIII (8) concentrates to treat hemophilia A include:

• Advate
• Adynovate
• Afstyla
• Altuviiio
• Eloctate
• Esperoct
• Jivi
• Kovaltry
• Novoeight
• Nuwiq
• Recombinate
• Xyntha

Brands of recombinant factor IX (9) concentrates for hemophilia B include:

• Alprolix
• Benefix
• Idelvion
• Ixinity
• Rebinyn
• Rixubis

Brands of recombinant factor concentrates for rare bleeding disorders include:

• Recombinant factor VIIa (7a) concentrate (NovoSeven RT)
• Recombinant factor XIIIa (13a) (Tretten)

Plasma-Derived Clotting Factors

Factor replacement concentrates are made from donated human plasma. The plasma is checked for diseases and heated to kill any germs. Alphanate, Humate-P, and Wilate are used to treat hemophilia A and von Willebrand disease. Mononine and Profilnine (factor IX), Coagadex (factor X, or 10), and Corifact (factor XIII) are examples of other factor replacement concentrates made from human plasma.

Fresh Frozen Plasma and Cryoprecipitate

There are fewer products available to treat rare bleeding disorders, such as deficiencies in clotting factors I, II, and V (factors 1, 2, and 5), as well as those caused by deficiencies in multiple factors.

Rare bleeding disorders may be treated with fresh frozen plasma or prothrombin complex concentrate, both of which contain a combination of clotting factors. Fresh frozen plasma is often needed in large quantities to be effective. Cryoprecipitate (“cryo”) — a product made from frozen blood plasma — may also be used, but unlike the other two multifactor products mentioned, cryoprecipitate does not undergo treatment to kill viruses.

Among the treatments listed above, recombinant clotting factors are typically the most expensive. However, they are also considered the safest option because they are made without human plasma, which eliminates the risk of bloodborne infections.

2. Nonfactor Replacement Therapies

Nonfactor replacement therapies are hemophilia treatments that support clotting or prevent bleeding in other ways.

Antifibrinolytics

Antifibrinolytics are drugs that prevent bleeding by preventing the breakdown of clots. These medications may be recommended to improve healing or help with excessive menstrual bleeding. Epsilon aminocaproic acid (Amicar) and tranexamic acid (Cyklokapron) are examples of antifibrinolytics. Both epsilon aminocaproic acid and tranexamic acid can be taken by mouth or injection.

Hormones

Desmopressin is a human-made version of the hormone vasopressin. It can be used to treat mild hemophilia A and mild to moderate von Willebrand disease. Desmopressin is believed to work by increasing the levels of von Willebrand factor and factor VIII in the blood. Desmopressin is available as an injection (sold as DDAVP). The nasal spray (sold as Stimate) was recalled and isn’t currently available.

Hormonal contraceptives, such as the birth control pill or intrauterine devices (IUDs), can help control excessive menstrual bleeding in some individuals with bleeding disorders.

Vitamin K

In some people, bleeding disorders are related to vitamin K deficiency. This can lead to a deficiency of multiple clotting factors, such as factors II, VII, IX, and X. Vitamin K may be taken orally or injected.

3. Treatments for Inhibitors

Inhibitors are a serious complication of bleeding disorders. Inhibitors are antibodies (immune proteins) that attack and destroy replacement clotting factors. The body creates inhibitors when the immune system recognizes infused factor treatments as foreign substances and starts to destroy them.

Hemophilia treatments in people who develop inhibitors are far less effective. About 30 percent of people with severe hemophilia A develop inhibitors. It’s less common for people with hemophilia B to develop inhibitors — about 5 percent — but inhibitors can be harder to treat in those with hemophilia B.

Treatment for people with inhibitors can be complicated. In some cases, a higher dose of a clotting factor may be needed to overcome the inhibitor. If this treatment isn’t effective, there are other treatment options.

Bypassing Agents

Bypassing agents help to form a clot without the need for factor VIII or factor IX. Activated prothrombin complex concentrate (Feiba) contains a combination of clotting factors to help treat and prevent bleeds. NovoSeven RT and Sevenfact are recombinant factor VIIa concentrates approved to treat people with hemophilia A and hemophilia B with inhibitors.

Immune Tolerance Induction

Inhibitors themselves can be treated in some people via immune tolerance induction (ITI). ITI involves taking large, regular doses of replacement factor for months or years. The goal of this treatment is to “train” the immune system to stop making inhibitors.

ITI is successful in 70 percent to 80 percent of people with hemophilia A. However, this treatment requires a large time and money commitment.

4. Biologics

Biologics are special treatments made from living sources, like cells or proteins. Emicizumab-kxwh (Hemlibra) is a biologic drug made of a monoclonal antibody (laboratory-made immune protein). It can be used to treat hemophilia A in people with and without inhibitors. Emicizumab-kxwh is believed to work by activating the coagulation process by connecting factors IXa and X. Since emicizumab-kxwh isn’t a clotting factor, it can help prevent bleeding even in people with inhibitors.

This medication is given as a subcutaneous (under the skin) injection.

5. Rebalancing Agents

Rebalancing agents are a type of nonfactor therapy used to treat hemophilia A and B with and without inhibitors. They work by restoring the balance between factors that help the blood clot and factors that prevent the blood from clotting.

Concizumab-mtci (Alhemo) and marstacimab-hncq (Hympavzi) help blood clot better by blocking a protein called tissue factor pathway inhibitor, which normally slows down clotting. Fitusiran (Qfitlia) works by blocking antithrombin, a protein that usually slows clotting. This helps the blood clot better.

These medications are given as a subcutaneous injection at home or in the clinic.

6. Gene Therapy

Gene therapy is a treatment that provides a working copy of the defective gene causing hemophilia. With a working copy of the gene, a person with hemophilia may be able to make the missing clotting factor on their own.

Valoctocogene roxaparvovec-rvox (Roctavian) is FDA-approved to treat severe hemophilia A. Etranacogene dezaparvovec-drlb (Hemgenix) is a gene therapy approved for hemophilia B. An FDA-approved test is required to check if a person is eligible for these treatments. These medications are given as a one-time intravenous infusion.

What Should You Do if Someone With Hemophilia Gets Cut?

A small cut or scrape typically isn’t more difficult to treat in a person with hemophilia compared to a person without a blood disorder. While people with hemophilia may bleed longer, they don’t bleed faster. Most of the time, people with hemophilia don’t need to take factor concentrate for a small cut.

First-aid for a cut should include:

• Washing the cut with soap and water
• Pressing firmly on the cut for several minutes until the bleeding stops
• Placing a bandage on the cut

Call your healthcare provider for cuts that are very deep or if the cut doesn’t stop bleeding.

Talk With Others Who Understand

On MyHemophiliaTeam, the social network for people with hemophilia and their loved ones, members come together to ask questions, give advice, and share their stories with others who understand life with hemophilia.

What treatments have you tried for hemophilia? What has or hasn’t worked for you? Share your experience in the comments below, or start a conversation by posting on your Activities page.