​Symptoms

Symptoms of a bleeding disorder include:

• Bleeding into joints, muscles and soft tissues

• Excessive bruising

• Prolonged, heavy menstrual periods (menorrhagia)

• Unexplained nosebleeds

• Extended bleeding after minor cuts, blood draws or vaccinations, minor surgery or dental procedures

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Treatment

Treatment for bleeding disorders varies, depending on the condition and its severity. For some bleeding disorders, there are clotting factor concentrates that can be infused prophylactically or on-demand at home, to prevent or treat bleeds. Some women with bleeding disorders may use hormone therapy (birth control pills) to manage heavy periods. For other bleeding disorders, there are topical products, nasal sprays, and fresh frozen plasma, which is administered in a hospital setting.

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Why Treatment Matters

People with bleeding disorders face serious health risks from everyday bumps and injuries. Without treatment, internal bleeding can cause:

• Chronic pain

• Permanent joint damage

• Disability or even death

Access to treatment allows children and adults to attend school, work, and fully participate in daily life. These therapies are not optional — they are lifesaving.

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Why Access Matters

• Bleeding disorder treatments can cost hundreds of thousands of dollars per year.

• Consistent access reduces emergency room visits, lowers long-term disability, and saves overall healthcare costs.

• Insurance coverage and patient assistance programs are essential to ensure Texans receive the care they need.

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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.) In fact, there are several human plasma-derived factor replacement products and recombinant products available to treat and prevent bleeds in people with hemophilia A, hemophilia B,  von Willebrand disease, and more rare bleeding disorders. 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.

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Non-factor replacement therapies: These products help prevent bleeding or assist in better clotting using other methods in the body besides factor replacement therapy. It is important to note that non-factor therapies do not treat bleeds, and factor replacement products may still be needed in the event of a bleed or injury.

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Rebalancing Agents are treatments that work to prevent too much bleeding by blocking proteins that slow down the clotting process. These treatments do not replace missing clotting factors. They take away, or block proteins that prevent clots. These treatment options can help people with hemophilia A or B. Some of them are currently indicated for persons with inhibitors, while others are prescribed for those without inhibitors. Currently, this type of treatment is for individuals who are age 12 and older.

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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. Current approved gene therapies utilize gene transfer technology.