Factor replacement therapy is the primary treatment for people with hemophilia A. But a doctor may also prescribe a combination of other medications and physical therapy.
Hemophilia A is a severe genetic disorder that affects your blood’s ability to clot. It’s due to insufficient levels of a protein called clotting factor VIII in your blood. Hemophilia A is also called classical hemophilia or factor VIII deficiency.
Hemophilia A doesn’t have a cure. This condition requires lifelong treatment to prevent and control severe bleeding — one of its main complications.
This article will take a closer look at different treatment options for hemophilia A and provide useful information to help people with hemophilia A and their caregivers manage the condition.
According to the
Factor replacement therapies are concentrates that contain factor VIII. Factor VIII can come either from a healthy blood donor (plasma-derived) or be made artificially in a lab (recombinant).
Doctors can prescribe factor replacement therapy for both episodic and prophylactic care. Episodic treatment helps stop bleeding. Prophylactic treatment helps prevent future bleeding episodes.
It’s possible to have IV infusions at home. Their frequency depends on the medication type and severity of your condition.
In addition to factor replacement therapies, doctors often use other medications for both episodic and prophylactic care. These are commonly called non-factor replacement therapies.
Bypassing agents
Some people who use factor replacement therapy develop an
People with inhibitors can use
Emicizumab (Hemlibra)
Emicizumab is a lab-made protein (monoclonal antibody) that
This therapy can prevent or reduce the frequency of your bleeding episodes.
Desmopressin (DDAVP or Stimate)
Desmopressin is a synthetic analog of a hormone called vasopressin, also known as antidiuretic hormone (ADH). This hormone controls the level of factor VIII in your blood. It’s a prophylactic treatment for people with mild hemophilia A.
It comes as an injectable as well as a nasal spray.
Aminocaproic acid (Amicar)
Aminocaproic acid prevents the breakdown of blood clots. Some doctors recommend it before dental procedures, such as tooth extraction. This is because it blocks molecules in saliva that break down clots.
It comes as a pill or liquid taken by mouth.
According to the
Hemophilia A is a genetic disorder due to a mutation (error) in the gene that contains information about factor VIII. Gene therapy provides people with a working copy of the affected gene. It has the potential to become the first cure for hemophilia.
Gene therapy for hemophilia A is currently in the latest stages of development. As of April 2023, it’s not available to the general public yet.
But this may change soon. Gene therapy treatment for hemophilia A is currently available in Europe. In November 2022, the
Hemophilia treatment centers
Hemophilia treatment centers (HTCs) are specialized clinics that provide comprehensive care for people with hemophilia A and other bleeding disorders. They provide a range of services for people with hemophilia, for example:
- diagnosis
- treatment
- side effect management
- patient and caregiver education
HTCs can also offer support services, for example, from social workers and genetic counselors. They can help you and your family manage the emotional and practical aspects of living with hemophilia A.
Side effects depend on the type of treatment. They may include:
- headache
- abdominal (stomach) pain
- nausea
- skin rash or itchiness at the injection site
Be sure to ask your doctor about the complete list of side effects associated with your treatment.
The outlook for people with hemophilia A has improved significantly in recent years, thanks to advances in management. With proper care, people with hemophilia A can lead full and active lives.
Still, some people have bleeding into the joints, which may affect their activity levels. A small number of people with hemophilia A may die of severe bleeding.
Hemophilia A is a genetic bleeding disorder. Although there’s no cure for it yet, effective therapies are available.
Factor replacement therapy is the most common treatment option, but other medications and therapies can also help prevent bleeding episodes and manage complications.
It’s important to work closely with a healthcare professional to develop and follow a treatment plan that meets your specific needs.