Hemophilia A treatment
Fact-checked by There is no cure for hemophilia A, but several treatments are available to help prevent and control bleeds, reduce the risk of complications, and improve quality of life.
People with hemophilia A are susceptible to excessive and prolonged bleeding episodes because their bodies don’t make enough functional factor VIII (FVIII), an important blood-clotting protein. The disease is caused by mutations in the F8 gene.
After a diagnosis, individuals will work with their care team to develop a tailored hemophilia A treatment strategy based on their individual needs. When possible, it is a good idea to seek input from a dedicated hemophilia treatment center, where specialists with expertise in hemophilia medical care and bleeding disorder therapies are available.
Prophylaxis vs. on-demand treatment strategies
There are two main treatment approaches for hemophilia A: prophylaxis and on-demand.
Prophylaxis, or preventive therapy, involves routinely administering medications to prevent or reduce the frequency of bleeds. This includes:
- Factor replacement therapies: Medications that contain a version of FVIII that are regularly infused to restore blood clotting
- Nonfactor therapies: Medications that promote blood clotting through alternative mechanisms, which can help people who develop neutralizing antibodies (inhibitors) against FVIII
On-demand treatment involves giving medication only to control an active bleed, instead of regularly on a set schedule. People on prophylactic therapy sometimes also need on-demand treatment if a breakthrough bleed arises. On-demand treatment may include:
- factor replacement therapies
- bypassing agents, which promote blood clotting through mechanisms that bypass the need for FVIII, for people with FVIII inhibitors
Choosing between prophylaxis and on-demand therapy in hemophilia A generally comes down to disease severity. Treatment guidelines recommend prophylaxis for people with more severe disease who are more susceptible to frequent or serious bleeding, while on-demand treatment may be suitable for people with milder disease who experience minor or infrequent bleeding.
Cost, convenience, and other factors may also be considered.
Gene therapies and emerging approaches
In recent years, gene therapy has emerged as a promising treatment option for hemophilia A. These therapies aim to deliver a healthy version of the F8Â gene to patient cells to enable long-term FVIII production. This can provide long-term bleed control with a single infusion into the bloodstream. However, these therapies are not suitable for all people.
Ongoing research is focused on the development of gene therapies and other experimental treatment approaches. Individuals interested in participating in a clinical trial can ask their care team to connect them with studies for which they may be eligible.
Hemophilia treatment centers
Hemophilia treatment centers, or HTCs, are healthcare facilities where people with hemophilia A can receive specialized, multidisciplinary care.
In addition to hematologists — the blood specialists who typically oversee hemophilia treatment — these centers also provide patients with access to other healthcare professionals who may be required for managing hemophilia A, such as orthopedists, physical therapists, social workers, and mental healthcare professionals.
Receiving targeted and appropriate care is key to preventing long-term complications. After diagnosis, patients are advised to ask for a referral to an HTC if they have not already. The World Federation of Hemophilia offers a global directory for individuals to find a center in their location.
Working with your hematologist to customize care
There is no one-size-fits-all approach to treating hemophilia A. Individuals should work closely with their hematologist to determine which medications will best control bleeding and improve quality of life, given their bleeding patterns, activity levels, and personal goals. Needs may change over time, so it’s important to maintain consistent and open communication.
While the hematologist is typically at the core of the care team, treatment often goes beyond hemophilia medications. Many patients benefit from nondrug interventions, such as physical therapy, mental healthcare, and working with a dietitian to develop a healthy diet.
A person’s hematologist can help refer a person to other specialists as needed and coordinate the multidisciplinary care team.
Hemophilia News Today is strictly a news and information website about the disease. It does not provide medical advice, diagnosis, or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website.
