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The importance of balance in hemophilia
Without Hemophilia
With Hemophilia
In order to achieve hemostasis, a balance is required between procoagulants (proteins that promote clotting) and anticoagulants (proteins that prevent clotting).
In hemophilia A or B, factor VIII or factor IX, respectively, is not working properly, leading to an imbalance between procoagulants and anticoagulants. This leads to excessive bleeding because the body is not creating enough thrombin.
Achieving a balance between procoagulants and anticoagulants allows the body to generate sufficient thrombin, which is critical to stop bleeds.
Rebalancing therapies are currently under investigation
Innovative rebalancing therapies may one day offer different types of nonfactor treatment.
- Rebalancing therapies are designed to increase the amount of thrombin in the body by lowering anticoagulants
- Lowering proteins in the blood that slow down the body's ability to make clots may help increase thrombin generation and rebalance hemostasis
The role of antithrombin
Antithrombin prevents the generation of thrombin.
By blocking antithrombin, more thrombin can be generated to form a blood clot.
Researchers are currently investigating a rebalancing treatment option in clinical trials that targets antithrombin.
The role of tissue factor pathway inhibitor, or TFPI
TFPI is an anti-clotting protein that stops the initial blood clotting process prior to thrombin being made.
- Rebalancing treatments that target TFPI are currently being studied in clinical trials
- Targeting TFPI may be a potential way to help increase thrombin production
Innovative approaches that target different parts of the coagulation cascade may help achieve hemostasis.
Hemophilia treatments and thrombin
There are different treatments to help prevent bleeding episodes, all with the objective of increasing thrombin. These include:
- Factor replacement therapy
- Nonfactor therapy
- Gene therapy
Factor mimetic therapy is a type of nonfactor therapy.
- Factor mimetic therapy increases the amount of thrombin in the body by mimicking the activity of factor VIII
Current treatment options
- Factor replacement therapy is given via an IV therapy (an infusion in the veins)
- There’s a risk of developing inhibitors, which may require a different treatment
- Factor replacement therapies may require frequent dosing to maintain desired levels of factor
- Factor mimetic therapy is given through a subcutaneous injection, an injection just under the skin. The dosage of this treatment is based on your body weight
- Some people taking factor mimetic therapy may encounter unexpected (or breakthrough) bleeds and require additional therapy on an as-needed basis to help resolve bleeds
- May be an effective long-term treatment option for eligible adults living with hemophilia even though gene therapy cannot cure hemophilia or be given more than once
- Is approved only for adults with either hemophilia A or hemophilia B without inhibitors
- Monitoring may reveal a need for additional treatment with steroids or other medications that keep the immune system in check
Factor replacement therapy ...
... is a well-known treatment that can help prevent bleeds and treat unexpected (or breakthrough) bleeding episodes that may occur. It works through an infusion in the veins that replaces the missing or defective clotting factor in the body.
Factor mimetic therapy ...
... is a different kind of treatment that mimics the function of clotting factors in your body. It can be used to protect against bleeds in people with hemophilia A, even if they have inhibitors.
Gene therapy ...
... could be a longer-term treatment option for eligible adults living with hemophilia. They are one-time treatments that work by replacing a missing or faulty gene to create factor VIII for hemophilia A or factor IX for hemophilia B.
Gene therapy:
Monitoring factor levels and liver function regularly is recommended after treatment with gene therapy.
How could discussing the whole experience make an impact?
Each patient story reflects the real-life experiences of individuals diagnosed with hemophilia. Individual experiences may vary. These patients were compensated for their time creating this content.
MAT-US-2307290-v1.0-11/2023
Last Updated: November 2023
