Behind the Science: A Closer Look at How Current and Innovative Approaches Can Help Achieve Hemostasis in Hemophilia

Let's start with Hemostasis

Hemostasis is the body's ability to stop and prevent bleeds. 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.

Procoagulants are proteins that promote clotting (like Factor VIII or IX) while anticoagulants are proteins that prevent clotting (like antithrombin and tissue factor pathway inhibitor (TFPI)).

The importance of balance in hemophilia

How could targeting anticoagulants help prevent bleeds?

Hemostasis is the goal of any hemophilia treatment—and treatments can work by targeting procoagulants or anticoagulants.

Innovative treatments called rebalancing therapies are being investigated and may offer a different type of nonfactor treatment in the future.

These rebalancing therapies are designed to increase the amount of thrombin in the body by lowering anticoagulants like antithrombin and TFPI – lowering proteins in the blood that slow down the body's ability to make clots may help increase thrombin generation and rebalance hemostasis.

How do current hemophilia treatments achieve hemostasis?

Factor replacement therapy is a well-known treatment that works by replacing missing or defective factor VIII or factor IX.

Other treatments include factor mimetic therapy, which mimics the function of factor VIII in the body, and gene therapy, which replaces the missing or faulty gene to create factor VIII or factor IX for hemophilia A or hemophilia B, respectively.

The science behind hemophilia management is continuously advancing. In addition to currently available treatment options, potential new approaches to restoring hemostasis may offer more ways for PwH to achieve balance.

Learn more at MyHemophiliaTruth.com

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