Published by Hemophilia News Today, March 5, 2025
“You have to use what’s right for you. Don’t feel pressured to try one thing or another. Talk to many knowledgeable people. Do your research.” – Janet Brewer, Co-founder and CEO of CHES
A hemophilia inhibitor is like a firewall in a computer network. Clotting factor replacement therapy is supposed to enter the bloodstream and stop bleeding, just like data should pass through a network without interruption. However, an inhibitor acts like an overly aggressive firewall—it misidentifies the helpful clotting factor as a threat and blocks it from doing its job.
Just as a malfunctioning firewall can disrupt communication and prevent essential information from reaching its destination, an inhibitor stops the clotting factor from working properly, making bleeding harder to control. Just as IT specialists must find ways to bypass or reconfigure a firewall, doctors and patients must use specialized treatments to overcome inhibitors and restore normal function.
“If you know one inhibitor patient, you know one inhibitor patient,” says Janet Brewer, Co-founder and CEO of the CHES Foundation. This organization, which stands for Comprehensive Health Education Services, provides education and support for those with chronic health conditions such as inhibitors and ultra-rare bleeding disorders. The CHES Foundation offers a range of resources, from informational materials and in-person retreats, to help those affected by inhibitors navigate their unique challenges.
Janet and I have stories of raising our sons with inhibitors. During the years when my youngest son, Caeleb (19), was struggling with a high titer inhibitor, she was also in the trenches with her son. From 1996 to 2014, I developed a network of friends, including Janet, who parented children with inhibitors. We were a tight-knit group that communicated through social media, met at national and local events, and called each other in the middle of the night when needed. We had an inhibitor diagnosis in common, but none had the same journey. Every body is different. Our shared experiences have shown us the resilience and strength of patients and families dealing with inhibitors.
The language regarding inhibitor treatment has changed dramatically. When treating my son, Immune Tolerance Therapy (ITT) was the goal. ITT involves infusing large amounts of factor into the bloodstream in the hope of eradicating the inhibitor. Unfortunately, not all patients can begin ITT. For ITT to succeed, most doctors will not attempt the treatment until inhibitor levels are less than 10 Bethesda units (BUs). Caeleb’s levels, like many others, were in the thousands.
Until Caeleb’s levels were lowered, treating bleeds was difficult. Because factor VIII could not be used, bypassing agents were used, but this treatment often took weeks for a bleed to subside. Most times, the bleeding would recur shortly after it improved. This is an all-too-familiar story for inhibitor patients.
The changing landscape of inhibitor treatment has brought new products to help inhibitor patients have less frequent bleeding. Emicizumab is the first non-clotting factor treatment for hemophilia A with an inhibitor (and is also approved for patients with hemophilia A without an inhibitor). It replaces part of the function of factor VIII. It brings activated factor IX (FIXa) and factor X together to help prevent and/or reduce bleeding episodes.
Emicizumab has proven life-changing for patients like my son, who endured years of bleeding into their joints by reducing bleeding episodes and infusions. While most patients using emicizumab are bleeding less and have a better quality of life, joint damage remains, as well as chronic pain. Emicizumab is not ITT. It is not factor VIII and is not used to treat a bleed once started.
There is no one standardized treatment for managing an inhibitor. The approach can vary based on the hematologist’s preference, the country’s region, and the patient’s specific needs. Some Hemophilia Treatment Centers may be more aggressive or conservative than others, and patients and families need to understand these differences and advocate for the best possible care.
The language of inhibitors has changed dramatically. The bottom line is that patients and families must do their research. Understanding the latest developments and treatment options is crucial in managing and living with inhibitors. Finding the inhibitor community through social media and local and national bleeding disorder organizations, such as CHES, is the first step to understanding the journey.
