What it’s like managing hemophilia with an inhibitor, part 2
The treatment landscape has changed drastically in recent years
Second in a series. Read part one.
A hemophilia inhibitor is like a firewall in a computer network. Clotting factor replacement therapy is supposed to enter the bloodstream and help form clots to stop bleeding, just as data should pass through a network without interruption. However, an inhibitor acts like an overly aggressive firewall, misidentifying the helpful clotting factor as a threat and blocking it from doing its job.
Just as information technology specialists must find ways to bypass or reconfigure a firewall, doctors and patients must use specialized treatments to overcome inhibitors and restore normal functioning.
“If you know one inhibitor patient, you know one inhibitor patient,” Janet Brewer, co-founder and CEO of the CHES Foundation, told me during a recent phone call.
The CHES Foundation, which stands for Comprehensive Health Education Services, provides education and support to those with chronic health conditions such as inhibitors and bleeding disorders. The organization offers a range of resources, from informational materials to in-person retreats, to help those affected by inhibitors navigate their unique challenges.
Janet and I have both raised sons with inhibitors. During the years when my youngest son, Caeleb, now 19, was struggling with a high-titer inhibitor, she was also in the trenches with her son.
After my oldest son, Julian, was born and diagnosed with hemophilia and an inhibitor in 1996, 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 of us had the same journey. As I noted in my last column, every body is different. Our shared experiences have shown us the resilience and strength of patients and families dealing with inhibitors.
The changing landscape of inhibitor treatment
But in the past decade, inhibitor treatment has changed dramatically. When we began treating my sons, 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, most doctors won’t attempt the treatment until inhibitor levels are less than 10 Bethesda units (BUs). While the treatment worked for Julian, whose level was less than 10 BUs, Caeleb’s level was in the thousands.
As a result, treating Caeleb’s bleeds was difficult. Doctors used bypassing agents instead of factor VIII, but even with this treatment, it often took weeks for a bleed to subside. Most times, Caeleb’s bleeding would recur shortly after it improved. This story is all-too-familiar for inhibitor patients.
However, the changing landscape of inhibitor treatment has brought new products to help patients have less frequent bleeding.
In 2017, Hemlibra (emicizumab-kxwh) became the first nonclotting factor treatment to be approved in the U.S. to treat hemophilia A, which both my sons have, with an inhibitor. (Its approval was extended in 2018 to hemophilia A patients without inhibitors.) The therapy works by mimicking the function of factor VIII and binding to factors IX and X to initiate blood clotting — thus preventing or reducing bleeding episodes.
Hemlibra has proven life-changing for many patients like Caeleb who endured years of bleeding into their joints. While Caeleb now bleeds less and enjoys a better quality of life, the joint damage remains, as well as chronic pain. Hemlibra is not an ITT. It is not factor VIII, and it’s not used to treat an active bleed.
There’s no single, standard treatment for an inhibitor. The approach can vary depending on the hematologist’s preference, the 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.
As Janet explained, “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.”
Understanding the latest developments and treatment options is crucial to managing hemophilia with an inhibitor. Connecting with the inhibitor community through social media and local and national bleeding disorder organizations, such as the CHES Foundation, can be a helpful first step.
Note: 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 another 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. The opinions expressed in this column are not those of Hemophilia News Today or its parent company, Bionews, and are intended to spark discussion about issues pertaining to hemophilia.
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