What it’s like managing hemophilia and an inhibitor, part 3
Taking note of the advancements for my sons and the larger community
Last in a series. Read parts one and two.
Guinea pigs are similar to humans. Their body chemistry has led to testing that’s revolutionized research, with the development of vaccines, replacement heart valves, and asthma medications, to name a few. The term “guinea pig” has become synonymous with human experimental subjects. This feeling of being a guinea pig is familiar in the hemophilia community.
Newer factor products on the market have longer half-lives, requiring fewer weekly infusions. Despite case studies conducted to prove the efficacy of these products, many patients are leery of the newest and most promising treatments. Using new products immediately after their approval by the U.S. Food and Drug Administration (FDA) makes many patients feel like guinea pigs, as the community watches to see how others respond before making the switch.
My sons’ different inhibitor journeys
My oldest son, Julian, who’s now 28 years old, has hemophilia and developed an inhibitor at 11 months. Since he had a low-titer inhibitor, his hematologist immediately sent him to the hospital to place a port-a-cath, which would enable my husband and I to infuse Julian daily, as required by the protocol for immune tolerance induction (ITI).
Daily infusions for two and a half years eradicated the inhibitor. Julian was then able to receive factor VIII three times a week for prophylaxis. The inhibitor hasn’t resurfaced. ITI worked, the best-case scenario, for which I’m deeply grateful.
Years later, longer half-life products came to market, but my husband and I were not what we call “early adopters” of the new products. We, too, had the wait-and-see approach. Switching to a new product brought the fear of an inhibitor reappearing. If we used the new products immediately, we worried we’d be treating our son like a guinea pig. However, the potential relief and comfort these new products could bring to Julian and our family gave us hope and reassurance.
One of the most groundbreaking advancements in inhibitor treatment has been Hemlibra (emicizumab-kxwh), which changed the lives of many inhibitor patients because they stopped bleeding.
My youngest son, Caeleb, who’s 19, has hemophilia as well and was diagnosed with a high-titer inhibitor at 11 months and continues to live with one. His complex case prompted his hematologist to take a conservative approach, closely observing how her inhibitor patients reacted to emicizumab. Eventually she recommended that Caeleb switch to Hemlibra, which has improved his quality of life tremendously.
Emicizumab is used for patients with hemophilia A with inhibitors or without. This product has changed the outlook for the hemophilia A community by preventing bleeds. Hemophilia B (factor IX deficiency) patients with an inhibitor haven’t had an option until recently.
Alhemo (concizumab-mtci) is FDA-approved as a new treatment from Novo Nordisk for patients with hemophilia A or B with an inhibitor. Alhemo is injected subcutaneously daily, a breakthrough method of administration for hemophilia patients.
Some patients prioritize bleeding prevention and opt for treatments like Hemlibra or Alhemo, despite their inhibitor remaining present. Others prefer ITI, aiming to eradicate the inhibitor to allow the natural benefit of factors VIII or IX, benefits the body is missing.
Inhibitor education advances
Education and community are critical on the inhibitor journey. The Comprehensive Health Education Services (CHES) Foundation provides education, support, and connection for the inhibitor community via two invaluable programs, webinars, and its annual publication, Lifelines for Health.
After the Shock is a weekend family camp program that offers a vital support network for parents, siblings, and children, fostering a familylike community of understanding in what can be an overwhelming journey. Sometimes called “a camp to call our own,” it reflects the inclusive and highly personalized model of support needed on an inhibitor journey.
InhibitCon provides tailored learning tracks for caregivers, adult men, teens, and kids with inhibitors. This weekend-long national event offers topics such as information on existing products and those in development, insurance issues, mental health support, pain management, and access.
Having experienced both a successful ITI outcome with Julian and the transformative impact of Hemlibra on Caeleb, I understand the complexities of these decisions. While there’s no universal standard of care for inhibitor patients, one thing remains certain: Education and community support are essential.
There’s no one standard of care for inhibitor patients, and immune systems are highly individualized. Thus, treatment protocols should reflect personal preferences. Most importantly, families and patients must be educated. Take advantage of the CHES Foundation’s offerings. Talk to your hemophilia treatment center, get involved in your local chapter, and connect with organizations like the National Bleeding Disorders Foundation and the Hemophilia Federation of America.
I’ve barely scratched the surface of inhibitors with this series. I plan to return to this conversation sometime later and share stories of how quality of life has changed for those with inhibitors.
A special thank you to my friend, Janet Brewer, co-founder of CHES, for sharing her expertise.
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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