How improved treatments may affect young people with inhibitors
While the future looks promising, we can't forget the past, this columnist says
My happy place is in front of an audience. It’s where I feel most comfortable. As I’m a self-proclaimed introvert, that may sound odd. Yet I’ve had numerous opportunities to answer my call to speak. It’s not about accolades and praise; it’s about the message.
Regardless of the size of the audience, I encourage participation. At a recent hemophilia event, however, I had a realization. When I asked for a show of hands of how many people lived with inhibitors, few hands were raised. I never anticipate many people with inhibitors to be in an audience, but this was an unusually small number. I was surprised.
According to the U.S. Centers for Disease Control and Prevention:
“Approximately 1 in 5 people with hemophilia A and about 3 in 100 people with hemophilia B will develop an antibody — called an inhibitor — to the treatment product (medicine) used to treat or prevent their bleeding episodes. People with [von Willebrand disease] type 3 may also develop inhibitors. Developing an inhibitor is one of the most serious and costly medical complications of a bleeding disorder because it becomes more difficult to treat bleeds.”
My youngest son, Caeleb, is 17 and has lived through the most challenging circumstances, even at his young age, due to hemophilia and an inhibitor.
The impact of improved treatments
The landscape for treating bleeding disorders has changed in recent years. Some medications can now be injected subcutaneously rather than infused peripherally or via a port. New products for those without inhibitors require less frequent dosing. Gene therapy is in development, and treatment options are expanding.
At a recent bleeding disorders event, I spoke with a woman who expressed her gratitude that I’d spoken about inhibitors. During our conversation, I realized there are people with inhibitors who now have access to treatments that may keep them bleed-free. As a result, many may never experience joint bleeds or loss of mobility. Treatment can even help severe hemophilia become mild.
I tell stories of raising my sons (always with their permission) so that others know they’re not alone in their struggles. But some people with inhibitors may never face those struggles.
Of course, not bleeding is great! I don’t wish for anyone to go through the nightmare that Caeleb has experienced, but it’s hard to comprehend that there are people with inhibitors who’ve never experienced a major bleed. Although Caeleb is dealing with severe issues resulting from years of joint bleeds, he hasn’t had an active bleed in five years.
However, the bleeding disorders community mustn’t forget its past.
The Hemophilia Federation of America developed a beautiful exhibition that toured the country, depicting the history of hemophilia from 1000 to 2019. It highlighted the 1980s AIDS crisis and how a generation of men in the community lost their lives due to the bad blood epidemic, also known as the hemophilia holocaust.
As a new mom in the community in 1996, I soon learned about this tragic chapter in our history. But because I hadn’t experienced it firsthand, it was confusing, and I didn’t understand the impact until many years later.
Now I see something slightly similar happening. With multiple new products on the market changing the lives of those with bleeding disorders, a new generation of boys and girls may never experience a significant joint bleed. Will they ever understand all the possible complications of their condition? I’ve witnessed the nightmare of an inhibitor, and Caeleb is still suffering its effects.
While many with inhibitors now live empowered, healthy lives without bleeds, many, like my son, will never forget the implications of this complication.
I can’t let stories like Caeleb’s be forgotten.
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.