Hemophilia’s treatment menu is longer, but patients must put in the work
For most of my life, the phrase 'treatment options' was a contradiction in terms
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When I was diagnosed with hemophilia decades ago, I was 10 years old, and nobody asked me what I wanted.
That isn’t a complaint about my doctors. There was nothing to ask. There was hemophilia B and von Willebrand disease, and one way to treat them. You took what existed or you bled. For most of my life, the phrase “treatment options” would have been a contradiction in terms.
Now there’s a menu
We now have factor products engineered to last longer between doses; non-factor prophylaxis given by injection instead of IV; rebalancing agents; gene therapy; options for people with inhibitors; and options for children.
This is what my community spent 50 years fighting for. I want to be clear about that before I say the next part.
Choice is work.
Every option on that menu comes with a trade-off — how often you dose, how it’s delivered, what it requires you to monitor, what it does to your bleed plan. And somebody has to weigh those against the life you actually want to live. Your hematologist can tell you what a therapy does, but only you know whether you’re planning a pregnancy, taking a job with no sick leave, or hoping to get back on a bike at 60.
All of this means the decision has quietly moved. It sits with us now. And most of us were never trained for it.
I work in this community. I read about this constantly. And I still scan a conference agenda and find a product name I don’t recognize, or realize something I explained to someone a year ago has already been revised. If it’s hard for me, what is it like for the family three hours from the nearest treatment center, seeing a hematologist twice a year? What is it like for a woman who spent a decade just getting someone to believe she bleeds?
A series built for that gap
“Navigating Hemophilia: A Webisode Series on Treatment Options and Overcoming Barriers,” from CME Outfitters, is aimed squarely at explaining new treatment options. It’s free.
It covers non-factor prophylaxis for hemophilia A and B, with and without inhibitors. It takes on gene therapy — not as a headline, but as an option carrying a safety profile and monitoring requirements. And it spells out something I rarely see addressed anywhere: how your bleed plan changes when your prophylaxis changes. What you do for a breakthrough bleed? Or before a procedure? What you tell an emergency room at 2 a.m?
The faculty are Maya Bloomberg, of the University of Miami Hemophilia Treatment Center; Guy Young, MD, of Children’s Hospital Los Angeles; and Mark Skinner, who has spent a career building the case that what patients experience counts as data.
The course features downloads for you to print and use; a prophylaxis comparison chart covering treatment type, dosing, monitoring needs, and lifestyle trade-offs; a plain-language FAQ, “Moving Beyond ‘One-Size-Fits-All'”; a treatment center locator; and a shared decision-making tool from The World Federation of Hemophilia — a real framework for steering an appointment instead of receiving it.
Bring more than symptoms
Shared decision-making takes two prepared people in the room. We spend a lot of energy asking our care teams to show up ready. We owe ourselves the same.
I can’t keep up with this field, and it’s half my job to try. But I can walk into an appointment knowing what I want my life to look like, and I can make someone account for how their recommendation fits it. That has never required a medical degree.
The 10-year-old I once was didn’t get a say. She didn’t have one to give. I do now — and so does every person reading this, whether or not anyone has told them so.
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 consult your physician or another qualified healthcare 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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