Women with Mild Hemophilia Deserve Prophylactic Treatment
When someone has a hemophilia diagnosis, they receive treatment in one of two ways: on demand, in which they are given products to help their blood clot after an injury or before a scheduled medical procedure; or prophylaxis, which is regular, preventive treatment. The latter is often used in those with severe or moderate hemophilia, and as most of these are men, few women are treated prophylactically.
Prophylactic treatment for mild hemophilia
Studies of those with severe hemophilia show that the number of bleeds is reduced when trough levels increase. However, many people with mild hemophilia — factor levels below 40 percent, and even as low as 5 percent — may not receive prophylactic treatment. I wonder if individuals with severe hemophilia on prophylaxis are more protected than those with mild hemophilia who are treated on-demand?
In a Phase 3 trial, published in 2018, participants with severe hemophilia A without inhibitors given regular doses of the medication Hemlibra (emicizumab-kxwh) reported a 96 to 97 percent reduction in bleeds.
Meanwhile, those with mild hemophilia, both male and female, are still getting multiple bleeds a year. I average six to 12 bleeds a year as a woman with mild hemophilia.
Treatment improves lifestyle choices
I know of teens with severe hemophilia A who are on prophylactic treatment to optimize their factor levels. One young man plays basketball and is far more active than I could ever dream of being. He can play a contact sport because he is protected. His factor levels are increased before a game for safety.
My factor levels are continually low as I am not on prophylactic treatment. I ride a bike 14 to 16 miles a day and struggle with mobility issues from joint microbleeds. I love to run on the beach with my dog, but can’t risk sustaining an ankle or knee bleed that would take weeks to heal.
Recently, I had an elbow bleed that required factor. The bleed did not limit my leg mobility, so the day that I infused I ran with my dog on the beach as I was protected. It was a wonderful, freeing moment. My companion asked me, “Why are you able to run today?” I answered, “I have factor in me. I am safe.”
It felt so good to let loose and feel “normal.” Afterward, my knees and ankles felt better than on the days I ride my bike because I was protected. Why do we have a system in which someone with severe hemophilia is supported and another with mild hemophilia has low protection? This potentially limits the activity of the person with mild hemophilia more than the person with severe disease.
Women want treatment
I know that many women with mild hemophilia question why they are denied access to prophylactic treatment. They want it for many reasons: to be physically active, to prevent injuries, and to mitigate the effects of heavy prolonged monthly bleeding. They struggle with being intimate with their partners. They want prophylactic options to protect them so they can have an active, safe sexual life with their significant others.
Early treatment options
As new treatments enter the market, the standard of care for hemophilia is rapidly changing. Many infants with severe or moderate hemophilia are being placed on prophylactic regimens prior to any injury with the aim of preventing long-term joint damage and increasing overall health and mobility.
Studies are showing that women carriers of hemophilia, even those with higher levels of factor, have joint damage as they age. They are less likely to have access to treatment for bleeds, and may not realize when they are bleeding — possibly due to years without treatment and having learned to live with the pain of bleeds.
Women deserve to be free from fear
Women with mild hemophilia are asking about prophylactic treatment. They want to live their lives without pain, to protect their joints, and to not worry about injuries while being active. While we are not demanding a weekly infusion routine, we want options to infuse before activities that may cause bleeding. We want to be active without fear. Women with mild hemophilia deserve to be just as protected as someone with severe hemophilia.
Shouldn’t we all be able to run on the beach or be intimate with a partner without fear of bleeds?
What do you think? Please share your thoughts in the comments below.
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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 Services, and are intended to spark discussion about issues pertaining to hemophilia.
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AnnK
Thank you for raising this important issue!
Mary Haugen
Many women with hemophilia have alot of bleeds and damage to their bodies. Micro vleeds are hard to treat to stop them (not less treatment). I think all people with hemophilia need to decide for themselves and not have a prophy decision made for them. I am on prophy and think it is great. Many aches and pain have showed me that they are bleeds. I can walk without pain right now (my L. knee is destroyed from old bleeds and micro bleeds). My shoukder ccontinues to have bleeds off and on but much better with factor). I am thankful for a slight increase in factor amount has greatly helped bleeds to be much improved). Factor is what all bleeders need, and adequately treated to stop them, otherwise, they continue to leak. Very good article.
Paul Clement
In response to your question "I wonder if individuals with severe hemophilia on prophylaxis are more protected than those with mild hemophilia who are treated on-demand?" the answer is no. In the US, most people on prophylaxis with clotting factor still have a target trough level of about 1%--this is too low to offer protection against spontaneous joint bleeds. It has been recommended that the trough level be raised to 15% to prevent most spontaneous joint bleeds. The main impediment to raising the trough level--and why women with mild hemophilia are often denied prophylaxis--is cost.
In the next sentence you mention Hemblibra. This drug is not a clotting factor but a bi-specific antibody that mimics the function of factor VIII. It can only be taken prophylactically, and has remarkable results and proven to be more effective at preventing bleeds than prophylaxis with factor.
Finally, everyone should be treated based on their bleeding phenotype (how frequently they bleed) not on their factor level or gender (some women with factor levels of 60% still suffer from bleeding issues). That being said, you might also consider using Stimate Nasal Spray, which could boost your factor level from your current 20% to between 30% and 50%, and which might be easier to obtain than factor.
G Shellye Horowitz
Thank you, Paul, for your comments!
I agree with you re: the target trough. One might argue that the target trough is that low point prior to the next infusion. Therefore, at some point immediately after infusion and before half life realities set in, someone with severe hemophilia would have factor levels at higher levels (some above 100%, it depends on their treatment plan). This is the prophy I advocate for women, situationally, as needed for protection immediately before activity. As you say, really this should be for all based on their bleeding realities.
Unfortunately, my personal variant does not respond to DDAVP. Interestingly, some research is now showing that, as a whole, women do not respond as well to DDAVP.
I definitely agree with your comment, also, re: cost/. I think if cost was not a factor (no pun intended) the factor products would be more readily available to all. (or, Hemlibra for that matter, as well - that costs even more, but may be the ultimate better option for most if cost were not an issue). I hope that, at some point, cost can be put aside and we can make sure that all people with hemophilia get exactly what they need to be safe and protect their joints (among other things) long term.
Do you have any links to research re: the minimum tough levels at 15% and spontaneous bleeds reduced at those levels? Would love to quote that in the future.
Thank you, again, for your comments!
Paul Clement
Hi Shellye,
The 15% trough level has been championed by Mark Skinner, former president of the World Federation of Hemophilia, for several years. Here is a link to an article that outlines the reasoning behind the 15% trough level:
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4111811/
You might also be interested in this article that recommends a trough level of 30% (given that about 6% of people with a 15% factor level still suffer from joint bleeds): https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6113607/
G Shellye Horowitz
Thank you!!!