One of the challenges for individuals with hemophilia — whether severe, moderate, or mild — is bleeds. Often people with hemophilia will struggle with bleeds into their joints. The bleeds are painful and limit mobility. If left untreated, they can cause long-term damage, such as arthritis.
Hemophilia bleeds are most often managed in two ways. Individuals treat with factor replacement products on demand or prophylactically to prevent a bleed. The severer the hemophilia, the more likely a person will need to use prophylactic treatment.
On-demand treatment provides people with factor replacement products when they are injured or have an active bleed. During a bleed, a person is often directed by their hemophilia treatment center (HTC) to take factor once a day to increase their levels.
Due to short, half-life realities of these products (often fewer than 12 hours), people need to infuse frequently. By the time they infuse 24 hours later, many are back at their base level of factor (which, for severe hemophiliacs, is less than 1 percent).
Prophylactic treatment provides routine factor replacement. Pharmacokinetic studies are sometimes conducted prior to prophylaxis. Factor is then prescribed from twice a week to daily for protection.
Doctors work to maintain a trough level — the lowest level of factor before the next infusion — to prevent bleeds. While there is a debate about how high the trough should be, many researchers agree that higher trough levels correlate with fewer bleeds.
A peak number is the highest amount of factor in a person’s system after they infuse. If a person metabolizes factor quickly, they may need a higher peak level to reach the target trough level.
With hemophilia, treatment can be tricky. Factor levels (or disease severity) alone cannot determine whether a person needs on-demand or prophylactic care. Some people with severe hemophilia rarely bleed, and some with mild hemophilia bleed a lot. Severity can be a guide, but bleeding patterns must also be taken into account.
Thoughts about treatment
As I think about prophylactic treatment, I find myself wondering about treatment efficacy and emphasis on trough levels. Perhaps the peak level — that time spent at more normal factor levels — is just as critical.
I had bleeding issues arise when stitches dissolved after a major surgery. When I started hemorrhaging, I was on factor every 12 hours. That brought my factor levels close to 60 percent each time. And I kept bleeding.
After a few weeks, my HTC changed my plan and had me infuse once a day, up to 100 percent. Their theory was that my body needed time at higher peak levels to aid in my healing. They were right.
Perhaps even a short time spent at normal peak levels affords a person with severe hemophilia a chance to heal in ways those with moderate and mild hemophilia (who are not on prophylaxis) do not.
Perhaps the individual with severe hemophilia who takes factor to 100 percent before a basketball game is more protected than a person with mild hemophilia, who stays at their continual 20 percent or 30 percent during the game.
Researchers are learning more about women with mild and moderate hemophilia and carriers who suffer from arthritis and joint pain later in life. Would these women have had fewer issues if they had been afforded access to peak factor levels that allowed their bodies to heal?
We have a lot to learn about treating hemophilia. I know women with mild hemophilia who struggle with regular bleeds. Some are gaining access to prophylactic treatment, which is changing their lives. Women with treatment access anecdotally report less pain, more mobility, and more physical activity.
Cost prohibits access
This brings me to the biggest issue: cost. Factor replacement products are among the most costly treatments on the market. If they were affordable, I believe all people with hemophilia would have equal access to prophylactic treatment to bring their factor levels to a normal peak and protective trough daily. But this is not the case.
I believe cost is the largest barrier to treatment access for those who do not have a severe form of hemophilia. Perhaps one day this barrier will disappear.
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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