Life Between Peak and Trough

Life Between Peak and Trough

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.

Managing hemophilia

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.

Shellye Horowitz is a licensed school counselor and school administrator with over 25 years of experience in the field of education. Shellye has strong ties to the bleeding disorders community with six traceable generations of hemophilia A in her family.
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Shellye Horowitz is a licensed school counselor and school administrator with over 25 years of experience in the field of education. Shellye has strong ties to the bleeding disorders community with six traceable generations of hemophilia A in her family.
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10 comments

  1. Great article! As a mild, I can relate to what you said there… Almost all of my bad bleeds when I was younger could have been controlled, but my doctor always said, “He’s just a mild, so let’s use ice and elevation…” We usually ended up in the ER dealing with a much bigger issue because we didn’t factor to start with. Women have an even tougher hurdle of sometime not being believed about bleeding problems as well. Thanks for sharing this and spreading awareness!

  2. Priscilla Oren says:

    Really could identify with this as a now senior citizen female with mild 20-30% all of my life and was not even diagnosed until mid 20’s and never treated until my 40’s. Results of course are advanced arthritis in all joints and many muscle bleeds that left hard lumps of scar tissue.
    Thanks for posting this!

  3. Mary Jo Haugen says:

    This article was very nice to read. Thank you!! Our family has mostly milds. We have had many trageties of bad treatment and serious, occasionally fatal bleeds. Pprophy for all is the best way to treat and I am now on Prophy after 9 months of lack of care for a smashed L. Shoulder (100s of fractures) and now I have prophy and a longer trough elevation. It has been difficult but factor has saved my arm!! Prophy is the best. Hope others start it if they can.

  4. David Clark says:

    Hi Shellye, First, I want to thank you for your columns. I’m a volunteer with the Coalition for Hemophilia B, and I’ve publicized your articles within our group. We’re trying to do more to raise awareness about issues for women with hemophilia, too.

    In my younger days, I was a researcher who believed the literature that said women don’t get hemophilia. Then I got involved with the Coalition and couldn’t understand why many of the female family members I met had bleeding problems!

    The Coalition was also involved in the B-HERO-S study a few years ago, which was one of the first to survey quality of life issues for milds and moderates. We found (surprisingly?) that milds and moderates can have significant problems that are different from those of severes. I agree that factor level alone is not the best indicator of bleeding risk.

    Anyway, thanks for your columns and keep up the good work!

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