Why knowing which hemophilia treatments don’t work is valuable

I groggily sat up in the hospital bed, sipped some water, and stared into the distance. Suddenly, a nurse pointed at my gown and shouted, “You’re bleeding!” But I’d taken so many painkillers that I barely reacted.

This was 2003, when I had laparoscopic surgery to treat endometriosis. Doctors had approached me as a carrier of hemophilia, rather than a hemophilia patient, and the plan didn’t work. I don’t remember how the bleeding stopped, but I do know what went wrong.

Thankfully, what happened to me is probably less likely to happen to someone today, thanks to the advancements in hemophilia advocacy for women, although we still have a long way to go.

The gynecological surgeon had been concerned about performing surgery on someone who was a carrier for hemophilia and sent me for a consultation with a hematologist. Desmopressin acetate (DDAVP) was prescribed to help me get through surgery safely. That was a high standard of care for a woman with hemophilia at the time.

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DDAVP is a fantastic treatment option for some people with mild bleeding disorders. It works by releasing the body’s storage of factor VIII, a protein that helps to clot blood and is insufficiently produced in my body. DDAVP shouldn’t be used several days in a row because these storages need time to be replenished. Additionally, we now know that not everyone responds well to DDAVP, so factor VIII levels may not rise or be sustained long enough to prevent or stop a bleed.

Today, doctors will do a DDAVP challenge before prescribing the medication to see if it works, which is important information, particularly in emergency situations.

One of the hard things about this drug is that it causes fluid retention, so patients should restrict their fluid intake when using it to avoid the risk of a dangerous drop in sodium levels. Unfortunately, when I first received this medication, I don’t think this fact was well known, because I received a lot of IV fluids. When the DDAVP finally wore off about a day later, I found myself quickly limping to the bathroom and urinating about every 30 minutes for hours.

Why DDAVP didn’t work for me

I think it was groundbreaking that in 2003, doctors thought to treat me to keep me safe during surgery. I wish it’d worked.

I have a rare hemophilia variant that causes reduced factor VIII binding to von Willebrand factor (VWF). When I describe this trait to people, I explain that factor VIII and VWF “hold hands” in the bloodstream (forming a noncovalent complex). VWF helps stabilize factor VIII, keep it in the blood, and carry it to the site of injury. The factor VIII in my body has reduced binding to VWF, so it doesn’t “hold hands” well, causing it to leave my body faster and decrease its effectiveness.

DDAVP didn’t work for me because it releases storage of factor VIII, and given my problem with that factor, the action wouldn’t offer increased protection. I didn’t learn about my genetic variant and the implications of it until 15 years later.

My experiences reinforced the critical importance of requesting pharmacokinetic testing to understand how medications work in my body. That can be different from the way the same medication works in someone else’s body.

I wish the surgery I mentioned above was the last one I had, but 15 years later, I also had a hysterectomy. It happened at a time when people were more aware that women with hemophilia should be treated. While I was still called a “symptomatic carrier” at the time, replacement factor VIII was given to me to keep me safe during surgery.

Still, after the surgery, I ended up in the emergency room, pouring blood, because we’d assumed that standard half-life products worked for me. My official hemophilia diagnosis came when I’d bled for 30 days post-surgery. It’d be another two years before a wonderful doctor discovered that standard half-life factor VIII products aren’t effective at stopping bleeds for me.

I’ve learned a lot over the years. Understanding what doesn’t work in my body is as critical as knowing what does. Though I’m still fine-tuning my treatment regimen, it’s much better than the old ones. I try not to get discouraged. Knowledge is power, even if it’s bad news. I’m thankful to have access to medication and a care team that works with me to evaluate our next steps to improve my care.

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.