Why more women with hemophilia are needed in clinical trials

Clinical trials for hemophilia are essential to advance medical knowledge and develop treatments. But for decades, trials related to hemophilia enrolled only men, given the assumption that women were only carriers of the disease. Consequently, women with the disorder were denied the opportunity to participate in trials that could potentially enhance their quality of life.

This neglect stemmed from a lack of awareness and misconceptions about the prevalence and severity of hemophilia in women. As a result, women have struggled to access treatment, even though they face unique bleeding complications during menstruation and childbirth.

I have a dual diagnosis of hemophilia B and von Willebrand disease (VWD). As such, I’ve encountered limitations in trial participation; many years ago, for instance, my hemophilia diagnosis prevented me from participating in a clinical trial for a medication to treat VWD, as such trials often have specific inclusion and exclusion criteria to ensure the results’ validity and the participants’ safety.

Despite feeling disappointed, my hematologist allowed me to experiment with the medication, aminocaproic acid, off-label. I immediately noticed a significant improvement in my menstrual bleeding. Amicar, the medicine’s brand name, has since become a standard treatment for women with bleeding disorders.

Recommended Reading

March 4, 2022 Columns by Jennifer Lynne

Spreading the Word About Bleeding Disorders in Women and Girls

Women bleed differently than men

Women of childbearing age bleed every month, for instance, which can create problems for those with bleeding disorders. Including women with hemophilia in clinical trials allows researchers to better understand hemophilia’s specific impact on women’s bodies while providing participants access to cutting-edge therapies in development and aiding them in their own healthcare advocacy. In this way, we can ensure that women’s unique needs are met, contributing to a more inclusive and equitable system.

Thankfully, the medical community and researchers have made a slow but significant shift in recent years toward recognizing and addressing the needs of women with hemophilia, including specialized care and more tailored treatments, by gathering data on them in trials. This paradigm shift aims to empower women and provide them with the support they need to manage their condition effectively.

Including women with hemophilia in clinical trials marks a significant step toward addressing the historical neglect we’ve faced. It’s time to break down the barriers and ensure that no one is left behind in the pursuit of better health for all.

The website of the National Hemophilia Foundation has a helpful resource about clinical trials. To find ones on bleeding disorders, you may visit the ClinicalTrials.gov website and search for relevant keywords, such as “hemophilia” or “von Willebrand disease.”

Have you been curious about clinical trials? Please share in the comments below. 

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.