Do you worry about
breakthrough bleeds?
DESPITE ADVANCEMENTS
IN THERAPY
51%
had 1 or more spontaneous bleeds during 18 months of follow-up
based on a study of 70 people on non-factor therapy that works like factor 8b
Every bleed
matters
Joint bleeds can lead to joint damage, long-term pain, and reduced mobility, and can impact your activities
bIn a study conducted by the Israeli National Hemophilia Center, 70 people with hemophilia A who had received prophylactic factor 8 mimetic therapy. The study analyzed the number of spontaneous and traumatic bleeds over an 18-month follow-up period.
In another study with factor therapy prophylaxis over the course of a yearc
cFrom July 2023 to February 2024, people with hemophilia A and their doctors took a survey about managing hemophilia and the challenges with current treatment. In total, 62 doctors and 348 patients with hemophilia A completed the survey. Among the patients with bleeding data of more than 1 year, 28 were using EHL treatment, and 40 were using SHL treatment. This study only included patients receiving hemophilia care, which may not reflect the full range of patient experiences, and since it looks at data from a single point in time, we are unable to observe how health conditions might change over time.
Why do breakthrough bleeds happen?
People with hemophilia A do not have enough Factor 8, a protein that helps blood clot. Without enough Factor 8, the body cannot make stable clots, so bleeding may happen, even when on prophylaxis treatment. That is why the primary goal of today’s hemophilia treatments, from Factor 8 replacement to non-factor therapies, is to prevent bleeds by restoring thrombin levels in the normal range.
What is thrombin and why is it important?
Like Factor 8, thrombin is a key protein in the clotting cascade. Factor 8 helps generate thrombin which helps to build stable clots. Stable clot formation is important because it is your body’s way to help prevent bleeding. Since people with hemophilia A can't make enough Factor 8, they can't generate enough thrombin to effectively form clots and prevent bleeds. The goal of hemophilia care is to restore thrombin to normal levels.
ONGOING RESEARCH IN BLEED PROTECTION
Scientists are exploring ways to optimize treatments so they can help people with hemophilia A generate a normal amount of thrombin. A normal amount of thrombin can help people with hemophilia A with or without inhibitors to effectively form clots.
SIMPLIFIED CLOTTING CASCADE
Are you reporting all of your bleeds?
Results show that some bleeds may not be reported to doctors. A study found that people reported more bleeds in a survey than what was recorded in their medical records.d
Take control of your hemophilia A by reporting every bleed—including minor ones, and talk with your doctor about the damage even microbleeds may cause.
dA study asked people with hemophilia how many bleeds they had in the last 2 weeks, and then checked if the answers matched what was recorded in their medical records. These results might show that some bleeds are not always reported to the doctor. Limitations of this data may include patients’ willingness to report their bleeds, whether patients’ accurately recalled their number of bleeds, or the correctness of their medical records.
David lives with
hemophilia A.
Your prophylaxis treatment should be
Helping you prevent bleeds
Some people have admitted to limiting activities to reduce bleeds, according to a survey.
Is this true for you? Share your concerns with your care team if you’ve faced similar challenges.
Speak up about your
bleed concerns
Reduced physical activity may lead to further limitations
Reduced physical activity can lead to weight gain, which may then lead to an increased load on joints. That load can then be followed by further bleeds and mobility problems.
Even with treatment, daily life can still feel limited
WHAT WOULD FEWER LIMITATIONS TO YOUR ACTIVITIES MEAN TO YOU?
IN A STUDY, PATIENTS WITH HEMOPHILIA A WITH OR WITHOUT INHIBITORS
ON NON-FACTOR THERAPY THAT WORKS LIKE FACTOR 8 (N=43) REPORTED A
29%
ON FACTOR 8 PROPHYLAXIS THERAPY (N=210) REPORTED A
30%
reduction in ability to do daily activitiese
eSecondary analysis of data collected as part of a repeated cross-sectional, retrospective observational study of adult patients with hemophilia in the US. Data collected as part of the CHESS US data platform were used, encompassing 2019, 2023, and 2024 waves. The analysis was carried out on 655 patients with hemophilia, including those with hemophilia A (N=431). The aim of this analysis was to estimate the indirect and societal cost burden of hemophilia in the United States. Activity impairment was measured by the degree to which hemophilia affected the patient’s ability to do their regular activities, other than working at a paid job, in the last 7 days, rated on a scale of 0 to 10, where 0 is ‘hemophilia had no effect on my daily activities’ and 10 is ‘hemophilia completely prevented me from doing my daily activities’. Limitations included the retrospective, non-interventional design and voluntary participation, which may introduce selection and recall bias.
DIFFERENT WAYS TO TREAT HEMOPHILIA today
Replaces clotting protein that is missing in people with hemophilia A.
The purpose of Factor 8 replacement therapy is to prevent and treat bleeds.
Treatments that work like Factor 8, but don’t replace Factor 8 in the body. By mimicking Factor 8, these treatments help increase the production of thrombin, a protein that plays a crucial role in forming blood clots. It is given with a syringe injection that goes under the skin.
These treatments block proteins that inhibit clotting, which helps restore the balance between the body's clotting and anticoagulant pathways. Current options are injected subcutaneously (under the skin).
Examples of these treatment approaches include anti-tissue factor pathway inhibitors, activated protein C inhibitors, protein S inhibitors, and antithrombin inhibitors.
Because hemophilia A is caused by a change in your genes, gene therapy works by providing a new copy of the gene needed to make Factor 8.
Investigating TREATMENTS FOR TOMORROW
Researchers continue to explore different ways to prevent bleeds
While non-factor therapies have improved options for people living with hemophilia A, researchers are still investigating a few key areas, like:
How therapies work with the clotting cascade: Can therapies be more effective at helping clots form when needed?
How therapy is administered: Are there ways to make treatments more manageable, like fewer injections or different methods of administration?
How much and how often treatments are dosed: Could therapies be adjusted so you don’t need as many doses or as much product?
Why is this evolution important? Many people desire less frequent dosing, and you may feel the same.
Researchers are also exploring anticoagulation inhibition, which aims to block different proteins to support the body’s natural clotting process. Gene therapy is being studied as a way to provide a new copy of the gene needed to make Factor 8.
These efforts reflect a strong commitment to improving overall care for those living with hemophilia A.
What other changes could improve your treatment experience? Sharing your thoughts could help shape your future care.
David lives with
hemophilia A.
WHAT TREATMENT CHALLENGES ARE HOLDING YOU BACK?
Explore the administration struggles that deserve attention
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