Sanofi

Women and hemophilia: evolving knowledge and raising awareness

admin June 4, 2023

I love sharing stories from our community, and have been a voice for hemophilia patients since 1990, when we published Raising a Child with Hemophilia. It included stories from 125 families, the first ever such publication. Let’s continue that tradition! In this blog, Sanofi introduces you to a woman with hemophilia, Kyrie, who shares her unique story.

Sponsored by Sanofi

While hemophilia is more common in men, women can also experience symptoms and receive a diagnosis. Because of a traditional misconception that women are only carriers of hemophilia, they have historically not been tested and treated for the condition.¹ This is changing, thanks to emerging understanding and awareness of hemophilia in women.

Kyrie Smith lives with hemophilia and works as a Community Relations and Education (CoRe) manager at Sanofi. We sat down with her to hear about her hemophilia journey.

Please note that Kyrie’s story is hers alone. While there are common threads, everybody’s experiences with hemophilia will be different.

A legacy of advocacy

Kyrie’s family history with hemophilia goes back to her grandmother. According to Kyrie, her grandmother was one of the first women to receive access to on-demand factor treatment for bleeds as they occurred. Kyrie credit’s her grandmother’s self-advocacy with helping her get treatment at a time when it was uncommon for women to be diagnosed, let alone treated.

“The ability for my grandmother to be prescribed factor to control a bad leg bleed was quite novel at the time,“ Kyrie says. “While she is no longer with us anymore, I often find myself thinking about her strength and resilience.”

Hemophilia affects both women and men in Kyrie’s family. Since Kyrie was a carrier of hemophilia and could become symptomatic, her mother advocated for Kyrie’s factor activity levels to be checked during her brother’s annual appointments.

“I’m thankful for my mother’s persistence, as I was diagnosed so early in my childhood, when many women— including my mother—are often diagnosed later in life,” Kyrie says. “I am very proud to come from a long line of strong women who have advocated for the health of their family as well as themselves.”

Strength in sisterhood

Kyrie was first diagnosed as a symptomatic carrier of hemophilia and was prescribed a treatment to address bleeds and other symptoms as they occurred. A few years later, her diagnosis was updated to mild hemophilia.

“This change was due to the strong women and advocates in the hemophilia community that helped evolve the definition of symptomatic carrier to mild hemophilia, which is defined by factor activity levels,” Kyrie says. “I am honored to now join them in their work to increase visibility and voices of women with hemophilia.”

By working with her healthcare team, Kyrie has been able to manage her condition. That doesn’t mean, however, that the situation is without its challenges.

“From missing sports practices in high school to switching cardio workouts to avoid overworking my joints and managing pain, I’ve had to make some compromises along the way,” Kyrie says.

She’s grateful for the support and encouragement she’s received from the hemophilia community to help her get through difficult times. Kyrie started attending hemophilia camps at age 7, an experience she considers central to her continued sense of community.

“By having a mentor to look up to, or a peer to talk through an issue with, I’ve made lifelong friends from my time at hemophilia camps and other chapter or national events,” says Kyrie.

When to start asking questions

Awareness, testing, and diagnosis of hemophilia in women are increasing.¹ It can be difficult, however, to know whether the bleeding you experience is normal or abnormal. When in doubt, it’s best to speak to your doctor to discuss what’s going on.

The common signs of hemophilia in women include:²

Heavy menstrual periods, such as soaking through one or more pads or tampons every 2 hours or less³
Low in iron or have anemia
Frequent nosebleeds that last longer than 10 minutes
Bleeding from cuts lasting longer than 5 minutes
Easily bruised (raised and larger than a quarter, happening on a weekly basis)
Joint bleeds (pain, swelling, unusual sensations, warmth, loss of motion)
Family history of a bleeding disorder
Heavy bleeding from surgery

If you or someone you care about is experiencing the signs and symptoms above, consider reaching out to a doctor to explain what’s happening and get more information.

Coming full-circle

Kyrie now works as a Sanofi CoRe, which gives her the opportunity to pay forward the information and connection she has experienced from within the hemophilia community.

“Because the hemophilia community has played such a large role in my life, I want to continue giving back, being a resource for the next generation,” Kyrie says. “As a CoRe, I am able to connect with various members of the community no matter where they might be in their hemophilia journey. It could be a new parent navigating hemophilia care for their child, or it could be a young adult working to manage their hemophilia care independently.”

While everyone’s experiences with hemophilia are unique, Kyrie’s personal path gives her extra insight into what members of the community are going through.

“When I have the opportunity to connect with a young girl who is having hemophilia symptoms and needs to advocate for herself, I can really relate because I’ve been in her shoes,” Kyrie says. “The road to diagnosis and treatment can be long and confusing, but there are so many reasons to keep going and so many people to help you do just that. I hope by sharing my story, others will too, and together, we can continue empowering the hemophilia community to advocate for their care and supporting those in the earliest stages of their hemophilia journey.”

If you have a family history of hemophilia or you’re experiencing symptoms, you’re not alone. Reach out to your doctor for more information.

To learn more about women and hemophilia, including how it’s inherited, how it presents, and options for treatment, visit RedefiningHemophilia.com.

This is a paid public announcement from Sanofi and does not constitute an endorsement of products or services. When you click on the links in this blog entry, you will be directed to a Sanofi website. LA Kelley Communications always advises you to be a savvy consumer when contacting any company; do not reveal identifying information against your will.

“Women can have hemophilia, too.” Centers for Disease Control and Prevention, https://www.cdc.gov/ncbddd/hemophilia/features/women-and-hemophilia.html
“Women and bleeding disorders.” National Hemophilia Foundation, https://www.hemophilia.org/bleeding-disorders-a-z/overview/women-and-bleeding-disorders
Menorrhagia (heavy menstrual bleeding).” Mayo Clinic, https://www.mayoclinic.org/diseases-conditions/menorrhagia/symptoms-causes/syc-20352829

MAT-US-2303664-v1.0-05/2023

Discover a treatment that could change how you manage your hemophilia A

admin May 28, 2023

This is a paid public announcement from Sanofi and does not constitute an endorsement of products or services. When you click on the links in this blog entry, you will be directed to a Sanofi website. LA Kelley Communications always advises you to be a savvy consumer when contacting any company; do not reveal identifying information against your will.

If your life has been touched by hemophilia A, you know it can be full of unknowns. Bleeds can be painful, happen without warning, and limit physical activity, which is why it’s critical to stay on top of them. Sanofi launched ALTUVIIIO [antihemophilic factor (recombinant), Fc-VWF-XTEN fusion protein-ehtl]—a hemophilia A Factor VIII replacement therapy that provides proven bleed protection.

Here are 4 reasons to consider ALTUVIIIO:

1. HIGHER FACTOR LEVELS THAT LAST LONGER
With just one weekly ALTUVIIIO infusion, factor levels remain in the near-normal to normal range (>40%) for most of the week and stay above 18%,^* on average, in adults.

*Average trough levels were 18% for adults 18 years and older, 9% for adolescents aged 12 years to under 18 years, 10% for children aged 6 years to under 12 years, and 7% for children aged 1 year to under 6 years.

^‡This is information from a study of 13 previously treated adults with severe hemophilia A that had the goal of comparing how long ALTUVIIIO, Adynovate^® [Antihemophilic Factor (Recombinant), PEGylated], and Advate^® [Antihemophilic Factor (Recombinant)] stayed in the body after 1 dose. Half-life was 43 hours for ALTUVIIIO, 15 hours for Adynovate, and 11 hours for Advate.
Adynovate and Advate are registered trademarks of Baxalta Incorporated, a Takeda company.

EHL=extended half-life; SHL=standard half-life.

ALTUVIIIO offers not only weekly prophylaxis use, but also on-demand bleed control and perioperative management. Regardless of how you use it, you can expect the same infusion process.

2. STUDIED—AND PROVEN—BLEED PROTECTION
Before we dig into the numbers, it’s helpful to know how ALTUVIIIO was studied and to understand its safety profile.

For one year, the XTEND-1 study looked at treatment in 159 adults and adolescents. Participants were divided into 2 groups. Both groups used mean and median annualized bleed rates (ABRs) to evaluate the efficacy of ALTUVIIIO. Finding people’s mean ABR was the primary goal of the study.

Safety evaluated in 159 people taking ALTUVIIIO in the XTEND-1 study showed that:

21% of people had headache (33 people)
16% of people had joint pain (26 people)
6% of people had back pain (9 people)

In 67 children taking ALTUVIIIO in the ongoing XTEND-Kids study:

1% of children had headache (1 child)

In XTEND-1 and XTEND-Kids, people taking ALTUVIIIO had:

0 inhibitors
0 serious allergic reactions

Although no inhibitors were found, and no serious allergic reactions occurred in clinical studies, inhibitors and serious allergic reactions are possible with ALTUVIIIO.

Group 1
This group consisted of 133 people aged 12 years and older who had prior prophylaxis therapy and switched to ALTUVIIIO weekly prophylaxis. This group included 1 female participant. Efficacy of prophylaxis was evaluated in 128 of these patients.

The primary outcome showed a mean of <1 (0.7) bleeds per year (the median ABR was 0).^‡

Here’s a look at how the study measured bleed rates:

Median ABR was the middle number of all ABRs, when ABRs were ordered from least to greatest
Mean ABR was the average number based on everyone’s ABRs

It’s also worth noting that 78 of the people in Group 1 participated in a separate study to measure their ABRs on their prior prophylaxis. These 78 people went from 3 bleeds to less than 1 bleed a year. That’s a 77% reduction in yearly bleeds!^‡

Group 2
People in this group (28 participants) switched to ALTUVIIIO on demand from prior on-demand therapy for 26 weeks, and then were treated with ALTUVIIIO prophylaxis for another 26 weeks.

This group also saw striking results. On average, people who switched from ALTUVIIIO on demand to ALTUVIIIO prophylaxis went from 21 bleeds to less than 1 bleed a year (mean ABR 0.7). That’s a 97% mean reduction in yearly bleeds.^‡

And over the 26 weeks on ALTUVIIIO prophylaxis, 77% of people had 0 bleeds.^‡

Both groups showed significant improvement in bleed protection with ALTUVIIIO prophylaxis.

^‡Data based on treated bleeds.

3. FEEL CONFIDENT YOUR JOINTS ARE PROTECTED

The XTEND-1 study also examined target joint bleeds. When evaluating joint results at 52 weeks in the 128 people who participated in the XTEND-1 study, 72% of people had 0 joint bleeds‡ on prophylaxis after switching to ALTUVIIIO. 100% of target joints were resolved.

Target joints:

Are 3 or more spontaneous bleeds in a major joint within a period of 6 consecutive months
Were considered resolved if 2 or fewer bleeds occurred in the target joint within 12 months

4. THE FEWEST WEEKLY INFUSIONS AMONG FACTOR VIII PROPHYLAXIS TREATMENTS

While most people with hemophilia grow accustomed to infusing, fewer infusions are generally preferred. In studies, ALTUVIIIO clearly outlasted other Factor VIII replacement therapies, meaning ALTUVIIIO takes longer to be reduced by half in the body, and therefore lasts for a longer period.

So instead of needing up to 4 infusions a week with other treatments, patients on ALTUVIIIO infused only once per week.

ALTUVIIIO offers the fewest weekly infusions among Factor VIII prophylaxis treatments.

^†This is information from a study in 13 previously treated adults with severe hemophilia A that had the goal of comparing how long ALTUVIIIO, Adynovate^® [Antihemophilic Factor (Recombinant), PEGylated], and Advate^® [Antihemophilic Factor (Recombinant)] stayed in the body after 1 dose. Half-life was 43 hours for ALTUVIIIO, 15 hours for Adynovate, and 11 hours for Advate.
^§Doses and dosing intervals may be adjusted.
Adynovate and Advate are registered trademarks of Baxalta Incorporated, a Takeda company.

Now that you’ve learned about a few of the ways ALTUVIIIO protects you from bleeds, you may be considering a conversation with your doctor about your treatment plan. Our Doctor Discussion Guide can help. It offers a list of helpful questions to help you jump-start the conversation. You can also connect with your local Sanofi Community Relations and Education (CoRe) Manager, who can share additional resources and provide education.

INDICATION
ALTUVIIIO™ [antihemophilic factor (recombinant), Fc-VWF-XTEN fusion protein-ehtl] is an injectable medicine that is used to control and reduce the number of bleeding episodes in people with hemophilia A (congenital Factor VIII deficiency).

Your healthcare provider may give you ALTUVIIIO when you have surgery.

IMPORTANT SAFETY INFORMATION

What is the most important information I need to know about ALTUVIIIO?
Do not attempt to give yourself an injection unless you have been taught how by your healthcare provider or hemophilia center. You must carefully follow your healthcare provider’s instructions regarding the dose and schedule for injecting ALTUVIIIO so that your treatment will work best for you.

Who should not use ALTUVIIIO?
You should not use ALTUVIIIO if you have had an allergic reaction to it in the past.

What should I tell my healthcare provider before using ALTUVIIIO?
Tell your healthcare provider if you have had any medical problems, take any medications, including prescription and non-prescription medicines, supplements, or herbal medicines, are breastfeeding, or are pregnant or planning to become pregnant.

What are the possible side effects of ALTUVIIIO?
You can have an allergic reaction to ALTUVIIIO. Call your healthcare provider or emergency department right away if you have any of the following symptoms: difficulty breathing, chest tightness, swelling of the face, rash, or hives.

Your body can also make antibodies called “inhibitors” against ALTUVIIIO. This can stop ALTUVIIIO from working properly. Your healthcare provider may give you blood tests to check for inhibitors.

The common side effects of ALTUVIIIO are headache, joint pain, and back pain.

These are not the only possible side effects of ALTUVIIIO. Tell your healthcare provider about any side effect that bothers you or does not go away.

Please see full Prescribing Information.

MAT-US-2209658-v1.0-05/2023

5 things to know about hemophilia, factor activity, and exercise

Laurie Kelley February 5, 2023

Sponsored by Sanofi

With 2023 now under way, getting more exercise is probably on a lot of people’s minds. It’s a great time to make new plans to get moving! For people living with hemophilia, there are extra considerations to make sure you can safely participate in physical activities. Below are 5 things to know about exercising when you have hemophilia—and how factor activity levels play a role. Remember that you should always speak to your doctor before starting any new type of activity.

Exercise is important for people living with hemophilia. Engaging in physical activity is associated with both physical and mental health benefits¹—and that goes for people who have hemophilia, too. In fact, exercising can improve joint health and reduce joint bleeds.² Hemophilia severity, factor activity, and other personal health considerations may all affect what kinds of exercises you and your doctor decide are the best fit for you.
Factor activity levels determine hemophilia severity. Your factor activity level is how much clotting factor you have in your blood at a given time. These levels are used to help determine how severe your hemophilia is at the time of diagnosis.³ Higher factor levels in your body over time means better bleed protection. Factor activity levels and severity may affect the amount of risk that comes with certain forms of exercise.⁴
- Categories of hemophilia severity and how they affect lifestyle,
  - Normal (non-hemophilia range)^{5, 6, 7, 8, 9}
  - Factor activity: 50% to 150%*†
  - Lifestyle: normal
  - Activity: High impact activity possible with no pain
- Near normal (non-hemophilia range)
  - Factor activity: >40% to <50%*†
  - Factor activity levels between 40-50 percent are currently undefined by World Federation of Hemophilia guidelines
- - Factor activity: >5% to <40%*†
  - Lifestyle: minor adjustments
  - Activity: limited activity with some pain and risk of spontaneous or micro bleeds
  Moderate hemophilia^6,10
  - Factor activity: >1% to 5%*†
  - Lifestyle: significant adjustments
  - Activity: limited activity with some pain and risk of spontaneous or micro-bleeds
- Severe hemophilia^6,10
  - Factor activity: <1%*†
  - Lifestyle: vulnerable
  - Activity: high risk of spontaneous bleeds with low activity and pain with target joints
Keep your hemophilia severity in mind when choosing types and levels of exercise. There are all kinds of ways to get exercise, depending on your hemophilia severity, your preferences, and your doctor’s recommendations. For example, people with moderate hemophilia may be better off with lower risk activities such as swimming, walking, or stationary biking.¹¹ People with mild hemophilia may be able to participate in activities with more moderate risk, like weight lifting, hiking, rowing, or snorkeling.¹¹
There are also many ways to get exercise that aren’t the recreational activities that might immediately come to mind. Everyday activities like gardening or cooking are good ways to move your body and be productive at the same time.¹² While there are many benefits to exercise and many ways to get active, no activity is completely risk free. Work with your doctor to choose the best kind of exercise for you.
Treatment may affect factor activity and lifestyle. When your factor levels stay above 40% (in the near-normal to normal non-hemophilia range), you may be able to engage in activities longer.^7,8,9 This is why many people look for treatments that improve bleed protection to allow them to spend more time doing the activities they enjoy. You may want to talk to your doctor about what your treatment is doing for your factor activity levels and how that may impact your lifestyle.
Every person is unique. No two people living with hemophilia are identical. There are differences between hemophilia A and B, everybody’s factor activity levels are different, and everyone’s body reacts differently to exercise. Talk to your doctor to explore the options that are right for you. No matter what you decide together, it’s a great time to start the conversation.

To learn more, visit Levels Matter, a Sanofi website dedicated to helping people understand factor activity levels, how blood clots, how hemophilia is inherited, and more. The more you know, the better you will be equipped to make the right decisions for your lifestyle.

*Severity classifications may be different for women with hemophilia
†Factor levels may not reflect bleeding patterns

REFERENCES

Centers for Disease Control and Prevention. Benefits of physical activity.
https://www.cdc.gov/physicalactivity/basics/pa-health/index.htm.
Harris S, Boggio LN. Exercise may decrease further destruction in the adult haemophilic joint.
Haemophilia. 2006;12(3):237-240. doi:10.1111/j.1365-2516.2006.01214.x
Centers for Disease Control and Prevention. What is hemophilia
https://www.cdc.gov/ncbddd/hemophilia/facts.html.
World Federation of Hemophilia. WFH guidelines for the management of hemophilia.
https://www1.wfh.org/publications/files/pdf-1863.pdf.
National Hemophilia Foundation. Hemophilia A.
https://www.hemophilia.org/bleeding-disorders-a-z/types/hemophilia-a.
Martin AP, Burke T, Asghar S, Noone D, Pedra G, O’Hara J. Understanding minimum and ideal factor levels for participation in physical activities by people with haemophilia: An expert elicitation exercise.
Haemophilia. 2020;26(4):711-717. doi:10.1111/hae.13985
Iorio A, Iserman E, Blanchette V, et al. Target plasma factor levels for personalized treatment in haemophilia: a Delphi consensus statement. Haemophilia. 2017;23(3):e170-e179.doi:10.1111/hae.13215
Skinner MW, Nugent D, Wilton P, et al. Achieving the unimaginable: Health equity in haemophilia. Haemophilia. 2020;26(1):17-24. doi:10.1111/hae.13862
Weyand AC, Pipe SW. New therapies for hemophilia. Blood. 2019;133(5):389-398. doi:10.1182/blood-2018-08-872291
Centers for Disease Control and Prevention. Diagnosis of hemophilia.
https://www.cdc.gov/ncbddd/hemophilia/diagnosis.html.
National Hemophilia Foundation. Playing it safe.
https://stepsforliving.hemophilia.org/sites/default/files/playing-it-safe.pdf#overlay-context=resources/physical-activity.
World Federation of Hemophilia. Psychosocial Care for People with Hemophilia.
https://www1.wfh.org/publication/files/pdf-1198.pdf.