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Chayse, Ashley, Brian, and Chrissy share their stories

admin May 18, 2025

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.

People living with hemophilia A may share a diagnosis, but their experiences living with a bleeding disorder are completely unique. And that includes their treatment journeys. As life and health change over time, treatment goals may change. So they may find they need to switch to a new treatment to achieve their most important goals.

Since 2023, more than 1500 people living with hemophilia A have switched to ALTUVIIIO® [antihemophilic factor (recombinant), Fc-VWF-XTEN fusion protein-ehtl], the #1 most switched-to Factor VIII replacement therapy* that keeps factor activity levels higher (above 40%) for multiple days of the week with one weekly infusion.

Find out why these 4 real ALTUVIIIO patients made the switch, and see if you should talk to your doctor about whether ALTUVIIIO might be right for you:

*Based on specialty pharmacy channel and HTC-reported data from October 2023 to January 2025.

CHAYSE SWITCHED FOR FEWER INFUSIONS

For decades, Chayse used the same factor treatment every other day, and his factor levels were stable. But after his career as a professional musician took off, he realized that his treatment schedule was becoming a real burden.

He did some math and was shocked to realize that in 28 years he’d stuck himself more than 5000 times—more than 180 times a year, on average.

He heard about ALTUVIIIO through the hemophilia community and learned that it was a once-weekly infusion with a 48-hour half-life in adults, which was important both for his lifestyle and his veins. So, he decided to do more research and talk to his doctor.

He says, “If ALTUVIIIO was right for me, that would mean sticking myself as few as 52 times in a year—and less worrying about transporting and packing doses of factor while we travel. Using ALTUVIIIO has simplified my routine, and the peace of mind knowing that I’m covered for longer has made me so happy.”

Check out Chayse’s story.

MOM, ASHLEY, PRIORITIZED PROTECTION

He did some math and was shocked to realize that in 28 years he’d stuck himself more than 5000 times—more than 180 times a year, on average.

Because Ashley’s older brother has severe hemophilia A, she knew there was a possibility that her own children could be born with hemophilia. So she and her husband had medical plans in place when her middle son, 5-year-old Harrison, was born. He was diagnosed at birth with hemophilia, and in the first year of his life, they treated him on demand until they realized he needed more protection. In their search for the right treatment, they tried prophylaxis treatments—both factor and non-factor.

Then Ashley heard about ALTUVIIIO at a conference for bleeding disorders, and Harrison’s doctor supported making another switch. Harrison started treating with ALTUVIIIO in December 2023.

Ashley says, “Switching was worth it. The schedule is a lot more efficient for our life, and he hasn’t had any breakthrough bleeds while on this product. We don’t worry about supplementing with factor like we used to. We spend less time infusing, and more time together.”

ALTUVIIIO was studied over 1 year in the XTEND-Kids study in children under 12 years of age. Routine prophylaxis with ALTUVIIIO resulted in a mean ABR of 0.6 and a median ABR of 0 based on treated bleeds. Efficacy of prophylaxis was evaluated in 72 of these children.^†

Check out Ashley’s story.

^† ALTUVIIIO was studied in XTEND-Kids, which enrolled 74 previously treated male children 1 year to under 12 years of age with severe hemophilia A. The primary goal of the XTEND-Kids study was to determine whether or not children under 12 years of age developed inhibitors to ALTUVIIIO. Zero inhibitors were detected in the 74 children.

BRIAN WANTED ONE PRODUCT

Brian became involved in the hemophilia community in his senior year of high school, learning about the history of the community and the newest treatments and studies. At the time, he was treating with a factor mimetic therapy and supplementing regularly with factor.

Armed with new perspective and knowledge about available treatment options, Brian decided he wanted to use a single product and it needed to be factor. So, as soon as he heard about ALTUVIIIO, he talked to his doctor and made the switch in August 2023.

Remembering that time, Brian says, “Because I wasn’t having any significant issues with my previous treatment, people would ask me, ‘Why switch?’ To some people, it may have seemed like a change that wasn’t necessary. I think it’s important to talk to your healthcare team about changing treatment simply because it isn’t right for you at this stage of your life.”

His passion for advocacy has become a future career. He’s currently pursuing a master’s degree in public health, with a focus on health policy. And he leaves us with this reminder: “You are your own best advocate. Hemophilia is not a one-size-fits-all diagnosis.”

Check out Brian’s story.

CRISSY NEEDED TO CONTROL HER BLEEDS

Chrissy is a critical care nurse with mild hemophilia A, who struggled since childhood to get a medical diagnosis. As a result, she had a tough time controlling her bleeds. She also experienced long-term injuries, and hemorrhaged during childbirth despite warning her doctor about her physical symptoms and status as a person who carries the hemophilia gene. After years of trying to get the right medical attention, she was discouraged and felt depressed.

Finally, she was able to make an appointment with a new hematologist who is also a woman with hemophilia. She underwent genetic testing and was diagnosed with mild hemophilia A. With an official diagnosis in hand and treatment goals in mind, she searched for the right treatment. She tried both factor and non-factor therapies, but she was bleeding frequently and couldn’t get control of it. She needed to get a handle on frequent bleeds she suffered due to her physically demanding career.

Then, her brother started using ALTUVIIIO, and her hematologist recommended that she give it a try. She started on ALTUVIIIO in April 2023. She says, “I have been on ALTUVIIIO for over a year now, and I infuse ALTUVIIIO once weekly. I like only having to use one product and being able to make it through my work week without getting bleeds as often.”

ALTUVIIIO was studied in 159 adults and adolescents (12 years and older) with severe hemophilia in the XTEND-1 study. Routine prophylaxis with ALTUVIIIO resulted in a mean ABR of 0.7 and a median ABR of 0 based on treated bleeds. Efficacy was evaluated in 128 of these patients.

Check out Chrissy’s story.

Now that you’ve met real ALTUVIIIO patients and learned how it’s helped them reach their treatment goals, you may be considering talking with your doctor about making the switch. You can sign up to speak with an ALTUVIIIO Peer Mentor about their experiences switching. And, our Doctor Discussion Guide can help. It offers a list of questions to jump-start the conversation with your doctor.

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 and joint 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.

Please consult your healthcare professional if you have any questions about your health or treatment.

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Information about Hemophilia and Gene Therapy

admin March 30, 2025

This is a paid public announcement from CSL Behring 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 the CSL Behring website. LA Kelley Communications always advises you to be a savvy consumer when contacting any company; do not reveal identifying information against your will.

About Hemophilia
Hemophilia is a rare disorder in which blood doesn’t clot in the typical way because it doesn’t have enough blood-clotting proteins (clotting factors).

The two most common types of hemophilia are categorized as types A and B. These conditions are often inherited, occurring because of gene variations that affect different blood clotting factors. Both have similar symptoms, including bruising and bleeding into joints, which causes stiffness, pain, and mobility issues, and bleeding after procedures such as dental surgery.

Hemophilia is considered a good candidate for gene therapy because it is a monogenic disorder (which means it is caused by an alteration to a single gene) and even a minimal increase in clotting factor can significantly improve quality of life.

About Gene Therapy
Although the concept of gene therapy is not new, it is an innovative treatment option compared to traditional therapies as the first gene therapy was approved by the U.S. Food and Drug Administration (FDA) in 2017. As of 2024, there are 38 cellular and gene therapies approved by the FDA. These therapies cover a range of conditions, from certain types of cancers to genetic disorders such as hemophilia.

How Gene Therapies Work for Hemophilia
Gene therapies for hemophilia introduce a working, or functioning, gene into the body, which allows people with hemophilia to produce their own factor, which can lower the risk of bleeding and reduce or eliminate the need for ongoing, regular prophylactic treatment.

Candidate Screening for Gene Therapy
There are various factors involved in understanding if someone may be the right candidate for gene therapy, including current course of treatment, the severity of the condition, and the types of bleeding episodes one may experience. There are also pre-screening tests to check overall liver health and possible adeno-associated virus (AAV) neutralizing antibody levels. If a person tests positive for AAV neutralizing antibodies, they should discuss their gene therapy treatment options with their doctor.

How Gene Therapies for Hemophilia Are Administered
These gene therapies are administered via a one-time IV infusion, which takes about one to two hours, or longer depending on the treatment. Once administration is completed, patients will stay for monitoring for at least three hours. Following administration, regular lab tests are required to track progress including overall health, liver function, and factor activity level. These can often be completed at a local lab.

Cost of Gene Therapies for Hemophilia
Cost for treatment will vary based on coverage policies and eligibility for financial assistance programs. Payers covering the vast majority of the U.S. population have established clear medical policies for gene therapy. Additionally, manufacturers offer copay assistance programs, which may allow people with commercial insurance to pay $0 out-of-pocket for treatment.

Interested in watching real-world stories from people living with hemophilia B who have received gene therapy? Visit BeyondHemB.com

From Disability to Doctor: Cristian of Romania

admin December 1, 2024

My name is Cristian Dragusin and I am a person with severe hemophilia from Romania. I am 25 years old and have recently graduated medical school. Things are looking up for my life but there were moments in the past when I could have never seen myself achieve everything I did. It was only with the support of the people around me that I was able to get this far and look forward to the future, in spite of my condition.

My early childhood was not as affected by my disorder as the later years, with only some occasional bleeding episodes. As I started school, however, the bleedings became more frequent and debilitating, the right elbow being the first target joint. Access to treatment was limited because I developed inhibitors, and there was no prophylaxis to speak of. The pain would keep me up at night and treatment often only consisted of cold compresses.

When I was 12, I had a severe episode that involved both my knees. I was in and out of the hospital for several months and there was long lasting damage. Around that time, my parents divorced and my mother was left alone to take care of me with help from my grandparents. The frequent and long periods of hospitalization were difficult to say the least. It was also at that age when I went to my first hemophilia camp, where I got to meet and befriend many other children like me. I was using a wheelchair when camp first started and, by the time I returned home, thanks to the treatment I received, I could walk with crutches. Every year afterwards I would look forward to Camp Ray of Hope, the place where I could move and have fun without worries.

The second turning point was in high school, when I had another severe event involving my knees. There were times when not even the hospital had any treatment available. Both of my knees were now permanently affected and I couldn’t walk without crutches anymore. This was the most hopeless time of my life.

As high school was nearing its end, I had to choose what I was going to do next. After long considerations and changes of heart, medical school seemed to be the path for me. Working in this field was something that just resonated with me after all the time spent in the hospital. And by the time high school ended, I finally gained access to prophylaxis and the number of bleeds decreased drastically: better later than never.

University came with its own set of problems. Courses and rotations were often in different parts of the city and there were many costs associated with school and living in a different place on my own. It was thanks to my family and the support offered by Save One Life that I managed to finish it without a hitch. The sponsorships and scholarships I received from Save One Life over the years were especially of great help in easing the financial burden.

Looking back, the journey to this point in time wasn’t the happiest so I am extraordinarily grateful to the people that were by my side, and especially to my mother, who has done everything in her power to make my life easier. Now I have proper access to treatment and will start working in a couple of months, a scenario which was merely a dream years ago, and I intend to live this dream to the fullest.

The Hemophilia Viking

admin November 20, 2024

There are a lot of colorful characters in the hemophilia community—and competitive athletes. Put them together you have Cobus Visser.

Cobus is from South Africa, has hemophilia and has quite a story to tell. From our wonderful archivist, Richard Atwood of North Carolina, comes a review of Cobus’s new book, Walking Through Fire. Read on and get motivated!

A quick bio: Cobus was born on November 2,1982 in Vereeniging, South Africa, to Theunis and Ansie Visser. He had a Christian upbringing. His great-grandfather was Charles Robert Swart, the first state president of the Republic of South Africa (1961-1969). Bruises at three months led to a diagnosis of hemophilia A. Both Cobus and his brother, Riann, had hemophilia; Pierre, the third brother, did not. Cobus matriculated from Hoerskool Piet Potgieter in Potgietersrus, Limpopo. He earned a BBA in Marketing Management from IMM Graduate School, and is currently working on an MBA in Marketing from the University of Edinburgh. In 2005, Cobus married Mar-nelle, and the couple now have two sons: Tiann, born in 2009, and Wihan, born in 2014.

Growing up in South Africa in the 1980s wasn’t easy. Cobus attended a hemophilia clinic and had numerous hospital stays in Johannesburg due to bleeds in his ankles, knees and elbows. He did not play sports. He grew tall and became heavy, weighing over 260 pounds, and faced physical limitations. At age seven, Cobus learned how to self-infuse to treat bleeds. The product was plasma-based. A brain bleed in 1996 led to migraines and a coma, including an ICU stay, and three operations on the right side of his head. This was followed by subsequent brain bleeds in 2008 and 2015, and later an epileptic attack in 2017 that was treated with medicine.

In 2003, Cobus underwent radioactive utrum for his joints. He took Vioxx for pain and creatine for strength. The prognosis was to fuse both ankles and replace both elbows. Even with physiotherapy, Cobus relied on crutches and wheelchairs for mobility. A diagnosis of hepatitis C in 2012 led to injections of Copegus and Pegasys for 12 months. His father Theunis retired from the police force and opened a family business, called Furn4U/Modern Living, in Pretoria. Cobus’s younger brother Pierre already owned a dozen gyms. Cobus was expected to eventually run the family business, yet brother Riann would assume that role, when Cobus quit, in 2014, to be self-employed.

Starting in 2011, Cobus took courses in Mind Power, Sales Explosion, Unleash the Power, and NLP. He took the Firewalking Instructor Training (FIT) course, and by 2014 was the only Master Firewalking Instructor in Africa!

Cobus became a speaker, trainer, facilitator, life coach, mentor, author of three books, and instructor. He learned to walk on hot embers and shards of glass. In 2016, Cobus had an idea: to climb Mount Kilimanjaro in Tanzania on crutches! But how to fund the $3,300 to $4,300 fee? The hemophilia clinic doctors would not sign the medical clearance certificate. Cobus, suffering from depression, planned to let the mountain take his life. After two years of preparation, eight team members, plus 31 guides and porters, climbed Mount Kilimanjaro from July 9-18, 2018. Cobus injected 6,000 IU of factor every morning. The crutches chafed his underarms. He walked on the front of his feet and toes, which led to calf cramps. Stomach cramps and diarrhea complicated matters. He could not acclimatize, and suffered altitude sickness. He hired a personal porter for an extra fee to carry his bags and day pack. Cobus reached Stella Point at 18,884 feet on crutches, though never achieving the summit on Uhuru Peak. He then descended in freezing temperatures, facing altitude sickness, joint pain, and delirium. The porters carried Cobus on a stretcher, then used a “Kili Taxi” (a wooden wheelbarrow stretcher) before a helicopter flew him to a hospital for a short stay. Cobus left the mountain with a certificate and renewed zest.

Cobus eventually expanded his business, and in 2022, rebranded himself from “Superman of Africa” to “The Viking.” Cobus climbed Mafadi (11,305 feet), the highest peak in South Africa. He had a major medical procedure in 2019, then a severe case of Covid-19 in 2021. But nothing seems to keep him down for long. Cobus is recognizable for his height, hair, persona, smile, Afrikaans accent, and three-second hugs. And don’t forget— his exceptional skill in fire walking and glass walking!

You can order Walking Through Fire in Amazon.com. You can learn more at https://cobusvisser.com

Cobus Visser, 2024, Walking Through Fire. Centurion, South Africa. 289 pages.

Women and hemophilia: evolving knowledge, raising awareness, and sharing stories

admin October 27, 2024

Thanksgiving is all about quality time with loved ones. This year, November 28 is also National Family Health History Day, which encourages families to get together and share health experiences. Stories from relatives can help us better understand our own health risks—and its helpful information to share with your care team. It reminds me why it’s so crucial to share stories with each other. As we approach the 35th anniversary of my book Raising a Child with Hemophilia, which included stories from 125 families in the hemophilia community, I want to continue the tradition here! In this blog, Sanofi introduces you to a woman living with hemophilia, Kyrie, who shares how hearing about her mother and grandmother’s experiences with hemophilia shaped her own journey of self-advocacy.

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.¹This 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 how her family’s experience with hemophilia inspired her to get the care she needs, and how she’s empowering other women to do the same.

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

Photo caption: Kyrie and her grandmother, who was treated for hemophilia at a time when it was uncommon for women to be diagnosed.

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 credits 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. Hearing her grandmother’s story became her guiding light. “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. Her mother knew how women in the family experienced hemophilia, empowering her to have effective conversations with Kyrie’s doctor to get the care her daughter needed.

“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.” While opening up about hemophilia can feel vulnerable, it also creates space to inspire others to better manage their health, too.

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. If hemophilia runs in the family, it’s even more important to watch for these symptoms to help ensure all women get the care and support they need.

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. Make it a resolution to prioritize health in the new year and reach out to your doctor to start conversations about your care.

To learn more about women and hemophilia, including how it’s inherited, how it presents, and how to be your best self-advocate, 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

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