Uncategorized

See updated study results for kids with hemophilia A.

admin August 18, 2024

Treatments for hemophilia continue to evolve, making a positive impact on those living with the disease—including children. That’s why we’re excited to discuss the recently released complete data from XTEND-Kids, the pediatric study for ALTUVIIIO® [antihemophilic factor (recombinant), Fc-VWF-XTEN fusion protein-ehtl]. ALTUVIIIO is a once-weekly Factor VIII replacement therapy that provides bleed protection and high sustained factor levels above 40% for ~3 days in kids with hemophilia A.

Before we dive into the benefits and safety of ALTUVIIIO, let’s explore the XTEND-Kids study and how it was set up. This study was a clinical trial that took place over 1 year and enrolled 74 previously treated male children under 12 years of age who switched to ALTUVIIIO.

36 participants were 6 to <12 years old
38 participants were 1 to <6 years old
Efficacy was evaluated in 72 of these children

The primary goal of the XTEND-Kids study was to find out if children would develop inhibitors to ALTUVIIIO—and 0 inhibitors were detected! Keep in mind that, although none were detected, inhibitors are still possible with ALTUVIIIO.

Now that we’ve covered the study, keep reading to learn about higher-for-longer factor activity levels in kids. (You can also learn about the safety and benefits of ALTUVIIIO in adults and adolescents at ALTUVIIIO.com.)

Kids can achieve high sustained factor activity levels for ~3 days.

One of the more exciting findings from the XTEND-Kids study is that, on average, after the first dose of ALTUVIIIO, factor activity levels stayed in the near-normal to normal range (above 40%) for about 3 days. In the chart below, you can see how long factor activity levels lasted in this range for children who were between the ages of 1 to under 6 (the purple line) and 6 to under 12 (the pink line).

Proven bleed protection with ALTUVIIIO prophylaxis.

Bleed protection was measured over 1 year of ALTUVIIIO prophylaxis. Much like results for adults and adolescents, the study showed rates of less than 1 bleed per year.

How were bleeds and joint bleeds measured in the trials?

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

If a bleed did happen, 95% of bleeding episodes were resolved with just one infusion of ALTUVIIIO.

ALTUVIIIO has an established safety profile, but as with any medication, side effects can occur. In 233 people across multiple studies^†:

15% experienced headache (35 people)
13% experienced joint pain (31 people)
4% experienced fever (10 people)

4% experienced pain in extremities (10 people)
4% experienced back pain (9 people)
3% experienced vomiting (7 people)

In XTEND-1 and XTEND-Kids, people taking ALTUVIIIO had 0 inhibitors OR 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.

^†Clinical trials included adults and adolescents (XTEND-1) and children (XTEND-Kids).

Connect with a community that’s waiting for you.

When you’re part of the ALTUVIIIO community, you’re never alone! The ALTUVIIIO community includes Patient Educators (also known as Community Relations and Education [CoRe] Managers), ALTUVIIIO Peers, Peer Mentors, Therapeutic Education Managers (TEMs), and Case Managers—people who are with you every step of the way!

Your local ALTUVIIIO Patient Educator is a dedicated professional who provides personalized education and can point you toward helpful resources and support—connect with a local Patient Educator today!

If you’re interested in talking to a real patient or caregiver about what life is like with ALTUVIIIO, the Peer Mentor Program will connect you with someone who has had similar experiences. Together, you can discuss their treatment journey and get answers to some of your questions.

Hear from Peers below, see more stories, and sign up to have a conversation with an actual Peer Mentor here.

Meet Ashley and Harrison
Real caregiver and patient

Ashley’s house can be a bit hectic. With 3 kids, 2 of them with hemophilia—it’s a lot to juggle between activities like fishing and T-ball, and treatment schedules. Her family’s goal was better bleed protection with fewer infusions. By switching to once-weekly ALTUVIIIO, Ashley was able to send Harrison off to school knowing that he was better protected. Ashley is a promotional speaker compensated by Sanofi.

Meet Natalie and Samuel
Real caregiver and patient

As Samuel grew up, it became clear to Natalie that they needed a treatment that would protect him from bleeds and also allow him to stay busy. That’s why they were so interested when they heard about ALTUVIIIO, its fewer infusions, and how its higher sustained factor levels are associated with lower bleed risk. Now they have a treatment and a schedule that works for them—it allows Samuel to get the most out of being a kid.

Natalie and Samuel are promotional speakers compensated by Sanofi.

To learn about what ALTUVIIIO can do for your child with hemophilia A, connect with a Patient Educator today!

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.

© 2024 Sanofi. All rights reserved.
ALTUVIIIO and Sanofi are registered trademarks of Sanofi or an affiliate.
MAT-US-2406347-v1.0-07/2024

Richard’s Review: Fictional Family Members

Laurie Kelley January 24, 2021

by Ricard Atwood

What does a typical family in the bleeding disorder community look like? I don’t know. There’s so much variability in family composition that the range of normal seems boundless.

Novelists may base their fictional characters on people they know, or use their imagination to create characters. Some novelists want to make a bleeding disorder an important part of their novel’s plot, yet choose another family member as the leading character. I’m curious about these protagonists—wife, mother, sister of someone with a bleeding disorder—in the following novels.

A Place for Kathy

Henry Denker William Morrow, 1997

Tessa Taylor, 40, has a type-A personality and runs her own organic makeup company. She is married with three kids. Living on the Florida panhandle coast, Tessa hunkers down alone for a Category 4 hurricane, with her family elsewhere. Curious, she snoops on her architect husband Ethan’s iPad and discovers his affair with Lindsey, a 26-year-old nursing student. Tessa tricks Lindsey into coming to her home, even though she has no power and no gas for the generator. The two women fight, verbally and physically, over Ethan—only to discover that he has additional mistresses. During the storm, and in the middle of this fight, Tessa’s eldest child, Colt, who is 18 years old, 6’6″ tall, and 200 pounds, returns from Florida State University because he runs out of meds for his hemophilia. Colt knows that his mother always keeps some in the refrigerator. Colt arrives bleeding profusely from his leg. Both women stop their fighting to care for Colt by hanging blood-clotting factor bags on an IV pole, which I found to be a puzzling way to treat hemophilia. Fortunately, mothers always seem to be prepared, regardless of the age of their child with a bleeding disorder.

Mine

Courtney Cole. Gallery Books, 2019

This melodramatic novel is sure to produce misty eyes, if you overlook some of the contrived storyline. In 1994, Grace Cameron becomes a widow when her husband, Kip, dies unexpectedly from a brain hemorrhage caused by a fall while hiking. Kip taught literature at a midwestern university, but had no life insurance because of his pre-existing condition, hemophilia. Grace learned of Kip’s hemophilia when they first dated, and she knew that he had tested negative for HIV. So in 1996, she is shocked to learn that she is HIV positive. As her medical symptoms worsen over 18 months, despite numerous antiviral drugs (AZT, DDC, DDI), Grace is overwhelmed by the prospect of dying. She is desperate to find a home for her 13-year-old daughter, Kathy. For Grace, few people meet her strict criteria as caregivers for Kathy. As I read the book, I wondered why Grace never hired a lawyer to plan her daughter’s guardianship, and never joined an AIDS support group. Or why Kip never attended an HTC. Still, spouses of HIV-infected people with hemophilia need more recognition for their important caregiving roles.

The Key to Finding Jack

Ewa Jozeflowicz. Zepher, 2020

Felicity Chesterford, who goes by “Flick,” is enrolled in secondary school in London. She assists her older brother, Jack, in his quest to solve puzzles. Jack finishes his A-level exams in June and plans to spend a gap year traveling in South America. Their father, a barrister, pressures Jack to become a lawyer. Their mother, who runs a social media company, is overprotective of Jack because he has hemophilia. Jack is well prepared with a special medical kit and a doctor’s letter for his hemophilia. Unexpectedly, in January an earthquake hits Lima, Peru, where Jack is headed. Communication is impossible. Flick and her best friend, Kiera, decide to act like detectives to uncover clues that will locate her missing brother, who might be in a medical emergency. Flick is successful, while the foreign office and local police are not, in finding Jack. In this novel for young readers, the plot emphasizes finding clues to solve puzzles, while the often neglected role of sibling bonding, when bleeding disorders are involved, receives recognition.

***

As seen in these novels, family members without bleeding disorders are just as important as the ones with bleeding disorders. The novelists realize that, while the bleeding disorder provides the drama, other family members can play a leading role. All this creates a dynamic fictional situation, one that reflects reality.

Why I Choose Cannabis for My Pain

Laurie Kelley January 17, 2021

Felicia Carbajal

[I have to preface this article by saying that while I never used recreational drugs, lately I have had a consistent, disruptive backache that I am still trying to resolve. It’s interfering with my quality of life by now, and upon recommendation of some people, I’ve tried CBD cream. I have to say I love the results! So I thought I’d reprint this article from the February 2020 issue of PEN, by someone who knows her stuff! …Laurie]

The cannabis plant has been deeply engrained in American history since our country’s inception. Commonly called medical marijuana and hemp, cannabis sativa has been used in everything from textiles and paper to medicines and spiritual tools. Although cannabis has been viewed as harmful or illegal, it has the potential to combat our nation’s opioid crisis, repair some of the harm caused by the war on drugs, and offer Americans a natural alternative to pharmaceutical drugs.

Why do I believe so strongly that cannabis can do all of this? It’s pretty simple: cannabis has been a wonder drug for treating my chronic pain from a spinal cord injury over a decade ago. It has also revolutionized the treatment of my depression, anxiety, and PTSD, and has numerous therapeutic effects when used in its various forms.

After my last back surgery, a microdiscectomy, I took Vicodin to manage the pain, but this would alter my mood and only mask the pain momentarily. I wasn’t myself, and the pain always came back. My medical team said this would be my life. Between the epidural injections and physical therapy sessions, I began researching alternatives.

I tried everything to reduce my pain. I bought gadgets like seat and hand-held massagers, a laser acupuncture pen, and electrostimulation devices. I tried countless complementary therapies like chiropractic treatments and acupuncture. Up to that point, cannabis was last on my list of options. Fortunately, I was introduced to a world-renowned medical professional who was working with patients on low-dose cannabis options with controlled intake of THC. His team gave me a bottle of tincture to try. Three days later, I was pain-free, no longer needing Vicodin, and smiling.

This introduction marked the beginning of my journey with cannabis. I knew the power of sharing my story, and became even more intrigued by the potential of this plant as I weaned myself off a cocktail of anxiety, depression, and pain meds that caused more harm than good. I knew I had to keep learning.

First, I researched the legal history of cannabis. I grew up a DARE¹ evangelist during the 1980s and was unaware of cannabis’s history in the US dating back over a century. Hemp was a valuable crop in the American colonies, used for a variety of purposes, including paper and rope. Eventually, it entered American pharmacopeia as cannabis and became a tool for advancing conservative agendas. Today, more than half the country has some form of regulated cannabis, and a majority of states allow the sale and transportation of hemp-derived products.

Next I explored the science of cannabis. I had friends who’d been diagnosed with HIV and AIDS and knew that this was their medicine, but I didn’t understand why or how. I dove deep into the research and discovered the endocannabinoid system (ECS).²

The ECS is a network of neurotransmitters and receptors that work round-the-clock to help keep the body in homeostasis. Found throughout the bodies of mammals and other vertebrates, the ECS responds to the presence or deficiency of cannabinoids, which can be endogenous (produced within the organism) or exogenous (produced externally).

Endocannabinoids are produced internally and regulate the function of just about every physiological system within the body. Phytocannabinoids are endocannabinoids derived from plants, including but not limited to cannabis.

Naturally, I went to my physician and began asking about the ECS. To my surprise, she knew little about it. I shared some links from the National Institutes of Health (NIH)³ and other research bodies⁴ about current clinical trials and research.

My biggest recommendation: When you explore cannabis, be safe. The cannabis industry is in its infancy; with recent reports of cannabis-induced health concerns, it’s imperative to purchase from a licensed, regulated producer. You should be able to view the lab test results of any product you buy, so you know exactly what you’re putting in your body. There are lots of options everywhere, including websites like Amazon, so be mindful! And if possible, test what you’re using.

Finally, make sure you’re aware of the laws and regulations in your area. This is essential as more and more states regulate.

I’m fortunate to live in a state with regulated cannabis, which means I have access to clean, tested cannabis products—a privilege I don’t take lightly. I have the opportunity to explore other cannabinoids, including THCA (tetrahydrocannabinolic acid) and THCV (tetrahydrocannabivari) in conjunction with terpenes, aromatic plant essences found abundantly in cannabis that can provide therapeutic relief and enhance the efficacy of other compounds when combined (the “entourage effect”).

For those who are new to cannabis or who live in unregulated areas, take this chance to educate yourself. Check out sites like Project CBD,⁵ GreenFlower Media,⁶

and Leafly,⁷ and dive into the data. Go to the NIH website⁸ and type “cannabis” along with your condition to review the research. The reality is that cannabis is personalized medicine, and the one-size approach won’t work for everyone.

After years of taking opioids, I’m finally free. Cannabis can improve the quality of your life, too. Understand and explore the possibilities of cannabinoid therapies. Together we can fight the stigma and perception surrounding cannabis, save countless lives from opioid-related overdoses and deaths, and heal the harm from the war on drugs by voting for sensible drug policy.

Felicia Carbajal is a values-based community organizer, social entrepreneur, change-maker and innovator in the cannabis industry. Based in Los Angeles, the cannabis capital of the nation, Felicia has over two decades of experience in California’s cannabis market. Felicia has worked with world-renowned cannabis medical professionals, has consulted numerous cannabis brands, and is a trusted resource for multiple patient and consumer communities. Currently Felicia is executive director of the Social Impact Center: felicia@thesocialimpactcenter.org.

1. Drug Abuse Resistance Education

2. www.uclahealth.org/cannabis/human-endocannabinoid-system

3. nccih.nih.gov/health/marijuana

4. www.cancer.gov/about-cancer/treatment/cam/patient/cannabis-pdq
5. www.projectcbd.org

6. green-flower.com

7. www.leafly.com 8. nih.gov

When Compassion Meets Anger

Laurie Kelley January 10, 2021

This past week we witnessed anger vented at our government like we have never seen from Americans in our lifetime. I wondered how to address it in a blog meant for bleeding disorder topics. I couldn’t ignore that it happened. I tried to focus on something positive this week. I found myself thinking about compassionate heroes: those who put themselves in harm’s way to get footage of the riots; who tried to help people who were injured; who used kinder language to soothe anger and hate.

As I was thinking of this, I recalled our own situation in 1992, when I attended my first national hemophilia meeting in Atlanta. My book Raising a Child with Hemophilia was being promoted, and I was so focused on that, and raising my 5-year-old with hemophilia and 2-year-old daughter, that I was shocked when during an interview a newscaster asked me what I thought about what was happening at the NHF meeting down the road. What was happening? I didn’t know what she was talking about. I was oblivious.

I soon found out. Stepping into the exhibit hall, where pharmaceutical companies promoted their booths, I saw protesters yelling, waving their arms, scaring people. One had red paint smeared on him and wore a death mask. They then marched together throughout the hall, banging a drum, calling out companies. It was grim. Some pharmaceutical reps fled the scene. In fact, most did. The booths were being emptied quickly.

At one booth, a lone rep stood his ground. I was standing next to him when Michael Davon, a person with hemophilia and HIV from Boston, walked up to him and began yelling. Michael was quite taller than the rep and intimidating. He threw packages of needles in the man’s face and yelled, “And your needles are the wrong size!”

I wondered what could possible happen next.

Joe Pulgliese, general manager of Armour Pharmaceuticals, the man who had the needles thrown in his face, responded calmly, “I think there are bigger issues here to talk about instead of needle size. Tell me what the problem is.”

The rage, absolute rage about being infected with HIV from contaminated blood products; pharma’s role in this; cover-ups; not being believed and not being heard; the deaths of friends and colleagues.

By simply exposing himself to the anger, absorbing it, taking a stand and offering a listening ear, Joe calmed Michael down. They sat. I watched from the sidelines in amazement as Michael quieted but continued his list of accusations. When everyone else fled, Joe had stayed, unafraid.

I saw the power of compassion, even in the face of potential violence, and the power of listening. Angry people want to be heard. But often our first reaction is to retreat, or defend ourselves. Or even fight back. No one ends up listening. Angry people have a right to be heard. Violence is not acceptable, but not being heard and not feeling compassion can lead to heightened emotions, and even violence.

Today is Joe’s birthday, and I’m proud to say he has been a friend since 1989. He was my first contact in the hemophilia community, and he has been a friend to the community for decades. He secured funding for so many nonprofits for family programs and camps. He approved my book in 1990, when I was a complete unknown. And he works today still, overseeing the Hemophilia Alliance, which helps HTCs with funding, to keep them viable, to serve us.

There will always be angry people, there will always be protesters, and there will always be wrongs that need to be righted. But there aren’t many people who stand in the face of it all, look it in the eye, and say “Tell me what’s wrong.”

Happy birthday, Joe! We have been and still are blessed to have you in this community.

Mountain Madness

Laurie Kelley January 3, 2021

The pandemic rages, winter is here; in the Northeast, it’s dark by 5 pm, gloomy, cold. It’s a great time to snuggle up with loved ones after a week of vacation, to watch a movie at night. On social media, people are recommending The Crown, The Last Czars (little bit of hemophilia in there) and even The Ripper.

Here’s a great family movie that I’d recommend: Bombardier Blood.

This is the movie about Chris Bombardier, a young man with hemophilia B, who set out to summit the seven highest mountains on each continent. North America, South America, Asia, Europe, Africa, Australasia and Antarctic: different heights, climates and topographies. Although expensive, time-consuming and death-defying, Chris did it. And Bombardier Blood captured his journey, from training to traveling to hiking. It will make you sit in awe, cry, laugh and be so proud.

And if you look at my flat-screen collection of documentaries, there is a definite theme: mountain madness! I love mountains, have done a bit of climbing myself, and follow the history of mountain climbing, including the Seven Summits.

Put Chris’s accomplishments into perspective, ignoring that he has hemophilia: of the 108 billion humans who have ever been on this earth, less than 500 have ever completed the Seven Summits. And one of our own, Chris, has done it.

And I’ll add that he did it as a fundraiser for our nonprofit, Save One Life, and to highlight the hardship of those with bleeding disorders in developing countries. That alone is worth the watch.

Laurie Kelley in the Andes: “The mountains are calling, and I must go”

Bombardier Blood was made by Believe Ltd, founded by Patrick James Lynch, another man with hemophilia. And originally funded by Octapharma, a European company that manufactures clotting factor wilate® and nuwiq®.

I’m partial to superhero movies. I love the Marvel Universe and especially love X-Men, the Avengers and even Deadpool (despite his dreadful language). But in Chris we have a real-life superhero. And he is now executive director of Save One Life. His journey to new summits continues, all to benefit our family in need overseas.

Watch it, buy it. About 20% of proceeds go to Save One Life. A win-win for all!

Available on iTunes and Amazon Prime.