HemaBlog™

See updated study results for kids with hemophilia A.

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.

*Data based on treated bleeds.

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

ALTUVIIIO_AshleyHarrison_GradCircle
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

ALTUVIIIO_NatalieSamuel_GradCircle

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.

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

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