Expert Perspectives: Lucas, 3 years old

[Dr Roberts] My name is Jonathan Roberts. I'm the Associate Medical and Research Director for the Bleeding and Clotting Disorders Institute and Associate Professor of Pediatrics and Medicine at the University of Illinois College of Medicine in Peoria. I met Lucas's family at a time of a lot of stress for them. After he was just a few days old, he was transferred to our center because he was having uncontrolled bleeding.

Lucas is now a 3.5-year-old little boy who was diagnosed with severe hemophilia A at birth. He has a de novo mutation, so his family has no history of hemophilia, and the diagnosis for Lucas was kind of a shock initially to the family. In talking with the family initially, they felt very strongly they wanted to stay on a Factor VIII product.

And so as he grew and his initial bleeding from a heel stick and a circumcision were controlled, we initiated standard half-life and then extended half-life factor therapies. And then actually as Lucas got older, ALTUVIIIO became available and his parents learned how to do infusions at home and he was transitioned to ALTUVIIIO.

His father is a high school basketball coach, and Lucas is a de novo mutation. All of his siblings have been tested. His mom's been tested. No one else has hemophilia or the mutation for hemophilia. So he really needs to fit into a very active family. So all of his older siblings are already teaching their little brother how to play basketball, and he's at all the games, and he wants to be active.

So there's a lot of focus in our discussions about wanting to ensure that he can still do the things that he wants to do, obviously with making sure he's mindful of, you know, what his factor levels are and that he's protected to do the activities he wants to do. But really my goal is for him to not have to think about his hemophilia much as he gets older. Now, some of the things that offered them confidence in ALTUVIIIO was looking at data from the 2 phase 3 studies, from the XTEND-1 XTEND-Kids, and then the XTEND-ed phase 3 extension study. So the XTEND-1 study is the original study looking at children, adolescents, and adults greater than or equal to 12 years of age with severe hemophilia A.

That had a prophylaxis and an on-demand arm that then went to prophylaxis with ALTUVIIIO 50 units per kilo weekly. The XTEND-Kids study was a prophylaxis study for children less than 12 years of age, and both enrolled into the Phase 3 extension XTEND-ed study to continue ALTUVIIIO prophylaxis for up to 4 years.

The primary endpoints of the XTEND-Kids study was met with 0 neutralizing antibodies detected. And select secondary endpoints were determined looking at the Factor VIII activity being 3 days in the near normal range, and then looking at the trough levels of Factor VIII of 10% at day 7 at the 26 week time point and then 10.9% in children less than 6 and 16.5% in children 6 to 12 years of age at Week 52 at the end of study. 

When also looking at the bleed protection rate offered by ALTUVIIIO, the median annualized bleed rate, or ABR, was 0 with the estimated mean ABR of 0.6 and 64% of patients had 0 bleeds.

Going back to our patient Lucas, his parents switched him to ALTUVIIIO and he's been on it for nearly 2 years. As he's getting older, he's moving towards being more active, playing basketball with his family as his father is a basketball coach for the local high school. And having other siblings that are not affected with hemophilia, Lucas really wants to feel like a normal little kiddo and ALTUVIIIO allows him to do that with weekly infusions.

At initiation, his Factor VIII level was 28% at 4 days after infusion, and now he maintains his trough over 10% without any breakthrough bleeds. The only additional bleeding or factor doses he's had since initiation on ALTUVIIIO has been proactively from the family if he's had some additional playful trauma, that they wanted some better protection for him in that regard. The treatment burden for Lucas early on, I mean, he was getting once a week factor therapy with previously available extended half-life factor, but he would still have, you know, playful trauma, even, you know, small hematomas, things would happen, to the point that his mom would be worried. And there are many weeks that he received more than 1 dose of factor. Now, since being on ALTUVIIIO, that hasn't completely gone away because they're very vigilant about ensuring he's treated. So he does occasionally get a breakthrough dose when they're worried about bleeding risk, but it's not been for a frank bleeding event. It's been more precautionary, you know, that a brother throws a toy at him or something like that. And they're worried about bleed formation, so they may give him a dose a little bit early. But overall, their peace of mind is much improved with being on ALTUVIIIO.

[VO] INDICATION

ALTUVIIIO^® is indicated for use in adults and pediatric patients with hemophilia A (congenital factor VIII deficiency) for:

• Routine prophylaxis to reduce the frequency of bleeding episodes
• On-demand treatment and control of bleeding episodes
• Perioperative management of bleeding

Limitation of Use

ALTUVIIIO is not indicated for the treatment of von Willebrand disease.

IMPORTANT SAFETY INFORMATION

CONTRAINDICATIONS

ALTUVIIIO is contraindicated in patients who have had severe hypersensitivity reactions, including anaphylaxis, to the product or its excipients.

WARNINGS AND PRECAUTIONS

• Allergic-type hypersensitivity reactions, including anaphylaxis, have occurred with ALTUVIIIO. Discontinue use of ALTUVIIIO if hypersensitivity reaction occurs and manage symptoms as appropriate.
• Formation of neutralizing antibodies (inhibitors) to Factor VIII has been reported following administration of ALTUVIIIO. Monitor all patients for the development of Factor VIII inhibitors by appropriate clinical observations and laboratory tests.
• If assessment of plasma Factor VIII activity is needed, it is recommended to use a validated one-stage clotting assay. The ALTUVIIIO Factor VIII activity level is overestimated by the chromogenic assay and a specific ellagic acid-based aPTT reagent in one-stage clotting assay by approximately 2.5-fold. If these assays are used, divide the result by 2.5 to approximate the patient's ALTUVIIIO Factor VIII activity level.

ADVERSE REACTIONS

The most common adverse reactions (>10% of subjects) reported in clinical trials were headache and arthralgia.

Please see full Prescribing Information.