[Dr Xavier] Hi, my name is Dr Federico Xavier. I'm a pediatric hematology oncologist specialist, and I treat patients at The Hemophilia Center. I've been treating patients with hemophilia A for more than 2 decades.
When I was in school, there were only a few treatments available, and they had to be dosed 2 to 3 times per week. Many patients weren’t compliant, and they were afraid that they were not protected. Ultimately, they were still bleeding. They would tell me they were doing just fine on their treatment. They wouldn't even recognize that they were having bleeds, because they were so used to them.
And for others, they would stop doing certain things like going to the gym, playing sports, or they had never started at all. They were avoiding activity because of bleeds, or the fear of having them.
But, as I tell my patients, everyone bleeds, no one is a superhero. It's about finding a different treatment that would ideally help avoid the bleeds or would just ensure it's not as serious. So when ALTUVIIIO was approved, I started switching my patients right away.
ALTUVIIIO is a high sustained factor replacement therapy. When used once a week, my adult patients have been able to maintain Factor VIII levels closer to 40% for most of the week because of its 48.2 hour half-life, which is longer than any other Factor VIII replacement therapy. ALTUVIIIO has truly changed the treatment landscape and what's possible for patients.
As anyone who treats hemophilia knows, bleed protection is always top of mind. Not only does it impact our patients' long-term health, but also their day-to-day lives. ALTUVIIIO delivers what I wanted out of a treatment. In the clinical trial, ALTUVIIIO met its primary endpoint, and by Week 52, a subgroup of patients previously on a different prophylactic factory replacement therapy, who had an average of 3 bleeds a year prior to the study, switched to ALTUVIIIO prophylaxis and experienced fewer than 1 bleed a year. That's a 77% reduction in the mean ABR.
Also, patients who had been on a different on-demand therapy and switched to ALTUVIIIO on-demand before transitioning to ALTUVIIIO prophylaxis experienced a 97% reduction in their mean ABR. Essentially, going from having about 21 bleeds in 26 weeks with ALTUVIIIO on-demand to fewer than 1 with ALTUVIIIO prophylaxis. Plus, during the trial, most prophylaxis patients experience not only 0 bleeds, but 0 joint bleeds.
ALTUVIIIO also showed impressive results in the pediatric study, which is great news for my younger patients. Children less than 12 reached a mean steady state of Factor VIII levels at over 40% for about 3 days, and an unprecedented trough level by 10% at about day 7.
During the trial, ALTUVIIIO met its primary endpoint. No Factor VIII inhibitors were detected, and ALTUVIIIO demonstrated proven bleed protection.
Most patients treated with ALTUVIIIO prophylaxis experience 0 bleeds, 0 spontaneous bleeds, and 0 joint bleeds.
For my pediatric patients, less time managing bleeds means more time just being a kid.
ALTUVIIIO also has a proven safety profile across both adult and pediatric studies. The most common adverse events were headache and arthralgia.
ALTUVIIIO is only dosed once a week, so it gives my patients more flexibility, but sometimes my patients are hesitant because it's still an IV infusion. But to me, it's worth it. Knowing how to use an IV is a skill that you don't practice with the factor mimetic.
So, if you do end up needing an additional dose, without that skill, using the IV can be more intimidating. ALTUVIIO is dosed less frequently than other factor treatments, because it's a high sustained factor replacement. For adults and kids alike, proven bleed protection from fewer infusions is in a way freeing. It's made my decision to switch my patients to ALTUVIIIO even easier.
ALTUVIIIO shows how far hemophilia A treatments has advanced, and I feel optimistic about the future for my patients. It's the first high sustained factor therapy on the market, and it keeps my patients' factor level higher for longer. It provides proven bleed protection for my patients, and it's manageable because it's only dosed once a week. It has been wonderful to witness a change in my patients' health after they have switched to ALTUVIIIO. They can finally expect more from their treatment, and that's the goal.
[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.
