Clinical Case: DJ
Dr Shapiro introduces DJ, an adult patient with severe hemophilia A who values balancing disease management with family and daily life responsibilities.
[Dr Shapiro] I'm Dr Amy Shapiro. I'm a pediatric hematologist. I am Medical Director and CEO of Innovative Hematology and the Indiana Hemophilia and Thrombosis Center in Indianapolis, Indiana. This individual is DJ, a 36-year-old husband, father, and working professional.
He is a promotional speaker compensated by Sanofi. He is a full-time tech specialist at a tech company. He is married with a 2-year-old son. He was diagnosed with severe hemophilia A at birth, and his brother also has hemophilia. He started prophylaxis treatment in 2001.
He was previously on standard half-life Factor VIII therapies for 27 years, followed by extended half-life Factor VIII prophylaxis every other day for 9 years.
He has arthropathy on his bilateral elbows, his left knee, and right ankle.
He also has had no recent bleeds in 2023 or 2024 that were reported. But it is important to consider what DJ's aspirations and interests are. He wants to be able to balance his professional life, his family, and his hobby of drumming with flexibility and control.
He wants to be able to put his family first and his hemophilia second. For example, he wants to be able to play catch or frisbee in the park with his son without worrying as much about his hemophilia. He also wants to decrease his infusion burden. The question that we need to consider in this case is how DJ can balance being a father, a working professional, and a drummer.
In XTEND-1, the primary endpoint was the mean annualized bleed rate, or ABR. Mean ABR with ALTUVIIIO prophylaxis in arm A was 0.7. Median ABR with ALTUVIIIO prophylaxis in arm A was 0. In XTEND-1, ALTUVIIIO demonstrated target joint and joint health improvement.
The majority of patients had 0 joint bleeds with ALTUVIIIO prophylaxis in both study arms, 72% in the prior Factor VIII prophylaxis group and 81% in the prior Factor VIII on-demand group after switching to ALTUVIIIO prophylaxis. 14 patients in arm A prior Factor VIII prophylaxis group had a total of 45 target joints at baseline. All of these target joints resolved within 12 months of continuous ALTUVIIIO prophylaxis, demonstrating 100% target joint resolution. The Hemophilia Joint Health Score, the HJHS, which is a validated scoring system for hemophilia, total score decreased by 1.5 compared with baseline in the prior Factor VIII prophylaxis group.
The Haem-A-QoL total and physical health scores were improved in 3 years of follow-up in patients aged 17 years or older in the XTEND-ed trial. Low scores here represent an improvement in physical health, so a negative change from baseline represents an improvement over the course of the study.
This was consistent with the improvement in the Haem-A-QoL physical health score in XTEND-1 from baseline to Week 52.
Consideration for switching DJ's treatment were discussed with him. He desired higher Factor VIII levels, decreased infusion frequency, and better bleed control. He has arthropathy of his bilateral elbows, his left knee, and right ankle. After discussions with DJ, and based on these considerations, he was started in ALTUVIIIO in October 2024. So how has he been doing in the 6 months since initiation of ALTUVIIIO? What's been possible for him? DJ has had no bleeding episodes since starting ALTUVIIIO. He has had more confidence in his bleed protection and less concern with his factor levels and dosing frequency and the unpredictability that may sometimes come with hemophilia. He is experiencing and enjoying the everyday moments in life, keeping up with his son and family. We can also hear how he's doing directly from him.
[DJ] Hi, my name is DJ and I have severe hemophilia A. I've been married almost 6 years, and I have a 2-year-old son named Kal-el. I play the drums, and I love music and comic books. Being a drummer helped me build an identity that went beyond DJ the hemophiliac. I wasn't always great with taking care of myself and my hemophilia, but I realized that when I do, I'm more present for my family without limitations.
My grandfather had hemophilia. He died in 1996 from the bad blood era because his factor was contaminated. That's why my family is never quick to switch to new treatment options. We talk to our doctors and the community to make sure that we're making the right decision. So when we do switch, we're confident that it's right for us. When ALTUVIIIO came out, I was still hesitant. We decided to make the change in October of last year, and since then, it's been an incredible journey.
I love the half-life. The biggest thing for me is the downtime, how much time in between infusions, and really feel protected between the fusion days. At my last visit, my factor levels the day before I infused were 30%. On ALTUVIIIO, I know where I stand as far as my factor levels.
I know I have higher for longer protection. It's the confidence in knowing. I can focus on living life first. My hemophilia treatment can become secondary because of medical innovations, including ALTUVIIIO. I can infuse once a week when my son isn't awake. I haven't had any moment where he's waiting for me or I'm down for a bleed or I can't do the things that he wants me to do with him. Kal likes Frisbee now. He calls it Flisbee. So just tossing his Flisbee around or playing catch with him, that's a joy I couldn't imagine living without.
[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.
