Long-lasting hemophilia treatment may offer hope in hard-to-treat cases
Approved therapy shown to reduce bleeding when prevention wasn't enough
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- A real-world study found long-lasting Altuvoct, sold in the U.S. as Altuviiio, effectively reduces bleeding in hemophilia A when preventive treatment isn't enough.
- Weekly Altuvoct infusions were found to cut breakthrough and joint bleeds by more than 80%.
- The study also suggests that FVIII monitoring in hemophilia A needs to be standardized.
Once-weekly infusions of Altuvoct (efanesoctocog alfa) — a long-lasting hemophilia treatment marketed in the U.S. as Altuviiio — reduced bleeding in adults and children with hemophilia A for whom preventive treatment was not sufficiently effective.
That’s the key finding of a new French real-world study that tracked the therapy’s use in a small number of hemophilia A patients determined to have “unmet need” despite the use of prophylactic, or preventive, therapies, including Hemlibra (emicizumab).
In addition to cutting so-called breakthrough bleeding, defined as bleeding that occurs despite prophylaxis, treatment with Altuvoct was found effective for people with digestive-tract bleeds and for those for whom preventive therapies did not last long enough. In fact, for most patients, the treatment reduced bleeds overall by more than 80%, the data showed.
“Over a median [nine]-month follow-up, weekly [Altuvoct] prophylaxis resulted in a significant 89% reduction in treated bleeds … and an 85% reduction in joint bleeds … for the total [patient] population,” the researchers wrote.
The researchers noted, however, that accurately measuring clotting factor VIII (FVIII), the protein missing in hemophilia A, remains a challenge.
The study, “Unmet needs despite standard-of-care prophylaxis in hemophilia A: improved outcomes after switching to efanesoctocog alfa,” was published in the journal Blood Advances.
Among people with hemophilia, blood does not clot properly, resulting in unusually easy and prolonged bleeding. In hemophilia A, this is caused by a lack of FVIII, a protein that helps blood to clot. Despite standard preventive treatment, known as prophylaxis, some patients still experience symptoms.
Real-world study examined use of approved hemophilia A treatment
Altuvoct is a factor replacement treatment, meaning it replaces missing FVIII, thereby helping the blood clot and providing temporary control of bleeding. Its active ingredient, efanesoctocog alfa, was designed to remain active in the body for longer than the natural FVIII.
After receiving European approval, Altuvoct was first available in France through an early access program for patients whose needs were not met by standard prophylaxis. This program, monitored by the French National Network for Rare Bleeding Disorders, continued until Altuvoct was marketed. Now, a team of researchers examined its data.
According to the researchers, the ECHO study “provides a real-world evaluation of [Altuvoct], a first-in-class ultra-long-acting factor VIII (FVIII) concentrate, within the French compassionate access (CAA) framework.”
The study also described the clinical needs that supported early access: Of 90 requests, 54 were approved because doctors demonstrated that existing treatment options did not meet patients’ needs. In total, 50 adults and children from 17 hospitals joined the study.
The patients had a median age of 31, and most (84%) had severe hemophilia A. Before switching to Altuvoct, all were already receiving prophylaxis. Slightly more than half were on FVIII products, while the remaining 48% were receiving Hemlibra, a treatment that mimics the action of FVIII. Importantly, the researchers noted that an analysis “confirmed that … [Hemlibra] is highly effective for the broader French [hemophilia A] population.”
The team also noted that nine patients (18%) had previously developed inhibitors, which are antibodies that prevent FVIII from working.
The main reason for switching to Altuvoct was continued breakthrough bleeding. However, in most cases, doctors reported several reasons for switching rather than just one. For example, some patients also had serious bleeding in the digestive tract; in others, previous prophylaxis did not last long enough.
Some of the participants had other medical problems that increased their risk of bleeding or made treatment more difficult, such as difficult to find veins. Among patients with available joint imaging, 78% already had synovitis, inflammation of the joint lining. Synovitis is common after repeated bleeding into the joints and can lead to pain and long-term damage.
Breakthrough bleeding became uncommon for patients after Altuvoct use
Most patients received Altuvoct once a week at the recommended dose. After a median of nine months, the number of treated bleeds decreased by nearly 90%. The number of patients with no treated bleeds increased from 43% before switching to 64% after starting Altuvoct.
Moreover, the researchers noted that “this clinical benefit remained consistent across exploratory subgroups, including for patients previously on [Hemlibra].”
Benefits were observed among those with both spontaneous and traumatic bleeds, as well as among patients previously treated with other therapies. Breakthrough bleeding became uncommon, the researchers noted.
In addition, Altuvoct alone provided sufficient control of bleeding for five minor medical procedures without requiring additional FVIII replacement, the data showed.
In [the] French … program, weekly [Altuvoct] prophylaxis reduced treated bleeds rate in [hemophilia A] patients with unmet needs.
The treatment was generally safe and well tolerated. No patient developed new inhibitors against FVIII. One older patient died from a heart problem that was considered unrelated to Altuvoct. Another patient stopped treatment because Altuvoct did not work well enough for a pseudotumor, which is a large collection of blood that can damage nearby tissues.
The researchers also measured FVIII just before the next weekly dose, a measure known as trough level. Trough levels indicate how much FVIII remains in the blood at the lowest point between doses and help doctors assess whether treatment is providing adequate control of bleeding. Using a lab test designed specifically for Altuvoct, the median trough level was 7 IU/dL.
However, when using the more commonly used test performed in local hospitals, the median trough level was about twice as high, at 14 IU/dL. The difference was observed across several reagent types, indicating that measuring FVIII remains challenging. However, “As adoption of [Altuvoct] increases, standardized laboratory protocols will be essential to ensure precise monitoring and optimal clinical outcomes,” the researchers wrote.
Overall, the team concluded: “The ECHO study provides real-world evidence that [prophylaxis with Altuvoct] addresses therapeutic gaps for patients with complex medical requirements.”
“In [the] French … program, weekly [Altuvoct] prophylaxis reduced treated bleeds rate in [hemophilia A] patients with unmet needs,” the researchers wrote.
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