Adynovate controls bleeding in real world for hemophilia A patients
Preventive therapy zeroed out bleeding episodes in South Korean study
In a real-world study in South Korea, preventive treatment with Adynovate (rurioctocog alfa pegol) was shown to be effective at preventing bleeding episodes among most children and adults with hemophilia A — findings that line up with those from clinical testing.
In fact, “76.1% of patients on prophylactic treatment had zero bleeds,” the researchers wrote in the study highlights.
For patients who did experience a bleeding episode, one or two doses of on-demand Adynovate were enough to bring it under control in most cases, according to data from the postmarketing study. For doctors, Adynovate provided good or even excellent bleed control when used as an on-demand treatment, the study found.
Overall, Adynovate “was effective in children and adults, with no new safety signals,” the researchers concluded.
The work, titled “Real-world safety and effectiveness of rurioctocog alfa pegol in 338 patients with hemophilia A in South Korea: A postmarketing surveillance study,” was published in the journal Thrombosis Research. It was funded by Takeda Pharmaceuticals, which markets Adynovate.
Hemophilia A is caused by mutations in the F8 gene, which provides instructions for making factor VIII (FVIII), a blood clotting protein. When FVIII is faulty or missing, a person’s blood cannot clot properly, leading to prolonged bleeding episodes that can be difficult to control.
Treatment usually involves replacing the missing FVIII, either regularly to prevent bleeds — thus known as prophylaxis — or as needed to control bleeding episodes when they occur, as an on-demand therapy. Standard half-life factor replacement treatments require frequent infusions, but extended half-life products like Adynovate stay longer in the body, reducing the number of doses needed.
Adynovate has been approved in South Korea since 2018 for treating hemophilia A.
Results seen with Adynovate use mirrored those in clinical trials
While clinical studies showed that Adynovate is safe and effective for both children and adults with hemophilia A, “routine clinical practice data on its safety and effectiveness, especially in children, are limited,” the researchers wrote. Further, the team added, “no large, real-world study has been conducted in South Korea.”
Now, a research team, primarily from institutions across the Asian nation, reported data from a postmarketing surveillance study (NCT03824522) that assessed the safety and effectiveness of Adynovate in a real-world medical setting.
The study used data from 338 people with hemophilia A, including 54 children younger than 12. Overall, the patients had a mean age of 25. On average, the time since diagnosis was 19 years.
Most patients (92.9%) used Adynovate for prophylaxis, while 23 (6.8%) used it as an on-demand treatment. Nine (2.7%) used it to manage bleeding associated with surgery.
During preventive treatment, most patients (76.1%) had no bleeding episodes, and the mean annualized bleeding rate (ABR) — the number of bleeds adjusted to a one-year time frame — was 1.7. For those younger than 12, the mean ABR was 1.1; for those ages 12 to younger than 18, it was 1.6.
When breakthrough bleeding occurred, doctors rated the effectiveness of on-demand treatment with Adynovate as being excellent (70.9%) or good (29.1%). Most bleeding episodes that occurred were controlled with just one or two doses of the therapy. This was also observed in children and adolescents across all ages, the researchers noted.
Therapy rated ‘good’ or ‘excellent’ when used on demand
All 23 patients who received only on-demand treatment with Adynovate had bleeds — 322 in total — and doctors rated treatment effectiveness as being good (60.6%) or excellent (36.3%). Most bleeding episodes (70.8%) were controlled with just one infusion.
Two children ages 2-11 had 31 bleeds in total, and treatment was rated as good by doctors for all bleeds. Most (87.1%) were controlled with one infusion.
Nine patients received treatment with Adynovate before, during, or after a surgical procedure. Effectiveness was rated as good in 60%, excellent in 20%, and fair in 20%. None of the patients developed inhibitors, or neutralizing antibodies that can sometimes form in response to factor replacement therapies and limit how well they work, the data showed.
Altogether, 20 patients (5.9%) experienced side effects, most of which were not serious. Four patients had side effects that were considered to be potentially related to Adynovate. In children, no new or unexpected safety concerns were found.
“Findings from this real-world study … indicate that the safety profile of [Adynovate] was maintained in line with previous reports, with no new safety concerns identified,” the researchers wrote.
The team added that Adynovate was “found to be efficacious,” noting that preventive treatment continues to zero out the number of bleeding episodes for most patients.
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