A study presented at American Society of Hematology’s (ASH) annual meeting and exposition shows that Adynovate is effective for the prevention and treatment of severe pediatric Hemophilia A (HA).
Eric Mullins, MD and his colleagues from the Cancer and Blood Diseases Institute at Cincinnati Children’s Hospital Medical Center presented their work “Pegylated Full-Length Recombinant Factor VIII with Extended Half-Life in Previously Treated Pediatric Patients with Hemophilia Α: Efficacy of Prophylaxis and Treatment By Time (of Day/of Week) of Occurrence of Bleeding” at the ASH annual meeting in San Diego, Calif., in December.
Adynovate (Bax 855, Baxalta) is a PEGylated version of the full-length recombinant Factor VIII (FVIII), Advate. PEGylation masks the drug from the host’s immune system and increases its half-life, allowing it to remain in circulation longer.
In children with HA, avoiding additional complications such as bleeding into the joints and specific forms of arthritis, is a major concern. Timing FVIII infusions to cover periods of physical activity, as well as adherence to treatments, also are significant concerns. Adynovate, with its longer half-life, may alleviate some of these concerns.
The drug’s efficacy was assessed in a Phase 3, prospective, multi-center clinical trial that included patients under the age of 12 previously treated for severe HA. Sixty-six patients were enrolled in the study. Just fewer than half the patients were less than 6 years old.
The researchers assessed annualized bleeding rates (ABR) at different times of day (morning, afternoon, evening) and between weekdays and weekends. The mean dose of BAX 855 was 51.1U/kg. On average, 1.8 infusions per week were given for an average period of 48.5 days.
Results from this study show patients had higher bleeding rates in the evening when compared to either afternoon or morning bleeding rates. The time between prophylaxis infusion and the bleeding event was similar at all times of the day. The interval between infusion and bleeding event was lower on weekends compared to weekdays. The researchers suggest that infusion was timed to cover the higher amount of activity expected on the weekends.
Interestingly, higher ABRs were observed on weekdays than on weekends, although bleeding severity was comparable. This suggests that timing of Adynovate infusion may be important. In general, higher ABRs also were seen in the older patient population.
Treatment with Adynovate was regarded as excellent or good in more than 85% of bleeding events, regardless of when the bleeding event occurred.
The authors concluded: “BAX 855 was shown to be effective for the prevention and treatment of bleeding in this patient population.”