The benefits of extended half-life products for patients with severe hemophilia A were highlighted by the experiences of adult and pediatric patients who received turoctocog alfa pegol (Esperoct®) as prophylaxis. The findings were published in the journal Patient Preference and Adherence.
The patients were selected from the pivotal pathfinder clinical trials, the longest and largest clinical trial program conducted in hemophilia A to date. More precisely, adult patients were enrolled in pathfinder 2 and 3 and pediatric patients were enrolled in pathfinder 5.
In this case series, turoctocog alfa pegol, also known as N8-GP, was well tolerated by both adult and pediatric patients. Overall, it decreased the number of bleeding episodes and was associated with improved physical activity in patients.
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The authors highlighted the cases of 2 patients whose mean annualized bleeding rates (ABR) decreased from 35 and 22 (prior to enrollment) to 1 and 3, respectively. The factor VIII (FVIII) trough levels of these patients were 2.2% and 1.9%, and both patients were able to improve their physical activity. One of the patients experienced a moderate adverse event (AE) of rotator cuff syndrome, while the other only reported mild AEs.
Prophylaxis with turoctocog alfa pegol showed positive results when used in adults undergoing ≥3 major surgeries. The 3 patients in this situation received a single presurgery dose. In addition, 4 surgeries required a single postsurgery dose on the day of the intervention. All patients presented with an ABR of 0 at the end of the study, and their FVIII trough levels were 3.8%, 8.8%, and 1.1%.
Pediatric patients noted improvements in joint health. Overall, 31.7% of the pediatric patients who enrolled in pathfinder 5 did not experience joint bleeds during the trial. Klamroth et al highlighted the cases of 5 patients with 7 target joints at baseline who had no bleeds during the duration of the trial (mean 4.5 years).
For instance, a pediatric patient who enrolled in pathfinder 5 at 11 years old presented with an ABR of 1 and an FVIII trough level of 2.9% during the 5-year treatment with turoctocog alfa pegol. During the 12 months before enrollment, his ABR was 3, with all bleeds involving traumatic injuries to joints. The patient transitioned to pathfinder 8 at around 16 years of age, and his treatment regimen was then updated to 3 injections per week to better support his physical performance in American football and rugby.
The safety profile of turoctocog alfa pegol was uneventful for all pediatric cases described by Klamroth et al.
“The strength of our case series is that all on-trial data were collected systematically as part of the pathfinder program. However, as for all case series, our study is limited by selection bias and potentially recall bias for subjective outcomes,” the authors acknowledged.
Klamroth R, Hampton K, Saulyte Trakymiene S, Korsholm L, Carcao M. Illustrative cases from the pathfinder clinical trials of patients with hemophilia A treated with turoctocog alfa pegol (N8-GP). Patient Prefer Adherence. 2021;15:2443-2454. doi:10.2147/PPA.S326282