Among patients with hemophilia A, targeting elevated factor VIII (FVIII) levels using personalized rurioctocog alfa pegol prophylaxis appears to be effective for reducing annualized bleed rates (ABRs), according to research published in Therapeutic Advances in Hematology.
Previous research has established that standard half-life (t1/2) FVIII treatments are necessary at least once every 2 to 3 days to maintain FVIII trough levels sufficiently for bleed protection. Rurioctocog alfa pegol — an extended t1/2 recombinant FVIII — appears to maintain FVIII trough levels for longer periods, warranting further study.
For the randomized phase 3 PROPEL study (ClinicalTrials.gov Identifier: NCT02585960), researchers evaluated the safety and efficacy of rurioctocog alfa pegol among patients with hemophilia A, relying on a pharmacokinetic-guided prophylaxis approach. For the present paper, researchers conducted a post-hoc analysis from PROPEL data to determine which patients with hemophilia A are the most likely to benefit from personalized prophylaxis with rurioctocog alfa pegol.
Overall, data from 95 patients were included, among whom 52 and 43 had FVIII trough levels of 1-3% and 8-12%, respectively, at baseline. Analysis showed that targeting an elevated trough (8-12%) led to higher FVIII levels, on average, over time, regardless of baseline FVIII t1/2 levels.
Patients with a FVIII t1/2 of between 6 and less than 12 had a greater decrease in ABRs over the study period compared with others (0.7 vs 3.5, respectively). Furthermore, more patients had 0 bleeds, and mean ABRs were lower in the 8-12% group compared with the 1-3% arm, regardless of baseline arthropathy status.
“Future studies could aim to confirm this by investigating the use of personalized treatment in these specific patient populations,” the authors wrote in their report.
Disclosures: This research was supported by Takeda. Please see the original reference for a full list of disclosures.
Escuriola-Ettingshausen C, Klamroth R, Escobar M, et al. Targeting an elevated FVIII level using personalized rurioctocog alfa pegol prophylaxis in specific patient populations with hemophilia A: post hoc subanalysis of the randomized, phase 3 PROPEL study. Ther Adv Hematol. 2023;14:20406207231178596. doi:10.1177/20406207231178596