Prophylaxis with Rurioctocog Alfa Pegol in Severe Hemophilia A
Rurioctocog alfa pegol represents a modern modality for the treatment of hemophilia from the group of factors with an extended half-life. The authors of a systematic review and meta-analysis published last year assessed its efficacy and safety in patients previously treated for hemophilia A.
Benefits of 3rd Generation rFVIII
Rurioctocog alfa pegol (BAX855, Adynovi) is a representative of the 3rd generation recombinant factor VIII (rFVIII) modified by attaching polyethylene glycol (PEG). This modification extends the half-life of FVIII by approximately 1.5 times compared to the original rFVIII. This then allows for reduced frequency of drug administration in the prophylaxis of hemophilia A (HA) and maintains better minimum FVIII levels, especially in the most severe hemophiliacs.
Meta-Analysis Results
The authors conducted a systematic review and meta-analysis of data on the efficacy and safety of rurioctocog alfa pegol in previously treated hemophiliacs following the PRISMA 2009 guidelines.
The data concerns 517 patients with severe HA from 4 studies. The cumulative average annual bleeding rate (ABR) reached 2.5 events/year (95% confidence interval [CI] 2.04–3.14), with an average efficacy of 92% (95% CI 85–97%). Only 30 (2.3%) non-serious adverse events related to the treatment with rurioctocog alfa pegol and 1 serious (1.4%) were described. No development of FVIII inhibitors was observed by the end of the studies; antibodies against FVIII, PEG, or FVIII-PEG were detected in 52 patients, but their presence did not correlate with the efficacy or safety of the treatment.
Conclusion
The authors conclude that, although we naturally do not have direct comparative studies, this analysis shows that rurioctocog alfa pegol is a safe and effective option for preventing bleeding episodes in previously treated individuals with hemophilia A. Furthermore, the data indicate low immunogenicity, which further improves the drug's safety profile.
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Source: Witarto B. S., Visuddho V., Witarto A. P. et al. Efficacy, safety, and immunogenicity of rurioctocog alfa pegol for prophylactic treatment in previously treated patients with severe hemophilia A: a systematic review and meta-analysis of clinical trials. F1000Res 2021 Oct 15; 10: 1049, doi: 10.12688/f1000research.73884.3.
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