Rurioctocog alfa pegol (Adynovate®) is safe, effective, and appears to cause only a weak immune response in previously treated patients with hemophilia A, according to a new study published in F1000Research. This finding suggests it could be a safe and effective alternative prophylactic treatment.
Patients with hemophilia A have to be treated with regular factor VIII (FVIII) prophylaxis. However, this causes a daily treatment burden on them.
In this study, a team of researchers conducted a systematic review and meta-analysis of rurioctocog alfa pegol clinical trials to evaluate its safety, efficacy, and immunogenicity in previously treated patients with severe hemophilia A.
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They analyzed 4 studies that involved a total of 517 patients and found that the pooled mean of total annualized bleeding rates among all patients was 2.59. The hemostatic efficacy of the treatment was 92% and, in terms of safety, 30 nonserious and 1 serious adverse event related to rurioctocog alfa pegol occurred. Patients did not develop inhibitors against the treatment.
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A network meta-analysis with a multiarm approach on the treatment of hemophilia A is needed to confirm these findings presented, the authors said.
Hemophilia A is caused by a mutation in the gene that encodes FVIII, 1 of the proteins that play an essential role during blood clotting. As a result of the mutation, there is not enough functional FVIII in the body. This leads to painful joint and soft tissue bleeding restricting the daily activities of patients.
Rurioctocog alfa pegol is a third-generation recombinant FVIII. It has a modification in its polyethylene glycol component that prolongs its half-life around 1.5 fold compared to original recombinant FVIII products. The treatment has been shown to have a safety profile comparable to that of other factor VIII products.
Reference
Witarto BS, Visuddho V, Witarto AP, Sutanto H, Wiratama BS, Wungu CDK. REVISED 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. [version 2; peer review: 1 approved]. F1000Research. 2021;10:1049. doi:10.12688/f1000research.73884.2
