The efficacy and safety of the drug Octofactor in prophylactic treatment of adolescents with severe hemophilia A

Effectiveness and safety of domestic recombinant factor VIII (FVIII) with removed B-domain (moroctocogum alfa, Octofactor®, JC “Generium”) usage for preventive treatment of 12 adolescents at age 12–17 years old with severe hemophilia A described in this article. Octofactor infused in dose 35 ± 5 ME/kg 3 times per week with the interval of 48 hours during the period of 21 ± 1 weeks. Effectiveness of therapy estimated at 11 patients because 1 patient was excluded after the first injection of Octofactor due to anaphylactic reaction. Seventeen episodes of bleeding at 6 patients were registered on the background of treatment with the help of Octofactor. Mean number of episodes of bleeding was 1.55 ± 2.12. Fourteen (82.4 %) hemorrhagic episodes of 17 were posttraumatic and 3 (17.6 %) were spontaneous. Mean number of spontaneous episodes (main criterion of effectiveness) was 0.27 ± 0.65, which confirms the low frequency of exacerbations of hemorrhagic syndrome on a background of prophylactic treatment by Octafactor. Among all registered hemorrhagic episodes, 10 (58.8 %) were mild, 5 (29.4 %) observations were intermediate and 2 (11.8 %) episodes were severe. Overwhelming majority of hemorrhagic episodes (15 or 88.2 %) were stopped after 1–2 infusions of Octafactor medication. Mean number of infusions of Octofactor medication for stopping of hemorrhagic episodes was 1.6 ± 0.86. It was needed mean 3808.8 ± 2022.4 МЕ of Octofactor medication to stop 1 episode of bleeding. Residual activity of FVIII after 48 hours after the Octofactor infusion was 2 % and more at overwhelming majority (63.6–90.9 %) of adolescents during the observation (3 visits). Common number of Octofactor medication infused for bleeding prevention was 1 500 500 МЕ, for bleeding stopping – 64 750 МЕ. Safety of therapy estimated at 12 patients. Nine adverse events (AE) were registered at 7 patients, clinical AE were predominant – 5 (55.6 %) cases. One patient had an anaphylactic reaction on the first infusion of Octofactor medication which was estimated as serious AE of mild severity. All other AE were not serious or mild and resolved without after-effects. Trombembolic complications and immunogenic reactions were not registered. Thus, received data demonstrate the effectiveness and safety of Octofactor medication both for prevention and stopping of bleeding at adolescents with severe hemophilia A.

adolescents aged 12–17 years, hemophilia А, recombinant preparation of factor VIII with deleted B-domain, Octofactor, preventive treatment, efficacy, safety

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