Erschienen in:
02.01.2019 | Original Article
Inhibitor development, safety and efficacy of Advate® among previously treated patients with hemophilia A in a postmarketing surveillance in Japan
verfasst von:
Katsuyuki Fukutake, Masashi Taki, Tadashi Matsushita, Keiji Nogami, Midori Shima, Akira Yoshioka, Junki Takamatsu, Haruhiko Uchikawa, Hiroshi Takagi, Morio Arai, Werner Engl, Akira Shirahata
Erschienen in:
International Journal of Hematology
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Ausgabe 3/2019
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Abstract
Rurioctocog alfa (recombinant factor VIII: Advate®) is available for the control of bleeding in patients with hemophilia A in Japan. To evaluate the inhibitor development, safety, and efficacy of rurioctocog alfa, a non-interventional and observational postmarketing surveillance was conducted on 352 previously treated Japanese patients aged 1–76 years with ≥ 4 exposure days under the conditions of routine clinical practice. A post-hoc comparison of the mean annualized bleeding rates which required treatment with rurioctocog alfa detected a statistically significant difference (P < 0.0001) between patients treated on regular prophylaxis (8.5 bleeds/year) and patients treated on an on-demand basis (36.6 bleeds/year). Favorable prophylactic and on-demand hemostatic efficacy (“excellent” or “good”) were shown in 88.5–100% of patients across all treatment regimens. A total of 22 events of adverse drug reactions were reported in 13 male patients. Of the 352 patients, 3 (0.9%) patients, all of whom had ≤ 50 exposure days before enrollment, developed de novo FVIII inhibitor. No deaths or allergic reactions were reported. Rurioctocog alfa was found to be well-tolerated and effective among patients with hemophilia A in a postmarketing routine clinical practice.