International Journal of Hematology

DOI: 10.1007/s12185-018-02574-x Pages: 336-345

Inhibitor development, safety and efficacy of Advate® among previously treated patients with hemophilia A in a postmarketing surveillance in Japan

1. Tokyo Medical University Hospital, Department of Laboratory Medicine

2. St. Marianna University School of Medicine Yokohama City Seibu Hospital, Department of Pediatrics

3. Nagoya University Hospital, Department of Transfusion Medicine

4. Nara Medical University, Department of Pediatrics

5. The Japanese Red Cross Tokai Hokuriku Block Blood Center

6. Shire, Department of Clinical Scientific Writing

7. Shire, Department of Medical Affairs

8. Shire, Department of Biostatistics

9. Kitakyushu Yahata-Higashi Hospital

Correspondence to:
Katsuyuki Fukutake
Tel: (81) 3-3342-6111



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.

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