Japanese Journal of Thrombosis and Hemostasis

Turoctocog alfa, a B-domain truncated recombinant factor VIII was developed as a 3rd generation product. A multi-center phase 3 trial including previously treated patients with severe hemophilia A above 12 years of age was conducted. The objectives of the trial were to demonstrate safety and efficacy of turoctocog alfa in treatment of episodic bleedings, in regular replacement therapy and in bleeding prevention during surgery. Total of 150 patients from 15 countries including 9 Japanese patients received regular replacement therapy. The annualized bleeding rate (median, bleeds/patient/year) was 3.66 in total patients and 0 in Japanese group. Of the 499 reported bleeds during the regular replacement therapy, 89.4% were stopped within two infusions of turoctocog alfa, in which all 6 bleeds of Japanese subjects were controlled with one infusion. The success rate for treatment of bleeds was 84.5% in total patients and was 100% in Japanese group. No inhibitors were detected and no clinically significant safety issues were identified during the study. In conclusion, turoctocog alfa was safe and effective in regular replacement therapy and treatment of bleeding episodes in patients with hemophilia A, providing consistent results between total patients and Japanese group.

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The Great East Japan Earthquake of March 11, 2011 destroyed the social infrastructures underlying pharmaceutical supplies and hospital functions that were indispensable to livelihood and medical care of patients with hemophilia. This study is a survey on disaster experiences and network states of the hemophilic patients who confronted the earthquake disaster. Subjects were 62 patients who answered the questionnaire survey conducted in March, 2012; 18 aged 20 years or younger and 44 aged 21 years or older; 13 residents in Iwate, 43 in Miyagi and 3 in Fukushima prefectures, respectively. Immediate damages such as injury or medicinal loss were experienced by approximately 10% of patients, but their troubles were exacerbated in poor evacuation surroundings. A half experienced difficulties in commuting, bleeding symptoms, drug obtainment, or home-injection and, even one year later, approximately 10% of patients still complained physical troubles and/or psychological difficulties. Their network of hospitals worked usefully, whereas networks of surrounding people, other hemophiliac or pharmaceutical company were useful as well as requisite. There observed a positive relation among these networks in regard to their good or bad judge. This survey record would be of valuable help for hemophilic patients or medical experts to prepare possible disasters in advance.

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