Surveillance study was performed in 24 (severe 10, moderate 6, and mild 8) previously untreated patients with hemophilia A to evaluate the safety and efficacy of plasma- derived factor VIII (CROSS EIGHT M, Japanese Red Cross) which has been widely used in Japan. In a total of 534 bleeding episodes during the study, 226 and 138 were judged to be “excellent” and “good”in hemostatic efficacy, respectively. The overall efficacy rate including more than “good” reached 96.4% without any “none” or “worse” cases. One severe patient transiently developed 0.9 BU/ml of inhibitor to factor VIII. Another severe patient developed transient anti-mouse IgG antibody did not show clinical symptoms. Regarding virus transmission, no HBV, HCV or HIV infection was reported, but human parvovirus B19 transmission through this product can not be excluded. The manufacturer has been improving the safety measures against this virus using a screening test by receptor-mediated hemagglutination (RHA) and introduction of nanofiltration during the manufacturing. These results indicate that CROSS EIGHT M has high ability in hemostatic efficacy and low activity in adverse reaction and inhibitor formation.

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The efficacy and the safety of a recombinant activated factor VII preparation (rFVIIa, NovoSeven®) were retrospectively analyzed in 13 patients with congenital factor VII(FVII) deficiency including 6 males and 7 females. Their age ranged from 60 days after birth to 78 years, and their plasma FVIIc levels were <3% in 7, 3-10% in 2 and > 10 in 4 patients. The bleeding episodes included hypermenorrhea in 2, joint bleeding in 3, bleeding related to minor surgery in 2 and major surgery in 6 patients. The rFVIIa was administered at an initial dose of 10-35 μg/kg of body weight and this dose was further added at every 2-8 hours as required to achieve efficient hemostasis. The rFVIIa preparation was found to be effective for the control of bleedings in all these patients, and no severe adverse events including thromboembolic complications were recorded except abnormal laboratory findings noted in a single patient. In Europe and USA, the dose of 15-30 μg/kg of body weight has been recommended for the control of bleeding in patients with FVII deficiency, and this recommendation seems to be legitimate based on our analysis data. The efficacy and the safety of this rFVIIa preparation were confirmed in this study as well.

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