インヒビター保有血友病A患者に対する乾燥濃縮ブタ血液凝固第VIII因子製剤「MTI-9002(Hyate: C)」の臨床評価

抄録

The efficacy, safety and usefulness of a highly purified concentrate of porcine factor VIII (MTI-9002; Hyate: C) was evaluated in 30 treatment episodes with 12 hemophilia A patients having inhibitor to human factor VIII in 11 institutes in Japan. A total of 44 infusions was given. Results were as follows.
1. The hemostatic efficacy was evaluated at 8 hours and 24 hours after the first infusion in each treatment episode. In 22 of 30 episodes (73.3%), excellent to good responses were obtained at both times. General hemostatic efficacy of each episode was also evaluated. In 26 of 30 episodes (86.7%), excellent to good responses were obtained.
2. When more units were infused than the calculated neutralizing dose based on the titer of porcine factor VIII inhibitor at the time of pre-infusion, an adequate rise in factor VIII: C was almost always seen after infusion of MTI-9002. However, even if factor VIII: C level remained low, desired hemostasis was occasionally obtained.
3. There were 8 infusions for 8 treatment episodes in 5 patients where a definite relationship between MTI-9002 and adverse reactions could not be eliminated. Since those patients treated with hydrocortisone responded well soon, it seemed that adverse reactions could be prevented and treated with hydrocortisone.
4. For patients who have difficulty controlling bleeding and low inhibitor cross-reactivity, it was felt that MTI-9002 was extremely useful.