Objective: To evaluate the efficacy and safety of homemade plasma derived coagulation factor Ⅷ in patients with hemophilia A.
Methods: Patients with congenital hemophilia A who met the inclusive and exclusive criteria were enrolled in the study after informed consent. The doses of factor Ⅷ were calculated according to the weight, disease severity etc. FⅧ activity and infusion efficacy value at 10 min and 60 min after infusion were recorded, as well as adverse events and validity rating according to the improvement of clinical syndromes. Viral infections including HBV, HCV, HIV and FⅧ inhibitor were determined after 3 and 6 months.
Results: A total of 65 patients were enrolled in this study, all of whom were evaluable for drug safety. Treatment efficacy was evaluated in 60 patients and 57 cases completed the trial finally. In this 57 cases, most (52/57) subjects were of middle and severe hemophilia A mainly characterized by joint bleeding. Overall response rate of acute bleeding events was classified as "excellent" (70.00%) or "better" (30.00%). The non-responder was 0. FⅧ activity and infusion efficiency value of first administration after 10 min and 60 min improved significantly [10 min: (123.66±47.54)%; 60 min: (108.05±43.24)%]. The incidence of adverse events was 1.54%. Neither allergic reaction nor reactivation of HBV, HCV, HIV was detected after treatment of 3 and 6 months. No FⅧ inhibitor negative patients converted to positive during follow-up.
Conclusion: This homemade plasma derived coagulation factor Ⅷ is safe and effective for the treatment of acute bleeding in patients with hemophilia A.