1.A phase Ⅲ multi-center clinical trial on safety and efficacy of a domestic plasma derived factor Ⅸ for the treatment of patients with hemophilia B.
Wei LIU ; Rong Feng FU ; Ya Wei ZHOU ; Yun CHEN ; Jie YIN ; Zi Qiang YU ; Lin Hua YANG ; Mei Fang WANG ; Hui BI ; Ze Ping ZHOU ; Xin Sheng ZHANG ; Jie Lai XIA ; Ren Chi YANG
Chinese Journal of Hematology 2018;39(5):404-407
Objective: To evaluate the efficacy and safety of a domestic human plasma derived coagulation Factor Ⅸ concentrate (pd-FⅨ) in patients with hemophilia B. Methods: The study was a multicenter, open-label and single-arm study. The efficacy of pd-F Ⅸ was evaluated by objective performance criteria. The doses of pd-FⅨ were calculated according to the bleeding symptom and disease severity. The infusion efficiency of pd-FⅨ and improvement of bleeding symptoms were measured at 30 minutes and (24±4) h after the first infusion, respectively. Adverse events were recorded. Viral infection and FⅨ inhibitor were detected 90 d after the first infusion. Results: All 36 subjects with hemophilia B were enrolled in the study. The median age of these patients was 31 years old and the median injection doses were 4 (1-17) times. The hemostatic effect of 27/36 (75.00%) and 9/36 (25.00%) acute bleeding events were rated as "excellent" and "better" , respectively. The recovery rate was 111.92% (65.55%-194.28%) at 30 minutes after infusion of FⅨ. There was no adverse event related to FⅨ. No reactivation of HBV, HCV or HIV and FⅨ inhibitor was detected at 90-104 d after the first FⅨ infusion. Conclusion: This domestically made human plasma derived FⅨ concentrate is safe and effective in the treatment of acute bleeding in patients with hemophilia B. Clinical trial registration: China food and Durg Administration, 2016L08027.
Adult
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China
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Factor IX
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Hemophilia A
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Hemophilia B/therapy*
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Hemorrhage
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Humans
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Plasma