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
10.3760/cma.j.issn.0253-2727.2018.05.011
- VernacularTitle: 国产血源性人凝血因子Ⅸ治疗血友病B的多中心Ⅲ期临床研究
- Author:
Wei LIU
1
;
Rongfeng FU
;
Yawei ZHOU
;
Yun CHEN
;
Jie YIN
;
Ziqiang YU
;
Linhua YANG
;
Meifang WANG
;
Hui BI
;
Zeping ZHOU
;
Xinsheng ZHANG
;
Jielai XIA
;
Renchi YANG
1
Author Information
1. Institute of Hematology & Blood Diseases Hospital, CAMS & PUMC, Tianjin 300020, China
- Publication Type:Journal Article
- Keywords:
Hemophilia B;
Factor Ⅸ;
Clinical Trial, Phase Ⅲ
- From:
Chinese Journal of Hematology
2018;39(5):404-407
- CountryChina
- Language:Chinese
-
Abstract:
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.
