重组人凝血因子Ⅸ预防性治疗中国儿童中、重型血友病B的单中心临床观察  

Efficacy and safety of recombinant coagulation factor IX in moderate and severe hemophilia B children in China ：a signal centre observation study

作　　者：李刚[1] 吴润晖[1] 吴心怡[1] 甄英姿[1] 张宁宁[1] 王岩[1] 何雯雯[1] 彭芸[1] LUKE Koon-hung POON Man-Chiu ANDREA Doria 

机构地区：[1]首都医科大学附属北京儿童医院血友病组,100045 [2]University of Ottawa, Children's Hospital of Eastern Ontario, Ottawa, Ontario, Canada [3]University of Calgary, Foothills Hospital, Calgary, Al- berta,Canada [4]University of Toronto, Sickkids Children's Hospital of Toronto,Toronto. Canada

出　　处：《中华实用儿科临床杂志》2013年第15期1128-1130,共3页Chinese Journal of Applied Clinical Pediatrics

摘　　要：目的应用重组人凝血因子Ⅸ（FIX）预防性治疗中国儿童中、重型血友病B，了解其疗效及安全性。方法使用重组人FIX开展儿童血友病B小剂量预防治疗，重组人FIX20IT]／（kg·次），1次／周，I临床观察指标包括：（1）疗效：出血减少率、日常活动参与改善情况；（2）安全性：用药中不良事件及抑制物产生情况。结果共收集11例病例，观察时间18～27周（平均25．5周）；年龄9个月～15岁（平均7岁1个月）；重型9例，中间型2例；开始观察前接受按需治疗6例，预防治疗4例，无治疗1例；中位暴露日（ED）63ED（范围0～〉150ED）。（1）疗效：总出血减少率67．7％（与原按需治疗／无治疗比较减少78．6％；与原预防治疗比较减少47．8％）；9例中8例日常活动参与改善（与原按需／无治疗比较6例中5例改善，与原预防治疗比较3例全部改善）；（2）安全性：应用过程中无不良事件发生；治疗过程中仅有1例有1次抑制物检测滴度达0．6BU／mL，治疗结束后降至0BU／mL。结论重组人FIX应用于中国中、重型血友病B儿童的小剂量预防治疗安全、有效。Objective To explore the efficacy and safety of application of recombinant coagulation factor IX （FIX） in Chinese severe and moderate hemophilia B children for low-dose prophylaxis. Methods FIX for low-dose prophylaxis 20 IU/kg, once a week for severe and moderate hemophilia B children in Beijing Children＇s Hospital. （1） Efficacy： reduction of bleeding, improvement of daily life participation; （2） Safety： the side-effect during the recombi- nant coagulation FIX intervention and monitor the inhibitor during the prophylaxis period. Results There were 11 ana- lyzable cases, the duration was 18 -27 weeks （mean 25.5 weeks） ;9-month to 15-year-old （mean 7 year and 1 month） ; 9 severe cases and 2 moderate cases;6 cases received on-demand,4 cases received prophylaxis intervention methods and 1 case without any treatment before the observation,median exposure day was（ED） 63 ED（ range 0 to 〉 150 ED）. （ 1 ） Efficacy：the total reduction of bleeding was 67.7% （78.6% for original on-demand treatment and 47.8% for origi- nal prophylaxis cases） ;8/9 cases daily life participation improved （5/6 cases for original on-demand or without treat- ment and 3/3 cases for original prophylaxis treatment） ;（2） Safety：there was no side-effect event during the recombi- nant coagulation FIX intervention and only 1 case had a 0.6 BU/mL transit inhibitor presented and low-downed to 0 BU/mL in the end of observation for all cases. Conclusion Low-dose prophylaxis with recombinant coagulation FIX is a safe and effective treatment for Chinese pediatric patients with severe and moderate hemophilia B.

关 键 词：血友病B 重组人凝血因子IX 预防治疗 儿童 

分 类 号：R725.5[医药卫生—儿科]