重型血友病A患儿19例个体化初级预防治疗效果分析  

Analysis of individualized primary prophylactic treatment of 19 cases of children with severe hemophilia A

作　　者：刘国青[1] 唐凌[1] 吴心怡[1] 甄英姿[1] 李刚[1] 陈振萍[1] 王岩[2] 张宁宁[3] 张纪水[4] 于国霞[5] 吴润晖[1] Liu Guoqing Tang Ling Wu Xinyi Zhen Yingzi Li Gang Chen Zhenping Wang Yan Zhang Ningning Zhang Jishui Yu Guoxia Wu Runhui(Hematology Oncology Center, Beijing Children＇s Hospital Affiliated to Capital Medical University, Beijing 100045, China)

机构地区：[1]首都医科大学附属北京儿童医院血液肿瘤中心,100045 [2]首都医科大学附属北京儿童医院康复科,100045 [3]首都医科大学附属北京儿童医院影像中心,100045 [4]首都医科大学附属北京儿童医院心理科,100045 [5]首都医科大学附属北京儿童医院口腔科,100045

出　　处：《中华儿科杂志》2016年第12期923-926,共4页Chinese Journal of Pediatrics

基　　金：国家自然科学基金（81341018）;首都卫生发展科研专项（2014-2-2092）;血友病疾病管理系统项目（IHECC2014HEM04）

摘　　要：目的 了解中国重型血友病A患儿初级预防治疗现状,探讨合理的治疗方案,为开展初级预防治疗提供依据.方法 回顾性收集201 1年2月至2015年9月在北京儿童医院血友病门诊接受初级预防的19例重型血友病A患儿病例资料并对方案实施、疗效进行评估.结果 （1）初级预防方案：开始中位年龄1.8（0.5 ～2.9）岁;中位凝血因子（FⅧ制剂）剂量16.7（8.0 ～23.5） U/（kg.次）;l中位注射频率1.0（1.0～3.0）次/周.其中8例因出血控制不满意曾进行预防治疗升级.（2）疗效：中位年总出血频率（ABR）为1.9（0 ～6.0）次/年,中位年关节出血频率（AJBR）为0（0 ～3.3）次/年,严重出血0次.所有病例保持北京儿童医院日常活动4级;中位年凝血因子消耗量为1 844（840 ～5 040）U/（kg·年）.结论 小剂量、低频次并根据出血情况进行调整的初级预防治疗模式可有效降低血友病A患儿的ABR及MBR,保持患儿正常生活能力,减少因子用量.Objective To study the current situation of primary prophylaxis in severe hemophilia A children and to explore rational regimen in order to provide evidence for the development of primary prophylaxis in China.Method A retrospective clinical data collection and analysis was conducted for 19 severe hemophilia A children who received primary prophylaxis in Beijing Children＇s Hospital outpatient clinic between February 2011 and September 2015 and evaluated the regimen and efficacy.Result （1） Primary prophylaxis regimen：the median beginning age 1.8 （range 0.5-2.9） years,the median F Ⅷ preparation using dosage 16.7 （8.0-23.5） U/（kg · time）,the median using frequency was 1.0 （1.0-3.0） time/week.Eight cases among the patients received escalation of treatment intensity because of the poor bleeding control.（2） Efficacy：the median annual bleeding rate （ABR） was 1.9 （0-6.0） times/ year,the median annual joint bleeding rate （AJBR） was 0 （0-3.3） times/year,without life threatening bleeding.All of them kept in 4th scale of Beijing Children Hospital daily activity level.The median annual factor consumption was 1 844 （840-5 040） U/kg.Conclusion Low-dose primary prophylaxis regimen which were in low-dose/low frequencies and adjusted by bleeding frequency could decrease bleeding and joint bleeding frequency significantly,maintained the normal daily activity capacity and saved the factor consumption compared to standard regimen in severe hemophilia A children.

关 键 词：儿童 出血 

分 类 号：R725.5[医药卫生—儿科]