血栓弹力图评估艾美赛珠单抗治疗后重型血友病A患儿凝血功能的单中心研究  

Application value of thromboelastography in assessing coagulation function in children with severe hemophilia A after emicizumab therapy:a single-center study

作　　者：彭冬 王缨[2] 周桂池 李倩 罗美珠 罗丽萍 邝雅贤 付笑迎[1] PENG Dong;WANG Ying;ZHOU Gui-Chi;LI Qian;LUO Mei-Zhu;LUO Li-Ping;KUANG Ya-Xian;FU Xiao-Ying(Department of Laboratory Medicine,Shenzhen Children's Hospital,Shenzhen,Guangdong 518038,China)

机构地区：[1]深圳市儿童医院医学检验科,广东深圳518038 [2]深圳市儿童医院血液肿瘤科,广东深圳518038

出　　处：《中国当代儿科杂志》2025年第3期293-299,共7页Chinese Journal of Contemporary Pediatrics

基　　金：广东省高水平医院建设专项基金;深圳市医疗卫生“三名工程”(SZSM202211033)。

摘　　要：目的探讨血栓弹力图(thromboelastography,TEG)在评估重型血友病A(hemophilia A,HA)患儿接受艾美赛珠单抗(emicizumab,EMI)治疗后凝血功能中的应用价值。方法回顾性分析2023年1月—2024年7月于深圳市儿童医院接受EMI治疗的17例重型HA患儿在用药前后活化部分凝血活酶时间(activated partial thromboplastin time,APTT)和TEG的检测结果,并对凝血因子Ⅷ(factorⅧ,FⅧ)等效活性与TEG检测中的反应时间(reaction time,R)进行相关性分析。结果重型HA患儿接受EMI治疗后平均年出血率为1.6次,15例(88%)患儿为零自发性出血、零关节出血。EMI治疗后患儿APTT较治疗前缩短(P<0.05),且低于正常对照组(P<0.05);治疗后APTT与FⅧ等效活性无相关性(P>0.05)。EMI治疗后,TEG检测中的R、动力学时间、α角、最大振幅、血块强度和凝血指数等参数均由治疗前的低凝状态转为趋于正常(P<0.05),且R值与FⅧ等效活性具有强负相关性(r=-0.758,P<0.05)。结论重型HA患儿接受EMI治疗后的出血状况得到有效控制,常规APTT检测不能反映真实的凝血功能,TEG检测在EMI治疗重型HA患儿的凝血功能评估中具有一定的临床应用价值。Objective To investigate the application value of thromboelastography(TEG)in assessing coagulation function in children with severe hemophilia A(HA)after emicizumab(EMI)therapy.Methods A retrospective analysis was performed on the activated partial thromboplastin time(APTT)and TEG testing results of 17 children with severe HA before and after EMI treatment at Shenzhen Children's Hospital from January 2023 to July 2024.Correlation analysis was conducted between coagulation factor VIII(FVIII)equivalent activity and reaction time(R value)measured by TEG.Results After EMI treatment,the mean bleeding rate for children with severe HA was 1.6 events per year,with 15 children(88%)without spontaneous bleeding or joint bleeding.The children with severe HA showed a significant reduction in APTT after EMI treatment(P<0.05),with a significantly shorter APTT than the normal control group(P<0.05).There was no correlation between APTT and FVIII equivalent activity after treatment(P>0.05).After EMI treatment,TEG parameters,including R value,kinetic time,alpha angle(α),maximum amplitude,clot strength,and coagulation index,shifted from a hypocoagulable state before treatment to a nearly normal state after treatment(P<0.05).The R value demonstrated a strong negative correlation with FVIII equivalent activity(r=-0.758,P<0.05).Conclusions The bleeding condition of children with severe HA can be effectively controlled after EMI treatment.Routine APTT testing cannot reflect true coagulation function,whereas TEG testing is clinically valuable in assessing the coagulation function of children with severe HA undergoing EMI treatment.

关 键 词：血友病A 艾美赛珠单抗 血栓弹力图 实验室检测 儿童 

分 类 号：R72[医药卫生—儿科]