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[血栓弹力图在评估重度甲型血友病患儿接受艾美赛珠单抗治疗后凝血功能中的应用价值:一项单中心研究]

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

作者信息

Peng Dong, Wang Ying, Zhou Gui-Chi, Li Qian, Luo Mei-Zhu, Luo Li-Ping, Kuang Ya-Xian, Fu Xiao-Ying

机构信息

Department of Laboratory Medicine, Shenzhen Children's Hospital, Shenzhen, Guangdong 518038, China.

出版信息

Zhongguo Dang Dai Er Ke Za Zhi. 2025 Mar 15;27(3):293-299. doi: 10.7499/j.issn.1008-8830.2409099.

Abstract

OBJECTIVES

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 (<0.05), with a significantly shorter APTT than the normal control group (<0.05). There was no correlation between APTT and FVIII equivalent activity after treatment (>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 (<0.05). The R value demonstrated a strong negative correlation with FVIII equivalent activity (=-0.758, <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.

摘要

目的

探讨血栓弹力图(TEG)在评估重度甲型血友病(HA)患儿接受依美珠单抗(EMI)治疗后凝血功能中的应用价值。

方法

对2023年1月至2024年7月在深圳市儿童医院接受EMI治疗的17例重度HA患儿治疗前后的活化部分凝血活酶时间(APTT)及TEG检测结果进行回顾性分析。对TEG测定的凝血因子Ⅷ(FVIII)等效活性与反应时间(R值)进行相关性分析。

结果

EMI治疗后,重度HA患儿的平均出血率为每年1.6次,15例患儿(88%)无自发性出血或关节出血。重度HA患儿经EMI治疗后APTT显著降低(<0.05),且明显短于正常对照组(<0.05)。治疗后APTT与FVIII等效活性之间无相关性(>0.05)。EMI治疗后,TEG参数,包括R值、动态时间、α角(α)、最大振幅、凝血强度和凝血指数,从治疗前的低凝状态转变为治疗后的近正常状态(<0.05)。R值与FVIII等效活性呈强负相关(=-0.758,<0.05)。

结论

重度HA患儿经EMI治疗后出血情况可得到有效控制。常规APTT检测不能反映真实的凝血功能,而TEG检测在评估接受EMI治疗的重度HA患儿的凝血功能方面具有临床价值。

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Emicizumab: the hemophilia A game-changer.依美德珠单抗:血友病 A 的变革者。
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