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首页> 外文期刊>Pediatric blood & cancer >Emicizumab prophylaxis among infants and toddlers with severe hemophilia A and inhibitors—a single‐center cohort
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Emicizumab prophylaxis among infants and toddlers with severe hemophilia A and inhibitors—a single‐center cohort

机译：婴儿和小孩中的eMicizumab预防血液干病A和抑制剂 - 单中心队列

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Abstract Background Emicizumab is a bispecific antibody that bridges factor IXa and factor X to restore hemostasis in patients with hemophilia A (HA). Its efficacy and safety have been proven in multicenter trials. However, real world data regarding its use in very young children are currently lacking. Ancillary test results for monitoring emicizumab's hemostatic effect and their clinical correlations are scarce. Methods Children with HA and inhibitors treated by emicizumab were prospectively followed at our center. Laboratory follow‐up included rotational thromboelastometry (ROTEM) and thrombin generation (TG), prior to and during treatment. Results Eleven children whose median age was 26 months were treated by emicizumab and followed for a median of 36?weeks. During follow‐up, none experienced hemarthrosis or any other spontaneous bleeds. For 7/11 patients, emicizumab prophylaxis was sufficient to maintain hemostasis without additional supplemental therapy. Only 4/11 patients were occasionally treated with recombinant activated FVII for trauma. Two minor surgeries were safely performed without supplemental therapy while another procedure was complicated by major bleeding. TG parameters improved for all patients, correlating with their clinical status. Interestingly, the lowest TG values were obtained for patients experiencing bleeding episodes, while ROTEM parameters in all patients were close to the normal range. Conclusions This study confirms the safety and efficacy of emicizumab in reducing bleeds in young children with HA with inhibitors, including infants. However, surgeries warrant caution as emicizumab prophylaxis may not be sufficient for some procedures. TG may more accurately reflect the hemostasis state than ROTEM in pediatric patients treated with emicizumab.
机译：摘要背景Emicizumab是一种双特异性抗体，其桥接因子IXA和因子X恢复血友病患者的止血（HA）。它的疗效和安全性已被证明在多中心试验中。然而，目前缺乏关于其在非常幼儿中使用的现实世界数据。监测Emicizumab止血效果的辅助测试结果及其临床相关性是稀缺的。方法在我们的中心前后遵循emicizumab的HA和抑制剂的儿童。在治疗之前和期间，实验室随访包括旋转血栓放气管（Rotem）和凝血酶产生（Tg）。结果Emicizumab中位年龄为26个月的11个儿童，并进行36个月的中位数。在随访期间，没有经历过的血液，或任何其他自发的流血。对于7/11患者，Emicizumab预防足以保持止血而无需额外的补充疗法。只有4/11患者偶尔治疗重组活化的FVII用于创伤。在没有补充疗法的情况下安全地进行两次轻微的手术，而另一种程序因重大出血而变得复杂。所有患者的TG参数改善，与其临床状态相关。有趣的是，为经历出血发作的患者获得最低TG值，而所有患者的Rotem参数接近正常范围。结论本研究证实了Emicizumab在患有抑制剂（包括婴儿）的HA患有HA的幼儿肠道的安全性和功效。但是，由于emicizumab预防可能不足以足够，因此手术谨慎。 TG可以更准确地反映与用Emicizumab治疗的儿科患者的旋转止血状态。

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