Emicizumab治疗血友病A

Q4 Dentistry
I. Tijanić, Ivana Golubović, M. Vučić, M. Tijanić
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引用次数: 0

摘要

近几十年来,尽管使用浓缩因子VIII制剂治疗A型血友病取得了实质性进展,但出血事件仍然不时发生。抑制剂的发展显著降低了传统替代疗法的疗效,严重增加了这些患者的发病率和死亡率。Emicizumab (HEMLIBRA®)是一种嵌合双特异性人源化抗体,可桥接活化的FIX和FX,从而恢复缺失的活化FVIII的功能。目的:本研究的目的是分析Emicizumab治疗a型血友病疗效的文献资料。结果:多中心随机研究(称为HAVEN)显示,该药物在治疗a型血友病患者方面取得了优异的效果。FVIII抑制剂不与Emicizumab结合或中和,因此对药物的止血活性没有影响。与使用旁路药物预防的患者组相比,Emicizumab预防治疗的出血显著减少79%,而经过更长的观察后,这一比例甚至上升到95%。其他研究也证实了良好的治疗效果和对成人和儿童有利的安全性。在出血事件或准备立即手术干预的情况下,建议根据先前的指南使用FVII (NovoSeven)®。结论:迄今为止,emicizumab的预防性使用结果表明,它可能是一种革命性的制剂,可以显著减少出血发作,改善a型血友病患者的生活质量。然而,该药物还需要进一步的测试。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Emicizumab in the treatment of hemophilia A
Introduction: Despite substantial advances in the treatment of Hemophilia A with the use of concentrated factor VIII preparations during recent decades, bleeding episodes still occur from time to time. The development of inhibitors significantly reduces the efficacy of traditional replacement therapy, seriously increasing morbidity and mortality in these patients. Emicizumab (HEMLIBRA ®) is a chimeric bispecific humanized antibody that bridges activated FIX and FX and thus restores the function of missing activated FVIII. Aim: The aim of the study was to analyze the literature date of the effect of Emicizumab in the treatment of Hemophilia A. Results: Multicenter randomized studies called HAVEN have shown excellent results of this medication in the treatment of patients with Hemophilia A. FVIII inhibitors do not bind to or neutralize Emicizumab and therefore have no effect on the hemostatic activity of the drug. Emicizumab prophylaxis produced a significant reduction in treated bleedings of 79%, compared to with the group of patients on prophylaxis with bypassing agents, while the rate grew up to even 95% after longer observation. Other studies have also confirmed good treatment results and a favorable safety profile in both adults and children. In the cases of bleeding events or preparation for immediate surgical interventions, it is recommended to user FVII (NovoSeven) ® according to previous guidelines. Conclusion: The results of the prophylactic use of Emicizumabhave so far shown that it may be a revolutionary preparation that can significantly reduce bleeding episodes and improve the quality of life of patients with Hemophilia A. Nevertheless, further testing of this drug is required.
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来源期刊
Acta Stomatologica Naissi
Acta Stomatologica Naissi Dentistry-Dentistry (all)
CiteScore
0.10
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0.00%
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审稿时长
4 weeks
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