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真实世界病例系列研究及目前文献综述：婴幼儿重型 A 型血友病伴抑制物患者接受艾美赛珠单抗预防治疗的情况。

Real-world case series and summary of current literature of infants and toddlers with severe hemophilia A with inhibitor on prophylaxis with emicizumab.

机构信息

UT Southwestern, Dallas, Texas, USA.

Children's Medical Center, Dallas, Texas, USA.

出版信息

Pediatr Blood Cancer. 2021 May;68(5):e28942. doi: 10.1002/pbc.28942. Epub 2021 Feb 8.

DOI:10.1002/pbc.28942
PMID:33559300
Abstract

Emicizumab is a recombinant, humanized, and a bispecific monoclonal antibody that bridges activated factor (F) IX and FX in place of FVIII to restore hemostasis in persons with hemophilia A (PHA). Data on the efficacy and safety of emicizumab in young children is limited. Immunologic naivety, physiologically decreased production of vitamin K dependent proteins, specifically FIX, and enhanced clearance of emicizumab in infants may support decreased emicizumab effectiveness. We report on the facilitation of care rendered by using emicizumab in young PHA with inhibitors and extend data on the efficacy and safety in PHA < 3 years.

摘要

依库珠单抗是一种重组的、人源化的双特异性单克隆抗体，可替代 FVIII 将激活的因子 (F) IX 和 FX 桥接在一起，以恢复血友病 A (PHA) 患者的止血功能。依库珠单抗在幼儿中的疗效和安全性数据有限。免疫幼稚、生理性降低维生素 K 依赖性蛋白（特别是 FIX）的产生以及婴儿中依库珠单抗的清除增强可能支持依库珠单抗效果降低。我们报告了使用依库珠单抗治疗患有抑制剂的年轻 PHA 患者的便利性，并扩展了 PHA < 3 岁患者的疗效和安全性数据。

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Emicizumab is well tolerated and effective in people with congenital hemophilia A regardless of age, severity of disease, or inhibitor status: a scoping review.
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