INTRODUCTION:Patients affected by hemophilia A often require frequent prophylactic and therapeutic self-infusion. For those who develop inhibitors, treatment options are limited and mortality is increased. Emicizumab, a bispecific antibody to Factors IXa and X that carries out the function of Factor VIII (FVIII), represents a novel therapeutic approach. Areas covered: We review the clinical trials and key laboratory assay research for emicizumab. Emicizumab reduced the annualized bleeding rate by 87% compared to placebo in patients with inhibitors. For patients without inhibitors, emicizumab reduced the annualized bleeding rate 96-97% compared to no prophylaxis and 68% compared to prior FVIII prophylaxis. Three patients developed a thrombotic microangiopathy (TMA) and two patients had thrombotic events while on emicizumab in combination with activated prothrombin complex concentration (aPCC) alone or concurrent with activated recombinant factor FVII (rFVIIa). Expert opinion: Emicizumab represents a much-needed alternative approach to managing Factor VIII deficiency, especially for those with inhibitors or limited ability to self-infuse. For patients with inhibitors, thrombotic complications including TMA, not seen with other bypassing agents, raises concern about the use of emicizumab in combination with aPCC and how patients who have breakthrough bleeding can be safely managed.

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简介:受血友病A感染的患者通常需要频繁的预防和治疗性自我输注。对于那些产生抑制剂的人,治疗选择受到限制,死亡率增加。 Emicizumab是一种针对因子IXa和X的双特异性抗体,具有因子VIII(FVIII)的功能,代表了一种新颖的治疗方法。涵盖领域:我们回顾了艾米单抗的临床试验和关键实验室测定研究。与安慰剂相比,在使用抑制剂的患者中,Emicizumab的年出血率降低了87%。对于没有抑制剂的患者,艾米珠单抗与无预防措施相比,年出血率降低了96-97%,与先前的FVIII预防措施相比,降低了68%。 3例患者发生了血栓性微血管病(TMA),2例患者在使用艾米单抗联合单独使用活化凝血酶原复合物浓度(aPCC)或同时使用活化重组凝血因子FVII(rFVIIa)时发生了血栓事件。专家意见:Emicizumab是解决VIII因子缺乏症的一种急需的替代方法,特别是对于那些具有抑制剂或自我注入能力有限的患者。对于有抑制剂的患者,血栓并发症(包括TMA)(其他旁路药物未见)引起了人们对艾米单抗联合aPCC的使用以及如何安全治疗突破性出血的患者的关注。

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