Hansenne Amandine, Hermans Cedric
Division of Haematology, Hemostasis-Thrombosis Unit, Cliniques universitaires Saint-Luc, Université catholique de Louvain (UCLouvain), Brussels, Belgium.
Division of Haematology, Hemostasis-Thrombosis Unit, Cliniques universitaires Saint-Luc, Université catholique de Louvain (UCLouvain), 10 avenue Hippocrate, 1200 Brussels, Belgium.
Ther Adv Hematol. 2021 Aug 28;12:20406207211038193. doi: 10.1177/20406207211038193. eCollection 2021.
Acquired haemophilia A (AHA) is a rare and severe haemorrhagic autoimmune disease caused by autoantibodies directed against factor VIII (FVIII). Treatment is based on two principles, including haemostatic control to compensate FVIII inhibition and eradication of inhibiting antibodies using immunosuppressive therapy. Rapid recognition and proper management are essential to avoid excess morbidity and mortality. Effective and safe treatments can be challenging, given that AHA patients are often elderly, with multiple comorbidities. Emicizumab, a bispecific antibody that mimics the action of FVIII, has proven effective in managing patients with congenital haemophilia, with or without inhibitors. Likewise, its mode of action suggests theoretical efficacy in AHA patients. We herein describe two AHA cases with comorbidities that were treated effectively using emicizumab combined with immunosuppressive therapy. We have also reviewed the current literature regarding the promising use of emicizumab in this indication.
获得性血友病A(AHA)是一种罕见且严重的出血性自身免疫性疾病,由针对凝血因子VIII(FVIII)的自身抗体引起。治疗基于两个原则,包括通过止血控制来补偿FVIII抑制以及使用免疫抑制疗法消除抑制性抗体。快速识别和妥善管理对于避免过高的发病率和死亡率至关重要。鉴于AHA患者通常年事已高且合并多种疾病,有效且安全的治疗可能具有挑战性。艾美赛珠单抗是一种模拟FVIII作用的双特异性抗体,已被证明在治疗有或无抑制剂的先天性血友病患者方面有效。同样,其作用方式表明对AHA患者具有理论疗效。我们在此描述了两例合并多种疾病的AHA病例,这些病例使用艾美赛珠单抗联合免疫抑制疗法得到了有效治疗。我们还回顾了当前关于艾美赛珠单抗在该适应症中应用前景的文献。