Launois Amélie, Martin-Toutain Isabelle, Devaux Floriane, Lambert Juliette, Longval Thomas, Merabet Fatiha, Jaidi Rym, Le Dore Sophie, Ferre Emmanuelle, Rousselot Philippe, De Raucourt Emmanuelle, Flaujac Claire
Laboratoire de biologie médicale, Centre Hospitalier de Versailles, 122 rue de Versailles, 78157 Le Chesnay-Rocquencourt, France.
Centre de ressources et compétences maladies hémorragiques, Centre Hospitalier de Versailles, 122 rue de Versailles, 78157 Le Chesnay-Rocquencourt, France.
Ann Biol Clin (Paris). 2024 Aug 30;82(3):294-307. doi: 10.1684/abc.2024.1900.
Emicizumab is a bispecific antibody that mimics the function of factor VIII (FVIII) and is indicated for prophylactic use in patients with congenital hemophilia A with or without inhibitors. Acquired hemophilia A (AHA) is a rare and severe disorder causes by autoantibodies that inhibit FVIII. In AHA, acute bleeding are managed with bypassing agents but several reports described the off-label use of emicizumab. The aim of this article is to describe two cases of AHA treated with emicizumab and a review of the scientific littérature. Reports indicate that the use of emicizumab is efficacious to treat acute bleeding with less thrombotic events thant with bypassing agents and with a reduced hospitalisation duration. Nevertheless biological monitoring is more complicated with assay interferences and a persistent circulation more than 6 months after the last injection was observed for our two patients.
艾美赛珠单抗是一种双特异性抗体,可模拟凝血因子VIII(FVIII)的功能,适用于有或无抑制剂的先天性血友病A患者的预防性治疗。获得性血友病A(AHA)是一种由抑制FVIII的自身抗体引起的罕见且严重的疾病。在AHA中,急性出血用旁路制剂治疗,但有几份报告描述了艾美赛珠单抗的超说明书使用情况。本文旨在描述两例用艾美赛珠单抗治疗的AHA病例,并对科学文献进行综述。报告表明,使用艾美赛珠单抗治疗急性出血有效,与旁路制剂相比,血栓形成事件更少,住院时间缩短。然而,生物监测因检测干扰而更为复杂,我们的两名患者在最后一次注射后6个月以上观察到持续循环。