First reported use of immune checkpoint inhibitor for treatment of cancer in a patient with acquired hemophilia A

Mariam A Mostafa , Sheref Elseidy , Reham Ali Metwally , Amir Mahmoud , Ali Abdelhay , Farhan S. Imran
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Abstract

Acquired hemophilia A is a rare but serious bleeding disorder that occurs because of neutralizing autoantibodies, also called inhibitors that target coagulation factor VIII (FVIII). Although it is a rare disorder, it has high morbidity and mortality with serious, sometimes life-threatening bleeding, often occurring. Immunotherapy with immune checkpoint inhibitors (ICI) is now a key pillar in treatment of malignancies. They have improved outcomes in malignancy but given their mechanism of action, which stimulates the immune response, autoimmune-associated adverse effects are a concern. Several case reports have identified a risk of AHA occurrence in patients treated with ICI. There are no case reports documenting the use or outcomes of ICI in patients with pre-existing AHA. Here we present the first ever case of a patient with AHA in complete remission treated successfully with ICI for lung cancer without relapse in AHA.

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首次报道获得性血友病 A 患者使用免疫检查点抑制剂治疗癌症
获得性血友病 A 是一种罕见但严重的出血性疾病,是由于针对凝血因子 VIII(FVIII)的中和自身抗体(也称为抑制剂)引起的。虽然这是一种罕见的疾病,但它的发病率和死亡率很高,经常会发生严重出血,有时甚至危及生命。免疫检查点抑制剂(ICI)免疫疗法目前已成为治疗恶性肿瘤的关键支柱。它们改善了恶性肿瘤的治疗效果,但由于其作用机制是刺激免疫反应,因此与自身免疫相关的不良反应令人担忧。一些病例报告发现,接受 ICI 治疗的患者有发生 AHA 的风险。目前还没有病例报告记录 ICI 在已有 AHA 的患者中的使用情况或结果。在此,我们介绍了有史以来第一例完全缓解的 AHA 患者在接受 ICI 治疗肺癌后未复发 AHA 的成功病例。
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