Clinical characteristics and outcomes of acquired hemophilia A before and after emicizumab approval in Japan

Blood Vessels, Thrombosis & Hemostasis Pub Date : 2024-09-16 DOI:10.1016/j.bvth.2024.100027

Daichi Kishi , Masashi Nishikubo , Yoshimitsu Shimomura , Takayuki Ishikawa , Tadakazu Kondo

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Abstract

Acquired hemophilia A (AHA) is a rare and potentially fatal bleeding disorder. Although bypassing agents effectively control active bleeding, their disadvantages, such as high cost and frequent administration, necessitate an agent that prevents recurrent bleeding events requiring bypassing agents. Emicizumab, a recombinant, humanized, bispecific monoclonal antibody with coagulation factor VIII (FVIII)–mimetic activity, was approved in Japan in 2022 for preventing bleeding in patients with AHA. However, owing to the rarity of the disease, real-world data on emicizumab use in AHA are scarce. Therefore, we aimed to assess the clinical characteristics and outcomes of 19 patients who were newly diagnosed with AHA before (non-emicizumab group [non-emi group], n = 12) and after (emicizumab group [emi group], n = 7) emicizumab approval in Japan. The median age, FVIII coagulation activity, and FVIII inhibitor titer were 81 vs 76 years, 1.0% vs 1.0%, and 43.75 vs 622 Bethesda units per mL in the non-emi and emi groups, respectively. Severe bleeding occurred in 14% of patients in the emi group, compared with 58% of patients in the non-emi group. Additionally, the doses of bypassing agents per patient were 43.4 vs 7, and the units of red blood cell transfusion per patient were 26.7 vs 4 in the non-emi and emi groups, respectively. Their hospital stays were median 73.5 days and 44 days, respectively. All patients treated with emicizumab maintained their activities of daily living (ADLs) and experienced no side effects. This study suggests that emicizumab effectively prevents bleeding in patients with AHA. Moreover, emicizumab may lead to shorter hospital stays, maintained ADLs, reduced costs, and improved prognosis in patients with AHA.

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日本批准埃米珠单抗前后获得性 A 型血友病的临床特征和治疗结果

摘要后天获得性血友病 A（AHA）是一种罕见且可能致命的出血性疾病。虽然旁路药物能有效控制活动性出血，但由于其成本高、用药频繁等缺点，需要一种药物来预防需要旁路药物的反复出血事件。Emicizumab 是一种具有凝血因子 VIII（FVIII）模拟活性的重组人源化双特异性单克隆抗体，于 2022 年在日本获批用于预防 AHA 患者出血。然而，由于这种疾病的罕见性，有关埃米珠单抗用于 AHA 的实际数据很少。因此，我们旨在评估日本批准埃米珠单抗之前（非埃米珠单抗组[non-emicizumab group]，n=12）和之后（埃米珠单抗组[emicizumab group]，n=7）19 名新诊断为 AHA 患者的临床特征和预后。非埃米组和埃米组的中位年龄、FVIII 凝血活性和 FVIII 抑制剂滴度分别为 81 岁对 76 岁、1.0% 对 1.0%、43.75 对 622 贝塞斯达单位/毫升。埃米组有 14% 的患者发生严重出血，而非埃米组有 58% 的患者发生严重出血。此外，非埃米组和埃米组每名患者使用旁路药物的剂量分别为 43.4 vs 7，输注红细胞的单位分别为 26.7 vs 4。他们的中位住院时间分别为 73.5 天和 44 天。所有接受埃米珠单抗治疗的患者都能保持日常生活能力（ADL），并且没有出现副作用。这项研究表明，埃米珠单抗能有效预防 AHA 患者出血。此外，埃米珠单抗还可缩短 AHA 患者的住院时间、维持日常生活能力、降低费用并改善预后。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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Blood Vessels, Thrombosis & Hemostasis

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