Background Primary analysis of the phase III AGEHA study suggested a favorable benefit–risk profile for emicizumab prophylaxis in patients with acquired hemophilia A (PwAHA); however, only patients undergoing immunosuppressive therapy (IST; Cohort 1) were included.

Objectives To present final analysis results of AGEHA, including data on IST-ineligible patients (Cohort 2) and on long-term prophylaxis with emicizumab.

Methods For patients in both Cohorts 1 and 2, emicizumab was administered subcutaneously at 6 mg/kg on Day 1, 3 mg/kg on Day 2, and 1.5 mg/kg once weekly from Day 8 onward.

Results Twelve patients (Cohort 1) and two patients (Cohort 2) were enrolled. Duration of emicizumab treatment was 8 to 639 days (median: 44.5 days) in Cohort 1 and 64 and 450 days in Cohort 2. In both cohorts, no major bleeds were observed after initial emicizumab administration. Six patients started their first rehabilitation sessions during emicizumab treatment and no rehabilitation-related bleeds occurred. Twenty-three surgeries were performed under emicizumab prophylaxis and there were no bleeds related to surgeries. Although asymptomatic deep vein thrombosis was reported in one patient in the primary analysis, no other thrombotic events occurred thereafter. Two patients developed anti-emicizumab antibodies, one of whom showed accelerated emicizumab clearance. Tailored IST approaches (delayed initiation, no use, or reduced dose) were successfully executed in three patients undergoing emicizumab prophylaxis.

Conclusion These results suggest that emicizumab prophylaxis has a favorable benefit–risk profile in PwAHA regardless of eligibility for IST.

背景III 期 AGEHA 研究的初步分析表明，对于获得性 A 型血友病 (PwAHA) 患者，艾美珠单抗预防具有良好的获益-风险特征；然而，仅纳入接受免疫抑制治疗的患者（IST；队列 1）。

目的呈现 AGEHA 的最终分析结果，包括不符合 IST 条件的患者（队列 2）和艾美珠单抗长期预防的数据。

方法对于队列 1 和队列 2 中的患者，艾美珠单抗在第 1 天按 6 mg/kg 皮下注射，第 2 天按 3 mg/kg 皮下注射，从第 8 天起每周一次 1.5 mg/kg。

结果12 名患者（队列 1）和 2 名患者（队列 2）入选。队列 1 的艾美珠单抗治疗持续时间为 8 至 639 天（中位值：44.5 天），队列 2 为 64 和 450 天。在两个队列中，初次给予艾美珠单抗后均未观察到大出血。六名患者在艾美珠单抗治疗期间开始了第一次康复治疗，没有发生与康复相关的出血。 23 例手术是在艾美珠单抗预防下进行的，没有发生与手术相关的出血。尽管在初步分析中报告了一名患者出现无症状深静脉血栓形成，但此后没有发生其他血栓事件。两名患者出现了抗艾美珠单抗抗体，其中一名患者表现出艾美珠单抗清除加速。在三名接受艾美珠单抗预防的患者中成功执行了定制的 IST 方法（延迟启动、不使用或减少剂量）。

结论这些结果表明，无论是否符合 IST 资格，艾美珠单抗预防在 PwAHA 中都具有良好的获益-风险特征。