Emicizumab prophylaxis in haemophilia patients older than 50 years with cardiovascular risk factors: Real-world data
Emicizumab prophylaxis in haemophilia patients older than 50 years with cardiovascular risk factors: Real-world data
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DOI：
10.1111/hae.14261
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发表时间：
2021-02-17
期刊：
影响因子：
3.9
通讯作者：
Kenet, Gili
中科院分区：
文献类型：
--
作者：
Misgav, Mudi;Brutman-Barazani, Tami;Kenet, Gili
Emicizumab （Hemlibra (TM)）被批准用于有或无抑制剂的血友病A （HA）患者的预防。然而，emicizumab用于合并心血管危险因素的老年HA患者的实际数据缺乏。目的评价emicizumab在现实世界老年HA患者队列中的安全性和有效性。方法对50岁以上的HA患者进行前瞻性纵向观察研究，随访和监测在emicizumab预防期间。我们记录了任何出血或不良事件，并收集血浆样本检测emicizumab水平、aPTT和凝血酶生成（TG）。结果17例HA患者（2例合并抑制剂），中位年龄62.4岁（范围51.5 ~ 77.1岁），其中9/17例合并多种心血管危险因素（高危组）。7名患者患有慢性艾滋病毒感染。本队列的中位随访时间为400天（89-805天，IQR为211-479天）。所有患者的年平均出血率（ABR）均显著降低。在表现出明显出血倾向的患者中，emicizumab稳态水平和TG较对照组低。同时使用抗血小板药物治疗的4例患者的ABR明显高于其他患者。未见血栓形成或血栓性微血管病变（TMA）。结论：对于50岁以上HA患者，包括那些有心血管危险因素的患者，Emicizumab的预防耐受性良好。由于在出血患者中观察到较低的emicizumab和TG水平，我们建议监测实验室分析可能对该年龄组有价值。
Introduction Emicizumab (Hemlibra (TM)) is approved for prophylaxis of Haemophilia A (HA) patients with and without inhibitors. However, real-world data on emicizumab use in the elderly HA patients with concomitant cardiovascular risk factors are lacking.Aim To evaluate the safety and efficacy of emicizumab in a real-world cohort of elderly HA patients.Methods A prospective longitudinal observational study on HA patients over 50 years old treated, followed and monitored during emicizumab prophylaxis was conducted. We documented any bleeding or adverse events and collected plasma samples for emicizumab levels, aPTT and thrombin generation (TG).Results Seventeen HA patients (2 with inhibitor), whose median age was 62.4 years (range: 51.5-77.1) composed the cohort, including 9/17 with multiple cardiovascular risk factors (high risk group). Seven patients had chronic HIV infection. The median follow-up of our cohort was 400 days (range 89-805, IQR 211-479 days). The median annualized bleeding rate (ABR) significantly decreased for all patients. Among patients who displayed significant bleeding tendencies, emicizumab steady state levels as well as TG were lower as compared with the group. The ABR of four patients concomitantly treated by antiplatelet agents was significantly higher compared with the rest of the cohort. Neither thrombosis nor thrombotic microangiopathy (TMA) was encountered.Conclusions Emicizumab prophylaxis for HA patients older than 50 years including those with cardiovascular risk factors was well tolerated. As lower emicizumab and TG levels were observed among bleeding patients, we suggest that monitoring laboratory assays could be of value within this age group.