Intraindividual bleeding outcomes in patients with hemophilia A on emicizumab prophylaxis in Australia

Blood Vessels, Thrombosis & Hemostasis Pub Date : 2024-03-01 DOI:10.1016/j.bvth.2024.100005

Radha Ramanan , Sumit Parikh , Lwin Lwin Aung , James D. McFadyen , Huyen A. Tran

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Abstract

Emicizumab became routinely available in Australia in November 2020 as regular prophylaxis for certain patients with hemophilia A (HA). We performed an intraindividual comparison of bleeding outcomes in Australian patients with HA before and after commencement of emicizumab. Data regarding demographics, severity, treatment, inhibitors, and number and type of intraindividual treated bleeds before and after starting emicizumab in patients with HA were extracted from the Australian Bleeding Disorders Registry. As of April 2022, there were 459 eligible patients with HA on emicizumab in Australia, 397 of 459 (86%) of whom had severe disease. Overall, 59 of 459 (13%) had a current inhibitor. Adults (aged ≥18 years) composed 49% (223/459) of the population. The proportion of patients with zero bleeds increased from 54% to 63% after commencement of emicizumab (relative risk [RR], 1.24; 95% confidence interval [CI], 1.09-1.41; P < .01). RR for zero treated bleeds after commencement was significant in subgroups including pediatric patients (RR, 1.34; 95% CI, 1.13-1.59; P < .01) and those not on regular prophylaxis prior (RR, 1.75; 95% CI, 1.22-2.52; P < .01). There was no significant difference in zero-bleed prevalence in the adult, standard half-life, and extended half-life subgroups. Spontaneous bleeding was reduced (RR, 1.69; 95% CI, 1.34-2.13; P < .01), whereas provoked bleeding was not (P = .15). Real-world data from Australia shows a reduction in bleeding events with emicizumab prophylaxis for the overall population of patients with HA, although not in all subgroups. This reduction appears to be most pronounced in spontaneous bleeds within the pediatric population, and in those on on-demand therapy before switching.

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澳大利亚接受埃米珠单抗预防治疗的 A 型血友病患者的个体内出血结果

摘要埃米珠单抗于2020年11月在澳大利亚开始常规用于某些A型血友病（HA）患者的预防治疗。我们对开始使用埃米珠单抗前后澳大利亚 A 型血友病患者的出血结果进行了个体内比较。我们从澳大利亚出血性疾病登记处（Australian Bleeding Disorders Registry）提取了HA患者在开始使用埃米珠单抗前后的人口统计学、严重程度、治疗、抑制剂以及个体内治疗出血的数量和类型等方面的数据。截至2022年4月，澳大利亚有459名符合条件的HA患者在使用埃米珠单抗，459人中有397人（86%）病情严重。总体而言，459 名患者中有 59 人（13%）目前正在使用抑制剂。成人（年龄≥18 岁）占总人数的 49%（223/459）。开始使用埃米珠单抗后，出血为零的患者比例从 54% 增加到 63%（相对风险 [RR]，1.24；95% 置信区间 [CI]，1.09-1.41；P < .01）。在包括儿科患者（RR，1.34；95% CI，1.13-1.59；P <；.01）和之前未接受常规预防治疗的患者（RR，1.75；95% CI，1.22-2.52；P <；.01）在内的亚组中，开始治疗后出血为零的相对风险显著。成人、标准半衰期和延长半衰期亚组的零出血率没有明显差异。自发性出血有所减少（RR，1.69；95% CI，1.34-2.13；P < .01），而诱发出血则没有减少（P = .15）。来自澳大利亚的真实世界数据显示，尽管并非在所有亚组中，但在所有HA患者中，使用埃米珠单抗预防性治疗可减少出血事件。这种减少似乎在儿科自发性出血和换药前按需治疗的患者中最为明显。

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Blood Vessels, Thrombosis & Hemostasis

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