Ramanan Radha, Parikh Sumit, Aung Lwin Lwin, McFadyen James D, Tran Huyen A
Australian Centre for Blood Diseases, Monash University, Melbourne, VIC, Australia.
Department of Haematology, Alfred Hospital, Melbourne, VIC, Australia.
Blood Vessel Thromb Hemost. 2024 Feb 24;1(1):100005. doi: 10.1016/j.bvth.2024.100005. eCollection 2024 Mar.
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; < .01). RR for zero treated bleeds after commencement was significant in subgroups including pediatric patients (RR, 1.34; 95% CI, 1.13-1.59; < .01) and those not on regular prophylaxis prior (RR, 1.75; 95% CI, 1.22-2.52; < .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; < .01), whereas provoked bleeding was not ( = .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.
艾美赛珠单抗于2020年11月在澳大利亚常规供应，用于某些甲型血友病（HA）患者的定期预防。我们对澳大利亚HA患者在开始使用艾美赛珠单抗之前和之后的出血结局进行了个体内比较。从澳大利亚出血性疾病登记处提取了有关HA患者在开始使用艾美赛珠单抗之前和之后的人口统计学、严重程度、治疗、抑制剂以及个体内治疗出血的数量和类型的数据。截至2022年4月，澳大利亚有459名符合条件的HA患者正在使用艾美赛珠单抗，其中459名患者中有397名（86%）患有严重疾病。总体而言，459名患者中有59名（13%）目前有抑制剂。成年人（年龄≥18岁）占总人数的49%（223/459）。开始使用艾美赛珠单抗后，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）。开始治疗后零治疗出血的RR在包括儿科患者（RR，1.34；95% CI，1.13 - 1.59；P <.01）和之前未进行定期预防的患者（RR，1.75；95% CI，1.22 - 2.52；P <.01）的亚组中具有统计学意义。在成人、标准半衰期和延长半衰期亚组中，零出血患病率没有显著差异。自发性出血减少（RR，1.69；95% CI，1.34 - 2.13；P <.01），而诱发性出血则没有（P =.15）。来自澳大利亚的真实世界数据显示，对于HA患者总体人群，使用艾美赛珠单抗预防可减少出血事件，尽管并非在所有亚组中均如此。这种减少在儿科人群中的自发性出血以及转换前接受按需治疗的患者中似乎最为明显。
