Shen Ming-Ching, Chou Sheng-Chieh, Chiou Shyh-Shin, Lin Pei-Chin, Chen Yeu-Chin, Lin Hsuan-Yu, Lee Yang-Cheng, Huang Cih-En, Weng Te-Fu, Huang Ting-Huan, Chung Chih-Yuan, Chen Jiann-Shiuh, Chen Shu-Huey, Cheng Shin-Nan, Hsiao Chih-Cheng, Huang Yen-Min, Chen Shih-Hsiang, Yu Yuan-Bin, Lin Shih-Chiang, Lin Ching-Yeh, Peng Ching-Tien, Wang Jiaan-Der
Department of Internal Medicine, Changhua Christian Hospital, Changhua, Taiwan.
Division of Hematology, Department of Internal Medicine, National Taiwan University Hospital, Taipei, Taiwan.
Haemophilia. 2023 Nov;29(6):1499-1508. doi: 10.1111/hae.14880. Epub 2023 Oct 11.
Emicizumab mimicking the cofactor function of activated factor VIII (FVIII) restores haemostasis.
This nationwide observational study aimed to retrospectively investigate efficacy, safety, and cost in 1 year before and up to 3 years after emicizumab prophylaxis for haemophilia A (HA) patients with FVIII inhibitors.
A total of 39 severe HA patients with a median age of 23.0 years were enrolled. The median historical peak FVIII inhibitor titre was 174.2 BU/mL with an interquartile range of 56.5-578.8 BU/mL. The median annualized bleeding rate reduced from 24 to 0 events in the first year after emicizumab prophylaxis (p < .01) and sustained in the second and third years. The median annualized joint bleeding rate reduced to 0 and maintained up to 3 years (p < .01). Twenty-seven patients (69.2%) had target joints before emicizumab prophylaxis and only seven patients (17.9%) of them had target joints after prophylaxis. Medical costs, including cost of haemostatic therapy, frequency of outpatient department visits, emergency room visits and hospital admission, were significantly reduced after emicizumab prophylaxis (p < .01). FVIII inhibitor titre decreased after emicizumab prophylaxis. Overall, three (7.7%) patients experienced 202 grade 1 drug-related adverse events after emicizumab prophylaxis. No serious adverse events were reported during emicizumab prophylaxis period. The adherence to emicizumab prophylaxis was 100% up to 3 years.
HA patients with FVIII inhibitors treated with emicizumab prophylaxis resulted in a significant reduction in treated bleeds and associated costs. No new safety events were observed.
艾美赛珠单抗模拟活化因子VIII（FVIII）的辅因子功能，可恢复止血功能。
这项全国性观察性研究旨在回顾性调查艾美赛珠单抗预防治疗血友病A（HA）伴FVIII抑制剂患者前1年及之后长达3年的疗效、安全性和成本。
共纳入39例重度HA患者，中位年龄23.0岁。既往FVIII抑制剂峰值滴度中位数为174.2 BU/mL，四分位间距为56.5 - 578.8 BU/mL。艾美赛珠单抗预防治疗后的第一年，年化出血率中位数从24次降至0次（p < 0.01），并在第二年和第三年持续保持。年化关节出血率中位数降至0，并维持了3年（p < 0.01）。27例患者（69.2%）在艾美赛珠单抗预防治疗前有靶关节，其中只有7例患者（17.9%）在预防治疗后有靶关节。艾美赛珠单抗预防治疗后，包括止血治疗费用、门诊就诊频率、急诊就诊和住院费用在内的医疗成本显著降低（p < 0.01）。艾美赛珠单抗预防治疗后FVIII抑制剂滴度降低。总体而言，3例（7.7%）患者在艾美赛珠单抗预防治疗后发生202次1级药物相关不良事件。在艾美赛珠单抗预防治疗期间未报告严重不良事件。至3年时，艾美赛珠单抗预防治疗的依从性为100%。
接受艾美赛珠单抗预防治疗的HA伴FVIII抑制剂患者的治疗性出血和相关成本显著降低。未观察到新的安全事件。
