Abstract：
BACKGROUND: Low-dose emicizumab can potentially offer a cost-effective treatment option in persons with hemophilia A, especially in developing countries.
OBJECTIVE: To compare the efficacy and safety of low-dose emicizumab with those on low-dose factor (F)VIII prophylaxis via chart review.
METHODS: After ethics approval, chart data of 2 groups of patients were reviewed: group 1 (low-dose emicizumab, n = 10; 3 mg/kg monthly without a loading dose) and group 2 (low-dose FVIII prophylaxis, n = 10; 10-20 IU/kg of FVIII concentrates twice a week). Outcomes were target joints, annual bleeding rate, annual joint bleeding rate, Hemophilia Joint Health Score, nonactivated thromboelastometry-rotational thromboelastometry clotting time, plasma emicizumab levels, and direct costs of treatment.
RESULTS: All outcome measures were significantly better in the low-dose emicizumab group than in the low-dose FVIII prophylaxis group. For nonactivated thromboelastometry-rotational thromboelastometry, median values after 6 months in the low-dose emicizumab group were comparable with values seen in patients with mild hemophilia, while the values in the low-dose FVIII prophylaxis group were similar to those of patients with moderate hemophilia. The direct cost of low-dose emicizumab was found to be approximately US $6000 and that for low-dose recombinant FVIII prophylaxis used in our study was US $6282 (the cost may range from US $3432 to $7920 depending on the type of factor) when compared to approximately US $15 000 for standard-dose emicizumab.
CONCLUSIONS: Low-dose emicizumab offers a cost-effective treatment option and can improve access in developing countries. These findings need to be confirmed in a larger and better-controlled study.
OBJECTIVE: To compare the efficacy and safety of low-dose emicizumab with those on low-dose factor (F)VIII prophylaxis via chart review.
METHODS: After ethics approval, chart data of 2 groups of patients were reviewed: group 1 (low-dose emicizumab, n = 10; 3 mg/kg monthly without a loading dose) and group 2 (low-dose FVIII prophylaxis, n = 10; 10-20 IU/kg of FVIII concentrates twice a week). Outcomes were target joints, annual bleeding rate, annual joint bleeding rate, Hemophilia Joint Health Score, nonactivated thromboelastometry-rotational thromboelastometry clotting time, plasma emicizumab levels, and direct costs of treatment.
RESULTS: All outcome measures were significantly better in the low-dose emicizumab group than in the low-dose FVIII prophylaxis group. For nonactivated thromboelastometry-rotational thromboelastometry, median values after 6 months in the low-dose emicizumab group were comparable with values seen in patients with mild hemophilia, while the values in the low-dose FVIII prophylaxis group were similar to those of patients with moderate hemophilia. The direct cost of low-dose emicizumab was found to be approximately US $6000 and that for low-dose recombinant FVIII prophylaxis used in our study was US $6282 (the cost may range from US $3432 to $7920 depending on the type of factor) when compared to approximately US $15 000 for standard-dose emicizumab.
CONCLUSIONS: Low-dose emicizumab offers a cost-effective treatment option and can improve access in developing countries. These findings need to be confirmed in a larger and better-controlled study.
摘要：
背景：-低剂量Emicizumab可能为PwHA提供具有成本效益的治疗选择，尤其是在发展中国家。在本图表综述中，我们评估了与低剂量因子VIII预防相比6个月时的疗效和安全性。
方法：伦理批准后，回顾两组患者的图表资料。第1组[低剂量艾咪珠单抗，n=10][每月3mg/kg，无负荷剂量]和第2组[低剂量因子VIII预防，n=10][10-20IU/kg因子VIII每周浓缩两次]。结果是目标关节，年出血率（ABR），年关节出血率（AJBR），,血友病联合健康评分（HJHS），NATEMROTEM凝血时间,血浆emicizumab水平和治疗的直接成本。
结果：低剂量Emicizumab组的所有结果指标均明显优于低剂量因子VIII预防。对于NATEM-ROTEM，低剂量Emicizumab组6个月后的中位值与轻度血友病患者的中位值相当,而低剂量FVIII预防组的中位值与中度血友病患者的中位值相似.发现低剂量Emicizumab的直接成本约为6000美元，而在我们的研究中使用的低剂量重组因子VIII预防的直接成本为6282美元（成本可能在3432至7920美元之间，取决于因子的类型）与标准剂量Emicizumab的直接成本约为15000美元相比。
结论：低剂量Emicizumab提供了一种具有成本效益的治疗选择，可以改善发展中国家的可获得性。这些发现需要在更大和更好的对照研究中得到证实。
方法：伦理批准后，回顾两组患者的图表资料。第1组[低剂量艾咪珠单抗，n=10][每月3mg/kg，无负荷剂量]和第2组[低剂量因子VIII预防，n=10][10-20IU/kg因子VIII每周浓缩两次]。结果是目标关节，年出血率（ABR），年关节出血率（AJBR），,血友病联合健康评分（HJHS），NATEMROTEM凝血时间,血浆emicizumab水平和治疗的直接成本。
结果：低剂量Emicizumab组的所有结果指标均明显优于低剂量因子VIII预防。对于NATEM-ROTEM，低剂量Emicizumab组6个月后的中位值与轻度血友病患者的中位值相当,而低剂量FVIII预防组的中位值与中度血友病患者的中位值相似.发现低剂量Emicizumab的直接成本约为6000美元，而在我们的研究中使用的低剂量重组因子VIII预防的直接成本为6282美元（成本可能在3432至7920美元之间，取决于因子的类型）与标准剂量Emicizumab的直接成本约为15000美元相比。
结论：低剂量Emicizumab提供了一种具有成本效益的治疗选择，可以改善发展中国家的可获得性。这些发现需要在更大和更好的对照研究中得到证实。
