Comparisons of global coagulation potential and bleeding episodes in emicizumab-treated hemophilia A patients and mild hemophilia A patients
Comparisons of global coagulation potential and bleeding episodes in emicizumab-treated hemophilia A patients and mild hemophilia A patients
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DOI：
10.1007/s12185-021-03276-7
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发表时间：
2022-01-19
影响因子：
2.1
通讯作者：
Nogami, Keiji
中科院分区：
文献类型：
--
作者：
Nakajima, Yuto;Mizumachi, Kuniyoshi;Nogami, Keiji
Emicizumab可减少重度血友病A（HA）患者的出血事件。临床剂量下emicizumab的凝血潜能似乎相当于约15 IU/dL的因子VIII活性（FVIII：C），相当于从重度表型转化为轻度表型。然而，接受emicizumab治疗的HA患者与轻度HA患者（PwMHA）相比的临床和实验室特征仍有待确定。我们回顾了63例PwHA和15例PwMHA患者的临床资料，并使用钙离子触发旋转血栓弹力图（ROTEM）和鞣花酸/组织因子触发凝块波形分析（改良CWA）评价了综合凝血功能。PwMHA的中位FVIII：C为13.0（IQR 8.5-17.0）IU/dL。两组的出血模式相似，分为三类：（1）自发性出血，创伤后，（2）1-2天内出血，（3）1-2周后迟发性出血。有和无突破性出血的EMP-PwHA的凝血潜力相当。此外，尽管PwMHA的治疗与因突破性出血住院治疗之间的时间似乎长于PwMHA，但PwMHA的凝血功能与PwMHA相当。Emi-PwHA和PwMHA的凝血潜力和出血模式似乎相似，表明emicizumab驱动的凝血潜力反映了轻度HA。
Emicizumab reduces bleeding events in patients with severe hemophilia A (HA). The coagulation potential of emicizumab at a clinical dose appears to correspond to about 15 IU/dL of factor VIII activity (FVIII:C), the equivalent of converting from a severe to mild phenotype. However, the clinical and laboratory characteristics of HA patients receiving emicizumab (Emi-PwHA) compared with patients with mild HA (PwMHA) remain to be determined. We reviewed clinical data from Emi-PwHA (n = 63) and PwMHA (n = 15) and evaluated comprehensive coagulation function using Ca2+-triggered rotational thromboelastometry (ROTEM) and ellagic acid/tissue factor-triggered clot waveform analysis (modified CWA). The median FVIII:C in PwMHA was 13.0 (IQR 8.5-17.0) IU/dL. Bleeding patterns in both groups were similar and classified into three categories: (1) spontaneous bleeding, post-traumatic, (2) bleeding within 1-2 days, and (3) delayed bleeding after 1-2 weeks. The coagulation potential in Emi-PwHA with and without breakthrough bleeds was comparable. Furthermore, coagulation function in Emi-PwHA was equivalent to PwMHA, although time between treatment and hospitalization for breakthrough bleeds in PwMHA appeared to be longer than those in Emi-PwHA. The coagulation potential and bleeding patterns appeared to be similar in Emi-PwHA and PwMHA, indicating that emicizumab-driven coagulation potential reflected mild HA.