Levy-Mendelovich Sarina, Brutman-Barazani Tami, Budnik Ivan, Avishai Einat, Barg Assaf A, Levy Tamara, Misgav Mudi, Livnat Tami, Kenet Gili
National Hemophilia Center, Sheba Medical Center, Tel Hashomer, Ramat Gan 52621, Israel.
Amalia Biron Research Institute of Thrombosis and Hemostasis, Sackler School of Medicine, Tel Aviv University, Tel Aviv 52621, Israel.
J Clin Med. 2021 Sep 22;10(19):4303. doi: 10.3390/jcm10194303.
Emicizumab (Hemlibra™) is approved for prophylaxis of hemophilia A (HA) patients. The HAVEN studies addressed bleeding reduction in emicizumab-treated patients, but real-world data on bleeding patterns during emicizumab therapy are lacking. We aimed to compare the occurrence of breakthrough bleeding at different time points, starting from emicizumab initiation. This longitudinal prospective observational cohort study included HA patients ( = 70, aged 1 month to 74.9 years) that completed at least 18 months of follow-up in our center. We analyzed the number of spontaneous and traumatic bleeds during selected time points of the study ("bleeding periods"). The percentage of traumatic and spontaneous bleeding episodes was not significantly different among "bleeding periods" ( 0.053 and = 0.092, respectively). Most trauma-related treated bleeds resulted from either hemarthrosis (53%) or head trauma (33%). Spontaneous bleeding episodes were mostly hemarthroses (80%). Potential associations of the patients' age, annualized bleeding rate before emicizumab treatment, and the presence of inhibitors with spontaneous bleed occurrence were analyzed with binomial logistic regression. The odds of bleeding while on emicizumab increased by a factor of 1.029 ( = 0.034) for every one year of age. Conclusions: Our real-world data revealed that the risk of bleeding persists, especially in older patients, despite therapy with emicizumab. These data may help clinicians in counselling their patients and in planning their management.
艾美赛珠单抗（Hemlibra™）已被批准用于预防A型血友病（HA）患者。HAVEN研究探讨了接受艾美赛珠单抗治疗的患者出血减少情况，但缺乏关于艾美赛珠单抗治疗期间出血模式的真实世界数据。我们旨在比较从开始使用艾美赛珠单抗起不同时间点的突破性出血发生率。这项纵向前瞻性观察性队列研究纳入了在我们中心完成至少18个月随访的HA患者（n = 70，年龄1个月至74.9岁）。我们分析了研究选定时间点（“出血期”）内自发和创伤性出血的数量。“出血期”之间创伤性和自发性出血事件的百分比无显著差异（分别为P = 0.053和P = 0.092）。大多数与创伤相关的治疗性出血源于关节积血（53%）或头部创伤（33%）。自发性出血事件大多为关节积血（80%）。采用二项逻辑回归分析患者年龄、艾美赛珠单抗治疗前的年化出血率以及抑制剂的存在与自发性出血发生之间的潜在关联。年龄每增加一岁，接受艾美赛珠单抗治疗时出血的几率增加1.029倍（P = 0.034）。结论：我们的真实世界数据显示，尽管使用艾美赛珠单抗进行治疗，但出血风险仍然存在，尤其是在老年患者中。这些数据可能有助于临床医生为患者提供咨询并规划治疗方案。
