Real-World Data on Bleeding Patterns of Hemophilia A Patients Treated with Emicizumab.
Real-World Data on Bleeding Patterns of Hemophilia A Patients Treated with Emicizumab.
复制标题
关于血友病A的出血模式的现实世界数据。
DOI：
10.3390/jcm10194303
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发表时间：
2021-09-22
影响因子：
3.9
通讯作者：
Kenet G
中科院分区：
文献类型：
--
作者：
Levy-Mendelovich S;Brutman-Barazani T;Budnik I;Avishai E;Barg AA;Levy T;Misgav M;Livnat T;Kenet G
Emicizumab（Hemlibra™）获批用于预防血友病A（HA）患者。HAVEN研究探讨了emicizumab治疗患者的出血减少，但缺乏emicizumab治疗期间出血模式的真实数据。我们的目的是比较从emicizumab治疗开始的不同时间点突破性出血的发生率。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）。我们分析了在研究的选定时间点（“出血期”）自发性和创伤性出血的数量。创伤性和自发性出血事件的百分比在“出血期”之间没有显著差异（分别为P = 0.053和P = 0.092）。大多数创伤相关的治疗出血由关节积血（53%）或头部创伤（33%）引起。自发性出血主要为关节积血（80%）。采用二项logistic回归分析患者年龄、emicizumab治疗前的年出血率和抑制剂的存在与自发性出血发生的潜在相关性。使用emicizumab时出血的几率每增加一岁增加1.029倍（P = 0.034）。结论：我们的真实世界数据显示，尽管使用emicizumab治疗，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。
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 (n = 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” (P = 0.053 and P = 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 (P = 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.
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影响因子：
158.5
作者：
Shima, Midori;Hanabusa, Hideji;Nogami, Keiji
通讯作者：
Nogami, Keiji
影响因子：
3.9
作者：
Ebbert, Patrick T.;Xavier, Frederico;Ragni, Margaret V.
通讯作者：
Ragni, Margaret V.
影响因子：
3.9
作者：
Misgav, Mudi;Brutman-Barazani, Tami;Kenet, Gili
通讯作者：
Kenet, Gili
影响因子：
2.4
作者：
O'Hara, Jamie;Walsh, Shaun;Wilkinson, Lars
通讯作者：
Wilkinson, Lars
DOI：
10.1111/hae.13998
发表时间：
2020-05
期刊：
Haemophilia : the official journal of the World Federation of Hemophilia
影响因子：
--
作者：
Soucie JM;Miller CH;Dupervil B;Le B;Buckner TW
通讯作者：
Buckner TW