Inhibitor eradication and bleeding management of acquired hemophilia A: a single center experience in China
Inhibitor eradication and bleeding management of acquired hemophilia A: a single center experience in China
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获得性血友病 A 的抑制剂根除和出血管理：中国的单中心经验
DOI：
10.1080/16078454.2019.1663028
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发表时间：
2019-01
期刊：
影响因子：
1.9
通讯作者：
Wu Wenman
中科院分区：
文献类型：
--
作者：
Ye Naifang;Liu Zhenzhen;Xu Guanqun;Wang Xuefeng;Wu Fang;Xu Xiaoqian;Wu Wenman
摘要目的：获得性血友病A(AHA)是一种罕见的疾病，由抗凝血因子VIII的自身抗体引起的自发性出血。本研究报告了一组相对较大的AHA患者的临床特征和治疗结果。方法：对2014年1月至2018年12月收治的42例AHA患者的临床资料进行回顾性分析。结果：FVIII抑制剂(中位数8BU/mL；IQR：4.0~16.0)显著抑制FVIII活性(中位数1.5%；四分位数范围[IQR]：0.9~3.5)。14例患者使用PCC或FVIIa旁路止血，无任何不良反应，大多数患者(90.5%，38/42)接受免疫抑制方案，单用糖皮质激素或与环磷酰胺联合应用。单独使用皮质类固醇的患者的中位抑制物滴度(8BU/mL)低于联合应用环磷酰胺的患者(16BU/mL)(p <； 0.001)。97.4%(37/38)的患者在接受免疫抑制治疗后获得完全缓解(CR)，单用糖皮质激素治疗的患者达到CR的中位时间比联合应用环磷酰胺的患者短(中位数40天；智商：31~65天比51天；智商：38~83，p = 0.301)。10例(26.3%)复发后接受皮质类固醇和环磷酰胺联合治疗，其中8例(80%)二次缓解。两名患者死亡，一名死于手术后腹膜后未控制的出血，一名死于皮质类固醇治疗中的脓毒症。结论：糖皮质激素治疗效果满意，尤其是抑制剂效价低，联合应用环磷酰胺可促进高效价的重症患者的缓解。
ABSTRACT Objectives: Acquired hemophilia A (AHA) is a rare disease resulting from autoantibodies against coagulation factor VIII that leads to spontaneous bleeding. This study reports the clinical characteristics and treatment outcomes of a relatively sizable cohort of patients with AHA. Methods: We retrospectively analyzed the characteristics and outcomes of 42 patients with AHA diagnosed in our center from January 2014 through December 2018. Results: The FVIII activity (FVIII: C) was significantly suppressed (median 1.5%; interquartile range [IQR]: 0.9–3.5) by FVIII inhibitor (median 8 BU/mL; IQR: 4.0–16.0). Bypassing agents, PCC or FVIIa, were used in 14 patients for bleeding control without any adverse reaction; and most patients (90.5%, 38/42) were placed on immunosuppressive regimen, corticosteroid alone or in combination with cyclophosphamide. Patients treated with corticosteroids alone had a lower median inhibitor titer (8 BU/mL) than those treated with combination corticosteroids of cyclophosphamide (16 BU/mL) (p < 0.001). 97.4% (37/38) patients achieved complete remission (CR) after immunosuppression therapy, and the median time to CR in patients treated with corticosteroids alone was shorter than those with combination corticosteroids of cyclophosphamide (median 40 days; IQR: 31–65 vs. 51 days; IQR: 38–83, p = 0.301). 10 (26.3%) patients relapsed thereafter and were placed on combined corticosteroid and cyclophosphamide treatment, which yielded second remission in 8 patients (80%). Two patients died, one from uncontrolled post-surgical retroperitoneal hemorrhage and one from sepsis complicating corticosteroid therapy. Conclusion: The corticosteroid achieves a satisfactory outcome, particularly with low inhibitors titers; and combination of cyclophosphamide will facilitate remission in sever patients with high titers of inhibitors.
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使用重组激活因子VII用于获得性血友病中的急性出血事件：止血和血栓形成研究学会注册表的最终分析获得了血友病研究。
DOI：
10.1097/mbc.0000000000000471
发表时间：
2016-10
期刊：
Blood coagulation & fibrinolysis : an international journal in haemostasis and thrombosis
影响因子：
--
作者：
Ma AD;Kessler CM;Al-Mondhiry HA;Gut RZ;Cooper DL
通讯作者：
Cooper DL
影响因子：
5.7
作者：
Webert, Kathryn E.
通讯作者：
Webert, Kathryn E.
影响因子：
20.3
作者：
Tiede, Andreas;Klamroth, Robert;Knoebl, Paul
通讯作者：
Knoebl, Paul
影响因子：
1.9
作者：
Franchini, Massimo;Vaglio, Stefania;Liumbruno, Giancarlo Maria
通讯作者：
Liumbruno, Giancarlo Maria
DOI：
10.1177/1076029613488937
发表时间：
2015-01
期刊：
Clinical and Applied Thrombosis/Hemostasis
影响因子：
--
作者：
Yan-hui Yang;F. Xue;H. Shi;Hongmei Wang;Lei Zhang;L. Ji;R. Yang
通讯作者：
Yan-hui Yang;F. Xue;H. Shi;Hongmei Wang;Lei Zhang;L. Ji;R. Yang