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获得性血友病患者的免疫耐受治疗。

Immune tolerance therapy in patients with acquired hemophilia.

作者信息

von Depka Mario

机构信息

Department of Hematology, Haemostasis and Oncology, Hannover Medical School, Germany.

出版信息

Hematology. 2004 Aug;9(4):245-57. doi: 10.1080/10245330410001722087.

Abstract

Acquired hemophilia is a rare disorder with an estimated annual incidence of 0.2-1 cases per million individuals. The etiology of the disorder remains obscure, although approximately half of all cases are associated with other underlying conditions. In acquired hemophilia, the severe hemorrhagic diathesis is caused by the development of autoantibodies directed against a clotting factor, most commonly factor VIII. These autoantibodies inhibit normal coagulation and lead to bleeding complications, which can be life-threatening in a high percentage of cases. Prompt diagnosis and appropriate management of the disorder enable effective control; the short- and long-term aims of therapy are to terminate the acute bleed and eliminate or reduce the inhibitor, respectively. Immune tolerance therapy has been shown to successfully eradicate or suppress inhibitors in patients with congenital hemophilia A and may be applicable to patients with acquired hemophilia. Here we present preliminary data on the use of immune tolerance therapy in patients with acquired hemophilia and discuss possible treatment strategies.

摘要

获得性血友病是一种罕见疾病,估计年发病率为每百万人口0.2 - 1例。尽管所有病例中约一半与其他潜在疾病有关,但该疾病的病因仍不明确。在获得性血友病中,严重的出血素质是由针对凝血因子(最常见的是凝血因子VIII)的自身抗体的产生所引起的。这些自身抗体抑制正常凝血并导致出血并发症,在很大比例病例中可能危及生命。对该疾病的及时诊断和适当管理能够实现有效控制;治疗的短期和长期目标分别是终止急性出血和消除或减少抑制剂。免疫耐受疗法已被证明能成功根除或抑制先天性A型血友病患者体内的抑制剂,可能也适用于获得性血友病患者。在此,我们展示了关于在获得性血友病患者中使用免疫耐受疗法的初步数据,并讨论了可能的治疗策略。

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