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[先天性和获得性血友病的诊断与治疗]

[Diagnosis and treatment of congenital and acquired hemophilia].

作者信息

Higasa Satoshi

机构信息

Division of Hematology, Department of Internal Medicine, Hyogo College of Medicine.

出版信息

Rinsho Ketsueki. 2017;58(7):857-865. doi: 10.11406/rinketsu.58.857.

Abstract

In recent years, the treatment of congenital hemophilia is a transition period for treatment using extended half-life clotting factor formulations. Although the half-life of these formulations is longer than that of the standard formulations, the strength and rate of the effect remain unchanged. Therefore, it is thought that we can adapt to any kind of case if we treat various kinds of plans within the insurance adaptation. Furthermore, the factor VIII-mimetic bispecific antibody has also been currently developed as a therapeutic drug for hemophilia A. The antibody has a long half-life of approximately 30 days, is not neutralized by inhibitors, and can be subcutaneously administered. Acquired hemophilia A (AHA) is a disorder characterized by bleeding caused by autoantibodies that are developed against factor VIII. In recent years, the diagnostic method and treatment for AHA have undergone no major change. The major symptoms of AHA-subcutaneous bleeding and muscle hemorrhage-differ from those of congenital hemophilia. In AHA, PT and VWF activities are normal, APTT is extended, factor VIII activity is decreased, and factor VIII inhibitor is positive. Bypassing agents are the mainstay of hemostasis treatment, and immunosuppressive therapy is essential for inhibitor eradication.

摘要

近年来,先天性血友病的治疗正处于使用延长半衰期凝血因子制剂进行治疗的过渡阶段。尽管这些制剂的半衰期比标准制剂长,但其效力和起效速度保持不变。因此,人们认为,如果在保险适应范围内制定各种治疗方案,我们就能适应任何类型的病例。此外,目前还开发了一种VIII因子模拟双特异性抗体作为治疗甲型血友病的药物。该抗体半衰期约为30天,较长,不会被抑制剂中和,且可皮下给药。获得性甲型血友病(AHA)是一种由针对VIII因子产生的自身抗体导致出血的疾病。近年来,AHA的诊断方法和治疗没有发生重大变化。AHA的主要症状——皮下出血和肌肉出血——与先天性血友病不同。在AHA中,PT和VWF活性正常,APTT延长,VIII因子活性降低,VIII因子抑制剂呈阳性。旁路制剂是止血治疗的主要手段,免疫抑制治疗对于根除抑制剂至关重要。

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