Department of Medicine, Ohio State University, Columbus, OH 43210, USA.
Semin Hematol. 2011 Oct;48(4):242-50. doi: 10.1053/j.seminhematol.2011.08.004.
While our understanding of the pathophysiology of both congenital and acquired forms of thrombotic thrombocytopenic purpura (TTP) has increased, it is only over the past couple of years that this understanding has been translated into clinically significant advances in the diagnosis, management, and treatment of TTP. More specifically, our understanding of the central role of the ADAMTS13 protease in the pathophysiology of TTP has allowed ADAMTS13 testing to have a more prominent role in confirming or re-evaluating the clinical diagnosis of idiopathic TTP with the finding of severely deficient or measurable ADAMTS13, respectively. Additionally, measurement of ADAMTS13 activity at presentation and during remission may be useful to predict both the risk of exacerbation and relapse in patients with idiopathic TTP. There are also several novel approaches to the therapy of TTP including a recombinant ADAMTS13 protease and agents that target the A1 domain of von Willebrand factor (VWF), blocking its interaction with platelet glycoprotein (GP)Ib to prevent the formation of microthrombotic disease in patients with TTP. The addition of these exciting new therapies to conventional plasma-based and/or immune modulating therapies provide hope for improved treatment outcomes for TTP patients.
虽然我们对先天性和获得性血栓性血小板减少性紫癜（TTP）的病理生理学的理解已经增加，但只是在过去几年中，这种理解才转化为 TTP 的诊断、管理和治疗方面具有临床意义的进展。更具体地说，我们对 ADAMTS13 蛋白酶在 TTP 病理生理学中的核心作用的理解，使得 ADAMTS13 测试在确认或重新评估特发性 TTP 的临床诊断方面具有更重要的作用，其发现分别为严重缺乏或可测量的 ADAMTS13。此外，在发作和缓解期间测量 ADAMTS13 活性可能有助于预测特发性 TTP 患者的恶化和复发风险。TTP 的治疗也有几种新方法，包括重组 ADAMTS13 蛋白酶和针对血管性血友病因子（VWF）A1 结构域的药物，阻断其与血小板糖蛋白（GP）Ib 的相互作用，以防止 TTP 患者形成微血栓性疾病。将这些令人兴奋的新疗法与传统的基于血浆的和/或免疫调节疗法相结合，为 TTP 患者提供了改善治疗结果的希望。
