Department of Medicine, Ohio State University, Columbus, OH, USA.
Department of Pathology, Ohio State University, Columbus, OH, USA.
J Thromb Haemost. 2015 Jun;13 Suppl 1:S223-9. doi: 10.1111/jth.12934.
Advances in our understanding of the pathophysiology of both congenital and acquired thrombotic thrombocytopenic purpura (TTP) have led to both an increased understanding of the disease and novel approaches to therapy. The efficacy of rituximab in acquired TTP has led to consideration of rituximab as a prophylactic therapy to prevent relapse of TTP. Novel therapies that target the A1 domain of von Willebrand factor (VWF) to block the formation of microthrombotic disease have also entered clinical study and have demonstrated promise as potential therapeutic options. Additionally, a recombinant ADAMTS13 protease has been developed which may be an important therapeutic option for both congenital and acquired TTP. The development of these new therapeutic options for patients diagnosed with TTP has increased the importance of conducting prospective, randomized studies with these agents to both confirm their efficacy and more importantly understand their most appropriate role in the treatment of patients with TTP.
我们对先天性和获得性血栓性血小板减少性紫癜（TTP）的病理生理学的认识不断提高，这不仅加深了我们对该疾病的理解，也为治疗带来了新的方法。利妥昔单抗治疗获得性 TTP 的疗效，促使人们考虑将其作为预防 TTP 复发的预防性治疗。针对血管性血友病因子（VWF）A1 结构域以阻断微血栓形成疾病的新型疗法也已进入临床研究，并显示出作为潜在治疗选择的潜力。此外，还开发了一种重组 ADAMTS13 蛋白酶，它可能是先天性和获得性 TTP 的重要治疗选择。这些新的治疗方法为诊断为 TTP 的患者提供了更多的选择，因此进行这些药物的前瞻性、随机研究变得尤为重要，这不仅有助于确定其疗效，更重要的是了解它们在 TTP 患者治疗中的最佳作用。
