Hematology Service, University Hospital La Fe , Valencia, Spain.
Hematology Service, University Hospital Doctor Peset , Valencia, Spain.
Expert Rev Hematol. 2020 Nov;13(11):1153-1164. doi: 10.1080/17474086.2020.1819230. Epub 2020 Oct 12.
Acquired thrombotic thrombocytopenic purpura (aTTP) is a thrombotic microangiopathy caused by inhibitory autoantibodies against ADAMTS13 protein. Until recently, the combination of plasma exchange (PEX) and immunosuppression has been the standard front-line treatment in this disorder. However, aTTP-related mortality, refractoriness, and relapse are still a matter of concern. The better understanding of the pathophysiological mechanisms of aTTP has allowed substantial improvements in the diagnosis and treatment of this disease. Recently, the novel anti-VWF nanobody caplacizumab has been approved for acute episodes of aTTP. Caplacizumab is capable to block the adhesion of platelets to VWF, therefore inhibiting microthrombi formation in the ADAMTS13-deficient circulation. In this review, the characteristics of caplacizumab together with the available data of its efficacy and safety in the clinical setting will be analyzed. Besides, the current scenario of aTTP treatment will be provided, including the role of other innovative drugs. With no doubt, caplacizumab is going to change the way we treat aTTP. In combination with standard treatment, caplacizumab can help to significantly reduce aTTP-related mortality and morbidity and could spare potential long-term consequences by minimizing the risk of exacerbation.
获得性血栓性血小板减少性紫癜（aTTP）是一种由抗 ADAMTS13 蛋白抑制性自身抗体引起的血栓性微血管病。直到最近，血浆置换（PEX）和免疫抑制的联合治疗一直是该疾病的标准一线治疗方法。然而，aTTP 相关的死亡率、难治性和复发仍然是一个令人关注的问题。对 aTTP 病理生理机制的更好理解，使得对这种疾病的诊断和治疗有了显著的改善。最近，新型抗 VWF 纳米抗体 caplacizumab 已被批准用于急性 aTTP 发作。Caplacizumab 能够阻断血小板与 VWF 的黏附，从而抑制 ADAMTS13 缺乏循环中的微血栓形成。在这篇综述中，将分析 caplacizumab 的特性，以及其在临床环境中的疗效和安全性的现有数据。此外，还将提供 aTTP 治疗的现状，包括其他创新药物的作用。毫无疑问，caplacizumab 将改变我们治疗 aTTP 的方式。与标准治疗联合使用，caplacizumab 可以显著降低 aTTP 相关的死亡率和发病率，并通过最大限度地降低恶化风险，避免潜在的长期后果。
