PDF(Pubmed)
Abstract：
In patients with immune thrombotic thrombocytopenic purpura (iTTP), autoantibodies against the metalloprotease ADAMTS13 lead to catastrophic microvascular thrombosis. However, the potential benefits of recombinant human ADAMTS13 (rADAMTS13) in patients with iTTP remain unknown. Here, we report the clinical use of rADAMTS13, which resulted in the rapid suppression of disease activity and complete recovery in a critically ill patient whose condition had proved to be refractory to all available treatments. We also show that rADAMTS13 causes immune complex formation, which saturates the autoantibody and may promote its clearance. Our data support the role of rADAMTS13 as a novel adjunctive therapy in patients with iTTP.
摘要：
在免疫性血栓性血小板减少性紫癜（iTTP）患者中，抗金属蛋白酶ADAMTS13的自身抗体导致灾难性的微血管血栓形成。然而,重组人ADAMTS13(rADAMTS13)对iTTP患者的潜在益处尚不清楚.这里,我们报道了rADAMTS13的临床应用,该应用可快速抑制危重患者的疾病活动并完全康复,而危重患者的病情已被证明对所有可用的治疗无效.我们还表明rADAMTS13导致免疫复合物形成，饱和自身抗体，并可能促进其清除。我们的数据支持rADAMTS13作为iTTP患者的新型辅助治疗的作用。
