From the Blood Transfusion Service (P.K.B., K.N.R., P.A.R.B., W.H.D., J.A.S., R.S.M.), the Division of Hematology (P.K.B., R.K.L., W.H.D.), the Department of Pathology (B.H.F., J.L., M.Y.C., J.R.S., J.H.), and the Division of Cardiology (E.S.L., R.L.G.), Massachusetts General Hospital, the Division of Hemostasis and Thrombosis (P.K.B., L.M.F., K.E.B., I.T., S.C.W.) and the Division of Hematology and Apheresis Service (B.J.C.), Beth Israel Deaconess Medical Center, and Harvard Medical School (P.K.B., J.L., M.Y.C., E.S.L., R.L.G., I.T., S.C.W., B.J.C., J.R.S., K.N.R., P.A.R.B., R.K.L., J.H., W.H.D., J.A.S., R.S.M.), Boston, the Broad Institute of MIT and Harvard, Cambridge (P.K.B.), the Division of Hematology and Oncology, Lahey Hospital and Medical Center, Burlington (D.P.), and the Department of Hematology and Clinical Oncology (A.R.A.) and the Department of Pathology, Transfusion/Apheresis Medicine Services (S.H., C.A.), UMass Chan Medical School-Baystate Health, Springfield - all in Massachusetts; the Department of Laboratory Medicine and Pathology, University of Washington, Seattle (B.H.F.); and Versiti Blood Center of Wisconsin, Milwaukee (S.B.M., W.C., K.D.F.).
N Engl J Med. 2024 May 9;390(18):1690-1698. doi: 10.1056/NEJMoa2402567.
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.
在免疫性血栓性血小板减少性紫癜（iTTP）患者中，针对金属蛋白酶 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）在 iTTP 患者中的潜在益处尚不清楚。在这里，我们报告了 rADAMTS13 的临床应用，它导致了一位重症患者的疾病活动迅速抑制，并且完全恢复，而该患者的病情已被证明对所有现有治疗均无反应。我们还表明，rADAMTS13 导致免疫复合物形成，从而使自身抗体饱和并可能促进其清除。我们的数据支持 rADAMTS13 作为 iTTP 患者新型辅助治疗的作用。
