• Pavan K Bendapudi, Brody H Foy, Sarah B Mueller, Jun Liu, Louis M Feingold, Kristen E Burke, Wendy Cruz, Maria Y Chen, Emily S Lau, Rachel L Goldberg, Ishan Tatake, Shelby C Wilkinson, Brian J Carney, James R Stone, Doyun Park, Alzira R Avelino, Sajjad Hassan, Chester Andrzejewski, Kristen N Ruby, Kenneth D Friedman, Patricia A R Brunker, Rebecca K Leaf, John Higgins, Walter H Dzik, Jonathan A Stefely, and Robert S Makar.
• 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): 169016981690-1698.

AbstractIn 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.Copyright © 2024 Massachusetts Medical Society.

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