%0 Journal Article
%T Recombinant ADAMTS13 for Immune Thrombotic Thrombocytopenic Purpura.
%A Bendapudi PK
%A Foy BH
%A Mueller SB
%A Liu J
%A Feingold LM
%A Burke KE
%A Cruz W
%A Chen MY
%A Lau ES
%A Goldberg RL
%A Tatake I
%A Wilkinson SC
%A Carney BJ
%A Stone JR
%A Park D
%A Avelino AR
%A Hassan S
%A Andrzejewski C
%A Ruby KN
%A Friedman KD
%A Brunker PAR
%A Leaf RK
%A Higgins J
%A Dzik WH
%A Stefely JA
%A Makar RS
%J N Engl J Med
%V 390
%N 18
%D 2024 May 9
%M 38718359
%F 176.079
%R 10.1056/NEJMoa2402567
%X 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.