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, Robert S Makar
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...
May 9, 2024: New England Journal of Medicine