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Recombinant ADAMTS13 for Immune Thrombotic Thrombocytopenic Purpura.

Authors :
Bendapudi PK
Foy BH
Mueller SB
Liu J
Feingold LM
Burke KE
Cruz W
Chen MY
Lau ES
Goldberg RL
Tatake I
Wilkinson SC
Carney BJ
Stone JR
Park D
Avelino AR
Hassan S
Andrzejewski C
Ruby KN
Friedman KD
Brunker PAR
Leaf RK
Higgins J
Dzik WH
Stefely JA
Makar RS
Source :
The New England journal of medicine [N Engl J Med] 2024 May 09; Vol. 390 (18), pp. 1690-1698.
Publication Year :
2024

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.<br /> (Copyright © 2024 Massachusetts Medical Society.)

Details

Language :
English
ISSN :
1533-4406
Volume :
390
Issue :
18
Database :
MEDLINE
Journal :
The New England journal of medicine
Publication Type :
Academic Journal
Accession number :
38718359
Full Text :
https://doi.org/10.1056/NEJMoa2402567