Recombinant ADAMTS13 for Immune Thrombotic Thrombocytopenic Purpura.
case_report · Level V
Where this comes from
- Record sourced from PubMed, PMID 38718359.
- Also identified by DOI 10.1056/NEJMoa2402567 and PMC identifier 11128310.
- Licence recorded as CC BY-NC-ND.
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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.
Medical subject headings
- ADAMTS13 Protein
- Purpura, Thrombotic Thrombocytopenic