Narsoplimab Results in Excellent Survival in Adults and Children With Hematopoietic Cell Transplant Associated Thrombotic Microangiopathy ( TA ‐ TMA )

Abstract excerpt

Inappropriate complement activation is a key driver of hematopoietic cell transplant-associated thrombotic microangiopathy (TA-TMA). Treatment with narsoplimab, an inhibitor of MASP-2, the effector enzyme of the lectin pathway, resulted in a response rate of 61% in a Phase 2 clinical trial in adults with TA-TMA. Given these promising results, a global expanded access program (EAP) was established facilitating...

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