Michelle L Schoettler, Seema Patel, Elyse Bryson, Laura Deeb, Benjamin Watkins, Muna Qayed, Shanmuganathan Chandrakasan, Taylor Fitch, Katherine Silvis, Jayre Jones, Satheesh Chonat, Kirsten M Williams
BACKGROUND: Transplant-associated thrombotic microangiopathy (TA-TMA) is a common and potentially severe complication of hematopoietic cell transplantation. TA-TMA directed therapy with eculizumab, a C5 inhibitor, has resulted in a survival benefit in some studies. However, children with TA-TMA refractory to complement C5 inhibition with eculizumab (rTA-TMA) have mortality rates exceeding 80%, and there are no other known therapies. Narsoplimab, an inhibitor of the effector enzyme of the lectin pathway, MASP-2, has been studied in adults with TA-TMA as first line therapy with a response rate of 61%...
December 23, 2023: Transplantation and cellular therapy