Compassionate Use Narsoplimab for Severe Refractory Transplantation-Associated Thrombotic Microangiopathy in Children.
Schoettler ML, Patel S, Bryson E, Deeb L, Watkins B, Qayed M, Chandrakasan S, Fitch T, Silvis K, Jones J, Chonat S, Williams KM.
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Transplant Cell Ther. 2024 Mar;30(3):336.e1-336.e8. doi: 10.1016/j.jtct.2023.12.017. Epub 2023 Dec 23.
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PMID: 38145741
TA-TMA-directed therapy with eculizumab, a complement C5 inhibitor, has resulted in a survival benefit in some studies. However, children with TA-TMA refractory to C5 inhibition with eculizumab (rTA-TMA) have mortality rates exceeding 80%, and there ar …
TA-TMA-directed therapy with eculizumab, a complement C5 inhibitor, has resulted in a survival benefit in some studies. Howeve …