Shenzhen Hospital of Southern Medical University

Iptacopan Shows Promise in Treating Life-Threatening Transplant-Associated Thrombotic Microangiopathy

- A 17-year-old patient with high-risk transplant-associated thrombotic microangiopathy (TA-TMA) was successfully treated with iptacopan, an oral complement factor B inhibitor, after conventional therapy failed. - The patient showed significant clinical improvement after one month of iptacopan treatment, with key biomarkers including C5b-9 levels dropping from 739 ng/mL to 163 ng/mL and platelet count recovering to 146 × 10⁹/L. - This case demonstrates iptacopan's potential to overcome limitations of existing C5 inhibitors like eculizumab by targeting the proximal complement pathway and preventing persistent complement activation. - TA-TMA affects up to 90% of high-risk patients with mortality rates of 50-90%, making this therapeutic breakthrough particularly significant for improving outcomes in this devastating post-transplant complication.