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

ABSTRACT: 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 compassionate use treatment. Herein, we report the survival of children (< 16 years old) and adults (≥ 16 years old) enrolled in the EAP from October 2017 to October 2023 (n = 136). Among children (n = 50), the majority underwent allogeneic HCT (n = 44); 37 were considered high-risk (HR) and 30 (81.1%) had organ dysfunction at the time of TA-TMA diagnosis. One-year overall survival (O

SUBMITTER: Schoettler ML 

PROVIDER: S-EPMC12516664 | biostudies-literature | 2025 Nov

REPOSITORIES: biostudies-literature