Use of eculizumab in pediatric patients with high-risk transplant-associated thrombotic microangiopathy: outcomes and risk factors associated with response and survival. A retrospective study on behalf of the Spanish Group for Hematopoietic Transplantation and Cellular Therapy (GETH-TC)

Transplant-associated thrombotic microangiopathy (TA-TMA) is a major complication of hematopoietic stem cell transplantation (HSCT) associated with high morbidity and mortality1 –3. It is characterized by an endothelial damage consisting of microangiopathic hemolytic anemia and microvascular thrombosis, which ends up causing tissue ischemia and organ damage 4–7. In TA-TMA, the complement activation and endothelial damage are triggered by multiple insults after transplan tation: chemotherapy, total body irradiation (TBI), human leukocyte antigen (HLA) mismatch, calcineurin inhibitors (CNIs), graft versus host disease (GvHD) and viral infections3,8,9.

Source: Biology of Blood and Marrow Transplantation - March 21, 2024 Category: Hematology Authors: MI Ben ítez-Carabante, D Bueno, L Alonso García, I López Torija, J Marsal, Fernandez NavarroJM, ML Uria Oficialdegui, M Panesso, B Molina, C Beléndez Bieler, P Palomo, A Pérez Martínez, C Diaz-de-Heredia Source Type: research