Mechanisms of endothelial injury and transplant-associated thrombotic microangiopathy in tandem autologous hematopoietic stem cell transplant for neuroblastoma

Our data indicate that endothelial injury is increased in auto-stem cell transplant patients receiving the specific conditioning regimen carboplatin, etoposide and melphalan and is associated with clinical TA-TMA. Moreover, our data identify specific pathways that can be targeted to treat or prevent TA-TMA. RNAseq was performed on glomerular endothelial cells cultured with serum from neuroblastoma patients who developed TA-TMA (n=5), timepoint-matched serum from neuroblastoma patients who did not develop TA-TMA (n=4) and complete media alone (no human serum, n=1).