Risk factors, prevention, and outcomes of VOD in pediatric patients undergoing SCT for acute leukemia
Mentor Name: Caitlin Elgarten
Stem cell transplantation is a curative treatment for some children with acute leukemia. Antibody-drug conjugates are a new class of medications that can delivery therapy in a targeted way directly to leukemia cells. As a result, these medications may improve outcomes for children with leukemia. But these medications also cause new side effects. The goal of this project is to understand how antibody-drug conjungate medications lead to specific complications after transplant, including a devastating liver disease called veno-occlusive disease. In this clinical research project, we will leverage a multi-institution consortium and retrospectively review the charts of children with acute leukemia who have undergone transplantation in order to define the current epidemiology of veno-occlusive disease in children transplanted for acute leukemia and the risk associated with antibody drug conjugates, as well as to assess the effectiveness of prophylaxis for veno-occlusive disease. The results of this study will help inform strategies to prevent veno-occlusive disease and ensure that more children can be cured of their leukemia without this dangerous side effect.
