Prospective Multi-Center Study of Pediatric Cellular Therapy Patients at Risk for Endothelial Dysfunction, Sinusoidal Obstruction Syndrome, and Multi-Organ Dysfunction Syndrome (MODS)

What this Trial Is About

Researchers are conducting an international, multi-center observational study to track children, adolescents, and young adults undergoing hematopoietic cell transplantation (HCT). The study focuses on understanding how endotheliopathies—such as sinusoidal obstruction syndrome (SOS), thrombotic microangiopathy (TMA), diffuse alveolar hemorrhage (DAH), and idiopathic pneumonia syndrome (IPS)—and multi-organ dysfunction syndrome (MODS) affect morbidity and mortality in this population. It also examines how these conditions correlate with abnormal levels of blood factors related to blood vessel function and inflammation within 100 days after transplant. Participants will have peripheral blood samples collected at eight specific time points to build a biorepository. The study includes patients receiving autologous and allogeneic HCT with any preparative regimen. Researchers will track and analyze these samples to assess the presence and impact of endotheliopathies and MODS, as well as how prior exposure to immunotherapies like CAR-T or PD-1 inhibitors may influence outcomes. Throughout the study, participants will be monitored for up to one year to measure the proportion developing endotheliopathies or MODS and to evaluate their overall survival. Data collection involves blood tests to measure angiogenic, vascular permeability, and inflammatory factors. The study aims to provide a better understanding of these complications to improve future care for pediatric cellular therapy patients.

A Prospective Registry of Pediatric Cellular Therapy Patients at Risk for Endothelial Dysfunction, Sinusoidal Obstruction Syndrome and/or Multi-Organ Dysfunction Syndrome (MODS).