The goal of this is to learn more about stem cell transplant and complications that some people have after their transplants, in particular sinusoidal obstruction syndrome (SOS), also called veno-occlusive disease of the liver.
This is a multicenter, prospective, observational trial. We will measure biomarkers and determine thresholds that will predict increased risk for SOS in pediatric patients receiving HCT or high intensity chemotherapy/irradiation with the future goal of a randomized, interventional, open-label, multicenter trial that will test the preemptive use of defibrotide for prevention of SOS in an enriched high-risk population.
Study Type
OBSERVATIONAL
Enrollment
80
Day 0 and Day 3 blood draw for SOS biomarkers. If subject develops SOS blood draw prior to dose of Defibrotide, Day 14 after Defibrotide and Day 21 after Defibrotide.
Children's National Medical Center
Washington D.C., District of Columbia, United States
Indiana University
Indianapolis, Indiana, United States
University of Michigan
Ann Arbor, Michigan, United States
Baylor College of Medicine
Houston, Texas, United States
SOS proteomic markers
Measure for 3 SOS proteomic markers, L-Ficolin, HA, and ST2, as early predictors of SOS incidence through study completion.
Time frame: Until the end of the study evaluation, day 180
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