Introduction
ascites, hepatomegaly, VOD, pain, weight gain, sinusoidal obstruction syndrome
SINUSOIDAL OBSTRUCTION SYNDROME, ASCITES, VENO-OCCLUSIVE DISEASE, DISEASE, COMPLICATION, BLOOD DISORDERS, ENDOTHELIAL DYSFUNCTION
Allogeneic hematopoietic cell transplantation (HCT) is a potentially curative therapy for blood disorders. However, the efficacy of this procedure has been impeded by early endothelial dysfunction that can lead to a severe and potentially lethal complication called sinusoidal obstruction syndrome (SOS), also known as veno-occlusive disease (VOD) (Defibrotide has received Food and Drug Administration (FDA) approval for SOS treatment (SOS diagnosis and its severity are assessed late in the course of the disease by nonspecific clinical signs and laboratory assays (ascites, weight gain, hepatomegaly, right upper quadrant pain, and bilirubin ≥ 2 mg/dL) (Currently, no validated laboratory test exists to stratify patients at high risk for developing SOS. Neither pretransplant clinical characteristics (recipient or donor) nor transplant characteristics have proven to be reliable predictors of SOS (L-ficolin, hyaluronic acid (HA), and stimulation 2 (ST2) were identified and validated in 3 retrospective cohorts as risk biomarkers for SOS (Herein, we prospectively assessed L-ficolin, HA, and ST2 at 2 early time points following HCT in a multicenter pediatric cohort. The optimal biomarker cutpoints were determined by exhaustive grid search and Youden’s index using biomarker data from all retrospective cohorts from our previously published study (