Biomarkers of Endothelial Dysfunction in Pediatric Patients Receiving High Intensity Chemotherapy/Irradiation
Overview
- Phase
- Not Applicable
- Intervention
- Not specified
- Conditions
- Sinusoidal Obstruction Syndrome
- Sponsor
- Indiana University
- Enrollment
- 80
- Locations
- 4
- Primary Endpoint
- SOS proteomic markers
- Status
- Completed
- Last Updated
- 2 years ago
Overview
Brief Summary
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.
Detailed Description
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.
Investigators
Sophie Paczesny
Professor, Department of Pediatrics
Indiana University
Eligibility Criteria
Inclusion Criteria
- •Age ≤ 25 years undergoing HCT for any reason who fulfill any ONE (1) of the following criteria:
- •History of hepatic disease as defined by:
- •Viral hepatitis (i.e., hepatitis C virus \[HCV\])
- •Liver tumor before HCT
- •Hepatic fibrosis or cirrhosis before HCT as proven by liver biopsy
- •High aspartate aminotransferase (AST) (\> 2x ULN) before HCT (pre-transplant evaluation)
- •High alanine transaminase (ALT) (\> 2x ULN) before HCT
- •High bilirubin (\> 1.2x ULN) before HCT
- •HCT high-risk features including:
- •a. Conditioning with high-risk modalities including: i. Busulfan (BU)-containing regimen particularly with oral BU + cyclophosphamide ii. TBI-containing regimen, particularly cyclophosphamide + total-body irradiation (TBI) b. ≥ 2 HCT c. Allo-HCT for leukemia \> or = second relapse d. Unrelated donor (URD) HCT e. Human leukocyte antigen (HLA) mismatch HCT (less than 10 of 10 for bone marrow/peripheral blood stem cell \[BM/PBSC\] or anything less than 6 of 6 for UCB) f. Use of sirolimus + tacrolimus prophylaxis for GVHD
Exclusion Criteria
- •Patients who are transplanted but do not fulfill any of the above mentioned criteria.
Outcomes
Primary Outcomes
SOS proteomic markers
Time Frame: Until the end of the study evaluation, day 180
Measure for 3 SOS proteomic markers, L-Ficolin, HA, and ST2, as early predictors of SOS incidence through study completion.