Sinusoidal Obstruction Syndrome for Stem Cell Transplant Patients Biomarker Study
SOSBiomarker
Biomarkers of Endothelial Dysfunction in Pediatric Patients Receiving High Intensity Chemotherapy/Irradiation
1 other identifier
observational
80
1 country
4
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P50-P75 for all trials
Started Apr 2017
Typical duration for all trials
4 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
Study Start
First participant enrolled
April 1, 2017
CompletedFirst Submitted
Initial submission to the registry
April 24, 2017
CompletedFirst Posted
Study publicly available on registry
April 27, 2017
CompletedPrimary Completion
Last participant's last visit for primary outcome
January 14, 2021
CompletedStudy Completion
Last participant's last visit for all outcomes
January 14, 2021
CompletedDecember 19, 2023
December 1, 2023
3.8 years
April 24, 2017
December 18, 2023
Conditions
Outcome Measures
Primary Outcomes (1)
SOS proteomic markers
Measure for 3 SOS proteomic markers, L-Ficolin, HA, and ST2, as early predictors of SOS incidence through study completion.
Until the end of the study evaluation, day 180
Study Arms (1)
Stem Cell Transplant
Serial Blood Draws
Interventions
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.
Eligibility Criteria
Stem Cell Transplant patients
You may qualify if:
- 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
- High-risk disease states including:
- Juvenile myelo-monocytic chronic leukemia (JMML)
- Primary hemophagocytic lymphohistiocytosis (HLH)
- Adrenoleukodystrophy
- Osteopetrosis
- +6 more criteria
You may not qualify if:
- Patients who are transplanted but do not fulfill any of the above mentioned criteria.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (4)
Children's National Medical Center
Washington D.C., District of Columbia, 20010, United States
Indiana University
Indianapolis, Indiana, 46202, United States
University of Michigan
Ann Arbor, Michigan, 48109, United States
Baylor College of Medicine
Houston, Texas, 77030, United States
Related Publications (1)
Han Y, Bidgoli A, DePriest BP, Mendez A, Bijangi-Vishehsaraei K, Perez-Albuerne ED, Krance RA, Renbarger J, Skiles JL, Choi SW, Liu H, Paczesny S. Prospective assessment of risk biomarkers of sinusoidal obstruction syndrome after hematopoietic cell transplantation. JCI Insight. 2023 May 22;8(10):e168221. doi: 10.1172/jci.insight.168221.
PMID: 37071469DERIVED
Biospecimen
Blood
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Study Design
- Study Type
- observational
- Observational Model
- COHORT
- Time Perspective
- PROSPECTIVE
- Sponsor Type
- OTHER
- Responsible Party
- PRINCIPAL INVESTIGATOR
- PI Title
- Professor, Department of Pediatrics
Study Record Dates
First Submitted
April 24, 2017
First Posted
April 27, 2017
Study Start
April 1, 2017
Primary Completion
January 14, 2021
Study Completion
January 14, 2021
Last Updated
December 19, 2023
Record last verified: 2023-12
Data Sharing
- IPD Sharing
- Will not share