SOSBiomarker: Sinusoidal Obstruction Syndrome for Stem Cell Transplant Patients Biomarker Study
Study Details
Study Description
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.
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
|
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.
Study Design
Arms and Interventions
| Arm | Intervention/Treatment |
|---|---|
| Stem Cell Transplant Serial Blood Draws |
Other: Serial Blood Draws
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.
|
Outcome Measures
Primary Outcome Measures
- SOS proteomic markers [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.
Eligibility Criteria
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:
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Viral hepatitis (i.e., hepatitis C virus [HCV])
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Liver tumor before HCT
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Hepatic fibrosis or cirrhosis before HCT as proven by liver biopsy
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High aspartate aminotransferase (AST) (> 2x ULN) before HCT (pre-transplant evaluation)
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High alanine transaminase (ALT) (> 2x ULN) before HCT
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High bilirubin (> 1.2x ULN) before HCT
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HCT high-risk features including:
- 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
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High-risk disease states including:
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Juvenile myelo-monocytic chronic leukemia (JMML)
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Primary hemophagocytic lymphohistiocytosis (HLH)
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Adrenoleukodystrophy
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Osteopetrosis
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Other high-risk features including:
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Prior treatment with gemtuzumab ozogamicin
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Use of hepatotoxic drugs 1 month before HCT and during HCT
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Iron overload (i.e., thalassemia/sickle cell) with serum ferritin > 1000ng/ml
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Deficit of ATIII, T-PA (i.e., < 30% normal values), and resistance to activated protein C if clinical indication (these values do not have to be specifically checked if no clinical history)
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Young age < 2 years but more than 1 month
Exclusion Criteria:
Patients who are transplanted but do not fulfill any of the above mentioned criteria.
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | Children's National Medical Center | Washington | District of Columbia | United States | 20010 |
| 2 | Indiana University | Indianapolis | Indiana | United States | 46202 |
| 3 | University of Michigan | Ann Arbor | Michigan | United States | 48109 |
| 4 | Baylor College of Medicine | Houston | Texas | United States | 77030 |
Sponsors and Collaborators
- Indiana University
Investigators
None specified.Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- 1701020549