Using Ultrasound Elastography to Predict Development of Hepatic Sinusoidal Obstruction Syndrome

NCT03865589 | Active Not Recruiting FDA Device

• 1 other identifier: STUDY0000548
• Study Type: interventional
• Target: 250
• Locations: 1 country
• Sites: 7

Brief Summary

To perform an receiver operating characteristic (ROC) analysis, define a threshold and quantify the sensitivity and specificity of US SWE for risk stratification of patients into three categories as defined by the European Bone Marrow Transplant (EBMT) adult and pediatric criteria: no sinusoidal obstruction syndrome (SOS), mild to moderate SOS, and severe to very severe SOS. Secondarily, the investigators would also like to quantify the temporal relationship between US SWE changes and SOS diagnosis according to various clinical criteria (Modified Seattle, Baltimore, EBMT consortium).

Conditions

Sinusoidal Obstruction Syndrome; Veno Occlusive Disease, Hepatic; Stem Cell Transplant Complications; Bone Marrow Transplant Complications

Keywords

Ultrasound Elastography

Outcome Measures

Primary Outcomes (1)

• Define Sensitivity and Specificity Threshold for US SWE Risk

  To define a threshold and quantify the sensitivity and specificity of US SWE for risk stratification of patients into three categories as defined by the EBMTC adult and pediatric criteria: no SOS, mild to moderate SOS, and severe to very severe SOS

  100 days post transplant

Secondary Outcomes (3)

• Quantify Temporal Relationship between SWE and SOS and Modified Seattle Criteria

  100 days post transplant

• Quantify Temporal Relationship between SWE and SOS and Baltimore Criteria

  100 days post transplant

• Quantify Temporal Relationship between SWE and SOS and EBMT Consortium

  100 days post transplant

Study Arms (1)

Patients Undergoing HCT

EXPERIMENTAL

All patients enrolled will undergo US SWE at specific time points as outlined in the protocol based on disease course.

Diagnostic Test: Ultrasound Elastography

Interventions

Ultrasound Elastography DIAGNOSTIC_TEST

Ultrasound shear wave elastography

Patients Undergoing HCT

Eligibility Criteria

• Age: 1 Month - 99 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Any patient undergoing a myeloablative conditioning regimen for HCT between 3/1/2019 and 12/31/2025 defined as one of the following:
• TBI \>= 1200 cGy (fractionated)
• Cyclophosphamide + TBI (\> 500 cGy (single) or \> 800cGy (fractionated))
• Cyclophosphamide + Etoposide + TBI (\> 500 cGy (single) or \> 800 cGy (fractionated))
• Cyclophosphamide + Thiotepa + TBI (\> 500 cGy (single) or \> 800 cGy (fractionated))
• Busulfan (Total dose \> 7.2 mg/kg IV or \>9.0mg/kg orally) + Cyclophosphamide
• Busulfan (Total dose \>7.2 mg/kg IV or \>9.0 mg/kg orally) + Melphalan
• Busulfan (Total dose \>7.2 mg/kg IV or \>9.0 mg/kg orally) + Thiotepa
• NOTE: Busulfan cumulative plasma AUC of \>75 mg/L per hour or \>18270 microMolar per minute could be used in the preceding criteria in lieu of the mg/kg doses.
• \. Any patient who has a myeloablative conditioning regimen (as defined by the local HCT team) that includes sirolimus and tacrolimus for GVHD prophylaxis.
• \. Any patient who is high risk for SOS irrespective of conditioning regimen: Neuroblastoma, HLH, Osteopetrosis, Thalassemia, treatment with inotuzumab or gemtuzumab within 3 months prior to HCT admission, 2nd HCT if it is myeloablative and within 6 months of prior, iron overload, steatohepatitis, active inflammatory or infection hepatitis or any other condition which puts the patient at a higher risk of developing SOS.
• Subjects aged 1 month through 99 years will be eligible for the study. Patients who receive defibrotide and/or ursodiol for VOD/SOS prophylaxis will also be eligible for the study. At most 175 children (ages 1 month to 18 years) and 175 adults (\>18 years old) can be enrolled into the study.

You may not qualify if:

• Any patient who has contraindication to ultrasound shear wave elastography (e.g. unable to hold still) Adults unable to consent Pregnant women Prisoners Wards of the state

Sponsors & Collaborators

• Jazz Pharmaceuticals: collaborator
• Nationwide Children's Hospital: collaborator
• Dana-Farber Cancer Institute: collaborator
• University of California, San Francisco: collaborator
• University Hospitals Cleveland Medical Center: collaborator
• Duke University: collaborator
• Children's Mercy Hospital Kansas City: lead

Study Sites (7)

University of California, San Francisco

San Francisco, California, 94143, United States

Location

Dana-Farber Cancer Institute

Boston, Massachusetts, 02215, United States

Location

Children's Mercy

Kansas City, Missouri, 64108, United States

Location

Duke University

Durham, North Carolina, 27705, United States

Location

University Hospitals Cleveland Medical Center

Cleveland, Ohio, 44106, United States

Location

Nationwide Children's Hospital

Columbus, Ohio, 43205, United States

Location

St. Jude Children's Research Hospital

Memphis, Tennessee, 38105, United States

Location

Related Publications (23)

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MeSH Terms

Conditions

Hepatic Veno-Occlusive Disease

Condition Hierarchy (Ancestors)

Liver Diseases; Digestive System Diseases; Vascular Diseases; Cardiovascular Diseases

Study Officials

• Sherwin S Chan, MD, PhD

  Children's Mercy Hospital Kansas City

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: DIAGNOSTIC
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Physician, MD, PhD

Model Details: All patients undergoing myeloblative conditioning regimen as part of hematopoietic cell transplant will be consecutively studied

Study Record Dates

• First Submitted: March 1, 2019
• First Posted: March 7, 2019
• Study Start: April 1, 2019
• Primary Completion (Estimated): June 30, 2026
• Study Completion (Estimated): June 30, 2027
• Last Updated: January 8, 2026

Record last verified: 2026-01

Data Sharing

• IPD Sharing: Will not share

Locations

US (7)