Ultrasound Elastography to Predict Development of SOS

NCT03858530 | Terminated Results FDA Device

• 1 other identifier: 17110684
• Study Type: interventional
• Target: 21
• Locations: 1 country
• Sites: 1

Brief Summary

The long-term goal of our research is to accurately identify SOS patients who would benefit from defibrotide treatment using US SWE. The overall objective of this study is to validate SWE as an early diagnostic marker for SOS. Our central hypothesis is that SWE changes will precede clinical and conventional US diagnostic criteria for SOS. Our hypothesis has been formulated on the basis of our own preliminary data. The investigators completed the first prospective cohort trial demonstrating that US SWE provides SOS diagnosis (80% sensitivity and 67% specificity) nine days earlier than current clinical criteria. SWE is widely available, has no known side effects, and is easy to learn and interpret. Our study enrolled 25 high-risk BMT patients over 18 months (five with SOS and two with severe SOS). More data is needed to determine the optimal window for testing to balance between improved test characteristics and early detection of disease. The investigators propose conducting a prospective cohort study with 80 additional patients, 12 of which will likely develop SOS (including four with severe SOS) to optimize SWE timing. This study will increase the confidence in the findings from our preliminary study and allow us to test SWE against newly published clinical criteria. The rationale for the proposed research is that, if SWE can diagnose SOS earlier than clinical criteria, then SWE can guide early initiation of SOS treatment.

Conditions

Bone Marrow Transplant Complications; Sinusoidal Obstruction Syndrome; Veno-occlusive Disease; Stem Cell Transplant Complications

Keywords

Ultrasound elastography

Outcome Measures

Primary Outcomes (1)

• Number of Days Between SWE Exams for VOD/SOS

  We performed SWE exams once a week for all of our patients. We looked at when patients met clinical criteria for VOD/SOS and then we looked at the SWE exams that were performed before the clinical diagnosis. If the exam immediately preceding the date of clinical diagnosis crossed the threshold for SWE diagnosis, then a frequency of 7 days was sufficient. If it did not, then testing should be more frequent. If multiple preceding exams were positive by SWE, then the optimal testing frequency could be longer than 7 days.

  Once a week through study completion assessed for up to 14 weeks

Secondary Outcomes (3)

• Determine if SWE Can Provide Earlier SOS Diagnosis Compared to Clinical Criteria.

  Daily through study completion assessed for up to 100 days

• Difference in Maximum SWE Value in Patients With Severe and Very Severe VOD Compared to Those With Mild and Moderate VOD.

  Weekly through study completion assessed for up to 14 weeks

• CEUS Variables Versus Grayscale and Doppler Variables

  Study period

Study Arms (1)

All Patients Enrolled

EXPERIMENTAL

All patients will undergo limited abdominal US with Doppler and SWE once a week upon admission for conditioning until the patient day +30 BMT or discharge, whichever comes first. Additional ultrasounds will also be performed if SOS is suspected.

Diagnostic Test: Ultrasound Examination

Interventions

Ultrasound Examination DIAGNOSTIC_TEST

Grayscale ultrasound, Doppler ultrasound, ultrasound elastography with or without ultrasound intravenous contrast will be performed.

All Patients Enrolled

Eligibility Criteria

• Age: 1 Month - 21 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Children and adults, ages 1 month through 21 years who are undergoing allogenic or autologous myeloablative stem cell transplant.

You may not qualify if:

• Any other medical or social condition that in the opinion of the investigator would make them unsuitable to participate.
• Inability to properly image patient by ultrasound (e.g. uncooperative)

Sponsors & Collaborators

• Children's Mercy Hospital Kansas City: lead

Study Sites (1)

Children's Mercy Hospital

Kansas City, Missouri, 64108, United States

Location

Related Publications (23)

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Related Links

• D'Souza A ZX. Current Uses and Outcomes of Hematopoietic Cell Transplantation (HCT): CIBMTR Summary Slides, 2016.
• Cozadd B (2018) Jazz Pharmaceuticals. 36th Annual JP Morgan Annual Healthcare Conference, San Francisco, California.
• (2016) In Brief: Defibrotide (Defitelio) for Hepatic Veno-Occlusive Disease. The Medical Letter

MeSH Terms

Conditions

Hepatic Veno-Occlusive Disease

Condition Hierarchy (Ancestors)

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

Limitations and Caveats

Early termination and data necessary for statistical analysis was not collected before research approval expired. The IRB closed the study, requiring the data to since be de-identified. There is no accessible data available to report.

Results Point of Contact

• Title: Dr. Sherwin Chan
• Organization: Children's Mercy Kansas City

sschan@cmh.edu

816-234-3273

Study Officials

• Shewin Chan, MD, PhD

  Children's Mercy Hospital Kansas City

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes

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

Study Record Dates

• First Submitted: December 10, 2018
• First Posted: February 28, 2019
• Study Start: May 1, 2018
• Primary Completion: January 26, 2022
• Study Completion: January 16, 2024
• Last Updated: September 4, 2024
• Results First Posted: September 4, 2024

Record last verified: 2024-08

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)