Validating Ultrasound Biomarkers for Hepatic Sinusoidal Obstruction Syndrome in Pediatric Hematopoietic Cell Transplant Patients

Summary

Hepatic veno-occlusive disease/sinusoidal obstructive syndrome (VOD/SOS) is a potentially fatal complication of hematopoietic cell transplant (HCT). Historically VOD/SOS has been clinically diagnosed using the modified Seattle criteria or the Baltimore criteria. The modified Seattle Criteria define VOD/SOS diagnosis is made when two of the following three criteria are present in a patient within 21 days of transplantation: hyperbilirubinemia (total serum bilirubin \> 2 mg/dL), hepatomegaly or right upper quadrant liver pain, and weight gain (\> 2% of baseline) or ascites. Other conditions like graft versus host disease, sepsis syndrome (fever and hypotension), cardiac failure, or tumor infiltration) have to be excluded. This definition was from a well-designed retrospective cohort study on 255 adult and pediatric HCT patients in which the VOD/SOS incidence was 21%. McDonald et al followed up this work with a prospective cohort study of 355 patients noting an incidence of VOD/SOS of 54%. These seminal studies have had a major impact on the field by defining clinical diagnostic criteria. An alternative diagnostic criteria (Baltimore criteria) was proposed by Jones et al as a part of a well-designed retrospective review of 235 HCT patients finding a VOD/SOS incidence of 22%. Jones defined VOD/SOS as the presence of hyperbilirubinemia (total serum bilirubin \> 2 mg/dL) along with at least 2 of 3 other findings: hepatomegaly, ascites, and weight gain (\> 5% of baseline).

Conditions

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

Interventions

DIAGNOSTIC_TEST

Ultrasound Elastography

Ultrasound shear wave elastography

DRUG

Contrast Enhanced Ultrasound (CEUS)

Contrast-enhanced ultrasound (CEUS) uses an intravenous injection of microbubble contrast agent.

Sponsors & Collaborators

• Nationwide Children's Hospital

  collaborator OTHER

• St. Jude Children's Research Hospital

  collaborator OTHER

• Children's Hospital Colorado

  collaborator OTHER

• Children's Mercy Hospital Kansas City

  lead OTHER

Principal Investigators

• Sherwin S Chan, MD, PhD · Children's Mercy Hospital Kansas City

Study Design

Allocation

NA

Purpose

DIAGNOSTIC

Masking

NONE

Model

SINGLE_GROUP

Eligibility

Min Age

1 Month

Max Age

25 Years

Sex

ALL

Healthy Volunteers

No

Timeline & Regulatory

Start

2020-04-01

Primary Completion

2024-04-30

Completion

2025-04-01

FDA Drug

Yes

Countries

• United States

Study Locations