Stem Cell Transplant Complications Clinical Trial
Official title:
Validating Ultrasound Biomarkers for Hepatic Sinusoidal Obstruction Syndrome in Pediatric Hematopoietic Cell Transplant Patients
Verified date | January 2020 |
Source | Children's Mercy Hospital Kansas City |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
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).
Status | Withdrawn |
Enrollment | 0 |
Est. completion date | April 1, 2025 |
Est. primary completion date | April 30, 2024 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 1 Month to 25 Years |
Eligibility |
Inclusion Criteria: - HCT patient under 25 years of age with - myeloablative conditioning, - prior liver damage or - other high risk factor: - Neuroblastoma, - HLH, - Osteopetrosis, - Thalassemia, - Treatment with inotuzumab or gemtuzumab within 3 months prior to HCT admission, - Hepatic iron overload, - Steatohepatitis, - Active inflammatory or infection hepatitis or - Any other condition which puts the patient at a higher risk of developing VOD). Exclusion Criteria: - Any patient who has contraindication to any of the ultrasound procedures (e.g. unable to hold still). |
Country | Name | City | State |
---|---|---|---|
United States | Children's Mercy Hospital | Kansas City | Missouri |
Lead Sponsor | Collaborator |
---|---|
Children's Mercy Hospital Kansas City | Children's Hospital Colorado, Nationwide Children's Hospital, St. Jude Children's Research Hospital |
United States,
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* Note: There are 19 references in all — Click here to view all references
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Define Sensitivity and Specificity of US (grayscale, Doppler and SWE) for Diagnosis and Severity Grading for SOS/VOD | Determine the sensitivity and specificity of US for diagnosis and severity grading of VOD/SOS in a cohort of 250 pediatric HCT patients. | 100 days post transplant | |
Primary | Define Sensitivity and Specificity of CEUS for Diagnosis and Severity Grading for SOS/VOD | Determine the sensitivity and specificity of contrast enhanced ultrasound (CEUS) variables for diagnosis and severity grading of VOD/SOS as compared to the pediatric EBMT criteria. | 100 days post transplant | |
Primary | Define Optimal Timing for US for Early Diagnosis and Risk Stratification of VOD/SOS | Determine the optimal timing for ultrasound measurements to help predict early diagnosis and risk stratification of VOD/SOS. | 100 days post transplant |
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