Using ultrasound to predict liver complications after bone marrow transplant
Using Ultrasound Elastography to Predict Development of Hepatic Sinusoidal Obstruction Syndrome
This study is testing if ultrasound can help predict liver problems in patients getting a bone marrow transplant to improve early detection and treatment.
Quick facts
| Phase | Not applicable |
|---|---|
| Study type | Interventional |
| Enrollment | 250 (estimated) |
| Ages | 1 Month to 99 Years |
| Sex | All |
| Sponsor | Children's Mercy Hospital Kansas City Academic / other |
| Drugs / interventions | inotuzumab, gemtuzumab, Cyclophosphamide |
| Locations | 7 sites (San Francisco, California and 6 other locations) |
| Trial ID | NCT03865589 on ClinicalTrials.gov |
What this trial studies
This study aims to evaluate the effectiveness of ultrasound elastography in predicting hepatic sinusoidal obstruction syndrome (SOS) in patients undergoing hematopoietic stem cell transplant (HCT). It will perform a receiver operating characteristic (ROC) analysis to establish a threshold for risk stratification into categories of SOS severity based on established clinical criteria. The study will also investigate the temporal relationship between changes in ultrasound elastography measurements and the diagnosis of SOS. Given the high morbidity and mortality associated with SOS, this research seeks to improve early detection and management of this serious complication.
Who should consider this trial
Good fit: Ideal candidates for this study are patients undergoing myeloablative conditioning regimens for hematopoietic stem cell transplant.
Not a fit: Patients who are not undergoing myeloablative conditioning regimens or those with pre-existing severe liver conditions may not benefit from this study.
Why it matters
Potential benefit: If successful, this study could lead to improved early detection and management of hepatic complications in patients undergoing bone marrow transplants.
How similar studies have performed: Other studies have shown promise in using ultrasound elastography for similar applications, indicating potential for success in this approach.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * 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. OR 2. Any patient who has a myeloablative conditioning regimen (as defined by the local HCT team) that includes sirolimus and tacrolimus for GVHD prophylaxis. OR 3. 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. Inclusion dates: Patients seen between 1/1/2019 and 12/31/2025 Exclusion Criteria: 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
Where this trial is running
San Francisco, California and 6 other locations
- University of California, San Francisco — San Francisco, California, United States (Recruiting)
- Dana-Farber Cancer Institute — Boston, Massachusetts, United States (Recruiting)
- Children's Mercy — Kansas City, Missouri, United States (Recruiting)
- Duke University — Durham, North Carolina, United States (Recruiting)
- University Hospitals Cleveland Medical Center — Cleveland, Ohio, United States (Recruiting)
- Nationwide Children's Hospital — Columbus, Ohio, United States (Recruiting)
- St. Jude Children's Research Hospital — Memphis, Tennessee, United States (Completed)
Study contacts
- Principal investigator: Sherwin S Chan, MD, PhD — Children's Mercy Hospital Kansas City
- Study coordinator: Maura Sien, MSML, CCRC
- Email: mesien@cmh.edu
- Phone: 8163028311
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.