Hypertension, Portal
Conditions
Brief summary
Early diagnosis of portal hypertension is difficult as symptoms rarely manifest until the later stages of liver disease. Both cirrhotic and non-cirrhotic portal hypertension can result in life-threatening complications, the most frequent of which is bleeding from esophageal varices. In children, variceal bleeds are associated with mortality rates of 1-3 %, while life-threatening complications have been reported in up to 20 % of children with cirrhosis. Despite the high incidence of portal hypertension in children with liver disease, a noninvasive modality to monitor disease progression and risk of complications is currently lacking. Hence, this trial will investigate the safety and efficacy of subharmonic aided pressure estimation (SHAPE) as a noninvasive ultrasound technique for diagnosing portal hypertension in children.
Interventions
DRUGSHAPE measurement using the ultrasound contrast agent Sonazoid (perfluorobutane microbubbles)
The ultrasound contrast agent will be infused thorough an IV line and SHAPE ultrasound imaging and data acquisition will be performed with a Logiq E10 (GE Medical Systems, Waukesha, WI) ultrasound scanner.
DRUGSHAPE measurement using the ultrasound contrast agent Lumason (sulfur hexafluoride lipid-type A microspheres)
The ultrasound contrast agent will be infused thorough an IV line and SHAPE ultrasound imaging and data acquisition will be performed with a Logiq E10 (GE Medical Systems, Waukesha, WI) ultrasound scanner.
Sponsors
Children's Hospital of Philadelphia
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Thomas Jefferson University
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
DIAGNOSTIC
Masking
NONE
Intervention model description
A prospective randomized cohort study with a longitudinal arm. There will be 2 cohorts.
Eligibility
Sex/Gender
ALL
Age
6 Years to 21 Years
Healthy volunteers
No
Inclusion criteria
* Patients with a diagnosis of chronic liver disease without portal hypertension. * Patients with a diagnosis of chronic liver disease with portal hypertension.
Exclusion criteria
* Subjects who are pregnant. * Patients with known or suspected hypersensitivity to egg phosphatidyl serine or with a history of anaphylactic allergy to eggs or egg products. * Subjects with allergy to egg products or other components of the ultrasound contrast agents will be excluded. * History of allergic reaction to Lumason®, sulfur hexafluoride, sulfur hexafluoride lipid microsphere components, or other ingredients in Lumason (polyethylene glycol, distearoylphosphatidylcholine (DSPC), dipalmitoylphosphatidylglycerol sodium (DPPG-Na), palmitic acid) * History of allergic reaction to Sonazoid * Patients with biliary atresia with asplenia or polysplenia. * Patients with prior liver transplant. * Patients with cystic fibrosis. * Patients with chronic lung disease. * Patients with portal vein thrombosis, cavernous transformation of the portal vein or absent portal vein. * Adults not competent/impaired. * Patients with significant heart disease or severe congenital heart disease
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| The rate (%) of adverse events that occur with Sonazoid compared to the current rate of adverse events that have been reported in the Lumason package insert for pediatric use (0.001%) | 2 hours |
| Calibrated subharmonic microbubble signals (in dB) between the portal and hepatic veins will differentiate between the portal hypertension and non-portal hypertension groups with an accuracy of 94% | 2 hours |
Secondary
| Measure | Time frame |
|---|---|
| Changes in calibrated subharmonic microbubble signals (in dB) between the portal and hepatic veins over time will predict the development of complications of portal hypertension (variceal bleeding, ascites, hepatopulmonary syndrome, etc) | on average 18 months |
| Calibrated subharmonic microbubble signals (in dB) between the portal and hepatic veins and liver fibrosis stage (on a scale from 0-4 as determined by liver biopsy) will correlate with a p<0.05 | 2 hours |
| Compare the calibrated subharmonic microbubble signals (in dB) between the portal and hepatic veins obtained with Lumason and Sonazoid, respectively, for their ability to differentiate between the portal hypertension and non-portal hypertension groups | 2 hours |
| Compare calibrated subharmonic microbubble signals (in dB) between the portal and hepatic veins to shear wave elastography values (in m/s) using regression analysis | 2 hours |
Countries
United States
Contacts
Primary ContactMorgan L Gabbert, MA
Outcome results
None listed