Quantitative Ultrasound To Diagnose Nonalcoholic Fatty Liver Disease in Children.

NIH RePORTER · NIH · R01 · $703,391 · view on reporter.nih.gov ↗

Abstract

SUMMARY Our broad long-term objective is to improve the health of children with nonalcoholic fatty liver disease (NAFLD), a condition characterized by accumulation of fat in liver cells, by developing and validating accurate, precise, practical, and widely available tools to detect, measure, and monitor liver fat. Over 5 million American children have NAFLD, placing them at risk for type 2 diabetes, cirrhosis, and premature death. Addressing this health crisis is crucial for our nation. Clinical practice guidelines recommend screening for NAFLD in American children aged 9 to 17 years with overweight or obesity because early diagnosis, prompt specialist referral, and NAFLD- tailored management can improve outcomes. However, there is no accurate, precise, practical, and widely available diagnostic tool to implement these guidelines, a major unmet need in pediatric NAFLD. Quantitative ultrasound (QUS, US) holds promise to address this unmet need, but notable gaps in QUS technology persist: Gap 1) unsatisfactory accuracy of current commercial QUS methods for estimating liver fat content in children; Gap 2) availability of commercial QUS technology mainly on full-size (US) systems, primarily located within radiology departments, limiting access for many children at risk NAFLD; and Gap 3) poor cross-vendor reproducibility, which undermines utility. Our immediate goal is to address these gaps by developing advanced QUS analysis models for use with full-size US or inexpensive POCUS systems in children at risk for NAFLD to estimate liver fat fraction and classify presence/absence of fatty liver. The models will incorporate technical innovations to improve QUS accuracy and efficiency. To develop and test these models, we propose a cross- sectional accuracy and precision clinical study in 140 children (ages 9 to 17 years) suspected to have NAFLD, who will undergo full-size US and POCUS exams with contemporaneous MRI-proton density fat fraction as reference. Our three specific aims are: Aim 1. To improve accuracy of QUS on full-size US for liver fat assessment in children. Aim 2. To translate QUS liver fat assessment in children to POCUS. Aim 3. To improve cross-vendor reproducibility of QUS liver fat assessment in children. We hypothesize that our QUS models using either full-size US or POCUS will estimate liver fat content more accurately than other US methods. We also expect our models to classify presence/absence of fatty liver in children more accurately than other US methods. Exploratory aims are to: a) explore associations between QUS and histology; b) explore effects of potential confounders on accuracy and precision of QUS models; c) create a repository of US data, plasma, and digitized histology for future research. Impact: Achieving our study aims will enable multi-center, multi-vendor trials, which collectively have the potential to transform clinical practice and therapeutic discovery in children by enabling widespread dissemination of reliable,...

Key facts

NIH application ID
10882535
Project number
1R01DK135951-01A1
Recipient
UNIVERSITY OF CALIFORNIA, SAN DIEGO
Principal Investigator
CLAUDE B SIRLIN
Activity code
R01
Funding institute
NIH
Fiscal year
2024
Award amount
$703,391
Award type
1
Project period
2024-07-01 → 2029-04-30