Evidence map›Paper›PMID 31633654›Full record

ArticleJournal of pediatric gastroenterology and nutrition2020

Evaluation of Quantitative Imaging Biomarkers for Early-phase Clinical Trials of Steatohepatitis in Adolescents.

Nidhi P Goyal, Mary Catherine Sawh, Patricia Ugalde-Nicalo, Jorge E Angeles, James A Proudfoot, Kimberly P Newton, Michael S Middleton, Claude B Sirlin, Jeffrey B Schwimmer

Open access · greenAbstract readEvaluation Study
In one paragraph

Article in Journal of pediatric gastroenterology and nutrition, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 9 papers, 2 of them syntheses that pooled it.

0numbers the graph read from it
0cells of the map it votes in
9citing papers in PubMed, 2 pooled it
0.6field-weighted citation impact, top 29% of its field
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

9 citing papers in PubMed, 2 syntheses or guidelines pooled it, 11 citations in OpenAlex.

  1. Pooled it
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  5. Review
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4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

9 authors at 3 institutions in 1 country.

Nidhi P GoyalDivision of Gastroenterology, Hepatology, and Nutrition, Department of Pediatrics, University of California, San Diego School of Medicine.
Mary Catherine SawhDivision of Gastroenterology, Hepatology, and Nutrition, Department of Pediatrics, University of California, San Diego School of Medicine.
Patricia Ugalde-NicaloDivision of Gastroenterology, Hepatology, and Nutrition, Department of Pediatrics, University of California, San Diego School of Medicine.
Jorge E AngelesDivision of Gastroenterology, Hepatology, and Nutrition, Department of Pediatrics, University of California, San Diego School of Medicine.
James A ProudfootClinical and Translational Research Institute.
Kimberly P NewtonDivision of Gastroenterology, Hepatology, and Nutrition, Department of Pediatrics, University of California, San Diego School of Medicine.
Michael S MiddletonLiver Imaging Group, Department of Radiology, University of California, San Diego School of Medicine, San Diego, CA.
Claude B SirlinLiver Imaging Group, Department of Radiology, University of California, San Diego School of Medicine, San Diego, CA.
Jeffrey B SchwimmerDivision of Gastroenterology, Hepatology, and Nutrition, Department of Pediatrics, University of California, San Diego School of Medicine.
University of California, San Diego · USRady Children's Hospital-San Diego · USSouthern California Clinical and Translational Science Institute · US

Funding

UC San Diego Clinical and Translational Research InstituteUL1TR001442 · NCATS · UNIVERSITY OF CALIFORNIA, SAN DIEGO · PI FIRESTEIN, GARY S, HOGARTH, MICHAEL · 2015 to 2024
$88.3M
NCATS NIH HHS UL1 TR001442
6 · The paper itself

Abstract

objectivesEarly-phase pediatric nonalcoholic fatty liver disease (NAFLD) clinical trials are designed with noninvasive parameters to assess potential efficacy. Increasingly, these parameters include magnetic resonance imaging (MRI)-derived proton density fat fraction (PDFF) and MR elastography (MRE)-derived shear stiffness as biomarkers of hepatic steatosis and fibrosis, respectively. Understanding fluctuations in these measures is essential for calculating trial sample sizes, interpreting results, and planning clinical drug trials in children with NAFLD. Lack of such data in children constitutes a critical knowledge gap. Therefore, the primary aim of this study was to assess whole-liver MRI-PDFF change in adolescents with nonalcoholic steatohepatitis (NASH) over 12 weeks.

methodsAdolescents 12 to 19 years with biopsy-proven NASH undergoing standard-of-care treatment were enrolled. Baseline and week-12 assessments of anthropometrics, transaminases, MRI-PDFF, and MRE stiffness were obtained.

resultsFifteen adolescents were included (mean age 15.7 [SD 2.9] years). Hepatic MRI-PDFF was stable over 12 weeks (mean absolute change -0.8%, P = 0.24). Correlation between baseline and week-12 values of MRI-PDFF was high (ICC = 0.97, 95% CI 0.90-0.99). MRE stiffness was stable (mean percentage change 2.7%, P = 0.44); correlation between baseline and week-12 values was moderate (ICC = 0.47; 95% CI 0-0.79). Changes in weight, BMI, and aminotransferases were not statistically significant.

conclusionIn adolescents with NASH, fluctuations in hepatic MRI-PDFF and MRE stiffness over 12 weeks of standard-of-care were small. These data on the natural fluctuations in quantitative imaging biomarkers can serve as a reference for interventional trials in pediatric NASH and inform the interpretation and planning of clinical trials.

Indexed as

Patient SelectionAdolescentBiomarkersChildClinical Trials as TopicElasticity Imaging TechniquesFemaleHumansLiverMagnetic Resonance ImagingMaleNon-alcoholic Fatty Liver DiseaseYoung AdultBiomarkers

Identifiers

PMID31633654
PMCPMC8053386
OpenAlexW2980832793

What OpenQuestion holds

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Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.