Prospective Multicenter Longitudinal Measurement of Liver Stiffness in School-Age Children With Cholestatic Liver Disease

Authors

Benjamin L Shneider
Nathan P Goodrich
Wen Ye
Jean P Molleston
Daniel H Leung
Ronald J Sokol
Laurel Cavallo
Kasper Wang
Pamela L Valentino
Jeffery H Teckman
James E Squires
Shikha S Sundaram
Philip Rosenthal
Rene Romero
Alexander G Miethke
Kathleen M Loomes
Rohit Kohli
Saul J Karpen
Binita M Kamath
M Kyle Jensen
Jorge A Bezerra
Lee M Bass
John C Magee
ChiLDReN

Language

English

Publication Date

1-1-2025

Journal

Gastro Hep Advances

DOI

10.1016/j.gastha.2025.100788

PMID

41142527

PMCID

PMC12547242

PubMedCentral® Posted Date

9-2-2025

PubMedCentral® Full Text Version

Post-print

Abstract

Background and aims: A multicenter prospective longitudinal study of vibration-controlled transient elastography (VCTE) in school-age children with biliary atresia (BA), alpha-1 antitrypsin deficiency (a1-AT) and Alagille syndrome (ALGS) was undertaken to test the hypothesis that there would be measurable disease progression over 2 years.

Methods: Vibration-controlled transient elastography was performed annually for 2 years in children with BA, a1-AT and ALGS.

Results: Valid liver stiffness measurement (LSM) was determined at baseline/second follow-up in 254/180 (71%), 104/58 (56%) and 100/61 (61%) participants (mean elapsed time 2.27 years) with BA, a1-AT and ALGS, respectively. Modeling did not reveal a relationship between LSM and time since baseline: BA 1.2% (-1.6, 4.2%), a1-AT 0.1% (-3.8, 4.2%), and ALGS 3.6% (-2.9, 10.5%) LSM (% change/year; mean [95% confidence interval]). Similarly, mean LSM did not change significantly from baseline to visit 2 (BA 13.6 + 11.0 vs 15.1 + 12.8; a1-AT 7.8 + 5.1 vs 8.5 + 7.6; ALGS 10.6 + 9.4 vs 12.2 + 12.1 kPa, mean + standard deviation). Albumin and total bilirubin levels did not change in these participants. Platelet counts dropped at rates that were similar to a national representative sample, the National Health and Nutrition Examination Survey (ie, 5000 to 7000/μL per year).

Conclusion: Surprisingly, longitudinal measurement of LSM and laboratory parameters of liver disease severity over 2 years in school-age children with compensated BA, a1-AT, and ALGS did not reveal significant change, consistent with slow progression of cholestatic liver disease in this age group. These findings have implications for both clinical care and interventional trials in this patient population.

Keywords

Biliary Atresia, Cirrhosis, Fibrosis, Alagille Syndrome, Alpha-1 Antitrypsin Deficiency

Published Open-Access

yes

Recommended Citation

Shneider, Benjamin L; Goodrich, Nathan P; Ye, Wen; et al., "Prospective Multicenter Longitudinal Measurement of Liver Stiffness in School-Age Children With Cholestatic Liver Disease" (2025). Faculty and Staff Publications. 5805.
https://digitalcommons.library.tmc.edu/baylor_docs/5805