Prospective Multicenter Longitudinal Measurement of Liver Stiffness in School-Age Children With Cholestatic Liver
Benjamin L Shneider1, Nathan P Goodrich2, Wen Ye3
1Texas Children's Hospital and Department of Pediatrics Baylor College of Medicine, Houston, Texas.
Insights
This study found that liver stiffness measurements and lab results did not significantly change over two years in children with biliary atresia (BA), alpha-1 antitrypsin deficiency (a1-AT), and Alagille syndrome (ALGS). These findings suggest slow disease progression in this pediatric cholestatic liver disease group.
Area of Science:
- Pediatric Gastroenterology
- Hepatology
- Medical Imaging
Background:
- Biliary atresia (BA), alpha-1 antitrypsin deficiency (a1-AT), and Alagille syndrome (ALGS) are significant causes of pediatric cholestatic liver disease.
- Longitudinal disease progression in these conditions in school-age children is not well-characterized.
Purpose of the Study:
- To assess disease progression over two years in school-age children diagnosed with BA, a1-AT, or ALGS using vibration-controlled transient elastography (VCTE).
- To evaluate changes in liver stiffness measurement (LSM) and key laboratory parameters indicative of liver disease severity.
Main Methods:
- A multicenter prospective longitudinal study involving annual VCTE measurements over two years.
- Participants included school-age children with BA, a1-AT, and ALGS.
- Liver stiffness measurement (LSM) and laboratory tests (albumin, bilirubin, platelet count) were analyzed.
Main Results:
- Valid LSMs were obtained from a significant proportion of participants at baseline and follow-up.
- No significant change in mean LSM was observed over the two-year study period for any of the conditions.
- Laboratory parameters, including albumin and total bilirubin, remained stable, while platelet counts decreased similarly to a national sample.
Conclusions:
- Longitudinal VCTE and laboratory assessments revealed minimal disease progression over two years in compensated pediatric BA, a1-AT, and ALGS.
- These findings suggest a slow progression rate for cholestatic liver diseases in this age group.
- The results have implications for clinical management and the design of interventional trials for pediatric liver disease.
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.
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