Published Research

Total Records: 118; Page 1 of 6
TitleMediaAuthorsPublicationPublication Year Descending order
Neonatal cholestasis in children with Alpha-1-AT deficiency is a risk for earlier severe liver disease with male predominance
Teckman JH, Rosenthal P, Ignacio RV, Spino C, Bass LM, Horslen SP, Wang K, Magee JC, Karpen SJ, Asai A, Molleston JP, Squires RH, Kamath BM, Guthery SL, Loomes KM, Shneider BL, Sokol RJ, Childhood Liver Disease Research and Education Net
Hepatology Communications (2023)
Teckman JH, Rosenthal P, Ignacio RV, Spino C, Bass LM, Horslen SP, Wang K, Magee JC, Karpen SJ, Asai A, Molleston JP, Squires RH, Kamath BM, Guthery SL, Loomes KM, Shneider BL, Sokol RJ, Childhood Liver Disease Research and Education Net Hepatology Communications2023
Cytomegalovirus in biliary atresia is associated with increased pretransplant death, but not decreased native liver survival
Kemme S, Canniff JD, Feldman AG, Garth KM, Li S, Pan Z, Sokol RJ, Weinberg A, Mack CL
Hepatology Communications (2023)
Kemme S, Canniff JD, Feldman AG, Garth KM, Li S, Pan Z, Sokol RJ, Weinberg A, Mack CLHepatology Communications2023
Sarcopenia is associated with osteopenia and impaired quality of life in children with genetic intrahepatic cholestatic liver disease
Boster JM, Goodrich NP, Spino C, Loomes KM, Alonso EM, Kamath BM, Sokol RJ, Karpen SJ, Miethke A, Shneider BL, Molleston JP, Kohli R, Horslen SP, Rosenthal P, Valentino PL, Teckman JH, Hangartner TN, Sundaram SS, Childhood Liver Disease Research and Education Net
Hepatology Communications (2023)
Boster JM, Goodrich NP, Spino C, Loomes KM, Alonso EM, Kamath BM, Sokol RJ, Karpen SJ, Miethke A, Shneider BL, Molleston JP, Kohli R, Horslen SP, Rosenthal P, Valentino PL, Teckman JH, Hangartner TN, Sundaram SS, Childhood Liver Disease Research and Education Net Hepatology Communications2023
Genotype-phenotype relationships of truncating mutations, p.E297G and p.D482G in bile salt export pump deficiency
Felzen A, van Wessel DBE, Gonzales E, Thompson RJ, Jamkowska I, Sheider BL, Sokal E, Grammatikopoulos T, Kadaristiana A, Jacquemin E, Spraul A, Lipinski P, Czubkowski P, Rock N, Shagrani M, Broering D, Nicastro E, Kelly D, Nebbia G, Arnell H, Fischler B, Hulscher JBF, Serranti D, Arikan C, Polat E, Debray D, Lacaille F, Goncalves C, Hierro L, Munoz Bartolo G, Mozer-Glassberg Y, Azaz A, Brecelj J, Dezsofi A, Calvo PL, Grabhorn E, Hartleif S, van der Woerd WJ, Kamath BM, Wang JS, Li L, Durmaz O, Kerkar N, Jorgensen MH, Fischer R, Jimenez-Rivera C, Alam S, Canazi M, Laverdure N, Targa Ferreira C, Ordonez Guerrero F, Wang H, Sency V, Mo Kim K, Chen HL, de Carvalho E, Fabre A, Bernabeu JQ, Zellos A, Alonso EM, Sokol RJ, Suchy FJ, Loomes KM, McKiernan P, Rosenthal P, Turmelle YP, Horslen SP, Schwarz KB, Bezerra JA, Wang KS, Hansen BE, Verkade HJ, NAtural course and Prognosis of PFIC and Effect of
JHEP Reports (2023)
Felzen A, van Wessel DBE, Gonzales E, Thompson RJ, Jamkowska I, Sheider BL, Sokal E, Grammatikopoulos T, Kadaristiana A, Jacquemin E, Spraul A, Lipinski P, Czubkowski P, Rock N, Shagrani M, Broering D, Nicastro E, Kelly D, Nebbia G, Arnell H, Fischler B, Hulscher JBF, Serranti D, Arikan C, Polat E, Debray D, Lacaille F, Goncalves C, Hierro L, Munoz Bartolo G, Mozer-Glassberg Y, Azaz A, Brecelj J, Dezsofi A, Calvo PL, Grabhorn E, Hartleif S, van der Woerd WJ, Kamath BM, Wang JS, Li L, Durmaz O, Kerkar N, Jorgensen MH, Fischer R, Jimenez-Rivera C, Alam S, Canazi M, Laverdure N, Targa Ferreira C, Ordonez Guerrero F, Wang H, Sency V, Mo Kim K, Chen HL, de Carvalho E, Fabre A, Bernabeu JQ, Zellos A, Alonso EM, Sokol RJ, Suchy FJ, Loomes KM, McKiernan P, Rosenthal P, Turmelle YP, Horslen SP, Schwarz KB, Bezerra JA, Wang KS, Hansen BE, Verkade HJ, NAtural course and Prognosis of PFIC and Effect of JHEP Reports2023
TWEAK/FN14 promotes profibrogenic pathway activation in Prominin-1-expressing hepatic progenitor cells in biliary atresia
Short C, Zhong A, Xu J, Mahdi E, Glazier A, Malkoff N, Noriega N, Yeo T, Asahina K, Wang KS
Hepatology (2023)
Short C, Zhong A, Xu J, Mahdi E, Glazier A, Malkoff N, Noriega N, Yeo T, Asahina K, Wang KSHepatology2023
Background and aims: Detailed investigation of the biological pathways leading to liver fibrosis and identification of liver fibrosis biomarkers may promote early interventions for children with cholestasis. Approach and results: A targeted panel of nine biomarkers (lysyl oxidase, tissue inhibitor matrix metalloproteinase (MMP) 1, connective tissue growth factor [CTGF], IL-8, endoglin, periostin, Mac-2-binding protein, MMP-3, and MMP-7) was studied in children with biliary atresia (BA; n = 187), alpha-1 antitrypsin deficiency (A1AT; n = 78), and Alagille syndrome (ALGS; n = 65) and correlated with liver stiffness (LSM) and liver labs.. Median age and LSM were 9 years and 9.5 kPa. There were positive correlations among LSM and endoglin ( p = 0.04) and IL-8 ( p < 0.001) and MMP-7 ( p < 0.001) in participants with BA. The addition of IL-8 and MMP-7 to clinical and lab measurements improved prediction of LSM in children with BA. Interestingly, among A1AT, CTGF and LSM were negatively correlated ( p = 0.004) and adding CTGF improved the LSM prediction model. Biomarkers did not correlate with LSM in ALGS. Conclusions: Endoglin, IL-8, and MMP-7 significantly correlate with increased LSM in children with BA, whereas CTGF inversely correlates with LSM in participants with A1AT; these biomarkers appear to enhance prediction of LSM beyond clinical tests. Future disease-specific investigations of change in these biomarkers over time and as predictors of clinical outcomes will be important.
Leung DH, Devaraj S, Goodrich NP, Chen X, Rajapakshe D, Ye W, Andreev VP, Minard CG, Guffey D, Molleston JP, Bass LM, Karpen SJ, Kamath BM, Wang KS, Sundaram SS, Rosenthal P, McKiernan P, Loomes KM, Jensen MK, Horslen SP, Bezerra JA, Magee JC, Merion RM, Sokol RJ, Shneider BL, Childhood Liver Disease Research and Education Net
Hepatology (2023)
Leung DH, Devaraj S, Goodrich NP, Chen X, Rajapakshe D, Ye W, Andreev VP, Minard CG, Guffey D, Molleston JP, Bass LM, Karpen SJ, Kamath BM, Wang KS, Sundaram SS, Rosenthal P, McKiernan P, Loomes KM, Jensen MK, Horslen SP, Bezerra JA, Magee JC, Merion RM, Sokol RJ, Shneider BL, Childhood Liver Disease Research and Education Net Hepatology2023
In biliary atresia, serum bilirubin is commonly used to predict outcomes after Kasai portoenterostomy (KP). Infants with persistently high levels invariably need liver transplant, but those achieving normalized levels have a less certain disease course. We hypothesized that serum bile acid levels could help predict outcomes in the latter group. To investigate this, we studied 137 participants with biliary atresia from the Childhood Liver Disease Research Network who had normalized bilirubin levels 6 months after KP and stored serum samples from the 6-month post-KP clinic visit. Bile acids were measured from the stored serum samples and used to divide participants into ≤40 μmol/L (n = 43) or >40 μmol/L (n = 94) groups. At 2 years of age, the ≤40 μmol/L compared with >40 μmol/L group had significantly lower total bilirubin, aspartate aminotransferase, alanine aminotransferase, gamma-glutamyltransferase, bile acids, and spleen size, as well as significantly higher albumin and platelet counts. Furthermore, during 734 person-years of follow-up, those in the ≤40 μmol/L group were significantly less likely to develop splenomegaly, ascites, gastrointestinal bleeding, or clinically evident portal hypertension. The ≤40 μmol/L group had a 10-year cumulative incidence of liver transplant/death of 8.5% (95% CI: 1.1%-26.1%), compared with 42.9% (95% CI: 28.6%-56.4%) for the >40 μmol/L group (p = 0.001). Our results suggest that serum bile acid levels may be a useful prognostic biomarker for infants achieving normalized bilirubin levels after KP.
Harpavat S, Hawthorne K, Setchell KD, Narvaez Rivas M, Henn L, Beil CA, Karpen SJ, Ng VL, Alonso EM, Bezerra JA, Guthery SL, Horslen SP, Loomes KM, McKiernan P, Magee JC, Merion RM, Molleston JP, Rosenthal P, Thompson RJ, Wang KS, Sokol RJ, Shneider BL
Hepatology (2022)
Harpavat S, Hawthorne K, Setchell KD, Narvaez Rivas M, Henn L, Beil CA, Karpen SJ, Ng VL, Alonso EM, Bezerra JA, Guthery SL, Horslen SP, Loomes KM, McKiernan P, Magee JC, Merion RM, Molleston JP, Rosenthal P, Thompson RJ, Wang KS, Sokol RJ, Shneider BLHepatology2022
Impact of long-term administration of maralixibat on children with cholestasis secondary to Alagille syndrome
Shneider BL, Spino C, Kamath BM, Magee JC, Ignacio RV, Huang S, Horslen S, Molleston JP, Miethke A, Kohli R, Leung DH, Jensen MK, Loomes KM, Karpen SJ, Mack CL, Rosenthal P, Squires RH, Baker A, Rajwal S, Kelly D, Sokol RJ, Thompson RJ, ChiLDReN , UK IMAGO/IMAGINE Investigators
Hepatology Communications (2022)
Shneider BL, Spino C, Kamath BM, Magee JC, Ignacio RV, Huang S, Horslen S, Molleston JP, Miethke A, Kohli R, Leung DH, Jensen MK, Loomes KM, Karpen SJ, Mack CL, Rosenthal P, Squires RH, Baker A, Rajwal S, Kelly D, Sokol RJ, Thompson RJ, ChiLDReN , UK IMAGO/IMAGINE Investigators Hepatology Communications2022
Serum proteomics uncovers biomarkers of clinical portal hypertension in children with biliary atresia
Osborn J, Mourya R, Thanekar U, Su W, Fei L, Shivakumar P, Bezerra JA
Hepatology Communications (2022)
Osborn J, Mourya R, Thanekar U, Su W, Fei L, Shivakumar P, Bezerra JAHepatology Communications2022
Use of funded multicenter prospective longitudinal databases to inform clinical trials in rare diseases—Examination of cholestatic liver disease in Alagille syndrome
Shneider BL, Kamath BM, Magee JC, Goodrich NP, Loomes KM, Ye W, Spino C, Alonso EM, Molleston JP, Bezerra JA, Wang KS, Karpen SJ, Horslen S, Guthery SL, Rosenthal P, Squires RH, Childhood Liver Disease Research and Education Net
Hepatology Communications (2022)
Shneider BL, Kamath BM, Magee JC, Goodrich NP, Loomes KM, Ye W, Spino C, Alonso EM, Molleston JP, Bezerra JA, Wang KS, Karpen SJ, Horslen S, Guthery SL, Rosenthal P, Squires RH, Childhood Liver Disease Research and Education Net Hepatology Communications2022
Risk of variceal hemorrhage and pretransplant mortality in children with biliary atresia
Bass LM, Ye W, Hawthorne K, Leung DH, Murray KF, Molleston JP, Romero R, Karpen SJ, Rosenthal P, Loomes KM, Wang KS, Squires RH, Miethke A, Ng VL, Horslen SP, Jensen MK, Sokol RJ, Magee JC, Shneider BL, Childhood Liver Disease Research and Education Net
Hepatology (2022)
Bass LM, Ye W, Hawthorne K, Leung DH, Murray KF, Molleston JP, Romero R, Karpen SJ, Rosenthal P, Loomes KM, Wang KS, Squires RH, Miethke A, Ng VL, Horslen SP, Jensen MK, Sokol RJ, Magee JC, Shneider BL, Childhood Liver Disease Research and Education Net Hepatology2022
To evaluate the neurodevelopmental status of children with inherited cholestatic liver diseases and features to predict impairment. Participants with Alagille syndrome (ALGS), progressive familial intrahepatic cholestasis (PFIC), and alpha 1 antitrypsin deficiency (A1AT) enrolled in a longitudinal, multicenter study and completed the Wechsler Preschool and Primary Scale of Intelligence-III or Intelligence Scale for Children-IV. Full Scale Intelligence Quotient (FSIQ) was analyzed continuously and categorically (>100, 85–99, 70–84, <70). Univariate linear regression was performed to study association between FSIQ and risk factors, stratified by disease. Two hundred and fifteen completed testing (ALGS n = 70, PFIC n = 43, A1AT n = 102); median age was 7.6 years (3.0–16.9). Mean FSIQ in ALGS was lower than A1AT (94 vs 101, P = 0.01). Frequency of FSIQ < 85 (>1 standard deviation [SD] below average) was highest in ALGS (29%) versus 18.6% in PFIC and 12.8% in A1AT, and was greater than expected in ALGS based on normal distribution (29% vs 15.9%, P = 0.003). ALGS scored significantly lower than test norms in almost all Wechsler composites; A1AT scored lower on Working Memory and Processing Speed; PFIC was not different from test norms. Total bilirubin, alkaline phosphatase, albumin, hemoglobin, and parental education were significantly associated with FSIQ. Patients with ALGS are at increased risk of lower FSIQ, whereas our data suggest A1AT and PFIC are not. A1AT and ALGS appear vulnerable to working memory and processing speed deficits suggestive of attention/executive function impairment. Malnutrition, liver disease severity, and sociodemographic factors appear related to FSIQ deficits, potentially identifying targets for early interventions.
Leung DH, Sorensen LG, Ye W, Hawthorne K, Ng VL, Loomes KM, Fredericks EM, Alonso EM, Heubi JE, Horslen SP, Karpen SJ, Molleston JP, Rosenthal P, Sokol RJ, Squires RH, Wang KS, Kamath BM, Magee JC
Journal of Pediatric Gastroenterology and Nutrition (2022)
Leung DH, Sorensen LG, Ye W, Hawthorne K, Ng VL, Loomes KM, Fredericks EM, Alonso EM, Heubi JE, Horslen SP, Karpen SJ, Molleston JP, Rosenthal P, Sokol RJ, Squires RH, Wang KS, Kamath BM, Magee JCJournal of Pediatric Gastroenterology and Nutrition2022
To determine the frequency and natural history of infantile idiopathic cholestasis (IC) in a large, prospective, multi-center cohort of infants. We studied 94 cholestatic infants enrolled up to 6 months of age in the NIDDK ChiLDReN (Childhood Liver Disease Research Network) "PROBE" protocol with a final diagnosis of IC; they were followed up to 30 months of age. Male sex (66/94; 70%), preterm birth (22/90 with data; 24% born at < 37 weeks' gestational age), and low birth weight (25/89; 28% born at <2500 g) were frequent, with no significant differences between outcomes. Clinical outcomes included death (n = 1), liver transplant (n = 1), biochemical resolution, partial resolution, and exited healthy. Biochemical resolution occurred at median of 9 months of age. GGT was < 100 U/L at baseline in 34/83 participants (41%). Frequency of IC and of death or liver transplant was less common in this cohort than in previously published cohorts, likely due to recent discovery and diagnosis of genetic etiologies of severe/persistent cholestasis that previously were labeled as idiopathic. Preterm birth and other factors associated with increased vulnerability in neonates are relatively frequent and may contribute to IC. Overall outcome in IC is excellent. Low/normal GGT was common, possibly indicating a role for variants in genes associated with low-GGT cholestasis. This warrants further study.
Hertel PM, Hawthorne K, Kim S, Finegold MJ, Shneider BL, Squires JE, Gupta NA, Bull LN, Murray KF, Kerkar N, Ng VL, Molleston JP, Bezerra JA, Loomes KM, Taylor SA, Schwarz KB, Turmelle YP, Rosenthal P, Magee JC, Sokol RJ, Childhood Liver Disease Research and Education Net
Journal of Pediatric Gastroenterology and Nutrition (2021)
Hertel PM, Hawthorne K, Kim S, Finegold MJ, Shneider BL, Squires JE, Gupta NA, Bull LN, Murray KF, Kerkar N, Ng VL, Molleston JP, Bezerra JA, Loomes KM, Taylor SA, Schwarz KB, Turmelle YP, Rosenthal P, Magee JC, Sokol RJ, Childhood Liver Disease Research and Education Net Journal of Pediatric Gastroenterology and Nutrition2021
High mobility group box 1 release by cholangiocytes governs biliary atresia pathogenesis and correlates with increases in afflicted infants
Mohanty SK, Donnelly B, Temple H, Ortiz-Perez A, Mowery S, Lobeck I, Dupree P, Poling HM, McNeal M, Mourya R, Jenkins T, Bansal R, Bezerra JA, Tiao G
Hepatology (2021)
Mohanty SK, Donnelly B, Temple H, Ortiz-Perez A, Mowery S, Lobeck I, Dupree P, Poling HM, McNeal M, Mourya R, Jenkins T, Bansal R, Bezerra JA, Tiao GHepatology2021
Impact of genotype, serum bile acids, and surgical biliary diversion on native liver survival in FIC1 deficiency
van Wessel DBE, Thompson RJ, Gonzales E, Jankowska I, Shneider BL, Sokal E, Grammatikopoulos T, Kadaristiana A, Jacquemin E, Spraul A, Lipinski P, Czubkowski P, Rock N, Shagrani M, Broering D, Algoufi T, Mazhar N, Nicastro E, Kelly D, Nebbia G, Arnell H, Fischler B, Hulscher JBF, Serranti D, Arikan C, Debray D, Lacaille F, Goncalves C, Hierro L, Munoz Bartolo G, Mozer-Glassberg Y, Azaz A, Brecelj J, Dezsofi A, Luigi Calvo P, Krebs-Schmitt D, Hartleif S, van der Woerd WL, Wang JS, Li LT, Durmaz O, Kerkar N, Hørby Jørgensen M, Fischer R, Jimenez-Rivera C, Alam S, Cananzi M, Laverdure N, Targa Ferreira C, Ordonez F, Wang H, Sency V, Mo Kim K, Chen HL, Carvalho E, Fabre A, Quintero Bernabeu J, Alonso EM, Sokol RJ, Suchy FJ, Loomes KM, McKiernan PJ, Rosenthal P, Turmelle Y, Rao GS, Horslen S, Kamath BM, Rogalidou M, Karnsakul W, Hansen BE, Verkade HJ, NAtural course and Prognosis of PFIC and Effect of
Hepatology (2021)
van Wessel DBE, Thompson RJ, Gonzales E, Jankowska I, Shneider BL, Sokal E, Grammatikopoulos T, Kadaristiana A, Jacquemin E, Spraul A, Lipinski P, Czubkowski P, Rock N, Shagrani M, Broering D, Algoufi T, Mazhar N, Nicastro E, Kelly D, Nebbia G, Arnell H, Fischler B, Hulscher JBF, Serranti D, Arikan C, Debray D, Lacaille F, Goncalves C, Hierro L, Munoz Bartolo G, Mozer-Glassberg Y, Azaz A, Brecelj J, Dezsofi A, Luigi Calvo P, Krebs-Schmitt D, Hartleif S, van der Woerd WL, Wang JS, Li LT, Durmaz O, Kerkar N, Hørby Jørgensen M, Fischer R, Jimenez-Rivera C, Alam S, Cananzi M, Laverdure N, Targa Ferreira C, Ordonez F, Wang H, Sency V, Mo Kim K, Chen HL, Carvalho E, Fabre A, Quintero Bernabeu J, Alonso EM, Sokol RJ, Suchy FJ, Loomes KM, McKiernan PJ, Rosenthal P, Turmelle Y, Rao GS, Horslen S, Kamath BM, Rogalidou M, Karnsakul W, Hansen BE, Verkade HJ, NAtural course and Prognosis of PFIC and Effect of Hepatology2021
Deleterious variants in ABCC12 are detected in idiopathic chronic cholestasis and cause intrahepatic bile duct loss in model organisms
Pham DH, Kudira R, Xu L, Valencia CA, Ellis JL, Shi T, Evason KJ, Osuji I, Matuschek N, Pfuhler L, Mullen M, Mohanty SK, Husami A, Bull LN, Zhang K, Wali S, Yin C, Miethke A
Gastroenterology (2021)
Pham DH, Kudira R, Xu L, Valencia CA, Ellis JL, Shi T, Evason KJ, Osuji I, Matuschek N, Pfuhler L, Mullen M, Mohanty SK, Husami A, Bull LN, Zhang K, Wali S, Yin C, Miethke AGastroenterology2021
Unique cholangiocyte-targeted IgM autoantibodies correlate with poor outcome in biliary atresia
Luo Y, Brigham D, Bednarek J, Torres R, Wang D, Ahmad S, Mack CL
Hepatology (2021)
Luo Y, Brigham D, Bednarek J, Torres R, Wang D, Ahmad S, Mack CLHepatology2021
Cholestasis due to USP53 deficiency
Bull LN, Ellmers R, Foskett P, Strautnieks S, Sambrotta M, Czubkowski P, Jankowska I, Wagner BE, Deheragoda M, Thompson RJ
Journal of Pediatric Gastroenterology and Nutrition (2021)
Bull LN, Ellmers R, Foskett P, Strautnieks S, Sambrotta M, Czubkowski P, Jankowska I, Wagner BE, Deheragoda M, Thompson RJJournal of Pediatric Gastroenterology and Nutrition2021
Analyses included participants with pathogenic biallelic mutations in adenosine triphosphate (ATP)-binding cassette subfamily B member 11 (ABCB11) (bile salt export pump; BSEP) or adenosine triphosphatase (ATPase) phospholipid transporting 8B1 (ATP8B1) (familial intrahepatic cholestasis; FIC1), or those with monoallelic or biallelic mutations in adenosine triphosphate (ATP)-binding cassette subfamily B member 4 (ABCB4) (multidrug resistance; MDR3), prospectively enrolled in the Longitudinal Study of Genetic Causes of Intrahepatic Cholestasis between November 2007 and December 2013. Summary statistics were calculated to describe baseline demographics, history, anthropometrics, laboratory values, and mutation data. Ninety-eight participants with FIC1, BSEP, or MDR3 deficiency were analyzed. Thirty-five had a surgical interruption of the enterohepatic circulation (sEHC), including 10 who underwent liver transplant (LT) after sEHC. Onset of symptoms occurred by age 2 years in most with FIC1 and BSEP deficiency, but was later and more variable for MDR3. Pruritus was nearly universal in FIC1 and BSEP deficiency. In participants with native liver, failure to thrive was common in FIC1 deficiency, high ALT was common in BSEP deficiency, and thrombocytopenia was common in MDR3 deficiency. sEHC was successful after more than 1 year in 7 of 19 participants with FIC1 and BSEP deficiency. History of LT was most common in BSEP deficiency. Of 102 mutations identified, 43 were not previously reported. In this cohort, BSEP deficiency appears to be correlated with a more severe disease course. Genotype-phenotype correlations in these diseases are not straightforward and will require the study of larger cohorts.
Hertel PM, Bull LN, Thompson RJ, Goodrich NP, Ye W, Magee JC, Squires RH, Bass LM, Heubi JE, Kim GE, Ranganathan S, Schwarz KB, Bozic MA, Horslen SP, Clifton MS, Turmelle YP, Suchy FJ, Superina RA, Wang KS, Loomes KM, Kamath BM, Sokol RJ, Shneider BL
Journal of Pediatric Gastroenterology and Nutrition (2021)
Hertel PM, Bull LN, Thompson RJ, Goodrich NP, Ye W, Magee JC, Squires RH, Bass LM, Heubi JE, Kim GE, Ranganathan S, Schwarz KB, Bozic MA, Horslen SP, Clifton MS, Turmelle YP, Suchy FJ, Superina RA, Wang KS, Loomes KM, Kamath BM, Sokol RJ, Shneider BLJournal of Pediatric Gastroenterology and Nutrition2021
Elastographic measurement of liver stiffness is of growing importance in the assessment of liver disease. Pediatric experiences with this technique are primarily single center and limited in scope. The Childhood Liver Disease Research Network provided a unique opportunity to assess elastography in a well-characterized multi-institutional cohort. Children with biliary atresia (BA), alpha-1 antitrypsin deficiency (A1ATD), or Alagille syndrome (ALGS) followed in a prospective longitudinal network study were eligible for enrollment in a prospective investigation of transient elastography (FibroScan). Studies were performed in participants who were nonfasted and nonsedated. Liver stiffness measurements (LSMs) were correlated with standard clinical and biochemical parameters of liver disease along with a research definition of clinically evident portal hypertension (CEPH) graded as absent, possible, or definite. Between November 2016 and August 2019, 550 participants with a mean age of 8.8 years were enrolled, 458 of whom had valid LSMs. Invalid scans were more common in participants <2 years old. There was a positive correlation between LSM and total bilirubin, international normalized ratio (INR), aspartate aminotransferase (AST), alanine aminotransferase (ALT), gamma-glutamyl transpeptidase (GGT), GGT to platelet ratio (GPR), pediatric end-stage liver disease score, AST to platelet ratio index, and spleen size, and a negative correlation with albumin and platelet count in BA, with similar correlations for A1ATD (except AST, ALT, and albumin) and ALGS (except for INR, GGT, GPR, and ALT). Possible or definite CEPH was more common in BA compared to ALGS and A1ATD. LSM was greater in definite versus absent CEPH in all three diseases. Disease-specific clinical and biochemical characteristics of the different CEPH grades were observed. It is feasible to obtain LSMs in children, especially over the age of 2 years. LSM correlates with liver parameters and portal hypertension, although disease-specific patterns exist.
Shneider BL, Goodrich NP, Ye W, Sawyers C, Molleston JP, Merion RM, Leung DH, Karpen SJ, Kamath BM, Cavallo LA, Wang K, Teckman JH, Squires JE, Sundaram SS, Rosenthal P, Romero R, Murray KF, Loomes KM, Jensen MK, Bezerra JA, Bass LM, Sokol RJ, Magee JC
Hepatology Communications (2020)
Shneider BL, Goodrich NP, Ye W, Sawyers C, Molleston JP, Merion RM, Leung DH, Karpen SJ, Kamath BM, Cavallo LA, Wang K, Teckman JH, Squires JE, Sundaram SS, Rosenthal P, Romero R, Murray KF, Loomes KM, Jensen MK, Bezerra JA, Bass LM, Sokol RJ, Magee JCHepatology Communications2020
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