TY - JOUR
T1 - A longitudinal assessment of non-invasive biomarkers to diagnose and predict cystic fibrosis-associated liver disease
AU - Karnsakul, Wikrom
AU - Wasuwanich, Paul
AU - Ingviya, Thammasin
AU - Vasilescu, Alexandra
AU - Carson, Kathryn A.
AU - Mogayzel, Peter J.
AU - Schwarz, Kathleen B.
N1 - Funding Information:
We would like to thank Saachi Nangia for her help with the early data collection during her time at Johns Hopkins University. K. A. Carson's work on the project was funded by the Johns Hopkins Institute for Clinical and Translational Research, under grant number UL1 TR001079 from the National Center for Advancing Translational Sciences, a component of the National Institutes of Health ( NIH ) and the NIH Roadmap for Medical Research.
Funding Information:
K. A. Carson's work on the project was funded by the Johns Hopkins Institute for Clinical and Translational Research, under grant number UL1 TR001079 from the National Center for Advancing Translational Sciences, a component of the National Institutes of Health (NIH) and the NIH Roadmap for Medical Research.We would like to thank Saachi Nangia for her help with the early data collection during her time at Johns Hopkins University. K. A. Carson's work on the project was funded by the Johns Hopkins Institute for Clinical and Translational Research, under grant number UL1 TR001079 from the National Center for Advancing Translational Sciences, a component of the National Institutes of Health (NIH) and the NIH Roadmap for Medical Research.
Publisher Copyright:
© 2020 European Cystic Fibrosis Society
PY - 2020/7
Y1 - 2020/7
N2 - Background & Aims: A practical, inexpensive, and non-invasive biomarker of liver fibrosis is needed as a reliable screening test for cystic fibrosis-associated liver disease (CFLD). Studies have shown the utility of AST to Platelet Ratio Index (APRI), fibrosis index based on 4 factors (FIB-4), and gamma-glutamyl transferase (GGT) as good biomarkers for identifying CFLD. The goal of the study was to evaluate the effectiveness of APRI, FIB-4, AST/ALT ratio, platelet count, GGT, and GGT platelet ratio (GPR) in predicting CFLD development. Methods: Data was collected from CF Foundation Patient Registry for patients aged 3–21 years at Johns Hopkins from January 1, 2002 to December 31, 2014. Collected data included demographic characteristics, presence of splenomegaly, hepatomegaly, ascites, and variceal bleeding, AST, ALT, GGT, platelet count, and FEV1. The sensitivity and specificity of each biomarker were analyzed and reported by the area under receiver operating characteristic (AUROC) curve. Results: By the end of the study, 144 “healthy” CF, 12 CFLD, 19 CF-associated pulmonary disease (CFPD), and 4 CFLD with CFPD cases were identified. APRI scores were higher in CFLD, 0.85 versus 0.28 in “healthy” CF and 0.23 in CFPD groups (p<0.001). GPR had the highest AUROC curve at 0.91. Conclusions: GPR, GGT, APRI score, and platelet count were potentially useful biomarkers while FIB-4 did not predict CFLD development. Cost-effectiveness studies are needed to analyze the utility of these biomarkers in clinical practice.
AB - Background & Aims: A practical, inexpensive, and non-invasive biomarker of liver fibrosis is needed as a reliable screening test for cystic fibrosis-associated liver disease (CFLD). Studies have shown the utility of AST to Platelet Ratio Index (APRI), fibrosis index based on 4 factors (FIB-4), and gamma-glutamyl transferase (GGT) as good biomarkers for identifying CFLD. The goal of the study was to evaluate the effectiveness of APRI, FIB-4, AST/ALT ratio, platelet count, GGT, and GGT platelet ratio (GPR) in predicting CFLD development. Methods: Data was collected from CF Foundation Patient Registry for patients aged 3–21 years at Johns Hopkins from January 1, 2002 to December 31, 2014. Collected data included demographic characteristics, presence of splenomegaly, hepatomegaly, ascites, and variceal bleeding, AST, ALT, GGT, platelet count, and FEV1. The sensitivity and specificity of each biomarker were analyzed and reported by the area under receiver operating characteristic (AUROC) curve. Results: By the end of the study, 144 “healthy” CF, 12 CFLD, 19 CF-associated pulmonary disease (CFPD), and 4 CFLD with CFPD cases were identified. APRI scores were higher in CFLD, 0.85 versus 0.28 in “healthy” CF and 0.23 in CFPD groups (p<0.001). GPR had the highest AUROC curve at 0.91. Conclusions: GPR, GGT, APRI score, and platelet count were potentially useful biomarkers while FIB-4 did not predict CFLD development. Cost-effectiveness studies are needed to analyze the utility of these biomarkers in clinical practice.
KW - Blood chemical analysis
KW - Blood platelets
KW - Liver cirrhosis
KW - Respiratory tract infections
KW - Transferases
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U2 - 10.1016/j.jcf.2020.05.002
DO - 10.1016/j.jcf.2020.05.002
M3 - Article
C2 - 32482593
AN - SCOPUS:85085615194
SN - 1569-1993
VL - 19
SP - 546
EP - 552
JO - Journal of Cystic Fibrosis
JF - Journal of Cystic Fibrosis
IS - 4
ER -