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Circulating miR-184 is a potential predictive biomarker of cardiac damage in Anderson–Fabry disease by Irene Salamon; Elena Biagini; Paolo Kunderfranco; Roberta Roncarati; Manuela Ferracin; Nevio Taglieri; Elena Nardi; Noemi Laprovitera; Luciana Tomasi; Marisa Santostefano; Raffaello Ditaranto; Giovanni Vitale; Elena Cavarretta; Antonio Pisani; Eleonora Riccio; Valeria Aiello; Irene Capelli; Gaetano La Manna; Nazzareno Galiè; Letizia Spinelli; Gianluigi Condorelli is a Biochemistry, Genetics and Molecular Biology article available to read on EtoBox.

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## Abstract Enzyme replacement therapy (ERT) is a mainstay of treatment for Anderson–Fabry disease (AFD), a pathology with negative effects on the heart and kidneys. However, no reliable biomarkers are available to monitor its efficacy. Therefore, we tested a panel of four microRNAs linked with cardiac and renal damage in order to identify a novel biomarker associated with AFD and modulated by ERT. To this end, 60 patients with a definite diagnosis of AFD and on chronic ERT, and 29 age- and sex-matched healthy individuals, were enrolled by two Italian university hospitals. Only miR-184 met both conditions: its level discriminated untreated AFD patients from healthy individuals (c-statistic = 0.7522), and it was upregulated upon ERT (P < 0.001). On multivariable analysis, miR-184 was independently and inversely associated with a higher risk of cardiac damage (odds ratio = 0.86; 95% confidence interval [CI] = 0.76–0.98; P = 0.026). Adding miR-184 to a comprehensive clinical model improved the prediction of cardiac damage in terms of global model fit, calibration, discrimination, and classification accuracy (continuous net reclassification improvement = 0.917, P < 0.001; integrated di

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Author

Irene Salamon; Elena Biagini; Paolo Kunderfranco; Roberta Roncarati; Manuela Ferracin; Nevio Taglieri; Elena Nardi; Noemi Laprovitera; Luciana Tomasi; Marisa Santostefano; Raffaello Ditaranto; Giovanni Vitale; Elena Cavarretta; Antonio Pisani; Eleonora Riccio; Valeria Aiello; Irene Capelli; Gaetano La Manna; Nazzareno Galiè; Letizia Spinelli; Gianluigi Condorelli

Publisher

Springer Science and Business Media LLC

Published

2021

Language

EN

Field

Biochemistry, Genetics and Molecular Biology (Life Sciences)