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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\u2009=\u20090.7522), and it was upregulated upon ERT (<jats:italic>P<\/jats:italic>\u2009&lt;\u20090.001). On multivariable analysis, miR-184 was independently and inversely associated with a higher risk of cardiac damage (odds ratio\u2009=\u20090.86; 95% confidence interval [CI]\u2009=\u20090.76\u20130.98; <jats:italic>P<\/jats:italic>\u2009=\u20090.026). 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\u2009=\u20090.917, <jats:italic>P<\/jats:italic>\u2009&lt;\u20090.001; integrated discrimination improvement [IDI]\u2009=\u20090.105, <jats:italic>P<\/jats:italic>\u2009=\u20090.017; relative IDI\u2009=\u20090.221, 95% CI\u2009=\u20090.002\u20130.356). Thus, miR-184 is a circulating biomarker of AFD that changes after ERT. Assessment of its level in plasma could be clinically valuable in improving the prediction of cardiac damage in AFD patients.<\/jats:p>","DOI":"10.1038\/s41419-021-04438-5","type":"journal-article","created":{"date-parts":[[2021,12,11]],"date-time":"2021-12-11T13:02:43Z","timestamp":1639227763000},"update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":15,"title":["Circulating miR-184 is a potential predictive biomarker of cardiac damage in Anderson\u2013Fabry disease"],"prefix":"10.1038","volume":"12","author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-2689-4340","authenticated-orcid":false,"given":"Irene","family":"Salamon","sequence":"first","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Elena","family":"Biagini","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-3636-3391","authenticated-orcid":false,"given":"Paolo","family":"Kunderfranco","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Roberta","family":"Roncarati","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-1595-6887","authenticated-orcid":false,"given":"Manuela","family":"Ferracin","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Nevio","family":"Taglieri","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-4245-912X","authenticated-orcid":false,"given":"Elena","family":"Nardi","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-2737-4665","authenticated-orcid":false,"given":"Noemi","family":"Laprovitera","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Luciana","family":"Tomasi","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Marisa","family":"Santostefano","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Raffaello","family":"Ditaranto","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Giovanni","family":"Vitale","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Elena","family":"Cavarretta","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Antonio","family":"Pisani","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Eleonora","family":"Riccio","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Valeria","family":"Aiello","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Irene","family":"Capelli","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Gaetano","family":"La Manna","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Nazzareno","family":"Gali\u00e8","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-9774-6268","authenticated-orcid":false,"given":"Letizia","family":"Spinelli","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-0481-6843","authenticated-orcid":false,"given":"Gianluigi","family":"Condorelli","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"297","published-online":{"date-parts":[[2021,12,11]]},"reference":[{"key":"4438_CR1","doi-asserted-by":"publisher","first-page":"668","DOI":"10.1097\/GIM.0b013e3181f13b75","volume":"12","author":"O Lidove","year":"2010","unstructured":"Lidove O, West ML, Pintos-Morell G, Reisin R, Nicholls K, Figuera LE, et al. 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