{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,28]],"date-time":"2026-10-28T03:45:00Z","timestamp":1793159100715,"version":"4.5.0"},"reference-count":28,"publisher":"MDPI AG","issue":"13","license":[{"start":{"date-parts":[[2024,6,26]],"date-time":"2024-06-26T00:00:00Z","timestamp":1719360000000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["JCM"],"abstract":"<jats:p>Background: This study aimed to evaluate the effect of treatment with tafamidis on clinical, laboratory, functional, and structural cardiovascular imaging parameters at the 12-month follow-up timepoint in patients with wild-type transthyretin amyloid cardiomyopathy (ATTRwt-CM) and to assess the response to treatment in terms of disease progression. Methods: Patients with ATTRwt-CM undergoing treatment with tafamidis for &gt;12 months were included. The patients underwent a comprehensive evaluation (including echocardiography, cardiac magnetic resonance imaging, six-minute walking test, assessment of quality of life, and laboratory tests) at baseline and the 12-month follow-up timepoint. Disease progression was assessed using a set of tools proposed by an international panel of experts, evaluating three main domains (clinical, biochemical, and structural). Results: The study cohort consisted of 25 patients (mean age of 75.9 \u00b1 6.1 years, with 92% males). At the 12-month follow-up timepoint, an improvement in quality of life calculated with the KCCQ overall score (64 \u00b1 20 vs. 75 \u00b1 20, p = 0.002) and a reduction in pulmonary artery pressure (34 \u00b1 10 mmHg vs. 30 \u00b1 5 mmHg, p-value = 0.008) and in native T1 time were observed (1162 \u00b1 66 ms vs. 1116 \u00b1 52 ms, p-value = 0.001). Clinical, biochemical, and structural disease progression was observed in 6 (24%), 13 (52%), and 7 (28%) patients, respectively. Overall disease progression was observed in two patients (8%). Conclusions: This study described the impact of tafamidis treatment on clinical, laboratory, and functional parameters. Disease progression, assessed using a multiparametric tool recommended by a recent position paper of experts, was observed in a minority of patients.<\/jats:p>","DOI":"10.3390\/jcm13133730","type":"journal-article","created":{"date-parts":[[2024,6,26]],"date-time":"2024-06-26T09:29:33Z","timestamp":1719394173000},"page":"3730","update-policy":"https:\/\/doi.org\/10.3390\/mdpi_crossmark_policy","source":"Crossref","is-referenced-by-count":5,"title":["Impact of Tafamidis on Delaying Clinical, Functional, and Structural Cardiac Changes in Patients with Wild-Type Transthyretin Amyloid Cardiomyopathy"],"prefix":"10.3390","volume":"13","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-5102-7460","authenticated-orcid":false,"given":"Giuseppe","family":"Palmiero","sequence":"first","affiliation":[{"name":"Department of Translational Medical Sciences, Inherited and Rare Cardiovascular Diseases, University of Campania \u201cLuigi Vanvitelli\u201d, 80131 Naples, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-9304-988X","authenticated-orcid":false,"given":"Emanuele","family":"Monda","sequence":"additional","affiliation":[{"name":"Department of Translational Medical Sciences, Inherited and Rare Cardiovascular Diseases, University of Campania \u201cLuigi Vanvitelli\u201d, 80131 Naples, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Federica","family":"Verrillo","sequence":"additional","affiliation":[{"name":"Department of Translational Medical Sciences, Inherited and Rare Cardiovascular Diseases, University of Campania \u201cLuigi Vanvitelli\u201d, 80131 Naples, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Francesca","family":"Dongiglio","sequence":"additional","affiliation":[{"name":"Department of Translational Medical Sciences, Inherited and Rare Cardiovascular Diseases, University of Campania \u201cLuigi Vanvitelli\u201d, 80131 Naples, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Chiara","family":"Cirillo","sequence":"additional","affiliation":[{"name":"Department of Translational Medical Sciences, Inherited and Rare Cardiovascular Diseases, University of Campania \u201cLuigi Vanvitelli\u201d, 80131 Naples, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-3805-4170","authenticated-orcid":false,"given":"Martina","family":"Caiazza","sequence":"additional","affiliation":[{"name":"Department of Translational Medical Sciences, Inherited and Rare Cardiovascular Diseases, University of Campania \u201cLuigi Vanvitelli\u201d, 80131 Naples, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Marta","family":"Rubino","sequence":"additional","affiliation":[{"name":"Department of Translational Medical Sciences, Inherited and Rare Cardiovascular Diseases, University of Campania \u201cLuigi Vanvitelli\u201d, 80131 Naples, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Annapaola","family":"Cirillo","sequence":"additional","affiliation":[{"name":"Department of Translational Medical Sciences, Inherited and Rare Cardiovascular Diseases, University of Campania \u201cLuigi Vanvitelli\u201d, 80131 Naples, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Adelaide","family":"Fusco","sequence":"additional","affiliation":[{"name":"Department of Translational Medical Sciences, Inherited and Rare Cardiovascular Diseases, University of Campania \u201cLuigi Vanvitelli\u201d, 80131 Naples, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Gaetano","family":"Diana","sequence":"additional","affiliation":[{"name":"Department of Translational Medical Sciences, Inherited and Rare Cardiovascular Diseases, University of Campania \u201cLuigi Vanvitelli\u201d, 80131 Naples, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-6919-899X","authenticated-orcid":false,"given":"Giovanni","family":"Ciccarelli","sequence":"additional","affiliation":[{"name":"Vanvitelli Cardiology Unit, Department of Translational Medical Sciences, Monaldi Hospital, 80131 Naples, Italy"},{"name":"Sbarro Institute for Cancer Research and Molecular Medicine, Center of Biotechnology, College of Science and Technology, Temple University, Philadelphia, PA 19122, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Santo","family":"Dellegrottaglie","sequence":"additional","affiliation":[{"name":"Advanced Cardiovascular Imaging Unit, Ospedale Medico-Chirurgico Accreditato Villa dei Fiori, 80131 Naples, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-5018-830X","authenticated-orcid":false,"given":"Paolo","family":"Calabr\u00f2","sequence":"additional","affiliation":[{"name":"Department of Translational Medical Sciences, Inherited and Rare Cardiovascular Diseases, University of Campania \u201cLuigi Vanvitelli\u201d, 80131 Naples, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-3590-6983","authenticated-orcid":false,"given":"Paolo","family":"Golino","sequence":"additional","affiliation":[{"name":"Vanvitelli Cardiology Unit, Department of Translational Medical Sciences, Monaldi Hospital, 80131 Naples, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-8291-9517","authenticated-orcid":false,"given":"Giuseppe","family":"Limongelli","sequence":"additional","affiliation":[{"name":"Department of Translational Medical Sciences, Inherited and Rare Cardiovascular Diseases, University of Campania \u201cLuigi Vanvitelli\u201d, 80131 Naples, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"1968","published-online":{"date-parts":[[2024,6,26]]},"reference":[{"key":"ref_1","doi-asserted-by":"crossref","first-page":"2872","DOI":"10.1016\/j.jacc.2019.04.003","article-title":"Transthyretin Amyloid Cardiomyopathy","volume":"73","author":"Ruberg","year":"2019","journal-title":"J. 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