{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,1,10]],"date-time":"2026-01-10T01:16:17Z","timestamp":1768007777327,"version":"3.49.0"},"reference-count":24,"publisher":"Oxford University Press (OUP)","issue":"2","license":[{"start":{"date-parts":[[2020,11,9]],"date-time":"2020-11-09T00:00:00Z","timestamp":1604880000000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"funder":[{"DOI":"10.13039\/100004319","name":"Pfizer","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100004319","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["European J of Heart Fail"],"published-print":{"date-parts":[[2021,2]]},"abstract":"<jats:title>Abstract<\/jats:title>\n                  <jats:sec>\n                    <jats:title>Aims<\/jats:title>\n                    <jats:p>Hereditary (ATTRv) and wild\u2010type (ATTRwt) transthyretin amyloidosis are severe and fatal systemic diseases, characterised by amyloid fibrillar accumulation principally in the heart or peripheral nerves (or both). Since 2012, tafamidis has been used in France to treat patients with ATTRv with neuropathy (alone or combined with cardiomyopathy). Recently, the Phase III ATTR\u2010ACT trial showed that tafamidis decreased the relative risk of mortality in ATTR amyloidosis with cardiomyopathy. The aims of this study were to assess the clinical characteristics of ATTR amyloidosis in a real\u2010life population in comparison to the population included in the ATTR\u2010ACT trial and to assess the impact of tafamidis treatment on major cardiovascular outcome (MCO)\u2010free survival time without cardiac decompensation, heart transplant, or death.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Methods and results<\/jats:title>\n                    <jats:p>From June 2008 to November 2018, 648 patients with ATTR amyloidosis (423 ATTRwt and 225 ATTRv) consecutively referred to the French Referral Center for cardiac amyloidosis were included. A total of 467 (72%) patients matched the inclusion criteria of the ATTR\u2010ACT trial. For the 631 patients with cardiomyopathy, tafamidis treatment was<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:label\/>\n                    <jats:p>\n                      associated with a longer median MCO\u2010free survival time (\n                      <jats:italic>n<\/jats:italic>\n                      \u00a0=\u00a098): 1565 (1010\u20132400) days vs. 771 (686\u2013895) days without treatment (log\u2010rank\n                      <jats:italic>P<\/jats:italic>\n                      \u2009&lt;\u20090.001). This association was confirmed after considering confounding factors (age at inclusion, N\u2010terminal pro\u2010B\u2010type natriuretic peptide and amyloidosis type) with a propensity score (hazard ratio 0.546;\n                      <jats:italic>P<\/jats:italic>\n                      \u00a0=\u00a00.0132).\n                    <\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Conclusion<\/jats:title>\n                    <jats:p>In a large cohort of ATTRwt and ATTRv patients, representative of the inclusion criteria of the ATTR\u2010ACT trial, the present results show an association between tafamidis treatment and a lower occurrence of cardiovascular outcomes in a real\u2010life population.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1002\/ejhf.2028","type":"journal-article","created":{"date-parts":[[2020,10,23]],"date-time":"2020-10-23T10:33:19Z","timestamp":1603449199000},"page":"264-274","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":45,"title":["Natural history and impact of treatment with tafamidis on major cardiovascular outcome\u2010free survival time in a cohort of patients with transthyretin amyloidosis"],"prefix":"10.1093","volume":"23","author":[{"given":"M\u00e9lanie","family":"B\u00e9zard","sequence":"first","affiliation":[{"name":"AP\u2010HP (Assistance Publique\u2010H\u00f4pitaux de Paris), Cardiology Department, DHU\u2010ATVB Henri Mondor University Hospital  Cr\u00e9teil France"},{"name":"AP\u2010HP (Assistance Publique\u2010H\u00f4pitaux de Paris), French Referral Centre for Cardiac Amyloidosis, Cardiogen Network Henri Mondor University Hospital  Cr\u00e9teil France"},{"name":"AP\u2010HP (Assistance 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de la Haute Autorit\u00e9 de Sant\u00e9. Recommandation relative a la prise en charge a titre derogatoire du tafamidis dans le cadre d'une recommandation temporaire d'utilisation. HAS;2018.https:\/\/www.has\u2010sante.fr\/upload\/docs\/application\/pdf\/2019\u201001\/cteval421_reco_rtu_annexe_vyndaqel_cd_2018_12_19_v1.pdf(26 October 2020)."},{"key":"e_1_2_7_14_1","doi-asserted-by":"publisher","DOI":"10.1080\/13506129.2017.1330744"},{"key":"e_1_2_7_15_1","doi-asserted-by":"publisher","DOI":"10.1016\/0197-2456(96)00075-X"},{"key":"e_1_2_7_16_1","unstructured":"ParsonsLS.Performing a 1:N case\u2010control match on propensity score. SUGI 29 Proceedings: SAS Users Group International Conference. Cary NC: SAS Institute Inc.; 2004. 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