{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,22]],"date-time":"2026-10-22T19:09:52Z","timestamp":1792696192733,"version":"4.5.0"},"reference-count":33,"publisher":"Ovid Technologies (Wolters Kluwer Health)","issue":"19","content-domain":{"domain":["www.ahajournals.org"],"crossmark-restriction":true},"short-container-title":["JAHA"],"published-print":{"date-parts":[[2020,10,6]]},"abstract":"<jats:sec xml:lang=\"en\">\n            <jats:title>Background<\/jats:title>\n            <jats:p xml:lang=\"en\">The emergence of specific therapies for transthyretin cardiac amyloidosis (CA) warrants the need for a systematic review of the literature.<\/jats:p>\n          <\/jats:sec>\n          <jats:sec xml:lang=\"en\">\n            <jats:title>Methods and Results<\/jats:title>\n            <jats:p xml:lang=\"en\">A systematic review of the literature was conducted according to Preferred Reporting Items for Systematic Reviews and Meta\u2010Analyses (PRISMA) guidelines. A systematic search was performed on MEDLINE, PubMed, and Embase databases on November 29, 2019. Studies were selected based on the following predefined eligibility criteria: English\u2010language randomized controlled trials (RCTs), non\u2010RCTs, or observational studies, which included adult patients with variant\/wild\u2010type transthyretin\u2010CA, assessed specific therapies for transthyretin\u2010CA, and reported cardiovascular outcomes. Relevant data were extracted to a predefined template. Quality assessment was based on National Institute for Health and Care Excellence recommendations (RCTs) or a checklist by Downs and Black (non\u2010RCTs). From 1203 records, 24 publications were selected, describing 4 RCTs (6 publications) and 16 non\u2010RCTs (18 publications). Tafamidis was shown to significantly improve all\u2010cause mortality and cardiovascular hospitalizations and reduce worsening in 6\u2010minute walk test, Kansas City Cardiomyopathy Questionnaire\u2014Overall Summary score, and NT\u2010proBNP (N\u2010terminal pro\u2010B\u2010type natriuretic peptide) in variant\/wild\u2010type transthyretin\u2010CA. Patisiran showed promising results in a subgroup analysis of patients with variant transthyretin\u2010CA, which have to be confirmed in RCTs. Inotersen showed conflicting results on cardiac imaging parameters. The one study on AG10 had only a 1\u2010month duration and cardiovascular end points were exploratory and limited to cardiac biomarkers. Limited evidence from noncomparative single\u2010arm small non\u2010RCTs existed for diflunisal, epigallocatechin\u20103\u2010gallate (green tea extract), and doxycycline+tauroursodeoxycholic acid\/ursodeoxycholic acid.<\/jats:p>\n          <\/jats:sec>\n          <jats:sec xml:lang=\"en\">\n            <jats:title>Conclusions<\/jats:title>\n            <jats:p xml:lang=\"en\">This systematic review of the literature supports the use of tafamidis in wild\u2010type and variant transthyretin\u2010CA. Novel therapeutic targets including transthyretin gene silencers are currently under investigation.<\/jats:p>\n          <\/jats:sec>","DOI":"10.1161\/jaha.120.016614","type":"journal-article","created":{"date-parts":[[2020,9,24]],"date-time":"2020-09-24T09:06:28Z","timestamp":1600938388000},"update-policy":"https:\/\/doi.org\/10.1161\/crossmarkpolicy","source":"Crossref","is-referenced-by-count":18,"title":["Specific Therapy for Transthyretin Cardiac Amyloidosis: A Systematic Literature Review and Evidence\u2010Based Recommendations"],"prefix":"10.1161","volume":"9","author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-0275-2807","authenticated-orcid":false,"given":"Nuno","family":"Marques","sequence":"first","affiliation":[{"name":"Algarve Biomedical Center Algarve Portugal"},{"name":"Biomedical and Medical Department Algarve University Algarve Portugal"},{"name":"Cardiology Department Centro Hospitalar Universit\u00e1rio do Algarve Algarve Portugal"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-6784-3403","authenticated-orcid":false,"given":"Olga","family":"Azevedo","sequence":"additional","affiliation":[{"name":"Cardiology Department Hospital Senhora da Oliveira Guimar\u00e3es Portugal"},{"name":"Life and Health Sciences Research Institute (ICVS) School of Medicine University of Minho Braga Portugal"},{"name":"ICVS\/3Bs PT Government Associate Laboratory Braga\/Guimar\u00e3es Portugal"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ana Rita","family":"Almeida","sequence":"additional","affiliation":[{"name":"Cardiology Department Hospital Garcia de Orta Almada Portugal"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Dina","family":"Bento","sequence":"additional","affiliation":[{"name":"Algarve Biomedical Center Algarve Portugal"},{"name":"Biomedical and Medical Department Algarve University Algarve Portugal"},{"name":"Cardiology Department Centro Hospitalar Universit\u00e1rio do Algarve Algarve Portugal"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"In\u00eas","family":"Cruz","sequence":"additional","affiliation":[{"name":"Cardiology Department Hospital Garcia de Orta Almada Portugal"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Emanuel","family":"Correia","sequence":"additional","affiliation":[{"name":"Cardiology Department Centro Hospitalar Tondela Viseu Viseu Portugal"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Carolina","family":"Louren\u00e7o","sequence":"additional","affiliation":[{"name":"Cardiology Department Centro Hospitalar Universit\u00e1rio de Coimbra Coimbra Portugal"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Lu\u00eds Rocha","family":"Lopes","sequence":"additional","affiliation":[{"name":"St. Bartholomew\u2019s Hospital\u2010Barts Heart Centre Barts Health NHS Trust London United Kingdom"},{"name":"Centre for Heart Muscle Disease Institute of Cardiovascular Science University College of London United Kingdom"},{"name":"Centro Cardiovacular Universidade Lisboa Lisboa Portugal"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"276","reference":[{"key":"e_1_3_1_2_2","doi-asserted-by":"publisher","DOI":"10.1074\/jbc.M115.659912"},{"key":"e_1_3_1_3_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.jacc.2016.03.596"},{"key":"e_1_3_1_4_2","doi-asserted-by":"crossref","DOI":"10.1161\/CIRCHEARTFAILURE.119.006075","article-title":"Expert consensus recommendations for the suspicion and diagnosis of transthyretin cardiac amyloidosis","volume":"9","author":"Maurer MS","year":"2019","unstructured":"Maurer MS, Bokhari S, Damy T, Dorbala S, Drachman BM, Fontana M, Grogan M, Kristen AV, Lousada I, Nativi\u2010Nicolau J, et al. 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