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Specific therapy for transthyretin cardiac amyloidosis: a systematic literature review and evidenceābased recommendations
| dc.contributor.author | Marques, Nuno | |
| dc.contributor.author | Azevedo, Olga | |
| dc.contributor.author | Almeida, Ana Rita | |
| dc.contributor.author | Bento, Dina | |
| dc.contributor.author | Cruz, InĆŖs | |
| dc.contributor.author | Correia, Emanuel | |
| dc.contributor.author | LourenƧo, Carolina | |
| dc.contributor.author | Lopes, LuĆs Rocha | |
| dc.date.accessioned | 2020-11-03T11:03:52Z | |
| dc.date.available | 2020-11-03T11:03:52Z | |
| dc.date.issued | 2020 | |
| dc.description.abstract | Background The emergence of specific therapies for transthyretin cardiac amyloidosis (CA) warrants the need for a systematic review of the literature. Methods and Results A systematic review of the literature was conducted according to Preferred Reporting Items for Systematic Reviews and Meta-Analyses (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-language randomized controlled trials (RCTs), non-RCTs, or observational studies, which included adult patients with variant/wild-type transthyretin-CA, assessed specific therapies for transthyretin-CA, 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-RCTs). From 1203 records, 24 publications were selected, describing 4 RCTs (6 publications) and 16 non-RCTs (18 publications). Tafamidis was shown to significantly improve all-cause mortality and cardiovascular hospitalizations and reduce worsening in 6-minute walk test, Kansas City Cardiomyopathy Questionnaire-Overall Summary score, and NT-proBNP (N-terminal pro-B-type natriuretic peptide) in variant/wild-type transthyretin-CA. Patisiran showed promising results in a subgroup analysis of patients with variant transthyretin-CA, which have to be confirmed in RCTs. Inotersen showed conflicting results on cardiac imaging parameters. The one study on AG10 had only a 1-month duration and cardiovascular end points were exploratory and limited to cardiac biomarkers. Limited evidence from noncomparative single-arm small non-RCTs existed for diflunisal, epigallocatechin-3-gallate (green tea extract), and doxycycline+tauroursodeoxycholic acid/ursodeoxycholic acid. Conclusions This systematic review of the literature supports the use of tafamidis in wild-type and variant transthyretin-CA. Novel therapeutic targets including transthyretin gene silencers are currently under investigation. | pt_PT |
| dc.description.version | info:eu-repo/semantics/publishedVersion | pt_PT |
| dc.identifier.doi | 10.1161/JAHA.120.016614 | pt_PT |
| dc.identifier.uri | http://hdl.handle.net/10400.1/14807 | |
| dc.language.iso | eng | pt_PT |
| dc.peerreviewed | yes | pt_PT |
| dc.publisher | American Heart Association | pt_PT |
| dc.rights.uri | http://creativecommons.org/licenses/by/4.0/ | pt_PT |
| dc.subject | Amyloid | pt_PT |
| dc.subject | Transthyretin-related amyloidosis | pt_PT |
| dc.subject | Cardiac amyloidosis | pt_PT |
| dc.subject | Therapy | pt_PT |
| dc.subject | Transthyretin | pt_PT |
| dc.title | Specific therapy for transthyretin cardiac amyloidosis: a systematic literature review and evidenceābased recommendations | pt_PT |
| dc.type | journal article | |
| dspace.entity.type | Publication | |
| oaire.citation.issue | 19 | pt_PT |
| oaire.citation.startPage | e016614 | pt_PT |
| oaire.citation.title | Journal of the American Heart Association | pt_PT |
| oaire.citation.volume | 9 | pt_PT |
| person.familyName | Marques | |
| person.familyName | Bento | |
| person.givenName | Nuno | |
| person.givenName | Dina | |
| person.identifier.orcid | 0000-0003-0275-2807 | |
| person.identifier.orcid | 0000-0001-6383-7228 | |
| rcaap.rights | openAccess | pt_PT |
| rcaap.type | article | pt_PT |
| relation.isAuthorOfPublication | 1b66ba1f-d295-4211-b41e-c0f0b622eea0 | |
| relation.isAuthorOfPublication | 7f5dd19b-baed-4cfe-807f-e8bb2267cf74 | |
| relation.isAuthorOfPublication.latestForDiscovery | 7f5dd19b-baed-4cfe-807f-e8bb2267cf74 |
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