Specific Therapy for Transthyretin Cardiac Amyloidosis: A Systematic Literature Review and Evidence‐Based Recommendations

Author:

Marques Nuno123ORCID,Azevedo Olga456ORCID,Almeida Ana Rita7,Bento Dina123,Cruz Inês7,Correia Emanuel8,Lourenço Carolina9,Lopes Luís Rocha101112

Affiliation:

1. Algarve Biomedical Center Algarve Portugal

2. Biomedical and Medical Department Algarve University Algarve Portugal

3. Cardiology Department Centro Hospitalar Universitário do Algarve Algarve Portugal

4. Cardiology Department Hospital Senhora da Oliveira Guimarães Portugal

5. Life and Health Sciences Research Institute (ICVS) School of Medicine University of Minho Braga Portugal

6. ICVS/3Bs PT Government Associate Laboratory Braga/Guimarães Portugal

7. Cardiology Department Hospital Garcia de Orta Almada Portugal

8. Cardiology Department Centro Hospitalar Tondela Viseu Viseu Portugal

9. Cardiology Department Centro Hospitalar Universitário de Coimbra Coimbra Portugal

10. St. Bartholomew’s Hospital‐Barts Heart Centre Barts Health NHS Trust London United Kingdom

11. Centre for Heart Muscle Disease Institute of Cardiovascular Science University College of London United Kingdom

12. Centro Cardiovacular Universidade Lisboa Lisboa Portugal

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.

Publisher

Ovid Technologies (Wolters Kluwer Health)

Subject

Cardiology and Cardiovascular Medicine

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