Evidence map›Paper›PMID 41059800›Full record

SynthesisArquivos brasileiros de cardiologia2025

Disease-Modifying Therapies for Transthyretin Amyloid Cardiomyopathy: A Systematic Review and Meta-Analysis.

Ligia Carolina Facin, Igor Pacheco Fiuza Romeiro, Kaushiki Sapahia, Beatriz Austregésilo de Athayde de Hollanda Morais, Juliana Queiroz Vasconcelos Muniz, Juliana Domenes Pereira, Cintia Gomes, Celina Borges Migliavaca, André Zimerman, Andreia Biolo

Abstract readSystematic ReviewMeta-Analysis
In one paragraph

Synthesis in Arquivos brasileiros de cardiologia, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 2 papers.

0numbers the graph read from it
0cells of the map it votes in
2citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

2 citing papers in PubMed.

  1. Review
  2. To Silence or to Stabilize: That is the Question.Arquivos brasileiros de cardiologia · 2025
    Article
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

10 authors.

Ligia Carolina FacinServiço de Cardiologia, Hospital Moinhos de Vento, Porto Alegre, RS - Brasil.ORCID 0000-0001-6060-8403
Igor Pacheco Fiuza RomeiroDepartamento de Medicina, Universidade Federal do Ceará, Fortaleza, PE - Brasil.
Kaushiki SapahiaDepartment of Medicine, University College of Medical Sciences of New Delhi, Delhi - Índia.
Beatriz Austregésilo de Athayde de Hollanda MoraisDepartamento de Medicina, Centro Universitário de Maceió (Cesmac), Maceió, AL - Brasil.
Juliana Queiroz Vasconcelos MunizDepartment of Internal Medicine, Schmieder Klinik Heidelberg, Heidelberg - Alemanha.
Juliana Domenes PereiraDepartamento de Clínica Médica, Universidade Estadual de Campinas, São Paulo, SP - Brasil.
Cintia GomesDepartment of Internal Medicine, University of Colorado Health Parkview Medical Center, Colorado - EUA.
Celina Borges MigliavacaPrograma de Pós-Graduação em Cardiologia, Universidade Federal do Rio Grande do Sul, Porto Alegre, RS - Brasil.
André ZimermanServiço de Cardiologia, Hospital Moinhos de Vento, Porto Alegre, RS - Brasil.
Andreia BioloServiço de Cardiologia, Hospital Moinhos de Vento, Porto Alegre, RS - Brasil.ORCID 0000-0002-8579-4158

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

backgroundTransthyretin (TTR) amyloid cardiomyopathy (ATTR-CM) is the most common form of restrictive cardiomyopathy. Emerging pharmacological therapies aim to alter the natural history of disease and delay its advancement. However, data directly comparing the efficacy of different drug classes versus placebo remain limited.

objectivesThis systematic review assessed the efficacy of TTR stabilizers and silencers compared with placebo on all-cause mortality, hospitalizations, functional outcomes, and serum levels of the biomarker NT-proBNP in patients with ATTR-CM.

methodsA comprehensive search of PubMed, Embase, and Cochrane databases was conducted for randomized controlled trials (RCTs) published through April 2025. Eligible studies compared patisiran, tafamidis, inotersen, revusiran, acoramidis, or vutrisiran to placebo in patients with ATTR-CM. Analyses were stratified by drug class, and statistical significance was set at p<0.05.

resultsSeven RCTs involving 2,526 participants were included; 42.5% received TTR stabilizers and 57.5% received TTR silencers. Compared with placebo, TTR stabilizers significantly reduced all-cause mortality (RR: 0.71; 95% CI 0.59-0.87; p=0.0006) and hospitalizations (RR: 0.81; 95% CI 0.73-0.89; p<0,0001). TTR silencers did not significantly reduce mortality (RR: 0.79; 95% CI 0.37-1.68; p=0.54) or hospitalizations (RR: 1.11; 95% CI 0.83-1.48; p=0.48). Both therapies were associated with improvements in 6-minute walk distance, quality of life, and reductions in serum NT-proBNP levels.

conclusionTTR stabilizers significantly reduced all-cause mortality and hospitalizations in patients with ATTR-CM compared with placebo. These benefits were not observed with TTR silencers, potentially due to shorter follow-up durations in the studies evaluated. Both therapies improved functional status and serum levels of NT-proBNP.

Indexed as

Amyloid Neuropathies, FamilialCardiomyopathiesBenzoxazolesBiomarkersHospitalizationHumansMaleNatriuretic Peptide, BrainPeptide FragmentsPrealbuminRandomized Controlled Trials as TopicTreatment OutcomeBenzoxazolesBiomarkersNatriuretic Peptide, BrainPeptide FragmentsPrealbuminpro-brain natriuretic peptide (1-76)tafamidis

Identifiers

PMID41059800
PMCPMC12677762

What OpenQuestion holds

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Registered trials

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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.