Evidence map›Paper›PMID 40251502›Full record

SynthesisBMC cardiovascular disorders2025

The efficacy and safety of specific therapies for cardiac Transthyretin-mediated amyloidosis: a systematic review and meta-analysis of randomized trials.

Alonzo Armani Prata, Eric Shih Katsuyama, Pedro Gabriel Scardini, Ana Carolina Covre, Wilson Falco Neto, Julia Marques Fernandes, Gabriel Scarpioni Barbosa, Chris Fukunaga, Rafael Petri Pinheiro, Vanio L J Antunes and 2 more

Abstract readSystematic ReviewMeta-Analysis
In one paragraph

Synthesis in BMC cardiovascular disorders, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 3 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
3citing papers in PubMed, 1 pooled it
–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

3 citing papers in PubMed, 1 synthesis or guideline pooled it.

  1. Pooled it
  2. Article
  3. Cardiac Amyloidosis: Tribulations and New Frontiers.Journal of personalized medicine · 2025
    Review
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

12 authors.

Alonzo Armani PrataFederal University of Espírito Santo (UFES) - Campus Maruípe, Avenida Marechal Campos, 1468, Vitória, ES, 29043-900, Brazil. alonzo.prata@edu.ufes.br.
Eric Shih KatsuyamaDepartment of Medicine, FMABC University Centre, São Paulo, Brazil.
Pedro Gabriel ScardiniEscola Superior de Ciências da Santa Casa de Misericórdia de Vitória, Vitória, Brazil.
Ana Carolina CovreFederal University of Espírito Santo (UFES) - Campus Maruípe, Avenida Marechal Campos, 1468, Vitória, ES, 29043-900, Brazil.
Wilson Falco NetoFAMECA University Center, Catanduva, Brazil.
Julia Marques FernandesAlbert Einstein Israeli Faculty of Health Sciences, São Paulo, Brazil.
Gabriel Scarpioni BarbosaFASM, Faculdade Santa Marcelina, São Paulo, Brazil.
Chris FukunagaDepartment of Medicine, FMABC University Centre, São Paulo, Brazil.
Rafael Petri PinheiroFederal University of Rio de Janeiro, UFRJ, Rio de Janeiro, Brazil.
Vanio L J AntunesPorto Alegre Health Sciences Federal University, Porto Alegre, Brazil.
Luciana Gioli-PereiraAlbert Einstein Israeli Faculty of Health Sciences, São Paulo, Brazil.
Fabio FernandesHeart Institute - InCor - University of Sao Paulo, Sao Paulo, Brazil.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

backgroundTransthyretin (TTR) Cardiomyopathy (ATTR-CM) is characterized by the deposition of misfolded TTR monomers in the heart, leading to progressive heart failure. TTR-specific therapies offer a pharmacological approach to slow disease progression. However, there remains limited data on the efficacy, comparative effectiveness, and safety of these therapies. Therefore, we aim to perform a systematic review and meta-analysis of randomized controlled trials (RCTs) comparing TTR-specific therapies with placebo in patients with ATTR-CM.

methodsWe searched through Pubmed, Cochrane, and Embase databases. Our primary outcome was: (1) All Cause Mortality. We also performed a subgroup analysis comparing TTR stabilizers versus TTR knock-down therapies (RNA inhibitors and antisense oligonucleotides).

resultsNine RCTs were included, involving 2,713 patients, of whom 1,160 (59.34%) were assigned to the TTR-specific therapies group. In the pooled analysis, TTR-specific therapies were associated with a significant reduction in all-cause mortality (RR 0.70; 95% CI 0.60, 0.83; p < 0.01; I² = 0%), with both TTR stabilizers and knock-down therapies showing equally effective reductions (p = 0.97). Additionally, TTR-specific therapies improved LV longitudinal strain (SMD - 0.22; 95% CI -0.34, -0.10; p < 0.01; I² = 17%) and reduced LV mass (SMD - 9.11 g; 95% CI -16.4 g, -1.82 g; p = 0.01; I² = 0%).

conclusionThis meta-analysis highlights the potential of TTR-targeting therapies as an effective option for managing ATTR-CM, with significant improvements in survival. No efficacy differences were found between TTR stabilizers and knock-down therapies.

Indexed as

Amyloid Neuropathies, FamilialCardiomyopathiesPrealbuminRNAi TherapeuticsAgedFemaleHumansMaleMiddle AgedRandomized Controlled Trials as TopicRecovery of FunctionRisk FactorsTreatment OutcomePrealbuminTTR protein, humanTransthyretin cardiac amyloidosisTransthyretin gene antisense oligonucleotidesTransthyretin gene RNA inhibitorsTransthyretin stabilizers

Identifiers

PMID40251502
PMCPMC12007281

What Socratic holds

Textmetadata
LicenceCC BY-NC-ND
Read underepoch 390

Registered trials

None linked

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 Socratic graph.