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

ABSTRACT:

Background

Transthyretin (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.

Objectives

This 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.

Methods

A comprehensive search of PubMed, Embase, and Cochrane databases was conducted for randomized controlled trials (RCTs) published through April 2025. Eligible studies compared patisiran, tafamidis, i

SUBMITTER: Facin LC 

PROVIDER: S-EPMC12677762 | biostudies-literature | 2025

REPOSITORIES: biostudies-literature