Structural outcomes among patients receiving targeted therapy for ATTR cardiac amyloidosis - A Systematic Review and Meta-Analysis
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Background
Targeted pharmacologic therapies for transthyretin amyloid cardiomyopathy (ATTR-CM) improve survival; however, their effects on cardiac structural parameters remain incompletely defined.
Objectives
To evaluate the pooled effects of disease-modifying therapies for ATTR-CM on echocardiographic structural parameters.
Methods
In accordance with PRISMA guidelines, we performed a systematic review and meta-analysis of randomized controlled trials and observational studies published through March 2025 assessing transthyretin stabilizers and RNA-silencing therapies in adults with cardiac amyloidosis. Outcomes included changes in global longitudinal strain (GLS), left ventricular ejection fraction (LVEF), interventricular septal (IVS) thickness, left ventricular mass, stroke volume, E/e′ ratio, and LV end-diastolic volume. Pooled between-group mean differences were calculated using random-effects models. Sensitivity analyses were performed.
Results
Eighteen studies (11 randomized, 7 observational) encompassing 3,646 patients were included. Compared with control, drug therapy was associated with attenuation of GLS decline (mean difference [MD] -0.69%; 95% CI -1.10 to -0.29; P <0.001) and preservation of LVEF (MD 1.62%; 95% CI 0.73 to 2.51; P <0.001). Treatment was also associated with reduced worsening of E/e′ ratio, and preservation of stroke volume. No significant between-group differences were observed for IVS thickness, LV mass and LV end-diastolic volume. Within-group analyses showed no change in echocardiographic parameters between baseline and follow-up in treated patients, in contrast to significant worsening in the control cohort.
Conclusions
Disease-modifying therapies for ATTR-CM are associated with stabilization and attenuated progression of cardiac remodeling rather than reversal of structural abnormalities.
