Heart failure & cardiomyopathies
Effects of Vutrisiran on Cardiac Function and Outcomes in Patients With Transthyretin Amyloidosis With Cardiomyopathy
Authors' conclusion
Echocardiographic measures of biventricular systolic and diastolic function provide important prognostic information beyond National Amyloidosis Centre stage in patients with ATTR-CM. Vutrisiran improved diastolic function and attenuated declines in left ventricular and right ventricular systolic function over 18 months.
Study context
- Population
- Patients with transthyretin amyloid cardiomyopathy (ATTR-CM)654 with echocardiographic data
- Participants
- 655 patients
- Comparison
- Vutrisiran vs placebo; echocardiographic measures and outcomes
- Primary endpoint
- Associations of echocardiographic measures of cardiac structure and function with the primary outcome (all-cause death and recurrent CV events)
Methodological detailslandmark analyses
- Landmark analyses
Primary result
As reported in the abstract
- LVEF, per 5% increase: HR 0.90, 95% CI 0.86-0.95
- Absolute global longitudinal strain, per 1% increase: HR 0.92, 95% CI 0.89-0.96
- Tricuspid annular systolic myocardial velocity, per 1-cm/s increase: HR 0.94, 95% CI 0.90-0.98
- Average E/e', per 1-U increase: HR 1.03, 95% CI 1.01-1.04
- Adjusted for age, sex, ATTR disease type and National Amyloidosis Centre stage
Further findings
| Outcome | Least squares mean difference (%) (95% CI) | |
|---|---|---|
| Change in cardiac function, vutrisiran vs placebo · 18 monthsVutrisiran vs Placebo · LVEF | 1.6 (0.1–3.2) | |
| Change in cardiac function, vutrisiran vs placebo · 18 monthsVutrisiran vs Placebo · absolute global longitudinal strain | 0.7 (0.3–1.2) | |
| −2024Difference, % (linear scale) |
| Outcome | Least squares mean difference (cm/s) (95% CI) | |
|---|---|---|
| Change in cardiac function, vutrisiran vs placebo · 18 monthsVutrisiran vs Placebo · tricuspid annular systolic myocardial velocity | 0.5 (0.1–0.9) | |
| −0.500.51Difference, cm/s (linear scale) |
Worsening of echocardiographic parameters at 18 months and primary outcome
- Worsening associated with heightened risk of the primary outcome
- Landmark analyses
Original publication
Effects of Vutrisiran on Cardiac Function and Outcomes in Patients With Transthyretin Amyloidosis With Cardiomyopathy. J Am Coll Cardiol. Published 12 August 2025. doi:10.
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