Heart failure & cardiomyopathiesSecondary analysis

Effects of Vutrisiran on Cardiac Function and Outcomes in Patients With Transthyretin Amyloidosis With Cardiomyopathy

J Am Coll CardiolPublished

  • Secondary analysis of the HELIOS-B randomised trial
  • landmark analyses

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)

Primary result

As reported in the abstract

Baseline echocardiographic parameters and primary outcome (HR per unit increase)
  • 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

Outcomeleast squares mean difference (%) (95% CI)
Change in cardiac function, vutrisiran vs placebo · 18 months — Vutrisiran vs Placebo · LVEF1.6 (0.1–3.2)
Change in cardiac function, vutrisiran vs placebo · 18 months — Vutrisiran vs Placebo · absolute global longitudinal strain0.7 (0.3–1.2)
Outcomeleast squares mean difference (cm/s) (95% CI)
Change in cardiac function, vutrisiran vs placebo · 18 months — Vutrisiran vs Placebo · tricuspid annular systolic myocardial velocity0.5 (0.1–0.9)

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:10.1016/j.jacc.2025.06.022

Summarised from the published abstract; every number is checked against it