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Heart failure & cardiomyopathiesSecondary analysis

Effects of vutrisiran on cardiac structure and function in patients with transthyretin amyloidosis with cardiomyopathy: secondary outcomes of the HELIOS-B trial

Nat MedPublished

  • Secondary analysis of the HELIOS-B randomised trial
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Authors' conclusion

Consistent with its clinical benefits, these echocardiographic findings indicate favorable effects of vutrisiran on cardiac structure and function in patients with ATTR-CM.

Study context

Population
Patients with transthyretin amyloidosis with cardiomyopathy (ATTR-CM)93% men
Participants
654 patients
Comparison
Vutrisiran vs placebo
Primary endpoint
Echocardiographic measures of cardiac structure and function
Follow-up
30 months

Primary result

As reported in the abstract

Echocardiographic cardiac structure and function
Vutrisiran vs PlaceboLeast squares mean difference, mm (95% CI)P value
mean LV wall thickness
−0.4(−0.8 to 0.0)
P = 0.03
Vutrisiran vs PlaceboLeast squares mean difference, g m-2 (95% CI)P value
LV mass index
−10.6(−18.0 to −3.3)
P < 0.01
Vutrisiran vs PlaceboLeast squares mean difference (95% CI)P value
LVEF (attenuated decline)
2.0%(0.3–3.7)
P = 0.02
absolute global longitudinal strain (attenuated decline)
1.2%(0.7–1.7)
P < 0.01
average E/e′ ratio
−2.0(−2.9 to −1.2)
P < 0.01
E/A ratio
−0.3(−0.6 to −0.0)
P = 0.04
Vutrisiran vs PlaceboLeast squares mean difference, ml (95% CI)P value
LV stroke volume (attenuated decline)
4.1(1.7–6.4)
P < 0.01

Original publication

Effects of vutrisiran on cardiac structure and function in patients with transthyretin amyloidosis with cardiomyopathy: secondary outcomes of the HELIOS-B trial. Nat Med. Published 6 August 2025. doi:10.1038/s41591-025-03851-z
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Summarised from the published abstract; every number is checked against it