Vutrisiran Improves Quality of Life Measures in ATTR-CM: HELIOS-B Analysis from ACC.26
Key Highlights:
- Vutrisiran improved nearly all Kansas City Cardiomyopathy Questionnaire (KCCQ) components versus placebo.
- The greatest benefits were observed in physical limitations and quality of life domains.
- The treatment effect corresponded to an estimated 11-year delay in expected KCCQ decline.
Vutrisiran improved multiple domains of patient-reported quality of life in individuals with transthyretin amyloidosis with cardiomyopathy (ATTR-CM), according to a prespecified analysis of the HELIOS-B trial presented at the American College of Cardiology’s Annual Scientific Session (ACC.26) in New Orleans, LA. The findings highlight broad-based benefits across nearly all components of the Kansas City Cardiomyopathy Questionnaire (KCCQ), a validated tool widely used in heart failure populations.
In this double-blind, randomized trial, investigators evaluated the impact of vutrisiran compared with placebo on quality of life over 30 months. The analysis focused on mean changes in each of the 23 individual KCCQ components.
Among 654 participants enrolled in HELIOS-B, 486 completed the KCCQ at both baseline and 30 months. The cohort had a mean age of 75±6 years, was predominantly male (93%), and largely consisted of patients with wild-type ATTR (91%). The baseline KCCQ overall summary score (KCCQ-OSS) averaged 74.6±18.9. Using multivariable linear regression models, researchers assessed changes in mean scores over time and compared observed outcomes with expected declines associated with aging.
Study Findings
Vutrisiran demonstrated improvement across nearly all KCCQ components compared with placebo, with a median increase of +3.8 points (range: −0.9 to +7.6). Only 1 component did not show improvement. The largest treatment-related gains were observed in domains related to physical limitations and overall quality of life, indicating meaningful functional and symptomatic benefits for patients.
At 30 months, KCCQ-OSS was inversely associated with age. The magnitude of benefit associated with vutrisiran corresponded to an estimated delay of approximately 11 years (95% CI: 3–20 years) in the expected decline of KCCQ-OSS.
Expert Commentary
“Vutrisiran demonstrated benefit in nearly all KCCQ components in ATTR-CM, with an overall benefit comparable to reversing more than a decade of aging,” the researchers concluded.
Reference:
Hamatani Y, Fontana M, Claggett B, et al. Impact of vutrisiran on the components of quality of life in transthyretin cardiomyopathy (ATTR-CM). Presented at: American College of Cardiology Annual Scientific Session (ACC.26); March 29, 2026; New Orleans, LA.
