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Effect of Acoramidis on Heart Failure–Related Health Status

JAMA Cardiology·July 29Open Access
Cardiac & Cardiovascular SystemsPractice changingHeart FailureTransthyretin Amyloid CardiomyopathyRandomized Controlled TrialTransthyretin StabilizerOlder AdultAcoramidis

Summary

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What was studied

This secondary analysis of the ATTRibute-CM phase 3 RCT evaluated the effect of acoramidis 800 mg twice daily vs placebo on HF-related health status (KCCQ-OS score) in 611 adults with ATTR-CM over 30 months.

Key findings

Acoramidis produced a statistically significant, clinically meaningful improvement in KCCQ-OS vs placebo (LSM difference +9.9 points; 95% CI, 6.0–13.9; P < .001). At 30 months, 47% of acoramidis recipients were "alive and not worse" vs 30% on placebo (NNT = 6), and 46% were "alive and well" vs 31% (NNT = 7).

Study limitations

- This is a secondary/post hoc analysis of the trial, so some comparisons ("alive and better") were not pre-specified. - The population was predominantly older (mean age 77.2 years) and male (90.8%), limiting generalizability to women and younger patients. - KCCQ data were only available in surviving patients at each time point, which may introduce survivorship bias given the mortality difference between arms.

Clinical implications

In ATTR-CM, acoramidis not only reduces mortality and hospitalizations but also meaningfully preserves patient-reported quality of life — roughly 1 in 6 additional patients remain alive and not worse compared to placebo (NNT = 6). Clinicians can use these KCCQ data to counsel patients on expected functional benefits over a 2.5-year treatment course.

Related Questions

Explore related topics

What is the overall survival and hospitalization benefit of acoramidis in ATTR-CM from the ATTRibute-CM trial?How does acoramidis compare to tafamidis for quality of life and outcomes in transthyretin amyloid cardiomyopathy?What KCCQ score thresholds are clinically meaningful in heart failure patients?

Publication Details

Year
2026
Journal
JAMA Cardiology
Sample Size
n=611
Source
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