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.
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).
- 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.
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.
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