Effect of vutrisiran on components of health status in transthyretin amyloidosis with cardiomyopathy: the HELIOS-B study.
Yasuhiro Hamatani, Brian L Claggett, Muthiah Vaduganathan et al.
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In 30 seconds
This analysis of HELIOS-B, a double-blind, randomized, placebo-controlled trial, examined individual health-status components in patients with transthyretin amyloidosis with cardiomyopathy (ATTR-CM). Among 486 participants with KCCQ assessments at baseline and 30 months, vutrisiran was associated with more favourable changes than placebo in most of the 23 components (median difference +3.8 points; range -0.9 to +7.6). The largest differences favoured vutrisiran for physical activities and feeling discouraged or down because of heart failure. The age–KCCQ-OSS relationship was shifted by approximately 11 years (95% CI: 3-20 years).
Key findings
- Vutrisiran was associated with more favourable changes than placebo in most KCCQ components (median: +3.8, range: -0.9 to +7.6).
- The greatest differences favouring vutrisiran involved hurrying or jogging, walking on level ground, and feeling discouraged or down with heart failure.
- At 30 months, vutrisiran shifted the age–KCCQ-OSS relationship by approximately 11 years (95% CI: 3-20 years).
- Similar or greater benefits were observed among participants not taking tafamidis at baseline.
Why it matters
Overall health-status scores can obscure which aspects of daily life change with treatment. These findings suggest that vutrisiran’s health-status benefit in ATTR-CM was evident across multiple patient-reported domains, particularly physical limitations and quality of life. The component-level results help contextualize the overall score, while remaining patient-reported outcomes from a trial analysis rather than evidence of benefit in every individual.
What to keep in mind
The component analysis included the 486 of 654 trial participants with KCCQ assessments at both baseline and 30 months; findings therefore reflect this assessed subgroup.