Clinical Report: Cardiac MRI Assessment of Changes in Cardiac Measures and Amyloid Burden During Vutrisiran Treatment
Overview
In a post hoc analysis of 43 HELIOS-B participants at the UK National Amyloidosis Centre, serial cardiovascular magnetic resonance (CMR) was used to assess cardiac structure, function, and amyloid burden during vutrisiran or placebo treatment. The supplied article text reports statistically significant, directionally favorable changes in biventricular ejection fraction with vutrisiran versus placebo; it does not provide the numerical estimates or the remaining CMR results.
Background
Transthyretin amyloid cardiomyopathy (ATTR-CM) involves progressive deposition of amyloid fibrils in the myocardial extracellular space and is associated with heart failure, declining quality of life, and mortality. Vutrisiran is a subcutaneous RNA interference therapy that suppresses hepatic transthyretin synthesis. In HELIOS-B, vutrisiran reduced risks of all-cause mortality and cardiovascular events versus placebo while preserving functional capacity and quality of life, as reported in the supplied context. CMR, including extracellular volume (ECV) mapping, can assess cardiac structure, function, and amyloid burden; ECV changes have been associated with prognosis in ATTR-CM.
Data Highlights
Measure
Finding reported in supplied article text
Baseline CMR cohort
43 participants; mean age 75.0 (SD 5.7) years; 2 (4.7%) female and 41 (95.3%) male
Treatment allocation
Vutrisiran: 21 (48.8%); placebo: 22 (51.2%)
CMR follow-up completion
Year 1: 39 (21 vutrisiran, 18 placebo); year 2: 26 (14, 12); year 3: 17 (9, 8)
Deaths during follow-up
2 in the vutrisiran group and 6 in the placebo group
Reported mixed-model result
Vutrisiran was associated with statistically significant, directionally favorable changes in biventricular ejection fraction measures versus placebo. Numerical estimates are not included in the supplied article text.
Key Findings
The analysis included 43 HELIOS-B participants with baseline CMR: 21 assigned to vutrisiran and 22 to placebo.
Serial CMR completion declined from 39 participants at year 1 to 17 at year 3; the supplied text reports 21 and 18 participants at year 1, and 9 and 8 at year 3, in the vutrisiran and placebo groups, respectively.
Follow-up deaths were reported in 2 vutrisiran recipients and 6 placebo recipients. The article text states that, apart from the lower frequency of death in the treatment group, attrition was balanced between arms and consistent with expectations for this population.
No participant received tafamidis during the study.
Mixed-model analysis found statistically significant, directionally favorable changes in biventricular ejection fraction measures with vutrisiran relative to placebo; the supplied excerpt ends before giving the specific measures and estimates.
The supplied related-article context describes CMR case and tafamidis reports, but provides no quantitative findings from those reports that can be compared with this analysis.
Clinical Implications
The supplied article describes serial CMR, including ECV mapping, as a means of objectively assessing cardiac structure, function, amyloid burden, and treatment response in ATTR-CM. The reported ejection-fraction findings are limited to the direction and statistical significance in the provided excerpt; numerical effect estimates and other CMR outcomes are not available here.
Conclusion
In this small post hoc UK cohort from HELIOS-B, vutrisiran was associated with favorable changes in biventricular ejection fraction measures compared with placebo. The supplied text does not include the full CMR results, limiting further characterization of changes in cardiac measures or amyloid burden.
by Yousuf Razvi, Awais Sheikh, Rishi K. Patel, Anouk Achten, Josephine Mansell, Ana Martinez-Naharro, Lucia Venneri, Philip N. Hawkins, James Moon, John Vest, Sameer Bansilal, Satish Eraly, Emre Aldinc, Brian L. Claggett, Scott D. Solomon, Julian D. Gillmore, Marianna Fontana
Vascular surgeon Eva Maria Urrechaga, M.D., has joined Baptist Health Heart & Vascular Care at Baptist Health Miami Cardiac & Vascular Institute. She specializes in evaluating and treating conditions affecting the arteries and veins through open surgery and minimally invasive endovascular procedures.
