Masri, Ahmad;
Maurer, Mathew S;
Claggett, Brian L;
Kulac, Ian;
Cruz, Marcia Waddington;
Conceição, Isabel;
Weiler, Markus;
... Solomon, Scott D; + view all
(2023)
Effect of Eplontersen on Cardiac Structure and Function in Patients with Hereditary Transthyretin Amyloidosis.
Journal of Cardiac Failure
10.1016/j.cardfail.2023.11.016.
(In press).
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1-s2.0-S1071916423008941-main.pdf - Accepted Version Access restricted to UCL open access staff until 8 December 2024. Download (2MB) |
Abstract
BACKGROUND: Hereditary transthyretin amyloidosis (ATTRv) is associated with polyneuropathy, cardiomyopathy, or both. The effects of eplontersen on cardiac structure and function were assessed. METHODS: NEURO-TTRansform was an open-label trial involving 144 adults with ATTRv polyneuropathy (49 patients (34%) with cardiomyopathy) receiving eplontersen throughout and compared to a historical placebo group (n=60, 30 patients (50%) with cardiomyopathy) from the NEURO-TTR trial at Week 65. Treatment effect (eplontersen vs placebo), presented as mean difference (95% confidence interval) was analyzed after adjusting for age, sex, region, baseline value, ATTRv disease stage, previous ATTRv treatment, and V30M transthyretin variant. RESULTS: There were notable differences at baseline between the eplontersen group and historical placebo. In the cardiomyopathy subgroup, 65 weeks of eplontersen treatment was associated with improvement from baseline relative to placebo in left ventricular ejection fraction (LVEF) 4.3% (1.40 to 21.01, p=0.049) and stroke volume 10.64 ml (3.99, 17.29, p=0.002) while the remainder of echocardiographic parameters remained stable. CONCLUSIONS: Eplontersen was associated with stable or improved measures of cardiac structure and function vs historical placebo in patients with ATTRv polyneuropathy and cardiomyopathy. Further investigation into eplontersen's effect on transthyretin amyloid cardiomyopathy is being conducted in the CARDIO-TTRansform trial.
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