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Effect of eplontersen on cardiac structure and function in patients with hereditary transthyretin amyloidosis

Background - Hereditary transthyretin amyloidosis (ATTRv) is associated with polyneuropathy, cardiomyopathy, or both. The effects of eplontersen on cardiac structure and function were assessed. - Methods and Results - NEURO-TTRansform was an open-label trial involving 144 adults with ATTRv polyneuro...

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Main Authors: Masri, Ahmad (Author) , Maurer, Mathew S. (Author) , Claggett, Brian L. (Author) , Kulac, Ian (Author) , Waddington Cruz, Marcia (Author) , Conceição, Isabel (Author) , Weiler, Markus (Author) , Berk, John L. (Author) , Gertz, Morie (Author) , Gillmore, Julian D. (Author) , Rush, Stephen (Author) , Chen, Jersey (Author) , Zhou, Wunan (Author) , Kwoh, Jesse (Author) , Duran, Jason M. (Author) , Tsimikas, Sotirios (Author) , Solomon, Scott D. (Author)
Format: Article (Journal)
Language:English
Published: August 2024
In: Journal of cardiac failure
Year: 2024, Volume: 30, Issue: 8, Pages: 973-980
ISSN:1532-8414
DOI:10.1016/j.cardfail.2023.11.016
Online Access:Verlag, lizenzpflichtig, Volltext: https://doi.org/10.1016/j.cardfail.2023.11.016
Verlag, lizenzpflichtig, Volltext: https://www.sciencedirect.com/science/article/pii/S1071916423008941
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Author Notes:Ahmad Masri, Mathew S. Maurer, Brian L. Claggett, Ian Kulac, Marcia Waddington Cruz, Isabel Conceição, Markus Weiler, John L. Berk, Morie Gertz, Julian D. Gillmore, Stephen Rush, Jersey Chen, Wunan Zhou, Jesse Kwoh, Jason M. Duran, Sotirios Tsimikas and Scott D. Solomon
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Summary:Background - Hereditary transthyretin amyloidosis (ATTRv) is associated with polyneuropathy, cardiomyopathy, or both. The effects of eplontersen on cardiac structure and function were assessed. - Methods and Results - NEURO-TTRansform was an open-label trial involving 144 adults with ATTRv polyneuropathy (49 patients [34%] with cardiomyopathy) receiving eplontersen throughout and compared with 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. 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 of 4.3% (95% confidence interval 1.40-21.01; P=.049) and stroke volume 10.64 mL (95% confidence interval 3.99-17.29; P=.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.
Item Description:Online verfügbar: 7. Dezember 2023, Artikelversion: 13. August 2024
Gesehen am 10.07.2025
Physical Description:Online Resource
ISSN:1532-8414
DOI:10.1016/j.cardfail.2023.11.016

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