医学
转甲状腺素
心肌病
内科学
射血分数
安慰剂
心脏淀粉样变性
心脏病学
淀粉样变性
多发性神经病
置信区间
心功能曲线
心力衰竭
病理
替代医学
作者
Ahmad Masri,Mathew S. Maurer,Brian Claggett,Ian J. Kulac,Márcia Waddington Cruz,Isabel Conceição,Markus Weiler,John L. Berk,Morie A. Gertz,Julian D. Gillmore,Stephen Rush,Jersey Chen,Wunan Zhou,Jesse Kwoh,Jason Duran,Sotirios Tsimikas,Scott D. Solomon
标识
DOI:10.1016/j.cardfail.2023.11.016
摘要
Hereditary transthyretin amyloidosis (ATTRv) is associated with polyneuropathy, cardiomyopathy, or both. The effects of eplontersen on cardiac structure and function were assessed.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.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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