作者
Scott D Solomon, David Adams, Arnt Kristen, Martha Grogan, Alejandra González-Duarte, Mathew S Maurer, Giampaolo Merlini, Thibaud Damy, Michel S Slama, Thomas H Brannagan III, Angela Dispenzieri, John L Berk, Amil M Shah, Pushkal Garg, Akshay Vaishnaw, Verena Karsten, Jihong Chen, Jared Gollob, John Vest, Ole Suhr
发表日期
2019/1/22
期刊
Circulation
卷号
139
期号
4
页码范围
431-443
出版商
Lippincott Williams & Wilkins
简介
Background
Hereditary transthyretin-mediated (hATTR) amyloidosis is a rapidly progressive, multisystem disease that presents with cardiomyopathy or polyneuropathy. The APOLLO study assessed the efficacy and tolerability of patisiran in patients with hATTR amyloidosis. The effects of patisiran on cardiac structure and function in a prespecified subpopulation of patients with evidence of cardiac amyloid involvement at baseline were assessed.
Methods
APOLLO was an international, randomized, double-blind, placebo-controlled phase 3 trial in patients with hATTR amyloidosis. Patients were randomized 2:1 to receive 0.3 mg/kg patisiran or placebo via intravenous infusion once every 3 weeks for 18 months. The prespecified cardiac subpopulation comprised patients with a baseline left ventricular wall thickness ≥13 mm and no history of hypertension or aortic valve disease. Prespecified exploratory cardiac end …
学术搜索中的文章
SD Solomon, D Adams, A Kristen, M Grogan… - Circulation, 2019