亲爱的研友该休息了!由于当前在线用户较少,发布求助请尽量完整地填写文献信息,科研通机器人24小时在线,伴您度过漫漫科研夜!身体可是革命的本钱,早点休息,好梦!

Aficamten for Symptomatic Obstructive Hypertrophic Cardiomyopathy

医学 心脏病学 心室流出道 肥厚性心肌病 内科学 安慰剂 瓦萨尔瓦机动 血压 射血分数 临床终点 心室流出道梗阻 肺动脉高压 心力衰竭 随机对照试验 替代医学 病理
作者
Martin S. Maron,Ahmad Masri,Michael E. Nassif,Roberto Barriales‐Villa,Michael Arad,Nuno Cardim,Lubna Choudhury,Brian Claggett,Caroline Coats,Hans‐Dirk Düngen,Pablo García‐Pavía,Albert Hagège,James L. Januzzi,Matthew M.Y. Lee,Gregory D. Lewis,Changsheng Ma,Michelle Michels,Iacopo Olivotto,Artur Oręziak,Anjali Owens
出处
期刊:The New England Journal of Medicine [Massachusetts Medical Society]
卷期号:390 (20): 1849-1861 被引量:109
标识
DOI:10.1056/nejmoa2401424
摘要

BackgroundOne of the major determinants of exercise intolerance and limiting symptoms among patients with obstructive hypertrophic cardiomyopathy (HCM) is an elevated intracardiac pressure resulting from left ventricular outflow tract obstruction. Aficamten is an oral selective cardiac myosin inhibitor that reduces left ventricular outflow tract gradients by mitigating cardiac hypercontractility.MethodsIn this phase 3, double-blind trial, we randomly assigned adults with symptomatic obstructive HCM to receive aficamten (starting dose, 5 mg; maximum dose, 20 mg) or placebo for 24 weeks, with dose adjustment based on echocardiography results. The primary end point was the change from baseline to week 24 in the peak oxygen uptake as assessed by cardiopulmonary exercise testing. The 10 prespecified secondary end points (tested hierarchically) were change in the Kansas City Cardiomyopathy Questionnaire clinical summary score (KCCQ-CSS), improvement in the New York Heart Association (NYHA) functional class, change in the pressure gradient after the Valsalva maneuver, occurrence of a gradient of less than 30 mm Hg after the Valsalva maneuver, and duration of eligibility for septal reduction therapy (all assessed at week 24); change in the KCCQ-CSS, improvement in the NYHA functional class, change in the pressure gradient after the Valsalva maneuver, and occurrence of a gradient of less than 30 mm Hg after the Valsalva maneuver (all assessed at week 12); and change in the total workload as assessed by cardiopulmonary exercise testing at week 24.ResultsA total of 282 patients underwent randomization: 142 to the aficamten group and 140 to the placebo group. The mean age was 59.1 years, 59.2% were men, the baseline mean resting left ventricular outflow tract gradient was 55.1 mm Hg, and the baseline mean left ventricular ejection fraction was 74.8%. At 24 weeks, the mean change in the peak oxygen uptake was 1.8 ml per kilogram per minute (95% confidence interval [CI], 1.2 to 2.3) in the aficamten group and 0.0 ml per kilogram per minute (95% CI, −0.5 to 0.5) in the placebo group (least-squares mean between-group difference, 1.7 ml per kilogram per minute; 95% CI, 1.0 to 2.4; P<0.001). The results for all 10 secondary end points were significantly improved with aficamten as compared with placebo. The incidence of adverse events appeared to be similar in the two groups.ConclusionsAmong patients with symptomatic obstructive HCM, treatment with aficamten resulted in a significantly greater improvement in peak oxygen uptake than placebo. (Funded by Cytokinetics; SEQUOIA-HCM ClinicalTrials.gov number, NCT05186818.)
最长约 10秒,即可获得该文献文件

科研通智能强力驱动
Strongly Powered by AbleSci AI
更新
PDF的下载单位、IP信息已删除 (2025-6-4)

科研通是完全免费的文献互助平台,具备全网最快的应助速度,最高的求助完成率。 对每一个文献求助,科研通都将尽心尽力,给求助人一个满意的交代。
实时播报
4秒前
芝麻发布了新的文献求助10
5秒前
昏睡的沛柔完成签到 ,获得积分10
7秒前
Aliya完成签到 ,获得积分10
16秒前
酷波er应助科研通管家采纳,获得10
16秒前
42秒前
44秒前
45秒前
李李发布了新的文献求助10
46秒前
bksqc发布了新的文献求助10
48秒前
要文献啊完成签到 ,获得积分10
48秒前
53秒前
WUHUIWEN完成签到,获得积分10
57秒前
Liiiiiiiiii发布了新的文献求助10
57秒前
58秒前
1分钟前
尊敬夜南发布了新的文献求助10
1分钟前
韦雪莲完成签到 ,获得积分10
1分钟前
wz完成签到,获得积分10
1分钟前
1分钟前
Liiiiiiiiii完成签到,获得积分10
1分钟前
杜德爽发布了新的文献求助10
1分钟前
好好搞科研完成签到 ,获得积分10
1分钟前
1分钟前
热情的寄瑶完成签到 ,获得积分10
1分钟前
yanglinhai完成签到 ,获得积分10
1分钟前
饱满含玉完成签到,获得积分10
1分钟前
1分钟前
2分钟前
CipherSage应助bksqc采纳,获得10
2分钟前
2分钟前
大神完成签到,获得积分0
2分钟前
爆米花应助科研通管家采纳,获得10
2分钟前
研友_VZG7GZ应助科研通管家采纳,获得10
2分钟前
2分钟前
2分钟前
2分钟前
2分钟前
科研通AI2S应助PEITON采纳,获得10
2分钟前
bksqc发布了新的文献求助10
2分钟前
高分求助中
The Mother of All Tableaux: Order, Equivalence, and Geometry in the Large-scale Structure of Optimality Theory 3000
A new approach to the extrapolation of accelerated life test data 1000
ACSM’s Guidelines for Exercise Testing and Prescription, 12th edition 500
Indomethacinのヒトにおける経皮吸収 400
Phylogenetic study of the order Polydesmida (Myriapoda: Diplopoda) 370
基于可调谐半导体激光吸收光谱技术泄漏气体检测系统的研究 350
Robot-supported joining of reinforcement textiles with one-sided sewing heads 320
热门求助领域 (近24小时)
化学 材料科学 医学 生物 工程类 有机化学 生物化学 物理 内科学 纳米技术 计算机科学 化学工程 复合材料 遗传学 基因 物理化学 催化作用 冶金 细胞生物学 免疫学
热门帖子
关注 科研通微信公众号,转发送积分 3990031
求助须知:如何正确求助?哪些是违规求助? 3532108
关于积分的说明 11256334
捐赠科研通 3270943
什么是DOI,文献DOI怎么找? 1805146
邀请新用户注册赠送积分活动 882270
科研通“疑难数据库(出版商)”最低求助积分说明 809228