Real-world experience with mavacamten in obstructive hypertrophic cardiomyopathy: Observations from a tertiary care center

IF 5.6 2区 医学 Q1 CARDIAC & CARDIOVASCULAR SYSTEMS
{"title":"Real-world experience with mavacamten in obstructive hypertrophic cardiomyopathy: Observations from a tertiary care center","authors":"","doi":"10.1016/j.pcad.2024.02.001","DOIUrl":null,"url":null,"abstract":"<div><h3>Background</h3><div>In symptomatic obstructive hypertrophic cardiomyopathy<span> (oHCM) patients, mavacamten is commercially approved to help improve left ventricular (LV) outflow tract (LVOT) gradients, symptoms, and reduce eligibility for septal reduction therapy (SRT) under the risk evaluation and mitigation strategy (REMS) program. We sought to prospectively report the initial real-world clinical experience with the use of commercially available mavacamten in a multi-hospital tertiary healthcare system.</span></div></div><div><h3>Methods</h3><div><span><span>We studied the first 150 consecutive oHCM patients (mean age 65 years, 53% women, 83% on betablockers and 61% in New York Heart Association [NYHA] class III) who were initiated on 5 mg of mavacamten with dose titrations using symptom assessment and echocardiographic measurements of LVOT gradient and </span>LV ejection fraction (LVEF) measurements. We measured changes in NYHA class, </span>LVEF, LVOT gradients (resting and Valsalva) at baseline, 4, 8 and 12 weeks.</div></div><div><h3>Results</h3><div>At 261 ± 143 days (range of 31–571 days), 69 (46%) patients had ≥1 NYHA class, and 27 (18%) additional patients had ≥2 NYHA class improvement. The mean Valsalva LVOT gradient decreased from 72 ± 43 mmHg at baseline to 29 ± 31 mmHg at 4 weeks, 29 ± 28 mmHg at 8 weeks and 30 ± 29 mmHg at 12 weeks (<em>p</em> &lt; 0.001). At baseline, 100% patients had Valsalva LVOT gradients ≥30 mmHg, which reduced to 29% at 4 weeks, 28% at 8 weeks and 30% at 12 weeks. In 40 patients who reported no symptomatic improvement, the mean Valsalva LVOT gradient decreased from 73 ± 39 mmHg at baseline to 34 ± 27 mmHg at 4 weeks, 35 ± 28 mmHg at 8 weeks and 30 ± 24 mmHg at 12 weeks (<em>P</em> &lt; 0.001). The mean LVEF at baseline was 66 ± 6% and changed to 64 ± 5% at 4 weeks, 63 ± 5% at 8 weeks and 62 ± 7% at 12 weeks (<em>p</em> &lt; 0.0001). No patient underwent SRT, developed LVEF ≤30% or developed heart failure requiring admission. Three (2%) patients needed temporary interruption of mavacamten due to LVEF&lt;50%.</div></div><div><h3>Conclusions</h3><div>In a real-world study in symptomatic oHCM patients at a multi-hospital tertiary care referral center, we demonstrate the efficacy and safety, along with the logistic feasibility of prescribing mavacamten under the REMS program.</div></div>","PeriodicalId":21156,"journal":{"name":"Progress in cardiovascular diseases","volume":null,"pages":null},"PeriodicalIF":5.6000,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"Progress in cardiovascular diseases","FirstCategoryId":"3","ListUrlMain":"https://www.sciencedirect.com/science/article/pii/S0033062024000227","RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q1","JCRName":"CARDIAC & CARDIOVASCULAR SYSTEMS","Score":null,"Total":0}
引用次数: 0

Abstract

Background

In symptomatic obstructive hypertrophic cardiomyopathy (oHCM) patients, mavacamten is commercially approved to help improve left ventricular (LV) outflow tract (LVOT) gradients, symptoms, and reduce eligibility for septal reduction therapy (SRT) under the risk evaluation and mitigation strategy (REMS) program. We sought to prospectively report the initial real-world clinical experience with the use of commercially available mavacamten in a multi-hospital tertiary healthcare system.

Methods

We studied the first 150 consecutive oHCM patients (mean age 65 years, 53% women, 83% on betablockers and 61% in New York Heart Association [NYHA] class III) who were initiated on 5 mg of mavacamten with dose titrations using symptom assessment and echocardiographic measurements of LVOT gradient and LV ejection fraction (LVEF) measurements. We measured changes in NYHA class, LVEF, LVOT gradients (resting and Valsalva) at baseline, 4, 8 and 12 weeks.

Results

At 261 ± 143 days (range of 31–571 days), 69 (46%) patients had ≥1 NYHA class, and 27 (18%) additional patients had ≥2 NYHA class improvement. The mean Valsalva LVOT gradient decreased from 72 ± 43 mmHg at baseline to 29 ± 31 mmHg at 4 weeks, 29 ± 28 mmHg at 8 weeks and 30 ± 29 mmHg at 12 weeks (p < 0.001). At baseline, 100% patients had Valsalva LVOT gradients ≥30 mmHg, which reduced to 29% at 4 weeks, 28% at 8 weeks and 30% at 12 weeks. In 40 patients who reported no symptomatic improvement, the mean Valsalva LVOT gradient decreased from 73 ± 39 mmHg at baseline to 34 ± 27 mmHg at 4 weeks, 35 ± 28 mmHg at 8 weeks and 30 ± 24 mmHg at 12 weeks (P < 0.001). The mean LVEF at baseline was 66 ± 6% and changed to 64 ± 5% at 4 weeks, 63 ± 5% at 8 weeks and 62 ± 7% at 12 weeks (p < 0.0001). No patient underwent SRT, developed LVEF ≤30% or developed heart failure requiring admission. Three (2%) patients needed temporary interruption of mavacamten due to LVEF<50%.

Conclusions

In a real-world study in symptomatic oHCM patients at a multi-hospital tertiary care referral center, we demonstrate the efficacy and safety, along with the logistic feasibility of prescribing mavacamten under the REMS program.
阻塞性肥厚型心肌病患者使用马伐康坦的真实体验:一家三级医疗中心的观察结果。
背景:对于有症状的梗阻性肥厚型心肌病(oHCM)患者,mavacamten已获得商业批准,有助于改善左心室流出道(LVOT)梯度和症状,并根据风险评估和缓解策略(REMS)计划降低室间隔减容疗法(SRT)的适用性。我们试图前瞻性地报告在一个多医院三级医疗系统中使用市售马伐康坦的初始实际临床经验:我们研究了首批连续使用 5 毫克马伐康坦的 150 名 oHCM 患者(平均年龄 65 岁,53% 为女性,83% 正在使用贝特类受体阻滞剂,61% 属于纽约心脏协会 [NYHA] III 级),他们通过症状评估、左心室出口梯度超声心动图测量和左心室射血分数 (LVEF) 测量进行剂量滴定。我们测量了基线、4周、8周和12周时NYHA分级、LVEF、左心室出口梯度(静息和Valsalva)的变化:在 261 ± 143 天(31-571 天不等)时,69 例(46%)患者的 NYHA 分级≥1 级,另有 27 例(18%)患者的 NYHA 分级提高≥2 级。平均 Valsalva LVOT 梯度从基线时的 72 ± 43 mmHg 降至 4 周时的 29 ± 31 mmHg、8 周时的 29 ± 28 mmHg 和 12 周时的 30 ± 29 mmHg(p 结论:患者的平均 Valsalva LVOT 梯度从基线时的 72 ± 43 mmHg 降至 4 周时的 29 ± 31 mmHg、8 周时的 29 ± 28 mmHg 和 12 周时的 30 ± 29 mmHg:在一项针对多医院三级医疗转诊中心有症状的 oHCM 患者的实际研究中,我们证明了在 REMS 计划下处方马伐康坦的有效性、安全性和后勤可行性。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
求助全文
约1分钟内获得全文 求助全文
来源期刊
Progress in cardiovascular diseases
Progress in cardiovascular diseases 医学-心血管系统
CiteScore
10.90
自引率
6.60%
发文量
98
审稿时长
7 days
期刊介绍: Progress in Cardiovascular Diseases provides comprehensive coverage of a single topic related to heart and circulatory disorders in each issue. Some issues include special articles, definitive reviews that capture the state of the art in the management of particular clinical problems in cardiology.
×
引用
GB/T 7714-2015
复制
MLA
复制
APA
复制
导出至
BibTeX EndNote RefMan NoteFirst NoteExpress
×
提示
您的信息不完整,为了账户安全,请先补充。
现在去补充
×
提示
您因"违规操作"
具体请查看互助需知
我知道了
×
提示
确定
请完成安全验证×
copy
已复制链接
快去分享给好友吧!
我知道了
右上角分享
点击右上角分享
0
联系我们:info@booksci.cn Book学术提供免费学术资源搜索服务,方便国内外学者检索中英文文献。致力于提供最便捷和优质的服务体验。 Copyright © 2023 布克学术 All rights reserved.
京ICP备2023020795号-1
ghs 京公网安备 11010802042870号
Book学术文献互助
Book学术文献互助群
群 号:481959085
Book学术官方微信