Efficacy of aficamten in obstructive hypertrophic cardiomyopathy: A systematic review and meta-analysis

IF 1.3 Q3 CARDIAC & CARDIOVASCULAR SYSTEMS
Muhammad Nabeel Saddique , Hamza Irfan , Saad Ashraf , Maria Qadri , Hammad Javaid , Sana Iftikhar , Muhammad Zain ul Haq , Laveeza Fatima , Qasim Mehmood , Anurag Jha , Saad Masood , Muhammad Safiullah , Muhammad Imaz Bhatti , Sheena Shamoon , Javed Iqbal
{"title":"Efficacy of aficamten in obstructive hypertrophic cardiomyopathy: A systematic review and meta-analysis","authors":"Muhammad Nabeel Saddique ,&nbsp;Hamza Irfan ,&nbsp;Saad Ashraf ,&nbsp;Maria Qadri ,&nbsp;Hammad Javaid ,&nbsp;Sana Iftikhar ,&nbsp;Muhammad Zain ul Haq ,&nbsp;Laveeza Fatima ,&nbsp;Qasim Mehmood ,&nbsp;Anurag Jha ,&nbsp;Saad Masood ,&nbsp;Muhammad Safiullah ,&nbsp;Muhammad Imaz Bhatti ,&nbsp;Sheena Shamoon ,&nbsp;Javed Iqbal","doi":"10.1016/j.ahjo.2025.100535","DOIUrl":null,"url":null,"abstract":"<div><h3>Background</h3><div>Obstructive hypertrophic cardiomyopathy (oHCM), a morbid hereditary condition, is characterized by asymmetrical intraventricular septum enlargement, obstructing blood flow from the left ventricle outflow tract (LVOT) and lowering cardiac output. Aficamten, a novel selective, oral myosin inhibitor, has been suggested to reduce myocardial hypercontractility and decrease LVOT gradient in oHCM.</div></div><div><h3>Methods</h3><div>A comprehensive search was conducted through PubMed, Embase, Scopus, and Cochrane databases for relevant literature from inception up to May 2024. Six studies focusing on efficacy and safety aficamten was included. Pooled outcome estimates were reported as mean difference (MD) and 95 % CI using random effect model. Statistical heterogeneity was assessed using I<sup>2</sup> and X<sup>2</sup> statistics.</div></div><div><h3>Results</h3><div>We found a significant change of −143.23 (pg/ml) NT-proBNP [95 % CI −564.8 to 278.4, I<sup>2</sup> = 97.65 %, <em>P</em> &lt; 0.001], −50.9 mmHg Valsalva LVOT gradient [95 % CI −55.2 to −46.6, I<sup>2</sup> = 0 %, <em>P</em> = 0.44], −38.5 mmHg resting LVOT gradient [95 % CI −49.9 to −27.6, I<sup>2</sup> = 0 %, <em>P</em> = 0.64], −5.98 % mean LVEF [95 % CI −9.4 to −2.6, I<sup>2</sup> = 64.18 %, <em>P</em> = 0.06] and −2.32 (ng/dl) Hs-Troponin I [95 % CI −7.55 to −2.91, I<sup>2</sup> = 0 %, <em>P</em> = 0.97] from the baseline. We found significant 64.9 % ≥ 1 NYHA class improvement [95 % CI 45.8 %–84.1 %, I<sup>2</sup> = 90.6 %, <em>P</em> &lt; 0.001] in aficamten. There was ‘low’ overall risk of bias in included studies.</div></div><div><h3>Conclusion</h3><div>We found that aficamten significantly reduced myocardial stress surrogates and functional disability parameters. The small sample sizes, diverse study designs and single-arm analysis limit our findings. More robust trials with larger sample sizes are required to establish conclusive evidence.</div></div>","PeriodicalId":72158,"journal":{"name":"American heart journal plus : cardiology research and practice","volume":"53 ","pages":"Article 100535"},"PeriodicalIF":1.3000,"publicationDate":"2025-03-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"American heart journal plus : cardiology research and practice","FirstCategoryId":"1085","ListUrlMain":"https://www.sciencedirect.com/science/article/pii/S2666602225000382","RegionNum":0,"RegionCategory":null,"ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q3","JCRName":"CARDIAC & CARDIOVASCULAR SYSTEMS","Score":null,"Total":0}
引用次数: 0

Abstract

Background

Obstructive hypertrophic cardiomyopathy (oHCM), a morbid hereditary condition, is characterized by asymmetrical intraventricular septum enlargement, obstructing blood flow from the left ventricle outflow tract (LVOT) and lowering cardiac output. Aficamten, a novel selective, oral myosin inhibitor, has been suggested to reduce myocardial hypercontractility and decrease LVOT gradient in oHCM.

Methods

A comprehensive search was conducted through PubMed, Embase, Scopus, and Cochrane databases for relevant literature from inception up to May 2024. Six studies focusing on efficacy and safety aficamten was included. Pooled outcome estimates were reported as mean difference (MD) and 95 % CI using random effect model. Statistical heterogeneity was assessed using I2 and X2 statistics.

Results

We found a significant change of −143.23 (pg/ml) NT-proBNP [95 % CI −564.8 to 278.4, I2 = 97.65 %, P < 0.001], −50.9 mmHg Valsalva LVOT gradient [95 % CI −55.2 to −46.6, I2 = 0 %, P = 0.44], −38.5 mmHg resting LVOT gradient [95 % CI −49.9 to −27.6, I2 = 0 %, P = 0.64], −5.98 % mean LVEF [95 % CI −9.4 to −2.6, I2 = 64.18 %, P = 0.06] and −2.32 (ng/dl) Hs-Troponin I [95 % CI −7.55 to −2.91, I2 = 0 %, P = 0.97] from the baseline. We found significant 64.9 % ≥ 1 NYHA class improvement [95 % CI 45.8 %–84.1 %, I2 = 90.6 %, P < 0.001] in aficamten. There was ‘low’ overall risk of bias in included studies.

Conclusion

We found that aficamten significantly reduced myocardial stress surrogates and functional disability parameters. The small sample sizes, diverse study designs and single-arm analysis limit our findings. More robust trials with larger sample sizes are required to establish conclusive evidence.
阿非曲坦治疗梗阻性肥厚性心肌病的疗效:一项系统回顾和荟萃分析
梗阻性肥厚性心肌病(oHCM)是一种病态的遗传性疾病,其特征是室间隔不对称扩大,阻碍左心室流出道(LVOT)的血流和心输出量降低。Aficamten是一种新型的选择性口服肌球蛋白抑制剂,被认为可以降低oHCM的心肌过度收缩性和LVOT梯度。方法综合检索PubMed、Embase、Scopus、Cochrane等数据库自成立至2024年5月的相关文献。纳入了6项关注afamten疗效和安全性的研究。合并结果估计报告为平均差异(MD)和95% CI使用随机效应模型。采用I2和X2统计量评估统计异质性。结果NT-proBNP变化为- 143.23 (pg/ml), 95% CI为- 564.8 ~ 278.4,I2 = 97.65%, P <;0.001],−50.9 mmHg并发LVOT梯度(95% CI 55.2−−46.6,I2 = 0%, P = 0.44),−38.5 mmHg休息LVOT梯度(95% CI 49.9−−27.6,I2 = 0%, P = 0.64),意味着LVEF−5.98% (95% CI 9.4−−2.6,I2 = 64.18%, P = 0.06)和−2.32 (ng / dl) Hs-Troponin我[95% CI 7.55−−2.91,I2 = 0%, P = 0.97)的基线。我们发现64.9%≥1 NYHA分级改善[95% CI 45.8% - 84.1%, I2 = 90.6%, P <;0.001]在非洲。纳入研究的总体偏倚风险为“低”。结论aficamten可显著降低心肌应激指标和功能失能指标。小样本量、多样化的研究设计和单臂分析限制了我们的发现。需要更大样本量的更可靠的试验来建立结论性证据。
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
CiteScore
1.60
自引率
0.00%
发文量
0
审稿时长
59 days
×
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