[1]焦哲豪 吴铿.Aficamten治疗肥厚型心肌病研究进展[J].心血管病学进展,2024,(7):585.[doi:10.16806/j.cnki.issn.1004-3934.2024.07.003]
 JIAO Zhehao,WU Keng.Aficamten in Treatment of H ypertrophic Cardiomyopathy[J].Advances in Cardiovascular Diseases,2024,(7):585.[doi:10.16806/j.cnki.issn.1004-3934.2024.07.003]
点击复制

Aficamten治疗肥厚型心肌病研究进展()
分享到:

《心血管病学进展》[ISSN:51-1187/R/CN:1004-3934]

卷:
期数:
2024年7期
页码:
585
栏目:
综述
出版日期:
2024-07-25

文章信息/Info

Title:
Aficamten in Treatment of H ypertrophic Cardiomyopathy
作者:
焦哲豪 吴铿
(广东医科大学附属东莞第一医院,广东 东莞 523000 )
Author(s):
JIAO ZhehaoWU Keng
(The First Dongguan Affiliated Hospital of Guangdong Medical University,Dongguan 523000,Guangdong,China)
关键词:
Aficamten肥厚型心肌病肌球蛋白
Keywords:
AficamtenHypertrophic cardiomyopathyMyosin
DOI:
10.16806/j.cnki.issn.1004-3934.2024.07.003
摘要:
aficamten是一种新型的心肌肌球蛋白抑制剂,可通过降低肥厚型心肌病患者的心肌收缩能力以及改善其舒张期功能,从而安全地降低左心室流出道压差,进而改善肥厚型心肌病患者的心力衰竭症状。2022年,aficamten获得了美国食品药品监督管理局和国家医疗产品管理局的突破性治疗称号,与上一代心肌肌球蛋白抑制剂mavacamten相比,aficamten具有更短的的血浆半衰期和更少的药物相互作用。现基于已完成和正在进行的临床试验数据,对aficamten治疗肥厚型心肌病的研究进展进行综述。
Abstract:
Aficamten is a novel myocardial myosin inhibitor that safely reduces left ventricular outflow tract pressure by reducing cardiac contractility and improving diastolic function in patients with hypertrophic cardiomyopathy,thereby improving symptoms of heart failure in patients with hypertrophic cardiomyopathy. In 2022,aficamten received Breakthrough Treatment Designation from U.S. Food and Drug Administration and National Medical Products Administration for having a shorter plasma half-life and fewer drug interactions than the previous generation myocardial myosin inhibitor mavacamten. Based on data from completed and ongoing clinical trials,this paper reviews the progress of aficamten in the treatment of hypertrophic cardiomyopathy.

参考文献/References:

[1]. Chuang C,Collibee S,Ashcraft L,et al. Discovery of aficamten(CK-274),a next-generation cardiac myosin inhibitor for the treatment of hypertrophic cardiomyopathy[J]. J Med Chem,2021,64(19):14142-14152.
[2]. Semsarian C,Ingles J,Maron MS,et al. New perspectives on the prevalence of hypertrophic cardiomyopathy[J]. J Am Coll Cardiol,2015,65(12):1249-1254.
[3]. Geisterfer-Lowrance AA,Kass S,Tanigawa G,et al. A molecular basis for familial hypertrophic cardiomyopathy:a beta cardiac myosin heavy chain gene missense mutation[J]. Cell,1990,62(5):999-1006.
[4]. Spudich JA. Three perspectives on the molecular basis of hypercontractility caused by hypertrophic cardiomyopathy mutations[J]. Pflugers Arch,2019,471(5):701-717.
[5]. Sebastian SA,Panthangi V,Singh K,et al. Hypertrophic cardiomyopathy:current treatment and future options[J]. Curr Probl Cardiol,2023,48(4):101552.
[6]. Ommen SR,Mital S,Burke MA,et al. 2020 AHA/ACC Guideline for the diagnosis and treatment of patients with hypertrophic cardiomyopathy:executive summary:a report of the American College of Cardiology/American Heart Association Joint Committee on Clinical Practice Guidelines[J]. J Am Coll Cardiol,2020,76(25):3022-3055.
[7]. Daniels MJ,Fusi L,Semsarian C,et al. Myosin modulation in hypertrophic cardiomyopathy and systolic heart failure:getting inside the engine[J]. Circulation,2021,144(10):759-762.
[8]. Day SM,Tardiff JC,Ostap EM. Myosin modulators:emerging approaches for the treatment of cardiomyopathies and heart failure[J]. J Clin Invest,2022,132(5):e148557.
[9]. Nag S,Gollapudi SK,Del Rio CL,et al. Mavacamten,a precision medicine for hypertrophic cardiomyopathy:from a motor protein to patients[J]. Sci Adv,2023,9(30):eabo7622.
[10]. Malik FI,Robertson LA,Armas DR,et al. A Phase 1 dose-escalation study of the cardiac myosin inhibitor aficamten in healthy participants[J]. JACC Basic Transl Sci,2022,7(8):763-775.
[11]. Maron MS,Masri A,Choudhury L,et al. Phase 2 study of aficamten in patients with obstructive hypertrophic cardiomyopathy[J]. J Am Coll Cardiol,2023,81(1):34-45.
[12]. 美通社.箕星宣布aficamten在中国获得 3 期临床试验申请批[EB/OL].(2022-07-06)[2022-10-30].https://baijiahao. Baidu.com/s?id=1737553375253566846&wfr=spider&for=pc.
[13]. Tsukamoto O. Direct sarcomere modulators are promising new treatments for cardiomyopathies[J]. Int J Mol Sci,2019,21(1):226.
[14]. Desai MY,Owens A,Geske JB,et al. Myosin inhibition in patients with obstructive hypertrophic cardiomyopathy referred for septalreduction therapy[J]. J Am Coll Cardiol,2022,80(2):95-108.
[15]. Keam SJ. Mavacamten:first approval[J]. Drugs,2022,82(10):1127-1135.
[16]. Grillo MP,Erve JCL,Dick R,et al. In vitro and in vivo pharmacokinetic characterization of mavacamten,a first-in-class small molecule allosteric modulator of beta cardiac myosin[J]. Xenobiotica,2019,49(6):718-733.
[17]. Wang A,Spertus JA,Wojdyla DM,et al. Mavacamten for obstructive hypertrophic cardiomyopathy with or without hypertension:post-hoc analysis of the EXPLORER-HCM trial[J]. JACC Heart Fail,2024,12(3):567-579.

相似文献/References:

[1]冯坤,刘春霞,熊峰,等.超声心动图在肥厚型心肌病诊治中的应用进展[J].心血管病学进展,2016,(4):409.[doi:10.16806/j.cnki.issn.1004-3934.2016.04.021]
 FENG Kun,LIU Chunxia,XIONG Feng,et al.Progress in Application of Echocardiography in Diagnosis and Treatment of Hypertrophic Cardiomyopathy[J].Advances in Cardiovascular Diseases,2016,(7):409.[doi:10.16806/j.cnki.issn.1004-3934.2016.04.021]
[2]黄淮滨 刘甲兴.肥厚型心肌病治疗新靶点——钙脱敏治疗[J].心血管病学进展,2019,(7):1047.[doi:10.16806/j.cnki.issn.1004-3934.2019.07.021]
 HUANG HuaibinLIU Jiaxing.A Novel Target for Therapy in Hypertrophic Cardiomyopathy: Ca2+ desensitizer[J].Advances in Cardiovascular Diseases,2019,(7):1047.[doi:10.16806/j.cnki.issn.1004-3934.2019.07.021]
[3]马秀英 贾锋鹏.肥厚型心肌病并发心房颤动的危险因素研究进展[J].心血管病学进展,2020,(12):1243.[doi:10.16806/j.cnki.issn.1004-3934.2020.12.005]
 MA XiuyingJIA Fengpeng.Update about the Risk Factors of Hypertrophic Cardiomyopathy Complicated with Atrial Fibrillation[J].Advances in Cardiovascular Diseases,2020,(7):1243.[doi:10.16806/j.cnki.issn.1004-3934.2020.12.005]
[4]邓清文,何森.肥厚型心肌病合并心房颤动的研究进展[J].心血管病学进展,2021,(4):27.[doi:10.16806/j.cnki.issn.1004-3934.2021.04.010]
 DENG Qingwen,HE Sen.Hypertrophic Cardiomyopathy Complicated with Atrial Fibrillation[J].Advances in Cardiovascular Diseases,2021,(7):27.[doi:10.16806/j.cnki.issn.1004-3934.2021.04.010]
[5]尚依一 刘罗 庞明杰 张艳.基因检测在肥厚型心肌病中的应用进展[J].心血管病学进展,2021,(6):512.[doi:10.16806/j.cnki.issn.1004-3934.2021.06.009]
 SHANG Yiyi,LIU Luo,PANG Mingjie,et al.Gene Detection for Hypertrophic Cardiomyopathy[J].Advances in Cardiovascular Diseases,2021,(7):512.[doi:10.16806/j.cnki.issn.1004-3934.2021.06.009]
[6]王娟 杨艳敏.肥厚型心肌病发生缺血性卒中的危险因素和管理策略研究进展[J].心血管病学进展,2021,(7):582.[doi:10.16806/j.cnki.issn.1004-3934.2021.07.002]
 WANG Juan,YANG Yanmin.Risk Factors and Management Strategies of Ischemic Stroke in Hypertrophic Cardiomyopathy[J].Advances in Cardiovascular Diseases,2021,(7):582.[doi:10.16806/j.cnki.issn.1004-3934.2021.07.002]
[7]武志刚 孙佳莉 王巍.基于心脏磁共振成像的肥厚型心肌病患者早期峰值充盈率与舒张功能相关性的研究[J].心血管病学进展,2021,(7):658.[doi:10.16806/j.cnki.issn.1004-3934.2021.07.020]
 WU Zhigang,SUN Jiali,WANG Wei.Correlation Between Early Peak Filling Rate and Diastolic Function in Patients with Hypertrophic Cardiomyopathy Based on Cardiac Magnetic Resonance Imaging[J].Advances in Cardiovascular Diseases,2021,(7):658.[doi:10.16806/j.cnki.issn.1004-3934.2021.07.020]
[8]蓝庆肃 武锋超 马兰虎 韩冰 徐金 彭瑜 张钲.Mavacamten治疗肥厚型心肌病的研究进展[J].心血管病学进展,2021,(11):1028.[doi:10.16806/j.cnki.issn.1004-3934.2021.11.000]
 LAN Qingsu,WU Fengchao,MA Lanhu,et al.Mavacamten in Treatment of Hypertrophic Cardiomyopathy[J].Advances in Cardiovascular Diseases,2021,(7):1028.[doi:10.16806/j.cnki.issn.1004-3934.2021.11.000]
[9]李传伟,罗浩,曾春雨.肥厚型心肌病室间隔消减策略的研究进展[J].心血管病学进展,2022,(1):7.[doi:10.16806/j.cnki.issn.1004-3934.2022.01.002]
 LI Chuanwei,LUO Hao,ZENG Chunyu.Septal Reduction Therapy of Hypertrophic Cardiomyopathy[J].Advances in Cardiovascular Diseases,2022,(7):7.[doi:10.16806/j.cnki.issn.1004-3934.2022.01.002]
[10]李珂 赵海娟 赵耀 黄松群 郭志福.肥厚型心肌病合并心房颤动的导管消融[J].心血管病学进展,2022,(5):399.[doi:10.16806/j.cnki.issn.1004-3934.2022.05.004]
 LI Ke,ZHAO Haijuan,ZHAO Yao,et al.Catheter Ablation of Hypertrophic Cardiomyopathy with Atrial Fibrillation[J].Advances in Cardiovascular Diseases,2022,(7):399.[doi:10.16806/j.cnki.issn.1004-3934.2022.05.004]

更新日期/Last Update: 2024-08-09