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玛伐卡坦对梗阻性肥厚型心肌病左心室收缩和舒张功能影响的真实世界评估:一项为期1年的单中心观察性研究。

Real-World Assessment of Mavacamten's Impact on Left Ventricular Systolic and Diastolic Functions in Obstructive Hypertrophic Cardiomyopathy: A 1-Year Single-Center Observational Study.

作者信息

Abood Zaid, Jan M Fuad, Ashraf Muddasir, Hundal Prabhjot, Howard Lauren, Sanders Heather, Misicka Amanda, Ghafoor Asad, Jahangir Arshad, Galazka Patrycja, Tajik A Jamil

机构信息

Aurora Cardiovascular and Thoracic Services, Aurora Sinai/Aurora St. Luke's Medical Centers, Aurora Health Care, Milwaukee, WI.

Aurora Cardiovascular and Thoracic Services, Aurora Sinai/Aurora St. Luke's Medical Centers, Aurora Health Care, Milwaukee, WI; Division of Cardiovascular Medicine, University of Wisconsin School of Medicine and Public Health, Milwaukee Clinical Campus, Milwaukee, WI.

出版信息

Am J Cardiol. 2025 May 1;242:68-74. doi: 10.1016/j.amjcard.2025.01.032. Epub 2025 Jan 31.

DOI:10.1016/j.amjcard.2025.01.032
PMID:39894330
Abstract

Real-world data on the effects of mavacamten on diastology and global longitudinal strain (GLS) in symptomatic patients with obstructive hypertrophic cardiomyopathy (oHCM) are limited. We share our experience with mavacamten over a 24-week period at an HCM Center of Excellence. Sixty-one adults with symptomatic oHCM who started on mavacamten between March 2023 and February 2024 were retrospectively identified. All patients had an electrocardiogram performed at each clinic visit, and 72-hour Holter monitoring was performed at 12- and 24-week visits. The mean age was 57.2 ± 14.5 years; 32 (51.6%) patients were female. Of the 61 patients, 45 completed a 24-week period and were the main subject of this study. After 6 months of treatment, the proportion of patients in Grade 1 diastolic dysfunction increased from 26.6% to 62.2%, p = 0.001, and the proportion in Grade 2 diastolic dysfunction decreased from 66.6% to 35.5%, p = 0.006; 26.7% (n = 12/45) of patients improved by 2 New York Heart Association functional classes and 46.7% (n = 21/45) by 1. GLS remained stable over time. At week 24, 35 of 45 patients (77.7%) had a left ventricular outflow tract gradient ≤30 mmHg. No arrhythmia burden or major side effects were reported. Left ventricular ejection fraction remained above 55% in all but 1 patient, who recovered within a month. In conclusion, our experience of significantly improved diastology and stable GLS after mavacamten treatment aligns with trial outcomes. Longitudinal investigations are needed to further assess the long-term impacts.

摘要

关于mavacamten对有症状的梗阻性肥厚型心肌病(oHCM)患者舒张功能和整体纵向应变(GLS)影响的真实世界数据有限。我们分享在一个肥厚型心肌病卓越中心使用mavacamten 24周期间的经验。回顾性确定了61例在2023年3月至2024年2月期间开始使用mavacamten的有症状oHCM成年患者。所有患者在每次门诊就诊时均进行心电图检查,并在第12周和第24周就诊时进行72小时动态心电图监测。平均年龄为57.2±14.5岁;32例(51.6%)患者为女性。61例患者中,45例完成了24周疗程,是本研究的主要对象。治疗6个月后,1级舒张功能障碍患者的比例从26.6%增加到62.2%,p = 0.001,2级舒张功能障碍患者的比例从66.6%降至35.5%,p = 0.006;26.7%(n = 12/45)的患者纽约心脏协会功能分级改善2级,46.7%(n = 21/45)改善1级。GLS随时间保持稳定。在第24周时,45例患者中有35例(77.7%)左心室流出道压力阶差≤30 mmHg。未报告心律失常负担或重大副作用。除1例患者外,所有患者的左心室射血分数均保持在55%以上,该例患者在1个月内恢复。总之,我们关于mavacamten治疗后舒张功能显著改善和GLS稳定的经验与试验结果一致。需要进行纵向研究以进一步评估长期影响。

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