使用马伐康汀治疗症状性阻塞性肥厚型心肌病时心室应变的序列变化:VALOR-HCM 试验的启示。

IF 6.5 1区 医学 Q1 CARDIAC & CARDIOVASCULAR SYSTEMS
Circulation: Cardiovascular Imaging Pub Date : 2024-09-01 Epub Date: 2024-09-02 DOI:10.1161/CIRCIMAGING.124.017185
Milind Y Desai, Yuichiro Okushi, Andrew Gaballa, Qiuqing Wang, Jeffrey B Geske, Anjali T Owens, Sara Saberi, Andrew Wang, Paul C Cremer, Mark Sherrid, Neal K Lakdawala, Albree Tower-Rader, David Fermin, Srihari S Naidu, Kathy L Lampl, Amy J Sehnert, Steven E Nissen, Zoran B Popovic
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引用次数: 0

摘要

研究背景在症状严重的阻塞性肥厚型心肌病患者中,VALOR-HCM(一项评估马伐康坦在符合室间隔减容治疗条件的症状性阻塞性肥厚型心肌病成人患者中应用情况的研究)证实,马伐康坦可减少室间隔减容治疗的需要,并能持续改善左心室流出道梯度和症状。整体纵向应变(GLS)是衡量区域心肌功能的指标,是更灵敏的收缩功能指标。在 VALOR-HCM 中,我们评估了左心室和右心室应变的序列变化:VALOR-HCM 包括 112 名有症状的梗阻性肥厚型心肌病患者(平均 60 岁;51% 为男性;左心室射血分数 68%)。基线时被分配服用马伐康坦的患者继续服药56周(n=56),被分配服用安慰剂的患者(n=52)从第16周到第56周(40周暴露)过渡到马伐康坦。LV-GLS 和 RV-GLS 评估使用供应商中立的软件进行。使用非缩短的心尖(四腔、三腔和两腔)切面获得 LV-GLS 峰值。RV 聚焦四腔切面用于计算 RV 四腔和游离壁应变。结果:基线时,左心室-GLS、左心室四腔和游离壁应变的平均值分别为-14.7%、-22.2%和-16.8%(均比报告的正常值差)。在所有研究样本中,LV-GLS 从基线到第 56 周显著改善(P=0.02)。12名患者的左心室射血分数出现短暂下降(P=0.64)。自由壁和四腔 RV-GLS 从基线到第 56 周均保持不变(分别为 P=0.62 和 P=0.56):结论:在 VALOR-HCM 中,使用马伐康坦治疗后,左心室-GLS 从基线到第 56 周均有所改善(左心室射血分数降低≤50% 的患者左心室-GLS 无明显恶化),这表明马伐康坦对区域性左心室收缩功能具有良好的长期影响。此外,对左心室收缩功能也没有不利影响:URL:https://www.clinicaltrials.gov;唯一标识符:NCT04349072。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Serial Changes in Ventricular Strain in Symptomatic Obstructive Hypertrophic Cardiomyopathy Treated With Mavacamten: Insights From the VALOR-HCM Trial.

Background: In severely symptomatic patients with obstructive hypertrophic cardiomyopathy, VALOR-HCM (A Study to Evaluate Mavacamten in Adults With Symptomatic Obstructive Hypertrophic Cardiomyopathy Who Are Eligible for Septal Reduction Therapy) demonstrated that mavacamten reduces the need for septal reduction therapy with sustained improvement in left ventricular (LV) outflow tract gradients and symptoms. Global longitudinal strain (GLS), a measure of regional myocardial function, is a more sensitive marker of systolic function. In VALOR-HCM, we assessed serial changes in LV and right ventricular (RV) strain.

Methods: VALOR-HCM included 112 patients with symptomatic obstructive hypertrophic cardiomyopathy (mean, 60 years; 51% male; LV ejection fraction, 68%). Patients assigned to mavacamten at baseline continued the drug for 56 weeks (n=56) and those assigned to placebo (n=52) transitioned to mavacamten from weeks 16 to 56 (40-week exposure). LV-GLS and RV-GLS assessment was performed using a vendor-neutral software. Non-foreshortened apical (4-, 3-, and 2-chamber) views were used to obtain peak LV-GLS. RV focused 4-chamber view was used to calculate RV 4-chamber and free wall strain. A more negative strain value is favorable.

Results: At baseline, the mean LV-GLS, RV 4-chamber, and free wall strain values were -14.7%, -22.2%, and -16.8%, respectively (all worse than reported normal means). In the total study sample, LV-GLS significantly improved from baseline to week 56 (P=0.02). Twelve patients had transient reduction in LV ejection fraction (<50%) requiring temporary drug interruption (including 3 permanent discontinuations). The LV-GLS in this subgroup was worse at baseline versus total study population (-11.4%), with no significant worsening from baseline through week 56 (P=0.64). Both free wall and 4-chamber RV-GLS remained unchanged from baseline to week 56 (P=0.62 and P=0.56, respectively).

Conclusions: In VALOR-HCM, treatment with mavacamten improved LV-GLS from baseline through week 56 (with no significant worsening of LV-GLS in patients with a reduction in LV ejection fraction ≤50%), suggesting a favorable long-term impact on regional LV systolic function. Additionally, there was no detrimental impact on RV systolic function.

Registration: URL: https://www.clinicaltrials.gov; Unique identifier: NCT04349072.

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来源期刊
CiteScore
6.30
自引率
2.70%
发文量
225
审稿时长
6-12 weeks
期刊介绍: Circulation: Cardiovascular Imaging, an American Heart Association journal, publishes high-quality, patient-centric articles focusing on observational studies, clinical trials, and advances in applied (translational) research. The journal features innovative, multimodality approaches to the diagnosis and risk stratification of cardiovascular disease. Modalities covered include echocardiography, cardiac computed tomography, cardiac magnetic resonance imaging and spectroscopy, magnetic resonance angiography, cardiac positron emission tomography, noninvasive assessment of vascular and endothelial function, radionuclide imaging, molecular imaging, and others. Article types considered by Circulation: Cardiovascular Imaging include Original Research, Research Letters, Advances in Cardiovascular Imaging, Clinical Implications of Molecular Imaging Research, How to Use Imaging, Translating Novel Imaging Technologies into Clinical Applications, and Cardiovascular Images.
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