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Real-World Assessment of Mavacamten's Impact on Left Ventricular Systolic and Diastolic Functions in Obstructive
Zaid Abood1, M Fuad Jan2, Muddasir Ashraf1
1Aurora Cardiovascular and Thoracic Services, Aurora Sinai/Aurora St. Luke's Medical Centers, Aurora Health Care, Milwaukee, WI.
Insights
Mavacamten significantly improved diastolic function in obstructive hypertrophic cardiomyopathy patients over 24 weeks, with stable global longitudinal strain. This real-world data supports mavacamten
Area of Science:
- Cardiology
- Pharmacology
- Clinical Medicine
Background:
- Limited real-world data exists on mavacamten's effects on diastolic function and global longitudinal strain (GLS) in symptomatic obstructive hypertrophic cardiomyopathy (oHCM).
- Obstructive hypertrophic cardiomyopathy (oHCM) is a significant cause of heart failure, necessitating effective therapeutic strategies.
- Mavacamten represents a novel therapeutic approach targeting the underlying pathophysiology of oHCM.
Purpose of the Study:
- To evaluate the real-world effects of mavacamten on diastolic function and GLS in symptomatic oHCM patients over a 24-week period.
- To assess changes in New York Heart Association (NYHA) functional class and left ventricular outflow tract (LVOT) gradient.
- To monitor safety and tolerability, including arrhythmia burden and major side effects.
Main Methods:
- Retrospective analysis of 61 adult patients with symptomatic oHCM initiating mavacamten treatment.
- Data collected over a 24-week period included electrocardiograms, 72-hour Holter monitoring, and echocardiographic assessments.
- Focus on 45 patients who completed the 24-week treatment duration.
Main Results:
- Significant improvement in diastolic dysfunction: Grade 1 increased from 26.6% to 62.2% (p=0.001), Grade 2 decreased from 66.6% to 35.5% (p=0.006).
- Symptomatic improvement observed: 26.7% improved by 2 NYHA classes, 46.7% by 1 NYHA class. GLS remained stable.
- Effective LVOT gradient reduction: 77.7% of patients achieved ≤30 mmHg at 24 weeks. No significant arrhythmia burden or major side effects reported.
Conclusions:
- Mavacamten treatment led to significant improvements in diastolic function and NYHA class in a real-world oHCM cohort.
- Global longitudinal strain (GLS) remained stable, suggesting preserved systolic function during treatment.
- Mavacamten demonstrated a favorable safety profile and efficacy in reducing LVOT gradients, aligning with clinical trial outcomes.
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.
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