Real-World Candidacy to Mavacamten in a Contemporary Hypertrophic Obstructive Cardiomyopathy Population
Key result
Real-world HOCM patients with persistent LVOTO were older and had lower LVEF than the EXPLORER-HCM trial population, with 51.2% found eligible for mavacamten treatment.
Why the study?
Following the EXPLORER-HCM trial, the potential real-world use and eligibility of mavacamten remained to be defined by comparing real-world HOCM patients with trial participants.
Population
662 HOCM patients with persistent LVOTO followed at 25 Italian HCM outpatient clinics
Comparison
Real-world HOCM patients vs EXPLORER-HCM trial population
Design
Multicentre observational study
Authors
Discussion
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Implication
Clinicians should note limited generalizability of EXPLORER-HCM to real-world HOCM; leaves open efficacy and safety questions in older patients with lower LVEF.
Key Points
- To define real-world clinical characteristics and assess mavacamten eligibility in hypertrophic obstructive cardiomyopathy patients compared with the EXPLORER-HCM trial cohort.
- Evaluated 1,706 HOCM patients followed across 25 Italian HCM outpatient clinics to identify 662 patients with persistent left ventricular outflow tract obstruction (gradient ≥30 mmHg at rest or ≥50 mmHg provoked) despite therapy.
- Compared baseline demographic, clinical, and echocardiographic variables against the EXPLORER-HCM trial population and determined eligibility according to trial criteria and European guidelines.
- Compared to the EXPLORER-HCM trial, real-world patients were older (62.1 ± 14.3 vs. 58.5 ± 12.2 years, p = 0.02), had lower BMI (26.8 ± 5.3 vs. 29.7 ± 4.9 kg/m2, p < 0.0001), and had more frequent atrial fibrillation (21.5% vs. 9.8%, p = 0.027).
- Real-world patients presented with lower LVEF (66 ± 7% vs. 74 ± 6%, p < 0.0001), higher resting LVOT gradients (60 ± 27 vs. 52 ± 29 mmHg, p = 0.003), and larger left atrial volume index (49 ± 16 vs. 40 ± 12 ml/m2, p < 0.0001).
- A total of 324 real-world patients (48.9%) were eligible for EXPLORER-HCM trial enrollment, and 339 patients (51.2%) met European guideline criteria for mavacamten treatment.
Study Design
Type
Observational (n=662)
Multicenter
Yes
Structured PICO
P
Population
662 real-world patients with hypertrophic obstructive cardiomyopathy and persistent LVOTO despite therapy, evaluated across 25 Italian outpatient clinics.
E
Exposure
Assessment of eligibility for mavacamten treatment based on EXPLORER-HCM trial criteria and European guidelines
C
Comparator
EXPLORER-HCM trial population
O
Outcome
Proportion of real-world HOCM patients eligible for mavacamten and comparison of clinical and echocardiographic characteristics with the EXPLORER-HCM trial population
Approximately half of real-world HOCM patients with persistent obstruction are eligible for mavacamten, but they represent a more advanced disease stage compared to the pivotal EXPLORER-HCM trial population.
Cite This Study
Bertero et al. (2023) conducted an observational in Hypertrophic Obstructive Cardiomyopathy (HOCM) (n=662). Real-world HOCM with persistent LVOTO vs. EXPLORER-HCM trial population was evaluated on Eligibility for treatment with mavacamten according to European guidelines. Real-world HOCM patients with persistent LVOTO were older and had lower LVEF than the EXPLORER-HCM trial population, with 51.2% found eligible for mavacamten treatment.