Heart failure & cardiomyopathies

Real-World Candidacy to Mavacamten in a Contemporary Hypertrophic Obstructive Cardiomyopathy Population

Eur J Heart FailPublished Cross-sectional study

Authors' conclusion

Real-world HOCM patients differ from the EXPLORER-HCM population for their older age, lower LVEF and larger atrial volume, potentially reflecting a more advanced stage of the disease. About half of real-world HOCM patients were found eligible to mavacamten.

Study context

Population
Hypertrophic obstructive cardiomyopathy (HOCM) patients with significant left ventricular outflow tract obstruction (LVOTO) (gradient ≥30 mmHg at rest or ≥50 mmHg after Valsalva or exercise), 25 Italian HCM outpatient clinics662 with persistent LVOTO
Participants
1,706 patients
Exposure
Real-world HOCM patients with persistent LVOTO vs EXPLORER-HCM trial population
  • Real-world HOCMn = 662
Outcome
Eligibility to mavacamten (EXPLORER-HCM enrolment criteria and European guidelines)
Methodological detailsmulticentre, 25 Italian HCM outpatient clinics
  • Multicentre
  • 25 Italian HCM outpatient clinics

Main finding

As reported in the abstract

Eligibility to mavacamten (patients with persistent LVOTO)

Eligible for enrolment in EXPLORER-HCM

324

48.9%

Eligible for mavacamten per European guidelines

339

51.2%

Observational data: associations, not effects.

Further findings

Age · older in real-world HOCM

P = 0.02

BMI · lower in real-world HOCM

P < 0.0001

History of AF · more frequent in real-world HOCM

P = 0.027

Persistent LVOTO despite therapy

LVOTO resolved by therapy
1044 (61.2%) of 1706
Persistent LVOTO
662 (38.8%)

Left ventricular ejection fraction (LVEF) (echocardiography), real-world HOCM vs EXPLORER-HCM

Lower LVEF in real-world HOCM vs EXPLORER-HCM
66 ± 7% vs. 74 ± 6%, p < 0.0001

Resting LVOT gradient (echocardiography), real-world HOCM vs EXPLORER-HCM

Higher resting LVOT gradient in real-world HOCM vs EXPLORER-HCM
60 ± 27 vs. 52 ± 29 mmHg, p = 0.003

Left atrial volume index (echocardiography), real-world HOCM vs EXPLORER-HCM

Larger LA volume index in real-world HOCM vs EXPLORER-HCM
49 ± 16 vs. 40 ± 12 ml/m² , p < 0.0001

Original publication

Real-World Candidacy to Mavacamten in a Contemporary Hypertrophic Obstructive Cardiomyopathy Population. Eur J Heart Fail. Published 2 January 2024. doi:10.1002/ejhf.3120

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