Heart failure & cardiomyopathies
Long-term effect of mavacamten in obstructive hypertrophic cardiomyopathy
Authors' conclusion
Long-term mavacamten treatment resulted in sustained improvements in cardiac function and symptoms in patients with obstructive HCM, with no new safety concerns identified. Transient, reversible reductions in LVEF were observed in a small proportion of patients during long-term follow-up.
Study context
- Population
- Patients with obstructive hypertrophic cardiomyopathy (HCM) who completed EXPLORER-HCM211 (91.3%) still on mavacamten
- Participants
- 231 patients
- Outcome
- Change from baseline to Week 180
- Follow-up
- Week 180
Methodological details5-year, ongoing, Median (range) time on study 166.1 (6.0-228.1) weeks; 185 (80.1%) and 99 (42.9%) completed Week 156 and 180 visits
- 5-year
- Ongoing
- Median (range) time on study 166.1 (6.0-228.1) weeks; 185 (80.1%) and 99 (42.9%) completed Week 156 and 180 visits
Main finding
As reported in the abstract
- Resting LVOT gradient, mean (SD) change
- −40.3 (32.7) mmHg
- Valsalva LVOT gradient, mean (SD) change
- −55.3 (33.7) mmHg
- NT-proBNP, median (IQR) change
- −562 (−1162.5, −209) ng/L
- EQ-5D-5L score, mean (SD) change
- 0.09 (0.17)
- Improved by ≥1 NYHA class
- 74 (77.9%) of 95
Further findings
| Outcome | Values |
|---|---|
| DeathAll considered unrelated to mavacamten | Mavacamten 2.2% |
Mean left ventricular ejection fraction (LVEF)
- Baseline
- 73.9%
- Week 24
- 66.6%
- Week 180
- 63.9%
Death (all considered unrelated to mavacamten)
- Deaths
- Five (2.2%)
Safety
Transient LVEF reduction to <50% with temporary treatment interruption (all recovered LVEF ≥50%) · over 739 patient-years exposure
- Patients with transient LVEF <50% (22 events)
- 20 patients (8.7%)
- Exposure-adjusted incidence
- 2.77/100 patient-years
- All recovered LVEF of ≥50%
Original publication
Long-term effect of mavacamten in obstructive hypertrophic cardiomyopathy. Eur Heart J. Published 1 September 2024. doi:10.
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