Heart failure & cardiomyopathies
Mavacamten in Patients With Hypertrophic Cardiomyopathy Referred for Septal Reduction: Week 56 Results From the VALOR-HCM Randomized Clinical Trial
Authors' conclusion
Results of this randomized clinical trial showed that in patients with symptomatic obstructive HCM, mavacamten reduced the need for SRT at week 56, with sustained improvements in LVOT gradients and symptoms.
Study context
- Population
- Patients with obstructive hypertrophic cardiomyopathy (HCM) (NYHA class III/IV) referred for septal reduction therapy (SRT), 19 US HCM centres108 qualified for week 56
- Participants
- 112 patients
- Comparison
- Mavacamten vs placebo crossover to mavacamten
- Mavacamtenn = 56
- Placebo crossovern = 52reference
- Primary endpoint
- Proportion undergoing SRT, remaining guideline eligible or unevaluable SRT status
- Follow-up
- Week 56
Methodological detailsdouble-blind, placebo-controlled, multicentre, placebo crossover at 16 weeks, Conducted July 2020 to November 2022
- Double-blind
- Placebo-controlled
- Multicentre
- Placebo crossover at 16 weeks
- Conducted July 2020 to November 2022
Primary result
As reported in the abstract
Further findings
| Outcome | Mean difference (mm Hg) (95% CI) | |
|---|---|---|
| Resting LVOT gradient, change · 56 weeks — Mavacamten vs Placebo crossover | −34.0 (−43.5 to −24.5) | |
| Resting LVOT gradient, change · 56 weeks — Placebo crossover vs Placebo crossover | −33.2 (−41.9 to −24.5) | |
| Valsalva LVOT gradient, change · 56 weeks — Mavacamten vs Placebo crossover | −45.6 (−56.5 to −34.6) | |
| Valsalva LVOT gradient, change · 56 weeks — Placebo crossover vs Placebo crossover | −54.6 (−66.0 to −43.3) | |
| −100−50050Difference, mm Hg (linear scale) |
Improvement in NYHA class ≥1
- Improvement in NYHA class ≥1: 51 of 55 vs 37 of 51
Continued mavacamten long term
- Continued mavacamten long term
- 96 of 108 patients (89%)
Safety
LVEF <50%
- LV ejection fraction (LVEF) <50% — Overall LVEF <50%
- 12 of 108 patients (11.1%; 95% CI, 5.87%-18.60%)
- LVEF <50% — LVEF ≤30%
- 2
- LVEF <50% — Died (LVEF ≤30%)
- 1
- LVEF <50% — Continued treatment after LVEF <50%
- 9 of 12 patients (75%)
Original publication
Mavacamten in Patients With Hypertrophic Cardiomyopathy Referred for Septal Reduction: Week 56 Results From the VALOR-HCM Randomized Clinical Trial. JAMA Cardiol. Published 1 October 2023. doi:10.
Summarised from the published abstract; every number is checked against it