Heart failure & cardiomyopathies
Mavacamten: Real-World Experience From 22 Months of the Risk Evaluation and Mitigation Strategy (REMS) Program
Authors' conclusion
The need for temporary interruption for LVEF <50% was low, including for patients on therapy ≥1 year, with even fewer LVEF reductions associated with heart failure requiring hospitalization.
Study context
- Population
- Patients with symptomatic NYHA class II–III obstructive hypertrophic cardiomyopathy treated with mavacamten under the US REMS program, April 28, 2022 to February 27, 20245,573 with Patient Status Forms
- Participants
- 6,299 patients
- Outcome
- LVEF <50% and HF requiring hospitalization (patients with submitted Patient Status Forms, n = 5573)
Methodological detailsREMS program database, real-world, Data on provider and pharmacy certification, patient monitoring (Patient Status Forms, partly echocardiogram-based) and drug-interaction screening before each dispense
- REMS program database
- Real-world
- Data on provider and pharmacy certification, patient monitoring (Patient Status Forms, partly echocardiogram-based) and drug-interaction screening before each dispense
Main finding
As reported in the abstract
Developed left ventricular ejection fraction (LVEF) <50%
256
4.6%
HF requiring hospitalization
71
1.3%
Observational data: associations, not effects.
Further findings
LVEF <50% and HF requiring hospitalization, patients with ≥1 year of treatment (n = 1929)
- LVEF reduction to <50%
- 78 (4.0%)
- LVEF <50% and HF requiring hospitalization, later resumed treatment
- 4 (0.2%)
Status forms (echocardiogram assessments, n = 29 111)
- LVEF <50% reported
- 276 (0.9%)
- HF requiring hospitalization reported
- 86 (0.3%)
Remained at 5 or 10 mg/d (initiated on 5 mg/d, treated ≥6 months, n = 3228)
- Remained at 5 or 10 mg/d
- 2391 (74.1%)
Post-Valsalva LV outflow tract gradient <30 mm Hg · 3 and 6 months
- At 3 months
- 57.2%
- At 6 months
- 70.3%
Original publication
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