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Standard-of-Care Medication Withdrawal in Patients With Obstructive Hypertrophic Cardiomyopathy Receiving Aficamten
Ahmad Masri1, Lubna Choudhury2, Roberto Barriales-Villa3
1Oregon Health and Science University, Portland, Oregon, USA.
Journal of the American College of Cardiology
|October 30, 2024
Summary
Standard-of-care (SoC) medication withdrawal was safely achieved in nearly half of obstructive hypertrophic cardiomyopathy patients on aficamten. This allowed for aficamten monotherapy without impacting clinical efficacy or safety outcomes.
Area of Science:
- Cardiology
- Pharmacology
- Clinical Trials
Background:
- Standard-of-care (SoC) medications for obstructive hypertrophic cardiomyopathy (oHCM) lack robust clinical trial evidence and have known side effects.
- Aficamten is an emerging treatment for oHCM.
Purpose of the Study:
- To evaluate the impact of reducing or stopping SoC therapy in patients receiving aficamten.
- To assess the safety and efficacy of aficamten monotherapy after SoC withdrawal.
Main Methods:
- Patients on aficamten underwent protocol-guided downtitration or withdrawal of SoC medications (beta-blockers, calcium-channel blockers, disopyramide).
- Successful withdrawal was defined as ≥50% dose reduction in at least one SoC medication.
- Adverse events and clinical outcomes were monitored post-withdrawal.
Main Results:
- Nearly half (47%) of patients attempted SoC withdrawal, with 92.2% achieving successful reduction or discontinuation.
- 79.2% of patients improved their NYHA functional class by at least one class.
- Aficamten monotherapy was achieved in 45.8% of patients, with no significant adverse events or negative impact on efficacy measures.
Conclusions:
- Downtitration and withdrawal of SoC medications are well-tolerated in oHCM patients treated with aficamten.
- Aficamten monotherapy can be safely achieved, maintaining or improving clinical outcomes.
- This strategy offers a potential alternative to long-term SoC polypharmacy in oHCM.
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