Mavacamten therapy managed in a pharmacist-led clinic significantly reduced Valsalva LVOT gradient from 83 mmHg to 50 mmHg (p<0.05) and improved NYHA class at 12 weeks.
Observational (n=16)
No
Does mavacamten managed in a pharmacist-led clinic improve symptoms and hemodynamics in patients with symptomatic obstructive hypertrophic cardiomyopathy?
A pharmacist-led clinic model for managing mavacamten therapy in oHCM patients is feasible, safe, and associated with significant early improvements in symptoms, quality of life, and hemodynamics.
Absolute Event Rate: 50% vs 83%
p-value: p=<0.05
Abstract Background The Inherited Cardiac Conditions (ICC) pharmacist-led clinic was established to manage patients with symptomatic obstructive hypertrophic cardiomyopathy (oHCM) eligible for cardiac myosin inhibitor therapy. Mavacamten requires pharmacogenomic testing, drug-drug interaction assessment, and echocardiography-guided dose adjustments. Purpose The aim of this project was to evaluate the feasibility of a novel integrated one-stop pharmacist-led clinic and to report safety and efficacy of treatment with mavacamten in this setting. Methods A one-stop clinic was established in September 2025, delivered by a sonographer, a clinical nurse specialist, and a pharmacist prescriber (Figure 1). Referrals are accepted from consultant cardiologists with cases discussed at a multidisciplinary team meeting. Each appointment includes a clinical evaluation with a nurse and pharmacist and the following investigations: echocardiography (with measurement of left ventricular ejection fraction LVEF, left ventricular outflow tract LVOT gradient at rest and with the Valsalva manoeuvre); electrocardiography; blood tests including N-terminal pro-B-type natriuretic peptide (NT-proBNP) and high-sensitivity cardiac troponin I (hs-TnI); functional assessment, including New York Heart Association (NYHA) class and 6-minute walk test; and completion of patient-reported outcome measures, including the KCCQ-12 (Kansas City Cardiomyopathy Questionnaire) and EQ-5D (EuroQol 5-Dimension). Results Of 49 patients referred, 16 have initiated treatment from September 2025-January 2026. Ten patients have completed 12 weeks or more of therapy. Between baseline and week 12, there was an improvement in NYHA Class from 2.7 to 2.2 (p0.05) and EQ-5D-5L health status from 46 to 60 (p0.001). This was accompanied by a reduction in Valsalva LVOT gradient from 83mmHg (± 27mmHg) to 50mmg (± 21mmHg) (p0.05) without a significant change in LVEF. Two patients required a protocol-directed dose reduction due to change in LVOT gradient at week 4, and two required temporary treatment interruption due to a reduction in LVEF. Conclusions Our findings demonstrate that this model is a safe, effective and feasible approach to enabling access to specialist cardiac medicines whilst reducing the need for cardiologist-led clinics. Pharmacists are experts in the therapeutic use of medicines, and data support the effectiveness of pharmacist prescribing in cardiology. Treatment was associated with early and sustained improvements in symptoms, quality of life, cardiac biomarkers, and haemodynamics, without adverse effects on left ventricular systolic function. Our early clinical experience with mavacamten supports the therapy’s favourable clinical and haemodynamic profile.Workflow at the clinic
Nowak et al. (Wed,) conducted a observational in Symptomatic obstructive hypertrophic cardiomyopathy (oHCM) (n=16). Mavacamten in a pharmacist-led clinic vs. Baseline was evaluated on Valsalva LVOT gradient (p=<0.05). Mavacamten therapy managed in a pharmacist-led clinic significantly reduced Valsalva LVOT gradient from 83 mmHg to 50 mmHg (p<0.05) and improved NYHA class at 12 weeks.