Introduction
Hypertrophic Obstructive Cardiomyopathy (HOCM) is an autosomal dominant cardiac condition characterised by left ventricular and septal hypertrophy resulting in dynamic left ventricular outflow obstruction (LVOTO) and consequently decreased exercise capacity, cardiac arrhythmias and sudden cardiac death. Sarcomeric gene mutations leading to increased actin-myosin contractility and hyper dynamic cellular disarray have been identified in the pathophysiology of HOCM. Mavacamten, a first of its kind cardiac myosin ATPase inhibitor has shown promising results in controlling disease progression and improving dynamic LVOTO in patients with significant symptom burden.
Mavacamten is a small molecule inhibitor of cardiac myosin ATPase that reduces the sarcomeric contractility power, hence reducing hypertrophy and fibrosis. In the EXPLORER-HCM trial, which pioneered the research around Mavacamten, the results showed a significant decrease in exercise burden and increase in exercise capacity (p=0.0005) in patients within the Mavacamten group as compared to the placebo group. There was also a significant reduction in post-exercise LVOT gradient in the Mavacamten group (p<0.0001) as well as a decrease in self-reported symptom burden as assessed on two standardized questionnaires.
Aim
The aims of our proposed presentation is to raise awareness for the use of Mavacamten and to recruit potential candidates into an extended arm of the original EXPLORER-HCM trial in Wagga. We have identified potential candidates in Wagga that may benefit from this novel therapy, which we show through our presentation. The awareness on Mavacamten also allows the drug to be made available to patients in rural settings with significant disease burden as an additive to symptom control measures and as a potential alternative to surgical interventions.