Novel Pharmacotherapy for Hypertrophic Cardiomyopathy

Key points

Current guideline-recommended medical therapy

The main goal of pharmacotherapy is to treat symptoms due to HCM. In patients with obstructive HCM, both beta-blockade and non-dihydropyridine calcium-channel blockade have been reported to decrease the LVOT gradient and improve symptoms, especially on exertion. Of note, the observation that beta and calcium-channel blockers for prevention of disease progression in asymptomatic patients has not been well established is specifically mentioned in the US guidelines.¹ Disopyramide is a useful

Potential applications of clinically available medications for disease modulation in hypertrophic cardiomyopathy

Several medications already approved for other indications have demonstrated the ability to modulate disease phenotype and/or the natural progression of disease in HCM animal models. Corresponding studies in humans have for the most part not demonstrated such strong findings, although most of these studies were small and exploratory in nature and some remain ongoing. Compounds for which ample preclinical and clinical data have been published are reviewed later, and agents with more limited data

Summary: current state of affairs and future directions

In summary, a variety of clinically available medications are in current use for the treatment of obstructive and nonobstructive HCM. Not all patients benefit from current therapy, and some progress to more advanced disease states of severe heart failure and other complications. Beyond guideline-recommended medications (beta-blockers, calcium-channel blockers, disopyramide) for symptomatic patients, several other medications have been studied with mixed results. Some have shown promise for