Current and emerging pharmacotherapy for the management of hypertrophic cardiomyopathy

ABSTRACT

Introduction

Hypertrophic cardiomyopathy (HCM) is one of the most common genetic causes of heart disease. Since the initial description of HCM, there have been minimal strides in management options. Obstructive HCM constitutes a larger subset of patients with increased left ventricular outflow tract gradients causing symptoms. Septal reduction therapy (SRT) has been successful, but it is not the answer for all patients and is not disease modifying.

Areas covered

Current guideline recommendations include beta-blockers, calcium channel blockers, or disopyramides for medical management, but there lacks evidence of much benefit with these drugs. In recent years, there has been the emergence of cardiac myosin inhibitors (CMI) which have demonstrated positive results in patients with both obstructive and non-obstructive HCM. In addition to CMIs, other drugs have been investigated as we have learned more about HCM’s pathological mechanisms. Drugs targeting sodium channels and myocardial energetics, as well as repurposed drugs that have demonstrated positive remodeling are being investigated as potential therapeutic targets. Gene therapy is being explored with vast potential for the treatment of HCM.

Expert opinion

The armamentarium of therapeutic options for HCM is continuously increasing with the emergence of CMIs as mainstays of treatment. The future of HCM treatment is promising.

KEYWORDS:

• Cardiac myosin inhibitors
• hypertrophic cardiomyopathy
• left ventricular
• outflow tract gradient
• mavacamten
• aficamten

Article highlights

• It is essential to perform comprehensive diagnostic evaluations for celiac disease before introducing any dietary gluten restrictions.

• Gluten challenge continues to play an important role in situations in which a gluten-free diet has already been initiated before appropriate investigations or when the celiac disease diagnosis is otherwise inconclusive.

• Due to limited scientific evidence, the timing, duration, and amount of gluten used in the challenge should be determined according to individual assessment and joint decision-making.

• It is important for clinicians to recognize that there are still significant limitations in the utilization and interpretation of the gluten challenge.

• Further studies with standardized protocols and outcome measures are warranted to determine best practices in the implementation of the challenge.

• Recent pharmaceutical studies have driven the development of more sensitive immunological methods and other outcome measures that may help to shorten or even eliminate the need for gluten challenges.

Declaration of interest

The authors have no relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript. This includes employment, consultancies, honoraria, stock ownership or options, expert testimony, grants or patents received or pending, or royalties.

Reviewer disclosures

Peer reviewers on this manuscript have no relevant financial or other relationships to disclose.

Additional information

Funding

This paper was not funded.