Key result
Preventative treatment strategies may offer greater efficacy than corrective approaches for HCM.
Why the study?
Current drug therapeutics for HCM relieve symptoms but do not prevent or reverse disease progression, and clinical heterogeneity leads to variable patient outcomes.
A preventative therapeutic approach, guided by mutation-specific mechanisms, may be more efficacious than corrective treatments for hypertrophic cardiomyopathy.
May warrant exploring preventative HCM strategies; leaves open prospective validation versus corrective approaches.
Sarcomeric gene mutations are associated with the development of hypertrophic cardiomyopathy (HCM). Current drug therapeutics for HCM patients are effective in relieving symptoms, but do not prevent or reverse disease progression. Moreover, due to heterogeneity in the clinical manifestations of the disease, patients experience variable outcomes in response to therapeutics. Mechanistically, alterations in calcium handling, sarcomeric disorganization, energy metabolism and contractility participate in HCM disease progression. While some similarities exist, each mutation appears to lead to mutation-specific pathophysiology. Furthermore, these alterations may precede or proceed development of the pathology. This review assesses the efficacy of HCM therapeutics from studies performed in animal models of HCM and human clinical trials. Evidence suggests that a preventative rather than corrective therapeutic approach may be more efficacious in the treatment of HCM. In addition, a clear understanding of mutation-specific mechanisms may assist in informing the most effective therapeutic mode of action.
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Solomon et al. (2020) conducted a review in Hypertrophic cardiomyopathy. Preventative therapeutic approaches was evaluated. Evidence from animal models and clinical trials suggests that a preventative rather than corrective therapeutic approach may be more efficacious in the treatment of hypertrophic cardiomyopathy.
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