Key result
Etiological treatments like mavacamten and tafamidis shift cardiomyopathy care toward targeting specific pathophysiological pathways.
Why the study?
Advances in diagnostic techniques have enabled cardiomyopathy management to move from a mechanistic or ejection fraction approach to phenotypic and etiological therapies.
This review outlines the evolution of cardiomyopathy treatments towards targeted etiological therapies based on specific pathophysiological pathways and genetic mutations.
Supports shift toward targeted cardiomyopathy therapies; leaves open need for long-term outcome data before routine adoption.
This review proposes to look at the evolution of cardiomyopathy treatments in the light of advances in diagnostic techniques, which have enabled to move from a mechanistic to a phenotypic and then etiological approach. The article goes beyond the ejection fraction approach, and look at new therapies that target the pathophysiological pathways of cardiomyopathies, either by targeting the phenotype, or by targeting the etiology. The evolution of HCM treatments is detailed, culminating in the latest etiological treatments such as mavacamten in sarcomeric HCM, tafamidis in transthyretin cardiac amyloidosis and migalastat in Fabry disease. Myosin stimulators are reviewed in the treatment of DCM, before opening perspectives for gene therapy, which proposes direct treatment of the culprit mutation.
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Lairez et al. (2024) conducted a review in Cardiomyopathies. Etiological treatments (e.g., mavacamten, tafamidis, migalastat) was evaluated. Etiological treatments such as mavacamten, tafamidis, and migalastat represent a shift towards targeting the specific pathophysiological pathways and underlying mutations of cardiomyopathies.
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