Pathophysiology and Treatment of Hypertrophic Cardiomyopathy: New Perspectives

Springer Science and Business Media LLC - Tập 18 - Trang 169-179 - 2021
Mattia Zampieri1, Martina Berteotti1, Cecilia Ferrantini2, Luigi Tassetti1, Martina Gabriele1, Benedetta Tomberli3, Gabriele Castelli1, Francesco Cappelli3, Pierluigi Stefàno4, Niccolò Marchionni2,5, Raffaele Coppini6, Iacopo Olivotto1,2
1Cardiomyopathy Unit, Careggi University Hospital, Florence, Italy
2Department of Experimental and Clinical Medicine, University of Florence, Florence, Italy
3Division of Interventional Structural Cardiology, Cardiothoracovascular Department, Careggi University Hospital, Florence, Italy
4Division of Cardiac Surgery, Careggi University Hospital, Florence, Italy
5Division of General Cardiology, Careggi University Hospital, Florence, Italy
6Department NeuroFarBa, University of Florence, Florence, Italy

Tóm tắt

We provide a state of the art of therapeutic options in hypertrophic cardiomyopathy (HCM), focusing on recent advances in our understanding of the pathophysiology of sarcomeric disease. A wealth of novel information regarding the molecular mechanisms associated with the clinical phenotype and natural history of HCM have been developed over the last two decades. Such advances have only recently led to a number of controlled randomized studies, often limited in size and fortune. Recently, however, the allosteric inhibitors of cardiac myosin adenosine triphosphatase, countering the main pathophysiological abnormality associated with HCM-causing mutations, i.e. hypercontractility, have opened new management perspectives. Mavacamten is the first drug specifically developed for HCM used in a successful phase 3 trial, with the promise to reach symptomatic obstructive patients in the near future. In addition, the fine characterization of cardiomyocyte electrophysiological remodelling has recently highlighted relevant therapeutic targets. Current therapies for HCM focus on late disease manifestations without addressing the intrinsic pathological mechanisms. However, novel evidence-based approaches have opened the way for agents targeting HCM molecular substrates. The impact of these targeted interventions will hopefully alter the natural history of the disease in the near future.

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Tập 18

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