Purpose of Review <p>Hypertrophic cardiomyopathy (HCM) is the most common inherited cardiac disease, characterized by unexplained left ventricular hypertrophy and a highly variable clinical course. There have been remarkable advances in diagnostic and therapeutic strategies in recent years. As novel disease-modifying therapies emerge, the identification and validation of robust biomarkers are of paramount importance.</p> Recent Findings <p>Multiple categories of biomarkers, including circulating, imaging, and molecular biomarkers, have shown correlations with disease severity, but their use as surrogate endpoints in clinical trials remain in its early stages. The limitations of biomarkers for clinical application are due to several factors, such as disease heterogeneity, biomarker nonspecificity, and lack of standardization.</p> Summary <p>This review provides a critical appraisal of recent studies evaluating circulating, imaging, and molecular biomarkers in HCM, particularly in the context of randomized clinical trials. We discuss how these biomarkers can predict disease progression, stratify risk, reflect therapeutic response, and serve as surrogate endpoints. Furthermore, we explore the limitations and future directions of biomarker research to optimize their utility in the precision-guided management of HCM.</p>

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Biomarkers as Clinical Endpoints in Hypertrophic Cardiomyopathy Clinical Trials

  • Sadia Tanami,
  • Ahmad Masri

摘要

Purpose of Review

Hypertrophic cardiomyopathy (HCM) is the most common inherited cardiac disease, characterized by unexplained left ventricular hypertrophy and a highly variable clinical course. There have been remarkable advances in diagnostic and therapeutic strategies in recent years. As novel disease-modifying therapies emerge, the identification and validation of robust biomarkers are of paramount importance.

Recent Findings

Multiple categories of biomarkers, including circulating, imaging, and molecular biomarkers, have shown correlations with disease severity, but their use as surrogate endpoints in clinical trials remain in its early stages. The limitations of biomarkers for clinical application are due to several factors, such as disease heterogeneity, biomarker nonspecificity, and lack of standardization.

Summary

This review provides a critical appraisal of recent studies evaluating circulating, imaging, and molecular biomarkers in HCM, particularly in the context of randomized clinical trials. We discuss how these biomarkers can predict disease progression, stratify risk, reflect therapeutic response, and serve as surrogate endpoints. Furthermore, we explore the limitations and future directions of biomarker research to optimize their utility in the precision-guided management of HCM.