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BACKGROUND Left ventricular (LV) myocardial extracellular volume (ECV), from cardiac magnetic resonance (CMR) T1 mapping, primarily reflects diffuse fibrosis and is a marker of adverse myocardial remodeling in hypertrophic cardiomyopathy (HCM). OBJECTIVES The authors sought to evaluate the prognostic value of ECV beyond established clinical and imaging variables in a large contemporary HCM cohort. METHODS We evaluated 1,050 consecutive patients with HCM and preserved LV ejection fraction (≥50%) who underwent CMR, including quantification of ECV (%) and late gadolinium enhancement (LGE) burden (% LV mass, >6 SDs above remote myocardium), between 2012 and 2021. The primary endpoint was a composite of mortality, appropriate implantable cardioverter-defibrillator (ICD) therapy, or heart transplantation. Statistical methods included multivariable Cox proportional hazards regression, restricted cubic spline analysis, assessment of incremental discrimination using changes in the C-statistic, and net reclassification improvement analysis. RESULTS Median age was 60 years (IQR: 49-68 years) with 44% women. Median follow-up was 5.6 years (IQR: 2.5-7.8 years). The primary endpoint occurred in 124 patients (11.8%), including 111 deaths (10.6%), 12 appropriate ICD discharges (1.1%), and 1 heart transplantation (0.1%). In multivariable Cox regression, older age (HR: 1.05, 95% CI: 1.03-1.06; P < 0.001), NYHA functional class ≥II (HR: 1.96, 95% CI: 1.23-3.11; P = 0.004), diabetes mellitus (HR: 1.89, 95% CI: 1.27-2.81; P = 0.002), and higher ECV (HR: 1.05 per 1% increase, 95% CI: 1.02-1.09; P = 0.001) were independently associated with the primary outcome. Patients with ECV ≥32% had lower event-free survival than those with ECV <32% (adjusted HR: 1.81, 95% CI: 1.24-2.64; P < 0.001). CONCLUSIONS Myocardial ECV was independently associated with long-term adverse outcomes. Multiparametric CMR assessment may improve risk stratification in HCM.
Hypertrophic cardiomyopathy (HCM) is a prototypical inherited cardiomyopathy with well-defined sarcomeric genetic underpinnings that make it an attractive target for molecular therapy. We review recent advances in gene-based approaches for HCM, including adeno-associated virus-mediate gene replacement, allele-specific silencing, and emerging gene editing strategies, and highlight the first demonstrations of in vivo target engagement and early clinical translation. Early-phase studies suggest that restoration of sarcomeric biology can favorably impact molecular and structural disease features. We further discuss key challenges related to immune responses, delivery efficiency, response durability, and patient selection that will shape the next phase of development. Together, these developments establish HCM as one of the important early models for cardiac gene therapy and highlight both the promise and complexity of translating genetic insight into durable clinical benefit.
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