KLHL24-Associated Hypertrophic Cardiomyopathy: When Genotype Outpaces Phenotype

KLHL24相关肥厚型心肌病:当基因型超越表型时

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Abstract

BACKGROUND: KLHL24 (Kelch-like family member 24)-associated hypertrophic cardiomyopathy (HCM) is a recently recognized genetic disorder characterized by early presentation and a disproportionate risk of malignant ventricular arrhythmias due to impaired cytoskeletal protein turnover. CASE SUMMARY: A 16-year-old asymptomatic young man without a family history of HCM was evaluated after detection of a cardiac murmur. Imaging revealed asymmetric septal hypertrophy with minimal fibrosis and preserved systolic function. Genetic testing identified compound heterozygous truncating KLHL24 variants. Despite low conventional risk markers, genotype-directed assessment indicated high arrhythmic risk, prompting prophylactic implantable cardioverter-defibrillator (ICD) placement. The patient remained clinically stable at follow-up. DISCUSSION: This case illustrates the limitations of phenotype-based risk stratification and highlights the importance of genotype-informed decision-making in arrhythmogenic cardiomyopathies. TAKE-HOME MESSAGES: KLHL24-associated HCM is uniquely arrhythmogenic, with malignant ventricular arrhythmias potentially preceding structural severity. Early genetic testing enables genotype-guided preventive strategies, including timely prophylactic ICD implantation, even in apparently low-risk phenotypes.

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