# Rethinking Childhood-Onset Hypertrophic Cardiomyopathy: A Review of Molecular Mechanisms and Unique Therapy Considerations

## TL;DR

Childhood-onset hypertrophic cardiomyopathy requires tailored treatment strategies due to unique molecular and clinical features that differ from adult-onset cases.

## Contribution

This review highlights the need for age-specific research and treatment approaches for childhood-onset HCM.

## Key findings

- Current treatment strategies for childhood-onset HCM are largely extrapolated from adult guidelines and lack evidence for efficacy in children.
- Molecular genetics has revealed age-specific disease modifiers and genotype-phenotype correlations in childhood-onset HCM.
- Emerging gene-targeted therapies and precision medicine show potential but require further investigation for pediatric use.

## Abstract

Childhood-onset hypertrophic cardiomyopathy (HCM) is a cardiac disorder presenting unique diagnostic and therapeutic challenges in children that require tailored clinical attention compared to HCM arising at other life stages. Despite this, current treatment strategies specific to childhood-onset HCM are lacking and are predominantly extrapolated from adult-specific treatment guidelines. This review explores the molecular basis, clinical implications, and management strategies specific to childhood-onset HCM. Advances in molecular genetics have elucidated diverse pathogenic pathways and genotype-phenotype correlations, revealing age-specific disease modifiers distinct from adult-onset forms. Current management includes pharmacologic, surgical, and device-based interventions, tailored to individualized needs. However, there is a lack of evidence for the efficacy and safety profiles of these treatments in children, meaning children may be receiving sub-optimal care. Emerging approaches, such as gene-targeted therapies and precision medicine frameworks, show promise, but require further investigation. Enhancing early diagnosis and personalized care is crucial for improving outcomes and reducing long-term disease burden in affected children. This review underscores the necessity for specific research to refine risk stratification and treatment paradigms for childhood-onset HCM.