Key result
HCM Risk-Kids poorly predicts SCD in RASopathy HCM, while unexplained syncope and NSVT remain strong predictors.
Why the study?
Sudden cardiac death occurs in RASopathy-associated hypertrophic cardiomyopathy, but its risk factors have not been systematically evaluated.
Does the HCM Risk-Kids model accurately predict sudden cardiac death in children with RASopathy-associated HCM?
Cohort (n=169)
Yes
Does the HCM Risk-Kids model accurately predict sudden cardiac death in children with RASopathy-associated HCM?
Effect estimate: C-index 0.60
The HCM Risk-Kids model may not be appropriate for children with RASopathy-associated HCM, in whom unexplained syncope and NSVT are significant predictors of sudden cardiac death.
HCM Risk-Kids may not reliably stratify SCD risk in RASopathy-associated HCM; challenges generalizability and leaves open subgroup-specific model development.
BACKGROUND: RASopathies account for nearly 20% of cases of childhood hypertrophic cardiomyopathy (HCM). Sudden cardiac death (SCD) occurs in patients with RASopathy-associated HCM, but the risk factors for SCD have not been systematically evaluated. AIM: To validate the HCM Risk-Kids SCD risk prediction model in children with RASopathy-associated HCM and investigate potential specific SCD predictors in this population. METHODS: Validation of HCM Risk-Kids was performed in a retrospective cohort of 169 patients with a RASopathy-associated HCM from 15 international paediatric cardiology centres. Multiple imputation by chained equations was used for missing values related to the HCM Risk-Kids parameters. RESULTS: Eleven patients (6.5%) experienced a SCD or equivalent event at a median age of 12.5 months (IQR 7.7-28.64). The calculated SCD/equivalent event incidence was 0.78 (95% CI 0.43-1.41) per 100 patient years. Six patients (54.54%) with an event were in the low-risk category according to the HCM Risk-Kids model. Harrell's C index was 0.60, with a sensitivity of 9.09%, specificity of 63.92%, positive predictive value of 1.72%, and negative predictive value of 91%; with a poor distinction between the different risk groups. Unexplained syncope (HR 42.17, 95% CI 10.49-169.56, p < 0.001) and non-sustained ventricular tachycardia (HR 5.48, 95% CI 1.58-19.03, p < 0.007) were predictors of SCD on univariate analysis. CONCLUSION: Unexplained syncope and the presence of NSVT emerge as predictors for SCD in children with RASopathy-associated HCM. The HCM Risk-Kids model may not be appropriate to use in this population, but larger multicentre collaborative studies are required to investigate this further.
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Boleti et al. (2023) conducted a cohort in RASopathy-associated hypertrophic cardiomyopathy (n=169). HCM Risk-Kids SCD risk prediction model was evaluated on Sudden cardiac death (SCD) or equivalent event (C-index 0.60). The HCM Risk-Kids model demonstrated poor discrimination for sudden cardiac death in children with RASopathy-associated HCM (C-index 0.60), while unexplained syncope and NSVT were strong predictors.
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