Development of a Novel Risk Prediction Model for Sudden Cardiac Death in Childhood Hypertrophic Cardiomyopathy (HCM Risk-Kids)

Gabrielle Norrish, Tao Ding, Ella Field, Lidia Ziólkowska, Iacopo Olivotto, Giuseppe Limongelli, Aristides Anastasakis, Robert Weintraub, Elena Biagini, Luca Ragni, Terence Prendiville, Sophie Duignan, Karen McLeod, Maria Ilina, Adrián Fernández, Regina Bökenkamp, Anwar Baban, Peter Kubuš, Piers E F Daubeney, Georgia Sarquella-BrugadaSergi Cesar, Chiara Marrone, Vinay Bhole, Constancio Medrano, Orhan Uzun, Elspeth Brown, Ferran Gran, Francisco J Castro, Graham Stuart, Gabriele Vignati, Roberto Barriales-Villa, Luis G Guereta, Satish Adwani, Katie Linter, Tara Bharucha, Pablo Garcia-Pavia, Torsten B Rasmussen, Margherita M Calcagnino, Caroline B Jones, Hans De Wilde, J Toru-Kubo, Tiziana Felice, Jens Mogensen, Sujeev Mathur, Zdenka Reinhardt, Constantinos O'Mahony, Perry M Elliott, Rumana Z Omar, Juan P Kaski

Research output: Contribution to journalArticlepeer-review


Importance: Sudden cardiac death (SCD) is the most common mode of death in childhood hypertrophic cardiomyopathy (HCM), but there is no validated algorithm to identify those at highest risk.

Objective: To develop and validate an SCD risk prediction model that provides individualized risk estimates.

Design, Setting, and Participants: A prognostic model was developed from a retrospective, multicenter, longitudinal cohort study of 1024 consecutively evaluated patients aged 16 years or younger with HCM. The study was conducted from January 1, 1970, to December 31, 2017.

Exposures: The model was developed using preselected predictor variables (unexplained syncope, maximal left-ventricular wall thickness, left atrial diameter, left-ventricular outflow tract gradient, and nonsustained ventricular tachycardia) identified from the literature and internally validated using bootstrapping.

Main Outcomes and Measures: A composite outcome of SCD or an equivalent event (aborted cardiac arrest, appropriate implantable cardioverter defibrillator therapy, or sustained ventricular tachycardia associated with hemodynamic compromise).

Results: Of the 1024 patients included in the study, 699 were boys (68.3%); mean (interquartile range [IQR]) age was 11 (7-14) years. Over a median follow-up of 5.3 years (IQR, 2.6-8.3; total patient years, 5984), 89 patients (8.7%) died suddenly or had an equivalent event (annual event rate, 1.49; 95% CI, 1.15-1.92). The pediatric model was developed using preselected variables to predict the risk of SCD. The model's ability to predict risk at 5 years was validated; the C statistic was 0.69 (95% CI, 0.66-0.72), and the calibration slope was 0.98 (95%, CI 0.59-1.38). For every 10 implantable cardioverter defibrillators implanted in patients with 6% or more of a 5-year SCD risk, 1 patient may potentially be saved from SCD at 5 years.

Conclusions and Relevance: This new, validated risk stratification model for SCD in childhood HCM may provide individualized estimates of risk at 5 years using readily obtained clinical risk factors. External validation studies are required to demonstrate the accuracy of this model's predictions in diverse patient populations.

Original languageEnglish
JournalJAMA Cardiology
Publication statusE-pub ahead of print - Aug 14 2019


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