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Multicenter Study
. 2019 Sep 1;4(9):918-927.
doi: 10.1001/jamacardio.2019.2861.

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

Gabrielle Norrish  1   2   3 , Tao Ding  4 , Ella Field  1   2   3 , Lidia Ziólkowska  5 , Iacopo Olivotto  6 , Giuseppe Limongelli  3   7 , Aristides Anastasakis  8 , Robert Weintraub  9   10   11 , Elena Biagini  12 , Luca Ragni  12 , Terence Prendiville  13 , Sophie Duignan  13 , Karen McLeod  14 , Maria Ilina  14 , Adrián Fernández  15 , Regina Bökenkamp  16 , Anwar Baban  3   17 , Peter Kubuš  18 , Piers E F Daubeney  19 , Georgia Sarquella-Brugada  3   20   21 , Sergi Cesar  3   20 , Chiara Marrone  22 , Vinay Bhole  23 , Constancio Medrano  24 , Orhan Uzun  25 , Elspeth Brown  26 , Ferran Gran  27 , Francisco J Castro  28 , Graham Stuart  29 , Gabriele Vignati  30 , Roberto Barriales-Villa  31 , Luis G Guereta  32 , Satish Adwani  33 , Katie Linter  34 , Tara Bharucha  35 , Pablo Garcia-Pavia  3   36   37 , Torsten B Rasmussen  38 , Margherita M Calcagnino  39   40 , Caroline B Jones  41 , Hans De Wilde  42 , J Toru-Kubo  43 , Tiziana Felice  44 , Jens Mogensen  45 , Sujeev Mathur  46 , Zdenka Reinhardt  47 , Constantinos O'Mahony  2   3   48 , Perry M Elliott  2   3   48 , Rumana Z Omar  4 , Juan P Kaski  1   2   3
Affiliations
Multicenter Study

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

Gabrielle Norrish et al. JAMA Cardiol. .

Abstract

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.

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Conflict of interest statement

Conflict of Interest Disclosures: Dr Barriales-Villa reports grants and personal fees from Sanofi-Genzyme and personal fees from Amicus, Pfizer, Health in Code, and Bayer outside the submitted work. No other conflicts were reported.

Figures

Figure.
Figure.. Performance of the HCM Risk-Kids Model
A, Kaplan-Meier curve showing cumulative probability of sudden cardiac death (SCD) end points within 5 years of baseline evaluation by clinical risk groups calculated by the pediatric SCD risk model. Patients with complete data for the calculation of 5-year SCD risk estimates (n = 527) were classified into 3 risk groups (<4%, 4%-<6%, and ≥6%). B, Comparison of observed and predicted risk by clinical risk group of the pediatric SCD risk model (for 1 imputed data set). Vertical bars represent observed (dark blue) and model-based predicted (light blue) probability of SCD by 5 years. HCM indicates hypertrophic cardiomyopathy.
See this image and copyright information in PMC

Comment in

References

    1. Colan SD, Lipshultz SE, Lowe AM, et al. . Epidemiology and cause-specific outcome of hypertrophic cardiomyopathy in children: findings from the Pediatric Cardiomyopathy Registry. Circulation. 2007;115(6):773-781. doi:10.1161/CIRCULATIONAHA.106.621185 - DOI - PubMed
    1. Alexander PMA, Nugent AW, Daubeney PEF, et al. ; National Australian Childhood Cardiomyopathy Study . Long-term outcomes of hypertrophic cardiomyopathy diagnosed during childhood: results from a national population-based study. Circulation. 2018;138(1):29-36. doi:10.1161/CIRCULATIONAHA.117.028895 - DOI - PubMed
    1. Norrish G, Field E, Mcleod K, et al. . Clinical presentation and survival of childhood hypertrophic cardiomyopathy: a retrospective study in United Kingdom. Eur Heart J. 2019;40(12):986-993. doi:10.1093/eurheartj/ehy798 - DOI - PMC - PubMed
    1. Gersh BJ, Maron BJ, Bonow RO, et al. ; American College of Cardiology Foundation/American Heart Association Task Force on Practice; American Association for Thoracic Surgery; American Society of Echocardiography; American Society of Nuclear Cardiology; Heart Failure Society of America; Heart Rhythm Society; Society for Cardiovascular Angiography and Interventions; Society of Thoracic Surgeons . 2011 ACCF/AHA guideline for the diagnosis and treatment of hypertrophic cardiomyopathy: a report of the American College of Cardiology Foundation/American Heart Association Task Force on Practice Guidelines. J Thorac Cardiovasc Surg. 2011;142(6):e153-e203. doi:10.1016/j.jtcvs.2011.10.020 - DOI - PubMed
    1. Elliott PM, Anastasakis A, Borger MA, et al. ; Authors/Task Force Members . 2014 ESC guidelines on diagnosis and management of hypertrophic cardiomyopathy: the Task Force for the Diagnosis and Management of Hypertrophic Cardiomyopathy of the European Society of Cardiology (ESC). Eur Heart J. 2014;35(39):2733-2779. doi:10.1093/eurheartj/ehu284 - DOI - PubMed

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