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Original Investigation
August 14, 2019

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

Author Affiliations
  • 1Centre for Inherited Cardiovascular Diseases, Department of Cardiology, Great Ormond Street Hospital, London, United Kingdom
  • 2Institute of Cardiovascular Sciences, University College London, London, United Kingdom
  • 3European Reference Network for Rare and Complex Diseases of the Heart, Amsterdam, the Netherlands
  • 4Department of Statistical Science, University College London, London, United Kingdom
  • 5Department of Cardiology, The Children’s Memorial Health Institute, Warsaw, Poland
  • 6Cardiothoracovascular Department, Careggi University Hospital, Florence, Italy
  • 7Department of Cardiothoracic Sciences, Monaldi Hospital, Naples, Italy
  • 8Department of Cardiology, Onassis Cardiac Surgery Center, Athens, Greece
  • 9Department of Cardiology, The Royal Children’s Hospital, Melbourne, Australia
  • 10Department of Clinical Sciences, The Murdoch Children’s Research Institute, Parkville, Australia
  • 11Department of Medical and Health Sciences, University of Melbourne, Melbourne, Australia
  • 12Department of Cardiology, S. Orsola-Malpighi Hospital, Bologna, Italy
  • 13The Children’s Heart Centre, Our Lady’s Children’s Hospital, Dublin, Ireland
  • 14Department of Paediatric Cardiology, Royal Hospital for Children, Glasgow, United Kingdom
  • 15Department of Ambulatory Cardiology, Favaloro Foundation University Hospital, Buenos Aires, Argentina
  • 16Department of Paediatric Cardiology, Leiden University Medical Center, Leiden, the Netherlands
  • 17Department of Paediatric Cardiology and Cardiac Surgery, Bambino Gesu Hospital, Rome, Italy
  • 18Children’s Heart Centre, University Hospital Motol, Prague, Czech Republic
  • 19Department of Paediatric Cardiology, Royal Brompton and Harefield NHS Trust, London, United Kingdom
  • 20Arrhythmia and Inherited Cardiac Diseases Unit, Hospital Sant Joan de Déu, University of Barcelona, Barcelona, Spain
  • 21Medical Sciences Department, School of Medicine, University of Girona, Girona, Spain
  • 22Department of Paediatric Cardiology, Papa Giovanni XXIII Hospital, Bergamo, Italy
  • 23The Heart Unit, Birmingham Children’s Hospital, Birmingham, United Kingdom
  • 24Department of Paediatric Cardiology, Hospital General Universitario Gregorio Marañón, Madrid, Spain
  • 25Children’s Heart Unit, University Hospital of Wales, Cardiff, United Kingdom
  • 26Department of Paediatric Cardiology, Leeds General Infirmary, Leeds, United Kingdom
  • 27Paediatric Cardiology Department, Val d’Hebron University Hospital, Barcelona, Spain
  • 28Department of Cardiology, University Hospital Virgen de la Arrixaca, Murcia, Spain
  • 29Department of Paediatric Cardiology, Bristol Royal Hospital for Children, Bristol, United Kingdom
  • 30Paediatric Cardiology Unit, Niguarda Hospital, Milan, Italy
  • 31Department of Cardiology, Complexo Hospitalario Universitario A Coruña, Centro de Investigación Biomédica en Red Enfermedades Cardiovasculares, A Coruña, Spain
  • 32Department of Cardiology, University Hospital La Paz, Madrid, Spain
  • 33Department of Paediatric Cardiology, John Radcliffe Hospital, Oxford, United Kingdom
  • 34Department of Paediatric Cardiology, Glenfield Hospital, Leicester, United Kingdom
  • 35Department of Paediatric Cardiology, Southampton General Hospital, Southampton, United Kingdom
  • 36Department of Cardiology, Hospital Universitario Puerta de Hierro Majadahonda, Centro de Investigación Biomédica en Red Enfermedades Cardiovasculares, Madrid, Spain
  • 37Department of Cardiology, University Francisco de Vitoria, Pozuelo de Alarcon, Spain
  • 38Department of Cardiology, Aarhus University Hospital, Aarhus, Denmark
  • 39Department of Cardiology, University Hospitals Parma, Parma, Italy
  • 40Cardiology Unit, IRCCS Ospedale Maggiore Policlinico, Milan, Italy
  • 41Department of Cardiology, Alder Hey Children’s Hospital, Liverpool, United Kingdom
  • 42Department of Paediatric Cardiology, Ghent University Hospital, Ghent, Belgium
  • 43Department of Cardiology and Geriatrics, Kochi Medical School, Kochi University, Kochi, Japan
  • 44Department of Paediatric Cardiology, Mater Dei Hospital, Msida, Malta
  • 45Department of Cardiology, Odense University Hospital, Odense, Denmark
  • 46Children’s Heart Service, Evelina Children’s Hospital, London, United Kingdom
  • 47Department of Paediatric Cardiology, The Freeman Hospital, Newcastle, United Kingdom
  • 48St Bartholomew’s Centre for Inherited Cardiovascular Diseases, Barts Heart Centre, St Bartholomew’s Hospital, West Smithfield, London, United Kingdom
JAMA Cardiol. Published online August 14, 2019. doi:10.1001/jamacardio.2019.2861
Key Points

Question  Can sudden cardiac death risk in children with hypertrophic cardiomyopathy be predicted?

Findings  In this cohort study of 1024 consecutively evaluated children (age ≤16 years), a prognostic model was developed using preselected predictor variables identified from the literature. The model’s ability to predict risk at 5 years was internally validated using bootstrapping.

Meaning  This new, validated risk stratification model for sudden cardiac death risk in childhood hypertrophic cardiomyopathy may provide individualized estimates of risk at 5 years using readily obtained data on clinical risk factors; external validation studies are required to demonstrate the accuracy of this model's predictions in diverse patient populations.

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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