Cardiomyopathies in children and adolescents: aetiology, management, and outcomes in the European Society of Cardiology EURObservational Research Programme Cardiomyopathy and Myocarditis Registry
Author:
Kaski Juan Pablo1ORCID, Norrish Gabrielle1ORCID, Gimeno Blanes Juan Ramon2ORCID, Charron Philippe3ORCID, Elliott Perry4ORCID, Tavazzi Luigi5ORCID, Tendera Michal6ORCID, Laroche Cécile7, Maggioni Aldo P78ORCID, Baban Anwar9ORCID, Khraiche Diala10, Ziolkowska Lidia11ORCID, Limongelli Giuseppe12ORCID, Ojala Tiina13ORCID, Gorenflo Matthias14ORCID, Anastasakis Aris15ORCID, Mostafa Shaimaa16ORCID, Caforio Alida L P17ORCID, , Ferrari R, Alonso A, Bax J, Blomström-Lundqvist C, Gielen S, Lancellotti P, Maggioni A P, Maniadakis N, Pinto F, Ruschitzka F, Tavazzi L, Vardas P, Weidinger F, Zeymer U, Vahanian A, Budaj A, Dagres N, Danchin N, Delgado V, Emberson J, Friberg O, Gale C P, Heyndrickx G, Iung B, James S, Kappetein A P, Maggioni A P, Maniadakis N, Nagy K V, Parati G, Petronio A-S, Pietila M, Prescott E, Ruschitzka F, Van de Werf F, Weidinger F, Zeymer U, Gale C P, Beleslin B, Budaj A, Chioncel O, Dagres N, Danchin N, Emberson J, Erlinge D, Glikson M, Gray A, Kayikcioglu M, Maggioni A P, Nagy K V, Nedoshivin A, Petronio A-P, Roos-Hesselink J W, Wallentin L, Zeymer U, Popescu B A, Adlam D, Caforio A L P, Capodanno D, Dweck M, Erlinge D, Glikson M, Hausleiter J, Iung B, Kayikcioglu M, Ludman P, Lund L, Maggioni A P, Matskeplishvili S, Meder B, Nagy K V, Nedoshivin A, Neglia D, Pasquet A A, Roos-Hesselink J W, Rossello F J, Shaheen S M, Torbica A, Caforio A lida, Blanes Juan Ramon Gimeno, Charron P hilippe, Elliott P erry, Kaski Juan Pablo, Maggioni Aldo P, Tavazzi Luigi, Tendera Michal, Komissarova S, Chakova N, Niyazova S, Mostafa S, Pihkala J, Ojala T, Hiippala A, Jarvinen T, Bonnet D, Khraiche D, Szezepanski I, Gorenflo M, Arnold R, Uhl S, Ziesenitz V, Jung A, Roesch E, Ilina M, Kaski J P, Norrish G, Field E, Anastasakis A, Ritsatos K, Vlagkouli V, Rammos S, Kourelis G, Vagenakis G, Papadopoulos G, Giannakopoulou A, Karanasios E, Papachristou P, Servos G, Biagini E, Corsini A, Gagliardi C, Graziosi M, Milandri A, Ragni L, Palmieri S, Calabro R, Pacileo G, Russo M G, Limongelli G, Caiazza M, Cirillo A, Del Giorno G, Esposito A, Tramonte S, Valente F, Gravino R, Marrazzo T, Masarone D, Pazzanese V, Rea A, Rubino M, Drago F, Baban A, Sinagra G, Carriere C, Merlo M, Ramani F, Bobbo M, Dagata B, Kavoliuniene A, Krivickiene A, Tamuleviciute-Prasciene E, Viezelis M, Asselbergs F W, De Jonge N, Kirkels J H, Van Der Heijden J, Van Laake L, Sammani A, Ajuluchukwu J, Olusegun-Joseph A, Ekure E, Brzezinska-Rajszys G, Ziolkowska L, Boruc A, Plodzien E, Zaklyazminskaya E, Dzemeshkevich S, Kolbasova E, Kotlukova N, Rusinova V, Cequier A, Salazar-Mendiguchia J, Gonzalez J, Manito N, Garcia-Pavia P, Briceno A, Cobo-Marcos M, Dominguez F, Gimeno Blanes J R, Castro F J, Munoz Esparza C, Sabater Molina M, Sorli García M, Lopez Cuenca D, Sanchez Fernandez P L, Villacorta E, Plata B, Avila C, Bravo L, Diaz-Pelaez E, Gallego-Delgado M, Garcia-Cuenllas L, Lopez-Haldon J E, Cantero Perez E M, Pena Pena M L
Affiliation:
1. Centre for Paediatric Inherited and Rare Cardiovascular Disease, University College London Institute of Cardiovascular Science and Great Ormond Street Hospital NHS Foundation Trust , 20 Guilford Street, WC1N 1DZ London , United Kingdom 2. Department of Cardiology, Hospital Clínico Universitario Virgen de la Arrixaca , Murcia , Spain 3. Assistance Publique-Hôpitaux de Paris, ICAN, Inserm UMR1166, Sorbonne Université, Centre de Référence des Maladies Cardiaques Héréditaires ou Rares, Hôpital Pitié-Salpêtrière , Paris , France 4. Centre for Inherited Cardiovascular Diseases, University College London and St. Bartholomew’s Hospital , London , United Kingdom 5. Department of Cardiology, Maria Cecilia Hospital, GVM Care&Research , Cotignola , Italy 6. Department of Cardiology and Structural Heart Disease, School of Medicine in Katowice, Medical University of Silesia , Katowice , Poland 7. EURObservational Research Programme, European Society of Cardiology , Sophia-Antipolis , France 8. Department of Cardiology, ANMCO Research Center , Florence , Italy 9. Cardiogenetic Centre, Medical and Surgical Department of Pediatric Cardiology, IRCCS, Bambino Gesù Children’s Hospital , Rome , Italy 10. Pediatric and Congenital Cardiology Unit, Hôpital Necker Enfants Malades, M3C-Necker , Paris , France 11. Pediatric Cardiology, The Children’s Memorial Health Institute , Warsaw , Poland 12. Inherited and Heart Disease Unit, Monaldi Hospital, A.O. Colli (University of Campania ‘Luigi Vanvitelli’) , Naples , Italy 13. Pediatric Cardiology, University of Helsinki and Helsinki University Hospital, Hospital for Children and Adolescents , Helsinki , Finland 14. Pediatric Cardiology and Congenital Heart Diseases, University of Heidelberg , Heidelberg , Germany 15. Unit of Rare and Inherited CVD—Department of Cardiology, Kappa Unit, Onassis Cardiac Surgery Centre , Athens , Greece 16. Benha Faculty of Medicine, Cardiovascular Department, Benha University , Benha , Egypt 17. Cardiology, Department of Cardiological, Thoracic and Vascular Sciences and Public Health, University of Padua , Padua , Italy
Abstract
Abstract
Background and Aims
Childhood-onset cardiomyopathies are rare and poorly characterized. This study examined the baseline characteristics and 1-year follow-up of children with cardiomyopathy in the first European Cardiomyopathy Registry.
Methods
Prospective data were collected on individuals aged 1–<18 years enrolled in the European Society of Cardiology EURObservational Research Programme Cardiomyopathy and Myocarditis long-term registry (June 2014–December 2016).
Results
A total of 633 individuals aged ≤18 years with hypertrophic [HCM; n = 388 (61.3%)], dilated [DCM; n = 206 (32.5%)], restrictive [RCM; n = 28 (4.4%)], and arrhythmogenic right ventricular cardiomyopathy [ARVC; n = 11 (1.7%)] were enrolled by 23 referral centres in 14 countries. Median age at diagnosis was 4.0 [interquartile range (IQR) 0–10] years, and there was a male predominance [n = 372 (58.8%)] across all subtypes, with the exception of DCM diagnosed <10 years of age; 621 (98.1%) patients were receiving cardiac medication and 80 (12.6%) had an implantable cardioverter-defibrillator. A total of 253 patients (253/535, 47.3%) had familial disease. Genetic testing was performed in 414 (67.8%) patients with a pathogenic or likely pathogenic variant reported in 250 (60.4%). Rare disease phenocopies were reported in 177 patients (28.0%) and were most frequent in patients under 10 years [142 (30.9%) vs. 35 (19.6%); P = .003]. Over a median follow-up of 12.5 months (IQR 11.3–15.3 months), 18 patients (3.3%) died [HCM n = 9 (2.6%), DCM n = 5 (3.0%), RCM n = 4 (16.0%)]. Heart failure events were most frequent in RCM patients (36.0%).
Conclusions
The findings confirm the heterogeneous aetiology of childhood cardiomyopathies and show a high frequency of familial disease. Outcomes differed by cardiomyopathy subtype, highlighting a need for disease-specific evaluation and treatment.
Funder
Abbott Vascular Int Amgen Cardiovascular AstraZeneca Bayer AG Boehringer Ingelheim Boston Scientific Bristol Myers Squibb Pfizer Alliance Daiichi Sankyo Europe GmbH Alliance Daiichi Sankyo Europe GmbH Eli Lilly and Company Edwards Gedeon Richter Plc Menarini Int. Op MSD-Merck & Co Novartis Pharma AG ResMed Sanofi SERVIER Vifor
Publisher
Oxford University Press (OUP)
Cited by
3 articles.
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