Cardiomyopathies in children and adolescents: aetiology, management, and outcomes in the European Society of Cardiology EURObservational Research Programme Cardiomyopathy and Myocarditis Registry

Juan Pablo Kaski(Great Ormond Street Hospital for Children NHS Foundation Trust), Gabrielle Norrish(Great Ormond Street Hospital for Children NHS Foundation Trust), Juan R. Gimeno(Hospital Universitario Virgen de la Arrixaca), Philippe Charron(Inserm), Perry Elliott(St Bartholomew's Hospital), Luigi Tavazzi(Maria Cecilia Hospital), Michał Tendera(Medical University of Silesia), Cécile Laroche(European Society of Cardiology), Aldo P. Maggioni(European Society of Cardiology), Anwar Baban(Bambino Gesù Children's Hospital), Diala Khraiche(Hôpital Necker-Enfants Malades), Lidia Ziółkowska(Children's Memorial Health Institute), Giuseppe Limongelli(University of Campania "Luigi Vanvitelli"), Tiina Ojala(University of Helsinki), Matthias Gorenflo(Heidelberg University), Aris Anastasakis(Onassis Cardiac Surgery Center), Shaimaa Mostafa(Benha University), Alida L.P. Caforio(University of Padua), the EORP Paediatric Cardiomyopathy Registry Investigators, Roberto Ferrari, Alvaro Alonso, Jeroen J. Bax, Carina Blomström‐Lundqvist, Stephan Gielen, Patrizio Lancellotti, A P Maggioni(European Society of Cardiology), Nikos Maniadakis, Fausto J. Pinto, Frank Ruschitzka, Luigi Tavazzi(Maria Cecilia Hospital), Panos Vardas, Franz Weidinger, Uwe Zeymer, Alec Vahanian, Andrzej Budaj, Nikolaos Dagres, Nicolas Danchin, Victoria Delgado, J Emberson, Örjan Friberg, C P Gale, Gr. Heyndrickx, Bernard Iung, Stefan James, A. Pieter Kappetein, Aldo P. Maggioni(European Society of Cardiology), Nikos Maniadakis, Klaudia Vivien Nagy, G Parati, A-S Petronio, Mikko Pietilä, Eva Prescott, Frank Ruschitzka, Frans Van de Werf, Franz Weidinger, Uwe Zeymer, C P Gale, B. Beleslin, Andrzej Budaj, Ovidiu Chioncel, Nikolaos Dagres, Nicolas Danchin, J Emberson, David Erlinge, M Glikson, Alastair Gray, M Kayikcioglu, Aldo P. Maggioni(European Society of Cardiology), Klaudia Vivien Nagy, А. О. Недошивин, A-P Petronio, Jolien W. Roos‐Hesselink, Lars Wallentin, Uwe Zeymer, Bogdan A. Popescu, David Adlam, Alida L.P. Caforio(University of Padua), Davide Capodanno, Marc R. Dweck, David Erlinge, M Glikson, Jörg Hausleiter, Bernard Iung, M Kayikcioglu, Peter Ludman, Lars H. Lund, A P Maggioni(European Society of Cardiology), С.Т. Мацкеплишвили, Benjamin Meder, Klaudia Vivien Nagy, А. О. Недошивин, Danilo Neglia, Agnès Pasquet, Jolien W. Roos‐Hesselink, Fernando J. Rossello, Sameh Shaheen, Aleksandra Torbica, Alp Caforio(University of Padua), Juan R. Gimeno(Hospital Universitario Virgen de la Arrixaca), P hilippe Charron(Inserm), Plachta Elliott(St Bartholomew's Hospital), Juan Pablo Kaski(Great Ormond Street Hospital for Children NHS Foundation Trust), Aldo P. Maggioni(European Society of Cardiology), Luigi Tavazzi(Maria Cecilia Hospital), Michał Tendera(Medical University of Silesia), С. М. Комиссарова, Н. Н. Чакова, S. S. Niyazova, Sadek Mostafa(Benha University), Jaana Pihkala, Tiina Ojala(University of Helsinki), Anita Hiippala, Tero A.H. Järvinen, Damien Bonnet, Diala Khraiche(Hôpital Necker-Enfants Malades), Isabelle Szézépanski, M Gorenflo(Heidelberg University), Richard Arnold, Sebastian Uhl, Victoria C. Ziesenitz, A. Jung, E. Roesch, Maria Ilina, Juan Pablo Kaski(Great Ormond Street Hospital for Children NHS Foundation Trust), Gabrielle Norrish(Great Ormond Street Hospital for Children NHS Foundation Trust), Ella Field, Aris Anastasakis(Onassis Cardiac Surgery Center), K Ritsatos, V Vlagkouli, S. Rammos, Georgios Kourelis, George A. Vagenakis, G. Papadopoulos, Anastasia Giannakopoulou, Evangelos Karanasios, Panagiotis Papachristou, George Servos, Elena Biagini, Anna Corsini, Christian Gagliardi, Maddalena Graziosi, Agnese Milandri, Luca Ragni, Stefano Palmieri, Raffaele Calabrò, Giuseppe Pacileo, Maria Giovanna Russo, Giuseppe Limongelli(University of Campania "Luigi Vanvitelli"), Martina Caiazza, Annapaola Cirillo, Giuseppe Del Giorno, Augusto Esposito, Stefania Tramonte, Fabio Valente, Rita Gravino, Tommaso Marrazzo, Daniele Masarone, Vittorio Pazzanese, A. Rea, Marta Rubino, Fabrizio Drago, Anwar Baban(Bambino Gesù Children's Hospital), Gianfranco Sinagra, Cosimo Carriere, Marco Merlo, Federica Ramani, Marco Bobbo, B Dagata, Aušra Kavoliūnienė, Aušra Krivickienė, Eglė Tamulevičiūtė-Prascienė, Mindaugas Vieželis, Folkert W. Asselbergs, Nicolaas de Jonge, J. H. Kirkels, Joris van der Heijden, L Van Laake, Arjan Sammani, Jayne N. Ajuluchukwu, Akinsanya Olusegun‐Joseph, Ekanem N. Ekure, Grażyna Brzezińska‐Rajszys, Lidia Ziółkowska(Children's Memorial Health Institute), Agnieszka Boruc, E Plodzien, E. V. Zaklyazminskaya, Dzemeshkevich Sl, Е. А. Колбасова, N.P. Kotlukova, Valeria Rusinova, Ángel Cequier, Joel Salazar‐Mendiguchía, Joseph E. Gonzalez, Nicolás Manito, Pablo García‐Pavía, Abelardo Morales Briceño, Marta Cobo Marcos, Fernándo Domínguez, Juan Ramón Gimeno Blanes(Hospital Universitario Virgen de la Arrixaca), F J Castro, Carmen Muñoz Esparza, Marcos Molina, M Sorli García, David López Cuenca, P L Sanchez Fernandez, Eduardo Villacorta, Beatriz Plata Izquierdo, C Avila, Loreto Bravo, Elena Díaz-Peláez, María Gallego‐Delgado, L Garcia-Cuenllas, José López‐Haldón, E M Cantero Perez, M L Pena Pena
European Heart Journal
March 1, 2024
10.1093/eurheartj/ehae109
Cited by 42Open Access
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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.

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