Catecholaminergic polymorphic ventricular tachycardia mediated by ryanodine receptor 2: a validated risk stratification

Background and Aims Patients with catecholaminergic polymorphic ventricular tachycardia (CPVT) are at risk for potentially life-threatening arrhythmic events (AEs) even while treated with beta-blockers. The aim was to develop a model for individualized prediction of AEs in patients with RYR2-mediate...

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Detalhes bibliográficos
Autores: Lieve, KV, van der Werf, C, Kallas, D, Denjoy, I, Bos, JM, Aiba, T, Behr, ER, van den Berg, MP, Bergeman, AT, Blom, NA, Borggrefe, M, Brugada, R, Mora, LMC, Chorin, E, Crotti, L, Davis, A, Drago, F, Dusi, V, Extramiana, F, Franciosi, S, Giudicessi, JR, Llopis, FAG, Haugaa, KH, van den Heuvel, F, Horie, M, Ingles, J, Kammeraad, J, Kannankeril, PJ, Khan, HR, Krahn, AD, MacIntyre, C, Maltret, A, Marjamaa, A, Ohno, S, Peltenburg, PJ, Perez, GJ, Probst, V, Roberts, JD, Robyns, T, Rootwelt-Norberg, C, Noguer, FRI, Roston, TM, Rydberg, A, Sacher, F, Sarquella-Brugada, G, Schwartz, PJ, Semsarian, C, Shimizu, W, Starling, L, Sumitomo, N, Skinner, JR, Tavacova, T, Tfelt-Hansen, J, Till, JA, Yap, SC, Wada, Y, Wangüemert, F, Zorio, E, Ackerman, MJ, Leenhardt, A, Sanatani, S, Tanck, MW, Wilde, AA
Formato: artículo
Estado:Versión publicada
Fecha de publicación:2025
País:España
Recursos:Fundación para el Fomento de la Investigación Sanitaria y Biomédica de la Comunitat Valenciana (FISABIO)
Repositorio:r-FISABIO. Repositorio Institucional de Producción Científica
OAI Identifier:oai:fisabio.fundanetsuite.com:p20302
Acesso em linha:https://fisabio.portalinvestigacion.com/publicaciones/20302
Access Level:acceso abierto
Palavra-chave:Catecholaminergic polymorphic ventricular tachycardia
beta-Blockers
Risk stratification
Sudden cardiac death
Ventricular arrhythmias
Descrição
Resumo:Background and Aims Patients with catecholaminergic polymorphic ventricular tachycardia (CPVT) are at risk for potentially life-threatening arrhythmic events (AEs) even while treated with beta-blockers. The aim was to develop a model for individualized prediction of AEs in patients with RYR2-mediated CPVT on beta-blocker monotherapy.Methods The derivation and independent validation cohorts included 743 and 129 patients, respectively. AEs were defined as arrhythmic syncope, appropriate implantable cardioverter-defibrillator shock, sudden cardiac arrest (SCA), and sudden cardiac death. Near-fatal or fatal AEs (nf/fAEs) included all AEs except for arrhythmic syncope. Prediction models using Cox regression were developed and internally and externally validated.Results A total of 102 (13.7%) patients in the derivation cohort and 24 (18.6%) patients in the validation cohort experienced >= 1 AE over a median follow-up of 5.1 [interquartile range (IQR), 7.7] and 2.4 (IQR, 4.4) years, respectively. Predictors of AE were arrhythmic syncope or SCA prior to diagnosis and age at beta-blocker initiation. In the derivation and validation cohorts, the optimism-corrected C-indices of the models for AE were 0.67 [95% confidence interval (CI) 0.62-0.72] and 0.59 (95% CI 0.48-0.71), respectively. For nf/fAEs, ventricular arrhythmia severity before beta-blocker initiation was a fourth independent predictor, and C-indices of the models in the derivation and validation cohorts were 0.74 (95% CI 0.68-0.80) and 0.60 (95% CI 0.47-0.72), respectively. In the derivation cohort, calibration slopes were 1.00 (95% CI 0.59-1.41) for AE and 1.00 (95% CI 0.69-1.32) for nf/fAE.Conclusions These externally validated risk prediction models using clinical parameters accurately distinguished CPVT patients on beta-blocker monotherapy at low and high risk for future AEs while treated with beta-blockers. These models provide guidance for implementation of clinical management therapies to prevent AEs in patients with CPVT.

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