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 ß-blockers. The aim was to develop a model for individualized prediction of AEs in patients with RYR2-mediated...

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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, Carrillo Mora LM, Chorin E, Crotti L, Davis A, Drago F, Dusi V, Extramiana F, Franciosi S, Giudicessi JR, González Llopis FÁ, 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, Roses I Noguer F, 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
Tipo de recurso: artículo
Fecha de publicación:2025
País:España
Institución:Fundació Sant Joan de Déu
Repositorio:r-FSJD. Repositorio Institucional de Producción Científica de la Fundació Sant Joan de Déu
OAI Identifier:oai:fsjd.fundanetsuite.com:p29651
Acceso en línea:https://fsjd.fundanetsuite.com/Publicaciones/ProdCientif/PublicacionFrw.aspx?id=29651
Access Level:acceso abierto
Palabra clave:Catecholaminergic polymorphic ventricular tachycardia
Risk stratification
Sudden cardiac death
Ventricular arrhythmias
ß-Blockers
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spelling Catecholaminergic polymorphic ventricular tachycardia mediated by ryanodine receptor 2: a validated risk stratification.Lieve KVvan der Werf CKallas DDenjoy IBos JMAiba TBehr ERvan den Berg MPBergeman ATBlom NABorggrefe MBrugada RCarrillo Mora LMChorin ECrotti LDavis ADrago FDusi VExtramiana FFranciosi SGiudicessi JRGonzález Llopis FÁHaugaa KHvan den Heuvel FHorie MIngles JKammeraad JKannankeril PJKhan HRKrahn ADMacIntyre CMaltret AMarjamaa AOhno SPeltenburg PJPerez GJProbst VRoberts JDRobyns TRootwelt-Norberg CRoses I Noguer FRoston TMRydberg ASacher FSarquella-Brugada GSchwartz PJSemsarian CShimizu WStarling LSumitomo NSkinner JRTavacova TTfelt-Hansen JTill JAYap SCWada YWangüemert FZorio EAckerman MJLeenhardt ASanatani STanck MWWilde AACatecholaminergic polymorphic ventricular tachycardiaRisk stratificationSudden cardiac deathVentricular arrhythmiasß-BlockersBACKGROUND AND AIMS: Patients with catecholaminergic polymorphic ventricular tachycardia (CPVT) are at risk for potentially life-threatening arrhythmic events (AEs) even while treated with ß-blockers. The aim was to develop a model for individualized prediction of AEs in patients with RYR2-mediated CPVT on ß-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 ß-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 ß-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 ß-blocker monotherapy at low and high risk for future AEs while treated with ß-blockers. These models provide guidance for implementation of clinical management therapies to prevent AEs in patients with CPVT.OXFORD UNIV PRESS2025info:eu-repo/semantics/articleaheadOfPrintVersionhttps://fsjd.fundanetsuite.com/Publicaciones/ProdCientif/PublicacionFrw.aspx?id=29651EUROPEAN HEART JOURNALISSN: 0195668XISSNe: 15229645reponame:r-FSJD. Repositorio Institucional de Producción Científica de la Fundació Sant Joan de Déuinstname:Fundació Sant Joan de DéuInglésinfo:eu-repo/semantics/openAccessoai:fsjd.fundanetsuite.com:p296512026-05-27T12:37:41Z
dc.title.none.fl_str_mv Catecholaminergic polymorphic ventricular tachycardia mediated by ryanodine receptor 2: a validated risk stratification.
title Catecholaminergic polymorphic ventricular tachycardia mediated by ryanodine receptor 2: a validated risk stratification.
spellingShingle Catecholaminergic polymorphic ventricular tachycardia mediated by ryanodine receptor 2: a validated risk stratification.
Lieve KV
Catecholaminergic polymorphic ventricular tachycardia
Risk stratification
Sudden cardiac death
Ventricular arrhythmias
ß-Blockers
title_short Catecholaminergic polymorphic ventricular tachycardia mediated by ryanodine receptor 2: a validated risk stratification.
title_full Catecholaminergic polymorphic ventricular tachycardia mediated by ryanodine receptor 2: a validated risk stratification.
title_fullStr Catecholaminergic polymorphic ventricular tachycardia mediated by ryanodine receptor 2: a validated risk stratification.
title_full_unstemmed Catecholaminergic polymorphic ventricular tachycardia mediated by ryanodine receptor 2: a validated risk stratification.
title_sort Catecholaminergic polymorphic ventricular tachycardia mediated by ryanodine receptor 2: a validated risk stratification.
dc.creator.none.fl_str_mv 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
Carrillo Mora LM
Chorin E
Crotti L
Davis A
Drago F
Dusi V
Extramiana F
Franciosi S
Giudicessi JR
González Llopis FÁ
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
Roses I Noguer F
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
author Lieve KV
author_facet 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
Carrillo Mora LM
Chorin E
Crotti L
Davis A
Drago F
Dusi V
Extramiana F
Franciosi S
Giudicessi JR
González Llopis FÁ
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
Roses I Noguer F
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
author_role author
author2 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
Carrillo Mora LM
Chorin E
Crotti L
Davis A
Drago F
Dusi V
Extramiana F
Franciosi S
Giudicessi JR
González Llopis FÁ
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
Roses I Noguer F
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
author2_role author
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author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
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author
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author
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dc.subject.none.fl_str_mv Catecholaminergic polymorphic ventricular tachycardia
Risk stratification
Sudden cardiac death
Ventricular arrhythmias
ß-Blockers
topic Catecholaminergic polymorphic ventricular tachycardia
Risk stratification
Sudden cardiac death
Ventricular arrhythmias
ß-Blockers
description BACKGROUND AND AIMS: Patients with catecholaminergic polymorphic ventricular tachycardia (CPVT) are at risk for potentially life-threatening arrhythmic events (AEs) even while treated with ß-blockers. The aim was to develop a model for individualized prediction of AEs in patients with RYR2-mediated CPVT on ß-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 ß-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 ß-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 ß-blocker monotherapy at low and high risk for future AEs while treated with ß-blockers. These models provide guidance for implementation of clinical management therapies to prevent AEs in patients with CPVT.
publishDate 2025
dc.date.none.fl_str_mv 2025
dc.type.none.fl_str_mv info:eu-repo/semantics/article
aheadOfPrintVersion
format article
dc.identifier.none.fl_str_mv https://fsjd.fundanetsuite.com/Publicaciones/ProdCientif/PublicacionFrw.aspx?id=29651
url https://fsjd.fundanetsuite.com/Publicaciones/ProdCientif/PublicacionFrw.aspx?id=29651
dc.language.none.fl_str_mv Inglés
language_invalid_str_mv Inglés
dc.rights.none.fl_str_mv info:eu-repo/semantics/openAccess
eu_rights_str_mv openAccess
dc.publisher.none.fl_str_mv OXFORD UNIV PRESS
publisher.none.fl_str_mv OXFORD UNIV PRESS
dc.source.none.fl_str_mv EUROPEAN HEART JOURNAL
ISSN: 0195668X
ISSNe: 15229645
reponame:r-FSJD. Repositorio Institucional de Producción Científica de la Fundació Sant Joan de Déu
instname:Fundació Sant Joan de Déu
instname_str Fundació Sant Joan de Déu
reponame_str r-FSJD. Repositorio Institucional de Producción Científica de la Fundació Sant Joan de Déu
collection r-FSJD. Repositorio Institucional de Producción Científica de la Fundació Sant Joan de Déu
repository.name.fl_str_mv
repository.mail.fl_str_mv
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