Catecholaminergic Polymorphic Ventricular Tachycardia in Children

Analysis of Therapeutic Strategies and Outcomes from an International Multicenter Registry

Thomas M. Roston, Jeffrey M. Vinocur, Kathleen R. Maginot, Saira Mohammed, Jack C. Salerno, Susan P. Etheridge, Mitchell Cohen, Robert M. Hamilton, Andreas Pflaumer, Ronald J. Kanter, James E. Potts, Martin J. LaPage, Kathryn K. Collins, Roman A. Gebauer, Joel D. Temple, Anjan S. Batra, Christopher C Erickson, Maria Miszczak-Knecht, Peter Kubuš, Yaniv Bar-Cohen & 11 others Michal Kantoch, Vincent C. Thomas, Gabriele Hessling, Chris Anderson, Ming Lon Young, Michel Cabrera Ortega, Yung R. Lau, Christopher L. Johnsrude, Anne Fournier, Prince J. Kannankeril, Shubhayan Sanatani

Research output: Contribution to journalArticle

84 Citations (Scopus)

Abstract

Background - Catecholaminergic polymorphic ventricular tachycardia is an uncommon, potentially lethal, ion channelopathy. Standard therapies have high failure rates and little is known about treatment in children. Newer options such as flecainide and left cardiac sympathetic denervation are not well validated. We sought to define treatment outcomes in children with catecholaminergic polymorphic ventricular tachycardia. Methods and Results - This is a Pediatric and Congenital Electrophysiology Society multicenter, retrospective cohort study of catecholaminergic polymorphic ventricular tachycardia patients diagnosed before 19 years of age. The cohort included 226 patients, including 170 probands and 56 relatives. Symptomatic presentation was reported in 176 (78%). Symptom onset occurred at 10.8 (interquartile range, 6.8-13.2) years with a delay to diagnosis of 0.5 (0-2.6) years. Syncope (P<0.001), cardiac arrest (P<0.001), and treatment failure (P=0.008) occurred more often in probands. β-Blockers were prescribed in 205 of 211 patients (97%) on medication, and 25% experienced at least 1 treatment failure event. Implantable cardioverter defibrillators were placed in 121 (54%) and was associated with electrical storm in 22 (18%). Flecainide was used in 24% and left cardiac sympathetic denervation in 8%. Six deaths (3%) occurred during a cumulative follow-up of 788 patient-years. Conclusions - This study demonstrates a malignant phenotype and lengthy delay to diagnosis in catecholaminergic polymorphic ventricular tachycardia. Probands were typically severely affected. β-Blockers were almost universally initiated; however, treatment failure, noncompliance and subtherapeutic dosing were often reported. Implantable cardioverter defibrillators were common despite numerous device-related complications. Treatment failure was rare in the quarter of patients on flecainide. Left cardiac sympathetic denervation was not uncommon although the indication was variable.

Original languageEnglish (US)
Pages (from-to)633-642
Number of pages10
JournalCirculation: Arrhythmia and Electrophysiology
Volume8
Issue number3
DOIs
StatePublished - Jun 4 2015

Fingerprint

Registries
Flecainide
Treatment Failure
Sympathectomy
Implantable Defibrillators
Channelopathies
Therapeutics
Electrophysiology
Syncope
Heart Arrest
Cohort Studies
Retrospective Studies
Polymorphic catecholergic ventricular tachycardia
Ions
Pediatrics
Phenotype
Equipment and Supplies

Keywords

  • flecainide
  • implanted cardioverter defibrillator
  • polymorphic catecholaminergic ventricular tachycardia
  • sudden unexpected death

ASJC Scopus subject areas

  • Cardiology and Cardiovascular Medicine
  • Physiology (medical)

Cite this

Catecholaminergic Polymorphic Ventricular Tachycardia in Children : Analysis of Therapeutic Strategies and Outcomes from an International Multicenter Registry. / Roston, Thomas M.; Vinocur, Jeffrey M.; Maginot, Kathleen R.; Mohammed, Saira; Salerno, Jack C.; Etheridge, Susan P.; Cohen, Mitchell; Hamilton, Robert M.; Pflaumer, Andreas; Kanter, Ronald J.; Potts, James E.; LaPage, Martin J.; Collins, Kathryn K.; Gebauer, Roman A.; Temple, Joel D.; Batra, Anjan S.; Erickson, Christopher C; Miszczak-Knecht, Maria; Kubuš, Peter; Bar-Cohen, Yaniv; Kantoch, Michal; Thomas, Vincent C.; Hessling, Gabriele; Anderson, Chris; Young, Ming Lon; Ortega, Michel Cabrera; Lau, Yung R.; Johnsrude, Christopher L.; Fournier, Anne; Kannankeril, Prince J.; Sanatani, Shubhayan.

In: Circulation: Arrhythmia and Electrophysiology, Vol. 8, No. 3, 04.06.2015, p. 633-642.

Research output: Contribution to journalArticle

Roston, TM, Vinocur, JM, Maginot, KR, Mohammed, S, Salerno, JC, Etheridge, SP, Cohen, M, Hamilton, RM, Pflaumer, A, Kanter, RJ, Potts, JE, LaPage, MJ, Collins, KK, Gebauer, RA, Temple, JD, Batra, AS, Erickson, CC, Miszczak-Knecht, M, Kubuš, P, Bar-Cohen, Y, Kantoch, M, Thomas, VC, Hessling, G, Anderson, C, Young, ML, Ortega, MC, Lau, YR, Johnsrude, CL, Fournier, A, Kannankeril, PJ & Sanatani, S 2015, 'Catecholaminergic Polymorphic Ventricular Tachycardia in Children: Analysis of Therapeutic Strategies and Outcomes from an International Multicenter Registry', Circulation: Arrhythmia and Electrophysiology, vol. 8, no. 3, pp. 633-642. https://doi.org/10.1161/CIRCEP.114.002217
Roston, Thomas M. ; Vinocur, Jeffrey M. ; Maginot, Kathleen R. ; Mohammed, Saira ; Salerno, Jack C. ; Etheridge, Susan P. ; Cohen, Mitchell ; Hamilton, Robert M. ; Pflaumer, Andreas ; Kanter, Ronald J. ; Potts, James E. ; LaPage, Martin J. ; Collins, Kathryn K. ; Gebauer, Roman A. ; Temple, Joel D. ; Batra, Anjan S. ; Erickson, Christopher C ; Miszczak-Knecht, Maria ; Kubuš, Peter ; Bar-Cohen, Yaniv ; Kantoch, Michal ; Thomas, Vincent C. ; Hessling, Gabriele ; Anderson, Chris ; Young, Ming Lon ; Ortega, Michel Cabrera ; Lau, Yung R. ; Johnsrude, Christopher L. ; Fournier, Anne ; Kannankeril, Prince J. ; Sanatani, Shubhayan. / Catecholaminergic Polymorphic Ventricular Tachycardia in Children : Analysis of Therapeutic Strategies and Outcomes from an International Multicenter Registry. In: Circulation: Arrhythmia and Electrophysiology. 2015 ; Vol. 8, No. 3. pp. 633-642.
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title = "Catecholaminergic Polymorphic Ventricular Tachycardia in Children: Analysis of Therapeutic Strategies and Outcomes from an International Multicenter Registry",
abstract = "Background - Catecholaminergic polymorphic ventricular tachycardia is an uncommon, potentially lethal, ion channelopathy. Standard therapies have high failure rates and little is known about treatment in children. Newer options such as flecainide and left cardiac sympathetic denervation are not well validated. We sought to define treatment outcomes in children with catecholaminergic polymorphic ventricular tachycardia. Methods and Results - This is a Pediatric and Congenital Electrophysiology Society multicenter, retrospective cohort study of catecholaminergic polymorphic ventricular tachycardia patients diagnosed before 19 years of age. The cohort included 226 patients, including 170 probands and 56 relatives. Symptomatic presentation was reported in 176 (78{\%}). Symptom onset occurred at 10.8 (interquartile range, 6.8-13.2) years with a delay to diagnosis of 0.5 (0-2.6) years. Syncope (P<0.001), cardiac arrest (P<0.001), and treatment failure (P=0.008) occurred more often in probands. β-Blockers were prescribed in 205 of 211 patients (97{\%}) on medication, and 25{\%} experienced at least 1 treatment failure event. Implantable cardioverter defibrillators were placed in 121 (54{\%}) and was associated with electrical storm in 22 (18{\%}). Flecainide was used in 24{\%} and left cardiac sympathetic denervation in 8{\%}. Six deaths (3{\%}) occurred during a cumulative follow-up of 788 patient-years. Conclusions - This study demonstrates a malignant phenotype and lengthy delay to diagnosis in catecholaminergic polymorphic ventricular tachycardia. Probands were typically severely affected. β-Blockers were almost universally initiated; however, treatment failure, noncompliance and subtherapeutic dosing were often reported. Implantable cardioverter defibrillators were common despite numerous device-related complications. Treatment failure was rare in the quarter of patients on flecainide. Left cardiac sympathetic denervation was not uncommon although the indication was variable.",
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T1 - Catecholaminergic Polymorphic Ventricular Tachycardia in Children

T2 - Analysis of Therapeutic Strategies and Outcomes from an International Multicenter Registry

AU - Roston, Thomas M.

AU - Vinocur, Jeffrey M.

AU - Maginot, Kathleen R.

AU - Mohammed, Saira

AU - Salerno, Jack C.

AU - Etheridge, Susan P.

AU - Cohen, Mitchell

AU - Hamilton, Robert M.

AU - Pflaumer, Andreas

AU - Kanter, Ronald J.

AU - Potts, James E.

AU - LaPage, Martin J.

AU - Collins, Kathryn K.

AU - Gebauer, Roman A.

AU - Temple, Joel D.

AU - Batra, Anjan S.

AU - Erickson, Christopher C

AU - Miszczak-Knecht, Maria

AU - Kubuš, Peter

AU - Bar-Cohen, Yaniv

AU - Kantoch, Michal

AU - Thomas, Vincent C.

AU - Hessling, Gabriele

AU - Anderson, Chris

AU - Young, Ming Lon

AU - Ortega, Michel Cabrera

AU - Lau, Yung R.

AU - Johnsrude, Christopher L.

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N2 - Background - Catecholaminergic polymorphic ventricular tachycardia is an uncommon, potentially lethal, ion channelopathy. Standard therapies have high failure rates and little is known about treatment in children. Newer options such as flecainide and left cardiac sympathetic denervation are not well validated. We sought to define treatment outcomes in children with catecholaminergic polymorphic ventricular tachycardia. Methods and Results - This is a Pediatric and Congenital Electrophysiology Society multicenter, retrospective cohort study of catecholaminergic polymorphic ventricular tachycardia patients diagnosed before 19 years of age. The cohort included 226 patients, including 170 probands and 56 relatives. Symptomatic presentation was reported in 176 (78%). Symptom onset occurred at 10.8 (interquartile range, 6.8-13.2) years with a delay to diagnosis of 0.5 (0-2.6) years. Syncope (P<0.001), cardiac arrest (P<0.001), and treatment failure (P=0.008) occurred more often in probands. β-Blockers were prescribed in 205 of 211 patients (97%) on medication, and 25% experienced at least 1 treatment failure event. Implantable cardioverter defibrillators were placed in 121 (54%) and was associated with electrical storm in 22 (18%). Flecainide was used in 24% and left cardiac sympathetic denervation in 8%. Six deaths (3%) occurred during a cumulative follow-up of 788 patient-years. Conclusions - This study demonstrates a malignant phenotype and lengthy delay to diagnosis in catecholaminergic polymorphic ventricular tachycardia. Probands were typically severely affected. β-Blockers were almost universally initiated; however, treatment failure, noncompliance and subtherapeutic dosing were often reported. Implantable cardioverter defibrillators were common despite numerous device-related complications. Treatment failure was rare in the quarter of patients on flecainide. Left cardiac sympathetic denervation was not uncommon although the indication was variable.

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KW - flecainide

KW - implanted cardioverter defibrillator

KW - polymorphic catecholaminergic ventricular tachycardia

KW - sudden unexpected death

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