An Evaluation of Age at Symptom-Onset, Proband Status and Sex as Predictors of Disease Severity in Pediatric Catecholaminergic Polymorphic Ventricular Tachycardia

Kód výsledku v IS VaVaI

<a href="https://www.isvavai.cz/riv?ss=detail&h=RIV%2F00064203%3A_____%2F21%3A10429947" target="_blank" >RIV/00064203:_____/21:10429947 - isvavai.cz</a>

Nalezeny alternativní kódy

RIV/00216208:11130/21:10429947

Výsledek na webu

<a href="https://verso.is.cuni.cz/pub/verso.fpl?fname=obd_publikace_handle&handle=usXf1Gpj-j" target="_blank" >https://verso.is.cuni.cz/pub/verso.fpl?fname=obd_publikace_handle&handle=usXf1Gpj-j</a>

DOI - Digital Object Identifier

<a href="http://dx.doi.org/10.1016/j.hrthm.2021.07.061" target="_blank" >10.1016/j.hrthm.2021.07.061</a>

Jazyk výsledku

angličtina

Název v původním jazyce

An Evaluation of Age at Symptom-Onset, Proband Status and Sex as Predictors of Disease Severity in Pediatric Catecholaminergic Polymorphic Ventricular Tachycardia

Popis výsledku v původním jazyce

BACKGROUND: Children with catecholaminergic polymorphic ventricular tachycardia (CPVT) are at risk for sudden death and a risk stratification tool does not exist. OBJECTIVE: To determine whether proband status, age of symptom-onset and/or sex are independent predictors of cardiac events. METHODS: A multicenter, ambispective, cohort of pediatric CPVT patients was categorized by sex, proband status and age of symptom-onset (D1-first decade of life [symptom-onset &lt;10 years] or D2-second decade of life [symptom-onset 10-18 years inclusive]). Demographics, therapy, genetics and outcomes were compared between groups. RESULTS: A total of 133 patients were included and stratified into 58 D1- and 75 D2-patients, 68 females and 65 males, and 106 probands and 27 relatives. Localization of RYR2 variants to hotspots differed based on proband status and age of symptom-onset. The cardiac event rate was 33% (n=44/133), inclusive of a 3% (n=4/133) mortality rate, over a median of 6-years (IQR:3-11) after time of symptom-onset. Proband status, rather than age of symptom-onset or sex, was an independent predictor of time to first cardiac event (p=0.008, HR=4.4). The 5-, 10- and 15- year event-free survival rate for probands were 77%, 56%, 46% and for relatives were 96%, 91%, 86%, respectively. Event-risk after diagnosis was 48% (n=32/67) in patients on β-blocker or flecainide alone versus 10% (n=5/48) on β-blocker plus flecainide and/or left cardiac sympathetic denervation (p&lt;0.001). CONCLUSION: Proband status, but not age of symptom-onset or male sex, independently predicted an earlier-onset of cardiac events. A larger sample size would enable a comprehensive investigation of other risk factors.

Název v anglickém jazyce

An Evaluation of Age at Symptom-Onset, Proband Status and Sex as Predictors of Disease Severity in Pediatric Catecholaminergic Polymorphic Ventricular Tachycardia

Popis výsledku anglicky

BACKGROUND: Children with catecholaminergic polymorphic ventricular tachycardia (CPVT) are at risk for sudden death and a risk stratification tool does not exist. OBJECTIVE: To determine whether proband status, age of symptom-onset and/or sex are independent predictors of cardiac events. METHODS: A multicenter, ambispective, cohort of pediatric CPVT patients was categorized by sex, proband status and age of symptom-onset (D1-first decade of life [symptom-onset &lt;10 years] or D2-second decade of life [symptom-onset 10-18 years inclusive]). Demographics, therapy, genetics and outcomes were compared between groups. RESULTS: A total of 133 patients were included and stratified into 58 D1- and 75 D2-patients, 68 females and 65 males, and 106 probands and 27 relatives. Localization of RYR2 variants to hotspots differed based on proband status and age of symptom-onset. The cardiac event rate was 33% (n=44/133), inclusive of a 3% (n=4/133) mortality rate, over a median of 6-years (IQR:3-11) after time of symptom-onset. Proband status, rather than age of symptom-onset or sex, was an independent predictor of time to first cardiac event (p=0.008, HR=4.4). The 5-, 10- and 15- year event-free survival rate for probands were 77%, 56%, 46% and for relatives were 96%, 91%, 86%, respectively. Event-risk after diagnosis was 48% (n=32/67) in patients on β-blocker or flecainide alone versus 10% (n=5/48) on β-blocker plus flecainide and/or left cardiac sympathetic denervation (p&lt;0.001). CONCLUSION: Proband status, but not age of symptom-onset or male sex, independently predicted an earlier-onset of cardiac events. A larger sample size would enable a comprehensive investigation of other risk factors.

Druh

J_imp - Článek v periodiku v databázi Web of Science

CEP obor

—

OECD FORD obor

30201 - Cardiac and Cardiovascular systems

Projekt

—

Návaznosti

I - Institucionalni podpora na dlouhodoby koncepcni rozvoj vyzkumne organizace

Rok uplatnění

2021

Kód důvěrnosti údajů

S - Úplné a pravdivé údaje o projektu nepodléhají ochraně podle zvláštních právních předpisů

Název periodika

Heart Rhythm

ISSN

1547-5271

e-ISSN

—

Svazek periodika

18

Číslo periodika v rámci svazku

11

Stát vydavatele periodika

US - Spojené státy americké

Počet stran výsledku

8

Strana od-do

1825-1832

Kód UT WoS článku

000729403800006

EID výsledku v databázi Scopus

2-s2.0-85113307351