Differences in Presentation and Outcomes between Children with Familial Dilated Cardiomyopathy and Children with Idiopathic Dilated Cardiomyopathy: A Report from the Pediatric Cardiomyopathy Registry Study Group

Paolo Rusconi, James D. Wilkinson, Lynn A. Sleeper, Minmin Lu, Gerald F. Cox, Jeffrey A. Towbin, Steven D. Colan, Steven A. Webber, Charles E. Canter, Stephanie M. Ware, Daphne T. Hsu, Wendy K. Chung, John L. Jefferies, Christina Cordero, Steven E. Lipshultz

Research output: Contribution to journalArticle

12 Citations (Scopus)

Abstract

Background - Research comparing the survival of children with familial dilated cardiomyopathy (FDCM) to that of children with idiopathic dilated cardiomyopathy (IDCM) has produced conflicting results. Methods and Results - We analyzed data from children with FDCM or IDCM using the National Heart, Lung, and Blood Institute-funded Pediatric Cardiomyopathy Registry. Compared to children with IDCM (n=647), children with FDCM (n=223) were older (mean 6.2 versus 4.5 years, P<0.001), less often had heart failure (64% versus 78%, P<0.001), had less-depressed mean left ventricular fractional shortening z scores (-7.85±3.98 versus -9.06±3.89, P<0.001) and lower end-diastolic dimension z scores (4.12±2.61 versus 4.91±2.57, P<0.001) at diagnosis. The cumulative incidence of death was lower for patients with FDCM compared with IDCM (P=0.04; hazard ratio 0.64, P=0.06), but no difference in risk of transplant or the combined death or transplant outcome. There was no difference in the proportion of children with echocardiographic normalization at 3 years of follow-up (FDCM, 30% versus IDCM, 26%; P=0.33). Multivariable analysis showed no difference in outcomes between FDCM and IDCM but for both groups older age, congestive heart failure, and increased left ventricular end-systolic dimension zscore at diagnosis were independently associated with an increased risk of death or heart transplantation (all Ps<0.001). Conclusions - There was no survival difference between FDCM and IDCM after adjustment for other factors. Older age, congestive heart failure, and greater left ventricular dilation at diagnosis were independently associated with increased risk of the combined end point of death or transplantation. Clinical Trial Registration - URL: https://clinicaltrials.gov. Unique identifier: NCT00005391.

Original languageEnglish (US)
Article numbere002637
JournalCirculation: Heart Failure
Volume10
Issue number2
DOIs
StatePublished - Feb 1 2017

Fingerprint

Dilated Cardiomyopathy
Cardiomyopathies
Registries
Pediatrics
Heart Failure
National Heart, Lung, and Blood Institute (U.S.)
Transplants
Survival
Heart Transplantation
Familial dilated cardiomyopathy
Dilatation
Age Groups
Transplantation
Clinical Trials
Incidence
Research

Keywords

  • dilatation
  • dilated cardiomyopathy
  • family history
  • genetics
  • incidence
  • pediatrics

ASJC Scopus subject areas

  • Cardiology and Cardiovascular Medicine

Cite this

Differences in Presentation and Outcomes between Children with Familial Dilated Cardiomyopathy and Children with Idiopathic Dilated Cardiomyopathy : A Report from the Pediatric Cardiomyopathy Registry Study Group. / Rusconi, Paolo; Wilkinson, James D.; Sleeper, Lynn A.; Lu, Minmin; Cox, Gerald F.; Towbin, Jeffrey A.; Colan, Steven D.; Webber, Steven A.; Canter, Charles E.; Ware, Stephanie M.; Hsu, Daphne T.; Chung, Wendy K.; Jefferies, John L.; Cordero, Christina; Lipshultz, Steven E.

In: Circulation: Heart Failure, Vol. 10, No. 2, e002637, 01.02.2017.

Research output: Contribution to journalArticle

Rusconi, P, Wilkinson, JD, Sleeper, LA, Lu, M, Cox, GF, Towbin, JA, Colan, SD, Webber, SA, Canter, CE, Ware, SM, Hsu, DT, Chung, WK, Jefferies, JL, Cordero, C & Lipshultz, SE 2017, 'Differences in Presentation and Outcomes between Children with Familial Dilated Cardiomyopathy and Children with Idiopathic Dilated Cardiomyopathy: A Report from the Pediatric Cardiomyopathy Registry Study Group', Circulation: Heart Failure, vol. 10, no. 2, e002637. https://doi.org/10.1161/CIRCHEARTFAILURE.115.002637
Rusconi, Paolo ; Wilkinson, James D. ; Sleeper, Lynn A. ; Lu, Minmin ; Cox, Gerald F. ; Towbin, Jeffrey A. ; Colan, Steven D. ; Webber, Steven A. ; Canter, Charles E. ; Ware, Stephanie M. ; Hsu, Daphne T. ; Chung, Wendy K. ; Jefferies, John L. ; Cordero, Christina ; Lipshultz, Steven E. / Differences in Presentation and Outcomes between Children with Familial Dilated Cardiomyopathy and Children with Idiopathic Dilated Cardiomyopathy : A Report from the Pediatric Cardiomyopathy Registry Study Group. In: Circulation: Heart Failure. 2017 ; Vol. 10, No. 2.
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abstract = "Background - Research comparing the survival of children with familial dilated cardiomyopathy (FDCM) to that of children with idiopathic dilated cardiomyopathy (IDCM) has produced conflicting results. Methods and Results - We analyzed data from children with FDCM or IDCM using the National Heart, Lung, and Blood Institute-funded Pediatric Cardiomyopathy Registry. Compared to children with IDCM (n=647), children with FDCM (n=223) were older (mean 6.2 versus 4.5 years, P<0.001), less often had heart failure (64{\%} versus 78{\%}, P<0.001), had less-depressed mean left ventricular fractional shortening z scores (-7.85±3.98 versus -9.06±3.89, P<0.001) and lower end-diastolic dimension z scores (4.12±2.61 versus 4.91±2.57, P<0.001) at diagnosis. The cumulative incidence of death was lower for patients with FDCM compared with IDCM (P=0.04; hazard ratio 0.64, P=0.06), but no difference in risk of transplant or the combined death or transplant outcome. There was no difference in the proportion of children with echocardiographic normalization at 3 years of follow-up (FDCM, 30{\%} versus IDCM, 26{\%}; P=0.33). Multivariable analysis showed no difference in outcomes between FDCM and IDCM but for both groups older age, congestive heart failure, and increased left ventricular end-systolic dimension zscore at diagnosis were independently associated with an increased risk of death or heart transplantation (all Ps<0.001). Conclusions - There was no survival difference between FDCM and IDCM after adjustment for other factors. Older age, congestive heart failure, and greater left ventricular dilation at diagnosis were independently associated with increased risk of the combined end point of death or transplantation. Clinical Trial Registration - URL: https://clinicaltrials.gov. Unique identifier: NCT00005391.",
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T2 - A Report from the Pediatric Cardiomyopathy Registry Study Group

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AU - Wilkinson, James D.

AU - Sleeper, Lynn A.

AU - Lu, Minmin

AU - Cox, Gerald F.

AU - Towbin, Jeffrey A.

AU - Colan, Steven D.

AU - Webber, Steven A.

AU - Canter, Charles E.

AU - Ware, Stephanie M.

AU - Hsu, Daphne T.

AU - Chung, Wendy K.

AU - Jefferies, John L.

AU - Cordero, Christina

AU - Lipshultz, Steven E.

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N2 - Background - Research comparing the survival of children with familial dilated cardiomyopathy (FDCM) to that of children with idiopathic dilated cardiomyopathy (IDCM) has produced conflicting results. Methods and Results - We analyzed data from children with FDCM or IDCM using the National Heart, Lung, and Blood Institute-funded Pediatric Cardiomyopathy Registry. Compared to children with IDCM (n=647), children with FDCM (n=223) were older (mean 6.2 versus 4.5 years, P<0.001), less often had heart failure (64% versus 78%, P<0.001), had less-depressed mean left ventricular fractional shortening z scores (-7.85±3.98 versus -9.06±3.89, P<0.001) and lower end-diastolic dimension z scores (4.12±2.61 versus 4.91±2.57, P<0.001) at diagnosis. The cumulative incidence of death was lower for patients with FDCM compared with IDCM (P=0.04; hazard ratio 0.64, P=0.06), but no difference in risk of transplant or the combined death or transplant outcome. There was no difference in the proportion of children with echocardiographic normalization at 3 years of follow-up (FDCM, 30% versus IDCM, 26%; P=0.33). Multivariable analysis showed no difference in outcomes between FDCM and IDCM but for both groups older age, congestive heart failure, and increased left ventricular end-systolic dimension zscore at diagnosis were independently associated with an increased risk of death or heart transplantation (all Ps<0.001). Conclusions - There was no survival difference between FDCM and IDCM after adjustment for other factors. Older age, congestive heart failure, and greater left ventricular dilation at diagnosis were independently associated with increased risk of the combined end point of death or transplantation. Clinical Trial Registration - URL: https://clinicaltrials.gov. Unique identifier: NCT00005391.

AB - Background - Research comparing the survival of children with familial dilated cardiomyopathy (FDCM) to that of children with idiopathic dilated cardiomyopathy (IDCM) has produced conflicting results. Methods and Results - We analyzed data from children with FDCM or IDCM using the National Heart, Lung, and Blood Institute-funded Pediatric Cardiomyopathy Registry. Compared to children with IDCM (n=647), children with FDCM (n=223) were older (mean 6.2 versus 4.5 years, P<0.001), less often had heart failure (64% versus 78%, P<0.001), had less-depressed mean left ventricular fractional shortening z scores (-7.85±3.98 versus -9.06±3.89, P<0.001) and lower end-diastolic dimension z scores (4.12±2.61 versus 4.91±2.57, P<0.001) at diagnosis. The cumulative incidence of death was lower for patients with FDCM compared with IDCM (P=0.04; hazard ratio 0.64, P=0.06), but no difference in risk of transplant or the combined death or transplant outcome. There was no difference in the proportion of children with echocardiographic normalization at 3 years of follow-up (FDCM, 30% versus IDCM, 26%; P=0.33). Multivariable analysis showed no difference in outcomes between FDCM and IDCM but for both groups older age, congestive heart failure, and increased left ventricular end-systolic dimension zscore at diagnosis were independently associated with an increased risk of death or heart transplantation (all Ps<0.001). Conclusions - There was no survival difference between FDCM and IDCM after adjustment for other factors. Older age, congestive heart failure, and greater left ventricular dilation at diagnosis were independently associated with increased risk of the combined end point of death or transplantation. Clinical Trial Registration - URL: https://clinicaltrials.gov. Unique identifier: NCT00005391.

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