TY - JOUR
T1 - Differences in Presentation and Outcomes between Children with Familial Dilated Cardiomyopathy and Children with Idiopathic Dilated Cardiomyopathy
T2 - A Report from the Pediatric Cardiomyopathy Registry Study Group
AU - Rusconi, Paolo
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.
PY - 2017/2/1
Y1 - 2017/2/1
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.
KW - dilatation
KW - dilated cardiomyopathy
KW - family history
KW - genetics
KW - incidence
KW - pediatrics
UR - http://www.scopus.com/inward/record.url?scp=85013482388&partnerID=8YFLogxK
U2 - 10.1161/CIRCHEARTFAILURE.115.002637
DO - 10.1161/CIRCHEARTFAILURE.115.002637
M3 - Article
C2 - 28193717
AN - SCOPUS:85013482388
SN - 1941-3289
VL - 10
JO - Circulation: Heart Failure
JF - Circulation: Heart Failure
IS - 2
M1 - e002637
ER -