Recovery of echocardiographic function in children with idiopathic dilated cardiomyopathy

Results from the pediatric cardiomyopathy registry

Melanie D. Everitt, Lynn A. Sleeper, Minmin Lu, Charles E. Canter, Elfriede Pahl, James D. Wilkinson, Linda J. Addonizio, Jeffrey A. Towbin, Joseph Rossano, Rakesh K. Singh, Jacqueline M. Lamour, Steven A. Webber, Steven D. Colan, Renee Margossian, Paul F. Kantor, John L. Jefferies, Steven E. Lipshultz

Research output: Contribution to journalArticle

62 Citations (Scopus)

Abstract

Objectives This study sought to determine the incidence and predictors of recovery of normal echocardiographic function among children with idiopathic dilated cardiomyopathy (DCM). Background Most children with idiopathic DCM have poor outcomes; however, some improve. Methods We studied children <18 years of age from the Pediatric Cardiomyopathy Registry who had both depressed left ventricular (LV) function (fractional shortening or ejection fraction z-score <-2) and LV dilation (end-diastolic dimension [LVEDD] z-score >2) at diagnosis and who had at least 1 follow-up echocardiogram 30 days to 2 years from the initial echocardiogram. We estimated the cumulative incidence and predictors of normalization. Results Among 868 children who met the inclusion criteria, 741 (85%) had both echocardiograms. At 2 years, 22% had recovered normal LV function and size; 51% had died or undergone heart transplantation (median, 3.2 months), and 27% had persistently abnormal echocardiograms. Younger age (hazard ratio [HR]: 0.92; 95% confidence interval [CI]: 0.88 to 0.97) and lower LVEDD z-score (HR: 0.78; 95% CI: 0.70 to 0.87) independently predicted normalization. Nine children (9%) with normal LV function and size within 2 years of diagnosis later underwent heart transplantation or died. Conclusions Despite marked LV dilation and depressed function initially, children with idiopathic DCM can recover normal LV size and function, particularly those younger and with less LV dilation at diagnosis. Investigations related to predictors of recovery, such as genetic associations, serum markers, and the impact of medical therapy or ventricular unloading with assist devices are important next steps. Longer follow-up after normalization is warranted as cardiac failure can recur. (Pediatric Cardiomyopathy Registry; NCT00005391).

Original languageEnglish (US)
Pages (from-to)1405-1413
Number of pages9
JournalJournal of the American College of Cardiology
Volume63
Issue number14
DOIs
StatePublished - Apr 15 2014

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Recovery of Function
Dilated Cardiomyopathy
Cardiomyopathies
Registries
Pediatrics
Heart Transplantation
Dilatation
Confidence Intervals
Delayed Diagnosis
Incidence
Heart Failure
Biomarkers
Equipment and Supplies

Keywords

  • cardiomyopathy
  • echocardiography
  • heart failure
  • pediatrics

ASJC Scopus subject areas

  • Cardiology and Cardiovascular Medicine

Cite this

Recovery of echocardiographic function in children with idiopathic dilated cardiomyopathy : Results from the pediatric cardiomyopathy registry. / Everitt, Melanie D.; Sleeper, Lynn A.; Lu, Minmin; Canter, Charles E.; Pahl, Elfriede; Wilkinson, James D.; Addonizio, Linda J.; Towbin, Jeffrey A.; Rossano, Joseph; Singh, Rakesh K.; Lamour, Jacqueline M.; Webber, Steven A.; Colan, Steven D.; Margossian, Renee; Kantor, Paul F.; Jefferies, John L.; Lipshultz, Steven E.

In: Journal of the American College of Cardiology, Vol. 63, No. 14, 15.04.2014, p. 1405-1413.

Research output: Contribution to journalArticle

Everitt, MD, Sleeper, LA, Lu, M, Canter, CE, Pahl, E, Wilkinson, JD, Addonizio, LJ, Towbin, JA, Rossano, J, Singh, RK, Lamour, JM, Webber, SA, Colan, SD, Margossian, R, Kantor, PF, Jefferies, JL & Lipshultz, SE 2014, 'Recovery of echocardiographic function in children with idiopathic dilated cardiomyopathy: Results from the pediatric cardiomyopathy registry', Journal of the American College of Cardiology, vol. 63, no. 14, pp. 1405-1413. https://doi.org/10.1016/j.jacc.2013.11.059
Everitt, Melanie D. ; Sleeper, Lynn A. ; Lu, Minmin ; Canter, Charles E. ; Pahl, Elfriede ; Wilkinson, James D. ; Addonizio, Linda J. ; Towbin, Jeffrey A. ; Rossano, Joseph ; Singh, Rakesh K. ; Lamour, Jacqueline M. ; Webber, Steven A. ; Colan, Steven D. ; Margossian, Renee ; Kantor, Paul F. ; Jefferies, John L. ; Lipshultz, Steven E. / Recovery of echocardiographic function in children with idiopathic dilated cardiomyopathy : Results from the pediatric cardiomyopathy registry. In: Journal of the American College of Cardiology. 2014 ; Vol. 63, No. 14. pp. 1405-1413.
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abstract = "Objectives This study sought to determine the incidence and predictors of recovery of normal echocardiographic function among children with idiopathic dilated cardiomyopathy (DCM). Background Most children with idiopathic DCM have poor outcomes; however, some improve. Methods We studied children <18 years of age from the Pediatric Cardiomyopathy Registry who had both depressed left ventricular (LV) function (fractional shortening or ejection fraction z-score <-2) and LV dilation (end-diastolic dimension [LVEDD] z-score >2) at diagnosis and who had at least 1 follow-up echocardiogram 30 days to 2 years from the initial echocardiogram. We estimated the cumulative incidence and predictors of normalization. Results Among 868 children who met the inclusion criteria, 741 (85{\%}) had both echocardiograms. At 2 years, 22{\%} had recovered normal LV function and size; 51{\%} had died or undergone heart transplantation (median, 3.2 months), and 27{\%} had persistently abnormal echocardiograms. Younger age (hazard ratio [HR]: 0.92; 95{\%} confidence interval [CI]: 0.88 to 0.97) and lower LVEDD z-score (HR: 0.78; 95{\%} CI: 0.70 to 0.87) independently predicted normalization. Nine children (9{\%}) with normal LV function and size within 2 years of diagnosis later underwent heart transplantation or died. Conclusions Despite marked LV dilation and depressed function initially, children with idiopathic DCM can recover normal LV size and function, particularly those younger and with less LV dilation at diagnosis. Investigations related to predictors of recovery, such as genetic associations, serum markers, and the impact of medical therapy or ventricular unloading with assist devices are important next steps. Longer follow-up after normalization is warranted as cardiac failure can recur. (Pediatric Cardiomyopathy Registry; NCT00005391).",
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T1 - Recovery of echocardiographic function in children with idiopathic dilated cardiomyopathy

T2 - Results from the pediatric cardiomyopathy registry

AU - Everitt, Melanie D.

AU - Sleeper, Lynn A.

AU - Lu, Minmin

AU - Canter, Charles E.

AU - Pahl, Elfriede

AU - Wilkinson, James D.

AU - Addonizio, Linda J.

AU - Towbin, Jeffrey A.

AU - Rossano, Joseph

AU - Singh, Rakesh K.

AU - Lamour, Jacqueline M.

AU - Webber, Steven A.

AU - Colan, Steven D.

AU - Margossian, Renee

AU - Kantor, Paul F.

AU - Jefferies, John L.

AU - Lipshultz, Steven E.

PY - 2014/4/15

Y1 - 2014/4/15

N2 - Objectives This study sought to determine the incidence and predictors of recovery of normal echocardiographic function among children with idiopathic dilated cardiomyopathy (DCM). Background Most children with idiopathic DCM have poor outcomes; however, some improve. Methods We studied children <18 years of age from the Pediatric Cardiomyopathy Registry who had both depressed left ventricular (LV) function (fractional shortening or ejection fraction z-score <-2) and LV dilation (end-diastolic dimension [LVEDD] z-score >2) at diagnosis and who had at least 1 follow-up echocardiogram 30 days to 2 years from the initial echocardiogram. We estimated the cumulative incidence and predictors of normalization. Results Among 868 children who met the inclusion criteria, 741 (85%) had both echocardiograms. At 2 years, 22% had recovered normal LV function and size; 51% had died or undergone heart transplantation (median, 3.2 months), and 27% had persistently abnormal echocardiograms. Younger age (hazard ratio [HR]: 0.92; 95% confidence interval [CI]: 0.88 to 0.97) and lower LVEDD z-score (HR: 0.78; 95% CI: 0.70 to 0.87) independently predicted normalization. Nine children (9%) with normal LV function and size within 2 years of diagnosis later underwent heart transplantation or died. Conclusions Despite marked LV dilation and depressed function initially, children with idiopathic DCM can recover normal LV size and function, particularly those younger and with less LV dilation at diagnosis. Investigations related to predictors of recovery, such as genetic associations, serum markers, and the impact of medical therapy or ventricular unloading with assist devices are important next steps. Longer follow-up after normalization is warranted as cardiac failure can recur. (Pediatric Cardiomyopathy Registry; NCT00005391).

AB - Objectives This study sought to determine the incidence and predictors of recovery of normal echocardiographic function among children with idiopathic dilated cardiomyopathy (DCM). Background Most children with idiopathic DCM have poor outcomes; however, some improve. Methods We studied children <18 years of age from the Pediatric Cardiomyopathy Registry who had both depressed left ventricular (LV) function (fractional shortening or ejection fraction z-score <-2) and LV dilation (end-diastolic dimension [LVEDD] z-score >2) at diagnosis and who had at least 1 follow-up echocardiogram 30 days to 2 years from the initial echocardiogram. We estimated the cumulative incidence and predictors of normalization. Results Among 868 children who met the inclusion criteria, 741 (85%) had both echocardiograms. At 2 years, 22% had recovered normal LV function and size; 51% had died or undergone heart transplantation (median, 3.2 months), and 27% had persistently abnormal echocardiograms. Younger age (hazard ratio [HR]: 0.92; 95% confidence interval [CI]: 0.88 to 0.97) and lower LVEDD z-score (HR: 0.78; 95% CI: 0.70 to 0.87) independently predicted normalization. Nine children (9%) with normal LV function and size within 2 years of diagnosis later underwent heart transplantation or died. Conclusions Despite marked LV dilation and depressed function initially, children with idiopathic DCM can recover normal LV size and function, particularly those younger and with less LV dilation at diagnosis. Investigations related to predictors of recovery, such as genetic associations, serum markers, and the impact of medical therapy or ventricular unloading with assist devices are important next steps. Longer follow-up after normalization is warranted as cardiac failure can recur. (Pediatric Cardiomyopathy Registry; NCT00005391).

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