Author + information
- Received September 7, 2013
- Revision received October 31, 2013
- Accepted November 5, 2013
- Published online April 15, 2014.
- Melanie D. Everitt, MD∗∗ (, )
- Lynn A. Sleeper, ScD†,
- Minmin Lu, MS†,
- Charles E. Canter, MD‡,
- Elfriede Pahl, MD§,
- James D. Wilkinson, MD, MPH‖,
- Linda J. Addonizio, MD¶,
- Jeffrey A. Towbin, MD#,
- Joseph Rossano, MD∗∗,
- Rakesh K. Singh, MD, MS††,
- Jacqueline Lamour, MD††,
- Steven A. Webber, MBChB‡‡,
- Steven D. Colan, MD§§,
- Renee Margossian, MD§§,
- Paul F. Kantor, MBBCh‖‖,
- John L. Jefferies, MD††,
- Steven E. Lipshultz, MD¶,
- Pediatric Cardiomyopathy Registry Investigators
- ∗Primary Children's Medical Center, Salt Lake City, Utah
- †New England Research Institutes, Inc., Watertown, Massachusetts
- ‡Washington University, St. Louis, Missouri
- §Ann & Robert H. Lurie Children’s Hospital, Chicago, Illinois
- ‖Department of Pediatrics, Miller School of Medicine, University of Miami, Miami, Florida
- ¶Columbia University, New York, New York
- #The Heart Institute, Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio
- ∗∗University of Pennsylvania, Philadelphia, Pennsylvania
- ††Children's Hospital at Montefiore, Bronx, New York
- ‡‡Vanderbilt University, Nashville, Tennessee
- §§Boston Children's Hospital, Boston, Massachusetts
- ‖‖University of Alberta, Stollery Children's Hospital, Edmonton, Alberta, Canada
- ↵∗Reprint requests and correspondence:
Dr. Melanie D. Everitt, Primary Children’s Medical Center, University of Utah, Division of Cardiology, Suite 1500, 100 North Mario Capecchi Drive, Salt Lake City, Utah 84113.
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)
This research was supported by grants from the National Heart, Lung, and Blood Institute (HL 53392 and NHLBI R01 087000) and the Children’s Cardiomyopathy Foundation. Dr. Towbin has received research grant(s) from the National Institutes of Health. The contents of this publication are solely the responsibility of the authors and do not necessarily represent the official views of the National Heart, Lung, and Blood Institute. All other authors have reported that they have no relationships relevant to the contents of this paper to disclose.
- Received September 7, 2013.
- Revision received October 31, 2013.
- Accepted November 5, 2013.
- American College of Cardiology Foundation