Author + information
- Received June 1, 2017
- Revision received August 30, 2017
- Accepted September 18, 2017
- Published online November 20, 2017.
- Rakesh K. Singh, MD, MSa,∗ (, )
- Charles E. Canter, MDb,
- Ling Shi, PhDc,
- Steven D. Colan, MDd,
- Debra A. Dodd, MDe,
- Melanie D. Everitt, MDf,
- Daphne T. Hsu, MDg,
- John L. Jefferies, MD, MPHh,
- Paul F. Kantor, MBBChi,
- Elfriede Pahl, MDj,
- Joseph W. Rossano, MD, MSk,
- Jeffrey A. Towbin, MDl,
- James D. Wilkinson, MD, MPHm,
- Steven E. Lipshultz, MDm,
- for the Pediatric Cardiomyopathy Registry Investigators
- aDepartment of Pediatrics, University of California–San Diego and Rady Children’s Hospital, San Diego, California
- bDepartment of Pediatrics, Washington University School of Medicine, St. Louis, Missouri
- cNew England Research Institutes, Watertown, Massachusetts
- dDepartment of Pediatrics, Boston’s Children's Hospital, Boston, Massachusetts
- eDepartment of Pediatrics, Vanderbilt University and Monroe Carell Jr. Children's Hospital, Nashville, Tennessee
- fDepartment of Pediatrics, Children’s Hospital Colorado and University of Colorado, Aurora, Colorado
- gDepartment of Pediatrics, Children's Hospital at Montefiore, Bronx, New York
- hDepartment of Pediatrics, The Heart Institute, Cincinnati Children’s Hospital Medical Center, Cincinnati, Ohio
- iDepartment of Pediatrics, Stollery Children’s Hospital, Edmonton, Alberta, Canada
- jDepartment of Pediatrics, Ann and Robert H. Lurie Children's Hospital of Chicago, Chicago, Illinois
- kDepartment of Pediatrics, The Children’s Hospital of Philadelphia, Philadelphia, Pennsylvania
- lDepartment of Pediatrics, Le Bonheur Children’s Hospital, Memphis, Tennessee
- mDepartment of Pediatrics, Wayne State University School of Medicine and Children’s Hospital of Michigan, Detroit, Michigan
- ↵∗Address for correspondence:
Dr. Rakesh K. Singh, Heart Failure and Transplantation, Rady Children’s Hospital, Division of Cardiology, 7960 Birmingham Drive, San Diego, California 92123.
Background Studies of children with dilated cardiomyopathy (DCM) have suggested that improved survival has been primarily due to utilization of heart transplantation.
Objectives This study sought to determine transplant-free survival for these children over 20 years and identify the clinical characteristics at diagnosis that predicted death.
Methods Children <18 years of age with some type of DCM enrolled in the Pediatric Cardiomyopathy Registry were divided by year of diagnosis into an early cohort (1990 to 1999) and a late cohort (2000 to 2009). Competing risks and multivariable modeling were used to estimate the cumulative incidence of death, transplant, and echocardiographic normalization by cohort and to identify the factors associated with death.
Results Of 1,953 children, 1,199 were in the early cohort and 754 were in the late cohort. Most children in both cohorts had idiopathic DCM (64% vs. 63%, respectively). Median age (1.6 vs. 1.7 years), left ventricular end-diastolic z-scores (+4.2 vs. +4.2), and left ventricular fractional shortening (16% vs. 17%) at diagnosis were similar between cohorts. Although the rates of echocardiographic normalization (30% and 27%) and heart transplantation (24% and 24%) were similar, the death rate was higher in the early cohort than in the late cohort (18% vs. 9%; p = 0.04). Being in the early cohort (hazard ratio: 1.4; 95% confidence interval: 1.04 to 1.9; p = 0.03) independently predicted death.
Conclusions Children with DCM have improved survival in the more recent era. This appears to be associated with factors other than heart transplantation, which was equally prevalent in both eras. (Pediatric Cardiomyopathy Registry [PCMR]; NCT00005391)
Supported by grants from the National Heart, Lung, and Blood Institute (HL53392) and the Children’s Cardiomyopathy Foundation. 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 or the Children’s Cardiomyopathy Foundation. Drs. Canter and Kantor have served as consultants to Novartis. Dr. Rossano has served as a consultant for HeartWare. All other authors have reported that they have no relationships relevant to the contents of this paper to disclose.
- Received June 1, 2017.
- Revision received August 30, 2017.
- Accepted September 18, 2017.
- 2017 American College of Cardiology Foundation
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