Rakesh K Singh1, Charles E Canter2, Ling Shi3, Steven D Colan4, Debra A Dodd5, Melanie D Everitt6, Daphne T Hsu7, John L Jefferies8, Paul F Kantor9, Elfriede Pahl10, Joseph W Rossano11, Jeffrey A Towbin12, James D Wilkinson13, Steven E Lipshultz13. 1. Department of Pediatrics, University of California-San Diego and Rady Children's Hospital, San Diego, California. Electronic address: rsingh@rchsd.org. 2. Department of Pediatrics, Washington University School of Medicine, St. Louis, Missouri. 3. New England Research Institutes, Watertown, Massachusetts. 4. Department of Pediatrics, Boston's Children's Hospital, Boston, Massachusetts. 5. Department of Pediatrics, Vanderbilt University and Monroe Carell Jr. Children's Hospital, Nashville, Tennessee. 6. Department of Pediatrics, Children's Hospital Colorado and University of Colorado, Aurora, Colorado. 7. Department of Pediatrics, Children's Hospital at Montefiore, Bronx, New York. 8. Department of Pediatrics, The Heart Institute, Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio. 9. Department of Pediatrics, Stollery Children's Hospital, Edmonton, Alberta, Canada. 10. Department of Pediatrics, Ann and Robert H. Lurie Children's Hospital of Chicago, Chicago, Illinois. 11. Department of Pediatrics, The Children's Hospital of Philadelphia, Philadelphia, Pennsylvania. 12. Department of Pediatrics, Le Bonheur Children's Hospital, Memphis, Tennessee. 13. Department of Pediatrics, Wayne State University School of Medicine and Children's Hospital of Michigan, Detroit, Michigan.
Abstract
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).
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).
Authors: M A Grenier; S K Osganian; G F Cox; J A Towbin; S D Colan; P R Lurie; L A Sleeper; E J Orav; S E Lipshultz Journal: Am Heart J Date: 2000-02 Impact factor: 4.749
Authors: Piers E F Daubeney; Alan W Nugent; Patty Chondros; John B Carlin; Steven D Colan; Michael Cheung; Andrew M Davis; C W Chow; Robert G Weintraub Journal: Circulation Date: 2006-11-20 Impact factor: 29.690
Authors: Jeffrey A Towbin; April M Lowe; Steven D Colan; Lynn A Sleeper; E John Orav; Sarah Clunie; Jane Messere; Gerald F Cox; Paul R Lurie; Daphne Hsu; Charles Canter; James D Wilkinson; Steven E Lipshultz Journal: JAMA Date: 2006-10-18 Impact factor: 56.272
Authors: Barry J Maron; Jeffrey A Towbin; Gaetano Thiene; Charles Antzelevitch; Domenico Corrado; Donna Arnett; Arthur J Moss; Christine E Seidman; James B Young Journal: Circulation Date: 2006-03-27 Impact factor: 29.690
Authors: Susan R Foerster; Charles E Canter; Amy Cinar; Lynn A Sleeper; Steven A Webber; Elfriede Pahl; Paul F Kantor; Jorge A Alvarez; Steven D Colan; John L Jefferies; Jacqueline M Lamour; Renee Margossian; Jane E Messere; Paolo G Rusconi; Robert E Shaddy; Jeffrey A Towbin; James D Wilkinson; Steven E Lipshultz Journal: Circ Heart Fail Date: 2010-09-10 Impact factor: 8.790
Authors: Elizabeth D Blume; David C Naftel; Heather J Bastardi; Brian W Duncan; James K Kirklin; Steven A Webber Journal: Circulation Date: 2006-05-16 Impact factor: 29.690
Authors: M Giulia Gagliardi; M Bevilacqua; C Bassano; B Leonardi; R Boldrini; F Diomedi Camassei; A Fierabracci; A G Ugazio; G F Bottazzo Journal: Heart Date: 2004-10 Impact factor: 5.994
Authors: Kimberly M Molina; Peter Shrader; Steven D Colan; Seema Mital; Renee Margossian; Lynn A Sleeper; Girish Shirali; Piers Barker; Charles E Canter; Karen Altmann; Elizabeth Radojewski; Elif Seda Selamet Tierney; Jack Rychik; Lloyd Y Tani Journal: Circ Heart Fail Date: 2013-10-16 Impact factor: 8.790
Authors: Kory J Lavine; Kathleen E Simpson; Melanie R F Gropler; Steven E Lipshultz; James D Wilkinson; Jeffrey A Towbin; Steven D Colan; Charles E Canter Journal: Pediatr Res Date: 2021-08-17 Impact factor: 3.953
Authors: Rabia S Khan; Elfriede Pahl; Lisa Dellefave-Castillo; Karen Rychlik; Alexander Ing; Kai Lee Yap; Casey Brew; Jamie R Johnston; Elizabeth M McNally; Gregory Webster Journal: J Am Heart Assoc Date: 2021-12-22 Impact factor: 5.501
Authors: Emily Morell; Mary Katherine Miller; Minmin Lu; Kevin G Friedman; Roger E Breitbart; Jeffrey R Reichman; Julie McDermott; Lynn A Sleeper; Elizabeth D Blume Journal: J Am Heart Assoc Date: 2021-01-14 Impact factor: 5.501
Authors: Stephanie J Nakano; John S Walker; Lori A Walker; Xiaotao Li; Yanmei Du; Shelley D Miyamoto; Carmen C Sucharov; Anastacia M Garcia; Max B Mitchell; Amrut V Ambardekar; Brian L Stauffer Journal: Am J Physiol Heart Circ Physiol Date: 2019-10-18 Impact factor: 4.733
Authors: Joseph W Rossano; Paul F Kantor; Robert E Shaddy; Ling Shi; James D Wilkinson; John L Jefferies; Jason D Czachor; Hiedy Razoky; Heidi S Wirtz; Christophe Depre; Steven E Lipshultz Journal: J Am Heart Assoc Date: 2020-07-30 Impact factor: 5.501