title: Cardiomyopathy Phenotypes and Outcomes for Children With Left Ventricular Myocardial Noncompaction: Results From the Pediatric Cardiomyopathy Registry.
abstract: BACKGROUND
  Left ventricular noncompaction (LVNC) is a distinct form of cardiomyopathy characterized by hypertrabeculation of the left ventricle. The LVNC phenotype may occur in isolation or with other cardiomyopathy phenotypes. Prognosis is incompletely characterized in children.
  
  
  RESULTS
  According to diagnoses from the National Heart, Lung, and Blood Institute-funded Pediatric Cardiomyopathy Registry from 1990 to 2008, 155 of 3,219 children (4.8%) had LVNC. Each LVNC patient was also classified as having an associated echocardiographically diagnosed cardiomyopathy phenotype: dilated (DCM), hypertrophic (HCM), restrictive (RCM), isolated, or indeterminate. The time to death or transplantation differed among the phenotypic groups (P = .035). Time to listing for cardiac transplantation significantly differed by phenotype (P < .001), as did time to transplantation (P = .015). The hazard ratio for death/transplantation (with isolated LVNC as the reference group) was 4.26 (95% confidence interval [CI] 0.78-23.3) for HCM, 6.35 (95% CI 1.52-26.6) for DCM, and 5.66 (95% CI 1.04-30.9) for the indeterminate phenotype. Most events occurred in the 1st year after diagnosis.
  
  
  CONCLUSIONS
  LVNC is present in at least 5% of children with cardiomyopathy. The specific LVNC-associated cardiomyopathy phenotype predicts the risk of death or transplantation and should inform clinical management.
authors.affiliation: Heart Institute, Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio. Electronic address: john.jefferies@cchmc.org.
authors.forename: John L
authors.initials: JL
authors.lastname: Jefferies
authors.affiliation: Department of Pediatrics, Wayne State University School of Medicine, Detroit, Michigan; Department of Pediatrics, University of Miami Miller School of Medicine and Holtz Children's Hospital, Miami, Florida.
authors.forename: James D
authors.initials: JD
authors.lastname: Wilkinson
authors.affiliation: New England Research Institutes, Watertown, Massachusetts.
authors.forename: Lynn A
authors.initials: LA
authors.lastname: Sleeper
authors.affiliation: Department of Cardiology, Boston Children's Hospital, Boston, Massachusetts.
authors.forename: Steven D
authors.initials: SD
authors.lastname: Colan
authors.affiliation: New England Research Institutes, Watertown, Massachusetts.
authors.forename: Minmin
authors.initials: M
authors.lastname: Lu
authors.affiliation: Division of Cardiology, Ann and Robert Lurie Children's Hospital, Chicago, Illionis.
authors.forename: Elfriede
authors.initials: E
authors.lastname: Pahl
authors.affiliation: Division of Pediatric Cardiology, Stollery Children's Hospital, University of Alberta, Edmonton, Alberta, Canada.
authors.forename: Paul F
authors.initials: PF
authors.lastname: Kantor
authors.affiliation: Division of Pediatric Cardiology, Primary Children's Hospital, Salt Lake City, Utah.
authors.forename: Melanie D
authors.initials: MD
authors.lastname: Everitt
authors.affiliation: Monroe Carell Jr. Children's Hospital at Vanderbilt, Nashville, Tennessee.
authors.forename: Steven A
authors.initials: SA
authors.lastname: Webber
authors.affiliation: Children's Hospital of Philadelphia, Philadelphia, Pennsylvania.
authors.forename: Beth D
authors.initials: BD
authors.lastname: Kaufman
authors.affiliation: Department of Pediatrics, Montefiore Children's Hospital, Bronx, New York.
authors.forename: Jacqueline M
authors.initials: JM
authors.lastname: Lamour
authors.affiliation: Division of Pediatric Cardiology, St. Louis Children's Hospital, St. Louis, Missouri.
authors.forename: Charles E
authors.initials: CE
authors.lastname: Canter
authors.affiliation: Department of Pediatrics, Montefiore Children's Hospital, Bronx, New York.
authors.forename: Daphne T
authors.initials: DT
authors.lastname: Hsu
authors.affiliation: Division of Pediatric Cardiology, Morgan Stanley Children's Hospital, New York, New York.
authors.forename: Linda J
authors.initials: LJ
authors.lastname: Addonizio
authors.affiliation: Department of Pediatrics, University of Miami Miller School of Medicine and Holtz Children's Hospital, Miami, Florida; Department of Pediatrics, Wayne State University School of Medicine and Children's Hospital of Michigan, Detroit, Michigan.
authors.forename: Steven E
authors.initials: SE
authors.lastname: Lipshultz
authors.affiliation: Heart Institute, Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio; Heart Institute, Le Bonheur Children's Hospital, University of Tennessee Health Science Center, Memphis, Tennessee.
authors.forename: Jeffrey A
authors.initials: JA
authors.lastname: Towbin
mesh_terms: D002170:Canada; D009202:Cardiomyopathies / Q000235:genetics* / Q000401:mortality* / Q000503:physiopathology; D002423:Cause of Death; D002648:Child; D002675:Child, Preschool; D005260:Female; D020022:Genetic Predisposition to Disease / Q000453:epidemiology; D006801:Humans; D015994:Incidence; D056830:Isolated Noncompaction of the Ventricular Myocardium / Q000235:genetics* / Q000401:mortality* / Q000503:physiopathology; D053208:Kaplan-Meier Estimate; D008297:Male; D015999:Multivariate Analysis; D010372:Pediatrics; D010641:Phenotype; D011237:Predictive Value of Tests; D011379:Prognosis; D016016:Proportional Hazards Models; D012042:Registries; D012189:Retrospective Studies; D016019:Survival Analysis; D014481:United States
keywords: Cardiomyopathy; heart failure; pediatrics