title: Competing risks for death and cardiac transplantation in children with dilated cardiomyopathy: results from the pediatric cardiomyopathy registry.
abstract: BACKGROUND
  Pediatric dilated cardiomyopathy (DCM) is the leading indication for heart transplantation after 1 year of age. Risk factors by etiology at clinical presentation have not been determined separately for death and transplantation in population-based studies. Competing risks analysis may inform patient prioritization for transplantation listing.
  
  
  RESULTS
  The Pediatric Cardiomyopathy Registry enrolled 1731 children diagnosed with DCM from 1990 to 2007. Etiologic, demographic, and echocardiographic data collected at diagnosis were analyzed with competing risks methods stratified by DCM etiology to identify predictors of death and transplantation. For idiopathic DCM (n=1192), diagnosis after 6 years of age, congestive heart failure, and lower left ventricular (LV) fractional shortening z score were independently associated with both death and transplantation equally. In contrast, increased LV end-diastolic dimension z score was associated only with transplantation, whereas lower height-for-age z score was associated only with death. For neuromuscular disease (n=139), lower LV fractional shortening was associated equally with both end points, but increased LV end-diastolic dimension was associated only with transplantation. The risks of death and transplantation were increased equally for older age at diagnosis, congestive heart failure, and increased LV end-diastolic dimension among those with myocarditis (n=272) and for congestive heart failure and decreased LV fractional shortening among those with familial DCM (n=79).
  
  
  CONCLUSIONS
  Risk factors for death and transplantation in children varied by DCM etiology. For idiopathic DCM, increased LV end-diastolic dimension was associated with increased transplantation risk but not mortality. Conversely, short stature was significantly related to death but not transplantation. These findings may present an opportunity to improve the transplantation selection algorithm.
authors.affiliation: Department of Pediatrics (D820), Miller School of Medicine, University of Miami, P.O. Box 016820, Miami, FL 33101, USA.
authors.forename: Jorge A
authors.initials: JA
authors.lastname: Alvarez
authors.forename: E John
authors.initials: EJ
authors.lastname: Orav
authors.forename: James D
authors.initials: JD
authors.lastname: Wilkinson
authors.forename: Lora E
authors.initials: LE
authors.lastname: Fleming
authors.forename: David J
authors.initials: DJ
authors.lastname: Lee
authors.forename: Lynn A
authors.initials: LA
authors.lastname: Sleeper
authors.forename: Paolo G
authors.initials: PG
authors.lastname: Rusconi
authors.forename: Steven D
authors.initials: SD
authors.lastname: Colan
authors.forename: Daphne T
authors.initials: DT
authors.lastname: Hsu
authors.forename: Charles E
authors.initials: CE
authors.lastname: Canter
authors.forename: Steven A
authors.initials: SA
authors.lastname: Webber
authors.forename: Gerald F
authors.initials: GF
authors.lastname: Cox
authors.forename: John L
authors.initials: JL
authors.lastname: Jefferies
authors.forename: Jeffrey A
authors.initials: JA
authors.lastname: Towbin
authors.forename: Steven E
authors.initials: SE
authors.lastname: Lipshultz
mesh_terms: D000293:Adolescent; D002311:Cardiomyopathy, Dilated / Q000209:etiology / Q000401:mortality* / Q000601:surgery*; D002648:Child; D002675:Child, Preschool; D005260:Female; D005500:Follow-Up Studies; D016027:Heart Transplantation / Q000401:mortality*; D006801:Humans; D007223:Infant; D007231:Infant, Newborn; D008297:Male; D009017:Morbidity; D009205:Myocarditis / Q000401:mortality; D018579:Patient Selection; D011237:Predictive Value of Tests; D012042:Registries / Q000706:statistics & numerical data*; D018570:Risk Assessment / Q000379:methods; D012307:Risk Factors; D016019:Survival Analysis