title: Management of children with dilated cardiomyopathy in The Netherlands: Implications of a low early transplantation rate.
abstract: BACKGROUND
  The policy for listing and transplant for children with dilated cardiomyopathy (DCM) in The Netherlands has been conservative because of low donor availability. The effects of this policy on outcome are reported.
  
  
  METHODS
  This was a multicenter, nationwide study performed in 148 children with DCM. The primary outcome was death or heart transplant.
  
  
  RESULTS
  Overall, 43 patients (29%) died or were transplanted. Within 1 year of diagnosis, 21 patients died, and only 4 underwent transplantation (3 on mechanical circulatory support). The 1-year survival was 85% (95% confidence interval [CI] = 79-91), and 5-year survival was 84% (95% CI = 78-90). Transplantation-free survival at 1 year was 82% (95% CI = 75-88) and at 5 years was 72% (95% CI = 64-80). Within 1 year of diagnosis, with death as the main end-point (21 of 25, 84%), intensive care unit admission (hazard ratio = 2.6, p = 0.05) and mechanical circulatory support (hazard ratio = 3.2, p = 0.03) were risk factors (multivariable Cox analysis); inotropic support was longer in patients reaching an end-point. At >1 year after diagnosis, with transplantation as the main end-point (15 of 18, 83%), age >6 years (hazard ratio = 6.1, p = 0.02) was a risk factor. There were 56 (38%) children who recovered, 50% within 1 year of diagnosis. Recovery was associated with younger age; was similar in patients with myocarditis (43%) and idiopathic disease (41%); and was similar in patients initially admitted to the intensive care unit, admitted to the ward, or treated as outpatients.
  
  
  CONCLUSIONS
  The transplantation rate in our cohort in the first year was low, with 1-year and 5-year survival rates similar to other cohorts. Our results suggest that a conservative approach to list children for transplantation early after presentation may be justifiable except for patients with prolonged intensive care unit or mechanical circulatory support.
authors.affiliation: Department of Pediatric Cardiology, Sophia Children's Hospital, Erasmus Medical Center, Rotterdam.
authors.forename: Susanna L
authors.initials: SL
authors.lastname: den Boer
authors.affiliation: Department of Pediatric Cardiology, Sophia Children's Hospital, Erasmus Medical Center, Rotterdam.
authors.forename: M
authors.initials: M
authors.lastname: Lennie van Osch-Gevers
authors.affiliation: Department of Pediatric Cardiology, Sophia Children's Hospital, Erasmus Medical Center, Rotterdam.
authors.forename: Gijs
authors.initials: G
authors.lastname: van Ingen
authors.affiliation: Department of Pediatric Cardiology, Beatrix Children's Hospital, University Medical Center Groningen, Groningen.
authors.forename: Gideon J
authors.initials: GJ
authors.lastname: du Marchie Sarvaas
authors.affiliation: Department of Pediatric Cardiology, Wilhelmina Children's Hospital, University Medical Center Utrecht, Utrecht.
authors.forename: Gabriëlle G
authors.initials: GG
authors.lastname: van Iperen
authors.affiliation: Department of Pediatric Cardiology, Radboud University Medical Center, Nijmegen.
authors.forename: Ronald B
authors.initials: RB
authors.lastname: Tanke
authors.affiliation: Department of Pediatric Cardiology, Emma Children's Hospital, Academic Medical Center, Amsterdam.
authors.forename: Ad P C M
authors.initials: AP
authors.lastname: Backx
authors.affiliation: Department of Pediatric Cardiology, Leiden University Medical Center, Leiden.
authors.forename: Arend D J
authors.initials: AD
authors.lastname: Ten Harkel
authors.affiliation: Department of Pediatric Cardiology, Sophia Children's Hospital, Erasmus Medical Center, Rotterdam.
authors.forename: Willem A
authors.initials: WA
authors.lastname: Helbing
authors.affiliation: Department of Pediatric Cardiology, Maastricht University Medical Center, Maastricht.
authors.forename: Tammo
authors.initials: T
authors.lastname: Delhaas
authors.affiliation: Department of Cardiothoracic Surgery, Erasmus Medical Center, Rotterdam.
authors.forename: Ad J J C
authors.initials: AJ
authors.lastname: Bogers
authors.affiliation: Department of Pediatric Cardiology, Free University Medical Center, Amsterdam, The Netherlands.
authors.forename: Lukas A J
authors.initials: LA
authors.lastname: Rammeloo
authors.affiliation: Department of Pediatric Cardiology, Sophia Children's Hospital, Erasmus Medical Center, Rotterdam. Electronic address: m.dalinghaus@erasmusmc.nl.
authors.forename: Michiel
authors.initials: M
authors.lastname: Dalinghaus
mesh_terms: D000293:Adolescent; D002311:Cardiomyopathy, Dilated / Q000401:mortality / Q000601:surgery*; D002648:Child; D002675:Child, Preschool; D005260:Female; D016027:Heart Transplantation / Q000706:statistics & numerical data*; D006801:Humans; D015994:Incidence; D007223:Infant; D008297:Male; D009426:Netherlands / Q000453:epidemiology; D012042:Registries; D012189:Retrospective Studies; D018570:Risk Assessment / Q000379:methods*; D012307:Risk Factors; D015996:Survival Rate / Q000639:trends; D013997:Time Factors; D014019:Tissue Donors / Q000600:supply & distribution; D014850:Waiting Lists
keywords: cardiomyopathy; heart failure; heart transplantation; pediatric cardiology