title: Predictors of outcomes in children awaiting heart transplantation: an experience from a National Paediatric Heart Transplantation Programme.
abstract: OBJECTIVE
  The risk of mortality while awaiting heart transplantation (HT) may be greater in the setting of a smaller population and lower donor organ rates. Optimizing allocation policy and identifying the most vulnerable patients will help reduce mortality. As such, we aimed to evaluate the predictors of outcomes in patients awaiting HT as part of a National Paediatric HT Programme.
  
  
  METHODS
  Between 1988 and 2015, 200 children were listed for HT as part of a National Paediatric HT Programme. We categorized patients as experiencing one of four competing events: (i) transplant, (ii) death, (iii) delisting for clinical deterioration and (iv) delisting for clinical improvement. Comparison was made across three study eras: Era 1: 1988-95; Era 2: 1996-2005; Era 3: 2006-15. A multivariable competing risk regression analysis was performed to determine the independent predictors of transplantation and the composite event of death or delisting for clinical deterioration.
  
  
  RESULTS
  Among the 200 patients listed, 60% (119/200) underwent transplantation, 22% (44/200) died on the waiting list, 7% (13/200) were delisted for clinical deterioration and 11% (21/200) were delisted due to clinical improvement, with 2% (3/200) being still active on the waiting list. The mortality-rate for patients who remained on the waiting list decreased from 27% in Era 1 to 18% in Era 3. The survival from wait-listing was 57, 47 and 39% at 5, 10 and 15 years, respectively. On multivariable competing risk regression, older age (HR 1.09, 95% CI 1.01-1.18, P = 0.029) and the absence of inotropic support (HR 2.22, 1.23-4.00, P = 0.0073) were predictors of reaching transplantation. Higher creatinine (per 20 µmol/l, HR 1.42, 1.03-1.95, P = 0.03) was a predictor of the composite endpoint of death or delisting for deterioration.
  
  
  CONCLUSIONS
  In this analysis of a National Paediatric HT Programme, waiting-list mortality has decreased over time though it remains higher than countries with higher donor rates. The requirement for inotropic support and worsening renal function were predictors of failure to reach transplantation. These patients are likely to benefit the most from ventricular-assist device therapy and higher priority listing.
authors.affiliation: Department of Cardiac Surgery, Royal Children's Hospital, Melbourne, Australia Murdoch Children's Research Institute, Melbourne, Australia University of Melbourne, Melbourne, Australia.
authors.forename: William Y
authors.initials: WY
authors.lastname: Shi
authors.affiliation: Department of Cardiac Surgery, Royal Children's Hospital, Melbourne, Australia.
authors.forename: Michael
authors.initials: M
authors.lastname: Rouse
authors.affiliation: Murdoch Children's Research Institute, Melbourne, Australia University of Melbourne, Melbourne, Australia Department of Cardiology, Royal Children's Hospital, Melbourne, Australia.
authors.forename: Robert G
authors.initials: RG
authors.lastname: Weintraub
authors.affiliation: Murdoch Children's Research Institute, Melbourne, Australia University of Melbourne, Melbourne, Australia.
authors.forename: Diana
authors.initials: D
authors.lastname: Zannino
authors.affiliation: Department of Cardiology, Royal Children's Hospital, Melbourne, Australia.
authors.forename: Anne
authors.initials: A
authors.lastname: Shipp
authors.affiliation: Department of Cardiac Surgery, Royal Children's Hospital, Melbourne, Australia Murdoch Children's Research Institute, Melbourne, Australia University of Melbourne, Melbourne, Australia.
authors.forename: Yves
authors.initials: Y
authors.lastname: d'Udekem
authors.affiliation: Department of Cardiac Surgery, Royal Children's Hospital, Melbourne, Australia Murdoch Children's Research Institute, Melbourne, Australia University of Melbourne, Melbourne, Australia igor.konstantinov@rch.org.au.
authors.forename: Igor E
authors.initials: IE
authors.lastname: Konstantinov
mesh_terms: D000293:Adolescent; D015415:Biomarkers / Q000097:blood; D009202:Cardiomyopathies / Q000401:mortality / Q000601:surgery*; D002648:Child; D002675:Child, Preschool; D003404:Creatinine / Q000097:blood; D015199:Extracorporeal Membrane Oxygenation; D005260:Female; D006330:Heart Defects, Congenital / Q000401:mortality / Q000601:surgery*; D016027:Heart Transplantation / Q000379:methods*; D006801:Humans; D007223:Infant; D008297:Male; D009313:National Health Programs; D011379:Prognosis; D012189:Retrospective Studies; D016896:Treatment Outcome; D014739:Victoria / Q000453:epidemiology; D014850:Waiting Lists
chemical_list: D015415:Biomarkers; D003404:Creatinine
keywords: Cardiac transplantation; Competing risk; Congenital heart disease; Heart failure; Paediatric; Paediatric cardiac surgery; Transplant waiting list; Waiting list