Current outcomes in US children with cardiomyopathy listed for heart transplantation.

BACKGROUND Previous studies have reported worse outcomes in children with nondilated cardiomyopathy (CMP) listed for heart transplant compared with children with dilated CMP. We sought to compare wait-list and posttransplant outcomes in these groups in the current era. METHODS AND RESULTS We analyzed all children <18 years of age with a diagnosis of CMP listed for heart transplant in the United States between July 2004 and December 2010. Multivariable risk factors for death on the wait-list (or becoming too sick to transplant) and posttransplant graft loss (median follow-up 2 years) were assessed using Cox models. Of the 1436 children analyzed, 1197 (83%) had dilated CMP and 239 (17%) had nondilated CMP (167 restrictive CMP, 72 hypertrophic CMP). In adjusted analysis, children with nondilated CMP were at higher risk of wait-list mortality only if they were on a ventilator support at listing (hazard ratio, 2.3; CI, 1.2-4.5). The risk was similar among children not on a ventilator support (hazard ratio, 0.6; CI, 0.3-1.1). Posttransplant 30-day and 1-year survival was 98% and 94%, respectively, in children with dilated CMP versus 95% and 89%, respectively, in children with nondilated CMP (P=0.17, log-rank test). In adjusted analysis, the risk of posttransplant graft loss was higher in nondilated CMP (hazard ratio, 1.8; CI, 1.2-2.7) versus dilated CMP. CONCLUSIONS The increased risk of wait-list mortality in children with nondilated CMP is limited to those on ventilator support at listing. Although the risk of graft loss is modestly higher in children with nondilated forms of CMP, their short-term transplant outcomes are good.