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Background: The course of pediatric hypertrophic cardiomyopathy (HCM) is variable, and the indications for heart transplantation (HTx) are ill defined. This study investigated outcomes and risk factors for adverse outcomes after listing for HTx., Methods: A multicenter, event-driven data registry of patients aged < 18 years listed for HTx between January 1993 and December 2007 was used., Results: During the study period, 3,147 children were listed for HTx (mean age, 7.6 +/- 0.8 years). Of l,320 with CM at listing, 77 (6%) had HCM (61% boys; 79% white); 59% were United Network of Organ Sharing (UNOS) status I, 30% were receiving inotropes, 27% were ventilated, and 8% required extracorporeal membrane oxygenation. Arrhythmia had occurred in 27%, and 14% had failure to thrive. Within 1 year, 65% underwent HTx. Overall, 25 patients died after listing: 11 (14%) while waiting and 14 of 49 (29%) after HTx. Pre-HTx survival was lower for those listed at age < 1 year (p = 0.0005). Risk factors for death after listing included UNOS status 1 (p = 0.01) and younger age (relative risk, 2.3; p = 0.001). Late (10-year) survival after HTx for HCM patients was 47% vs 63% for non-CM patients within the database., Conclusions: Children with HCM listed for HTx age < 1 year and UNOS status 1 have the highest mortality awaiting HTx. A more rigorous identification of additional risk factors should be performed to better define timing of listing and which patient sub-group may derive optimal benefit from HTx.

Background: Restrictive cardiomyopathy (RCM) in children often has a progressive nature, with a high risk of clinical deterioration and death. Heart transplantation (HTx) is a widely accepted therapy that offers long-term survival, but criteria for and outcomes after listing have not been well defined., Methods: A multi-institutional, prospective, event-driven data registry of 3,147 patients aged < 18 years listed for HTx from January 1993 to December 2006 was used to assess risk factors and survival of 145 listed RCM patients., Results: Mean age at listing was 8.1 years, with 44% listed as United Network of Organ Sharing status 1, 33% on inotropic support, 10% on a ventilator, and 5% on mechanical support. At 1 year, 82% of these patients survived to HTx, whereas 9% died waiting. Univariate risk factors for death while waiting included younger age (p < 0.001), ventilator dependence (p < 0.001), status 1 (p < 0.001), and inotrope usage (p < 0.001). Use of multiple support devices at listing (ventilator, extracorporeal membrane oxygenation, ventricular assist device, intraaortic balloon pump) was also an important risk factor for early phase death while waiting (relative risk; 9.01, p < 0.0001). Survival after listing was 63% at 10 years and compared favorably with survival for non-cardiomyopathy patients (p = 0.01)., Conclusions: Children with RCM awaiting HTx have a generally low waitlist mortality and reasonable overall survival. Children requiring mechanical support and infants had a significantly higher risk of death while waiting. Further study is warranted to identify factors important in determining the optimal timing of listing in children with RCM before the need for inotropic or mechanical support.