Survival and quality of life for children with end-stage heart failure who are not candidates for cardiac transplant.

BACKGROUND: Some pediatric patients referred for heart transplant (HTx) are sub-optimal candidates. Their outcomes without HTx are presumed to be dismal, but have not been well described. Knowledge about their outcomes is critical when weighing the risks between a high-risk transplant and "terminal" palliation. METHODS: We retrospectively reviewed all HTx referrals from January 2005 to July 2013. We excluded those who were listed for HTx, or who were denied HTx due to being "too well," seeking only those who were in need of but not suitable for HTx. End-points included mortality and length of survival. RESULTS: Of 212 referrals, 39 (19%) (age 0 to 19 years, median 3.5 years) were denied HTx for reasons other than being too well. Twenty-eight (72%) had palliated congenital heart disease. Overall mortality during the follow-up period was 38% (n = 15) with a median follow-up time of 195 days (8 to 2,832 days). Ten patients received subsequent cardiac surgery with 1 death (10%) and median follow-up of 2.6 years. Mortality risk was not influenced by age, weight, growth failure, congenital heart disease or single-ventricle physiology. Mechanical ventilation (hazard ratio 6.31, p = 0.001) and inotrope dependence (hazard ratio 4.79, p = 0.006) were associated with the highest risk of mortality. Quality of life was measured with the PedsQL cardiac module and completed by 11 of 16 eligible patients with an overall average score of 70.2 ± 23.9. CONCLUSIONS: An advanced heart failure program can achieve satisfactory results for pediatric patients who are not suitable candidates for HTx. For some children, high-risk palliative surgery can result in better outcome than high-risk HTx. Mortality was related to the degree of heart failure at presentation rather than underlying heart disease.

Interagency Registry for Mechanically Assisted Circulatory Support (INTERMACS)-defined morbidity and mortality associated with pediatric ventricular assist device support at a single US center: the Stanford experience.

BACKGROUND: The use of ventricular assist devices (VADs) to bridge pediatric patients to heart transplantation has increased dramatically over the last 15 years. In this report, we present the largest US single-center report of pediatric VAD use to date. We present detailed descriptions of morbidity and mortality associated with VAD support, using standard Interagency Registry for Mechanically Assisted Circulatory Support (INTERMACS) criteria for pediatrics to facilitate the comparison of these results to other studies. METHODS AND RESULTS: We retrospectively identified 25 patients younger than 18 years with 27 episodes of mechanical circulatory support using VADs as bridge to heart transplantation from January 1998 to December 2007. Survival to transplant for the entire cohort was 74%. The most common major morbidities, as defined by INTERMACS criteria for a pediatric population, were respiratory failure, major localized infections, major bleeding events, hepatic dysfunction, and right heart failure. Major neurological events occurred in 48% of the study population. The median time to the first occurrence of an adverse event was less than 14 days for respiratory failure, right heart failure, major localized infection, and major bleeding. Patients who died before transplantation had significantly more adverse events per day of support than did those who were successfully transplanted. Episodes of major bleeding, tamponade, acute renal failure, respiratory failure, and right heart failure were all associated with increased risk of mortality. CONCLUSIONS: INTERMACS criteria can be successfully used to analyze pediatric VAD outcomes. These data serve as a baseline for future studies of VAD support in children and indicate good survival rates but considerable morbidity.