Abstract 13241: Long-Term Clinical Outcome After Transcoronary Cardiosphere-Derived Cell Infusion in Patients With Single Ventricle Congenital Heart Disease

Abstract

Introduction: Although transcoronary infusion of cardiosphere-derived cells (CDCs) in patients with single ventricles provides short-term improvement in cardiac function, the long-term efficacy is still unknown. Hypothesis: We tested the hypothesis that CDC infusion is associated with improved long-term clinical outcome in patients with single ventricles and heart failure. Methods: Patients were enrolled in the TICAP phase 1 (Transcoronary Infusion of Cardiac Progenitor Cells in Patients With Single Ventricle Physiology) and PERSEUS phase 2 (Cardiac Progenitor Cell Infusion to Treat Univentricular Heart Disease) trials between January 5, 2011, and January 30, 2015. The inclusion criteria were that patients were aged <6 years who were diagnosed with single ventricle lesions undergoing stage 2 (bidirectional Glenn) or stage 3 (total cavopulmonary connection) palliation. Medical records of patients with single ventricles were obtained and analyzed as a control group who were eligible but were treated by staged palliation alone. We evaluated the effectiveness of CDCs using an integrated cohort study in 93 patients with single ventricles, including 40 patients who received CDC infusion and 53 controls. Results: At the time of this study, 35 patients with CDC infusion and 43 controls were alive without cardiac transplantation, with mean follow-up of 10.0±1.2 years. The absolute changes in brain natriuretic peptide ( P = 0.015), atrioventricular regurgitation grade ( P = 0.018), and weight for age ( P < 0.001) from baseline to last follow-up showed significant improvements in CDC-treated patients compared with controls. Survival rate did not differ between the 2 groups (log-rank P = 0.351), whereas overall patients treated by CDCs had lower incidents of late failure ( P = 0.043) and adverse events ( P = 0.036) compared with controls. Multivariate proportional hazard regression analyses showed that CDC infusion was associated with lower risks of late failure ( P = 0.003) and adverse events ( P = 0.013). Conclusions: Intracoronary delivery of CDCs in patients with single ventricles did not improve all-cause mortality rate but significantly reduced the incidence of late failure and adverse events at 8 years of follow-up.