Abstract 11909: Transcoronary Infusion of Cardiac Progenitor Cells in Hypoplastic Left Heart Syndrome: 3-year Results of the TICAP Trial
Backgrounds: Hypoplastic left heart syndrome (HLHS) is still one of the severe congenital heart defects. The first results of the TICAP trial (NCT01273857) conducted in our hospital have shown that intracoronary infusion of cardiosphere-derived cells (CDCs) in children with HLHS was feasible and safe; however, the mid-term safety and efficacy of CDCs infusion in these patients remain unsolved. The aim of this study is to evaluate the safety and efficacy of CDCs injection during mid-term period and to determine the factors that may be related to the beneficial effects of cardiac function after cell injection.
Methods and Design: This phase 1 trial is a prospective controlled study. Fourteen patients with HLHS undergoing staged-2 or -3 palliations were enrolled between January 2011, and January 2012. Seven patients assigned to receive intracoronary CDCs infusion 1 month after the cardiac surgery followed by 7 patients allocated to a control with standard care alone. The primary endpoint was to assess the safety and the secondary endpoint was to evaluate the cardiac function and heart failure status.
Results: No complications including tumor formation were reported within 3 years after CDC infusion. Echocardiogram showed a significantly greater improvement in right ventricular ejection fraction (RVEF) in children receiving CDCs than in controls at 3 years (+8.0 ± 4.7% vs. +2.2 ± 4.3%, P=0.03). In cardiac MRI study, improvement of RVEF at 3 years was significantly greater in the CDC-treated group than in controls (+5.7 ± 4.9 vs. -1.0 ± 4.6%, P=0.04). These cardiac function improvements resulted in reduced brain natriuretic peptide levels (P=0.04), lower incidence of unplanned catheter interventions (P=0.04), and higher weight-for-age Z score (WAZ) (P=0.02) at 3 years compared with those of controls. As independent predictors of treatment responsiveness, absolute changes in RVEF at 3 years were negatively correlated with age, WAZ, and RVEF at CDC infusion (age: r=-0.91, P=0.005; WAZ: r=-0.88, P=0.009; EF: r=-0.90, P=0.006).
Conclusions: Intracoronary CDC infusion in patients with HLHS was safe and improved RVEF during 3 years of follow-up. This therapeutic strategy may merit somatic growth enhancement and reduce the incidence of heart failure.
- Hypo plastic left heart syndrome
- heart failure
- cardiosphere derived cells
- cardiac regeneration
- mid-term follow up
Author Disclosures: S. Ishigami: None. S. Tarui: None. T. Goto: None. D. Ousaka: None. K. Baba: None. S. Kasahara: None. S. Ohtsuki: None. S. Sano: None. H. Oh: None.
- © 2015 by American Heart Association, Inc.