Abstract 11789: Intracoronary Delivery of Autologous Cardiac Progenitor Cells in Children With Hypoplastic Left Heart Syndrome: The Ticap Trial With 18-Month Follow Up
Backgrounds: Hypoplastic left heart syndrome (HLHS) is one of the severe malformations in congenital heart disease. This study is to investigate whether intracoronary delivery of autologous cardiosphere-derived cells (CDCs) is feasible and safe to treat the children with HLHS.
Methods and Design: This phase 1 trial (TICAP: NCT01273857) is a prospective controlled study. Four-teen patients with HLHS who are undergoing staged-2 or -3 surgical palliations were enrolled between January, 2011, and January, 2012. Seven patients assigned to receive intracoronary CDCs infusion 1 month after the shunt procedures followed by 7 patients allocated to a control group with standard palliations. The primary endpoint was to assess the safety and the secondary endpoint was to evaluate the cardiac function and heart failure status from the baseline through 18 months of follow-up.
Results: No complications, including cardiac death, myocardial ischemia, arrhythmia, re-hospitalization, and tumor formation, were reported within 18 months after CDCs infusion. Echocardiography showed that the absolute improvement of right ventricular ejection fraction (RVEF) was greater in the CDCs-treated group (+7.1±5.5%) than in controls (+2.1±0.7%, P=0.04) at 18 months. Compared with controls, cMRI analysis showed that patients with CDCs infusion had significantly increased RVEF (31.5±6.8% vs 40.4±7.6%, P=0.04) and reduced end-systolic volume index at 18 months (P=0.049). The improved mechanical output was addressed by a gain of end-systolic elastance (P=0.03) and ventriculo-arterial coupling (P=0.02) in CDC-treated group at 18-month compared with baseline. The increased cardiac performance in long-term resulted in greater somatic growth (weight-for-age z score, P=0.02), reduced heart failure status assessed by Ross classification (P=0.004) and NYU PHF index (P=0.04), decrease in BNP levels (P=0.049), and significantly lower incidence of coil occlusion for collaterals (P=0.007) by 18 months after CDC transfer than controls.
Conclusion: Transcoronary infusion of CDCs appeared to be feasible and safe to treat the patients with HLHS. These initial results provide the basis for larger studies to assess the efficacy of this novel therapeutic approach in children.
Author Disclosures: S. Ishigami: None. S. Tarui: None. M. Okuyama: None. D. Ousaka: None. S. Ohtsuki: None. T. Eitoku: None. J. Kobayashi: None. S. Kasahara: None. S. Sano: None. H. Oh: None.
This research has received full or partial funding support from the American Heart Association.
- © 2014 by American Heart Association, Inc.