Intracoronary Autologous Cardiac Progenitor Cell Transfer in Patients With Hypoplastic Left Heart Syndrome (TICAP): A Prospective Phase 1 Controlled Trial | Journal Scan

Study Questions:

What is the safety and feasibility of intracoronary delivery of autologous cardiosphere-derived cells (CDCs) in patients with hypoplastic left heart syndrome (HLHS)?


A prospective, nonrandomized, controlled trial was performed at a single center. Patients were prospectively assigned to receive intracoronary infusion of autologous CDCs or standard palliation. The primary endpoint was safety, including evaluation for procedural complications including distal coronary embolization, coronary artery injury, and sustained ventricular arrhythmia associated with CDC infusion. The primary safety endpoints at 3 months were death due to ventricular fibrillation, ventricular tachycardia, and myocardial infarction after CDC infusion. Efficacy endpoints included right ventricular (RV) function, somatic growth, heart failure status, and need for coil occlusion of collaterals. CDCs were harvested, validated, and prepared as 3.0 x 105 cells/kg of body weight. Heart catheterization was performed 4-5 weeks after staged shunt procedure, and CDCs were transferred into the coronary arteries using a balloon occlusion technique.


Of a total of 14 patients (1.8 ± 1.5 years) enrolled, 7 patients were assigned to receive intracoronary infusion of autologous CDCs 33.4 ± 8.1 days after staged procedures. There were no procedural complications, and no serious adverse events were reported during the 18-month follow-up period. Patients treated with CDCs showed improvement in RV ejection fraction (RVEF) from baseline to 3-month follow-up (46.9 ± 4.6% to 52.1 ± 2.4%, p = 0.008). Compared with controls at 18 months, cardiac magnetic resonance imaging (MRI) analysis of CDC-treated patients showed a higher RVEF (31.5 ± 6.8% vs. 40.4 ± 7.6%, p = 0.049), improved somatic growth, reduced heart failure status, and lower incidence of coil occlusion of collaterals.


Intracoronary infusion of autologous CDCs appears to be feasible and safe in children with HLHS after staged surgery.


This small phase 1 trial shows promising results of intracoronary infusion of autologous cardiac progenitor cells in infants with HLHS. Recent data from the single-ventricle reconstruction trial suggest that RV dysfunction is common, and likely worse in patients who have undergone RV-to-pulmonary artery shunt as part of their initial palliation. Although the sample size is small and the follow-up is short, this trial suggests safety and possible benefits of this novel therapy. Certainly, many questions remain about this therapy, including the possibility of differing impact based on early surgical repairs, as the majority of patients in this study had undergone RV-to-pulmonary artery shunts.

Clinical Topics: Arrhythmias and Clinical EP, Congenital Heart Disease and Pediatric Cardiology, Heart Failure and Cardiomyopathies, Noninvasive Imaging, SCD/Ventricular Arrhythmias, Congenital Heart Disease, CHD and Pediatrics and Arrhythmias, CHD and Pediatrics and Imaging, Acute Heart Failure, Magnetic Resonance Imaging

Keywords: Balloon Occlusion, Cardiac Catheterization, Heart Failure, Hypoplastic Left Heart Syndrome, Magnetic Resonance Imaging, Myocardial Infarction, Ventricular Function, Pulmonary Artery, Coronary Vessels, Stem Cells, Tachycardia, Ventricular, Ventricular Fibrillation, AHA Annual Scientific Sessions

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