Intracoronary delivery of cardiosphere-derived cells shows promise in children with hypoplastic left heart syndrome

CHICAGO — Intracoronary delivery of autologous cardiosphere-derived cells was safe and feasible in children with hypoplastic left heart syndrome.

Shuta Ishigami, MD, of the Okayama University Hospital, Okayama, Japan, and colleagues conducted a prospective, controlled study that enrolled 14 children with hypoplastic left heart syndrome (HLHS). Patients underwent stage 2 or 3 surgical palliations between January 2011 and January 2012, and were assigned to receive either infusion of intracoronary cardiosphere-derived cells (CDCs) 1 month after the shunt procedure (n=7) or standard palliations (control; n=7).

Safety served as the primary endpoint. Secondary endpoints included cardiac function and HF status from baseline to 18 months follow-up.

At 18 months, there were no complications in the infusion arm, including no reported cases of cardiac death, myocardial ischemia, arrhythmia, rehospitalization or tumor formation. Echocardiography demonstrated a greater absolute improvement in right ventricular ejection fraction (RVEF) in the CDCs group (7.1 ± 5.5% vs. 2.1 ± 0.7%; P=.04). The CDCs group also experienced an increase in RVEF (40.4 ± 7.6% vs. 31.5 ± 6.8%; P=.049) and a reduction in end-systolic volume index (P=.048) in cardiac MRI analysis.

In other 18-month data, patients who received CDCs had improved mechanical output as demonstrated by a gain of end-systolic elastance (P=.007) and ventriculo-arterial coupling (P=.006) vs. baseline. This resulted in greater somatic growth (P=.0005), reduced Ross classification HF status (P=.048) and New York University Pediatric Heart Failure index (P=.003), decreased brain natriuretic peptide levels (P=.049) and a lower incidence of coil occlusion for collaterals (P=.007) in the cell-therapy infusion group, according to the researchers.

“Transcoronary infusion of autologous cardiosphere-derived cells appeared to be feasible and safe to treat the patients with hypoplastic left heart syndrome,” Ishigami and colleagues wrote in the abstract. “These initial results provide the basis for larger studies to assess the efficacy of this novel therapeutic approach in children.” – by Brian Ellis

For more information:

Ishigami S. Abstract #11789. Presented at: American Heart Association Scientific Sessions; Nov. 15-19, 2014; Chicago.

Disclosure: Ishigami and colleagues report no relevant financial disclosures.