Transcoronary infusion of cardiac progenitor cells in hypoplastic left heart syndrome: Three-year follow-up of the Transcoronary Infusion of Cardiac Progenitor Cells in Patients With Single-Ventricle Physiology (TICAP) trial

J Thorac Cardiovasc Surg. 2015 Nov;150(5):1198-1207, 1208.e1-2. doi: 10.1016/j.jtcvs.2015.06.076. Epub 2015 Jul 8.


Objectives: Our aim was to assess midterm safety and clinical outcomes of intracoronary infusion of cardiosphere-derived cells (CDCs) after staged palliation in patients with hypoplastic left heart syndrome (HLHS).

Methods: In this prospective, controlled study, 14 consecutive patients with HLHS who were undergoing 2- or 3-stage surgical palliations were assigned to receive intracoronary CDC infusion 1 month after cardiac surgery (n = 7), followed by 7 patients allocated to a control group with standard care alone. The primary end point was to assess procedural feasibility and safety; the secondary end point was to evaluate cardiac function and heart failure status through 36-month follow-up.

Results: No complications, including tumor formation, were reported within 36 months after CDC infusion. Echocardiography showed significantly greater improvement in right ventricular ejection fraction (RVEF) in infants receiving CDCs than in controls at 36 months (+8.0% ± 4.7% vs +2.2% ± 4.3%; P = .03). These cardiac function improvements resulted in reduced brain natriuretic peptide levels (P = .04), lower incidence of unplanned catheter interventions (P = .04), and higher weight-for-age z score (P = .02) at 36 months relative to controls. As independent predictors of treatment responsiveness, absolute changes in RVEF at 36 months were negatively correlated with age, weight-for-age z score, and RVEF at CDC infusion.

Conclusions: Intracoronary CDC infusion after staged procedure in patients with HLHS is safe and improves RVEF, which persists during 36-month follow-up. This therapeutic strategy may enhance somatic growth and reduce incidence of heart failure.

Trial registration: NCT01273857.

Keywords: cardiosphere; congenital heart disease; heart failure; hypoplastic left heart syndrome; stem cell therapy.

Publication types

  • Controlled Clinical Trial
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Age Factors
  • Cardiac Catheterization
  • Cells, Cultured
  • Child Development
  • Child, Preschool
  • Feasibility Studies
  • Female
  • Fontan Procedure
  • Heart Failure / etiology
  • Heart Failure / physiopathology
  • Heart Failure / prevention & control
  • Humans
  • Hypoplastic Left Heart Syndrome / complications
  • Hypoplastic Left Heart Syndrome / diagnosis
  • Hypoplastic Left Heart Syndrome / physiopathology
  • Hypoplastic Left Heart Syndrome / surgery*
  • Infant
  • Infant, Newborn
  • Japan
  • Male
  • Palliative Care
  • Prospective Studies
  • Recovery of Function
  • Risk Factors
  • Stem Cell Transplantation / adverse effects
  • Stem Cell Transplantation / methods*
  • Stroke Volume
  • Time Factors
  • Treatment Outcome
  • Ventricular Function, Right*

Associated data