Eight-Year Outcomes of Cardiosphere-Derived Cells in Single Ventricle Congenital Heart Disease

Area of Science:

• Cardiology
• Regenerative Medicine
• Pediatric Cardiac Surgery

Background:

• Single ventricle heart disease (SVHD) presents long-term challenges post-palliation.
• Previous studies indicated potential benefits of cardiosphere-derived cell (CDC) infusion at 2 years.
• This study evaluates long-term (8-year) outcomes of CDC infusion in SVHD patients.

Purpose of the Study:

• To investigate the time-dependent effects of CDC infusion on clinical outcomes up to 8 years in SVHD patients.
• To assess the impact of CDC treatment on overall survival, late failure, and adverse events.
• To determine if CDC infusion provides durable benefits, especially in patients with heart failure with reduced ejection fraction (HFrEF).

Main Methods:

• A cohort of 93 SVHD patients undergoing stage 2 or 3 palliation were enrolled between January 2011 and January 2015.
• 40 patients received CDC infusion, while 53 served as controls.
• Primary outcomes assessed were time-dependent CDC treatment effects on death and late complications using restricted mean survival time (RMST) analysis over 8 years.

Main Results:

• Overall survival at 8 years did not differ significantly between CDC-treated and control groups (HR 0.60, P=0.35).
• CDC treatment showed a trend favoring survival by 4 years (RMST difference +0.33 years, P=0.043).
• CDC infusion was associated with significantly lower incidences of late failure (HR 0.45, P=0.027) and adverse events (sHR 0.50, P=0.036) at 8 years.
• In patients with HFrEF, CDC treatment demonstrated a greater survival benefit over 8 years (RMST difference +1.58 years, P=0.043).

Conclusions:

• CDC infusion in SVHD patients is associated with reduced late failure and adverse events by 8 years.
• The survival benefit observed by 4 years appears durable.
• CDC treatment offers a significant long-term clinical benefit for SVHD patients with HFrEF.