1. Among post-myocardial infarction patients with left ventricular dysfunction, an infusion of cardiosphere-derived cells did not significantly reduce scar size at six months.
2. Favorable changes among the infusion cohort, however, were noted in markers of left ventricular remodeling and heart failure.
Evidence Level Rating: 1 (Excellent)
Cardiovascular disease is responsible for significant morbidity and mortality worldwide. Novel treatments are always in the pipeline, including the use of cardiosphere-derived cells (CDCs), heart progenitor cells that have been studied for use in regenerative therapy post-myocardial infarction (MI), hypoplastic left heart syndrome, and dilated cardiomyopathy. This study was a double-blinded, randomized, placebo-controlled trial evaluating the safety and efficacy of intracoronary administration of allogeneic CDCs in patients with post-MI left ventricular (LV) dysfunction. The primary endpoint was the percent change in scar (infarct) size in an antibody-matched cohort at 12 months post-infusion as assessed by contrast-enhanced MRI. 90 patients (mean [SD] age = 55  years, 84.4% male) were randomized to receive an infusion of CDCs, while 44 patients (mean [SD] age = 54  years, 86.4% male) received a placebo. 121 patients were antibody-matched while 13 were antibody-mismatched. It was found that there was no statistically significant change in scar size between the CDC and placebo groups (-5.0±7.4% vs. -4.1±9.1%, p = 0.54). However, the infusion of CDCs had a favorable impact on six-month percent change in LV end-diastolic volume (p = 0.02) and LV end-systolic volume (p = 0.02), as well as NT-proBNP levels (p = 0.02). Adverse events were infrequent in both groups. Of note, no patient mounted an immune response to infused CDCs. The study was terminated early at six months due to concern for futility; thus, the primary endpoint was assessed at six rather than 12 months. Overall, this trial showed that an infusion of CDCs in patients with post-MI LV dysfunction did not significantly reduce scar size when compared with placebo, the primary endpoint. It did, however, favorably impact secondary endpoints, such as LV end-systolic/diastolic volumes, markers of LV remodeling, and NT-proBNP levels, a marker of heart failure. As the infusion was shown to be safe and well-tolerated, more research is needed to determine if alternate dosing regimens or routes of administration have any benefit for select patients.
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