A HEART-WISE: Allogeneic Wharton's Jelly-derived Mesenchymal Stromal Cells' Intracoronary Transplantation in Pediatric Patients with Dilated Cardiomyopathy: The First Case Reports.

BACKGROUND

Pediatric dilated cardiomyopathy (PDCM) is a severe cardiovascular disorder characterized by ventricular systolic dysfunction, with heart transplantation as the gold standard therapy. Mesenchymal stromal cells (MSCs) have demonstrated promise for treating cardiomyopathy, but previous studies in pediatric patients have focused exclusively on autologous cells such as MSCs, while allogeneic MSCs with better results were studied only in adults.

OBJECTIVE

The study “A HEART-WISE” is the first case reports to evaluate the safety and feasibility of intracoronary transplantation of allogeneic GMP-complierant Wharton’s jelly-derived MSCs (WJ-MSCs) in PDCM patients.

METHODS

Two pediatric patients with PDCM received a single intracoronary infusion of 1.5 × 10⁶ cells/kg WJ-MSCs. Clinical evaluations included NYHA functional class, six-minute walk test (6MWT), N Terminal pro-brain natriuretic peptide (NT-proBNP), and cardiac imaging over a 12-month follow-up period.

RESULTS

Both patients tolerated intracoronary WJ-MSC administration without serious adverse events related to the procedure. Patient 1 demonstrated marked improvements by 3 months (NYHA III to II, LVEF 33% to 48%, 6MWT 300 to 373 m, NT-proBNP 3502 to 1734 pg/mL) but passed away at 6 months follow-up due to a viral infection. Patient 2 had a persistently reduced LVEF in the 43–46% range for more than three years prior to therapy but achieved sustained improvement by 12 months (NYHA III to II, LVEF 43% to 64%, 6MWT 265 to 380 m).

CONCLUSION

Our preliminary findings suggest the safety and feasibility of intracoronary transplantation of allogeneic WJ-MSCs in PDCM, with potential off-the-shelf therapeutic benefit. Validation of these observations in larger randomized controlled trials is needed to better evaluate long-term safety and efficacy.