A Novel Cellular Therapy for Preterm Complications: Safety Profile of Allogeneic Cord Blood Mononuclear Cells in a Pilot Clinical Study.

BACKGROUND

Preclinical evidence supports the potential use of human umbilical cord blood-derived mononuclear cells (hUCB-MNCs) for preterm infants with preterm birth-associated complications (PBAC). Allogeneic hUCB-MNCs could extend this therapeutic option to infants without autologous cord blood available at birth.

METHODS

This phase I, open-label, single-arm, and single-center study enrolled 10 preterm infants (gestational age [GA] 27.1 ± 0.7 weeks) with two or more PBAC. All received a single intravenous infusion of allogeneic, partially or fully human leukocyte antigen (HLA)-mismatched hUCB-MNCs without preconditioning or immunosuppression. The median cell was (3.51 ± 0.93) × 106 cells/kg. Safety, adverse events, and clinical outcomes were assessed over a 2-year follow-up period.

RESULTS

All infants survived intensive care unit discharge. Four infants were lost to follow-up for parental reasons, with no in-hospital adverse events noted, their long-term outcomes remain unconfirmed. No infusion-related adverse events, acute reactions, or graft-versus-host disease (GVHD) were detected. One case of cerebral palsy was diagnosed during follow-up and was unrelated to cell infusion. Five of the six infants with evaluable long-term data had no respiratory, developmental, or neurological complications. Plasma inflammatory cytokines exhibited exploratory changes after infusion, without confirmed causality. All infants with follow-up data reached age-appropriate growth at 12 and 24 months of corrected age.

CONCLUSIONS

No major infusion-related adverse events were observed in preterm infants with PBAC receiving allogeneic hUCB-MNC infusion. Six infants completed 2-year follow-up and four were lost to follow-up. Constrained by small sample size and attrition, the results need further verification in larger controlled trials.

TRIAL REGISTRATION: Chinese Clinical Trial Registry: ChiCTR-OPN-15006932, ChiCTR2000035227.